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Competing for Patients

Increased interest in developing drugs for rare diseases is leading some trials to struggle to attract participants, writes Amy Dockser Marcus at the Wall Street Journal.

For instance, she notes that there are three trials gearing up to test treatments for Niemann-Pick Type C, but the pool of prospective participants is small; there's an estimated 500 people in the world with the condition. Scientists and patients' families worry that the three trials will fight over patients and that no drug will be able to be approved from any of them.

"This is a critical juncture for our community," Nadine Hill, executive director of the National Niemann-Pick Disease Foundation, tells the Journal. "To lose out on having an approved treatment is our worst-case scenario."

At the same time, not all patients will meet the trials' criteria — one trial is limiting enrollees by age, for instance, and by whether they've already received the drug under compassionate use — and, Dockser Marcus notes, some potential participants may shy away in fear that they won't be among the participants to receive the drug under investigation.

Still, Anders Mørkeberg Hinsby, the CEO of Orphazyme, one of the companies launching a NPC drug trial says the community can handle multiple trials, "but it has to be coordinated well."