This White Paper examines the potential risks and advantages of reforms to current policies related to orphan drug development, pricing, and coverage. 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 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.   

Date of publication: April 2022

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Final Documents

Below you will find the final documents from the policy paper review process:

“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. As a society, we must prioritize ongoing innovation and drug development for rare diseases—particularly those conditions with no available treatments today.  

Treatments for ultra-rare conditions remain particularly elusive, as current market dynamics often make it challenging for manufacturers to bring these products to market.  However, at the same time, there are widespread concerns about the perceived weakening of evidence requirements for orphan drugs, and questions are being raised about whether the health system can sustain access to orphan drugs if current pricing trends continue as the cumulative number of orphan drugs increases. To secure a future in which innovation and affordability are both ensured, policymakers are going to have to consider potential reforms.”