Speeding up drug-approval process could have downside

By Ed Silverman

Excerpted from The Wall Street Journal, May 30, 2015.

Would a congressional bill designed to jump-start medical innovation end up lowering standards for approving new uses of existing medicines?

Consumer advocates are raising this concern about the 21st Century Cures legislation, which passed the House Energy and Commerce Committee unanimously last week and, in part, is designed to reform the approval process for drugs. Supporters say the bill is a long overdue move that, among other things, will give the FDA the tools to ensure treatments reach patients faster.

But critics say that a section of the bill devoted to drug development is problematic. Specifically, they point to language that would allow the FDA to approve additional uses for drugs without having to rely on randomized controlled trials. These are considered to be the gold standard for determining whether a medicine offers a benefit, and they help gauge the extent to which there are risky side effects.

The bill, however, pushes aside evidence in favor of something called “clinical experience,” which is defined as a mix of observational studies, patient registries and therapeutic use. None of these, however, are viewed as scientifically rigorous for establishing whether a drug may be effective. Instead, critics say the language in the bill is sufficiently, perhaps deliberately, vague.

“Clinical experience is something that should be considered as additional information, but absolutely never take the place of scientific data,” says Diana Zuckerman, who heads the National Center for Health Research, a nonprofit think tank. “By urging FDA to get away from randomized clinical trials, drug makers may have more power to urge the FDA to consider data that is favorable to their product.”

To read the entire article, including similar concerns expressed by Dr. Steven Goodman from Stanford, see http://www.wsj.com/articles/speeding-up-drug-approval-process-could-have-downside-1432857506