tv Health Experts Testify on Neurodegenerative Diseases CSPAN July 30, 2021 8:02pm-1:39am EDT
now a hearing on degenerative brain diseases like alzheimer's, huntington's disease and als. officials with the national institution of health and food and drug administration testified about research and treatment efforts for the diseases. >> subcommittee on health will now come to order you to overnighting today's hearing is being held remotely as well as in person. for members and witnesses taking part in person here following the guidance of the cdc and
office of the attending physician so please wear your mask when you're not speaking and thank you for doing so. for members and witnesses taking part remotely, microphones will be set on mute to eliminate background noise and he will need to unmute your microphone when you wish to speak. members are participating from different locations at today's hearing recognition of members will be in the order of the subcommittee seniority. the e-mail address we provided to your staff and all documents will be entered into the record at the conclusion of the hearing. the chair now recognizes for five minutes for an opening statement. my colleagues, i called for today's hearing to discuss the challenge of advancing treatment
and cures for neurodegenerative diseases. my constituents, jamie perry was diagnosed with als one year ago. as she wrote a letter to me saying with als, a piece of you dies every single day. we are simply asking for a fighting chance to live the lives we were meant to live. today we're going to hear from for patients and caregivers who like jamie them are simply asking for a fighting chance against neurodegenerative diseases that have afflicted their families. ryan and sandra, thank you especially for traveling across the country to offer your testimony to us. the difficult journey you've made and we thank you for the profiles in courage and being
here to offer testimony. our work today is to help create the fighting chance against deadly diseases. according to the national institute of neurological disorders, each year 50 billion americans are affected by neurological disorders such as als, alzheimer's, huntington's disease and parkinson's. these diseases exact an enormous personal talk and cost to the u.s. economy as much as $800 billion annually. despite prevalent, deft and heartrending impact on families across our country, there are effective treatments for neurological disorders. lack of investment, difficult drug approval processes and limited understanding of extremely heterogeneous diseases keep effective drugs off the market. private companies invest one
fourth as much into neurological drugs as they do for oncology treatment. only 7.9% of drugs for neurological report disorders successfully make it from phase one approval. when they are successful, neurological drugs take on average, 50% longer to reach approval and drugs for other disease areas. there have been recent breakthroughs understanding genetic basis of the disease and potential biomarkers but this is yet to translate into effective treatment. for patients, a diagnosis can be a death sentence. i think every one of our members have heard from patients, set up with lack of options. two drugs, amx and 0035 and neuron captured attention and sparked a debate over whether potential benefits effects
outweigh the risk. everyone here shares the same goal -- full approval for effective drugs but the question still stands -- how do we best get there? an obvious first choice is funding to the fda to ensure complacent staff working at capacity. we made progress with fy 22 appropriations bill that increases fda's budget by nearly $2,150,000,000. but i'm still interested to hear from fda about what more should be done to support their mission. we may need better multi- kibbutz going ordination between fda, nih, academic researchers, private drug companies and patients. breakthroughs in cancer and hiv came from a better understanding of basic science of the disease but also better collaboration and coordinated strategy.
these efforts will bring breakthroughs from the bench to the bedside. that's why i am pleased to be working with the biden harris administration to create the advanced research project agency for health. new independent agency will take on projects like alzheimer's and als where the market failed to invest due to risk and bring new strategies and collaborations to our current system. i make there needs to be clarity and transparency about the standards for approval for deadly diseases with unmet medical needs. a promise of flexibility rings hollow when undefined. i believe these challenges are not insurmountable but these diseases are not incurable we can provide constituents set, a fighting chance for patients to live the lives they were meant
to live. that's our work today and for the days and years ahead. the chair now is pleased to recognize mr. guthrie, our distinguished ranking member on the committee of health, five minutes for his opening statement. >> thank you for holding this important hearing about advancing treatment and neural degenerative diseases. our to recognize and think it's up to the check to testify on her experience huntington's disease. construct a fine from kentucky while not advocating for patients from huntington's in the family, she can be found facepainting community offense. the fourth none suspected generation of her family and a strong advocate and voice for the huntington's disease community. we are here today to examine how we can further advanced treatments focus on cures for patients suffering with
neurodegenerative diseases. with my progress to create an environment in the united states and encourages innovation for treatments, thanks to representative in this committee, the 21st century cures modernized healthcare innovation infrastructure and included more flexibility to capitalize on this exciting time in science. enable private sector innovation. this committee has worked on preauthorization institutes of health and ensure the budget is adequate to foster research for treatments and cures. congress provided at the eighth the resources and refute drugs for serious unmet needs and rare diseases. we have continued to examine expanded access pathway to drug outside of clinical trials and are flexible like to try pathway for patients to access experimental drugs. while we have come so far, we have a long road ahead to help patients and families. one disease i focused on ever since coming to congress is
alzheimer's. alzheimer's is the sixth leading cause of death in the u.s. 2021, estimated 6.2 million americans age 65 and older are living with all summers. it is expected to reach 14.8 million, bearing the department of medical breakthrough to prevent in the disease. this alzheimer's act signed into law in 2018 creating a public health infrastructure across the country to support prevention treatment and care for alzheimer's patients and related neurodegenerative diseases. i have continued my commitment to this issue introduced in the copperheads of care care for alzheimer's congress. this reduces medical complications of these patients by creating a new way to medicare. it's not just alzheimer's but other neurodegenerative diseases devastating for the person who suffers with disease in the family, friends and five months. huntington's disease is
progressive, brain disorder caused by an inherited gene can appear as early as age two or as late as 80 years old. more than 200,000 americans are at risk of inheriting the gene from a parent with hd. parkinson's disease is another of the progressive brain disorder that affects 60000 americans here. an estimated 50 to 80% of individuals with parkinson's disease, expressing dementia in their lifetime. als referred to as lou gehrig's disease that affects vital nerve cells. for people with als, the average time is three years and report to 15000 americans with als. we can all agree we want to advanced treatments. hr three is not to do our path forward. i believe innovation to treat
these diseases will be in jeopardy, quite possibly decimated altogether. over ten years, life-saving drugs would not come to market due to hr three because it does incentivizes research and development. it would directly here patients like the ones before us today that i support the bipartisan alternative to reduce drug prices and protect innovative cures. i'm glad to have kayla with us today and ryan wallace to share how congress can a hopeful i, promote innovation for lifesaving cures and i look forward to working on this for american patients and with the chair and look forward to seeing a better future where people can live the life and day we are meant to live. >> the gentleman yelled back at the chair thanks him for his opening statement. the chair is pleased to recognize chairman of the full committee for his five minutes
for an opening statement. >> eq. our understanding of the human brain rapidly increasing in recent years thanks to the advancement in science and research. diseases and their causes continue to be a mystery in many ways. you treatments of the symptoms of neurodegenerative disease exist and no known cures by significantly slower progression. millions of americans and their families face heartbreaking daily challenges that come with these diseases. in congress, the committee in recent years have supported substantial investments in neurodegenerative disease research flexibility is for clinical research. in 2016 we passed the 21st century cures act which authorized $1.5 billion to put the national institutes of health brain research through advancing innovative neural technologies or brain
initiative. this mission is to revolutionize understanding of the human brain to discover new ways to treat, cure and prevent brain disorders including neurodegenerative diseases. by sullivan the department of novel technologies to map new picture and understand, providing revolutionary foundation for future research and clinical department. this would be augmented by the advanced research project agency for help. this plays a key role in the agency is responsible for the safety and efficacy of all drugs and treatments and development including those who treat brain disorders. he provides guidance clinical trial design, meaningful and consideration to determine whether treatment is beneficial, market approved. he works with physicians and patients when treatment options will be unavailable. for the 21st century cures and
reauthorization act, the committee encouraged greater guidance on the use of novel clinical trial and inclusion of patients in the drug development project. these are all promising but it's clear that a lot more must be done. to provide quality of affordable and equitable care, for patients and their families. to protect patients in the american public it's important we understand current state of science neurodegenerative diseases and how we can further improve access to clinical trial and treatment seekers. it is are responsible to provide agencies with necessary resource so we have two panels on our first panel, who will hear from government representatives at the fda, national institute agents and neurological disorders and strokes. i'm interested in hearing about the progress made since the
21st century cures act and fda reauthorization act of 2017 and how the problems are affecting research and drug development and what needs to be done. our second panel will hear from research industry patients caretakers and their expense as a critical to our work today. patients and caretakers with physical and emotion diseases everyday including the process of enrolling and participating in a clinical trial searching for available treatment. these are neighbors and friends and for many of us, our family. i look forward to hearing from those on the cutting edge research and als and neurodegenerative diseases who have conducted clinical trials treated patients with the disease. will hear from industry about the pipeline and manufacturers challenges for neurodegenerative use part.
thank you, madam chair for your role in making sure we have theirs and i yield back the balance of my time. >> the chair recognizes representative rogers. recognized for five minutes for opening statements. >> the money. we speak our work before the pandemic. the many meetings with all had, listen to advocates fighting for cures and treatments. hundreds of disease and breast disease groups came to the people's house so the opportunity to share their story, advocates met my friend, dale gleason, they like millions of other people have an extraordinary amount of hope in the promise of american innovation whether it als,
alzheimer's, huntington's, and other disease, the hope for lifesaving treatments and cures is in the united states of america. we cannot forget that, we have let the world, u.s. is where hope becomes reality. that brings me to hr three. speaker pelosi's harmful prescription drugs. one mom told the subcommittee, research will stall under hr three and said other countries have price control and innovation desert, they are limitless meaning access to a rare disease treatment to save your children. her son famous hunter, he has muscular atrophy and he is alive today, matches his birthday, his tenth birthday because of a breakthrough treatment. for children like hunter, it would be devastating if price
controls were jammed in the majority's reckless spending and lead to less innovation and no hope for many people who deserve a fighting chance at life. about a fighting chance, we should be working on bipartisan solutions like hr 19, lower cost and more cures act. in addition we should be leading the way in a bipartisan way to fund more basic research, support research around the causes of diseases and release the private sector like we did covid-19 vaccines. this is very personal for me. my son has down syndrome, the most common form of zonal abnormality. there is still a lot we don't know about that 21st chromosome. for example the scientific community has acknowledged one 100% of people with down syndrome will develop brain
pathology for alzheimer's in their lifetime but only about half will experience symptoms of dementia. the reason is still not understood. imagine what it would mean if we unlock the mysteries of the 21st chromosome to lead to major discovery and maybe even the cure for a disease like alzheimer's. why i was surprised to see the biden budget proposed to move nih's include program from the office of the director of the national institute of child health and human development without any explanation. what problem does this organization solve? will have the same sector collaboration and coordination? the program has been one of my top priorities. i am disappointed if there were concerns, and ih didn't consult with congress. one of these -- i want to be clear, i have starkly been a champion for nih supported government their funding, i
cochair for neuroscience carcass and i promoted brain initiative from the beginning that nih is on the verge of a trust, prices with this committee and the american people. this is a warning opposing living in apartment, the authorizing many promise is one thing. another is lack of respect for congressional oversight and how nih research money is received and spent. a scientific objective investigation into the origins of covered, i've made many requests to nih to be transparent and provide documents. we've received no documents including print documents releasable to the general public under federal law. it's unacceptable. i told doctor directly when we spoke about age, resident biden requested more than $6 billion for it with less accountability and transparency than we have
now. if i were to support this, i think our confidence and trust in the oversight management of the 44 billing and taxpayer funding going to nih now. including a clear picture of how much of that research is going to china. i'll close by thinking that patients and families and caregivers and researchers with us today, we are grateful and share your mission to unleash american innovation, support clinical trials, improve early diagnosis and improve the lives of millions of americans and that's why i'm passionate about making sure nih research is spent wisely and accountable. that's what we can do. on these, flew to unleash the private sector of these diseases with the same of urgency had with cobit and i back. gentlemen yelled back. the chair would like to remind members of the committee most all companies.
i would like to introduce our witnesses for our first colleagues witnesses today and i think the minority poor will bring you people forward as well. first, doctor richard is the director of the national institute on aging, the national to of health, talk to you. doctor walter is the director of the national institute of neurological disorders and stroke nih, welcome to your and i hope i have not butchered your name. doctor is the director of the center for drug evaluation research at the u.s. food and drug administration, welcome to
you, doctor. i think this is -- welcome to the committee. i think it's your first time here so the chair now recognizes the doctor for your five minutes. >> good morning members of the committee. thank you for the opportunity to be here, the director of the national to aging. identify ways to prevent, treat and care for those. one of the most common tragic or six-point people now by 2060. congressional appropriation taxpayers to make progress. understanding has ranged from basic fundamental science and
trajectory demonstrated disease give us new insight. from the most basic level and enterprises such as the 80 or partisan, are brought together pharmaceutical corporate fundamental basic science by nih as well as foundational promises, all to generate the open size and acceleration process identification of hundreds of new targets. changes that occur in strength represent a potential intervention phase move toward individual measurement and all from person-to-person. translating this into clinical
intervention to identify is a particular challenge and we've instituted infrastructure to celebrate private sectors of the transition from basic science to be studies. we support this, more than 50 drug trials targeting a variety including information and stability as well as how. these are moving through recognizing and understanding it's unlikely for suspicion to address all what we are looking at the size clinical research itself and the importance of recruiting in the population in the u.s. including -- down syndrome. we've come to realize diseases like alzheimer's with the progress that goes on for years and decades with the symptoms and therefore the importance of
being able to identify it intervene before their brain cells and their connections. until recently, alzheimer's was diagnosed at autopsy. now, markers have a lot to see what's going on in the brain early on and track the responses. most recently, being less intrusive and expensive to bring the new ability to recruit people into these tracker disease and track the outcomes and intervention. people currently living with alzheimer's, it's important research be conducted as we make priorities to understand the best ways to support those living with in caring for people with alzheimer's. identify infrastructure collaboration and partnerships with healthcare allow us to
conduct pragmatic trials short turnaround and identify success for the best way to care for people with alzheimer's. we understand prevention is also an important way and the disease was identified prevented interventions from a study that showed this approach has the ability to decrease this, a precursor mentioned. similarly, intervention with diet, exercise, combinations of them, to find ways to innovate and prevent disease and site behavior change to make sure we know how to best inform people for their lifestyles. thank you for the ability to hear and profoundly to support
progress as we come to understand and intervene. thank you so much. >> thank you. next we call on doctor walter for your five minutes of testimony and thank you for your work and being a witness today. >> thank you anguished members of the committee. leading the national institute for and i ds and disorders, to use that knowledge to reduce this in this disorder in the brain and unfortunately, once it
dies, it is not replaced so there are many forms of neurogenic degenerative diseases affecting different parts of the brain, they have the ability to take away their abilities to do anything and take care of themselves. they rob people of years of fives. this is among the research community to recover highly effective treatments. i'm hopeful with a host of new discoveries will lead to great things. i'm going to focus my remarks on just three. the first is genomic therapy as congressman mentioned recently we had almost miraculous impact of therapies, a genetic disease that causes degeneration of the same affected by ls. with gene treatment, and infant restored function and save lives but it's not a one off.
this should drive a whole new approach to the neurological disease. these therapies are already underway for huntington's disease and some forms of als. i believe we are on the step of a revolutionary era of therapy, especially as they become linked to these delivery tools developed by the brain initiative. second, these tools are now to inherit disorders but the promise is much broader. in most disorders, a subgroup of patients for these patients but most have a sporadic form. luckily, studies of the inherited disease patients have uncovered pathways of the degeneration and non- inherited forms as well. therapies are being developed
with common pathways to prevent neuron dysfunction and death. third, somewhat commenting and treatment, potential of multiple degenerative disorders. we look under the microscope and people who could die from these disorders, we almost always see of protein inside with the dying celebrate specific proteins, the brain areas involved with this disease, these aggravated proteins seem to think have the ability to spread to a healthy one and verify damage one brain region after another. as an example of parkinson's disease, there's some evidence that protein aggregation start in the nerves that supply pickup due to interaction with bacteria in our gut. over the course of years, these proteins are in the nerves of
wicca. treatment to block these proteins are being developed for parkinson's but for many other disorders. furthermore, this will allow identification of these individuals and enable early treatment that blocks before the disease in these three specific examples but let me turn to the overall strategy. once disorder or area on a nervous system as a whole and how it integrates with other body systems. elderly persons with diagnosed dimensions have alzheimer's disease along with blood vessels
caused by years of high blood pressure and evidence of injury and brains connecting fibers and white man disease. the nih study showed oppressive blood pressure reduces cognitive overtime and what we know now and how to control blood pressure, it's not only this book cognitive decline and potentially dementia. public health campaigns were targeting black americans in the late 20s to mid 40s and the greatest brain health due to hypertension. i would emphasize a tremendous scientific challenge as we strive in these orders by optimism stems from powerful new tools in the last five to ten years to combat these diseases.
>> thank you, status compelling testimony and we appreciate your work and being with us today. next we have doctor -- welcome to the committee and the chair recognizes you for your five minutes to present your testimony to us today do we know why the doctor is not -- what? you need to unmute, doctor. >> my apologies. >> there you are.
>> here i am from a apologies for the technical difficulties. members of the committee, to give her the opportunity to testify before you today. also, i want to thank my colleagues from nih for their ongoing support and willingness to collaborate. as we translate research therapies, in recent years, advancements have been life-changing. you therapies have brought patients faster, more efficient clinical trials and use of expedited pathways. we don't look at the development of the covid vaccines and therapeutics to see how quickly scientific research can spread benefit. many new cases which would have resulted a few years ago, it can now be treated and in some cases, cured by fda approved
therapies. while fda approved countless transformative therapies for life-threatening diseases, it's in stark contrast with the condition for which there are few treatment options. i applaud the subcommittee's attention to this by holding this hearing today. degenerative diseases caused tremendous suffering for patients and loved ones. we need to adopt additional innovative approaches to bring new drugs to people who desperately need them. there are three primary elements to our approach i want to highlight today. the need for more research and from regulatory stability and actively listening to the people stricken by these terrible foundations. neuroscience is an area of medicine whether it's safe and effective treatment and research to guide and inform the
development of the therapies. although there has been great progress in basic and preclinical research for these degenerative diseases we have yet to identify the underlying ways for many of these provisions. the client communication prevent significant challenges for the development. this is true for als. for instance, for many diseases, there are no easily measured biomarkers reliable predictors for this disease progression in individual patients. such tool will prove improve revisions with which drug response would be evaluated leading to more robust and insight for distinguished promising drugs for those less likely to succeed. researchers are continuing to make advances in understanding underlying process of neurodegenerative diseases and drug involvement.
as an agency we are using every tool at our disposal to help facilitate the development of treatment. we have long had a threat for regulatory flexibility in the standards when it comes to medical products or serious disease and medical needs while making sure these are effective and have favorable benefit to risk profile. this flows from regulation. in the meantime, we understand the need for access to therapies when people cannot participate in clinical trials. this is why the agency has almost all individuals expanding request. however, an essential step in this process is the company's willingness to provide this and there are instances when this
doesn't happen in ongoing clinical trials, financial burden or insufficient drug supply. we do all we can in these situations to help people who desperately need these drugs. finally, the son of all our efforts of the people who need therapies, experiences, perspective and priorities are a critical aspect of the development. this enables the delivery up therapeutics that have meaningful impacts on people's quality of life target with a consider the most important aspect of their diseases. i look forward to discussing these and other issues with you today. we recognize the impact of these diseases have on patients and their loved ones. we share the sense of urgency and we stand ready to make full use of authority in order to help renew therapies to people with these diseases as quickly
as possible. >> thank you, doctor. we are going to move to member questions and the chair recognizes herself for five minutes to do so. first, doctor, in your written statement you say treatment that provides meaningful incremental benefits would still be desirable. now many of the als advocates think the significant outcome in the trial that showed a three-point improvement was incremental but meaningful. now as we all know, these are individuals usually told they only have two -- five years to live. what i want to examine, because of the discrepancy between fda
and incremental meaningful benefits, being desirable and approving a drug that produces that so would you comment on that and tell us how you define meaningful incremental benefits? works you need to unmute. >> iq question. we generally don't comment on specific drug programs are some of the information is down. >> in general. i understand you can't comment
on specific drugs. i just use it as an example but in general, how do you define incremental? >> the context here is that incremental in these cases is small but represents a great deal of hope to people that are living with investment. >> in general we look at incremental benefit and dealings with several points of view. the perspective of the patient is very important because what we hear from the patient guides us for what is meaningful incremental gains. but we had heard from people suffering from als is
improvement in symptoms and quality of life is very important. in addition with my clinic survival so we have reflected these and how we guide our running programs for treatment for als and this is reflected in our guidance. we show that should be looking at a variety of endpoints. >> i need to interrupt because i need to get to another question that deals with the european conditional approval pathway. they have a different system when they approve, when the drug
is approved they will be evaluated further on the market. we don't do that in the united states and this means the drug may be available to european patients, two to three years before american patients so if fda had a similar authority for conditional approval pathway, would that give the fda more flexibility getting potentially promising parents to die patients sooner? if so, what the fbi's have that kind of legislation in congress? english conditional approval? >> it's a very important question. when it comes to expediting the
development, we think we have a lot of tools at our disposal and a limiting factor applying all of the tools we have for approval has been lack of understanding of the biology of the diseases and we are eager to work with them to identify some of the biomarkers and markers of the disease to allow us to utilize expedited pathway including accelerated approval and accelerate the development. >> my time has expired and the chair now recognizes mr. guthrie, ranking member for his five minutes of questions. >> thank you for the recognition if the first question, as you know nih brain initiative
intended to produce revolutionary new dynamic map of the brain that control how individual cells and complex neural circuit interact in time and space. how will this initiative improve our knowledge of generative disease and help researchers find new ways to treat, cure and even prevent these diseases? >> thank you, this is something i could spend hours talking about but i will say three things. one is we now have the ability, amazing discovery to get a census of the human brain. powers enabled by technologies that allow us to look at cells and analyze what's inside the
single cells but instead of 100 south over six months, we can do 1 million in two days. that's an amazing way to study the brain and degenerative diseases we can tell what cells are missing but also be able to tell the difference between a sick and healthy self. the second thing, inside the cells are like genomic heat and what we are trying to do is find the keys and link them up when we give to the person will always go into where it needs. the last one is that as a neurologist, we see the patient and what symptoms they have and
we look in the grain and see what we see and read say it because her symptoms but in between is the circuit and we have no way of seeing the circuit but with this in action. for instance, this is a study of people who do not have dementia, their perfectly normal but they have terrible alzheimer's disease. an example is pulling us, the circuits are still healthy. >> okay, thank you. now instead of answering that question, you can emphasize from her back if you would like.
caring for a person with alzheimer's or another disease poses unique challenges. they express at least one narrow psychotic symptoms including siding or agitation, depression, hallucinations and delusions these are charges that prompt family characteristics signed their leptons into institutional care centers. the question is, people read living with these family members would be safe and effective treatment is important but also could you update us on the search for treatment options. >> thank you. research have ways in which to maximize life and care for those living with dementia. there are currently over 80 studies trying to understand
which of the most effective. some already have reached number one example this five years ago and went through the va and health service is one example but we are constantly looking for ways in which with the pragmatic randomized trial trying to look at the very best time of intervention in learning more about everyday so our ability to translate this is on the horizon. i can just briefly -- >> does look at some.your scene coming. >> in terms of caregiver report alexis. >> justin alzheimer's. >> in general, a good place to
start as described enthusiasm gently named as the ability to look up on the ev lease that was never happy for you and begins with basic science. normal brains. animals and humans providing opportunity for breakthrough for diseases such as alzheimer's only to find out what we are reformed, we look at the profiles and biology and brain space. they are not off the same so we are now poised to look forward i look at ways to target these strategies. >> i think i'd let you run overtime some going to have to stop you here. i gulped back. >> the gentleman you expect. thank you, doctor. the chair recognizes the chairman of the full committee for his five minutes of questions. >> thank you.
one of the strategies discussed in testimony today bolstering expanded access, compassionate use and allows these conditions with no satisfactory alternatives could cap role in the clinical clinical trial for investigational drug and assess the potential benefit justifies potential risk and providing for have an ongoing clinical trial. i have some questions about this. first, in 2017 from a former fda commissioner scott testified before the committee and set fda approves expanded access to 90% of time and wanted to know two questions. so, why did i still have difficulty accessing trucks under the program? what recent manufacturers have for declining to participate expanded access.
>> thank you for that question. i confirmed that we are still approving the overwhelming majority of expanded access application in between the past five years we have approved 8%. there are reasons for companies not making the investigation of drugs available through expanded access. the first one is the fact that there might be clinical trials and the patient's who are asking for expanded access for that clinical trial. make the concerns about slowing down recruitment of the clinical trial docket provide important
aspects on the drug. for small companies what we have also seen as they may be financial constraints in supporting an expanded access program sometimes we see some issues with limitations in the drug supply drug supplies for an ongoing clinical trial. oregon and financial considerations, particularly with the smaller companies. >> expanded access and neurodegenerative disease because more data would be generated patient's on the treatment so let me go to doctor, can you speak about nih on weather data generated through expanded access program would be useful for scientific research and how would nih consider research based on data
generated from an expanded access program? >> status a good way to go over the finances of expanded access and the scientific value so nih, in a trial, the value would be in continuing access patient's involved, they have finished the trial because then you have a comparative group you can check for durability and also do a crossover and have active treatment from that. very hard to get, a muster is a tremendous effect, scientific value out of expanded access must the treatment has an effective size.
a smaller size you wouldn't be able to tell there has been a change in conditions. >> thank you so much. let me go back to doctor. can you explain why clinical research might choose to exclude a patient who participated in expanded access program from participating in our trial? is a possible put safeguards in place to ensure participation doesn't harm clinical trial enrollment? >> i cannot hypothesize why individual researchers may decide to exclude a patient with a specific clinical trials. however, in general, studies routinely about prior exposure to investigational agent, including potentially expanded access after. of time when the patient has an
agent. there may be some situations where the developers may have concerns about prior exposure with an investigational agent. or the interpretation of their results. when it comes to what we do, we are very sensitive to this routinely work with sponsors to attempt to ensure they do not use overly restrictive or unnecessary criteria to exclude patients who have been on investigational agent. this is part of our commitment to making sure that trials are inclusive when it comes to the diversity of patient included in clinical trials and fullscope of
guidance when it comes to the progress of our over the past several years we still have more work to do and we are very focused and working with developers to make sure that they deploy all tools that are available to them or even new tools to identify to expand the diversity. >> thank you. i wanted to highlight the individuals because as i mentioned in my opening statement 100 percent are developing alzheimer's but yet
they are not being included the way i believe they should that we should embrace what we can learn for those with down syndrome with those extra 21st chromosome those that have limited kenya and no tumor cancers let's embrace what we could learn from those of down syndrome. >> individuals of down syndrome experience a lifelong auto information. with accelerated approval to explore alternatives with use of therapies that modulate the immune system to slow down or reverse alzheimer's disease or neurodegenerative degenerative diseases. >> for that specific question the diversity of targets involved in clinical studies and clinical trials more than
those from birth to life and those to identify early life events that would predispose for alzheimer's disease? >> and then to include those that are participating but a multifaceted and then to collaborate in those efforts. >> thank you for being with us and your work still the gentlewoman yields back the
chair is pleased to recognize the gentle man from north carolina. mr. butterfield. thank you for the witnesses for your testimony today and your dedication on a bipartisan basis take you for your incredible work at fda you noted in your testimony your progress has been made to me say that progress has not been even. and with caregivers and researchers and then to recognize the cost with that
are so rapidly progressive and we also as we look and that they put in front of us that there has to be a higher pressure for risk with some degree of uncertainty. >> and with that five minute timeframe. and have the opportunity to and that was right near my district. having taking care of 3000 als patients in his career most
cannot find a place in a clinical trial not only but the research community suffers because it is not properly studied everyone would agree the access program with appropriate oversight would be preferable to experimentation. i realize fda cannot require of expanded access. fda incentivize person precipitation on —- parts participation from the extended access program. >> we work actively was sponsors that do not willing
from michigan from the fall committee and the number that we all have a deep regard you have five minutes for questions. >> thank you madame chair and this incredibly important hearing and i appreciate the testimony by the witnesses not only on this panel and those that are coming on the second panel as well. but i remind my colleagues that when we embarked on 21st century chairs that important legislation that we supported we work with the edge and state agencies and we ask questions what can we do to advance cares that impacts everybody? >> my neighbor next-door died of als and other friends of
parkinson's and to talk about along with mr. guthrie that can be appropriated and then we will get to the president's desk and we are working on a cures 2.0 bill that would be included but as part of 21st century cares what can we do to help you do your job better? how do we help you approve the chairs earlier to deal with these folks are with cystic
fibrosis and that remarkable achievement. so you have an important important role and looking at your testimony of the next panel that is coming especially the als community is so frustrating. there is not a cure. and that is more functional to provide the help that we will have a chair. in the next panel because there is a whole series of votes but the fda must be fully funded and to provide
the regulatory authority and janet woodcock and others so what could we do and we did it to increase it. but i guess the question i have for you on scandal is what can we do now for those folks who have als to help that their lives will be bettered with that research that duke has done so what can we do to help you to provide the hope that these folks want? that's my question.
and how the als community are with those patients with als and at the same tools of the disposal with regulatory with flexibility and those are the same tools. and with some limitations and challenges and we don't have as good of an an understanding of the biomarkers and many more degenerative diseases and those are the elements that allowed us to fully deploy but
>> i like to make a remark that even though you don't have the biomarkers so that is an area we need to hear more about from you. maybe is not a leapfrogging advance but it is in advance it demonstrates something and that means that there is a god awful disease so the chair is pleased to recognize the gentlewoman for her questions. >> thank you madame chairwoman for calling the important hearing. and the expressions of emotions that we have around
not only provide a light of information for when it comes to the endpoint that they could use to study drugs for als in the absence of biomarkers or evening that grieving function and someone a host that they could use quickly. and in addition to that we also emphasize those patient reported outcomes. and recognizing that caregiver is important and then to
and those conditional improve full pathways that exist. and that we both recognize that when that pathway is used there is some residual uncertainty around the drug benefit. there is also some notable differences between the two. when we look at that tools that are currently at our disposal to have an array of tools with accelerated approval that allow us to make that determination a benefit risk to approve a drug before to complete those trials. where the drugs are approved.
>> and there is a situation that and those patient experiences that we have had. and i yield back. >> the gentlewoman yields back. the gentlewoman from texas is recognized. >> we don't have nearly enough hearings involving it's tough to get calls answered from the agency. so ask me a question and i noticed that my department but people just have to know so
for several weeks and then we get no response. and i was on the subcommittee it was a novel approach to that type of legislation and it was understood at the start it could take more than one congress we had hearings and briefings and personally attended 15 different fields hearings around the country hearing from people. and in the alzheimer's stakes
seemed that that we had outlined in teachers to reduce the from the bench to the bedside and to sign into law to identify als associated risk factors and granted are you aware of any efforts from the fda to utilize the data and the registry correct? >> . >> i appreciate that very much. that one of the most of
sensitive victories achieved in my 21st century c.a.r.e.s. act and those that have been suffering from a life altering illness. i just want to point out from the work on the cures there was a stand-alone bill to establish a national neurologic surveillance system to us passing the c.a.r.e.s. act there was no official structure in place that is to be established and now to take the next step and deliver the benefits. i yield back. >> the chair is pleased to recognize the gentleman from florida for her five minutes of questions. >> thank you chair for holding this important hearing and
take you to our witnesses today for your important work. i and committed with those degenerative diseases in florida, 580,000 floridians 65 and older suffer from alzheimer's the highest percentage in the country and in my neck of the woods we are very fortunate we have a research university with does it degenerative diseases with the department of neurology in
those advocates with the als patients that are focused and of course we have a number of clinical trials with university communities and industry as well. and that is paramount. want to ask you a little bit about recent guidance fda guidance and industry is very important to help direct for stakeholders seeking for als.
and in that context with that efficacy. and that they should make the point during drug development to encourage industry to work throughout the process. fda does not often release disease specific guidance why did they develop specific guidance for als. >> thank you for the question we have a host of guidance and we do so when we recognize there may be particular
>> the gentleman time is expired. and then the gentleman from virginia for his five minutes of questions. >> a friend of mine that has als recently participated in the national institute of health study and please the way it was conducted once it finally began and it studied one year before it began despite it only needing 25 participants to filled how is
with als is doing pretty well but if you sending in information i would be happy to look into it. >> i be happy to send you the information that you say als is doing good to get people into the study that my experience was a check over one year before they got enough participants that doesn't like me are doing good enough and maybe we need more doesn't sound like you have any ideas but we are here trying to help. if you need authorization to advertise for clinical trials
we want to help them solve this problem because he is not doing very well. >> and one year he waited for the study. when we were doing research on these degenerative diseases and trying to recruit people the impediment which can be the impediment is getting people what about those that are more difficult for them to participate. >> that's a really good question. the bright side of covid is that they had to remove to a remote business with the
patient. they can learn they can do things remotely which is a tremendous advance for people who have trouble coming in to the census to be enrolled in the trials. those are anywhere in the country to nih. >> i appreciate that and that is a follow-up question what can we do to use more telehealth? if it's a blood sample and me can be shipped in the nih. the more we can do with telehealth and then a speak for both sides of the aisle we are anxious for agency like yours to tell us what you need to put into languages to
facilitate to use more telehealth to make sure we do it right to get the studies and more participants in the studies. >> working with patients in one state with 15 different licenses in each state that i worked in. >> we have done that with special circumstances in the past. thank you for your testimony and i yield back. >> i word add the bill that we approved of with the additional funds for the large , i went to encourage all of the witnesses up front what
you need to make all of this work better. that is what the's hearings are the important aspect of the hearing. the chair is please recognize the gentle man from maryland and mr. sarbanes-oxley his five minutes of questions. >> we had others to speak to some degree to this hearing but the importance of exclusion criteria that were used by developers in who participates in these various clinical trials and in recent years it has been a push on —- a push to diversify to better represent the population but
the fda for the public meeting in the clinical trial and thereafter so to put these measures together in a way with those degenerative diseases researchers have to evaluate the populations of disease progression which is complicated to determine how to maximize what is brought to bear at each stage with that desire with the effects of
certain populations on the one hand and those populations on the other hand. so can you say why this tension exist? especially with neurodegenerative diseases we are discussing today? >> thank you for the question. i acknowledge the fact there is anxiety among the developers when it comes to broadening inclusion criteria in clinical trials. those underrepresented racial and ethnic minorities or inclusion of the subset of the population that are affected with the disease such as we heard earlier.
we can develop those ways to appropriately represent the subgroups that are affected by the disease but also be able to contact trials in a timely fashion. in addition to the guidance we have issued and then to have in place the appropriate outreach and those geographic areas to establish a network to refer clinical trial participants as well as making sure with the earlier
conversation important tools such as decentralized clinical trials and then to issue guidance during covid and we are working on a go forward guidance that one of the reasons some populations to be underrepresented in clinical trials can access clinical trials because as we have just heard. so therefore it is important we encourage developers to centralize those modalities including physical health in a way that doesn't require degenerative diseases that may be in a wheelchair to travel
but then have that procedure done remotely. so we think we can get to a point with greater representation well not slowing down clinical trials. >> thank you for your testimony and i yield back. >> the chair is pleased to recognize the gentle man from california for his five minutes of questions. >> i appreciate it very much thank you for holding this hearing. again thank you for holding's we can learn more about the challenges of the neurodegenerative diseases such as als which is a brutal
disease with no known cure or any real treatment i was particularly saddened to learn this past week of the passing of my constituents in good friend doug mcginnis a combat veteran two was diagnosed over 15 years ago with als he was an incredible warrior. i knew him very well managed to fight back for many years before the disease progressed. unfortunately since he experienced treatments that prolonged his lifetime he was barred from clinical trials and those that were effective for him. both he and his wife has been
at the fair front of the clinical fight fight the this disease and i agree you must act and do more. we have heard the concerns from the als community that the heterogeneity and others with huntington's can complicate participation in clinical trials to therapies. and with adult stem cell treatments and i am very sad
benefits and nine dares positive. and with that function and muscle strength and so on because we really want to make sure developers use multiple and to evaluate these therapeutics. >> i have a question with parkinson's disease. i know we don't have a lot of time but i have to summit for the record. thank you very much.
nonaddictive the pain therapies. and to build need new research capacity. >> thank you very much. and what about expediting development and quickly as possible. >> thank you for the question. we have been working closely with developers to advise them on how to have clinical trials to identify endpoints for clinical trials in a way that
allows those trials to deliver answers as quickly as possible depending on the endpoint. severe very engage with the developers so we are in particular with the development of therapeutics with a large unmet medical need and we guide them on the details on the clinical trials that it can be using to yield faster answers. and then looking at the impact of a potential therapeutic. we have heard from people suffering from als that what matters to them is improvement
of symptoms and quality of life so we work with developers to identify ways to evaluate the drug that could yield answers that we know are meaningful to people suffering from the disorder. >> thank you both very much. i appreciate the work you are doing. alzheimer's is just devastating that if you can't afford that their pricing power of $55000 and it would
cost and spent all medications for all patients. so i appreciate the work you are doing for patients to have reasonable pricing so it is affordable for taxpayers i yield back. >> seeing no republicans available i recognize the gentleman from illinois for five minutes of questions and then make remarks about impending votes.
>> we will see if we can squeeze you in the gentle man from california is recognized for five minutes with questions. >> sorry about that madame chair and i could see myself on the screen but it was not connecting to the committee. i appreciate this opportunity to the panel for your expertise and advice and i would like to cover various issues when it comes to clinical trials with the inclusion and exclusion eligibility require and external factors from participating in clinical trials will include geographical limitations of financial burdens, transportation difficulties and the ability for caregivers to assist patients in enrolling and participating. in the second panel we will
hear testimony from a witness who was faced with all of these issues who describes in her written testimony that she had to help her mother with alzheimer's disease from the bronx to manhattan every week while she maintained a full-time job. the clinical job barely compensated enough for the cost of a cab to the research site. these issues are systemic in the federally funded alzheimer's disease research centers tend to be in the wealthiest neighborhoods for those who would love to be a part of these trials on the other side of town. if we will discover new treatments available to the widest population, we need to do more to expand access for the trial so what guidance has the fda to provided and how
health and encouraging developers to define clinical trials in a way they are simple as possible that doesn't require multiple unnecessary visits. assess the situation, it could be cumbersome to describe in the caregiver. >> thank you, doctor. my time is limited, i would like to get a question in. the ability of affecting the patient and accessing critical trials for older adults those with alzheimer's disease. has nih evaluated the role of caregiver's plate and patient access to trials and what can be done to support characters so more patients can participate? >> thank you for the question. it's critical that there be provision made for someone to
accompany the person with dementia. it's part of the fund, we are doing our best to locate it as close as possible to affected communities. that's established a standard infrastructure, we don't have it, we need to identify and redesign. in some areas at the national level, we haven't had alzheimer's disease is applicable in all parts of the country so prevalent in mexico and alabama and tennessee, all of these have the affected populations at the lab for companions needed to do as much as we can to produce the burdens of dissipation in trials.
>> health equity is something very important to every single person on this committee and i'm sure, it may not be on the minds of every american but i think every american would agree everybody deserves not only equal access but also equal carousel thank you so much and i look forward to seeing you continuing. thank you for being here today. i got back from a creditor. >> it's my understanding doctor ruiz is with us and i recognize him for five minutes for questions. >> thank you for holding this important hearing today. as a physician and chair of the congressional hispanic caucus powerbrokers who grew up in practice medicine in the medically underserved coachella valley area in california, happy to hear some new members have diversity in clinical trials. gender, race, ethnicity, age or lifestyle playing a role in how
bodies move on to different medications and what's more that this proportion of the impact on communities of color highlighted vital importance of inclusion, diverse, participation in clinical trials addressing social determinants of health and creating greater health equity has long been a top priority of the congressional hispanic caucus. for the past several months i've been working with fellow hispanic caucus members from senator menendez on the important legislation aimed at improving diversity and inclusion in clinical trials for engagement of communities of color and decentralized clinical trials in developing solutions to better recruit racially and ethnically diverse populations. nih and fda engagement will be key in approving outcome and participation of communities of color in clinical trials narrow degenerative diseases and
beyond. the first question is doctor catatonia, how do you determine of the cortical trials is even a drop application representative of the u.s. population and when, if ever you require sponsors to increase the size of clinical trials to accurately understand their different outcomes and demographic groups. >> we have issued guidance to encourage them and make sure entry criteria for clinical trials as much as possible and encourage them to ensure the representation of racial ethnic subgroups that are relevant and represented. when we talk to sponsors about
the design of clinical trials, it's very much top of mind and re- discuss with sponsors approaches to make sure the clinical trial population is as close as possible representing the scope of groups that suffer. this is an area that's very important to us. we have made gains the past few years but shown in the clinical trial that a while ago and we need to continue to focus -- >> thank you. given narrow degenerative diseases, and use of critical trials improve the experience and make proper dissipation more accessible for more diverse communities?
>> we think, as you say decentralized trial approaches in the reading controls with the participation and inclusion of patients, particularly patients in underserved communities in melted denver-based diseases. diseases debilitating such as the one we are discussing that are dependent on caregivers going with the patient to the clinic, we think these centralized telehealth simplify clinical trials and make it easier to connect. from the patient's home or to go to their home to collect, they are critical to ensuring conclusion. >> i-40 seconds left. i'd like to ask what your perspective on the importance of
diverse predicted summation for therapies being developed for neurodegenerative diseases and what you think about improved community outreach and increase diverse precipitation. >> it's important not just for all because we already know risk factors differ across the population. cardiovascular ones is imperative. as far as outreach, the nuance will help to rather than the current situation we may have to bring people in to do a pet scan and blood biomarkers. we also have assistant in real time for tracking individual studies for demographic characteristics so we can have an annual report and see if we are on track.
>> the gentleman time has expired. colleagues, we are going to send witnesses, we are not going to break because we have seven bills on the floor that we have to vote on so we will take a recess. i think we will come back, let me just make this announcement. robin kelly and then we will recess coming back at approximately 3:30 p.m., 15 minutes after the last vote is taken. hopefully books will, and sooner than that so i encourage members and witnesses to return in a timely way. one more member, congresswoman robin kelly and then we will
recess, i think everyone needs a break anyway. the judge a woman from illinois has five minutes. her questions and then we will take her break. >> thank you so much. i want to talk about clinical trials and the concern about how they reflect diversity about populations so we need to ensure clinical trials are reflective of racial disparities neurodegenerative diseases according to the als. black patients are 2.5 years younger than white patients at the time of the onset. when here delayed in receiving als diagnosis. this is one of the many facts that highlight the need for clinical trials. what guidance has nih provided to sponsors to ensure demographic diversity in clinical trials are reflective
of disease disparities including race, ethnicity and gender? >> thank you. at nih, the trials we've done, we are obliged to report what the population is on a yearly basis and re-examine those and if falling short, we put in changes to approve diversity of participants. we also find particularly relevant here, there is research that tries to understand the best way affecting these in populations because as you mentioned there is a problem for and as these treatments come along, it's for the diagnosis to be delayed because it something called detect private of impairment which is trying to figure out how to know if somebody is developing these diseases and that's done in many
different regions for that purpose. >> is any additional benefit for having them work with nih to establish clear and measurable diversity and funding applications and have the goals publicly available and employed throughout the trial? >> my understanding is that nih we do what we have to do we do it to say i don't know that we have any leverage on the industry. the fda might be able to seek with the fibers are, we don't have any leverage that. >> we do need to improve clinical trial in the process. basic research to understand how these devastating diseases work. this will ensure we have continuous pipeline of novel therapy. what are some promising areas of research? how can we increase the
likelihood that this translates to novel therapy? >> we come in the last few years to recognize multiple pathways at a level that contributes to alzheimer's as a clinical syndrome remake seek progress translating the information already in clinical trials that target so maybe the best in others maybe inflammatory. in others are proteins ranging from drugs and small molecules to individual combination behavior therapies as necessary and we are talking multiple such pathways with the notion that a personalized precision level we are going to have it not just in racial and individual designing optimal ways. >> thank you and i will yield back the balance of my time.
describe it. i just leaned over and said to the ranking member i've learned something the week that proceeds heading into a major break. i don't think i will ever schedule a hearing up again again because we have absolutely no control what's going to take place on the floor so apologies to everyone especially our unisys who have flown across the country to be in the room to testify in a wheelchair waiting. our deepest apologies. so now back to where we left off. the chair now has the pleasure of recognizing the judgment from oklahoma, mr. mullen for five minutes of questions he's not
ready? all right. then we will go to doctor joyce. you need to unmute. can't hear. i think we will go to the gentleman from pennsylvania, doctor joyce for your five minutes. >> thank you for yielding. to this panel of witnesses, her testimony today, a long day of incredibly important mission. these diseases devastate the lives of those diagnosed the
impact family members and all around them. it is critical we are putting forth the proper set of policies that will foster the research and development of cutting edge therapeutics cures that will help for these diseases. need to impact my first question, my home state of pennsylvania has been a global pioneer gene -based therapies which could offer incredible hope to patients with neurodegenerative diseases. does the fda have necessary resources and workforce expertise to ensure timely review and approval of these new medicines for years to come? >> thank you for that question
and i will assume you would like me to comment broadly on gene therapies for neurodegenerative diseases so i can semi- touch on that. this area is an area that is exploding when it comes to research and a number of programs coming to fda for review. the review is done not by my center for the center of biologics so area where we need to continue to be able to invest in a highly specialized workforce that would allow us to pace with both volume and scientific advances we see in gene therapy understanding this area is particular the important for advancing therapies for
neurodegenerative diseases. in order to do so, we will meet resources as well, who will need to be able to recruit the right scientists and personnel and experts and be competitive with the private sector and acknowledge the tremendous support by congress by passing, by giving the hr tours authority which is the hiring authority that allows us to be much more speedy and competitive with the private sector recruiting scientists such as the one we need to review applications and therapeutics and gene therapy.
>> we are going to be hearing with our second panel of witnesses, the fda has provoked commitments, provide regulatory flexibility or consider risks and life-threatening nature of diseases when making regulatory decisions about drugs to treat als. as outlined in the september 2019 guidance, als clinical trials, what is your response to this? >> thank you for the question, i think it's important to this country how we are operating in a manner that's entirely consistent with als guidance we've put in place. when it comes to what we ask and develop clinical trials and
conduct them. the guidance is about how to develop fourth als. at the end of the day, our decisions are based on the data derived from this clinical trial. >> do you feel guidances necessary and is it reliable? i can't wait to hear the comment on this. >> we think the guidance is very important for developers. at the end of the day we have to make decisions on the basis of the strength of the data derived from those developments and clinical trials, they have been in compliance and according to the guidance. however, sometimes we are disappointed by what the clinical trials are in terms of results.
sometimes they are very complex to understand or sometimes insufficient for us to make the determination to be approved. >> my time has expired. my time has expired, i yelled. >> the gentleman yields back. the chair now recognizes the gentleman from oklahoma, mr. mullen followed by the gentleman from utah, mr. curtis taking to republicans in order because he's waiting on the committee so we will hear from both of you first. >> i'm not letting john getting front of me. >> i'm ready to go. >> thank you so much, adam
chair. let me throw out some questions doctor, there numerous documents from the fbi to discuss the importance of the flexibility when it comes to approval for als therapy. >> excuse me, i'm sorry, can you speak up? we are having a hard time hearing you and we don't want to miss a word. >> i am so sorry. hold on. is that better? >> it's a little better. just speak up. >> okay, i've turned my bike up all the way. can you hear me now? >> we can hear you. thank you. >> there are numerous documents from the fda discussing the importance of exercising regular try flexibility and approval of als therapy.
do you know how how the fda has exercised regulatory flexibility when it comes to reviewing clinical trials with als? >> we exercise regular flexibility as we review data from all life-threatening disease including als. our ability to fully exercise regulatory flexibility within the confines of statutory ways is at the end of the day, it's depend on the data in front of us and what the clinical trial results are telling us so there are times when despite the fact that we are very committed and willing to be flexible as we can within our standards, the data are not sufficient to exercise
the flexibility we would want to be able to in a situation als where there is such a huge medical need. >> the issue you have we feel like it is possible the drug were work well as some stations and not others. just like chemotherapy treatments work well for some cancer patients and some others. the fear is sometimes we are waiting on the fda to have approval but yet als patients are saying listen, we will track whatever. if it's working in some patients even though it may not be working in all patients, the ability to try, we passed a bill for pediatrics that the right to try pediatric cancer patients, the right to try drugs available. i think that's what als patients are wanting to do.
>> these are very important points but it's important to distinguish the access and importance to provide access for investigational therapies through expanded access programs are right to track. the importance of being able to study drugs and controlled clinical trials and way that would allow us to understand whether they work sufficiently faced so we try to balance that need by working with sponsors to put in place clinical trials that have the best chance to get sensors including subpopulations and i recognize and agree that this is heterogeneous. in parallel to that, who work with sponsors to facilitate access to treatment for expanded
access into the effect we try to do everything we can to facilitate those programs. >> i visited with some consistency that have responded well to some of the als treatments they preserved is freighted with critical trials some would love the opportunity to do that and i wonder if the fda doing things to ensure treatments that are available they some success with are available to other patients who would like to try it because i know we have had several reach out to us and say spoken to constituent a they spotted will but we've tried to get involved they were being denied and i'm wondering if the fda could be more flexible on the. >> we try to be as possible as we can within our authorities.
for instance, we work with sponsors and encourage to have open label extensions and clinical trials after the controlled base of the trial so that patients can move into extension and have access to the drug. we also encourage sponsors to make the drug available through expanded access after the trial is completed or even only after the control stage of the trial is completed. ultimately we need sponsors to be willing to work with us and obviously we engage regularly because we really understand tremendous urgency and mathematical need. >> i am out of time if we can be helpful to you in any way, if there are any barriers in place to do that, let us know.
thank you, adam chair. >> the gentleman yields back. the chair is pleased to recognize the judgment from utah. >> thank you and i'm pleased to be here and it's an important hearing. you're going to hear my question the theme heard throughout the day, i would like to start with doctor cap mazzoni. my colleagues and is to trust cap homes often communicate to me that the fda needs to be less risk-averse in the a buddy to access treatment under expanded access the right to try is one reason why. i have here in my notes without undermining patient safety but i'm going to put an asterisk by that because our patients are not exactly worried about their safety, they are worried about living. with that in mind, is there more congress can do to make the fda and pharmaceutical community more comfortable offering these
for instance we do have a higher threshold when it comes to safety. has so much rescue. >> and i know that your nature is not to take risks and we need a different paradigm i have been noticed and touched by all of my colleagues you have asked questions that somebody that is afflicted by ms or als or down syndrome als
has ravaged my neighborhood. very good friend the he has been able to travel worldwide that some are unable to receive those that are under clinical investigation he is willing to travel to spend more the average person can do in that can be a challenge ask special he participation additionally is hard how many react to the experimental drug is the more we should consider doing without undermining the integrity of the trials when i am referring specifically to congress. i know you are doing your job what can we do to expedite
this speed back when it comes to understanding that is very important to advance the therapies for als and continuing to support us as we continue to focus on expanding the criteria for clinical trials would be important we want to have as many patients as possible to access clinical trials and also continuing to support us in the efforts to engage for drugs with expanded access more available. >> i want to make one.with something that you brought up and we have talked about treatment. but i am not convinced we
don't exactly know that cause if it is environmental or not and how important your work is in that area i yield my time spent the chair is pleased to recognize the gentleman from delaware for five minutes spent thank you madame chair women. thank you to the distinguished panel on neurodegenerative diseases. and then to be personally touched i lost a friend and a loved one to als and a grandmother and great-grandmother who several dementia and the neighbor who died from alzheimer's and i have seen firsthand the devastating impacts the diseases have on loved ones in the told they taken working
families. with the non-latino white americans. and by 2030 nearly 40 percent of all americans living with alzheimer's will be latino or black. black and latino americans living with dementia are less likely to receive a timely diagnosis and have discrimination and less likely to be enrolled in cutting-edge research. and as i just mentioned in less than ten years and then
to increase the participation with clinical trials by a outreach populations and encouraging the participation burden. and as the national institute of aging funds research centers that major middle institutions between the united states however that analysis has shown that geographic distribution scroll screwed on —- skewed to those neighborhoods with your gear that characterization? >> . >> with the extreme importance of what you said and then to
that were not living up to standards. and then to increasing the visibility and with that among the things we are doing and then to with a higher concentration of historically black colleges and universities. and underrepresented populations so that they could establish the clinics that we believe so that funded research centers with clinics
and then we take that approach to be flexible as possible. and then we also accept and with that accelerated approval and in many situations with ecology and rare diseases and infectious diseases. >> i yield back. >> the chair is pleased to recognize the gentleman from washington state five minutes for her questions. >> spec take you to the
>> and we hope to get data from the recover study where congress has appropriated funds. to study tens of thousands of people who had covid to understand what the systems are. including children and pregnant women and people from diverse backgrounds and i would say that the cognitive problems that people with the persistent symptoms have are different than what you would see with alzheimer's. much more than what you would experience or if you had a concussion with more that attention and concentration issues one study day looked at the risk of dementia on they do see an increase whether or not people were having trouble
but that is still a hypothesis. >> thank you for that. but then related to children as the pediatrician i will say it most often with the seizure disorders so if you could just touch on the state of research pediatric neurological diseases and ideology if we have any more information there are any new treatment for epilepsy? >> i guess what we have learned from the large genetic studies there is an overlap between epilepsy and also with
developmental delay. so that is getting at is the problems that occur as the brain is developing even as the fetus. and we do have some clues and the question is how to go from those genetic clues to treatments i would say there are some very new genomic techniques that are coming out because we know the different mutations are they are very mutations specific and more precise especially young children with severe epilepsy and mental disorders are cognitive disorders. spent the gentlewoman yield back. >> . >> .
and all those in the committee with the input that we received from the neurology advisor committee. so we looked at the data over many months. after exhaustive and detailed review of the data provided over the course of the many months and also in different all of that together a concludes at the data on —- the data for accelerated approval of the condition and
the accelerated approval pathway of those degenerative diseases and that data made us comfortable with all the criteria for accelerated approval have been met. we views this to bring it potentially beneficial treatment but also incremental steps in finding ways to leverage the expedited pathways to bring therapies
for those degenerative diseases for patients who are desperately needing them. >> i yield back. >> i should know more about this but i am struck of the irony of that conditions under which the alzheimer's drug was approved understanding that it had limited help to patients which is one of the reasons we are having a hearing today to examine what fda is doing to bring about limited help with the als communities so i can
that so far? there are people over this country if i sound upset my constituents are here i have been getting calls from their friends all over the country and you will hear them and i hope you will stay to hear them. i know once we moved to the next panel that i beg you then those drugs that are there now
advice for developers so they can plan in place excuse me. >> and when you have people that created man's why we wouldn't do this and it's in your power what a blessing that is its in your power. >> i am begging you i guess. to give some help that there will be some accessibility due to the flexibility of the guidance. is there any more that you can
do? >> i hear you and we are working every day and how we help them put in place and also as flexible as we can and how we look at the data from those clinical trials. and dealing with als there has to be a higher threshold for risk and also greater tolerance and make discoveries that could help others and to extend their lives and i yield
back. >> the gentlewoman yield back and this concludes the first panel. thank you for your testimony and have heard a lot of frustration with the interim is challenge and as i said in my opening statement we cannot only make progress but as we make progress that we bring hope to the patient population
expanded access to the investigational therapies and those that will not clarify for trials. and 130 patients receiving nine different treatments with expanded access. one could found out they could summon the pool again and with the ventilator. and that does not interfere with clinical trials and with can additional approval we
degenerative diseases. largest trade organization developing the first product under multinational companies. and then across different diseases to identify those barriers with cures and treatments and then to focus with the goal to provide insight with is very important conversation. we counted 653 clinical development programs and with those oncology developments programs and with those approvals and that analysis
provides us with those any given disease area that needs to be resolved and then emerging companies as they are responsible for the partnership with the biopharmaceutical companies 47 percent of the pipeline over the past five years and with the recent increase of neurology companies to go public which is a way to generate investment dollars is not a single company and with all those companies to go public and to provide context 75 oncology companies went
public. it is not just at the level we would like to see or need. when we look at clinical success rates to seven.9 percent. with that success rate with these categories for example those biomarkers have a twofold higher success rate. looking quickly at alzheimer's and conversely other individuals may have similar but not exhibit dementia.
delivered it is welcome here. is a any recommendation with the legislation. >> i have to agree and the als community agrees that as mentioned these bills on the floor commit transformational change to help preserve clinical trial integrity and help access to experimental therapies and actually in those cases of expanded access to gain data to help understand the disease and improve our understanding of the biology and those that
have you and we look forward to your testimony now. i want to add because i did not recognize our colleagues from illinois the democratic lead on the legislation. and i'll look forward to this committee considering it and thank you for the work that you have done. thank you for being here for the second time today. if you go over time you will not lower the gavel. >> we would be here all night and into the morning it is a
critically important hearing with tens of thousands of als patients have been waiting for this moment and we are so grateful to be here. >> and to testify that before today. >> i'm a caregiver and brian's wife and as you are here today i will be his voice. we have two young daughters one turn 62 weeks ago and her second daughter turns one on saturday. professionally i have served as a staff member in the house , in the senate, federal agency and brian and i together served as staff members in the white house. at the time he was diagnosed at 37 years old assistant
science that currently exist or therapies i can keep patients alive. and i'm excited to tell you that the answer is abundantly simple. make the fda act. to approve the drugs that are stalled right now. it is abundantly simple. with a biomarker to use the measurements to approve therapies in the past survival
link to walk up the stairs. it's extremely important this committee to know the date of the facts and with that als rf scale. one of those to drug stalled now also show dad it did the same thing. and that gave them longer to live a compared to the placebo and with approval in canada and europe that the fda had one year ago it begs the
question why didn't fda approved this one year ago? the trial for the second therapy being stalled that showed the patients that started the trial with a score of 35 or higher on the same scale and received the drug is two points higher. we need science and data and he did say the data is publicly available for patients who entered the trial with 35 or higher they scored two points better than the placebo go back and look at the fda website it is the exact same type a success rate when it was approved in 2017 that begs the question, why has this era be which is successful for a subset of the patients not being approved?
>> a friend of ours who can now buckle his seatbelt and now take his own pills. and with expanded access on that flight you will see very clearly in order to be eligible for expanded access on the website patient moment is not possible that may be super clear. expanded access in no way endangers a clinical trial.
if that is true i would ask the fda to publicly announce their support and doctor andrews also said is all aligned uniform expanded access is not affordable to those that are we need the funding if anyone was to see expanded accents and that is the vehicle to do so. all the more shocking of their own september 19 guidance i'm
so grateful so many members brought up today. however to exercise flexibility and urgency and what we have seen is even less urgency and less flexibility the two drugs i have repeatedly mentioned to run another large placebo-controlled trial. that means these therapies will not be approved for four years. let me remind you. so how can congress help?
>> fda cannot answer that question so we as the patients who are dying and patient on —- lives on the line million threat for you. and then urge the fda to approve today. two, urge fda to approve medicine for the subgroup today. three. and that expands access today. impasse the promising act to have rapidly progressing fatal diseases even beyond als. spent tens of thousands of patients are watching this from their homes wheelchair-bound some on life
support, watching this today. there hope this in this hearing. some have waited and postponed their decision for suicide to see this hearing. i don't thank you understand what this hearing means to us. please do not let another generation of als patients die in pursuit of the purpose. please let this be the first generation to survive we want to live. you have the power to make it possible. thank you. >> i think there is applies
from the entire als community offering your testimony. we are so in debt to you thank you for your clarity and your courage thank you for the patients you have exhibited today and i don't think that every single member of the subcommittee is moved by what you said and that we are determined to pursue exactly what you set out to do in your testimony. thank you. thank you. thank you. thank you. next we welcome our guest and willing to testify them a
and those health issues of high blood pressure and heart disease diabetes and stroke they are two times more likely to get the disease than white people but this disease is not who my mother is even though it has taken control of her life. the city on the coast she grew up poor in the eighth grade. my mom was a big dreamer who wanted more so she wanted out of santo domingo with no work or no money and no hope. in 1960 and then went to beauty squirrel school.
and then they were married nine months later in my dad and my sister both died so it's just been the two of us for a long time. and then began to hallucinate of a boy and top of the refrigerator she gave all her money away to miller psychics promising her riches and promised her every day. and with black and brown people after series of test telling us he had alzheimer's
disease. gave us some url told us to google it and centers on her way less than ten minutes. clearly he had no time for us and barely looked at my mom. i went home and cried. and then to access the form all diagnosis and those that are less likely to receive a timely diagnosis compared to non-hispanic whites. i was ready to fight this disease i switch neurologist and another one for clinical trials to help with hallucinations. to make up less than 2 percent of the participants currently
enrolled in research funded by the national institute of health. i was so that you could be part of one but it was grueling she was in the bronx and i was in philadelphia i had to get her to new york presbyterian hospital every week for six weeks to have psychological exams before the meant one —- medicine was administered. we barely had enough compensation to carry on —- cover and huber. with that geographic distribution of what the research centers toward the
most wealthy neighborhoods balancing that participation my full-time job was another challenge and then to be at every appointment. and then to make it work my employer has paid family medical leave and flextime but millions are not that lucky with the urgent health equity for caregivers. according to a national survey and have access to paid family medical leave and more than half of caregivers who utilize benefits reported better emotional well-being according to the 23 percent to didn't have access. it is critical and it became
and then i had to placement a nursing home it was the hardest thing ever had to do in my life. and every floor was affected with covid-19 and no physical contact with her she is now nonverbal and cannot feed herself and every day i wait for the call that says she has passed. but here's i'm asking on her behalf. for equity and diagnosis and detection and with men to give
your mother and as her daughter is the story so many americans and i look forward to the day when the policies that you articulated that are needed because it would just show the american people so well these are struggle 20 farmer struggles on a daily basis in people's lives have the privilege to take care of my parents and it was a huge responsibility and i was is working and everyone else was working as well. there were so many things that you brought up those are huge burdens am by no means that
some people think of the handouts i say go walk in your shoes. >> and then to recognize this came up with was a huntington's disease patient and i caregiver. >> we are prepared to have your five minutes of testimony thank you for your extraordinary patients. >> good evening. and we have huntington's
disease that only secondly officially diagnosed. i am a caregiver today over the years i taught myself how to emotionally disconnect to allow me to be able to separate the huntington's disease and with that situation it is unbearable. huntington's disease is a rare disease that causes to jen did on —- degeneration the brain it affects each patient differently the progression time is different but it is fatal with no care. every other neurological disease happens in the prime of their life, in their forties the highest income
there with the members of the subcommittee we apologize for keeping you waiting all day and into the evening. but you enriched your hearing with your testimony. but now we have completed the testimony of the witnesses what you need from congress and the fda to continue to innovate on the clinical trial design. with the existing regulatory
authority with those clinical trial results with the disease they are reviewing for it's important to note something like als that has a 0 percent chance of survival that is something shows a clinical benefit to retain function and slow disease progression and extend survival and is safe and well tolerated that the framework that is typically used by the fda doesn't work for fatal degenerative disease like als. in doing so, this can spare innovation and to come into the space and then we are stuck and then it drives people from that disease and in that way the bills that we
talked about to urge some regulatory flexibility and abiding by the fda guidance that was finalized in 2019 can help that among the crowd on —- the clinical trials. >> . >> and that was biomarkers. it seems to me the fda stood on that with the biomarkers with the drug development it seems to me the needle is stuck. can you cast some light on this? i pointed out in the comments of what was stated there seems
to be an irony of results even though they are limited even they are desperately needed they don't move in that direction but back to the biomarkers. >> he was to know it is true you don't fully understand the biology if we did we can cure today. . . . . we can do both. >> this sounds like the european
model to me. >> this is part of the reasons why the als act can help in this situation. it's difficult for us as americans to seek potential therapies get faster approval because regulatory review process by different registration could help harmonize locally in developing innovative therapies for these conditions. >> thank you very much. how can we get more companies to participate expanded access? >> thank you, chairwoman for the question. generally speaking, they do have unique multiple factors they have to evaluate including the resource capacities to see in
the programs including rationale on which basis they would be denied. the questions around the ability to scale up as well as thinking through impacts of clinical trial enrollment so there's not a one-size-fits-all for each company for how they approach, medicine approved so they are available to all. i will note that requirement for companies to provide information, there may be something to think through to make sure more people are aware to find these programs start something we can maybe work with you all on. >> thank you, my time -- i've
exceeded my time. wonderful ranking member of the subcommittee, you have five minutes. >> thank you for being here and i know when we talk this week i was talking how important this for patients to come before congress, it vitally important people get the impression there well off and over that testimony, it moves us and hopefully inspires most of us, the most important meeting i've had in 12 years, we have eight or ten a day in the one i could choke was most moving, nine or 10-year-old, which and her brother taylor, she came with als and she said my father cannot hug me anymore but i want to make sure no other little girl go through what i'm going
through. a great family and i'm always been an advocate because they showed up. i do have personal experience with als, unfortunately, many of us do have personal experience, i have alzheimer's in my family. thank you for being here and sharing your story and making a tremendous effort whether you came with the tremendous effort to be here, and roger here. earlier this week you mentioned your involvement with the clinical trial and shared exciting news. your mother had her first clinical trial. can you describe the opportunity to pipe to participate in some of the changes you would like to see happen reflect the needs of the community?
you may be on mute. >> you have to unmute. >> can you hear me? the clinical trial up next find my pack. i think god wants me to help others and call this interaction. this brings a lot of challenges. in clinical trials, it shown that i have been able to help those at risk. families are looking for ways to treat the disease. this element has helped me, my family and many other families. now my mother is enrolling in
her first clinical trial. it does damage to your brain and can't be fixed. part of what can be done in working with my local group, fda has been a good partner with the community. i would like to have the fda think about ways to work with the community to recognize the difference patient the patient some we are not missing out on patient. >> thank you. my friend and member of this committee is working on social security we talked about and it sounds like your family had a difficult time getting your mom's disability application approved and it felt like they didn't understand hd, how does that impact the expense of your
family? >> the disability process is very difficult and frustrating. it took 16 months to get my mom's application approved. she was denied twice and it wasn't until we hired a lawyer she was finally able to go. the judge backdated her eligibility and medicaid right away. however, are the families cannot afford a lawyer and it takes much longer. many individuals and families including mine how to support this case so they can work as long as i can to try to prove progression and help insurance through their employer so that by the time they apply for disability, they really need it. men wait years to get through even longer is unacceptable.
devastating. access to medicare immediately as an added hardship the families do not have to endorse. i'm asking congress to pass this act. most people have not heard of huntington's and even those who have don't understand. is our experience with the process. while hd is on the compassion catalyst, to my state. for hg patients, their physically disabled. many patients experience
cognitive and behavioral. they are don't understand the impacts on the person. to confirm they have it, it makes the process even more challenging. it's about how we can ensure patients get what they need quickly. the center of excellence experts. even if it only takes six months to get through the process, there are still delays in cash make payment for five years. >> i think i'm running out of time to ask questions but thank you for answering that. i will go back.
>> the chair is pleased to recognize the chairman of the full committee for your five minutes of questions. >> thank you, chairwoman and everybody for testifying today. this was incredibly informative. heard from some of you in the last two months but today it's made a difference in our understanding where we need to go so i wanted to mention obviously there was a lot of patience, a lot of information from patient and family access to clinical trial can be difficult based on disease progression and other factors. designing trials for people to participate our understanding clinical benefit developers up
als treatment. more patients can participate in clinical trials criteria run for one group to be considered for primary analysis of the product group for secondary support of analysis. you acknowledge, how has the industry responded to this? greater crossings from the agency needed invest space? >> thank you. i don't know that i can answer that question with specificity but when you think about crimes, i know you had been some time since i had been updated. it's important to keep the process including engagement between regulators and patients in the medical community and pharmaceutical industry to make sure the kind with needs
understanding of science. also i don't want to take up all of your time but inclusion criteria, in addition to what you're saying from the larger conversation looking about how we through through basically what we learned during the pandemic amount of companies are re-examining how we should be thinking through to ensure does not bias and if we brought this income is how can we use the modern tools at our disposal and it will improve how we analyze data and diseases and outcomes? is a lot of interesting work going on we are ready to advance all concepts.
>> thank you. to get to doctor sanders as well. i know your organization is a lot to talk clinical trials and criteria, how would you say they're implementing that guidance and how can they do better? >> i think it's fair -- [inaudible] developers have taken the guidance apart but it takes two to tango and fda has not taken the guidance apart so what i mean by that for the neuron trial they have more patients.
the neuron trial had more patients further progress in the disease than any trial had before and that was used against them when they got approval so we need both for guidance. >> all right, thank you. >> doctor andrus, and you offer insight on how they could be better designed to allow for more inclusion? >> in collaboration with the fda have designed the called a platform trial and it has broadened inclusion exclusion criteria to allow more patients are people living with als to participate in clinical trials. the issue i think is its troop manufacturers are trying their best to broaden inclusion
exclusion criteria. many of the drugs testing are trying to slow down progression so often previously inclusion exclusion was stringent to get very early onset or early stage disease which meant more than half of people living with als were not eligible and that's what led to this lack of access and no other way to gain access to experimental therapy. that i think is changing across the field but as mentioned, it will take, in order to design clinical trials more inclusive, we will have to employ technology so there are ways to strategize and upset the populations you think might respond better to the particular drug but we need the help of our regulatory colleagues understand how to employ that and it will try. if there is a prespecified self
population, therapy your primary analysis, they have to accept that so we can include a broader population so there are ways to design but we need to work together with regulatory colleagues to ensure that would be acceptable to the. >> all right, thank you so much. they can, manager. >> the chair is pleased to recognize the gentleman from virginia for your five minutes of questions. >> thank you, appreciate it. doctor enders, i'll start with you. in regard to als, supposed to be relatively rare, not as rare as huntington's but rare. i know four or five people who have had it. is there any research you are aware of that's going on with like a geographic outbreak? at one time, about four or five year period, i had three people i knew who in the earlier part
of their life, had all lived within probably half mile of each other, is there any work on outbreak of als? >> historically and als we've identified populations specific geographic areas like warm for example, had high rates of als. an environmental exposure over the course of the study, there have been many epidemiological studies looking at environmental exposures and toxins that may increase your risk of als. it is actually from als association, one of the priorities of study risk factors and that's why funding to the cdc national als registry is quickly important to identify the clusters. one of the most important risk factors regarding identified and acknowledged is the service by veterans. military service, two times the
general population. >> is a thought today, and i know this may not be directly your area but it's the thought that it causes it or maybe a trigger for the onset of als? >> i think that still yet to be determined but there are definitely exposures identified as association. >> i'm sorry, i was talking about exposures to environmental -- remark the happen associations identified and further research could be helped. >> not going to go to huntington's but i'm going to ask a question because i think it's something we need to look at and i don't know the answer and you probably don't know either but it gives me a good platform if i ask. he set in regard to als, a cure today if we knew what caused it. with huntington's, we know what caused it and yet we can't still hear it. if you have any comment? >> i can only talk about als specialty but as i said there are als also has minority
patients have the genetic contributor to their disease and there are technologies that can be targeted and deployed for gene genetic diseases. >> thank you, i'm going to go to miss booth, i could ask you a couple questions. what i want to know is the clinical trial you are involved in the clinical trial her mother is involved in, are those drugs to treat huntington's? >> no -- can you hear me? >> yes, ma'am. >> both of progression now. there's nothing to treat right now but it's the progression of it now. >> so you don't know ever any genetic work they are doing to maybe figure out how to solve -- i bring it up because one of the biggest successes nih has had
recently is sickle-cell where they were able to completely change the genetic makeup. hannah huntington's would be more complicated and am wondering if you knew of any work that involved dealing with changing the genes that cause this, particularly if we can catch it before the onset. >> i'm not familiar and i know that's not the right answer. >> i understand, i appreciate that. i've been interested in huntington's sometime because there is a family, south to families i'm aware of in my community that have huntington's, one of them, the first family was good friends with the children of that family and her new mother who died of huntington's. this was, a lot of this happened before they had testing available. your mother who didn't want to be tested but always had a happy attitude until recently with the
onset, i'm curious if any of the data you've seen working with the association indication that there is a higher rate of suicide for those who do get tested and realize they have the disease. >> i think there is a higher rate, there is no cure in the only two medicines out there -- other than that, other drugs treating the symptoms so you get to helplessness or the area of the brain causes mental illnesses and stuff so there is a lot higher rate of suicide. >> my time is up but i want to ask the character so i can tell you for a couple more seconds as
a result of your testimony, i have instructed my staff to sign me onto hr 2050, one of the request you made so thank you for your testimony today. >> thank you so much. >> the gentleman yelled back. the chair is now pleased to recognize the gentlewoman from california for your five minutes of questions. >> thank you very much, madam chair and thank you for convening this hearing. it's been wonderful and lightning on the subcommittee. i want to thank the patients caregivers, advocates because your testimony here really does help us as we look to make sure we do everything we can to address the diseases involved in what are having to do with your work every single day. for me, i feel each one of us
could be involved in any way. to hear the testimony of the caregivers, but something all of us know that it could be something we can be involved in for some time. let me say this, i've had to follow-up on an earlier discussion from the first panel, a patient experience drug development and application with the fda. what steps did you take to correct patient expense data and how is it communicated to fda? >> thank you for that question. as you are very aware of, the 21st century cures, there's a tremendous effort to set the framework with this systematic approach to appropriate patient patient data and the entirety of
drug development and drug review. i would say we are making tremendous strides collecting patient experienced data on a much more regular basis provided as part of the application that can be in terms of primary, secondary endpoints and patient preference for context about how to think through benefit and risk. we want to advance a lot more so there's still work to be done. another requirement was patient perspective data is submitted as part of the package and review, it's required to report on that fact. we would like to see that reporting to provide more context to gain insight, not just that it was reviewed but what impact did it have on the review decision-making? that will help more of us understand how develop stronger
from the strongest possible data to continue to inform regulatory decision-making. we also enhance the a buddy for misinformation in the label to be communicated to the patient, their families and their position so these are things i think we still want to move forward and continue. >> fda has indicated data differently for different areas. patient experience data differently -- >> i'm sorry, i'm not sure i understand the question. >> fda indicated patient expense data different for different disease types, how are the developers collectively use data different also? >> i don't know that it's different, i probably depends on again if you're trying to support say a benefit risk
position where some of the patient's on the panel have talked about the value of being able to improve quality through daily function patient, it's very important and will focus on back so i don't know that it different from other might be different questions trying to be asked. does that make sense? >> thank you for your testimony, in your testimony, your view that fda could do more to consider patient experience risk benefit analysis so what is the best way to approach this given that experience the input in patients may vary? >> the best way for fda to approach patient reported
outcome to proactively work with drugs to incorporate new technology that allows us, as patients, to provide information that was never available before and that information, as doctor andrews mentioned, can help us understand it's a therapy that's helping us retain function which is everything. so be more forward leaning is the key here. >> thank you so very much because i believe very much the
patient experience is very important as we look ahead to address these issues. madam chair, i yield back. thank you very much. >> the gentlewoman yields back. the chair is pleased -- always pleased to recognize the judgment from georgia, mr. carter, our resident pharmacist. >> thank you, madam chair. let me begin by thanking you for being here and thanking those who have joined us virtually for your testimony. let me say that your presence here, virtually, brings a human touch to als, huntington's and that's extremely important. i echo the remarks of my colleague, representative
guthrie when i say that it is important for you to be here, it is important for us to understand. as chairwoman said, i am pharmacist and i practice pharmacy for over 32 years and i will tell you through research and development, i've witnessed nothing short of miracles in the way of medicine and it's extremely important that i continue to encourage companies in the united states federal government to invest in research and development because as i say, i've witnessed nothing short of miracles in the years i've practiced pharmacy it is extremely important and gives help to all of us and gives it to you so please understand i support you. doctor, i want to ask you, in your written testimony you suggest that investment in
>> these are risky investments. i have seen drugs get all the way up to the final stage of development and then have to be pulled. and companies understand this. not trying to give the pharmaceutical manufacturers a free pass. they need to do a better job with prescription pricing but at the same time they do play an important role come extremely important role of research and development that is something we have to encourage. in discussing your testimony the als drug but unfortunately 87 programs what do you which should be at this long-term lack of success in one of the biggest barriers to get drugs
to treat neurological diseases approved? >> . >> alzheimer's is a complex disease. there is over two dozen genes we have associated with alzheimer's. but i will say that what is different than the generality to do early safety studies. in terms of transitioning from phase two to phase three but in phase three is the brick wall so this is an exciting time to see the first disease modifying program get approved and when you think about what that approval did for cancer
it will allow us to gain further understanding and we do think it will have a positive impact on investment. >> research development is extremely important and thank you to those who are participating virtually. you bring a face and a voice to what otherwise is just a disease but it is real and impacts real people and we must commit to continuing research and development. thank you madame chair i yield back. >> i want to express not only my condolences to you congressman carter but all of the members of the subcommittee and you just tommy lost her father. you have our sympathies in the
approach expanded access and to outline in my earlier answer to the question and further that if they have the expanded access to provide the information on the website if they have a program and if there is an information to help navigate and work with extended access programs available you have a very important voice so paid
times we are caught between our children and parents so having a policy of paid medical leave could have a huge difference with so much pressure. because especially when she was first diagnosed it was complete chaos and i didn't know what was going on so i just beg you to please help us. help parents like me. >> thank you very much i yield
would reflect somehow i'm wondering if there's anything congress can do to help the pharmaceutical companies or transparency they would not necessarily need to be public or is it another way we could help them with a barrier they might have is that a barrier in your mind? >> it's important to know that some of the barriers we have talked about heavily expanded access mechanism and then to
make things easier to access the monitoring of safety within nine academic center that requires additional paperwork and resources at the clinical site are essential in and that the clinical sites not have use of any facilities and single resources are the biggest barrier and there is experimentation with remote
trials. is that something we should expand fuller and how big of a deal is that into here with the advocate for access to clinical trials. so anything we can do with specific issues that has been highlighted with limited access and then try to shift to patients that can be difficult across straight lines so the second is issues but regulation or principal investigators into make it that it's easier in the
practice of medicine research. >> thank you very much for the answers. >> the gentle manuals back. the chair is delighted to recognize the gentlewoman from delaware. she may thank you madame chair woman. first, thank you all for your patience. it has been a very harrowing day here but it was important for me to be present. i am so grateful and thankful for you to take the time and
sharing. kayla and mr. carter and i heard the word voice that you are the voice of millions. and as i sat here, i recalled losing my father-in-law to als and we did not even know for months what was wrong with him. it just reminds me how important our work, and a bipartisan way come is to the american people and to the families. and then as we talked about her mother and my mother and her mother had dementia and her mother's mentor on —- mother would wander and i
wanted to stay. i had a few questions of which i can submit in writing because i want you to know how grateful we are for you standing up and speaking up and in your testimony you mentioned the bill that i lead which is really about expanding clinical trials for alzheimer's i was hoping that would help the situation how can we strengthen engagement with communities of color and
working families to make sure there is an interest of research of clinical trials? but to explain to have a lot of compassion to give you the diagnosis. that's what i saw and it's really important to how you communicate with these communities, what type of messaging, website, handouts to make the communities safe
and the only reason i got that far with my mom is thanks to my mom i got a college education and they learn to advocate and to have that chronic - - clinical trial and what is happening to my mom. >> and the messaging is important. >> and in terms of whether research is done to break down those barriers. and when kaylee was - - kayla was speaking about social security disability and i recalled the intern for the current senator in 1988.
and this is a commercial for families that the offices have caseworkers that can help navigate social security disability. that was one of my jobs especially for those who might not have the resources. and specifically the two things that you talk about going across state lines maybe we could follow up with you afterwords with state and federal problems and are barriers and follow up with you afterwards science is important and i yield back.
>> thank you for your beautiful remarks. >> thank you for this important hearing we are approaching ten hours in the impact coming to the people's house is so important i will never forget your words if you had to choose between the problems we have addressed , would you look for more innovation, more accessibility , more flexibility, or more urgency and response?
european union and the conditional approval pathway and that we have a conditional approval pathway for animals in the last but not human beings. it begs the question why we are doing everything we can to get their fees to people who are dying and to have the chance to live to see possible cures. >> thank you for an honest and courageous answer. i will take that answer. the clinical trials process
can be long and incredibly resource intention and and especially with the delays covid has played which really exacerbated what was stated and nodding in affirmation of that are there actions that could be taken our policies enacted to redesign clinical trials to account for those operations of the covid pandemic. >> at the very onset of the pandemic and then to continue to make sure how to document any missing data that has
occurred to revamp being operations with the pandemic. currently operating under the assumption that usa did put out guidance very quickly. but then that is what we're monitoring very closely. >> thank you for addressing that these delays are so important for the's people and time is of the essence. time is limited. do you feel there should be other steps such as increased patient access for investigational drugs outside of the clinical trials and we should be mindful of the
disruptions that have occurred in this pandemic? >> again, thank you for the question and we are supportive of the fact that expanded access is a tool in the toolbox to make a decision by expanded access. but i just want to underscore the other revolutions i think are important with the entirety of this conversation. some of the things we learned under the pandemic how can we bring those clinical trials closer to the patient so in addition focusing on improvements based on tools and designs and that is so
important my time has expired but thank you madame chair for this incredibly important hearing today and thank you for being here at this late hour. i yield. >> the gentle man yields back now the chair is pleased to recognize one of the new members to the committee and a marvelous addition the gentlewoman from minnesota for your five minutes. >> thank you so much chairwoman. let me just say that this is my second current one —- term and congress this is been one of the most impactful subcommittee hearings i have been a part of as a member.
meeting with constituents regularly of those that have degenerative diseases that this is just a devastating set of disease states so while there are huge advancements an understanding of brain illnesses and limited treatment options. i will add the impact on families that my own family watched when my grandfather had alzheimer's but we are so fortunate to have a world-class health care system. in the first certified excellence and those with the
most cutting-edge treatment that are only available in a research setting. still my constituents suffering from those illnesses if there is anything you made clear to congress here today is that point. i would say to our witnesses and to those in our community your message has been received by the bipartisan nature of our work on that particular subcommittee. congress, nih and industry must work together to make sure we are doing everything we can for life-saving treatment and ultimately to
cures. thank you for sharing your story and advocating not just for yourself that everyone who can to be here. so from your perspective what should fda do better to incorporate that patient experience into decision-making? >> and then to write the 2019 guidance that we talked about today. so at some point in time you begin to wonder if those are
get the answer will get that answer you just gave me to the question that you and all witnesses for every single one of us those pieces of legislation you have asked us to review i commit my team and i will do that. i just want to close this out we know health disparities especially for black and brown americans. can you talk and how you believe what we should focus on to close the gap. >> and there has to be more
outreach in the clinical trials and paid family leave why so committed to doing this. at far higher rates to be compassionate understanding that the government needs to focus on alzheimer's disease to help black and brown americans. >> thank you so much and chairwoman thank you for your graciousness. and with that i yield back. >> and we have exhausted all
the members of the subcommittee and to recognize the gentlewoman from illinois who was waiting on. you are recognized for your five minutes. thank you i am here in my 22nd here in congress but i also feel this is one of the most impactful hearings and it could be a real game changer i want to say to people who are listening there were hundreds if not thousands of people who are being advocates i remember the aides academic on —- epidemic.
and actually it was a population of people. mostly gay men at first and then the committee organized and was out there and fighting and no easy and partners to make things happen and that you probably recruited all those diseases we were talking about today. there is a lot more intensity of interest. i did learn that that the ms diagnosis als is diagnosed as ms. is that true quick. >> .
hopeless we hope you have the courage to see this through and we look forward to celebrating the end of these diseases with you. >> thank you. i'm so proud to have you as constituents of mine. and i am honored and think the witnesses we are not powerless here to make a difference and to partner with you to make them in the right direction. >> the gentlewoman yield back. last but not least we will close out with the ranking member of the subcommittee.
>> i heard just the tail end of that. thank you for being here. thank you for your compelling advocacy and sharing your stories. just talk to me a couple of times about this hearing and what a long day this has been. you made the trip and you traveled is not easy to be here and to share your story and it is powerful. powerful and we have all been impacted by you and others that have received a devastating diagnosis with als and this is an opportunity for us to come together to do what you just stated to make a difference. and i am reminded again of the tremendous research that is underway right now and we are on the verge of amazing
breakthroughs and we need to say yes and what that could mean to so many individuals that you represent a bunch of other people. thank you so much. your family has faced a lot of challenges and particularly at the and given the symptoms experiencing how common is it to receive services in long-term care facilities and how challenging is it but
>> parting is sweet company and it has been difficult because there is an emotional tie between those of us that are seated here and those who gave testimony today. ledger brought this hearing forward. was highly intentional because it was desperately needed. you have been the stars. every member has hung on every word you have uttered. what a wife and a partner and mother you are. what is citizen you are. you are a source of pride to all of us. and it is humbling to have you
come into our committee room that you would repeat the words of your beautiful husband to the people of our country and the representatives all of the witnesses that were part of the second panel, thank you. >> i also went but your message has been received by this congress and i am so proud of the bipartisan nature of our work and on this particular subcommittee. congress, nih, we was make sure we're doing everything we can to improve patient access to life-saving treatment and
ultimately. your presence here is so commended. after hearing your story and advocating, not just for yourself but for everyone who can to be here, what do you think that fda should do better to incorporate that patient experience and perspective into their decision-making? >> it took fda six years to write the 2019 guidance we talked about today so at some point in time you begin to
will get the answer that you just gave me to that question. you and all of the witnesses here tonight and trust every single one of us and the pieces you have asked us to review i commit my team and i will do just that. i want to close this out by giving you the last word. we know health disparities in this country especially for black and brown americans. can you talk about your experiences and how you believe what we should focus on to close this gap? >> that such an important question.
think has put more effort for those to participate in clinical trials and that we need better medical care and paid family leave. >> and then the black and brown community. and then to have more compassion and understanding and then should have one does help all americans. >> thank you for your graciousness in giving me a few extra seconds and with that i yield back. >> any time. and now we have recognized all
the members of the subcommittee and i can recognize the gentleman from illinois who was waving onto the subcommittee. >> . >> i also feel this is the most impossible hearings i have heard and it could be a game changer with the intensity. hundreds if not thousands of those who are advocates i remember the aids epidemic and
it was a population of people of gay men that were not especially popular necessarily and then the community organized and out there and fighting and noisy and partners to make things happen and you probably recruited a lot of partners. and all those diseases talked about today there is a lot more intensity of interest. but i learned that ms diagnosis and als is diagnosed as much as ms. is that true?
>> that is true. >> so my guess is there is more money that goes into msn people live longer we may disagree across the aisle because in some ways big pharma gets to pick winners and losers and may be the fact that als patients don't live as long maybe it may not be as useful but i think the government and taxpayers with that accessibility this has
if any of the other witnesses have brief i have a little over minute left. >> i really wanted to thank the committee for this hearing. and for seeing all of us, and for hearing all of us. >> put your microphone on are closer. >> and for hearing all of us in seeing all of us. and acting on all the request and to make us advocates today this committee and congress has the chance to end diseases we hope you have the courage
to see this through and we look forward to celebrate the end of these diseases with you. >> i am so proud to have you as constituents of mine i am honored and we are not powerless to make a difference and to partner with you in the right direction thank you madame chair i appreciate it last but not least we will close out the members of the questions with the ranking member of the full committee.
>> i just heard the tail end of that and thank you for being here and thank you for your compelling advocacy and sharing your story. you made the trip we've all then impacted and this is an opportunity for us to come together to do what you just stated in this tremendous research underway and to be on the verge of amazing
breakthroughs. and what all that can mean to so many individuals representing a bunch of other people so thank you so much. your family has faced a lot of challenges getting your grandfather the best care possible and particularly at the end, giving the symptoms he was experiencing, how common is it for hd services for those families to locate a facility to provide the appropriate care?
that's what we need to expand that. >> i appreciate you sharing the day with us in sharing your story and advocating for so many others. this has been an important hearing for all of us. i look forward to working together to continue to advance solutions. >> i yield back. partying is sweet company and it's more difficult because it
is the emotional tie between those of us that are seated here and you gave the testimony today. it was highly intentional because it was desperately needed. every member has hung on every word you have uttered. what a wife you are. with a partner you are. what a mother you are. what is citizen you are. you are a source of pride to all of us. and is really humbling for you
to come into our committee room. and that you would repeat the words of your beautiful husband to the people of our country and their representatives. and to the doctor and witnesses that were a part of the second panel, thank you. you have been so instructive to us and have taken the very real stories of your day-to-day lives with the burdens of humanity and the personal stories taking care of your mother or your husband and then speaking for all of the advocates in patients across the country speaking for 750,000 people that you are speaking for millions in our country.
and with long drawnout frustrating day. i know that you are exhausted. but none of it has been a waste of time. none of it. not one minute. even the waiting is worth it and we will show you that it was worth it by taking up the legislation, working together to pass it. i think there is a rocksolid commitment to advance this legislation of doctor andrews and others as advocates have said be have been instructed to us. this is what we need is the right ingredients it's the right recipe to address what is ailing us. my hope more than anything else is that hope comes out of this. i have confidence that it
>> to the wonderful ranking member i have a request for unanimous consent to enter into the record wonderful list of documents national organizations i can read them all. no objection. thank you very much. so ordered all of these documents will be entered into the record thank you all of those that have submitted these documents we value it is contained in them and pursuant to committee rules, members have ten days to submit additional questions for the record to our witnesses. so please respond promptly to
any questions that you receive. at this time the subcommittee is adjourned. god bless all of you. thank you colleagues, you have been spectacular today. it is an honor to serve with you. and all of the collective things from committee members to the staff on both sides of the aisle. you have worked hard. you have put in many, many hours and we appreciate it. thank you. the subcommittee is adjourned. [inaudible conversations]