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Rare Pediatric Disease Voucher Program is Being Phased Out by FDA…

Will companies continue to develop new meds for rare pediatric diseases?

The FDA will be ending their Rare Pediatric Disease Priority Review Voucher Program. This program was designed to award priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. A sponsor who receives an approval for a drug or biologic for a "rare pediatric disease" may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product. Unfortunately, this voucher program is being phased out. Details of the phase out are as follows:

  • Pediatric disease designation for the drug must be granted by the FDA by September 30th, 2024.

  • After September 30th, 2026, the FDA may not award any rare pediatric disease priority review vouchers.

Well over 50 companies have sold these vouchers, in some cases for over $300M. More recently the vouchers have sold for around $100M (See Graph). Some companies who have sold vouchers are shown in (Table 1). Recently, SRPT announced that it sold one of its vouchers for around $100M that it obtained for a Duchenne's Muscular Dystrophy (DMD) approval. With this program ending, companies will now have to find alternative funding opportunities to justify the expense of these clinical trials, potentially hindering the number of companies developing drugs for rare pediatric diseases.

Table 1: Examples of companies that have used the Rare Pediatric Disease Priority Review Voucher (PRV) Program:

Figure 1:

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