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.

News Feed

"Children do not have the time to wait.

We must act now to reauthorize the rare pediatric disease PRV program."

The program spurred the development of new treatments for previously untreatable diseases.

Small + Start-Up Companies Supported

174

RPD Designations Granted

738
Estimated

PRVs Awarded

59

PRVs Sold

30

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 market caps. Each square represents a drug candidate, with colors indicating the company's market cap category. Hover over any square to see details about the company and indication, and click to filter the data by market cap.

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Data updated through April 5, 2025