FDA Reform

The FDA’s warning letter Anne Wojcicki received as the CEO of 23andMe in November 2013 came as a shock to the direct-to-consumer product industry dealing in the “peri-medical” device space. The Food and Drug Administration (FDA) had not previously made such a move, and perhaps 23andMe was being used to set an example, but nonetheless, the action was at the time unprecedented.  23andMe could no longer provide consumers with information about potential disease markers in their genome sequence.

The FDA was signaling it would now regulate the burgeoning field of consumer genomics. As Dr. Margaret Hamburg, the FDA Commissioner who stepped down from her role last week, explained, the FDA has “an enormous set of responsibilities that every day get bigger…”[1]  In fact, FDA-regulated products account for about 25 cents of every dollar spent by American consumers each year.  And the agency is continuing to expand its reach by announcing plans to regulate digital medical devices like apps and wearables, as well as Laboratory Diagnostic Tests (LDTs).

Why does this matter?  Today’s drug and device development process costs too much and takes too long.  It now takes a decade or more and well over a billion dollars for the average drug to make it to market—a dramatic jump compared to $300 million in 1987 and $100 million in 1975 (adjusted for inflation).[2] As a result, venture capital investment in medical technology has declined by 42% between 2007 and 2013.[3]  A 2011 survey by the National Venture Capital Association found that over 60% of their members cite FDA regulatory challenges as reason for shifting away from biotech & medical device investment.[4]

No one wants the FDA to approve drugs or devices that haven’t been properly vetted or could be dangerous to the public.  But we also must make sure we aren’t stifling innovation and deterring investment in the name of safety.  An inefficient, outdated FDA drives up costs for consumers, delays patient access to lifesaving medications and devices, deters private investment in medical innovation, and encourages companies and investors to move overseas.

What can we do to reverse this trend?  Congress must act.  The authorizing statute which established the FDA in 1906 saw its last major update in 1997.  I voted for that legislation as a member of the U.S. Senate; it was sorely needed at the time.  But that was 18 years ago. We have seen technological advances in the fields of health and medicine that don’t fit neatly into 20th century statute.  To bring the FDA into the 21st century, we need to see four things happen.

First, as with most reform discussion in government, we must simplify.  We can do this in part by clarifying the agency’s drug development standards so applicants understand expectations from the outset.  The FDA often issues draft guidance to disseminate information to industry – which often isn’t finalized or withdrawn.  This leaves uncertainty for private actors, who as a result design very broad (and expensive) clinical trials due to a lack of clarity regarding what the FDA will expect of them.  Increasing communication between the FDA and applicants earlier in the process has been shown to be effective.  We may also want to consider putting into statute an updated development process.  While Congress (and even the FDA) lack the scientific expertise to write guidance that reflects cutting-edge scientific methods, this responsibility could be effectively outsourced to groups such as the National Institute of Standards and Technology (NIST), the Institute of Medicine, or the National Institutes of Health.

Second, we can improve accepted clinical trial methodology to reduce one of the greatest drivers of cost in the drug approval process.  The FDA is currently looking into statistical techniques to identify how we make clinical trials more flexible, efficient, and quick while maintaining their safety and reliability. This needs to be heavily supported moving forward. Additionally, the FDA could liberalize its use of the conditional approval process followed by post-market analysis, which would allow consumers to receive needed treatments sooner. There is a particular demand for this with new treatments for terminal diseases. Another option is streamlining the multiple types of “expedited approval” available under the current regulatory scheme. The FDA rarely used its expedited approval powers.  Each pathway takes up time and resources which can be consolidated to avoid bureaucratic redundancy and private sector confusion.

Third, we must also reduce FDA mission creep by clarifying the FDA’s authorizing statue so that its definitions and boundaries reflect modern medicine.  It should not, for example, be delving into LDT regulation, which is already overseen by the Centers for Medicare and Medicaid Services.

Fourth, we need to set a clear goal for cooperation of the FDA with international sister agencies. The Ebola epidemic taught us many lessons but one that was especially embarrassing was our lack of agility regarding emergency approval for new drugs and devices. There needs to be a separate set of regulations regarding international disaster preparedness.

In January, Congress took up the charge of modernizing the FDA.  My fellow Tennessean Senator Lamar Alexander, Chairman of the Senate Health, Education, Labor and Pensions Committee recently released a report with Senator Richard Burr requesting public feedback on best methods for reform, and has announced plans to collaborate with his Democratic counterpart, Ranking Member Patty Murray of Washington. The House Energy & Commerce Committee Chairman Fred Upton has also seriously committed to this effort in the recently released 21st Century Cures discussion draft.  With bipartisan, bicameral support for reform and an Administration looking to cap off its final two years with some legislative achievements, the political climate is ideal for agency modernization.

Perhaps the FDA is getting the message that changes are needed.  The agency is beginning to take advantage of an expedited approval process for breakthrough therapies that Congress passed in 2012.  Breakthrough therapy approvals more than tripled in 2014 compared to 2013.[5]  This track record seems to be continuing into 2015, as the FDA just approved Pfizer’s new drug for terminal breast cancer at record pace.[6]

This a good sign.   However, we cannot let this agency continue to rely on 20th century methodologies when we’re on the cutting edge of new health technologies. New medical devices and sophisticated precision medicine have the potential to revolutionize health care.  We must give the hardworking FDA employees the tools and support they need to succeed in this rapidly evolving field.  The only thing standing in the way is red tape.



Bill Frist is both a former U.S. Senate Majority Leader and nationally recognized heart and lung transplant surgeon.



[2] Roy, Avik, “How the FDA Stifles New Cures, Part I: The Rising Cost of Clinical Trials,” Forbes, 4/24/12.





Morning Consult