Adaptive approval of drugs for rare diseases
Wendy Olsder1, Tugce Martagan1, Jan Fransoo2, Carla Hollak3
1Eindhoven University of Technology, School of Industrial Engineering, Eindhoven, The Netherlands; 2Tilburg University, Tilburg School of Economics and Management, Tilburg, The Netherlands; 3Department of Endocrinology and Metabolism, Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands
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. We present a Stackelberg game-theoretic model to understand why industry participation has been low. Our results inform healthcare policymakers on ways to redesign adaptive approval programs to likely increase industry participation and improve patients' welfare.