Alexander: FDA and NIH Must be Ready for the Medical Breakthroughs that are Coming

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“Our task is to help ensure that the exciting new technologies being developed and discoveries being made are reaching patients, and that the NIH is equipped to support the early-stage research required to make these advancements and that the FDA is equipped to review them.” –Lamar Alexander 

WASHINGTON, D.C., April 28 – U.S. Senator Lamar Alexander (R-Tenn.) today urged experts from the National Institutes of Health (NIH) and the Food and Drug Administration (FDA) to help Congress in its work to ensure that “the exciting new technologies being developed and discoveries being made are reaching patients-- that the NIH is equipped to support the early-stage research required to make these advancements and that the FDA is equipped to review them.”

“We want to make sure the FDA is ready for the developments coming, such as: bioelectric medicine, where nano-technology sends electric signals to restore nerve function; regenerative adult stem cell therapies derived and put back in the same patient; and therapies based on the whole genome sequence intended to prevent any clinical symptoms from ever occurring. FDA has quite the task before them to keep up with these and many other technologies, and being able to judge that the benefits of the technology outweigh any risk.”

The senator’s full remarks are below:

This is our committee’s third hearing examining how we can get safe drugs, medical devices, and treatments from the discovery process through the regulatory process into medicine cabinets and doctors’ offices for patients who need them. 

Today we have experts from the National Institutes of Health (NIH) and the Food and Drug Administration (FDA) here who can speak to specific challenges to that process, what NIH and FDA are working on to address those challenges, and barriers that remain in their way. 

Each of our witnesses today knows a great deal about innovation. 

Their daily work puts them up close with cutting-edge technologies that are changing the face of modern medicine – from researching spinal stimulation to help paralyzed people regain control of their limbs to approving the next breakthrough medication that could cure cystic fibrosis. 

In many cases, our witnesses have overseen advancements in their fields that have embraced innovation and moved the American medical field forward for patients.

Dr. Austin founded and directed the NIH Chemical Genomics Center while he was at National Human Genome Research Institute to advance the translation of discoveries of the Human Genome Project into insights on diseases and conditions and ultimately treatments.

Dr. Pettigrew established the Quantum Grants at the National Institute of Biomedical Imaging and Bioengineering to achieve "medical moon shots" by supporting high-risk, high-reward projects to address major health care problems, like microchips to capture circulating tumor cells for early detection and to monitor treatment.

Dr. Shuren has overseen a major advancement in heart valve replacement therapy, and the first approval of a next generation gene sequencing platform.

There is not much in drug innovation that Dr. Woodcock has not overseen or been involved in over the last 10 years, including the first personalized medicines. 

I’d like to hear today what FDA and NIH currently are doing and how Congress can help the NIH and FDA keep pace with today’s cutting-edge scientific advancements.

Senator Burr and I released a white paper in January that looked at the process of getting drugs and devices from discovery to medicine cabinet, and much of what the report covered is relevant here today. 

Today, medical products take more time and money to discover, develop, and reach the American patients than ever before. 

We have heard that the FDA has difficulty regulating the most cutting-edge medical products. This disparity between the pace of scientific discovery and FDA’s scientific knowledge is threatening America’s position as a global leader in medical innovation.  

We read in the paper and hear stories about drugs and devices available to patients outside the U.S. first, such as the heart valve mentioned earlier or a drug for multiple sclerosis.

Private investment is shifting away from early-stage drugs and devices in part due to increasing regulatory burden and uncertainty. 

And countries across the globe are seeking to capitalize on America’s shrinking competitive advantage in the biomedical space. 

In response to that report, we’ve gotten a glimpse of some of the exciting new technologies on the way.

We want to make sure the FDA is ready for the developments coming, such as: 

Bioelectric medicine, where nano-technology sends electric signals to restore nerve function;

Regenerative adult stem cell therapies derived and put back in the same patient; and

Therapies based on the whole genome sequence intended to prevent any clinical symptoms from ever occurring. 

FDA has quite the task before them to keep up with these and many other technologies, and being able to judge that the benefits of the technology outweigh any risk. 

At our first hearing, we heard from Dr. Collins, the head of the NIH, and Dr. Hamburg, the former FDA Commissioner. 

Dr. Collins highlighted the need for reforms on the travel of NIH scientists, and the ability to roll funds over from one year to the next.

Dr. Hamburg said that more needs to be done on regulatory science, and that FDA needs to be involved earlier in medical product development to ensure a most efficient process.

We know that opportunities exist today:

The ability to use real world data to improve health – both to shorten the time to get to market, and then also to make sure that medical products are safe once on the market.

The ability to really know about a disease, including the genetic and molecular impact, and target those markers before symptoms are ever even present.

Our task is to help ensure that the exciting new technologies being developed and discoveries being made are reaching patients, and that the NIH is equipped to support the early-stage research required to make these advancements and that the FDA is equipped to handle them.

I look forward to hearing from the panel how Congress can ensure that our biomedical research and review systems are ready for these opportunities and have the expertise and tools to address the challenges.   

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