‘Major flaws’ in research for drugs with accelerated approval

Evidence is lacking about drugs approved by the FDA through its fast track process.

Many U.S. patients with serious illnesses are being treated with fast-tracked drugs that have questionable evidence behind them, according to a new study.

The research, published Wednesday in the June issue of The Milbank Quarterly, exposes major flaws in the quick approval of some drugs available to the American public without any stringent clinical evidence that shows their benefits.

Researchers from the London School of Economics and Political Science, The Wharton School and Stanford University evaluated the clinical evidence on drugs receiving accelerated approval from the U.S. Food and Drug Administration (FDA). The agency can approve therapeutic agents that treat serious conditions as part of an accelerated process and those drugs often quickly become part of standard treatment, despite shortcomings in their evidence base, researchers found.


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The study, the first to provide a systematic evaluation of all clinical studies conducted on drugs receiving this type of approval, assessed 37 novel therapeutic agents that received accelerated approval between 2000 and 2013. The researchers examined the timing and characteristics of 7,757 available studies and assessed to what extent the objectives, nature, and timing of research activity were aimed at addressing the limitations of the data available. Randomized trials—the gold standard of evaluating a drug’s effectiveness—constituted only a small minority of existing evidence, they found.

“FDA’s accelerated approval pathway allows potentially promising drugs to receive marketing authorization on the basis of surrogate measures that are easy to obtain, rather than clinically meaningful outcomes,” said author Huseyin Naci, of the London School, in an announcement (PDF).

“Surprisingly, the vast majority of clinical studies including these drugs after market entry are not designed to directly evaluate their benefits. Currently available evidence on drugs given accelerated approval has major flaws and is inadequate to address the information needs of patients and doctors,” he added.

While there is an abundance of research on drugs receiving accelerated approvals, about 70% is of poor quality, he said in an email to FierceHealthcare.

Most clinical studies of the drugs that received accelerated approval are small and nonrandomized, the researchers said. Only about a third of studies are randomized controlled trials, Naci said. A substantial share of the research (about 30%) are not approved by the FDA. And only about half of the studies actually evaluate the effectiveness of the drugs.

RELATED: Hospital Impact: Drug companies win, patient safety loses with 21st Century Cures Act

The 21st Century Cures Act signed into law last year also further lowers the bar for approval of drugs under development by reducing the rigor required for approval of new drugs by the FDA. Traditionally, rigorous double-blinded peer review studies are required prior to consideration and approval, but under the new law pharmaceutical companies will only be required to submit a “data summary” for new pharmacological agents or new indications to existing products, which will make the introduction of new products far easier, according to Jonathan H. Burroughs, M.D.

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