A pair of recent studies of accelerated approvals by the Food and Drug Administration may collide with the Trump administration's push to speed the government's drug review process.
The Journal of the American Medical Association (JAMA) reports that a study of 22 drugs for 24 different indications were granted accelerated approvals from 2009 to 2013 but the trials were not as rigorous as normally required, less than the gold-standard of randomized controlled trials. Moreover, when the regulator ordered follow-up studies to ensure the efficacy of the drugs, only 50% had been completed three years later. Of the remaining studies, 11 were being done on time, six had been delayed and two were canceled. And the study also found that the follow-up trials did not meet accepted approval protocols, sometimes did not yield expected benefits or were shut down early.
Huseyin Naci of the London School of Economics and one of the authors of the drug study wrote, "Although (the acceleration) can be highly effective in facilitating the approval of certain new drugs . . . approved via expedited pathways may have greater safety risks to patients."
Naci and his co-authors said the FDA needs to identify data limitations caused by the abbreviated nature of the study and detail how the follow-up studies should be adapted to account for those limitations.
A second study by a trio of doctors at the University of California San Francisco showed modifications to already approved high-risk medical devices such as cardiac stents seldom using the most rigid standard, a so-called "panel track."
Over a 10 year period beginning in 2006, just 78-panel track approvals occurred. But from 1979 to 2012, 5,800 non-panel modifications happened for cardiac implantable electronic devices alone.
But even the panel-track studies may not be that strenuous. The UCSF study showed that less than have of the trails were randomized and just 30% of those trials were blinded. And almost 25% of the trials specified a specific endpoint.
PhRMA, a lobbying group for the pharma industry defended the accelerated approval process, saying that faster approvals of drugs by the FDA often provided treatments to patients who were suffering from diseases where standard care was falling short.
Deborah Katz, a spokeswomen for the FDA, said "We are considering this study as part of the larger body of evidence concerning medical products and will carefully review its findings."
President Trump has pledged to quicken the pace of drug approvals by the regulator, stating in his address to the joint session of Congress in March that he wanted to "slash the restraints" on drug development and that he intended to change the "slow and burdensome approval process."
Scott Gottlieb, Trump's choice to head up the FDA, has promised changes in the culture of the regulator and among the shifts would be an examination of the approval process.
Trump's remarks were not universally accepted. Noted FDA critic Dr. Michael Carome of Public Citizen's Health Research Group took issue with his remarks saying they demonstrated "complete ignorance about the FDA's current regulatory schemes for ensuring that medications and medical devices are safe and effective."
While drug companies have long complained that the journey to a drug approval can take a decade and cost north of $1 billion, the fact is that the median time for a FDA drug review is now just 10 months, down from 13 months in 2005, according to the regulator. The time can slide to 8 months if the drug has been given a priority review status.