A Catalyst for Rare Pediatric Disease Research
Like the Big Bang, the RPD Priority Review Voucher Program (RPD PRV) introduced new energy into a space that previously had fewer incentives for investment, catalyzing the development of rare pediatric disease treatments across more than 60 indications.
This dashboard provides insights into how PRVs have been awarded, used, and traded—helping researchers, policymakers, and industry leaders assess their impact within the evolving rare disease ecosystem.
Joe and Courtney Dion
parents of two children with limb-girdle muscular dystrophy 2C &
Founders of the Dion Foundation for Children with Rare Diseases
For parents of children with rare, untreatable conditions, easy answers are difficult to come by.
But the rare pediatric disease priority review voucher program is a policy we know works.
Joe and Courtney Dion
parents of two children with limb-girdle muscular dystrophy 2C &
Founders of the Dion Foundation for Children with Rare Diseases
The program spurred the development of new treatments for nearly four dozen previously untreatable diseases.
60
New Treatments for
47
Rare Diseases
231
Small + Start-Up Companies Supported
738 Estimated
RPD Designations Granted
Industry Perspectives
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New research sparked by the program yielded treatment options for 43 pediatric diseases which lacked previously-approved treatments.
Therapeutic Area Distribution
This chart shows the distribution of drug candidates across therapeutic areas. Each square represents a single drug candidate, with different color shades indicating different indications within the same therapeutic area. Hover over any square to see the specific indication, and click to view details about that therapeutic area.
The program overwhelmingly encouraged research and development from smaller teams and companies.
Company size distribution
This visualization shows the distribution of drug candidates across different company sizes. Each circle represents a drug candidate, with colors indicating the company's size and stage category. Hover over any circle to see details about the company and indication, and click to filter the data by size and stage.
Spurring Industry Innovation
Children do not have the time to wait.
We must act now to reauthorize the rare pediatric disease PRV program.
Stuart Siedman
Vice President, Patient Advocacy, Chiesi Global Rare Diseases
