Gottlieb Will Have to Decide Future of Efficacy Standard: Schultz

Former FDA deputy commissioner for policy William Schultz says that newly confirmed agency commissioner Scott Gottlieb will need to make an early decision on whether to accept industry proposals to reduce the standards under which drugs can be advertised, standards that Schultz says have “protected patients for more than 50 years.” Writing in a Washington Post op-ed column, he says the issue is whether to allow companies to market approved drugs for additional uses without the full clinical studies that FDA requires for a first indication.

“These companies claim that, as long as what is said is truthful and not misleading, they have a constitutional right to market a drug without ever showing it works for that particular condition,” Schultz writes. “The problem with this approach, as everyone who has worked with clinical studies knows, is that early tests on a small number of patients can suggest great promise, but subsequent, more robust studies often disappoint. This is why FDA generally requires two studies to demonstrate efficacy and that the studies be large enough that their results are unlikely to be due to chance.”

The most serious problem with reducing the efficacy standard, Schultz writes, is that we might never find out whether a drug being used to treat a disease works. He cautions that if drug companies are permitted to talk about promising test results of their drugs, many companies are likely to forgo the expensive, rigorous studies necessary to validate the hope of the early results. “Drug manufacturers typically propose reducing the efficacy standard only for the second use of a drug, which is not as extreme as the wholesale elimination of the standard,” Schultz says. “But such a policy change would still severely impair the ability of physicians to provide the highest quality health care to patients.”