The economics of moonshots: Value in rare disease drug development.

The authors review the literature surrounding the economics of rare disease drug development and access before advancing the case for novel approaches to funding treatments. To fund the next stage of rare disease drugs, which will likely center on gene therapies and molecular medicine, they discuss value frameworks as well as patient- led models of finance, and how these may fit into the existing frameworks in the US to incentivize rare disease drug development and access.