The EveryLife Foundation for Rare Diseases is committed to leading and supporting innovative policies to drive rare disease therapy development. The EveryLife Foundation is dedicated to pursuing policy solutions to close the innovation gap for the 95% of rare disease communities that have no FDA-approved treatment, and the 100% of patients seeking curative innovations. To that end, we are grateful for Congress’ 40+ year commitment to advancing innovative rare disease policy. The delicate rare disease therapy development ecosystem requires close evaluation of any legislative proposal to identify benefits and potential unintended negative consequences.
The Promising Pathways Act (S.4426) aims to support the critical goal of working to bring treatments to communities faster. The Promising Pathways Act proposes to create a new conditional approval pathway that would allow FDA to approve treatments for most rare diseases and other rapidly progressing diseases for up to four, two-year periods using very limited safety and efficacy data.
As the EveryLife Foundation worked to assess our position on the bill, it became clear that there is a segment of the rare disease community with fatal and devastating diagnoses, without approved treatments, and whose diseases are so rapidly progressive, that existing access pathways remain untenable. On behalf of that deserving and underserved segment of our community, we worked to engage in discussions around this legislation to seek options.
Despite our sincere interest, the EveryLife Foundation has serious concerns about the current version of the Promising Pathways Act. The unintended consequences of the broad eligibility to use the new pathway, the implications of creating a new pre-approval pathway with very limited efficacy requirements, the impact of requiring coverage for unapproved treatments without coverage for those that have met accelerated and traditional approval standards, and the lack of identified resources for the FDA to operationalize the new pathway could all be extremely harmful to our rare disease community.
While we are disappointed that our engagement efforts to address these concerns were not successful, the EveryLife Foundation remains committed to supporting policies that will deliver safe and effective treatments to patient communities as swiftly as possible. The EveryLife Foundation stands in opposition to the Promising Pathways Act at this time. We thank the rare community for their work and feedback on this issue and hope that we can continue to work with the full community to serve the needs of those currently being left behind.
