On September 30, 2020, the Creating Hope Act, a pediatric voucher program, is scheduled to expire. The Creating Hope Reauthorization Act (HR 4339) permanently reauthorizes this wildly successful pediatric voucher program. This program has created a pathway for small biotechs and even larger pharmaceutical companies to develop drugs for pediatric rare diseases.
At no cost to taxpayers, The Creating Hope pediatric voucher program has created well over $1 billion in incentives for pediatric drug development. When a company gets approval for a specific pediatric cancer or rare disease drug they receive a voucher from the FDA, which is transferable and it entitles the holder to put another drug, perhaps a major blood pressure or cholesterol drug, through the FDA’s priority review process, potentially allowing them to get to market more quickly. Pediatric rare disease vouchers have traded for between $67 million and $350 million each.
Another measure of success is the increase in the number of new drugs that have been approved for rare pediatric diseases before the passage of the Creating Hope Act and after. Since 2012, the program has seen 22 rare pediatric disease drugs that were approved after passage of the Creating Hope Act, a jump in FDA approval of rare pediatric disease drugs of over 120%.
The number of rare pediatric disease drugs that were in the drug development pipeline in 2019 was a 200% increase over 2016. Perhaps the most important, long-term impact of the Creating Hope Act has been to improve the risk/return ratio of biotech and pharmaceutical companies making them more interested in pediatric rare disease drug development.
Since 2012, the program has been reauthorized three times, each time for
only one to four years. This actually has not been the most favorable situation for providing incentives because researchers and executives at the start of a drug development plan cannot count on the existence of the pediatric voucher program, 7 to 10 years later, when their drug might be approved, therefore they are less likely to be incentivized to develop rare pediatric disease drugs, but if The Creating Hope Reauthorization Act voucher program were made permanent, it will have an even greater impact on the number of new rare pediatric disease drugs in development.
Please cosponsor HR 4439-Creating Hope Reauthorization Act to provide incentives in the form of non-taxpayer funded vounchers to promote development of rare pediatric drugs.