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FDA head is open to orphan drug law changes

In this image, FDA commissioner Scott Gottlieb sits with a hand covering his mouth as he frowns into the distance beyond the camera.
FDA Commissioner Scott Gottlieb. Photo: Drew Angerer/Getty Images

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

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