tv Health Experts Testify on Neurodegenerative Diseases CSPAN August 15, 2021 1:42pm-3:55pm EDT
war -- war correspondent at a time when it was a male dominated profession. >> there was no military censorship per se. the south made -- vietnamese had their censorship, but -- it was for women a gift. because it was only because of this lack of participation and openness that women could get through what had big the biggest barrier is a war correspondent that you were not allowed in the field. >> tonight at 8:00 eastern on c-span's q&a. you can also find all q&a interviews wherever you get your podcasts. >> a look into advances in research for neurological disorders.
we hear from health experts on research and cures and if that -- as they describe for the house committee. >> the subcommittee on health will now come to order. 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 i thank you for doing so. for members and witnesses taking part remotely, microphones will be set on mute to eliminate background noise and you will need to unmute your microphone when you wish to speak. since members are participating from different locations at today's hearing recognition of members will be in the order of
subcommittee seniority. documents for the record should be sent to megan mullen, 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 -- herself 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 constituent, 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. it was a 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 these deadly diseases. according to the national institute of neurological disorders, each year 50 billion -- 50 million americans are affected by neurological disorders such as als, alzheimer's, huntington's disease and parkinson's. these diseases exact an enormous personal goal -- toll and cost
to the u.s. economy as much as $800 billion annually. despite prevalent, deft and -- death 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 all 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 disorders successfully make it from phase one approval. when they are successful, neurological drugs take on average, 50% longer to reach approval van -- van -- than 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 member of our committee has heard from patients, set up with lack of options. two drugs, amx0035 and neuron captured attention and sparked a debate over whether potential benefits 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 fy22 appropriations bill that increases the fda's budget by $2,150,000,000.
but i'm still interested to hear from fda about what more should be done to support their mission mission. we need better multidisciplined coordination 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. this 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.
finally, 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 -- as my constituents stead -- said 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. rep. guthrie: thank you for holding this important hearing about advancing treatment and neural degenerative diseases. i want to recognize and think it's up to the check to testify on her experience huntington's
disease. kayla is a constituent of mine from kentucky while not advocating for patients from huntington's in the family, she can be found facepainting community events. she is 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. we have made progress to create an environment in the united states and encourages innovation for treatments, thanks to representative in this committee, the 21st centurycures robert modernized healthcare innovation infrastructure and included more flexibility to capitalize on this exciting time in science. and enable private sector innovation innovation. this committee has worked on reauthorization institutes of health and ensure the budget is adequate to foster research for treatments and cures.
current subs provided the fda 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 added a 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. in 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 -- development of a 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 by introducing this care act 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 loved ones. 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. -- 60,000 americans each year. 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 survival time is three years and report to 15000 americans with als. we can all agree we want to advanced treatments. let me point out hr three is not , to do our path forward. if this bill becomes law i , believe innovation to treat these diseases will be in jeopardy, quite possibly decimated altogether. we have seen estimates that 15 drugs over ten years, life-saving drugs would not come to market due to hr three because it does incentivizes -- this incentivizes -- this incentivizes -- disincentivizes 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, -- hopeful eye 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 they were meant to live. she dubbed -- >> the gentleman yields 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. >> you have neurodegenerative diseases that continue to affect us in many ways. the treatment of 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 -- to support the national institutes of health brain research through advancing innovative neural technologies or brain initiative. this mission is to revolutionize the understanding of the human brain to discover new ways to treat, cure and prevent brain disorders including neurodegenerative diseases. by accelerating the development of new technologies to map new picture and understand, providing revolutionary foundation for future research and clinical department. this work will be augmented by the advanced research project agency for help.
the fda also 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. they also provide guidance clinical trial design, meaningful and consideration to determine whether treatment is beneficial, market approved. it also works with physicians and patients when treatment options will be unavailable. through 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 for neurodegenerative diseases and how we can further improve access to clinical trial and treatment seekers. it is our responsibility 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 on aging 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 -- programs are affecting research and drug development and what needs to be done. on our second panel will hear from research industry patients caretakers and their expense as our -- are 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 and treated patients with the disease. we will also 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 this hearing and i yield back the balance of my time. >> the chair recognizes representative rogers. recognized for five minutes for opening statements. rep. rodgers: thank you madam chair.
we speak our work before the pandemic. the many meetings with all had, listen to advocates fighting for cures and treatments. hundreds of disease in rare disease groups came to the people's house so the opportunity to share their story story, advocates met my friend, gail 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 diseases, 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
>> she said other countries have earth controls and innovation deserts. innovation deserts are relentless when you need access to a treatment to save your children. our son's name -- her son's is alive today -- his 10th birthday because of a breakthrough treatment. for children like hunter, it would be devastating if price controls were jammed in majority tax and spending. it would have fewer cures and no hope for many people who deserve a fighting chance for life. for that fighting chance, we should be working on bipartisan solutions like hr19. in addition we should be leading the way to fund more basic
research, support research around the causes of diseases, and unleash the private sector like we did with covid-19 vaccines. this is personal for me. my son has down syndrome. it is the most common chromosomal abnormality. there is still a lot we do not know about that 21st chromosome. it's a difficult committee has acknowledged that 100% of people with down syndrome will develop the brain pathology for all in their lifetime. only half experienced symptoms of dementia. the reason is understood. imagine what it would meaningfully unlock the mistress of the 21st chromosome's. -- chromosome. i was surprised to see the biden budget proposed to move the include program from the office of the director to the national institute of child health and
human development without any explanation. what problem does this reorganization solve? it will have the same cross institute coordination. this program has been one of my top roadies. -- priorities. i am concerned that i did not consult with congress. i want to be clear, i have been a champion for nih. i have supported doubling their funding, i co-chair the neuroscience caucus. i promoted the brain initiative from the beginning. nih is on the verge of a crisis with the american people. this is a warning. moving a program like include with no authorization from congress is one thing, one is a lack of respect for congressional oversight on how nih money is received and spent. to inform an investigation into
the origins of covid-19, i have made many requests to nih to be transparent and provide arguments. -- documents. we have received no documents, including grant documents releasable under federal law. it is undetectable. president biden has requested more than $6 billion for arpa h with less transparency we have now -- then we will have now. i need more trust -- going into nih now. including a clear picture of how much of that research is going to china. i will close by thinking the patient's, the families, caregivers, and researchers who are with us today. we share your mission to unleash american innovation, support clinical trials, and improve the
lives of millions of americans. that is why i am passionate about making sure nih research dollars are spent wisely and accountable. we need to unleash the private sector to tackle these diseases with the same sense of urgency we had with covid-19. i yield back. >> the gentlewoman yields back. the chair would like to remind members that pursuant to committee rules all opening statements shall be made part of the record. i would like to introduce our witnesses for our first panel. colleagues, we have a terrific group of witnesses today. i think the minority for your role in bringing people forward as well. first, dr. richard hodes, the director of the national institute on aging.
dr. walter, is the director of the national institute of neurological disorders and stroke at mi h. i hope i have not butchered your name. dr. patrizio is the leader of drug research at the food and drug administration and welcome to you. i think this is a welcome to the committee. this is the first time you're testifying here. the chair recognizes the first dr.. -- doctpr. or. >> thank you for the opportunity to be here. the national institute on age and -- aging cares for those who
are inflicted with alzheimer's disease. is one the most commonly and tragic neurodegenerative diseases. it is better to double by 2050 if nothing changes. -- expected to double by 2050 if nothing changes. we have made enormous progress in understanding this disease. understanding has ranged from the digital trajectory -- traditional trajectory plus new insights into potential targets. from the most basic level, enterprises such as the medicine partnership for alzheimer's are brought together pharmaceutical, corporate, fundamental, basic supported by nih as well as philanthropic and foundational funds. all to generate an acceleration
in a process that has led to the entity because you and of hundreds of new novel targets for alzheimer's disease. changes that happen in the brand -- brain that are targets for intervention. it will move towards the individual medicine which recognizes will be discovered to be the laxity and differences -- complexity and differences from person-to-person. -- nih intern to accelerate the public and private sectors. the transition from basic science to clinical studies. we support for the drug tiles that are targeting a variety of commodities -- comorbidities. these are moving to recognize
the complexity of the disease and understanding it is unlikely one treatment that will address all. we are looking at the research as self, the importance of recruiting a diverse population representative of the u.s. include the most vulnerable. we have come to realize that the neurodegenerative diseases affect a process that goes on for years and decades prior to symptoms. the importance to identify and intervene before massive loss of brain cells and connections. until recently, alzheimer's was diagnosed only at postmortem autopsy. now biomarkers and scans show us the processes that go on in the brain and allow us to make interventions. blood markers which of the promise of being less intrusive and expensive will bring a new ability to recruit people into
studies, taxi disease, and track the outcomes of intervention. -- track the disease, and track the outcomes of intervention. the nih makes it a priority to understand the best ways to support those living with and caring for people with alzheimer's disease. laboratory now identified in infrastructure -- an infrastructure that allows us to conduct pragmatic trials with short turnaround and identify successes that are early to care for people with alzheimer's. including the quality of life program. prevention is also an important way -- the rest of the disease is identified and most recently in terms of preventive interventions we had promising news from a study which showed that intensive approach to
controlling blood pressure has the ability to decrease mild quantitive impairment. a precursor of dementia. we are pursuing interventions that affect diet, exercise, combinations of them, all in an attempt to find ways we can prevent disease. behavior changes in self so we know how to inform people so they can monitor their lifestyles. thank you for the ability to appear here and thank you for the support. [indiscernible] thank you. >> thank you. next, we call on dr. walter. thank you for your work and being here with us today. >> thank you.
i am a neurologist leading the national institute of neurological disorders and stroke. research to understand there are hundreds of disorders and use that to reduce the burden of illness. neurodegenerative disorders cause cells in the brain and spine to die. they are not replaced. the spinal cord, they rob people of the ability to move and communicate. they rob people of years of life. that causes urgency to uncover highly effective treatments. i am hopeful that a host of new discoveries and tools will reach
real breakthroughs. i want to focus on three. genome therapy, as congressman mentioned, we saw an almost miraculous effect of genomic therapies in scilly muscular atrophy -- skeletal muscular atrophy. especially those affected by als. gene treatment in infants have restored function and save lives. this should drive a whole new approach to the inherited neurological diseases. genomic therapy is already underway for huntington's disease and some forms of als. we are on the doorstep of a rush of illusionary -- of a revolutionary therapy. there are tools being developed by the brain initiatives. these genomic tools are now in
the clinic for inherited neurodegenerative disorders. in most disorders, there is a group of patients who have an inherited form due to a known mutation. most persons have what is called a sporadic form which is not inherited. fruitful studies of the inherited disease causing mutations have uncovered pathways that are common to the non-inherited forms as well. therapies are not developed to manipulate these common pathways -- now developed to manipulate these common pathways to prevent death. it has been a common theme in treatment of multiple neurodegenerative disorders. we look under the microscope of -- at the brains of people who have died. we see a clumps of a protein within dying cells. this protein in the brain very
disease to disease -- varies disease to disease. their ability to spread from a sick sell to a healthy one, it may damage one brain region after another. in parkinson's disease, there is evidence that protein aggregation may start in the nerve that supplies the gut to interactions with bacteria in our guts over the course of years. these aggravated proteins spread from the nerves of the gut, to the lower part of the brain, treatment to block the spread can help parkinson's and other neurodegenerative disorders. new biomarkers allow the entity vacation of at risk individuals and enable a rate -- early treatment that blocks the spread before the disease leads to disability. it is exciting examples.
-- they are exciting examples. on the basic science aside, we have learned we can place great emphasis on the nervous system as a whole, and how it integrates with the other body systems. elderly persons with a diagnosis of dementia commonly have signs of alzheimer's disease along with blood vessels caused by years of high blood pressure. at the nih -- the nih showed that aggressive blood pressure control reduces cognitive decline. we can decrease not only heart attack and stroke but cognitive decline and dementia. are public health campaign, my dearest we are targeting black americans in their late 20's to
mid 40's as this group servers from the greatest disparities in brain health. i would emphasize the tremendous scientific challenges that remain as we strive to solve neurodegenerative disorders. also to spread optimism about the new tools to combat these diseases. thank you. >> thank you. that is compelling testimony. we appreciate your work and you being with us today. , we have the next dr. -- next, we have the next dr.. that you recognize is you for the next five minutes to present your testimony to us today.
do we know why he is not on? you need to unmute, doctor. >> my apologies. we were muted. >> welcome to you. >> thank you. here i am, thank you. apologies for technical difficulties. let me start, thank you for the opportunity to testify before you today. i would like to thank my colleagues from nih for their ongoing support and willingness to collaborate with the fda as we translate research into therapies. in recent years, drug development advancements have been lysed -- life changing.
new therapies have been brought to patients faster thanks to the use of expedited regulatory pathways. we only need to look at the pace of element of the covid vaccine to see how quickly scientific research can result in widespread benefit. many diseases which would have resulted in a patient's death can be treated and some cases secured by fda approved therapies -- cured by fda approved therapies. these stand in stark contrast compared to available treatment options. i think you for holding this hearing today -- i thank you for holding this hearing today. we need to adopt additional
innovative approaches for these diseases to bring new drugs to people who desperately need them. there are three primary elements to our approach that i want to highlight today. the need for more research, employing regulatory flux ability, -- flexibility, and support for these people who are afflicted by these conditions. there is tremendous unmet need for safe and effective treatment. there is a need for research with second-rate new therapies. there has been -- create new therapies. there has been research in neurodegenerative, but we have not identified the defects that rise -- great these conditions -- create these conditions. this holds true for als and many other neurodegenerative diseases. there are no easily measured
biomarkers that are reliable pretty errors or surrogates -- markers or surrogates. and direct response could be evaluated leading to more robust and earlier insight to distinguish drugs from those who are politically to succeed. researchers are making advances in understanding the underlying causes of neurodegenerative diseases and this holds promise for drug development. we are using every tool at our disposal to help facilitate the development of treatment for these diseases. we have understood the need to have revelatory flexibility when it comes to medical products for theories diseases. -- for serious diseases. we want to make sure it has a favorable benefit.
flux ability flows in the statute into our regulations and guidance -- flexibility flows into our scituate and revelatory guidance. -- cannot persuade in clinical trials. this is why the agency grants almost all individual patients expended access requests. an essential step in the expended axis process is the company's willingness -- access process is the company's to provide the drug. we do all we can in the situations to help people who need these drugs. the capstone of all of our efforts are the people who need therapies. and their experiences, perspectives, and priorities are a critical aspect of drug development. we focus our development on
what they feel is the most important aspects of their diseases. i look forward to discussing these with you today. we recognize the impact these thesis have on patients and their loved ones. which share the sense of urgency , we stand ready to make full use of our resources to bring new therapies to people with these differences -- diseases as quickly as possible. >> thank you. we are going to move to questions. the chair recognizes herself for five minutes to do so. in your written statement you say a treatment that provides meaningful, incremental benefits would still be desirable. many of the als advocates think
that the statistically significant and come that shout come showed a three point -- outcome that was meaningful. these are individuals that are pulled only have 2-5 years to live. i want to examine, there is a discrepancy between the fda and incremental meaningful benefit being desirable and actually approving a drug that produces that. when you comment on that and tell us -- would you comment on that and tell us how you define meaningful, incremental benefit?
you need to unmute. >> thank you for that question. we do not comment on specific drug programs. some of the information is edited. >> in general, i understand that you cannot comment on specific drugs. i used that as an example. how do you define incremental? >> in general --. >> the context here is incremental in these cases is small. it represents a great deal of hope to people that are living with a death sentence. >> in general, we look at
incremental benefit and incremental being from several points of view. the perspective of the patient is important to us because what we hear from the patient really guides us as what they view as meaningful gains. what we have heard from people suffering from als is that improvement in symptoms and improvement of quality of life is important. in addition, lengthening of survival. we have reflected these perspectives in how we guide developers. running programs for treatments for als. this is reflected in our guidance. the governmental directive for
guest: where we -- the governmental directive for als is showing a gathering of endpoints. >> and need to get another question in -- i need to get another question in. they have a different system, when they approve, when the drug is approved on the condition they will be evaluated further while on the market. we do not do that in the united states. this means that the drug may be available to european patients before american patients. if fda had a similar authority for conditional approval pathways, or that give the fda more flexibility in getting,
saying therapies -- in getting promising therapies to patients that are dying sooner? does the fda seek that kind of legislation in congress? for conditional approval? >> thank you. that is an important question. when it comes to approaches to development of a drug. we think we have a lot of tools at our disposal. the limiting factor in applying the tools we have is the lack of understanding of the biology of the diseases. we are eager to work with sponsors to identify some of the biomarkers and markers of the disease that would allow us to
realize our expedited pathway including accelerated approval in neurodegenerative diseases. >> my time is expired. the chair now recognizes mr. guthrie, a ranking member. -- our ranking member. >> as you know, the nih brain initiative is intended to produce a revolutionary new my dynamic map of the brain that can show ohio individual cells -- how individual cells interact. how would this initiative improve our knowledge of neurodegenerative disease and help researchers find new way to prevent these seizes -- diseases?
>> thank you. there are three things. one is we have the ability to have amazing discoveries to get a understanding of the human brain. that was enabled by technology that allowed us to look at sales and analyze -- cells and have us analyze what are in those individual cells. we can do one million sales in two days. that is amazing. in the brains of people with neurodegenerative diseases, we can tell which cells are missing and the difference between a healthy cell and a six cell as the disease progresses. inside of these cells there are
like genomic keys that opened the door to the cell. but we are trying to do is -- what we are trying to do is find the keys to each cell and link them up to eight genaro mike -- to a genomic line that will give information to the cell that needs it. that targeting will be -- we see with symptoms they have and we look in the brain and we see what kind of pathology we see. we think the pathology causes the symptoms. in between them is the circuits. we had no way of seeing those circuits. now with the brain initiative technology, we can see those circuits in action which is exciting. the circuit becomes the target. there is a study of people who
do not have any dementia, they are perfectly normal but their brains look like they have alzheimer's disease. the pathology is fooling us. the circuits are still healthy. >> thank you. instead of answering that question, a second question you can emphasize. you can go back to that one if you would like. if a person -- a person with dementia faces challenges. they often have -- challenges leading reasons that causes people to place their loved ones in institutional care settings. do you believe that provide individuals living with psychiatric symptoms of dementia
is a brody -- is a priority? can you meet that unmet medical need? >> research is showing ways to maximize the quality of life and care for people living with dementia. it is imperative as we are searching for the care itself. there are over 80 studies that try to understand which are the most effective interventions. some already have reached, one example resulted in a program that has been complicated through the health service -- propagated through the health service. randomized trials, trying to look at the best kind of intervention. we are learning more about this every day.
the ability to translate this to real care is on the horizon. if i could briefly -- >> some breakthroughs you see coming? >> in terms of caregiver support? >> in terms of alzheimer's research, yes. >> in general, a good point to start is described with enthusiasm. it is an incredible ability to look at the levels of individual cells and circuits we never had before. the initial studies, it provides the opportunity for breakthrough for diseases such as alzheimer's to find out what has already learned. when we look at the profiles of silly molecular -- of molecular
biology, they are not all of the same. looking at ways to intervene. >> i have you run over time. i yield back. >> thank you. the general recognizes the next five minutes of questions. >> one of the strategies that has been discussed today is bolstering expanded access, sometimes called compassionate use. and allows patients who -- it allows patients who cannot enroll in a clinical trial to access an unapproved invasion nation will -- unapproved drug. as long as that axis will not interfere with an ongoing
clinical trial. in 2017, former fda commissioner testified before the committee and said that fda approves expanded access requests 99% of the time. is that still the case? if so, why do some patients still have difficulty accessing drugs through the program? and to companies refused to have drugs through extended access? >> i can confirm that we are still approving the overwhelming majority of expanded access individual patient applications. over the past five years we have approved close to 90% -- 98%. there are companies that are not
making the drug, and investigational drug available through expanded access -- an investigational drugs through fitted access. -- expanded access. the patients who are asking for expanded access may be eligible for a clinical trial or they may be concerned about slowing down the recruitment of a clinical trial that could provide important answers on the drug. smaller companies, there may be financial constraints in supporting and expanded access -- an expanded access program. there are issues with the limitations in the drug supply when all of the drug supply has to be devoted to an ongoing clinical trial. for instance, oregon had financial considerations,
particularly with the smaller companies. >> expanded access can improve research into neurodegenerative diseases because more data will be generated from patients on the treatment. let me go to the other doctor. can you speak on nih's view on how expanded access can be useful for scientific research? have what they generate data from an expanded access -- how would they generate data from an extended access trial? >> the finances of expanded access and the scientific value. for nih, in a trial, the value would be in continuing access to patients who were enrolled after they had finished the trial. then you have a comparative
coming can check for durability of results that are found. you can also do a crossover of patients who are on a placebo and get on active treatment. you get information from that. it is hard to get, unless there is a tremendous>>tonight on q&a, elizabeth becker, author of "you do not belong here." the story of female war correspondence when covering war was a male dominated profession. >>it was the first and last uncensored american war. the south vietnamese had their censorship in the boston telegraph. it was for women, a gift. because it was only because of this lack of codification, this openness, that women could get through what had been the biggest barrier as a war correspondent. that you were not allowed on the battlefield. >>elizabeth becker tonight on 8:00 eastern on q&a. you can find all q&a interviews --
>> is it possible to put safeguards in place to ensure that expanded access participations it does not harm clinical trial enrollment? >> i cannot why individual resed exclude a patient from a clinical trial. studies allow pyrex mosher during -- allow expanded access after a period of there may be s where the developers may have concerns that prior exposure during the agent may have on the interpretation of the results. when it comes to what we do at fda, we are sensitive to this. we work with sponsors to attempt
to ensure that that they do not -- that they do not use overly research of criteria to exclude patients who have been on investigational agents. this is part of our broader commitment, making sure that trials are inclusive when it comes to the diversity of patients that are included in clinical trials. the full scope of the many studies, -- >> the gentleman's time is expired. the chair recognizes miss morris rogers. >> the fda has been working on inclusivity of clinical trials. there is still a lot of patience
desperate for axis -- patients four desperate for access to drugs -- who are desperate for access to drugs. can you speak -- including charles intended -- including charles intended to create drug approval -- trials intended to create approval? >> we have been working on presentation in clinical trials. we have recently issued guidance to instruct or help developers on how to expand eligibility to clinical trials and find ways to make sure that underrepresented
populations, beginning with ethnic minorities are included in these clinical trials. we are continuing to work to make sure that the developers are following our guidance. when it comes to the progress we have done so far, we have seen there have been gains when it comes to representation of women, racial, and ethnic minorities in many therapeutic areas. there are certain areas where there is work to do. a trial for alzheimer's disease, we are focused in working with developers to make sure that they deploy all tools that are available to them. or even new tools they will be
able to identify to expand their diversity of the -- >> thank you. want to highlight the issue of individuals with down syndrome. hundred percent of them are building alzheimer's. they are not included the w ay they should. when it comes to research and including them in clinical trials. they develop leukemia at higher rates. let us learn from those with down syndrome. individuals with down syndrome experienced a lifelong chronic auto inflammation. the fda accelerated approval for
therapy, is in i-8 export alternatives -- is in i-8 exploring all -- is nih exploring alternatives for neurodegenerative diseases? >> the diversity of targets involved in clinical trials has expanded. more than 50 clinical trials for drugs, the majority of them are looking at -- among them are the pathways. [indiscernible] they also allude to the inclusion of down syndrome as the population is vulnerable to alzheimer's disease.
they currently -- the currently ongoing study is putting together individuals who are city for the progression of the disease through biomarkers and will provide an important basis for further inclusion in clinical trials. >> are there efforts being funded to investigate processes from birth to life at a to identify other early life events that may predispose individuals with down syndrome to alzheimer's disease? would you discuss further work in this space? >> there is a multifaceted study looking at the goal of inflammation in the nervous system and other autoimmune
disorders, cardiovascular disorders, we have been effective in collaborating with the folks in those efforts. the population is affected, it is very informative. [indiscernible] >> thank you. >> into woman yields back. -- the gentlewoman yields back. the chair acknowledges the man from north carolina. >> thank you. i thank you for convening this meeting -- hearing. thank you for your dedication. this is an issue we should be able to embrace on a bipartisan basis. i want to begin to develop this .
you noted in your testimony that all great progress has been in curing some conditions. the progress has not been even. we will hear from patients and caregivers and researchers who share what the human cost of this failed progress is. the fda recognize the human cost when it released its guidance on drug development. it has denied approval for two als drugs. will act to better understand how the fda is supplying -- applying its data into practice. -- was rejected by the fda even though it showed a 30% slowing in disability and a six-month elongation in survival for a subset of patients.
the basis of the rejection was a perceived need for a confirmatory trial. such a trial would take 3-4 years during which time half of the 20,000 americans currently living with als will leave us. the fda said it understood the appropriateness of exercising regulatory flexibility for serious diseases with unmet medical needs. here is my question, why hasn't fda employed this flexibility for a-list treatments when it has mistreated -- a-list treatments when it --als treatments when it has demonstrated flexibility with other treatments? >> the questions you raise are important and core to how we view our work.
we are operating in a matter that is -- manner that is consistent with our guidance. understanding there has been a lot of hope that has been pinned on certain therapies. unfortunately, they have been disappointed by programs. our guidance and the -- four risk and the top of the hour. they are so rapidly progressing and legal. as we look at how to ride developers in how we interpret the data they put in front of us, we take into consideration the fact that there has to be a higher threshold for risk. in situations where we have some
degree of uncertainty around the benefit. >> let me move on to the next question. my staff had the opportunity to speak with the director of duke university. the als clinic. he has taken over 3000 als patients. most who could not find a place in a clinical trial. is patients are self experiencing -- his patients are self experimenting. the research community suffers because this is not properly studied. everyone would agree that an access program with appropriate oversight would be preferable to select imitation.
-- self experimentation. -- fda devices participation or leveraged research generation from expanded access programs. >> we work actively with sponsors to establish expanded access programs. that have been cases where we have repeatedly asked sponsors to offer drugs under expanded access. the sponsor has not been willing or able to do so. we also see the utility of data that is gathered from expanded access programs. when it comes to rare diseases, we try to accelerate the development by not requiring as large of a safety database we would normally do. we value the expanded access
programs windows those are put in place. -- when those are put in place. >> i yield back. there is much more to go on this, we are on the second round. >> the gentleman yields back. thank you for your questions. the chair is pleased to recognize the gentleman from michigan. you are recognized for five minutes. >> thank you for sharing this portent hearing -- important hearing. paper sheet the testimony by the witnesses -- i appreciate the testimony by the witnesses. when we embarked on 21st century
cures, everyone on the committee supported back in 2016. we work with the fda, we work with the patient groups, we asked a lot of questions. what could we do to advance a cures for these diseases? my neighbor next door died of als, and a lot of friends with parkinson's. we know people who are getting cancer and hopefully cured. that rate has gone up, i recommend the reading of michael milken's piece in the op-ed page in the wall street journal. about where we can go from now. are but h is going to be -- arpa h will get to the president's
desk. where working on a cares act 2.0 bill that will be included. as part of 21st century cures, we asked the fda, what could we do to help you do your job better? had we find the cures -- how do we find the cures? so we cannot have these folks languish and die before their lives and be bettered or find a cure like we did elect sickle-cell -- like we did with sickle cell. you have been -- you have got an important role. we look at the testimony of the next panel that is coming, particularly the als is so frustrated. there is not a cure, can we
extend lives so they can do things that they can do things that are more functional and provide hope that we will have a cure? i think that is the frustration that all of us share with their group. when i look at the testimony from the als association which is coming on the next panel. they ask a number of questions. the fda must be fully funded and staffed to provide regulatory authority. we asked that question, we asked that question to director hamburg and to others. they gave us a dollar figure, and we did it. the question i have for you on this panel is, what can we do now to give those folks who have als the hope that their lives will be frozen or bettered while
they are still here? i am curious to know if this is following up on my good friend's comments about the researcher to consent and other universities as well. what can we do to help you to a better job to provide the hope that these folks want? that is my question. >> thank you. i am sensitive to the -- to how frustrated the als community is and share the sense of urgency to bring therapies to patients with als. when it comes to the tools we have at our disposal, we have the same tools at our disposal when it comes to revelatory flexibility.
it has led to tremendous advances in oncology. we are experiencing some limitations and challenges in the fact that we do not have as good of an understanding of the biology, genetic under pending -- underpinning, of many neurodegenerative diseases. that allowed us to fully deploy the expedited regulatory pathways we have available for accelerated approval such as breakthrough therapy and so on. therapeutic care in oncology has made tremendous gains over the past 20 years. the understanding of the biology improves, we are doing everything we can to work collaboratively and proactively
with als and other neurodegenerative diseases to advance clinical trials and understand the data we obtain from clinical trials. sometimes it is complex and requires working closely with the developers. >> i would like to make a remark. even though you do not have the biomarkers, you know what the outcomes are. i think that is an area we need to hear more about from you. maybe it is not a leapfrogginh advance but it -- leapfrogging advance but it demonstrates something.
i want to get that down for the record. the chair is now pleased to recognize the gentlewoman from california. >> thank you. as everyone can see, by the expressions and the emotions we have around the issue of neurodegenerative diseases, there are reasons why it so many from so many background have suffered in many ways from parkinson's, als, and on and on. this is critical for all of us. want to thank the witnesses for joining us today as we tried to untangle this and find the path forward. i introduced the benefit act, legislation that focuses on
we are very sensitive to drug development of how they are designed. so as an example looking at that guidance we have issued for the drugs around als, we not only provide a lot of information for when it comes to the endpoint that they could use to study drugs for als in the absence of biomarkers like muscle strength or function or even grieving function and so on so they have a host that they could use quickly.
and in addition to that we also emphasize those patient reported outcomes. not only for als, but for other diseases for instance come alzheimer's. and recognizing that caregiver is important and then to encourage the use of items that allow us in this shows to be so this is for full approval? and -- >> it's been allowed for proposal -- approval of drugs before data is developed for clinical trials is relevant
early evidence based on early evidence based on early-stage trial shows that there is a positive therapeutic outcome for the drug. can you explain how this standard is different from what the fda currently uses for full approval? and what are the risks of adopting a provisional or conditional standard? there -- >> there are some similarities and those conditional approval pathways that exist. and that we both recognize that when that pathway is used there is some degree of residual uncertainty around the drug benefit. there is also some notable differences between the two. when we look at the tools that
we currently have at our disposal to have an array of tools with accelerated approval that allow us to make that determination is about benefit risk and decide whether to approve a drug before we have for instance, some have completed trials. there are good examples in oncology where drugs are being approved. >> i think what -- what we talk to people about their particular situation and we are looking at that -- i hope with some focus along with how we might exploit this process more safely and look at some of those patients experiences. with that, i yelled back. >> the gentlewoman yields back.
the gentleman from texas is recognized. >> i think the chair and appreciate the fact that we have a conceit with us. we don't have nearly enough hearings involving agency personnel for whatever reason it is hard to get calls answered from the agency. so i am going to ask you a question and i know your answer will be that is not my department but people just have , to know so why are we still left guessing here?
>> you are on mute. >> my apologies. i did not have much to compare to in this area today. i would be pleased to come back to you and your staff with an answer. >> i would like that very much. it's very frustrating. we come with questions, we call into the cdc for several weeks and we get no response. and were in the middle of this pandemic, your agency and our committee needs to work closely. i'm getting the impression is not happening how it should. haven't got that off my chest, mr. upton's remarks similar to what i was going to bring up -- i was on this committee, we work through the 21st century cures act.
it was a novel approach to that type of legislation. we had -- it was understood that it would probably take more than one congress to work through and develop a bill and understand the processes. we had hearings, briefings, field hearings, i personally attended 15 different field hearings around the country. in the alzheimer's space, as you can imagine it has been intriguing for several years and makes an appearance and then it is withdrawn we go through an advisory committee and it was controversial and now it has received conditional approval. i would appreciate more information on this. it seems like that is exactly what we talked about with skewers. we have an endpoint. which is the development of an order tell and we have a drug to
which to apply it in the whole problem is, how do we get to some answers before everyone expires? it seems to take so long. how do we reduce the time from lab bench to bedside i understand i don't hundred standard billing that was involved with the approval and conditional approval, but it seems like that followed the pathway we outlined to reduce the time from the bench to the bedside. in 20 -- in 2008 it was signed into law with a goal of understanding and identifying als associated risk factors. granted, the registry is administered under the cdc, are you at the fda aware of any efforts from the fda to utilize
the data at the registry question mark -- at the registry ? >> i can't think of any specific instances right now. this is something i would be able to -- happy to get back to you after the hearing. >> i would appreciate that. i am just going to underscore, one of the most substantial victories achieved in my time in congress is the 21st-century cares act that provided so much help to families have been suffering from this life altering illness. i want to point out from the work on the cures there was a stand-alone bill to establish a national neurologic surveillance system prior to us passing the c.a.r.e.s. act there was no official structure in place that
i am grateful that has been established. thank you madam chair i yield , back. >> the chair is pleased to recognize the gentlewoman from florida. >> thank you chair for holding this important hearing and take -- your devotion and thank you to our witnesses today for your important work. i am committed to advancing treatments and cures for neurodegenerative diseases in florida, about 580,000 floridians age 65 and older suffer from alzheimer's the , over 1300 fullerenes are living with als. florida has the highest
percentage in the country and in my neck of the woods we are very fortunate we have a research university that is leading research on neuro- degenerative diseases with the department of neurology. that center does a lot of important research that is family centered, compassionate in partnership with patients and advocates. they do the same with als patients. they have a clinic solely focused on als. patients with families and caregivers there, they believe in the interdisciplinary approach. we have a number of clinical trials with the university, community, industry as you know. we have consistent support from
nah and fda. i want to ask you, doctor, a bit about recent guidance from the fda. guidance to the industry is important to help direct work. it is my understanding the fda has released guidance for industry stakeholders sing to develop treatment for als. that currently has no known cure and few approved treatments. the guidance says fda will consider patient tolerance for risk and the serious and life-threatening nature of the condition in this context of structural tory requirements for safety and efficacy. it also describes consideration for drugmakers that they should make points during drug annulment and encourage the industry to work with fda throughout the process. the fda does not often release disease specific guidance. why did they develop specific
guidance for als? >> thank you for the question. we have released a host -- we do so in the instance of als one we recognize that there might be particular challenges in the development of therapeutics for those diseases. certainly, neurodegenerative diseases are part of those situations. we have worked on the als guidance in collaboration, and listening carefully to the feedback from the community and what we heard from a community and researchers in treating physicians that annulment was
challenging. certainly, when we look at the -- what we lay out in the guidance, we do make the point that we recognize the tolerance for risk when we are developing drugs for diseases like als is greater and we recognize that and now thinking about the rest as we evaluate the data that is provided to us and are yielded by clinical trials. >> the guidance says developers should not unnecessarily exclude patients from trial moments based on characteristics such as age or disease stage unless scientifically justified. and that even if they are testing a subset of patients for a primary analysis of effectiveness, they can include
a broader population in the trial for secondary supportive analysis. how is the industry respond to the guidance question mark -- to the guidance? they have been receptive to the advice. some of the challenges that sponsors have encountered is in the fact that in the limitations in some instances of being able to identify biological markers for some of these populations. we know that als has a largely sporadic -- >> the gentlewoman's time has expired. the chair recognizes the gentleman from virginia. mr. griffith for his five minutes. >> thank you very much.
a friend of mine that has als recently participated in the national institute of health study and was pleased the way it was conducted once it began. recruitment for the study started a year before it began despite it only needing 25 participants. i think we can agree it should take weeks, maybe days to fill only 25 slots. it tell me about your recruitment process. who was involved? how was information disseminated with doctors and patients and advocacy groups so that they know there is a study available and is there inappropriate sense of urgency at the nih? >> there definitely is a sense of urgency. everybody who knows als -- in
general, als trials are actually better than most. the als community is a tight community, we have very good talks with the als association. generally, because the community is looking for answers we have generally not -- she has the platform and maybe they will have more truth on this. my understanding is that als is doing pretty well. if you send me any information about the particular one you mentioned, i would be happy to look into it. >> i would be happy to send you the information. you are saying that als is doing good in getting people into the studies, my friends experienced
was it took him over a year before they got enough participants and they were only looking for 25. that doesn't centigrade doing good enough and we might need to use more. it doesn't sound like we have any ideas. we are here trying to help, if there is something we need to be doing. if you need authorization to advertise it sometimes of you from uncle trials, you need to advertise and we have an approved minotaur that, let us know. we want to help. we want to try to solve this problem. he is not doing very well, not as well as i hoped. the year he waited for this study to get off and running. he was happy once it got started. the new switch gears a bit. when we are doing research on these types of diseases, the neurodegenerative diseases and we are trying to week three people from a wide background which i understand, one of the
things that is probably an impediment is getting people who are not located in the d.c. area up to nih. what are you doing to make sure people that you're having participation from people who live in rural areas? >> that is a good question. the one bright side of covid is that many of the trials had to move towards remote business with the patient's. he talked about als trials but i think they learn they can do things remotely which is a tremendous advance for people who live far away and have trouble coming in to the senses to be enrolled to the trials. at nah we are at the unusual position or we can use nih funds to transport people from anywhere in the country to nih. >> i appreciate that and that was going to be one of my follow-up questions is what can we do to facilitate more
telehealth? if it's a blood sample that needs to be drawn they can be done locally and shipped but i know there are other things. it might have to be an occasional visit. the more we can do with telehealth the better off all our patients will be. i think i speak for both sides of the aisle and this committee, we are anxious for agencies like yours to tell us what you need put into language in the law so we can facilitate you while using more telehealth and making sure we are doing it right? so we can get studies and more participants in these studies. >> one thing that might help is helping physicians in one state to be able to with patients in another state without having to get a license. when i did telehealth i had so many different medical licenses in each state i worked in.
>> we did that in special circumstances in the past, i will look into that. thank you for your testimony, i yield back. none of the gentleman yields back. i would add that the bill we recently approved of with mr. hudson and myself with additional funds for the lodge 12 also. i want to encourage all the witnesses to share with us up front what you need in order to make all of this work better. don't be shy about it, that is what these hearings are -- it is one of the important hearings of -- aspects of the hearing. are the chairs pleased to recognize the gentleman from maryland for five minutes? >> thank you very much, i appreciate the opportunity. we have had others speak to some
degree to this hearing but the importance of inclusion and exclusion criteria that were used by developers in who participates in these various clinical trials. in recent years, there has been a push to diversify to better represent the population but the fda for the public meeting in an clinical trial inclusion. putting these inclusion and exclusion guidance measures together in a comprehensive way
can present unique challenges. researchers have to evaluate populations of -- in different phases of disease progression which is complicated and determine how to maximize -- the fda noted in the report the statistical noise which can mask a finding of the effect for certain populations. on one hand and the desire to generate data that can be applied for patient population on the other. dr., can you tell us why this exists? and what developers in your view should do to bounce this market especially when it comes to neurodegenerative diseases that we are discussing today? >> thank you for the question.
i acknowledge the fact that there is anxiety among developers in some instances when it comes to broadening criteria in clinical trials. be that a system of underrepresented racial and ethnic minorities or inclusion of a subset of the population that are affected with the disease such as we heard earlier with down syndrome and alzheimer's. we can develop those ways to appropriately represent the subgroups that are affected by the disease while also being able to cut -- conduct clinical trials in a timely fashion without those trials slowing down. in addition to the guidance we have issued, when we meet with
developers we talked about tactics such as having in place the appropriate outreach to certain geographic areas or establishing a network of treating physicians we might be able to observe clinical trials participants as well as making sure that we deploy earlier conversations important to such as decentralized clinical trials. we have guidance on these trials during covid and we are working on guidance recognizing that one of the reasons for some subpopulations that have been underrepresented in clinical trial's cannot access clinical trials because they cannot travel there as we have heard.
they live in rural areas and so on. therefore, it is particularly important that we encourage developers to use decentralized -- like telehealth, digital technologies as a way to capture endpoints in a way that does not require people to suffer from neurodegenerative diseases who might be in a wheelchair and dedicated to travel to it the best your side but rather have those features done using telehealth when possible. we think that we can get to a point where we can have greater representation and appropriate representation of subgroups slowing down the threats development and timing of clinical trials. >> thank you very much, i appreciate your testimony. i yield back. >> the gentleman yields back.
scherrer is pleased to recognize the gentleman from florida. bilirakis, for his five and subquestion. >> thank you madam chair. thanks for holding this hearing. things were holding the hearing so we can learn more about the challenges involved with these neurodegenerative diseases such as als, which is a brutal disease that sadly has no known cure or any real treatment. i was particularly saddened to learn this past week that my constituent and good friend, doug mcginnis, a combat veteran diagnosed with als, he was an incredible warrior madam chair. i knew him well. he managed to fight back for many years before the disease
again progressed. unfortunately, since he received experiment with treatments that prompt his lifetime from the u.s. is barred from clinical trials in the u.s. and ultimately could not access the treatments that were effective. both he, his wife, and my good friend have been at the forefront of the critical fight against the disease. i agree we must act and do more. my question is for the doctor, we have heard and will hear concerns from the als community that the rareness of the disease and others like huntington can come kate participation in
clinical trials and access to investigational therapies. in doug's case, these included a dog stem cell treatment that proved effective for him. he had several treatments outside the u.s.. how can we better improve the drug development process side patients with varying stages of neurodegenerative diseases can participate in clinical trials? >> thank you for the question. let me convey how sorry i am about the passing of your constituent after a battle with als. i am very sad to hear that. when it comes to the ability to access drugs -- als is a good
example of a rare disease where we know there are certain forms that are genetic and where we have actually been able to underpin the genetic mutation that then allowed for development of targeted drugs. on the other hand, the 90% or 85 to 90% of als is actually sporadic. meaning we have not identified its specific genetic mutation. that holds some challenges when it comes to deponent. on the other hand, the way we have continued to advance development, even if we in many instances do not have a full understanding of the biology, is to work with developers and the disease community to identify
the modality that can allow patients to be evaluated in clinical trials. for instance, going back to the work we have done with als and the guidance, we have identified a host of clinical endpoints that even without an understanding of the molecular biology of the disease or having biomarkers can allow us to adequately and quickly evaluate drugs and determine whether they can be advanced and whether the benefits are -- and risk profile is positive. these tools for als include endpoints such as function, breathing, muscle strength, and
so on. we really want to make sure that developers can use multiple approaches to evaluating these potential therapeutics. >> madam chair, i have a question with regard to parkinson's disease and alzheimer's and i know we don't have a lot of time. i will submit it for the record. >> think the gentleman for his question. just mature written questions to our witnesses. the chair is pleased to recognize the gentleman from vermont for his five minutes. >> thank you very much madame chair. i want to estimate three areas. in one is migraine and headaches, to is a lack -- als, three is alzheimer's. often times we talk about headaches and it is way worse
than that. 50 million americans suffer from migraines, 46 with my colleagues bipartisan have written in support of the heel act. my question is, can you tell me what the nih is doing to help the millions of americans who are suffering from headache disorders and how can the nih work to engage more researchers, thank you. >> headache is the leading cause of missed work in the u.s., because the a lot of suffering. we find research trying to get at why the headache so care. we have some successes. in my career, there were not very effective drugs and i have japan's and the new drugs.
the industry has been good at picking up on any kind of discoveries that ms -- the neuroscience went to the three people who discovered -- the truth of the matter is we have a shortage of people who concentrate in pain and headaches. it's a difficult field to go into unfortunately, we have a few people that we need. the heel initiative which has a focus on building nonaddictive pain pills, there are a lot of programs out another to research including on headache and to build new research capacity, younger people, headaches, any kind of pain. >> thank you very much. director, could you explain the actions committee has taken to expedite developments in approval programs available to
bring new treatments to als patients? as quickly as possible smart >> thank you for the question. we have been working closely with developers who advise them on how to design clinical trials and including identifying endpoints for clinical trials, how to recruit for clinical trials in a way that allows those trials to deliver answers as quickly as possible. depending on the endpoint. we are very engaged with developers as we always are, particularly when it comes to develop mental air predicts -- where there is a large unmet medical need. we guide them on the details on the clinical trials, the
endpoints they could be using that might yield -- i'm going to give an example of als while we know that clinical programs have been looking at the impact on a potential therapeutic on survival. we have heard from people suffering from als that what matters to them is also improvement in symptoms, improvement in quality of life. 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. i so appreciate the work you are doing in your organizations are doing. i want to end by making a comment. alzheimer's is devastating,
there's a lot of controversy about the approval. the price is unreal, i want to say this. you get a drug and hope it works but is so pricey cannot afford it. and this one is. they set the price at $55,000, there are 6 million medicare double or triple people who suffer from alzheimer's. one third of those -- if one third of people to get drug it would cost 110 billion dollars. that is more than the medicare program spends on all medications for all patients. really appreciate the work you are doing in research, we have to have a reasonable pricing so it is affordable for individuals, taxpayers, and those providing health care. i yelled back. >> the gentleman yields back. i see no republicans available
for questioning or committee democrats. i will recognize the gentlewoman from illinois, miss kelly for five minutes of questions. and then i will make some remarks about impending bets. >> manager -- madame chairman -- >> i think that -- robin, i think we need to go to mr. garnets for five minutes in and let's see if we can squeeze you in also. the gentleman from california is recognized for five minutes. >> sorry about that madame chairwoman. i could see myself on the screen, ab it was not connecting to the committee. >> there you are. >> i appreciate the opportunity, thank you to all the panelists for your expertise and advice. i would like to cover theories
issues when it comes to clinical trials. outside of the exclusion exclusion criteria, a number of external factors exclude individuals from participating will include geographical limitations, financial burdens, transportation difficulties and the ability for caregivers to assist patients in enrolling in a trial in participating. in our second fiddle today, we will hear testimony from a witness who was faced with all these issues. one of these issues describes that she had to help her mother with alzheimer's disease commuting from the bronx to manhattan every week while she maintained a full-time job. to top it off, the clinical trial barely compensated participants enough to cover the cost of a cab from her mother's home to the research site. these issues are systemic and
the federally funded alzheimer's disease research centers tend to be in the wealthiest neighborhoods to add insult to injury for people who would love to be part of these trials but live on the other side of town. it is clear that if we are going to discover new treatments that are available to the widest popular -- possible patient population we need to do more to break down these barriers. what guidance has the fda provided to developers and how they should consider these nonclinical barriers, including financial barriers? >> this is an area where we are putting a lot of fault and we are putting a lot work into this , we recognize that it is critical to increasing the representation of underserved communities and underrepresented racial and ethnic subgroups. during covid, we very quickly
issued guidance on how developers could use decentralized clinical trials approaches to make it easier for patients -- those modalities entail using technologies, telehealth, digital health tools, and also include -- encouraging developers to design clinical trials so that they are as simple as possible and they don't require multiple unnecessary visits. the situation can be very cumbersome. >> thank you, doctor. my time is limited.
thank you so much. the ability of a caregiver to a cyst a patient in accessing a clinical trial can be critical. has nih evaluated the role of caregivers play in facilitating patient access to trials question mark and what can be done to support caregivers so that more patients can participate? it is critical there be revisions made that somebody accompany. >> is a part of the studies in the science. >> we have established a standing 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 applicable -- we have not had alzheimer's disease in all parts of the country where it's most prevalent. recently was funded for pilot centers and other areas in nevada, new mexico, alabama and tennessee. all of these designed to accomplish what you said to put the clinical signs as close as possible and allow for companions when needed and do as much as we can remotely to reduce the burdens of participation in trials. >> thank you, health equity is important to every person on this committee. i am sure it is -- it might not be on the minds of every american, but i think every american agrees everybody deserves not only equal access, but equal care. thank you so much, i look
forward to seeing you in the future. thank you for being here today. my time is expired, i yield back. over the gentleman yields back. it is my understanding that dr. louise -- dr. ruiz is with us. >> thank you for holding this important hearing today. as a physician and a chair of the congressional hispanic kosice -- hispanic caucus, and as someone who practiced medicine in an underserved community i am happy to hear members talk about the importance of diversity. factors like gender, race, ethnicity, age play an important role in how bodies respond to different medications and therapies. what is more, the disproportionate impact of the covid-19 pandemic on communities of colors has highlighted the vital importance of including diverse participation.
addressing the determinants of health and creating greater health equity has long been one of the top priorities of the congressional hispanic caucus. for the past several months, i have been working with fellow hispanic caucus members on important legislation aimed at improving diversity and inclusion in clinical trials supporting patient engagement of communities of color in it decentralized clinical trials in developing solutions to better recruit racially and ethnically diverse populations. nih and fda's engagement will be key in improving outcomes and the participation of communities of color in clinical trials for neurodegenerative diseases and beyond. the first question is to the doctor, how do you determine if the clinical trial is even representative of a u.s. population #and wendy require a sponsor to increase the size of
their trials to accurately understand if there are different outcomes in different payment -- demographic groups? >> we have issued guidance to sponsors to encourage them to make sure that the entry criteria for global trials are as broad as possible. and, to encourage them to ensure that there is representation of that racial ethnic and other subgroups that are relevant and represented in that particular disease. when we talk to sponsors about the design of clinical trials, this is very much at top of mind. we discussed with sponsors modalities and approaches to make sure that the clinical trial population is as close as
possible in representing the scope of groups that suffer from the disease. this is an area that is important to us. we have made gains over the past few years as shown in the clinical trials, that we have accomplished it while ago. and we need to continue to focus. >> thank you, given the nature of neurodegenerative diseases, how can greater use decentralized clinical trials improve the space for all patients and make trial participation more accessible for more diverse communities? >> we think using decentralized trials approaches can be an important tool in facilitating the participation and inclusion of patients, particularly patients in underserved
communities in a neuroscience and neurodegenerative diseases. particularly diseases that are debilitating, like the ones we are discussing that are dependent on caregivers going with the patient to a clinic or clinical site. we think decentralized moralities that use telehealth and semper fi clinical trials and make it easier to collect data from the patient's home or for a lab technician to go to a patient's home to collect a blood test, they are critical in ensuring -- >> thank you. i would like to ask the other doctor what your perspective in terms of the importance of diverse participation for those therapies being developed for neurodegenerative diseases and what you think about improve community outreach to increase diverse participation in this trials. >> not just for all -- we
already know risk factors differ across population. cardiovascular and other contributions -- as far as outreach, the new technologies will help. rather than the current situation will might have to bring people to a site that has access to a cat scan we have the ability to use blood biomarkers that takes one of our restrictions away. we also put in place a system in real-time for tracking each individual accruing through a study for ethnic demographic characteristics. we can track not just in your report but real-time and see if we are on track. >> colleagues, witnesses, we are now going to break because we
have seven bills on the floor that we vote on. we will take a recess, and i think we will come back -- let me make this announcement. we will recess coming back at approximately 3:30, 15 minutes after the last vote is taken. hopefully, boats will end sooner than that. i encourage members and witnesses to return in a timely way. we will take one more member, congresswoman robin kelly, and then we will recess, i think everybody needs a break anyway. the gentleman from illinois has five minutes for her questions, then we will take our break. >> thank you so much. i want to talk about clinical trials and, there is always the
concern about how they reflect diversity of our population. we need to ensure clinical trials are collective of the racial disparities and neurodegenerative diseases. according to the als association, black patients are 2.5 years younger than white patients at the time of symptom onset. they are up to when you're delayed in receiving a diagnosis, one of the many stats that highlight the need for increased diversity in clinical trials. what guidance has nih provided to sponsors to ensure that demographic diversity in clinical trials is reflective of disease disparities, including race, ethnicity, and gender? >> thank you. nih, the trials we run, we are obliged to report what the population is on a yearly basis.
we examine those, to improve the diversity. what's relevant here, there is research to try to understand what is the best way of detecting these conditions in different populations? 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. the gentleman yields back. the chair will now recess the committee and resume at approximately 3:30 p.m.