With PRV’s reauthorization, the rare disease community gets a rare win
The Give Kids a Chance Act was named in honor of Mikaela Naylon
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Frustration is found around every corner in my life with Friedreich’s ataxia (FA). I get frustrated about many things, macro and micro. This can range from the inaccessibility of public spaces (macro) to struggling to find the armhole in my hoodie when I get dressed in the morning (micro).
One macro thing I’ve been frustrated by is the rare disease community’s loss of the priority review voucher (PRV), an important incentive for pharmaceutical companies to develop treatments for rare pediatric diseases. I wrote about the voucher in a previous column.
But something really exciting happened on Feb. 3. After the efforts of many advocates, President Trump signed the Mikaela Naylon Give Kids a Chance Act into law last week. Included in that bill was a five-year reauthorization of the PRV. Not only will this help many, but it will also make advocates like me less frustrated.
Many of us with FA are beneficiaries of a PRV. Skyclarys (omaveloxolone), the only treatment for FA approved by the U.S. Food and Drug Administration, was developed with a PRV. It isn’t a stretch to say that Skyclarys wouldn’t be available without it.
A little history
The law reauthorizing the PRV was named after Mikaela Naylon, who spent the end of her life advocating for the needs of young people with rare cancers. Naylon, who died in October, was diagnosed with osteosarcoma in 2020.
The PRV was expected to be reauthorized for five years in December 2024, but was cut, along with other healthcare programs. I was one of many who advocated for its renewal, frustrated that a program that cost taxpayers nothing and helped children with rare diseases was being cut.
Our advocacy was successful, and the Give Kids a Chance Act, which was introduced in the House of Representatives last February with strong bipartisan support and 313 co-sponsors, was passed unanimously in December. The bill stalled in the Senate, but after advocates urged senators to move forward, the bill was included as part of a larger appropriations package to end a partial government shutdown.
The PRV is now available to companies developing therapies to treat and cure rare pediatric diseases, such as FA. Importantly, the budget the president signed also renewed several key provisions, including more robust funding for the National Institutes of Health, which are also important to the rare disease community. I am so grateful to all our advocates who worked so hard to make this happen, and to readers, friends, and family who called their legislators when I asked for their help.
Patience, perseverance, and advocacy work. I am trying to let that sink in as I move forward with my team of rare disease advocates, knowing we can make things happen. I can and will move through these frustrations.
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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