▶ 0:08:17Chair Cassidy: The senate committee of health, later -- labor, education and pensions will come to order. I was amazed that c-span is not here and could there be a better committee hearing. They were here. And I say that because maybe it is only the eyes of the physician, but I understand the importance of this hearing.
▶ 0:08:42Chair Cassidy: In my career, I graduated meadows -- medical school in 1983. There were diseases at the time that were common, debilitating and/or fatal that are now because of american innovation are like nobody sees it anymore and I can go further back. Because the pediatricians would speak about hospital boards being filled with children of measles of the complications and soon as the vaccine came out within six months whole awards were closed down.
▶ 0:09:13Chair Cassidy: That is the power of innovation. Another example I always give. When I was in med school cutting out a portion of stomach to treat a disease was common. And now, a drug is sold over-the-counter and within six months it was never done. My wife is a general surgery resident and is better about it. And we can go on.
▶ 0:09:38Chair Cassidy: There is now treatment for obesity, malignant melanoma and other conditions before untreatable. Care is for hepatitis c, spinal implants, tests to detect diseases faster and other interventions to improve outcomes.
▶ 0:09:55Chair Cassidy: Our country has led the world in these discoveries accounting for 40% of world biotech related patents for drugs and backed by the gold standard review we produce twice as many novel chemical or biological products as european countries.
▶ 0:10:11Chair Cassidy: The fda has approved over 30 cell and gene therapies including the first gene therapy for sickle cell disease in the hospital in which I managed patients, sickle cell disease was so awful with repeated hospitalizations for patients who were just in pain crisis in pain crisis until they became addicted to drugs and then their whole life spiraled down. Now there is a therapy for it.
▶ 0:10:42Chair Cassidy: It is incredible. We cannot rest on our lawyer looks -- laurels. If we have to maintain that edge there are things that we must do. Outdated framework and regulations push biotech to europe and recently communist china. In varying degrees they viewed china as a collaborator, competitor and as a threat.
▶ 0:11:12Chair Cassidy: When the developers were looking outside of our country for early-stage clinical work we need to pick up our game. It is important for the american biomedical industry and important for patients. We need to give innovators confidence and stability that they can create in the united states without hardship including eliminating unnecessary barriers in the drug approval process while ensuring the gold standard of review is maintained.
▶ 0:11:39Chair Cassidy: That means leveraging ai, machine learning a new technology platforms such as mrna to accelerate drug discovery and clinical development. We have to recognize that although outside the scope of this committee that posed fda, coming to market depends on cms streamlining the coding and reimbursement process so new technologies do not die a slow and regulatory debt -- death postapproval.
▶ 0:12:07Chair Cassidy: That is not in our jurisdiction, but it is critical and must be considered. Finally we must ensure fair competition for american biomedical companies with nations that do not share our values and engage in anticompetitive practices like forced technology transfer and ip threat -- theft. This will advance the goal of making america healthy again while strengthening our dominance in the american economy.
▶ 0:12:36Chair Cassidy: I look forward to discussing these ideas for the hearing and with that I recognize senator sanders.
▶ 0:12:40Sen. Sanders: Thank you very much. I agree with some of what you said. Certainly, I do not agree that president trump is trying to make us healthy again by throwing 50 million people off the health care they had and doubling premiums for 20 million more. That is not making america healthy. Over the last couple of years there have been a number of innovations that ease the pain and we thank all of the scientists and companies responsible for that.
▶ 0:13:12Sen. Sanders: Scientists who work on universities, the nih deserve our gratitude and respect for the work that they have done and discovers -- the new discoveries, treatments and cures. You must continue to support those evidence. There is enormous opportunity to ask -- to extend the life expectancy unpaid.
▶ 0:13:36Sen. Sanders: A life-saving drug no matter life-saving -- no matter how good it will be does no good for the people who cannot afford it. Yet Mr. chairman here is a reality. One out of four americans cannot afford to purchase the medicine prescribed by doctors because they cannot afford them. Think about how crazy that is.
▶ 0:14:03Sen. Sanders: People walk into the doctor's office, they are sick and they get diagnosed and they cannot afford to fill it. What happens? They suffer and end up in the emergency room or hospital at great expense to the health care system. That is crazy. So, in my view, one of the issues that must be looked at today is not only innovation but the cost of medicine in the united states.
▶ 0:14:28Sen. Sanders: And in my view, we can no longer tolerate the american people paying by far, not even close, the highest prices for prescription drugs. Why does merck charge americans and maybe some of our panelists can answer americans -- can help us answer it.
▶ 0:14:49Sen. Sanders: Why does merck charge and's of dollars a year when the same drug can be purchased for $42,000 in japan, pretty simple question. The american people would love to know the answer for that. Why does johnson & johnson charge americans with blood cancer $188,000 when that same exact drug can be purchased for just 82 thousand dollars, less than half in canada and $79,000 in japan.
▶ 0:15:19Sen. Sanders: Why does bristol-myers squibb charge americans with advanced melanoma 204,000 dollars for up tivo -- optivo when it can be purchased for $48,000 in japan? Pretty simple question and maybe our panelists will give us the answer.
▶ 0:15:42Sen. Sanders: According to reuters, the median price of new drugs has more than doubled over the past four years going from 100 $80,000 to more than $370,000 today. And the answer, in my view is -- can be summed up by that famous american expression, follow the money.
▶ 0:16:08Sen. Sanders: Since 1998, pharmaceutical companies have spent more than $6.3 billion on lobbying and we welcome all the lobbyists that are here today, to ensure that their profits come before the health of the american people. This is quite unbelievable. Today we have over 1800 and today we have over 1800 awell paid lobbyists and washington, D.C.
▶ 0:16:36Sen. Sanders: Including former leaders of the republican and democratic parties. That is three lobbyists for every member of congress. Wow. Three lobbyists for every member of congress which is why he probably paid by far the highest prescription -- prices for prescription drugs. During the same period, they spent hundreds of millions of dollars to campaign contributions to both political parties.
▶ 0:17:02Sen. Sanders: While millions of americans suffer or others die because they cannot afford the prescriptions they need 10 top pharmaceutical companies made over 100 billion dollars in profits. It is not just industry profits. Look at what their ceos are making and may be our friends will explain that to thus -- to us. After charging americans with diabetes high prices, the cio made over 113 million dollars in total compensation.
▶ 0:17:31Sen. Sanders: Americans cannot afford prescription drugs and the ceo of eli lilly made $113 million. The ceo of rejugeran -- the ceo of abbey made nearly $80 million in compensation. And here is another reality, since president trump took office in january the price of more than 680 prescription drugs have gone up and etc..
▶ 0:18:03Sen. Sanders: So, Mr. chairman we are in agreement, there is huge potential on the part of our scientists and resource search -- researchers to come up with drugs to deal with diabetes and cancer and that is what we want them to do. Let us also understand that there is maybe with the exception of wall street and I will give you that, may be no industry more greedy or selfish than the pharmaceutical industry and they have to stop their practices which are forcing millions of americans to go without the medicine they need. Thank you very much.
▶ 0:18:34Chair Cassidy: Thank you senator sanders. We are joined by our witnesses today and I will introduce each of them just before their testimony. First is Mr. lowell schiller, a nonresident senior scholar at the usc schaeffer institute for economics.
▶ 0:19:02Chair Cassidy: He is an fda policy expert who prior to his post held senior leadership positions from the fda from 2017 until 2020 he is also a former help committee staff. Welcome home. He served as senior counsel for chairman lamar alexander working on legislation. He has a law degree from harvard and undergraduate from princeton. Thank you, sir.
▶ 0:19:30Mr. Schiller: Thank you chairman, ranking members and members of the committee, thank you for the opportunity to testify on improving patient access while sustaining U.S. leadership. I serve as a nonresident senior scholar at the usc state -- schaefer institute. I held senior roles at the U.S. fda as the chairman noted including principal associate commissioner for policy and acting chief counsel.
▶ 0:19:59Mr. Schiller: We are at a pivotal moment for medical product innovation. Scientific and technological advances are accelerating the development of novel products from targeted therapies and regenerative medicine to increasingly sophisticated diagnostics.
▶ 0:20:17Mr. Schiller: The modern development tools can bring products to market faster and at lower cost while meeting the same regulatory standards for safety, effectiveness and the protection of human research participants to fully realize these opportunities, we must maintain a modern regulatory ecosystem which allows products to read patients efficiency -- efficiently. As technology evolves the fda must evolve with it.
▶ 0:20:42Mr. Schiller: When we does not -- when we do not weep face uncertainty about how products are reviewed meaning late stage project failures and less investment. The fda has made progress including delivering on congressional mandates of the 21st century cares act and other legislation but there is still much to be done. The fda can facilitate more use of advanced evidence generation techniques.
▶ 0:21:10Mr. Schiller: Innovative trial designs and sophisticated uses of real-world data. The pilot programs and guidance documents have laid the groundwork by explaining when and how these techniques can be used appropriately. There is no one silver bullet, these are tools in a toolkit and each has their own uses and challenges. Deployed as appropriate and using scientific best practices they can make our infrastructure more efficient. We are still early in adoption.
▶ 0:21:39Mr. Schiller: The next steps are for fda to expands its programs and refining policies. Issues around data formatting, the review of patient data, or regulations that need updating to accommodate approaches like decentralized and community-based trials.
▶ 0:21:57Mr. Schiller: Second, fda can do more to support the treatment of rare diseases or traditional trials are not possible due to small patient populations, uncertain degree -- disease progression and other factors. The agency can be more systematic and doing more to advance promising tools like using historical or real-world data when it is not feasible or ethical to randomize patients.
▶ 0:22:24Mr. Schiller: They can streamline review and the drugs and biologic centers and project share calm technology or when it can be individualized in the course of care. Third, the fda can modernize its internal processes to facilitate more innovation. The agency's building infrastructure to apply ai and advanced computing with refinement and appropriate guardrails, this offers the opportunity to make reviews more efficient, predictable, and higher quality.
▶ 0:22:52Mr. Schiller: The fda can and -- can update confidence. Which can enable earlier access to 300 dogs -- drugs for serious and life-threatening conditions, half of which is converted into traditional approvals including increasing procedural transparency and leveraging more real-world data which would help address challenges like retaining study participants.
▶ 0:23:20Mr. Schiller: The fda can update regulations to make operating in the united states more globally competitive, particularly in areas with the most off shoring like manufacturing and clinical research. The current regulations were written for technologies that can create uncertainty that make it harder to employ advanced manufacturing techniques and domestic facilities. There are opportunities to streamline requirements for early phase research while maintaining protections for human subjects research can get off the ground faster.
▶ 0:23:52Mr. Schiller: With reforms like these we can ensure that our regulatory system continues to instill confidence in novel products and remains a competitive advantage and ensure that patients benefit from the remarkable innovations. Thank you for the opportunity to testify and I look forward to your questions.
▶ 0:24:09Chair Cassidy: Thank you. Next is Mr. john crowley. He has the president and ceo of the biotechnology innovation organization, the largest advocacy organization representing biotech companies and industry leaders in the world.
▶ 0:24:35Chair Cassidy: He has decades of experience in pharmaceutical tech development beginning in the 90's when two of his children were diagnosed with a rare and fatal genetic disorder. He founded a startup that ultimately developed a life-saving treatment. He holds degrees from georgetown university, the university of notre dame law school and harvard business school. Please proceed.
▶ 0:24:59Mr. Crowley: Sorry. Again, chairman cassidy, ranking member sanders and distinguished vendor -- members, thank you for the opportunity to testify on behalf of the biotechnology innovation organization, we represent more than 1000 biotech companies, academic institutions and research centers across the united states.
▶ 0:25:28Mr. Crowley: As you also indicated, my experience is very personal going back to 1998 when our two youngest children were diagnosed with a fatal and rare -- rare neuromuscular disease. At the time there was no treatment and no hope.
▶ 0:25:45Mr. Crowley: Two years after the diagnosis and nearing what was supposed to be the end of their lives, I quit their jobs -- I quit my job and took a home equity loan and I partnered with a brilliant scientist in oklahoma city and started a small biotech. Within two years we partnered with a larger company based in boston. We successfully developed an enzyme replacement therapy that saved our children's lives and in the lives of thousands of others around the world.
▶ 0:26:17Mr. Crowley: That therapy was born of american innovation. It gave my children a chance to live their lives. My daughter went on to earn a degree from the university of notre dame and works as a social worker with the make-a-wish foundation. Patrick works in a flower shop, staffed entirely by people living with disabilities. Today this ecosystem that shape -- save our children's lives is one of our greatest strengths. Biotechnology is uniquely american-born and american-led.
▶ 0:26:47Mr. Crowley: This committee an entire nation must care about biotechnology for three important reasons. First for the public health, second as an engine of economic growth and third for our national security. America's biotechnology companies have a hundreds of experimental medicines and clinical studies and thousands more in earlier stages of research, each with a promise to extend and enhance human life and to alleviate suffering.
▶ 0:27:17Mr. Crowley: To give those with illness or disease a better quality of life and a chance to have more time, time with people they love. Biotechnology also drives more than $3 trillion in annual economic output. We employ 2.3 million americans and indirectly support more than 8 million others across all 50 states. U.S. biotechnology dominance is national security.
▶ 0:27:45Mr. Crowley: The world, the entire world is a better, safer, healthier and more prosperous pay -- place with the united states together with our allies lead in biotechnology. That american-led ecosystem today is greatly challenged and increasingly uncompetitive. At the same time china is catching up fast launching nearly as many clinical trials as the united states and expanding its share of the global pipeline eightfold.
▶ 0:28:14Mr. Crowley: China's biotech pushes not just economic but strategic and based upon a belief that controlling access to the world's most advanced medicines will provide them an enormous amount of global power and influence. How do we maintain and advance our lead? First we need to strengthen the fda and its role as a global regulatory gold standard.
▶ 0:28:41Mr. Crowley: We should modernize clinical trial processes and make early-stage development more efficient and improve review predictability, expand the use of modern clinical trial designs and biomarkers. We need to strengthen communication with our innovators and developers so that innovation is not delayed. We see encouraging signs from the fda and I have great confidence in our commissioner to boldly and smartly reform the fda.
▶ 0:29:10Mr. Crowley: Second, we must rebuild america's biotechnology manufacturing base. Small and midsize biotech's need help. That means targeted incentives, tax and capital support for contract manufacturers, supply chain investment, workforce development and streamlined infrastructure permitting. Third, we need to reauthorize the rare pediatric risk -- disease priority review program.
▶ 0:29:38Mr. Crowley: This has already helped lead to therapies for 39 pediatric rare diseases and cancers. It costs taxpayers nothing and it has proven to be one of the most effective market-based incentives for innovation. The united states has led the world in biotechnology for decades not by chance but with great purpose. Strong science and smart policy in the partnership between innovators and government has made it possible.
▶ 0:30:07Mr. Crowley: We stand ready to partner with congress to make this happen and continue to maintain and advance our lead as the world's global technology leader. Thank you.
▶ 0:30:17Chair Cassidy: Thank you. Next we are joined by Dr. josh makower. A bio medical engineer and professor at the stanford school of medicine and engineering.
▶ 0:30:40Chair Cassidy: He was the chief architect of the bio design process which has trained hundreds of thousands of students in the -- and faculty into developing manufacture and he has created 10 med tech companies. He holds a medical degree from nyu school of medicine and masters of business administration from columbia and a bachelor's degree in mechanical engineering from m.I.t.. Thank you.
▶ 0:31:04Dr. Makower: Thank you chair, ranking member sanders and members of the committee for this opportunity to testify. I am for over 36 years, have been dedicating my life to -- ok. Thank you. Over 36 years I have devoted my life to developing therapies and technologies to improve patient care.
▶ 0:31:28Dr. Makower: The impact on patients and their loved ones drives my passion to improve our innovation ecosystem and why am honored to be here today. In addition to being a physician, inventor and on up -- entrepreneur I am a cofounder and director of the stanford mussallem center for biodesign. Throughout my career I have had countless interactions with the fda and its teams.
▶ 0:31:54Dr. Makower: The fda like any large organization has had its challenges but its willingness to adapt has contributed to the united states being the global leader in health technology innovation. I know from personal experience that the people at the fda are resolutely focused and ensuring that patients gain access to care is an diagnostics that are ecosystem develops.
▶ 0:32:17Dr. Makower: For decades the united states has dominated global healthcare with cost-effective treatments and diseases improving patient lives everywhere. That leadership is now under threat from nations like china. We must not be complacent and should pursue policies to support this ecosystem. I submit a testimony that detail several suggestions but I do want to highlight a few.
▶ 0:32:43Dr. Makower: Patients are best served when the process is transparent, reasonable and consistent. A majority of biotech innovators indicated that the typical time for a new cell or gene therapy has six to 10 years. And the biggest challenges where issues such as reviewer or key staff turnover and lack of transparency during the approval process. These issues must be addressed. Adequate fda review staff as a priority.
▶ 0:33:13Dr. Makower: I appreciate the efforts by many of this committee to ensure that fda reviewers and inspectors are not impacted. I am concerned that many senior review staff have retired or left the agency. Each center must maintain a stable workforce to step -- to facilitate timely interactions.
▶ 0:33:34Dr. Makower: Industry pays user fees to maintain this workforce, but when the positions are vacated they need to be backfilled and we need to exempt user fees supported visit -- positions from cuts. I know that simply providing more staff does not result in optimization. We are experiencing a way -- a revolution in the way that decision-making is supported.
▶ 0:33:59Dr. Makower: The same holds incredible potential for medicine and surgery. Whether it is ai-assisted ekg interpretation and improving the detection of cancer or powering robotic procedures, it can be leveraged by fda staff to support and accelerate reviews. The issue of efficiency is essential as small companies are the engine of innovation in this country.
▶ 0:34:25Dr. Makower: And their funding is often not capable of braving -- bridging over substantial delays. Innovators should also explore ways to optimize interactions to prevent an adverse impact on patient care. There are growing concerns that the submission program designed to enhance opportunity and engage with teams is being over utilized for simple interactions for which it was not intended.
▶ 0:34:50Dr. Makower: Pre-submissions have tripled to over 4000 in just a few years leading to instances where innovators are waiting to schedule a meeting to discuss minor questions historically handled over a short phone call. This is inefficient for innovators in the fda. True patient access for novel health technology includes affective authorizations, but also a more rapid reimbursement system by private payers.
▶ 0:35:18Dr. Makower: Our research found an average of 5.7 years delay after the fda market authorization before the most nominal level of reimbursement is established. This harms innovators, investors and medicare beneficiaries. I strongly encourage congress to pass a bipartisan ensuring patient access to critical bait -- breakthrough products act and I'm thankful there is broad agreement that this legislation should move forward to narrow the gap.
▶ 0:35:47Dr. Makower: I had my fellow innovators remain committed to work closely to you -- with you to reach our shared goals to save effective health technologies and ensure that the united states remains a global leader in these important fields into the future. Thank you very much and I am happy to answer questions.
▶ 0:36:04Chair Cassidy: I defer to senator sanders to introduce his witnesses.
▶ 0:36:09Sen. Sanders: Are first is andrew -- aaron kessel heim who is a professor of medicine at harvard medical school and brigham and women's hospial -- hospital and he focuses on pharmaceutical policy and pricing. Thank you very much. >> thank you. Chairman, ranking member and members of the committee, my name is erin and I lead the program on regulation therapeutic sandlot harvard medical school.
▶ 0:36:36Sen. Sanders: Thank you for inviting me to speak on how to promote transformative biotechnology and of the U.S.. One of the primary ways the U.S. has ensured a vibrant pipeline of essential products is through high levels of public research funding through the nih and other federal sources. Kate -- take treatments for various forms of cancer arose from scientists at penn and came late via licensing agreements.
▶ 0:37:04Sen. Sanders: We found that one hundred million dollars in grants contributed to the development of the treatment for lymphoma. All fda approved gene therapies to this point can be traced to academic research institutions or their spin ups -- spinoffs that develop key know-how. A work for brightness was based on work from cornell in florida.
▶ 0:37:26Sen. Sanders: The federal government catalyze new sickle cell disease treatments with viral vector technology from the agent -- nih and technology embedded in academic institutions. We found 42% of new recent drugs had late stage reliance on public funding. Despite this track record the current administration has undercut nih funding and targeted universities where these innovations arose. Despite the successes, the health budget includes a 40% cut.
▶ 0:37:56Sen. Sanders: At this model one -- at this .1 model found that a large number of drugs would not be discovered. But the final decision should be up for -- up to you. Congress should establish the normal flow of research funding before the establishment is damage. Another way congress can support is by making sure that innovation is available to patients who need it which include -- which requires reasonable prices.
▶ 0:38:27Sen. Sanders: The ira offered important relief by allowing medicare to directly to negotiate. Just as the government negotiates and sets prices for any other product purchases. It only applied to drugs many years after the fda approved and it has secured in its first year real and useful priced productions that will affect large numbers of people. By contrast, recent moves on drug pricing will not result in nearly as meaningful price reductions.
▶ 0:38:54Sen. Sanders: The administration announced secret deals with drug companies in which they promised without providing details to introduce future drugs at prices comparable to those in other countries. But those unenforceable promises only applied to medicaid which gets major rebates and there are plenty of ways for manufacturers to game the system by launching drugs at different times are different formulations around the world or securing undisclosed rebates.
▶ 0:39:20Sen. Sanders: In these deals the administration also announced a website to help companies facilitate direct could -- direct to consumer sales rebranding but there is always a catch. Patients have to pay for these drugs directly outside of their insurance and most cannot afford brand-name drugs that cost thousands of dollars even at discounted prices. Manufacturers prices tend to increase when these deals are announced showing that investors think that they lack substance.
▶ 0:39:48Sen. Sanders: Because such actions will mostly be useless, congress can take its own steps to ensure that essential biotechnology innovations are accessible. First, congress should fix the biological bonus, and extra period of four years for delayed negotiations that only applies to biologic drugs beyond the standard nine year delay that applies to other drugs. Special treatments in the U.S.
▶ 0:40:12Sen. Sanders: Over small drugs is not justified by any difference in biological development costs or risks. Indeed biologic drugs make more revenue than regular drugs and progress through the approval process faster. Second, congress should revisit the move and the one big beautiful bill act to exclude rare disease drug for negotiation. Why should we be concerned about negotiating prices for rare disease drugs that are making billions of dollars a year?
▶ 0:40:44Sen. Sanders: They concluded this change will cost is $8.8 billion. Congress should pass the no big blockbuster bailouts act which allows for negotiations and annual medicare sales. Congress should take steps to promote timely price lowering competition like passing the ethic act to reduce large patent tickets. In the opposite direction, this patent office is making it harder to get bad patents off the books.
▶ 0:41:14Sen. Sanders: Finally, enjoying the benefits of innovation also requires that patients have adequate insurance products and it is currently poised to allow premiums to sort of unaffordable levels affecting insurance coverage for 50 million people. Congress should stop these changes. Making basic health insurance reasonably available and help ensure that biotechnology products reach the patients who need them. I look forward to the conversation about this.
▶ 0:41:42Sen. Sanders: The second witness is Dr. reshma ramachandran, who is a practicing primary care doctor at a community health center and assistant professor of medicine at the yale school of medicine and is an expert that ensure patients have access to safe and effective health technologies. Thank you for being with us.
▶ 0:42:01Dr. Ramachandran: Chairman cassidy, ranking member sanders and distinguished members of the committee, thank you for the opportunity to testify. I am an assistant professor at the yale school of medicine where I codirected into -- enter petitionary research. I am also a primary care physician, taking care patients at a federally qualified health center.
▶ 0:42:29Dr. Ramachandran: My remarks reflect my own views and not my employers organizations I work with. Based on my exam on how they are regulated and covered I have detailed specific solutions for congress and federal health and agencies including the fda to consider to ensure that patient received timely access to safe and affordable treatments. This future where we can work together to deliver lifesaving cures is being threatened by our present reality.
▶ 0:42:57Dr. Ramachandran: Several of my patient have showed me notices that they received announcing increases in their health insurance premiums. Too many of them including those with multiple chronic conditions requiring regular visits with me and their specialists as well as multiple prescriptions are considering or have decided to forgo health insurance due to these price hikes.
▶ 0:43:19Dr. Ramachandran: These have not just affected my patients who receive coverage through the aca marketplace or the -- but those on the employee -- employer plans. With the continued shutdown of the federal government and the expiration of critical health provisions, medicare patients can no longer receive telehealth services.
▶ 0:43:42Dr. Ramachandran: I see the harmful consequences of all of this in my patients, those who are brushing their care and those who are asking me to provide does prescribe bulk refills in anticipation of losing their coverage. My patientd with chronic and -- patients with chronic conditions who cannot walk or move without great difficulty are asking their family members if they might be able to borrow money to pay out-of-pocket for a telehealth visit out of desperation.
▶ 0:44:10Dr. Ramachandran: In considering the future one thing has become clear, new biotechnology discoveries will be of no use to my patients or any if they are unable to afford them. Under the inflation reduction act medicare was granted the authority to negotiate lower drug prices for older and disabled adults, setting a price benchmark for other health plans. Medicare can consider therapeutic value and how beneficial a treatment is in negotiating a fair price.
▶ 0:44:37Dr. Ramachandran: As the fda leaves unanswered for patients and their doctors whether their treatments worked or were safe, the ability to negotiate could serve as an incentive for manufacturers to generate robust evidence of value and compare its benefits and harms to other available options. This would not only help patients receive a fair price but incentivize better innovation. And when they are proven to be meaningful beneficial -- meaningfully beneficial we will prescribe them.
▶ 0:45:07Dr. Ramachandran: The ongoing crisis has only been worsened. Rather than strengthening the negotiation authorities, congress passed legislation to further exempt or delay negotiation for mower drugs approved for rare diseases. Price negotiations for a drug approved for bone marrow and other rare blood conditions are delayed until 2028. Medicare spent $28,000 on this drug.
▶ 0:45:35Dr. Ramachandran: Patients will have to pay 25 to 33% of the claim cost for 21 hundred dollars per prescription filled. The impacts are not only on patients but also in the health care system. The congressional budget office predicts that these exemptions and delays will cost medicare and taxpayers up to 8.8 billion dollars over the next decade. Proposed fixes in the form of backroom deals between the administration and branding pharmaceutical companies will fail to lower drug prices.
▶ 0:46:07Dr. Ramachandran: Platforms that directly sell select and branded prescription drugs at reduced prices will be no use to patients with limited fixed incomes and those facing a loss of health insurance coverage. Most of my patients who take multiple generic medications these platforms offer no real cost savings.
▶ 0:46:26Dr. Ramachandran: While policies to address ongoing supply and generics are welcome these are predicated by having a well resourced and staffed fda who can address the backlog of inspections that predate covid-19. On shoring of manufacturing facilities will not happen overnight and requires a robust fda to ensure that medicines are being produced and are safe and effective.
▶ 0:46:50Dr. Ramachandran: The recent firing and attrition of more than 20% of the fda workforce including 900 scientists and technical experts and 500 regulators, compliance officers and investigators threatens our ability to continue critical regulatory operations and implement solutions that deliver a sustainable supply of treatments that work and are without harm to patients. Industry reports have noticed delays in the fda meeting regulatory deadlines and a decrease in approvals.
▶ 0:47:21Dr. Ramachandran: Generic approvals that enable choice and affordability are also at a new low as of this year. The future of biotech cannot be centered around technical fixes that prioritize or hasten the market entry and of unclear benefit.
▶ 0:47:36Dr. Ramachandran: I would urge congress to century future around patients, where federal health agencies can assure that federal intervention -- innovation are aligned with those who needed have access to care is -- to cures that work and are safe and affordable.
▶ 0:47:56Chair Cassidy: I will defer to senator tuberville to begin.
▶ 0:48:01Sen. Tuberville: Good morning. Following president trump's domestic manufacturing executive order the fda launched the pre-check program. The program offers more frequent communication and simplifies some manufacturing information required in application. How can the fda use pre-check to include -- to improve inspections?
▶ 0:48:29Mr. Schiller: Thank you for the question. The program has a lot of potential. So do the challenges around investment in domestic manufacturing goes to a degree of uncertainty about how the regulations will apply to those facilities. So having earlier and more frequent interactions with the agency through the process can help. There are other things at the agency can be doing.
▶ 0:49:00Mr. Schiller: Things like updating manufacturing regulations to accommodate more advanced manufacturing techniques. They were written for conventional and older techniques so updating them to take into account use of ai and continuous manufacturing would be incredibly helpful.
▶ 0:49:21Sen. Tuberville: A global leader of genomics and precision medicine is in huntsville. It has generated over 3.5 billion of an economic impact in my state and supports 45 companies in two thousand high paying jobs and attracting 300 million dollars in competitive research funding. It acts as an incubator referred biotechnology research and development that's help resort -- that helps researchers lived -- move from idea to development.
▶ 0:49:54Sen. Tuberville: What are the institutes doing right and how can we encourage more similar efforts?
▶ 0:50:01Mr. Crowley: Thank you for the question and you bring up an important point and this is how medicines are made. If you look last year, three quarters of all medicines approved originated from start up companies. Over time well more than half of these medicines come from small businesses and incubators of research. When we look at what it takes to make newer and better medicines it is an entire ecosystem and a virtuous circle of innovation.
▶ 0:50:31Mr. Crowley: Grounded in our great academic universities and institutions like in alabama, often times funded and partnered with the important research at the national institutes of health, and that foundational research is often times what spurs the start up companies and over and over it is a great technology or a disease.
▶ 0:50:55Mr. Crowley: There are people giving angel money and start up capital and economic development funds in our state such as alabama to support the start up companies. Also having clusters in each of your states where innovators and entrepreneurs can come together where there is lab space provided and in proximity to academic research and often times the founders have joint appointments at local and state universities and the start up
▶ 0:51:25Mr. Crowley: Companies. This is what is so uniquely american. Biotechnology is only a 49-year-old industry. Our first company was founded by a scientist at the university of california, san francisco and a venture capitalist. This happens in every state. It happens in alabama.
▶ 0:51:45Mr. Crowley: As you go along the virtuous circle of innovation, it takes the small companies and contract research organizations and manufacturers, providers of capital and oftentimes wall street driving private capital to these incredibly risky and incredibly inspects dachshunds -- expensive and important gestures. I -- this is the heart and soul of biotechnology and what we need to strengthen and continue.
▶ 0:52:13Sen. Sanders: Thank you --
▶ 0:52:16Sen. Tuberville: Thank you. We talked a lot about united states history. We held that title for decades and we want to keep it for generations to come. Can you explain how america built and sustained this damage at -- dominance and what we can do to sustain it?
▶ 0:52:35Dr. Makower: Most importantly we need to remove unnecessary delays. Efficiency is key, as most of these innovations are coming as john crowley mentioned from small companies, that rely on venture capital and a very short timeline to achieve their goals.
▶ 0:52:59Dr. Makower: By improving the process and shortening it and allowing it to be as rigorous, but removing inefficiencies is one of the primary ways to do it.
▶ 0:53:07Sen. Tuberville: Thank you.
▶ 0:53:08Chair Cassidy: Senator sanders.
▶ 0:53:11Sen. Sanders: A lot of questions and limited time. I would appreciate brief answers, please. One out of four americans cannot afford the prescription drugs that their doctors prescribe. Do any of you have information into how many thousands of americans die because they cannot afford the outrageously high prices of american prescription drugs?
▶ 0:53:40Sen. Sanders: Do you know how many thousands died?
▶ 0:53:42Mr. Schiller: I do not have that statistic.
▶ 0:53:44Mr. Crowley: I do not know.
▶ 0:53:46Dr. Makower: No.
▶ 0:53:52Sen. Sanders: Ok. Let me -- the theory -- let me ask Mr. crowley a question, if I might. Health and human services secretary kennedy says there is "no vaccine that is safe and effective" and that the polio vaccine "killed many many many more people than polio ever did." do you agree with the secretary?
▶ 0:54:23Mr. Crowley: I believe that vaccines are incredibly important. I have in my office as a gift given to me many years ago, a picture of Dr. jonas and I keep that in my office. Vaccines have done as much or more to transform human health than ebony technology we have ever developed. They are safe and effective and they need to be rigorously studied and should continue to be as we did for covid.
▶ 0:54:52Mr. Crowley: We are perfectly aligned and we ran a campaign.
▶ 0:54:54Sen. Sanders: Thank you very much. Do you agree with Mr. crowley?
▶ 0:54:58Mr. Schiller: I do. Vaccines are one of the great success stories.
▶ 0:55:03Dr. Makower: I agree.
▶ 0:55:09Sen. Sanders: I find it strange that we have a situation where over recent 10 year period, 14 major pharmaceutical companies spent $87 billion more on stock buybacks and dividends than they did on research and development.
▶ 0:55:33Sen. Sanders: And ceo's as I mentioned a moment ago, the major drug companies make over $100 million in compensation. What does that tell us a little bit about the goals of -- and motives of the major drug companies?
▶ 0:55:48Dr. Kesselheim: As with all companies they are responsible to their shareholders and primarily responsible to their shareholders. What it also says if we are trying to stimulate innovation it makes most sense and is most efficient to give that money where the innovation comes from, which is at the academic institutions where it is discovered and not to the end product at the end of the stage where the large pharmaceutical companies are spending only 10%
▶ 0:56:18Dr. Kesselheim: To 20%. So for each dollar spent on through the nih, you will incentivize a lot more innovation than for each dollar spent.
▶ 0:56:27Sen. Sanders: So what you are saying is financing research should be the goal, not making stockholder is extremely wealthy or outrageous compensation packages for the ceos?
▶ 0:56:42Dr. Kesselheim: We need to make sure there is a reasonable market and there is an drug companies make money from it. As we think about incentives and encouraging innovation, the most important way to do that is to try and raise the ground and grow the innovation that comes out of where it comes from which is publicly funded research.
▶ 0:57:02Sen. Sanders: You are a practicing physician working with low income and working class people. What is the impact of high income -- high priced drugs.
▶ 0:57:15Dr. Ramachandran: It could be the difference between life and death. Because of the rising cost there are choices that they are making at the pharmacy counter or when they come to the clinic asking me if I could select which drugs among a lot of the drugs they are taking that they could maybe skip that month or not take because of the cost when they go to the pharmacy counter they have to make decisions for paying for the drug or paying for their rent or other needs as a part of their life.
▶ 0:57:43Dr. Ramachandran: It has made a huge difference and often times it has led to people showing up to the hospital because of delayed care and not treating their conditions and sometimes even death.
▶ 0:57:52Sen. Sanders: Thank you very much on the work you do.
▶ 0:57:55Chair Cassidy: Senator moody.
▶ 0:57:58Sen. Moody: Thank you for holding this hearing and for our witnesses for being here. Some of you I have met with personally. This country has blessed the world with numerous treatments and cures for all kinds of diseases. In the 50 years since biotech became a reality. American biotech is strong and growing in florida. I am the newest U.S.
▶ 0:58:19Sen. Moody: Senator from florida and I am told it is home now to nearly 3000 biotech pharmaceutical and medical development enterprises. We are home to 27 bio members. Immune bio is focused on developing two new drugs, one to treat alzheimer's and another to treat a rare skin inflammation disease.
▶ 0:58:43Sen. Moody: Like so many other companies, it shows to plant roots in this country because we have free markets, deep pools of capital and a highly talented workforce. Barda is only becoming more blessed with more talent in this area. So, we have seen progress from start ups having multiple drugs heading into phase three trials and that demonstrates how small, science driven companies can drive progress for patients.
▶ 0:59:12Sen. Moody: In september "the miami times" reported that florida is first in the nation ahead of investment for clinical trials. Biotech is a burgeoning sector of my state's economy and I agree with the chairman that congress should work to unleash the power of american life sciences.
▶ 0:59:34Sen. Moody: We are seeing this presidents and other initiatives that have invested 420 $5 billion in investments for r&d, manufacturing and jobs. I think that is incredible and I expect great things throughout this administration. I will start with you, Mr. crowley.
▶ 0:59:53Sen. Moody: I was touched by your story and it is truly amazing and it is emblematic of what we can do when americans have a personal cause and the resources to do something about it. And the policy that would support successful outcomes and that is why it is so important. I have heard from so many of my constituents that a faster and clear guidance from the fda particularly on rare neurological diseases would be greatly beneficial.
▶ 1:00:23Sen. Moody: They told me that additional support for accelerated approval pathways could contribute to houston -- two huge breakthroughs. There was an announcement of a new approval pathway for drugs which could further empower biotech to bring a greater diversity of cures to patients.
▶ 1:00:44Sen. Moody: How do you believe -- what is your opinion on that announcement and how do you believe that we has congress can make constructive changes to empower biotech and bring more breakthrough therapies to market?
▶ 1:00:57Mr. Crowley: Thank you for your question and everything in the state of florida when our children were originally diagnosed, one of the things we what -- one of the centers we went to was one in florida. You have the right ecosystem and environment.
▶ 1:01:13Sen. Moody: It is the university of florida. Go gators. My school but I am just saying that.
▶ 1:01:19Mr. Crowley: You are right. When we got into that.
▶ 1:01:22Chair Cassidy: The senator is out of order.
▶ 1:01:26Mr. Crowley: I will throw in a go irish. When I came into this world we focus on science, medical -- medicine and technology and now we have never had more technology or opportunity where doctors do not have to tell families what they told us years ago. There are a range of options, most of the challenges are man-made including regulatory. We need to modernize and strengthen. We do not want lower standards.
▶ 1:01:58Mr. Crowley: There are advanced regulatory toolsets, biomarkers and adaptive clinical studies and statistics. What we need is an fda that is modernized and uses the entire toolset and looks at every disease and every proposed molecule through a risk-benefit assessment. Nothing is without risk, but take a look at each of these diseases.
▶ 1:02:20Mr. Crowley: We are seeing very positive moves towards that and other moves that we will continue to see from the commissioner. We have provided ideas and we will continue to work with the fda. It is incredibly important to get as many therapies to everyone in need as quickly as possible.
▶ 1:02:39Sen. Moody: Thank you.
▶ 1:02:40Chair Cassidy: Senator murray.
▶ 1:02:43Sen. Murray: Thank you, as we talk about this really important topic of future biotech it is important to note that since president trump took office and secretary kennedy was confirmed, the trump administration has terminated $2.3 billion in life savings -- saving research from the nih and tried to cut more from the medical research labs by implementing a new indirect
▶ 1:03:14Sen. Murray: Cost policy and proposed gutting the nih budget by 43% which is about $20 billion and laid off thousands of scientists and grant administrators at nhf -- nih and slowed the fda's review of drugs by firing thousands of staff at the agencies and halted more than 100 clinical trials that were working on discovering promising new treatments and cures.
▶ 1:03:41Sen. Murray: It seems to me that this administration has declared war on public health and those examples are just a few of many. I wanted to ask you, does canceling more than 5000 nih grants on everything from cancer to alzheimer's disease increase or decrease american competitiveness and innovation.
▶ 1:04:03Dr. Kesselheim: It substantially decreases because we will lose the competitive battle that we have with academic institutions and research institutions and other countries if we do not continue to have that level of support and more that we need in this country that the support for research at those centers that would ultimately then leads to spinoff companies or other developments.
▶ 1:04:32Dr. Kesselheim: That is where we get the cures from and we really need that in order to stay competitive with the science going on around the world.
▶ 1:04:39Sen. Murray: We should vote -- note that 99% of all new drugs approved by the fda from 2010 through 2019 were built on a foundation of nih funded research. It is clear that our country's historical success that you all talked about relied on a strong pipeline from basic research through pharmaceutical development and are investments that we make.
▶ 1:05:08Sen. Murray: What do the administration's cuts at nih and fda mean for pharmaceutical companies' ability to bring new treatments and medicine to patients?
▶ 1:05:19Dr. Ramachandran: It is a short-term and long-term problem. In the short term you mention clinical trials being canceled and these are often times the late stage development steps for us to be able to see is a therapies safe and effective and that is not happening as often. And there have been delays in terms of awarding grants as well. And then the cut you mention to fda is causing real-time effects with therapies getting reviewed much less approved.
▶ 1:05:50Dr. Ramachandran: So that means therapies that are safe and effective are not reaching patients. Nih is the agency responsible for the foundational research that leads to the start up companies that Mr. crowley was talking about and other senators have mentioned that have come across various states. That is the catalyst for innovation in the first place. If we do not have that investment at nih in five to 10 years down the line we will not see new treatments that are going to be transformative.
▶ 1:06:20Dr. Ramachandran: We heard from the biotech industry that they do not have the resources to make up from shortfalls nor do they have the compulsion to do so because they have shareholders who want short term or terms. There is not a 10 year pipeline, they are looking for returns in one to five years.
▶ 1:06:39Dr. Ramachandran: On top of that the trainees and academic institutions are leaving the united states to go to other countries because they are not seeing opportunities both in the public sector and academic institutions to continue the research much less the private sector with huge cuts there as well.
▶ 1:06:57Sen. Murray: C1 thank you for sharing your family's personal experience. That was I think his story of us many need to remember that these investments pay off and we cannot ignore that. I hope everybody recognizes that.
▶ 1:07:16Sen. Murray: I want to ask you specifically because nih offers -- funds the river sees clinical network in the chaotic policies and firings of more than 1600 scientists and grad administrators has offended many of the clinical trial -- upended many of the clinical trials and researchers are hearing that may not be able to reapply for this funding for-five years from now. Talk about how canceling rare disease clinical trials and suspending funding impacts?
▶ 1:07:50Mr. Crowley: I thicket has a direct effect. The nih plays a role in foundational research and facilitates translational research as well. It remains incredibly important and we support continued funding of the nih.
▶ 1:08:08Sen. Murray: Thank you. I think we all as members of congress need to recognize we have a responsibility to fund those critical agencies and not decimate the future with these layoffs and mass firings and cuts in funding.
▶ 1:08:22Chair Cassidy: I will mention that to the ranking member of the appropriations committee.
▶ 1:08:27Sen. Murray: Thank you.
▶ 1:08:28Chair Cassidy: Senator hawley.
▶ 1:08:30Senator Hawley: Thanks to the witnesses for being here. Dr. ramachandran, I would like to start with you if I can and I want to come back to the price and availability of prescription drugs. I note that you are a researcher, professor, and clinician so you have up close and personal experience with this. Let me look at some statistics that caught my eye. The U.S.
▶ 1:08:59Senator Hawley: Lost 26,000 independent pharmacies. 45 million americans live in pharmacy deserts where there is no local follis -- pharmacy or any pharmacy including in my home state of missouri. We have two counties that have zero pharmacies. Cvs announced plans to close 271 retail stores. Walgreens says it will close 1200 stores over the next two years.
▶ 1:09:29Senator Hawley: We are seeing mass consolidation, three companies I think control 80% of U.S. prescription drugs. What is the effect on patients from this mass consolidation and lack of access?
▶ 1:09:41Dr. Ramachandran: It is devastating and I have personally seen this with patients having to figure out how to drive one hour or two hours to pick up prescription drugs, especially for things like that are emergent, like an asthma inhaler in particular. Sometimes this has landed them in the hospital or even worse. And results and consolidation.
▶ 1:10:12Dr. Ramachandran: And there is not proper oversight. We have a huge problem with our pharmaceutical supply chain in terms of who is controlling access.
▶ 1:10:30Sen. Hawley: Let's stick on pbm's. Is it a good idea to allow them to buy up independent pharmacies and other pieces of the medical supply chain? Should pbm's beaufort -- purchasing pharmacies and doctors practices and merging with hospitals? Is that kind of consolidation across vertical consolidation, across the medical supply chain, is that a good thing for patients?
▶ 1:10:55Dr. Ramachandran: No and we know this from various studies looking at patient outcomes. When that has occurred come outcomes in patients are worse. If appropriations were better in terms of access and quality of care, we would be having a different conversation. What can they do to be able to maximize profits instead of thinking about patient outcomes?
▶ 1:11:21Sen. Hawley: Let me read you from a recent article about private equity. When private equity enter self-care, closures are not side effects, they are the business model. Extracting value comes at the cost of extracting outcomes for patients.
▶ 1:11:42Sen. Hawley: We have a lot of companies and corporations who are rapidly consolidating and they are stripmining the american health care system for their profits, but not for the good of patients. Prescription prices go up, access declines. What are prescriptions you would give us as policymakers to address these problems?
▶ 1:12:03Dr. Ramachandran: I know you and the committee have been talking about reforms to various parts of the supply chain to address this issue around consolidation. Regulation of private equity, having oversight of these deals, which has been happening in some states to some extent, but usually after there is some harm occurring to patients.
▶ 1:12:28Dr. Ramachandran: Being proactive with these mergers and acquisitions and understand what the impact is on patients. And the federal trade commission stepping in so anticompetitive practices are not moving forward.
▶ 1:12:46Sen. Hawley: Free market works best when there is competition, when there is free and open competition. That is what keeps prices down and when you see the kind of consolidation we are seeing in of the pharmacy side or the big pharma or the production of pharmaceuticals, it is really knows rise we are seeing the increase of prices but the decrease in care to consumers. This is why I believe is we must prevent pbm's from buying up the supply chain.
▶ 1:13:16Sen. Hawley: Why should they be able to open up the supply chain? We don't allow it in every other aspect of our supply chain. We should put patients first.
▶ 1:13:33Chair Cassidy: Senator kim.
▶ 1:13:37Senator Kim: Mr. crowley would you say the U.S. is the leading country in the world when it comes to biotech?
▶ 1:13:46Mr. Schiller: Yes, we are the leading company in the world when it comes to biotech but china is a rising, competitive threat.
▶ 1:13:55Senator Kim: You can say definitively right now we are in the lead.
▶ 1:14:00Mr. Crowley: We are.
▶ 1:14:02Sen. Kim: What about 10 or 20 years?
▶ 1:14:04Mr. Crowley: Not at all. If you look at the national security report, they have 49 recommendations to maintain and advance our lead in biotechnology but we need to act boldly and act today. 10 years ago, 2% of the innovative medicines were being developed in china, now it is 25%.
▶ 1:14:32Sen. Kim: Mr. schiller, even within 2-3 years we are going to be losing our competitive edge here. I really think that the key is not just what are the steps to move us forward, what should we do to embrace the urgency of this moment? Are we operating with the level of urgency we should be?
▶ 1:14:58Mr. Schiller: Senator, thank you, I don't think we are. I think there is more we can do to ensure that the regulatory system, the ecosystem allows for faster trials getting off the ground in the united states without that research moving out to other countries.
▶ 1:15:15Sen. Kim: Mr. makower, you talked about reviewers and turnover on that front or just resources. Does the fda have the resources to meet this urgency in terms of our global lead on biotech?
▶ 1:15:44Mr. Makower: Thank you for the question, senator. We can do much better. We should be refilling vacancies 1-1. We need to deploy the latest technologies. Most importantly to be competitive, we need to reinforce in our clinical -- and invest in our clinical infrastructure. It is absolutely important.
▶ 1:16:17Mr. Makower: We need to stand behind our U.S. innovation and fight infringing entities that are utilizing our intellectual property to create their businesses.
▶ 1:16:35Sen. Kim: I think we need to be taking that on. When it comes to the fda, usa need to back build one for one. We are not just trying to tread water here, we are really trying to figure out how we can afford -- I feel like none of you would say we are own the trajectory we need to be, the urgency we need to be on? I would urge this committee to ask, are we on the right path?
▶ 1:17:08Sen. Kim: Backfilling 121 is part of it, but are we moving with the right velocity moving forward? Mr. schiller, what regulatory or statutory changes are necessary to encourage the innovative use of ai?
▶ 1:17:28Mr. Schiller: The most exciting uses of ai in drug development involve reducing the time and expense of drug discovery if we can move from that process into human studies more efficiently and move from computer trials to human trials, that would help substantially.
▶ 1:18:03Mr. Schiller: I think there is much more we can be doing to help with trial recruitment. If more of those tools are leveraged, the more powerful those tools will become.
▶ 1:18:16Sen. Kim: The one word for me is about agency -- urgency. I don't -- I don't just want to think about the urgency for geostrategic leadership or gain, but the urgency of a dad, the urgency of a parent, I hope our nation moves without urgency of a parent trying to save the lives of our children.
▶ 1:18:46Sen. Kim: This sometimes get lost and talk about industries. This should be about the ability to save lives.
▶ 1:18:58Chair Cassidy: I defer to senator baldwin.
▶ 1:19:03Senator Baldwin: Thank you, Mr. chairman. I want to take up what senator kim was saying about the urgency of a child and just note the moment we are in right now in day 29 of the shutdown. I thought that you, Dr.
▶ 1:19:26Senator Baldwin: Ramachandran, helped bring out the numbers of people we are talking about who won't be able to afford their health insurance when they shop on saturday or when the opening period of shopping for the affordable care act marketplace plans, they are going to have a real shock.
▶ 1:19:50Senator Baldwin: And I worry on both sides, I so support the biotechnology innovation that is going on across the country but we have an opportunity to act so that parents can afford to get health care.
▶ 1:20:22Senator Baldwin: I just wanted to emphasize that. To the topic of today's hearing, wisconsin is a leader thanks to investment in strong research institutions, advanced manufacturing, workforce training and industry partnerships.
▶ 1:20:44Senator Baldwin: According to the economic impact report, the industry supports jobs and generates over $37 billion in total annual economic impact. Because of the strong foundation in my state, in 2023, a consortium was selected as a regional tech have focusing on medicine and advancing the adoption of the next generation of theranos sticks.
▶ 1:21:15Senator Baldwin: Mr. crowley, you mentioned that each part of the biotech ecosystem is integrated and complex. I wonder if you can speak about funding programs like wisconsin's biotech hub that deals with all of the ecosystem parts?
▶ 1:21:42Mr. Crowley: It does. Wisconsin is anchored in great economic institutions. When it comes to funding these state-funded programs that often complement private and federal capital are incredibly important. It is a very risky environment when you are starting a company.
▶ 1:22:08Mr. Crowley: So often this state funding can provide the capital where there are failures in the capital market or the technology is simply too early. If you look at wisconsin, some of the therapeutic areas they are developing are areas where they have expertise. Not everybody is trying to do everything. Some are experts in gene therapy.
▶ 1:22:34Mr. Crowley: So yes, these are incredibly important and it is important to recognize the vast majority of capital that goes into development is private capital.
▶ 1:22:47Sen. Baldwin: In april of this year, Mr. crowley, the national security commission on emerging biotechnology released an action plan with a number of recommendations regarding the future of the field and the report aims to ensure the united states remains the global leader. We have seen this playbook before with china.
▶ 1:23:17Sen. Baldwin: They have made this sector a strategic priority and it can achieve global dominance in the space if left unchecked. We cannot let that happen in biotechnology. Which is so critical to our public health and economic strength and national security and for the children they love so much.
▶ 1:23:41Sen. Baldwin: Can you expand upon the dangers of losing our global leadership within the role within biotechnology? What would a world where china leads in biotechnology look like?
▶ 1:23:57Mr. Crowley: I think it would be a different world than the one we have today. We need to thing about where these are made? I don't care where my kids' or grandkids' toys are made eye care where our medicines are made. Where are we making our medicines? How are they distributed? Where is the research and develop coming from. We must win in biotechnology.
▶ 1:24:27Mr. Crowley: We have the lead today. If we don't continue that path, it will be a very different world.
▶ 1:24:36Sen. Baldwin: Thank you.
▶ 1:24:38Chair Cassidy: Great witnesses, great panel, a lot of agreement around this life of innovation beginning with research through the nih and our research institutions onto improvements in the validation and approval process. I'm proud to represent virginia which is a leader in biotech innovation.
▶ 1:25:11Chair Cassidy: I helped found the biotech authority which has helped grown interest in the richmond region. Also 52 million dollars from advanced pharmaceutical manufacturing through the american rescue plan and that
▶ 1:25:43Chair Cassidy: Has really enabled the biotech industry to take off in the last few months. We haven't benefited by announcements from major projects in virginia. Astrazeneca, eli lilly, merck making massive investments including small innovators doing great, celebrating the 20th anniversary of the howard hughes medical campus in loudoun. There is a lot going on in virginia.
▶ 1:26:14Chair Cassidy: I wanted to ask about one part of this innovation chain. Beginning with research through production. Those lawsuits threatened to shut down those efforts, so
▶ 1:26:44Chair Cassidy: Congress created the vaccine program which has been a substitute and created a no-fault mechanism for those who do suffer side effects from vaccines to receive compensation. By my thinking, that has worked pretty well. It has not only worked well to provide prompt recompense, it has done pretty well and enabling us to collect data.
▶ 1:27:18Chair Cassidy: It has also allowed us to look at how any vaccines there are in dpt and covid. I'm very worried that secretary kennedy has flirted with the idea of eliminating this program and returning us to the 1980's when it was a litigation free-for-all against vaccine manufacturers. I'm worried we will see what we saw in the 1980's.
▶ 1:27:47Chair Cassidy: And we would lose the ability to produce these vaccines here at home. I appreciated the acceptance and support for vaccines. How damaging would it be to the production of safe, reliable vaccines on our home shores if we were to eliminate this vaccine compensation program?
▶ 1:28:11Dr. Ramachandran: It would be disastrous, in particular manufacturers are very sensitive to what the government is asking for, what the regulations are. So the market is really dictated in many ways by how government sets policies. The compensation program has provided that security blanket for manufacturers to safely seed.
▶ 1:28:38Dr. Ramachandran: If they anticipate any disruptions to the market either because of removal of the program or changes in coverage as well, they will exit the market. We are seeing right now across different states some efforts of manufacturers to say that we can still have a place to provide vaccines for your constituents. At a federal level, that has a much more broad impact and does not provide any security.
▶ 1:29:05Dr. Ramachandran: That means patients will not have access to the vaccines that they need and we will have too few choices to offer them and the possibility of infections that we thought were gone coming back again potentially.
▶ 1:29:15Sen. Kaine: Are there any other members of the panel who would like to weigh in on the notion of what would happen if we eliminated the vaccine entry compensation? >> I would say would be extremely devastating.
▶ 1:29:29Sen. Kaine: We need confidence in vaccines so a large number of healthy people take vaccines to prevent the diseases from spreading and infecting people who can have problems with their immune systems and can't take vaccines and are at risk for it.
▶ 1:29:48Sen. Kaine: We need there to be confidence in vaccines so that healthy people take them so that there can be vaccines available to people who need them and so we can help prevent diseases that the vaccines of been extremely helpful in preventing.
▶ 1:30:05Sen. Kaine: Any significant disagreement? >> I'm in full agreement, senator. It would be devastating and harm many people. The system has worked well and it will strengthen -- and we should strengthen and preserve it.
▶ 1:30:20Chair Cassidy: Senator hickenlooper.
▶ 1:30:23Senator Hickenlooper: Thank you, Mr. chair. Thank you for being here and for all the work you've done. Mr. makower, you identified several recommendations to continue american leadership and dominance in biotech. Your testimony highlights the need for the fda to leverage a predetermined chain control plan policy that was enacted in congress in 2022.
▶ 1:30:54Senator Hickenlooper: We need to make it easier, not harder for medical devices to grow with the changing landscape of technology. What specifically do you think is preventing this policy from being fully leveraged within the fda?
▶ 1:31:14Mr. Makower: With respect to change control, we are talking about medical devices where they are utilizing ai and other types of machine learning, where there is potential for change for the software subsequent to the initial review, but those things can be anticipated.
▶ 1:31:36Mr. Makower: This is only going to increase as we utilize these technologies and really ai is enabling technology to allow for better access to health care at lower costs for americans. It is important for us to find ways of using predetermined chain control and other ways for allow ai to play a bigger role in health care.
▶ 1:31:58Sen. Hickenlooper: I appreciate that. Dr. kesselheim, you discussed the importance of breakthrough drugs. Being available to patients at fair prices, especially for those drugs which benefit from government investment in their development. The medicare drug pricing negotiation program, which was passed in the inflation reduction act, is an important part of getting treatment to patients that a fair price.
▶ 1:32:29Sen. Hickenlooper: Your testimony noted the negotiations for the discounts at almost 80%. It is a huge drop. A reflection of how much we have been overpaying relative to what the rest of the world is paying.
▶ 1:32:46Sen. Hickenlooper: When we were trying to pass that bill, we talked about developing new drugs, can you explain about how the negotiation is working and the impact it is having or not having on the impact of pharmaceutical companies to research?
▶ 1:33:10Dr. Kesselheim: Sure, the first year achieved meaningful reductions in prices and even those reductions are not as low for those drugs and other countries. There is still room to move in terms of the negotiation and we will see how the second year goes but I would also say that none of those dire warnings have come to fruition yet.
▶ 1:33:36Dr. Kesselheim: I think by virtue of any measure we look at it, of innovation, number of mergers and acquisitions, amount of venture capital investment, even the things the companies are saying, everybody seems to say that there have been no changes in innovation as a result of the inflation reduction act and investments have increased in those first few years afterward.
▶ 1:34:03Sen. Hickenlooper: It is becoming a bipartisan issue, there is a recognition that it is not trump's plan, it is our plan going forward. Mr. crowley, robust funding obviously for the national institutes of health has helped drive innovation in terms of biomedical innovation. The first two shingles vaccines were developed in colorado, which we are very proud of that.
▶ 1:34:31Sen. Hickenlooper: Doctors studying parkinson's where the first to use neuro-pixels, the special implants in the brain. The president's budget request puts all that future at risk of a 40% cut in the research funding. How would you describe the scientific risks of imposing a cut of this magnitude.
▶ 1:35:07Sen. Hickenlooper: How can we maintain our dominance in this area?
▶ 1:35:10Mr. Crowley: We think the nih plays an incredibly foundational role in basic research and development and something we should continue to fund and support, so that is something our industry broadly supports. When we do that at the nih, it encourages the university research, it vote -- it promotes research for start up.
▶ 1:35:34Sen. Hickenlooper: A sequence of miracles. I yield back to the chair.
▶ 1:35:38Chair Cassidy: I will defer to senator blunt rochester.
▶ 1:35:44Senator Blunt Rochester: While I agree this is a vitally important topic, our U.S. biotechnology is critical, even in the state of delaware, this is a big focus for us. Delivering lifesaving cures, that is the bottom line. I'm concerned that this moment is kind of overshadowing this really critical conversation we are having here.
▶ 1:36:12Senator Blunt Rochester: Right now as I sat here I saw that the windowshopping period for healthcare.gov has opened up in families right now are opening this website and seeing sticker shock for so many of them. I missed some of your testimony because I was on the phone with people in my state whether they are from community centers and government officials.
▶ 1:36:42Senator Blunt Rochester: One of our community centers said they ran out of food yesterday and this is before we make choices with what happens with the snap program. Things that make us healthy. Eating good, healthy food. To the witnesses, I thank you for your testimony, I'm looking forward to real, robust conversation on how to make sure our country is strong, both our health, our national security, our agriculture because biotechnology is connected to all of that.
▶ 1:37:17Senator Blunt Rochester: Dr. ramachandran, I want to start with you. With the president just leaving china, I think it is important to note that china is closing the gap. China has their 14th five-year plan which laid out a whole of government blueprint to becoming a global leader in biotech. It is one of their strategic priorities as a country for health care and agriculture. Can you highlight the priorities of that five-year plan?
▶ 1:37:47Dr. Ramachandran: You are absolutely right. China made very strategic targeted investments at every point of drug development, manufacturing in order to enhance the role in our global biomedical system.
▶ 1:38:10Dr. Ramachandran: They wanted to hit of targets and have exceeded them. They have put in several grants for clinical trials to be able to host clinical trials hosted another countries as well and prioritizing domestic pharmaceutical companies and manufacturing.
▶ 1:38:37Dr. Ramachandran: A lot of public investment and manufacturing facilities. At every stage of the pipeline, china put in a lot of investment and we are seeing returns from that. They are now a key player in terms of medicines. Almost a third of key medicines come from china. Several generics have come from china as well. They have positioned themselves to be a key player.
▶ 1:39:09Dr. Ramachandran: In terms of bio technical innovation.
▶ 1:39:11Sen. Blunt Rochester: I have a question for the panel in terms of yes or no? Have you seen in our country a comprehensive plan that is as strategic as this one in biotechnology for this country? Yes or no? >> no, senator. >> no. >> no. >> no. >> no.
▶ 1:39:33Sen. Blunt Rochester: That answers because we don't have one. I think watching and I'm just going to run down the cuts in research right now, the lack of focus on clinical trials and pulling away, getting rid of scientists and cuts to our institutions, our academic institutions, the talent pool, the workforce, and shuttering agencies and getting rid of grant.
▶ 1:40:01Sen. Blunt Rochester: I don't think that is a recipe for us to be a global leader in biotechnology, do you? Anybody? Again, thank you Mr. chairman for this very important hearing and I'm really looking forward to continuing this dialogue because this is vital for our health, our national security, and really for the world. I like what you said, Mr. crowley.
▶ 1:40:28Sen. Blunt Rochester: I don't mind if china makes our toys, I don't like them making our medicines. I yield back.
▶ 1:40:32Chair Cassidy: Senator? >> thank you, Mr. chairman. Totally agree with senator blunt rochester. On that note and thank you Mr. chairman for hosting this hearing. Such an incredibly important topic and I appreciate the feedback we have already received from so many of you today.
▶ 1:40:55Chair Cassidy: Indiana is a thriving hub for biotech already, we have 3200 life sciences companies that directly employ about 70,000 hoosiers in my state. The average salary is about 140 $8,000 and the total payroll is about $7.4 billion for indiana.
▶ 1:41:18Chair Cassidy: Iu, purdue, and notre dame bring in over $1.6 billion in research funding annually and hoosier biotech companies are leading the way in orthopedics, technology, radio pharmaceuticals, regenerative medicine, cancer research, and therapies for metabolic disease. In carmel, just north of indianapolis, mbx biosciences is developing a treatment for a rare hormone disorder.
▶ 1:41:46Chair Cassidy: In west lafayette, on target laboratories created the only fda approved imaging agent that helps surgeons better detect lung and ovarian cancer. And in indianapolis, a company is producing bone marrow for leukemia patients. Those are just a few of the success stories we have in our great state. Mr.
▶ 1:42:10Chair Cassidy: Schiller, how can the fda make it easier to build more biopharmaceutical manufacturing facilities in the U.S. like the ones I just mentioned? I know senator tuberville mentioned the pre-check program, but what else can we do?
▶ 1:42:26Mr. Schiller: I think there are a few things fda can be doing. It can be using the new advanced manufacturing program that congress just passed and enhancing the benefits for participating, so making the designation more of a tool for streamlining. It can also be updating regulations to make it clearer how manufacturing facilities can comply with outdated regulations that were in for older techniques.
▶ 1:42:56Mr. Schiller: There are things congress can be thinking about. Setting up a strategic stockpile of manufacturing capacity. To some amount of agile manufacturing capability that can be used in the case of national emergency or drug shortage.
▶ 1:43:18Sen. Banks: I recently wrote a letter to the fda -- fda urging them to step up the inspections and drug plants in china and india. Inspection of U.S. plants should also be faster than what they are. How can the fda adopt a more risk-based approach to inspections focusing on past problems?
▶ 1:43:42Mr. Crowley: I agree that parity in inspections of domestic and those abroad is incredibly important when you look at the rates when we go to facilities located in some of those jurisdictions. It is much harder for fda to get to those facilities unfortunately and often times there are diplomatic challenges.
▶ 1:44:09Mr. Crowley: Fda has been doing some work trying to increase a number of those inspections and leveraging the inspection tools we used during covid in order to free up some inspection resources, particularly lower risk facilities that have been inspected domestically to free up inspectors going to facilities that have not been inspected more recently.
▶ 1:44:33Sen. Banks: Iu, purdue, and notre dame are leaders for the whole country, but ip theft is a serious problem and we see it in our own backyard. How widespread is ip theft and how are we going to stop it?
▶ 1:44:52Mr. Crowley: Intellectual property is the foundation for a lot of what we do and the ability of us to fund and there is a reason why article one, section 8 clause, eight included intellectual property protections. We are seeing increased espionage and theft. We need to stop that, we need to support law enforcement agencies, the fbi or intelligence community to stop that.
▶ 1:45:18Mr. Crowley: We have enough trouble with the relatively fair competition in the plan for china and we need to strengthen intellectual property protections and that includes not just the threat from outside the united states, it is the threat from within, policies, proposals, proposed legislation that would undermine intellectual property would be cutting the foundation for biotechnology.
▶ 1:45:42Sen. Banks: Thank you, great conversation, I yield back.
▶ 1:45:46Chair Cassidy: Senator hassan.
▶ 1:45:49Senator Hassan: Mr. chair, I appreciate that very much and I appreciate our witnesses. I will associate myself with senator baldwin's and blunt rochester's comments about the moment we are in and the opportunity to take action to make health insurance significantly more affordable for our constituents and make sure they get the care that they need. Dr. ramachandran, drug-resistant superbugs are on the rise across the globe.
▶ 1:46:18Senator Hassan: Each year, tens of thousands of americans die after being infected by superbug bacteria that have outsmarted the antibiotics we have used for decades. To combat these superbugs, it is critical we support the development of antibiotics that are new and different from our existing options. How can congress target our investments to ensure we are investing in antibiotics that will perform better than the status quo?
▶ 1:46:43Dr. Ramachandran: Thank you, senator, for that question and this is very much tied to the current moment we are in right now in terms of where we are supposed to be putting our dollars and our resources to address the global rices of drug resistance that is hitting our shores here. In particular, the bid -- big roadblock we are seeing is actually finding novel compounds, novel treatments to treat drug-resistant infections.
▶ 1:47:11Dr. Ramachandran: Most of the new antibiotics that have come through the pipeline are usually different versions of existing antibiotics which means resistance will happen faster. This is a problem because we are not seeing new options coming onto the market and that is oftentimes because of a lack of drug discovery investment from the federal government and the need not just for nih funding, but cdc funding to identify drug-resistant infections in the first place so we can do the
▶ 1:47:41Dr. Ramachandran: Early-stage research to find new compounds to address the prices -- crisis we are seeing.
▶ 1:47:45Sen. Hassan: Some more investment and more cdc action? Ok. Dr. kesselheim, bio similar drugs are similar drugs that treat autoimmune system drugs, when a bio similar drug enters the market, it is half the price often, but brand-name drug makers often block low-cost alternatives from getting to agents by filing for dozens of frivolous patents and they force
▶ 1:48:16Sen. Hassan: Patients to pay their high prices. What policy changes can congress consider to get patients access to these lower cost bio similar drugs more quickly?
▶ 1:48:27Dr. Kesselheim: As I mentioned in my testimony, one of the things congress can consider is there are bills currently being discussed that would reduce the patents that are growing up around these drugs and allow legitimate innovations to be protected by meaningful intellectual property but not allowing companies to build dozens and hundreds of patents that impair the entry of bio similar products that can lead to competition and lower prices.
▶ 1:48:57Dr. Kesselheim: I would add to that once a bio similar drug is roof, there needs to be efforts to try to ensure patients are prescribed that medication and that when the science supports it and it seems like the science is the orting at that it can be made interchangeable with the original biologic product so it can be automatically substituted at the pharmacy level. Those kinds of steps could help those lower cost, bio similar products make an impact in patients lives.
▶ 1:49:26Sen. Hassan: Thank you. Mr. crowley, advances in genetic screening and the production of synthetic dna has the potential to your deadly diseases, but there are serious risks. These technologies could be misused to produce dangerous pathogens or targeted bio weapons. What actions are your members taking to ensure cutting-edge technologies do not end up in the hands of bad actors or foreign adversaries?
▶ 1:49:57Mr. Crowley: I appreciate and share that concern. A lot of the threat is the unknown. This is part of our trade associations responsibility, to educate. The only difference between doing good and incredibly wrong or evil with biotechnology is intent and purpose.
▶ 1:50:18Mr. Crowley: Much of this lies on the education, the awareness, together with all of the different ways in which our government can protect losing any of this technology to any foreign competitor.
▶ 1:50:34Sen. Hassan: And part of it it seems to me is working collaboratively with all of you to figure out what kind of guidelines and standards you already follow try to prevent the misuse of your technology moving forward and we would love to touch base with you about that and see what kind of standards --
▶ 1:50:57Mr. Crowley: Would be happy to collaborate with you.
▶ 1:51:00Chair Cassidy: I get to ask questions last. It is polite, but it is actually selfish because now I get to go as long as I want. [laughter] Dr. kesselheim, you said investment has not fallen biotech. Did I understand that correctly?
▶ 1:51:19Dr. Kesselheim: In relation to the ira --
▶ 1:51:22Chair Cassidy: Let me ask. You are from biotech. Mr. crowley just responded that much of this off shoring to china has had a very negative impact on biotech investment. Would you include that in your statement that there has not been a decrease?
▶ 1:51:41Dr. Kesselheim: I think it is very important to try to promote biotechnology development and manufacturing in the U.S., the question I answered was related to the ira.
▶ 1:51:54Chair Cassidy: Can you differentiate the ira from the impact of off shoring to china?
▶ 1:51:59Dr. Kesselheim: My response was not related to --
▶ 1:52:02Chair Cassidy: Have you seen decrease in investment and related to off shoring to china?
▶ 1:52:09Dr. Kesselheim: I thing we have seen a movement in off shoring of company investment manufacturing to china and we need to move that back to the U.S.
▶ 1:52:26Chair Cassidy: Mr. crowley, how can you parse the impact of the ira, and the masking totally, parse the difference between off shoring and the ira?
▶ 1:52:37Mr. Crowley: Just to frame it, this has been a tough 4.5 years for emerging biotechnology. We can provide you follow-up stats and statistics about the market value of companies, the number of ipos and programs being cut and there are a lot of factors. With respect to the ira, it is going to be really hard to understand how many programs would have been funded but for the ira, it is hard to prove a negative. I agree with Dr. kesselheim.
▶ 1:53:12Mr. Crowley: In the near term, programs continue to be funded, but it is a long-term we are worried about. We are talking to any private market investor, large companies looking at their portfolio is the shift away from small molecules were away from small molecules to large molecules.
▶ 1:53:31Chair Cassidy: Has there been a shift in on investment?
▶ 1:53:34Mr. Crowley: A disparity.
▶ 1:53:37Chair Cassidy: Castle heim -- Dr. kesselheim says he sees it moving.
▶ 1:53:49Mr. Crowley: I think it is more toward large molecules, particularly when small molecules are so important, they cross the blood rain barrier. -- blood-brain barrier.
▶ 1:54:00Chair Cassidy: I'm going to pause you because I have a lot of questions. Mr. schiller, you talk about domestic manufacturing. There was a generic drug plant in shreveport that was recently closed and they left all their equipment and the labor cost and whatever in the united states, it was cheaper to make it there and ship it.
▶ 1:54:23Chair Cassidy: They said you can do robotics and you mentioned this, but the upfront cost to invest in robotics is so much that still you are better off going to india. That is what I was told. Is that true? You are advocating a re-shoring of manufacturing, but will we always be at a competitive -- competitive disadvantage?
▶ 1:54:45Mr. Schiller: I do believe that is a problem. That the margins on many generic products have gotten so low that it is not profitable to manufacture many of them in the united states and if you have some amount of upfront investment in advanced manufacturing facilities where the unit cost does go down, that can help, but finding the capital to invest upfront.
▶ 1:55:12Chair Cassidy: Even if you found the capital to invest, would it level out over 20 years? Has anybody put finger to keyboard to see if that works out?
▶ 1:55:26Mr. Schiller: Part of the problem is the capital isn't sitting in an account upfront to invest.
▶ 1:55:32Chair Cassidy: Usually when you told me about this post fda marketing being a problem, I have talked to cms regarding another aspect of this and they told me the fda and I think you said nine-seven years between fda approval and getting insurance coverage? >> five .7 years.
▶ 1:55:54Chair Cassidy: That fda uses a safe and effective standard and cms uses a reasonable and necessary standard. They kind of overlap, if you will. Is that with the holdup is?
▶ 1:56:10Mr. Makower: That is part of it. Reasonable and necessary has not really been defined. But a lot of fda studies, safety and efficacy as agreed upon and negotiated in whatever trial or design and the study is either map or not. It is unclear how reasonable and necessary is fundamentally determined.
▶ 1:56:34Mr. Makower: The big challenge is that we have a system where these innovations can be created, proven to be safe and effective by the fda and there are substantial time delays even in accomplishing coding, going through the coverage decisions. It is just an extremely byzantine and very long process and during that time, investors are just funding these companies.
▶ 1:56:59Chair Cassidy: Is that something that could be reasonably addressed by ai?
▶ 1:57:04Mr. Makower: It could be exceptionally helpful. There needs to be process reform for sure.
▶ 1:57:09Chair Cassidy: But that is again cms. Mr. schiller, I want you to comment on what Mr. makower said, but I will let anybody take a crack. Under the biden administration, there was an increased emphasis upon third-party review, but you say that the fda will do the re -review. You strongly imply it. But it defeats the purpose.
▶ 1:57:36Chair Cassidy: Why the heck with a re-review if you just at a third-party review? >> thank you for the question. That has been an issue and there has been under utilization of the third-party pathway because the benefit is not clear and part of that is variable quality within third-party reviews.
▶ 1:58:02Chair Cassidy: The third parties have not been able to do it consistently, when there is inconsistency, it reduces the incentive and you can expect an easier process with the fda.
▶ 1:58:18Chair Cassidy: If the third-party review was inconsistent, there should be some sort of quality control and you get rid of the people who are lousy and get rid of the people who are good. -- and keep the people who are good.
▶ 1:58:32Dr. Ramachandran: Only about 2% of devices in recent years have actually undergone third-party reviews. A government audit was done to show discrepancies in terms of vendor quality which forced fda to step into do a review. There are things to address. Recommendations were put forward on how that might be used. A key aspect is that the third-party review is funded from leftover funding.
▶ 1:59:02Dr. Ramachandran: It is kind of whatever is left goes then to the third-party review to identify vendors to review that process in the first place, which is not a really efficient use of the funding quite frankly and it is compounded by a larger issue in terms of the quality of evidence submitted by medical device manufacturers. We did a big analysis of all breakthrough devices approved by the fda as of 2023 and we found that one in 10 medical breakthrough devices don't have any clinical testing to support them.
▶ 1:59:32Dr. Ramachandran: So no verification about safety and efficacy before fda market authorization and most of those did not have post-market requirements to make sure we actually could confirm safety and efficacy. For medicare in particular, reasonable and necessary also means reasonable and necessary for their beneficiaries, older adults. If you look at the authorization, older adults are often not represented in those trials. We are not even testing it in the population medicare covers.
▶ 2:00:02Dr. Ramachandran: We should also introduce complexities in terms of medicare coverage.
▶ 2:00:05Chair Cassidy: I was trained as a gastroenterologist. First, it seems like it has to be proven for people over 65, if you are looking about whether something detects polyps in cancer, if it detects a polyp and a cancer and someone who is 55, it is going to detected and someone who is 65.
▶ 2:00:36Chair Cassidy: There is nothing fundamentally different between a cancer shedding whatever it sheds in someone of a different age.
▶ 2:00:42Dr. Ramachandran: That's correct and I can grant you that and this is why we have fda authorization, but that is predicated on the fact that fda has the evidence and clinical testing that shows that effect.
▶ 2:00:55Chair Cassidy: Dr. makower, your testimony was calling for reform but that there is not adequate premarket or post-market. Any other comments?
▶ 2:01:07Dr. Makower: I think that the legislation in front of congress actually calls for accelerating coverage and concha meant studies that allow for these questions to be answered in that timeframe.
▶ 2:01:24Dr. Makower: I agree that very often there is no difference between the study population that was necessary for premarket authorization and what the results would be in an older population but it is reasonable to look at that and that is called for in the legislation.
▶ 2:01:41Chair Cassidy: I could keep going, but they have called votes. I really appreciate you while being here. We will submit some questions for the record. Senator markwayne mullin has a question for you. You have been very helpful though and I look forward to if any of you have continued input, please feel free to continue.
▶ 2:02:03Chair Cassidy: For any senator wishing to ask additional questions, questions will be due at 5:00 p.m., thursday, november 15. Thank you again for being here. [captioning performed by the national captioning institute, which is responsible for its caption content and accuracy. Visit ncicap.org]