The Give Kids a Chance Act of 2025 is a piece of legislation poised to transform drug development for children with rare diseases and cancer. Reintroduced in the House of Representatives on February 12, 2025, by Congressman Michael McCaul, this bipartisan effort aims to extend the Priority Review Voucher (PRV) program beyond its anticipated September 2026 sunset, preserving critical incentives for rare pediatric disease (RPD) drug developers and offering new hope to families across the nation.
What is the PRV Program?
The PRV program has been a game-changer in rare pediatric disease drug development since its inclusion in the program in 2012. It incentivizes companies to invest in treatments for rare pediatric diseases by allowing for expedited FDA review of future drug applications. These vouchers have become an important source of non-dilutive funding for rare disease drug developers, typically selling for around $100 million. As of April 30, 2024, a total of 53 RPD PRVs have been awarded, reflecting a steady upward trend in RPD drug development.
The impact of the PRV program has grown significantly over time. More than half of all RPD PRV designations, awards, and redemptions occurred in the last four years. Between 2013 and 2022, 569 therapies won RPD designation, with neurology (26%), metabolism (23%), and oncology (18%) being the top indications.
- What’s at Stake?
The Give Kids a Chance Act addresses several key areas in RPD drug development 1, including the reauthorization of the Pediatric Priority Review Voucher (PRV) program, mandated studies on combination therapies for pediatric cancers, and enhanced FDA enforcement capabilities for timely completion of pediatric studies. These provisions are crucial for maintaining momentum in developing life-saving treatments for children with rare conditions.
Why Does it Matter?
Every year, thousands of children battle rare diseases with limited treatment options. The Give Kids a Chance Act is an opportunity to turn the tide, offering accelerated development of life-saving therapies, increased investment in pediatric rare disease research, and a potentially brighter future for children affected by rare pediatric conditions.
For families affected by rare diseases, this legislation means the opportunity for additional treatment options for their children and potentially faster access to breakthrough therapies. More than 90 percent of all PRVs were awarded to therapies for indications with no approved therapy on the market, offering new hope for previously untreatable conditions. If passed, this legislation could enhance collaboration between researchers, pharmaceutical companies, and regulatory bodies, allowing them to focus on enhancing the quality of life for children with rare diseases
The Give Kids a Chance Act also amplifies the voice of rare disease communities in the drug development process, ensuring that their needs are heard and addressed.
How Should Drug Developers Respond?
This act could impact RPD drug development pipelines for pharma and life science companies. It will be essential for companies to take this into consideration as they plan out their strategies for the coming years. This includes evaluating PRV opportunities and understanding how they can be optimized in the current regulatory environment.
To do so, companies should have robust and real-time insights into the disease landscape. Leveraging real-world data will be critical to staying ahead of the curve, since it can:
- help identify new patients for clinical trial recruitment
- improve site selection
- enhance clinical feasibility strategies and clinical trials
- bring treatments to market more efficiently
Furthermore, it’s important to stay informed about PRV valuation trends. Strategizing the best timing for utilization or sale of your vouchers can significantly impact your bottom line and future research investments. By combining these insights with comprehensive data analytics, you can enhance your overall trial performance and ensure that your RPD drug development strategy is optimized for success.
Unlocking the Power of Real-World Data with PurpleLab
Looking for a trusted real-world data partner to help you navigate these regulatory changes and optimize your RPD drug pipelines? Look no further. PurpleLab provides access to real-world data that accurately reflects the dynamic nature of the specialty drug market.
Our clinical team constantly reviews and optimizes our expansive dataset, so you’re left with the most accurate and valuable insights across predominant specialty classes such as autoimmune disease, oncology, neurology, hematology and rare diseases.
We also have near-exclusive access to specialty pharma data from one of the three PBMs. But for a well-rounded strategy, our healthcare analytics platform compliments competitor and other vendor data by adding additional patients to the claim environment with pharmacy and medical, open, and closed specialty drug data, as well as specialty drug retail benefit (Rx) claims that can be blocked to competitors. This can augment your data sets and expand reach, helping you identify new patients and providers, as well as achieving more in-depth insights.
Don’t miss this opportunity to stay ahead in the rapidly changing world of RPD drug development. By optimizing your strategy with real-world data, you’re helping to bring much needed life-changing treatments to children who need them most.
Request a demo today to discover how we can work together to make this happen.