Study Finds Wide Variation in FDA Drug Approval Process

(Updated January 22, 2014)

Nicholas Downing, an ISPS Policy Fellow and Yale medical student, and Dr. Joseph Ross, an assistant professor at Yale Medical School and ISPS Health affiliate, along with colleagues at the Yale Center for Outcomes Research & Evaluation, have just released a ground-breaking study in JAMA that examines the FDA’s drug approval process.

Using publicly available FDA records to look at clinical trials conducted between 2005 and 2012, the authors found a noticeable lack of uniformity in the evidence used to approve a drug. For instance, over a third of the drugs in the study were approved on the basis of only one trial. They authors note the agency’s flexibility in drug testing: “The variation in the [amount and type] of clinical trial evidence used by the FDA to assess the efficacy of novel therapeutic agents highlights the agency’s flexible standards for approval.” This flexibility allows for a more customized approach to a drug’s approval, including “the ability to rapidly approve potentially effective therapies for life-threatening diseases, such as certain cancers, or those diseases for which there is no existing effective treatment, such as orphan diseases.”

Other authors in the JAMA article were Jenerius A. Aminawung, MD, MPH; Harlan Krumholz, MD, SM; and Nilay D. Shah, PhD.

The paper has generated media attention on  Yale News, Washington Post, Wall Street Journal, Reuters, Modern Healthcare and  USA Today.

 Nicholas Downing was interviewed (Jan. 23)  by Jenna Lee, host of Happening Now (Fox) on the segment “Not All FDA Approvals are Created Equally.”

Area of study