Dan Kracov and Eva Temkin Analyze FDA’s Proposed Conditional Approval Pathway for Rare Disease Drugs in BioSpace
Dan Kracov, chair of the firm's Global Life Sciences Industry group, and Life Sciences and Healthcare Regulatory partner Eva Temkin were quoted in the BioSpace article, “Makary’s ‘Conditional Approval’ Pathway for Rare Diseases Poses More Questions Than Answers.”
The story examines Food and Drug Administration (FDA) Commissioner Marty Makary’s proposal to grant conditional approval to rare disease drugs based on a “scientifically plausible mechanism.” Kracov and Temkin highlighted the legal, regulatory, and practical complexities the proposal raises, particularly regarding safety assessment and product liability.
Kracov emphasized that the proposal would likely require congressional legislation. While he acknowledged that lowering the bar to improve access may be a positive step, he stressed the need for meaningful standards to ensure payer reimbursement. Approving drugs based on a plausible mechanism, he cautioned, is “about as low a standard as you could possibly get.”
Temkin, former Acting Policy Staff Director at the FDA's Office of Therapeutic Biologics and Biosimilars, compared the proposal to the Right to Try law, but warned that the new pathway “may be less protective of patients.” She also raised concerns about safety monitoring and post-market enforcement, noting the difficulty of assessing adverse events without sufficient clinical data: “Separating the signal from the noise when you don’t even know very much about the safety or effectiveness of the drug becomes very difficult.”
