(Clinical) Trials and Tribulations


When Janet Woodcock declared the nation’s clinical trials system to be broken the first time, I wasn’t sure I had heard that correctly. But my ears definitely perked up.

Then she said it again.

“I personally believe, like many other people, that the clinical trial system we have right now is broken. It isn’t working to meet the objectives that we need it for,” she said Sept. 20 at a National Academies workshop on real-world evidence. “Our inability to generate the needed evidence efficiently and in a cost-effective manner will continue to be a barrier to innovation and to the quality of care around the world.”

Dr. Woodcock—which, if you are reading this, you probably already know—is a stalwart of the Food and Drug Administration. She is now the director of the Center for Drug Evaluation and Research, but she’s been everything from the acting commissioner, to the chief medical officer to the chief operating officer.

So it’s probably safe to say she knows a thing or two about the state of the clinical trials system…

(Side note: Satirical publication The Onion parodied her as a pregaming party animal in this hilarious piece.)

Dr. Woodcock was making the case for why the FDA should pursue real-world evidence. FDA Commissioner Scott Gottlieb said they are still coming up with a consensus definition, but it’s in short the use of existing data sources, like an electronic medical record, to make regulatory decisions. It’s a move, when feasible, to find alternatives to randomized controlled trials, which are a major contributor to the soaring costs and time incurred with drug development.

While randomized controlled trials are “the gold standard” for testing if the drug works because you are eliminating as many variables as possible, people also don’t take medicines in such isolated settings. The FDA says this step is especially critical for rare disorders, when getting a large enough sample size for a clinical trial is by definition challenging or impossible. But opponents of the push to real-world evidence say it will lower the level of rigor applied at the FDA to approve new treatments.

Real-world evidence has been and will continue to be a huge topic over the next few years. This, of course, means a lot more coverage of this issue, such as at the International Society for Pharmacoeconomics and Outcomes Research summit Oct. 20. So stay tuned…

Stay on top of new developments in health law and regulation with a free trial to the Health Law Resource Center.

Learn more about Bloomberg Law and sign up for a free trial.