FDA Commissioner Scott Gottlieb told Axios that while he thinks the orphan drug market is too small to seriously dampen competition, he's open to changes in the law.
Details: Gottlieb's top priority is giving drugmakers an incentive to develop treatments for the rarest of diseases, or diseases that aren't getting much pharma attention.
Where it stands: Right now, part of the definition of an orphan drug is that it's intended for a patient population of less than 200,000 people. One idea would be to lower that number, meaning orphan designation goes to treatments for even rarer conditions.
- In turn, there could be a "richer incentive" for drug companies to go after orphan approvals.
- "For a drug that's already on the market, maybe you don't need as much incentive to study a subsequent indication," Gottlieb said. "Maybe we make the designation harder to get."
- "I see a lot of things not getting studied or not getting into drug labels that should be. I'd like to have that clinical discussion," he added.
Go deeper: Blockbuster drugs are stacking up orphan approvals
