The white paper, published at the Florida State University College of Medicine’s Institute for Pediatric Rare Disease, outlines a patient-centered roadmap for regulatory reform and calls for greater regulatory flexibilities, including mechanism-driven approvals for new treatment innovations.

“Today’s regulatory and insurance systems were built for a different era of medicine,” said Patients Rising’s Executive Director MacKay Jimeson. “We now have the science to treat previously untreatable diseases, but if regulatory approval isn’t paired with guaranteed, affordable access, patients are still left waiting. It’s time to modernize healthcare to reflect 21st-century science, medicine, and technology.”

The paper highlights the urgent need for the FDA to consistently use its congressionally authorized flexibilities for rare diseases. It also calls for insurers to recognize as legitimate treatments therapies already approved by the FDA, including those approved through Accelerated Approval pathways. Key patient-focused reforms highlighted in the report include:

• Urging the FDA to fully use its authority to approve rare disease treatments swiftly, as supported by Commissioner Marty Makary.
• Demanding insurers cover FDA-approved therapies, including those via Accelerated Approval, without delays.
• Pushing for financing models that prioritize long-term patient health over short-term costs.

The paper also highlights the importance of access to new treatment innovations.

“Cutting-edge treatments are meaningless if patients face insurance barriers, delays or denials,”  said Terry Wilcox, co-founder at Patients Rising. “We have the science to save lives, but bureaucratic hurdles and insurance barriers stand in the way.”

Jimeson thanked the FSU’s Institute for Pediatric Rare Disease for its leadership in advancing research and innovation for the rare disease community, noting that partnerships between academic institutions, patient advocates, and policymakers are crucial to driving the next generation of lifesaving treatments.

Read the entire report: Unlocking Hope for the Few: Accelerating Access and Evidence Pathways for Rare and Ultra-Rare Disease Treatments