Next Generation of Rare Disease Drug Policy
NORC at the University of Chicago, in collaboration with the Institute for Clinical and Economic Review (ICER), produced a white paper that examines the potential risks and advantages of reforms to current policies related to orphan drug development, pricing, and coverage.
The white paper, “The Next Generation of Rare Disease Drug Policy: Ensuring Both Innovation and Affordability,” was informed by expert input from rare disease patient groups and other stakeholders. It highlights the ongoing need for incentives to drive innovation in rare disease treatments, particularly treatments for the smallest patient groups. A rising number of orphan drug approvals is a sign of success, but patients and the broader health system struggle with the cumulative effect of high orphan drug prices. The white paper analyzes the potential risks and benefits of reforms seeking to strengthen the evidence on orphan drugs and ensure that adequate incentives for orphan drug development are coupled with a more sustainable long-term trajectory for orphan drug pricing.
The white paper does not advocate for specific policy proposals. Instead, it analyzes the potential benefits and risks of reforms meant to improve evidence generation, boost the chances for treatments of ultra-rare diseases, and reduce the prices for treatments whose clinical benefits do not merit traditional orphan-drug-level pricing. The goal was to provide policymakers with an evaluation of different policy reform options and to ensure that potential risks and benefits are examined closely.
The white paper analyzes these potential policy reforms:
- Encouraging ultra-rare drug development
- Limiting incentives for partial orphans
- Strengthening evidence generation
- Reducing prices for rare disease products