Alexander: NIH and FDA Must Keep Pace with Today’s Cutting-edge Scientific Advances

“FDA has struggled to regulate the most cutting-edge medical products. The disparity between the pace of scientific discovery and FDA’s scientific knowledge is threatening America’s position as a global leader in medical innovation.”  –Lamar Alexander 

WASHINGTON, D.C., March 24 – At the second hearing on the committee’s bipartisan initiative to examine the process for getting medical products through discovery and development into medicine cabinets, U.S. Sen. Lamar Alexander (R-Tenn.) today told the story of 3-year-old Tennessean whose struggle with cystic fibrosis “illustrates why we’re embarking on this effort and why it’s so important for us to get a result.”

“Last week Ginger Birnbaum from Chattanooga visited my office and told me about her 3-year-old son, King. King has cystic fibrosis, and today there is no medicine to treat his form of the disease. King’s family must simply treat his symptoms. There is good news for some cystic fibrosis patients. There is a drug that can actually treat the underlying cause of cystic fibrosis in just about 9 percent of cases.”

Alexander continued: “This drug, the first personalized drug for cystic fibrosis, was approved in 2012, three months after the developer of the drug submitted to the FDA a new drug application. The bad news is that it took 15 years from discovery to FDA’s door. It also took another 3 years from that approval in 2012 to approve the same drug for children 2-5 years old.

“My question is: what can we do to shorten the discovery and development process so that King doesn’t have to wait another 15-18 years for the next personalized medicine?”

Witnesses at today’s hearing represented much of the biomedical research and development system.

The chairman’s prepared remarks follow:

This is the second hearing on our effort to examine how we get drugs, devices, and treatments from the discovery process through the regulatory process into our medicine cabinets and doctors’ offices.

I’d like to begin today by telling a story that illustrates why we’re embarking on this effort and why it’s so important for us to get a result.

Just last week, Ginger Birnbaum from Chattanooga visited my office and told me about her 3-year-old son, King.

King has cystic fibrosis, and today there is no medicine to treat his form of the disease. King’s family must simply treat his symptoms. His older sister Virginia, who is 6 years old, helps set up his feeding tube at night since the disease doesn’t allow him to digest and absorb the nutrients he needs. She walks with her friends to help raise money to try to fund research.

There is good news for some cystic fibrosis patients. There is a drug that can actually treat the underlying cause of cystic fibrosis in just about 9 percent of cases.

This drug, the first personalized drug for cystic fibrosis, was approved in 2012, three months after the developer of the drug submitted to the FDA a new drug application.

The bad news is that it took 15 years from discovery to FDA’s door. It also took another 3 years from that approval in 2012 to approve the same drug for children 2-5 years old.

The same company is currently studying other therapies for different forms of cystic fibrosis, and if all goes well, King could have a drug that treats his form of cystic fibrosis soon, and I hope in the next 5 years.

My question is: What can we do, here in Congress, to help shorten that process? 

Or, if that drug is not successful, what can we do to shorten the discovery and development process so that King doesn’t have to wait another 15-18 years for the next personalized medicine?

Earlier this month we heard from Dr. Collins, the head of the National Institutes of Health, and Dr. Hamburg, the Food and Drug Administration (FDA) Commissioner.  They provided insights into what NIH and FDA have been doing to try to improve the discovery, research and development, and regulatory processes from the government perspective.

Today, our goal is to hear from the researchers and innovators that interact with the NIH and FDA and can tell us how this is working and about potential solutions.

I’ve found the best ideas often come from outside of Washington.

The witnesses today are from outside Washington and represent much of the biomedical research and development system –

• We’ll hear from the academic who makes the discovery,
• From the venture capital community who funds further development, and
• From a company who takes discoveries through the regulatory process and makes them for patients.
• We will also hear from a group that has been studying how to improve the discovery and development process, from improving clinical trial efficiency to creating a more predictable FDA.

We plan to hear from patients and their families, like King, throughout this process as well.

I’m looking forward to hearing today about how to decrease red tape and administrative burden,

We’ll also hear about some of the exciting new technologies and one of our goals is to make sure that the FDA and others are ready for these technological 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.

We found that medical products take more time and money to discover, develop, and reach American patients than ever before. 

We also found that FDA has struggled to regulate the most cutting-edge medical products. The 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 reported that the venture capital community is shifting investments away from early-stage drugs and devices as a result of increasing regulatory burden and uncertainty. 

Without this venture capital investment, researchers are not able to scale up their research or bring their ideas from the laboratory to the development of medicines or medical devices. 

We also found that countries across the globe have sought to capitalize on America’s shrinking competitive advantage in the biomedical space. 

For example, in China, between 2007 and 2012, funding for biomedical research increased 33 percent, which has translated into rapid growth for its businesses.

These are big challenges that are slowing down the process for getting the cutting edge innovations we are discovering into the medicine cabinet and the doctor’s office.

The NIH and FDA must keep pace with today’s cutting-edge scientific advances.

I look forward to hearing your unique perspectives on these challenges, and others you see as standing in the way of innovation.

I am especially interested in your ideas for how to solve these challenges.

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For access to this release and Chairman Alexander’s other statements, click here.