The FDA and pharmaceutical companies want to improve drug development and approval for rare diseases, the majority of which lack approved treatments. There are over 7,000 rare diseases in the U.S. and only about 5% to 6% have FDA-approved treatments.
WASHINGTON — The Food and Drug Administration and pharmaceutical companies say they’re hoping to improve drug development and approval for rare diseases, the majority of which lack FDA-approved treatments.
“Establishing appropriate efficacy endpoints in rare diseases is often challenging because of a lack of regulatory precedent, small trial populations and limited understanding of a disease’s natural history,” said Heidi Ross, acting vice president of policy and regulatory affairs at the National Organization for Rare Disorders.
The RDEA pilot was proposed as part of the FDA performance goals outlined amid the reauthorization process of the Prescription Drug User Fee Act. “The medicine has already been approved, that means FDA already has efficacy and safety data, maybe for a different disease, but still it is familiar with the product,” PhRMA’s Vereshchagina said of the STAR program.
“NORD is pleased to see that FDA will consider the applicability of the endpoints for multiple diseases, which will help broaden the impact of the pilot beyond the selected applicants,” Ross said. “It is critical that the learnings from this program ultimately be made available so that future rare disease drug applications can build on the progress made within the program.”
Novartis NVS, +0.85% said in an email that it “supports the PDUFA VII goals letter as negotiated between FDA and industry, and [welcomes] new regulatory initiatives that further advances innovative product development and enables earlier patient access.” Sharp said in an interview that the agreement process for prescription drug user fees tends to be quick, as “there’s a lot of clarity on both sides about what the value of the fee is, what the rules of the road are, [and] what the scale of it is.”
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