Proposal Would Add a Real-World Evidence Phase to Drug Approvals

A random set of patients could “test” drugs nearing an OK

The drug approval process should include an only-in-research (OIR) step that would gauge real-world effectiveness. That’s the argument put forth by Anirban Basu, a professor at the University of Washington, in a Health Affairs blog post.

Basu says that at the completion of phase 2 trials, the manufacturer would apply to the FDA for a new drug application (NDA) to allow the drug to be included in the OIR process. With the FDA approval, the manufacturer would include the OIR designation on the label along with a random list of birthdays (for instance, odd or even days of the month) for patients who are eligible to take the drug.

“As the FDA surveils the OIR product, the manufacturer carries out effectiveness studies using real-world evidence and submits a final NDA to get full approval, provided the FDA surveillance does not decide to pull the product out of market,” Basu writes.

Clinical trials for drugs currently take anywhere from six to 11 years, Basu points out. The FDA approval after that stage takes about a year. He says that there are still ethical challenges, including the nonuniformity in access to the OIR product.

“But consider the status quo: Under the current process, a much smaller number of people have access—only those enrolled in phase 3 trials at very specific academic centers around the country,” Basu writes. “Also, in the phase 3 trials of the current process, the chances of getting assigned to the new drug (as opposed to a placebo) is essentially determined by a coin flip. In contrast, under this alternative process, a much larger patient population would get access to the new drug in the OIR phase, albeit still in a randomized manner.”

One of the challenges to the FDA would include creating an easily accessible system for doctors to find out who is eligible for the phase 3 drug. Basu says that health insurers should be able to help the FDA install such a system.

Source: Health Affairs; November 2, 2017.