Drugs

4 Ways to Reform FDA Drug Regulatory Reform

David McFarland

— 2 min read
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Former FDA principal deputy commissioner Joshua Sharfstein says there are four things FDA and Congress should do to enhance the benefits and value and reduce the inefficiency and risks of the U.S. system of drug approval. Sharfstein’s ideas appear in a JAMA editorial accompanying a research article discussing the evolution of drug approval in the U.S. over several decades. He says the research “raises a concern over whether … the search for new cures has weakened the evidence base for clinicians without yielding sufficient benefit for patients.”

Sharfstein notes that is not an easy question to answer because not all of the data points are discouraging. “FDA has tended to require fewer studies before approval,” he writes, “but the median number of patients in these studies did not decline. Approvals of new biologics increased from a median of 2.5 from 1990-1999 to 5 from 2000-2012 and to 12 from 2014 to 2018. While overall development times for new products have remained stable at about eight years, those with the breakthrough designation are approved in under five years.”

According to Sharfstein, the picture is not of a struggling FDA but rather of a regulatory process that has evolved over time into a thicket of special programs, flexible review criteria, and generous incentives. He suggests that: 

  •          Congress and FDA rationalize the various programs for expedited review to, at least, promote meaningful competition for non-orphan uses and update requirements for fast track and breakthrough status to increase the chance the treatments will be shown to offer major advances for patients;
  •          FDA should strengthen its oversight of postmarketing safety for medications that offer major benefits but also pose serious risks;
  •          Congress should recalibrate the programs that provide companies with special marketing protections; and
  •          Congress should use patent and pricing incentives to accelerate the generation of definitive evidence under accelerated approval.

“These changes would each reflect an evolution, not a revolution, of FDA’s approach to drug approval,” Sharfstein concludes. “These reforms also could bring greater order and thoughtfulness to the regulation of important new therapies while enhancing safety and creating a greater capability to afford truly transformative medical products. If the point of four decades of FDA reform was to find new cures faster, the point of reforming FDA reform should be to increase the effectiveness and efficiency of this search, propelling the discovery of needed therapies forward while supporting the ability of the healthcare system to bring these advances to the patients who need them.”

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