The FDA is often under pressure to approve new drugs quickly, as patients are eager for access to treatments that may dramatically improve outcomes and manufacturers are eager to start raking in those obscene profits.  The FDA’s primary charge is to ensure that drugs on the US market are safe, which can take long and complex trials.  There is obviously tension here.  Research in the Journal of the American Medical Association should make us question whether safety is being evaluated thoroughly before approval.   (JAMA Article)  The researchers examined novel drug approvals from 2001 to 2010.  There were 222 of these, 39 biologics and 183 traditional drugs. Many had accelerated or special review pathways.  71 or 32% of these novel drugs had a postmarket safety issue.  There were a total of 123 of these issues, so some drugs had more than one.  3 of the drugs had to be withdrawn from the market.  61 of the safety events resulted in boxed warnings, which identify major concerns, often life-threatening.  The median time from approval to first safety event for an affected drug was about 4 years.  The most common category with safety issues was mental health compounds and the least common were cancer and hematologic disease.  Biologics were more likely to have safety events than traditional drugs.  Priority review and orphan status were not associated with more safety problems, but accelerated review was as was approval near a regulatory deadline.

One reason safety issues are not always identified in clinical trials is that manufacturers have gotten very clever about limiting who the drug gets tested on to a population most likely to respond and least likely to have safety issues.  That would be fine if the population to whom the drug could be prescribed was similarly limited, but it usually isn’t.  The FDA may have to be more careful in examining clinical trial applicability to the real world of medicine.  And the study certainly supports mandatory post-approval safety monitoring, paid for by the manufacturers but conducted by independent researchers.  Given the price manufacturers are charging for their new drugs, the public is entitled to have them be as free of safety issues as possible.

Kevin Roche

Author Kevin Roche

The Healthy Skeptic is a website about the health care system, and is written by Kevin Roche, who has many years of experience working in the health industry through Roche Consulting, LLC. Mr. Roche is available to assist health care companies through consulting arrangements and may be reached at khroche@healthy-skeptic.com.

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