Faster U.S. Drug Approvals Come at Expense of Safety Oversight

Print 19 January 2016
Anna Edney / Bloomberg Business

Since 2012, an increasing number of drugs have been approved by U.S. regulators through programs meant to speed them to market. It’s good news for patients who benefit sooner from potentially life-saving treatments.

The downside, a government watchdog agency said Thursday, is that the Food and Drug Administration hasn’t been keeping up with monitoring what happens after the drugs are being prescribed to patients -- as it should. The Government Accountability Office, Congress’s investigative arm, foundshortfalls in the FDA’s ability to track whether safety issues arose or check that drugmakers conduct additional trials when they are needed.

“The GAO report confirms my greatest fear, that FDA lacks fundamental resources and leadership in ensuring that drugs brought quickly to market are truly safe and effective,” said Congresswoman Rosa DeLauro, a Democrat from Connecticut who asked for the study. “If FDA is shifting more of the safety risk to consumers by allowing fewer and shorter clinical trials on expedited drugs, adequate tracking of drug safety issues and review of post-market studies are absolutely vital.”

The report highlights the tension between the urge to speed up reviews for ground-breaking treatments that can save many lives and the potential risks those drugs, which on average get approved 3 1/2 months faster than through regular FDA programs, may pose down the road. Cancer treatments, in particular, have benefited from a sharp acceleration in approval times in recent years under the leadership of Richard Pazdur, the FDA’s director of the office of hematology and oncology products. It enabled a drug like Bristol-Myers Squibb Co.’s Opdivo to be cleared months ahead of schedule to treat a form of lung cancer.

What the GAO report pointed out is the FDA’s lack of follow-ups. The agency can request that drugmakers conduct further studies on treatments once they’re for sale. Yet more than half of submissions by pharmaceutical companies from March 2008 to September 2013 -- related to 1,400 post-market studies -- were either reviewed late by the FDA or not reviewed at all.

The FDA said in a response posted by the GAO that it has been working to improve its tracking and assessment abilities. Drugs approved through expedited programs must meet the same standards for safety and effectiveness as other drugs, the FDA added.

About a quarter of the 1,717 drugs approved from Oct. 1, 2006 through Dec. 31, 2014 went through at least one of the FDA’s four expedited programs -- which are priority review, accelerated approval, breakthrough and fast track. Those drugs underwent 8.6 months of review on average compared with 12.1 months for the others, the GAO said. Oncology was the biggest category for speedier reviews.

Better Drugs

In 2015, the FDA approved 30 drugs for cancer, which is more than three times the number in 2009, a decade low.

In an interview before the report came out, Pazdur said the science around cancer has improved drastically, allowing him to move treatments to patients much faster because drugmakers are able to better target those who will benefit.

“The drugs got better,” Pazdur said. “When I first came here, it was ‘Well, should we approve a drug or not?” he said. Now, with treatments that more effective and more tolerable, “it’s not so much a question of should we approve the drug, but how fast can we approve,” he said.

His career epitomizes the recent changes in the FDA review processes. Earlier in his 16-year tenure, Pazdur was cast as a barrier to getting much-needed cancer medications to patients. Now, he’s praised for his fast-track approvals. Pazdur knows from a personal level, too, having lost his wife to ovarian cancer in November, which he says gave him “a sense of urgency that things have to be done.”

Improve Oversigt

Although patients benefit from new treatments, not everyone thinks faster means better.

The FDA often approves drugs for cancer based on what are called surrogate endpoints -- for example, a tumor shrank or grew more slowly during clinical trials. But endpoints like a tumor size may not correlate with survival, said Vinay Prasad, an oncologist with the Knight Cancer Institute at Oregon Health and Science University.

Of 54 cancer treatments the FDA approved from 2008 through 2012, 67 percent were cleared based on a surrogate endpoint, according to a paper Prasad co-authored in the American Medical Association’s journal of internal medicine. After a median follow-up of 4.4 years, five drugs were shown to improve survival, 18 drugs failed to do so and 13 had unknown survival effects, meaning either the companies hadn’t tested them for that or hadn’t reported the results.

The GAO had raised concerns in 2009 about the FDA’s reliance on surrogate endpoints, saying the agency needed to enhance the oversight of post-market studies on those drugs. In the report Thursday, the GAO recommended the FDA develop plans to better track drugs once they’re on the market, including improved databases on safety issues.

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