Congress must reauthorize the priority review voucher program
We must continue incentivizing companies to develop rare disease treatments
During the final hours of the 2019 Friedreich’s Ataxia Symposium in Pennsylvania, the Friedreich’s Ataxia Research Alliance announced that the pharmaceutical company Reata had achieved statistically significant positive results in its Phase 2 trial of omaveloxolone (also known as “omav” at the time and now sold under the brand name Skyclarys). This meant Reata would submit omaveloxolone to the U.S. Food and Drug Administration (FDA) for review; if approved, the drug would be made available to Friedreich’s ataxia (FA) patients.
The room erupted with cheers, hugs, and tears. Joy overwhelmed me. Our community’s hard work was so close to bearing fruit. We’d done everything possible to get omav to the FDA, including fundraising and participating in clinical trials.
If omav were approved, it would be the first treatment available for FA — the first option I would have since my diagnosis in 1981. It was an ethereal day when the FDA approved the drug in February 2023.
One incentive Reata had to develop omav was a rare pediatric disease priority review voucher (PRV). Without that PRV, we likely wouldn’t have our first and only FA treatment.
What is PRV?
A PRV incentivizes pharmaceutical companies to develop drugs for rare diseases that affect children. This includes FA, as symptoms usually appear between the ages of 5 and 15.
The voucher grants the pharmaceutical company priority FDA review for one of its products, reducing the wait from the standard 10 months to within six months. This can be a crucial advantage, especially for companies developing treatments for rare conditions, which often have limited or no existing therapies. However, a company needn’t use the voucher on a drug with a rare pediatric disease designation. A PRV can be redeemed for other products or even sold to other pharmaceutical companies.
Importantly, PRVs cost the taxpayers nothing. Pharmaceutical companies still must pay hefty fees to redeem PRVs. According to the Federal Register, this fee is almost $2.5 million for the fiscal year 2025.
If you are like me, you may wonder how vital expedited review can be to pharmaceutical companies. It turns out to be very important! Some companies have purchased PRVs for over $100 million.
Rare diseases such as FA offer pharmaceutical companies a small market and, therefore, limited profits. However, incentives such as PRVs can make the development of rare disease treatments profitable.
When one company purchases a PRV from another, everyone benefits. The company developing the new treatment for a rare pediatric disease raises money, enabling it to bring the treatment to market and fund new therapy programs. The company purchasing the PRV gets a faster review for one of its investigative treatments. Most importantly, patients and families living with rare pediatric diseases gain options where once there were none.
More action is needed
Historically, government incentives to develop treatments and cures for rare diseases have significantly increased the chances of pharmaceutical and biological companies developing such drugs. This started with the passage of the Orphan Drug Act (ODA) in 1983.
According to a StatPearls article, before the ODA was passed, “only ten drugs for orphan diseases were accessible on the market for patient use.”
A 2023 article in the Orphanet Journal of Rare Diseases noted that the FDA approved 1,122 orphan-designated products between 1983 and 2022. This significant jump was partly due to incentives such as the ODA and PRVs.
However, more progress is needed. The goal is to find a cure for each of the estimated 10,000 rare and genetic diseases.
The PRV program is effective. A review by the Rare Disease Company Coalition showed that the program contributed to the development of innovative treatments benefiting more than 200,000 patients across 47 rare, pediatric indications. More than 90% of all PRVs were awarded to drugs indicated for conditions without approved treatments.
Unfortunately, the PRV program expired on Dec. 20, 2024, after Congress failed to renew it. Our legislators can reauthorize it by passing the Give Kids a Chance Act of 2025 (H.R. 1262/S. 932). To learn how you can help, visit the EveryLife Foundation for Rare Diseases website.
I hope that companies continue to develop drugs for rare diseases so that I can celebrate as more treatments cross the FDA finish line. A victory for one rare disease is a cause for celebration for the entire community.
As more treatments, or even cures, are discovered, I hope you’ll join me in my uncoordinated, yet joyful, wheelchair dance. Just think how sweet it will be knowing you helped make it happen.
Note: Friedreich’s Ataxia News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Friedreich’s Ataxia News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to Friedreich’s ataxia.
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