If new restrictions are placed on the FDA’s Accelerated Approval pathway, as many as two-thirds of the treatments approved by this pathway would never reach patients, according to research released at the BIO International Convention.
Accelerated Approval, in a nutshell: Established in the 1990s to encourage R&D for HIV/AIDS, the pathway accelerates drugs for serious conditions for which no effective treatments exist. Drugmakers gain approval based on achieving a surrogate endpoint; they’re ultimately required to prove clinical benefit.
Why it matters: 82% of Accelerated Approval drugs are for so-called orphan conditions, which affect fewer than 200,000 people and are thus commercially challenging to address, said Duane Schulthess, CEO of Vital Transformation, which released the report at the convention.
The key finding: “33% to 66% of accelerated approvals at the median delay of three years will no longer have a net positive NPV, and would be at high-risk of not coming to market or being developed at all,” the report finds. “We estimate these at-risk therapies address the needs of 850,000 to 3.6 million patients.”
But: “CMS just changed everything,” said Global Alzheimer's Platform Foundation President John Dwyer at the session—click to read what they did.
Patient advocates agree: The pathway must be preserved—with Dr. Eric Dube, CEO of Travere Therapeutics, telling a panel this week that it’s been “a gamechanger for patients.”
Watch: BIO’s John Murphy and Khushboo Sharma, as well as patient advocates, explain why it’s vital to protect Accelerated Approval.
