Problem definition: Adaptive approval is a novel regulatory program that enables earlier patient access to new drugs for rare diseases. The program has been in place for almost a decade, however, industry participation has been surprisingly low. Academic/Practical Relevance: The main purpose of...

There are more than 7,000 known rare diseases, but pharmaceutical manufacturers developed treatments for only 500 of them. To improve the availability and accessibility of treatments for rare diseases, governments have introduced several programs including subsidies, innovative pricing schemes,...