ICER paper considers rare disease drug policy

Unofficial US drug price watchdog the Institute for Clinical and Economic Review (ICER) has published a new white paper in collaboration with researchers at NORC at the University of Chicago.

This paper, called The Next Generation of Rare Disease Drug Policy: Ensuring Both Innovation and Affordability , examines the potential risks and advantages of reforms to current policies related to orphan drug development, pricing, and coverage.

"Tremendous unmet need remains"Steven Pearson, president of the ICER, said: “Since its passage, the Orphan Drug Act and accompanying scientific advancements have successfully increased the number of treatments available for patients with rare diseases, but tremendous unmet need remains.