Valuing Rare Pediatric Drugs: An Economics Perspective
There is a coming wave of novel genetic therapies aiming to treat rare pediatric disease. A large literature investigates the valuation of new treatments, but the valuation of treatments for rare pediatric illness raises a host of unique issues. In this paper, we review the challenges of applying both the standard economic model and standard approaches to estimating cost-effectiveness using the quality-adjusted life year (QALY) to this case. We argue that there are a large number of special issues that have only been partially addressed by past work and we conclude that more data and the development of new methods are vital as innovators, health technology assessment practitioners and policymakers confront the launch of these new drugs.
The origin of this study is a workshop on this topic sponsored by Sarepta Therapeutics. This study was motivated by, and summarizes many of the lessons from, this workshop. We are grateful to all participants in that workshop. Gruber, Neumann and Ollendorf acknowledge financial support from Sarepta Therapeutics. Conti did not receive financial support from Sarepta Therapeutics. This study and publication was not contingent on Sarepta’s approval or censorship of the manuscript. The authors thank Kao-Ping Chua for excellent comments on a previous draft. The views expressed herein are those of the authors and do not necessarily reflect the views of the National Bureau of Economic Research.