Rare pediatric diseases are an area with extreme unmet need in our health care ecosystem. The challenges associated with rare diseases, such as small populations, unknown cause and/or course of the disease, and widely dispersed patient populations, are exacerbated in children. Research and development of products for these diseases is extremely risky and faces tremendous odds. In order to incentivize the life sciences industry to undertake these risks, Congress created the Rare Pediatric Disease Priority Review Voucher program to help bring treatments to these children. Please help show your support for the Rare Pediatric Disease Priority Review Voucher program by sending the below letter to your Member of Congress. You can view and personalize your message on the right side of this page. Thank you!

---

I write to you today to urge you to support H.R. 1262, the Give Kids a Chance Act of 2025, and the pressing reauthorization of the Rare Pediatric Disease Priority Review Voucher (RPD PRV) program, which expired in December 2024. 

Rare pediatric diseases are an area of extreme unmet need in our health care system and are more common than often realized. A rare disease is one that impacts under 200,000 people in the U.S.; combined, 1 in 10, or 30 million Americans have a rare disease. Half of the 30 million rare disease patients are children, and all pediatric cancers are considered to be rare. Of those 15 million children with rare diseases, 30% will not live to see their 5th birthday, and they are responsible for 35% of deaths in the first year of life. Many children with rare pediatric diseases often face enormous odds of getting a diagnosis and a treatment, if one even exists, in a timely way. 

The process of developing products to treat rare pediatric diseases is extremely risky, and the challenges associated with rare disease product development, such as small populations, unknown cause and/or course of the disease, and widely dispersed patient populations, are all exacerbated in children. Additionally, developing treatments for these diseases can be especially challenging because of the severity of these diseases and their early onset.

Therefore, in order to incentivize the development of products for rare pediatric diseases, Congress created the RPD PRV program at FDA, which, upon approval for a product to treat a rare pediatric disease, awards a voucher that entitles the holder to priority review, or FDA review in 6 months, for another product. The RPD PRV program has demonstrated success in progressing products through clinical development and keeps investment flowing into these products, which increases their chances of making it to market. 

Preserving the RPD PRV program is critical to ensuring continued research and development into treatments and providing hope for patients with rare pediatric diseases. I thank you for considering my request and I reiterate my support for the RPD PRV program and for the pressing reauthorization of this important legislation.