Posted 20 January 2017
By Zachary Brennan
Writing in JAMA and FDA’s Voice blog late this week, outgoing US Food and Drug Administration (FDA) commissioner Robert Califf took to task any doubters on the necessity of an independent FDA that can assess an investigational drug’s safety and efficacy.
“Some argue that individuals and their physicians should be free to make these decisions without relying on FDA to make determinations about marketing approval. However, the complexity of the data as well as examples of historical tragedies in therapeutic development caused by erroneous assumptions together make a compelling case that a complete evaluation by unbiased experts is critical to protecting the public from harm,” Califf wrote in a JAMA article published Friday.
“Ever since this foundational protection was established in 1962, a body of evidence has supported the use of a flexible but consistent standard when making decisions about marketing approval: Do the benefits of a product outweigh the risks when used as intended and labeled?” he wrote, noting that a system lacking “this critical check by an independent FDA would unleash harmful products; eschewing premarket evaluation that included both safety and efficacy would be a major mistake.”
Califf also called to harmonize pharmaceutical and medical device approval frameworks.
“While the 2 frameworks for drugs and devices represent critical steps toward creating an agencywide understanding of standards for marketing approval, a harmonized framework could bring greater consistency to these key aspects of benefit-risk determinations,” he wrote.
“Legal standards for reviewing and approving drugs and devices are different, and the results of benefit-risk calculations necessarily reflect these differences. Although a harmonized benefit-risk framework across FDA’s medical product centers would not change statutory standards for approval, it would make explicit a common understanding of principles relevant to benefit-risk assessment and the specific factors undergirding those principles.”
Califf further discussed FDA’s use of independent advisory committees (ACs) composed of external experts, noting one of the “most common concerns raised when I meet with medical leaders” is improving them.
“ACs have been the subject of ongoing discussions concerning their impartiality, their transparency, and how they affect decisions made about FDA-regulated products. In response to these concerns, the FDA is taking a closer look at the AC meeting process to determine what changes may be needed to ensure that ACs remain able to provide crucial expert advice relevant to the uncertainties that prompt such meetings,” he wrote Thursday.