The Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) Program, launched by the FDA in 2012, has been a pivotal initiative in fostering the development of treatments for rare pediatric diseases. The third-of-its-kind, the RPD PRV program was responsible for nearly all 31 PRVs awarded to Sponsors in the first ten years of the programs, according to the US Government Accountability Office Report on FDA’s Priority Review Voucher Programs published in January 2020. That total is now up to 53 across 39 rare diseases as of April 2024, according to NORD’s latest white paper, “Reauthorize the Rare Pediatric Disease Priority Review Voucher Program.” Given its successes and ongoing challenges, the renewal of this program is a topic of significant interest within the industry and a regular conversation amongst RareMoon’s clients. Here’s why we at RareMoon believe the RPD PRV Program should, and likely will, be renewed.
Successes of the Priority Review Voucher Program (Mease et al. 2024)
Over its first decade, the RPD PRV Program resulted in 569 designations, of which RareMoon secured nearly 20%. There was a notable concentration in therapeutic areas such as neurology, metabolism, and oncology, underscoring the program’s success in directing attention and resources toward serious, life-threatening diseases that affect children. Conditions that have otherwise lacked effective treatments have seen increased research efforts, thanks to the incentives provided by this program.
Further, those PRVs have been awarded to multiple modalities: drugs, enzyme replacement therapies, and advanced therapies, including novel gene therapies and the first-ever gene editing therapy approved by the FDA. Since January of 2024, four Priority Review Vouchers have been awarded, RareMoon having secured one of those RPDs!
These positive outcomes are compelling arguments for the program’s effectiveness and necessity moving forward.
Challenges in drug development for rare pediatric diseases
Sponsors of clinical trials for rare pediatric diseases face numerous challenges that can impede the development and commercialization of new treatments. The fragility and rarity of these diseases mean that finding enough participants for clinical trials can be challenging, leading to increased costs.
Securing funding for research and development is often particularly challenging for rare disease treatments due to the lower potential return on investment than treatments for more common conditions. This makes it difficult to attract traditional investment sources. The added challenges pediatric drug developers face can amplify fundraising challenges.
The industry is encouraged by the RPD PRV Program, recognizing the dual benefits of accelerating the review process and bringing vital treatments to market more quickly. This reflects the program’s value in encouraging investment in high-risk areas that might otherwise be neglected due to financial and developmental challenges.
Meeting public health goals for children with rare diseases
As Mease and colleagues (2024) rightfully point out, renewing the RPD PRV Program aligns with broader public health goals and legislative priorities. Policymakers are increasingly focused on addressing unmet medical needs, particularly in vulnerable populations such as children with rare diseases. The program’s renewal would demonstrate a continued commitment to these priorities, ensuring that progress made over the past decade is not lost and that new innovations can continue to emerge.
Addressing challenges of the Priority Review Voucher Program
Despite its successes, the program faces challenges. Critics argue that PRVs can sometimes lead companies to act strategically in ways that don’t always align with the program’s original intent. However, the author suggests that these challenges can be overcome through program adjustments and stricter regulations to ensure the vouchers are used to maximize public health benefits (Mease et al. 2024). At RareMoon, we view these strategic behaviors as opportunities to support subsequent programs and reinvestments that drive further industry developments.
Our support for the Rare Pediatric Disease Priority Review Voucher Program
Given the substantial evidence of its impact, strong industry support, and alignment with public health goals, we agree with Mease and colleagues (2024) and Nord, and feel the likelihood of the RPD PRV Program renewal is high. There is an urgent need for continued development of treatments for rare pediatric diseases with severe, debilitating, and life-threatening outcomes. In our view, the program has proven effective in directing innovation towards this need.
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