Finding cures for rare diseases may become more difficult

MADISON, Wis. (WMTV) - Rare diseases impact one out of every 10 Americans, but a voucher program to incentivize drug companies to research rare disease treatments is on the brink of expiring.

Katie Moreau, a mother to five boys, has one child with a rare genetic condition called Prader-Willi syndrome.

“I was told by doctors to be prepared, he’s going to have a short life,” Moreau said.

95% of rare diseases, like Prader-Willi syndrome have no treatment.

A mother turned advocate, Moreau is hoping Congress will extend the Rare Pediatric Priority Review Voucher program to incentivize drug companies to find a cure for her son.

“Right now, they see the voucher program as a major financial incentive to develop treatments for rare disorders,” explained Dr. Stephen Meyn, the director of the UW Center for Genomics and Precision Medicine.

Rare disease treatments have a small market, Meyn said.

“You’re not going to create a blockbuster drug that everyone is going to be taking,” he said.

In order to create a financial incentive for drug companies to still research rare diseases, a voucher program was created in 2012. Essentially, if the FDA approves your rare disease treatment, then you can get priority review for another future drug you make for any disease, not just rare ones.

“So what you’re selling is the opportunity to get another drug on the market about six months sooner than otherwise,” Meyn said. “That is considered quite valuable.”

However, if no further action is taken to extend the program, pharmaceutical companies will have until Sept. 30, 2026 to receive vouchers for their qualifying drugs. After that, the FDA would no longer be able to give out Priority review vouchers.

Drug companies can sell these vouchers to each other for hundreds of millions of dollars, which some critics of the program say could ultimately drive up drug prices for consumers.

Meanwhile, Moreau sees the voucher program as being one step closer to finding a cure for her son, Kade.

“I can’t give up hope,” Moreau said.

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