Therapeutic Value of Drugs Lower for Supplemental Versus First Indications

For first three approved indications, second and third indication approvals less likely to have high value rating

By Elana Gotkine HealthDay Reporter

FRIDAY, July 7, 2023 (HealthDay News) — For drugs approved in the United States and Europe, the therapeutic value of supplemental indications is lower than for first indications, according to a study published online July 5 in The BMJ.

Kerstin N. Vokinger, M.D., J.D., Ph.D., from the University of Zurich, and colleagues analyzed the therapeutic value of supplemental indications compared with first indications for drugs approved in the United States and Europe in a retrospective cohort study. The study included 124 and 335 first and supplemental indications, respectively, approved by the U.S. Food and Drug Administration, and 88 and 215 first and supplemental indications, respectively, approved by the European Medicines Agency, between 2011 and 2020. The largest subset of indications was for cancer disorders. Therapeutic ratings were available for 86 percent of first and 53 percent of supplemental indications in the United States, and for 99 percent of first and 86 percent of supplemental indications in Europe.

The researchers found that among FDA approved indications, 41 versus 34 percent of first and supplemental indications had high therapeutic value ratings, compared with 47 versus 36 percent in Europe. Among FDA approvals, second and third indication approvals were less likely to have a high value rating (relative ratios, 0.64 and 0.55, respectively) compared with the first indication approval when the sample was restricted to the first three approved indications. Similar findings were seen for Europe.

“When first or supplemental indications do not offer added therapeutic value over other available treatments, that information should be clearly communicated to patients and physicians and reflected in the price of the drug,” the authors write.

Several authors disclosed ties to the pharmaceutical industry; one author is an expert witness in a qui tam suit against Biogen.