ICER White Paper – Rare Disease Drug Policy Reform, Drug Access, and Innovation

Despite the advancements in rare disease treatment, patients with many rare diseases still have extremely limited, if any, treatment options. Changes in regulatory approval for rare disease drugs like the Orphan Drug Act have helped to bring new treatments to the clinic, but some worry whether the evidence supporting such drugs is strong enough and if the current pricing trends are sustainable. A new ICER white paper explores these issues and more.

According to ICER, “The White Paper does not advocate for specific policy proposals. Instead, it analyzes the potential benefits and risks of reforms that might serve to reshape the incentives for orphan drug development in a way 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 ensure that potential risks and benefits are both examined closely.”

Read more and download the free whitepaper by clicking here.

(Source: ICER, April 7^th, 2022)