Andrea Alms Video:  Money In Motion 149 - Pediatric Rare Disease Market

TRANSCRIPT: Hello, my name is Andrea. Alms. I am a technology investor and financial manager and this is your money in motion. The future of pediatric rare disease is in question. The end. The U.S. Congress did not reauthorize the Rare Disease Priority Review Program at the end of 2024, despite bipartisan support. Advocates felt the ripple effect is being felt now across biopharma.

Rare disease. Pediatric disease has historically been overlooked in drug development, as pharmaceutical companies often prioritize research for conditions affecting larger patient populations. Before the 1983 Orphan Drug Act, there were very few financial incentives for companies to invest in rare diseases treatments, leading to significant unmet medical needs for children suffering life threatening conditions. The odds to help encourage research and development by providing tax credits, grant funding, market exclusivity for rare disease treatments.

However, there's still a gap when it comes to pediatric specific conditions, which typically involve even smaller patient populations and UN and unique scientific challenges. Recognizing this gap, Congress passed the rare pediatric disease PRV program as part of the FDA Safety and Innovation Act in 2012 to accelerate the approval process for rare pediatric treatments by offering priority review vouchers as a financial incentive.

While the Rare Pediatric Disease PRV program saw notable success, its legislative authorization included a sunset clause which resulted in the program expiring in September 2024. Congress did not act in time to extend or authorize it, despite strong advocacy from patient organizations, research, and industry stakeholders at Grandview Research. The global market was 195.2 billion in 2024, growing at a compound average growth rate of 11.6%.

Please note, pediatric rare disease is about 50% of the entire market. At Prophecy Market Insight, the market size was 161.4 billion in 2020 and projected to grow to 547.5 billion by 2030, at a growth rate of 13.1%. At Vision Research markets, the market was 119.7 billion in 2021 and expected to reach 335.99 billion in 2030, at a growth rate of 12.15%. Averaging the averages, the market was 158.8 billion at a 12.3% growth rate, triple digit billion dollar market growing a double digit growth rate assuming the rare pediatric disease is 50% of the entire market. It is still a double digit billion dollar market. It is odd the future of rare pediatric drug innovation would remain uncertain. Thank you. This is your money. Your money in motion.