Eva Temkin Quoted in BioSpace on FDA’s Expanding Rare Disease Regulatory Framework
Eva Temkin, Arnold & Porter Life Sciences & Healthcare Regulatory partner and former Acting Policy Staff Director at the U.S. Food and Drug Administration (FDA)’s Office of Therapeutic Biologics and Biosimilars, was quoted in the BioSpace article, “Busy FDA gives rare disease sector complementary pathways, unanswered questions.” The article discusses the U.S. Food and Drug Administration’s (FDA) recently introduced rare disease initiatives and how they fit within the agency’s broader expedited development programs.
Addressing the relationship between the FDA’s newer rare disease-focused mechanisms and existing regulatory pathways, Eva noted that sponsors should view them as “complementary,” though she advised that they “should also be mindful that they are not substitutes or alternatives.”
She explained that the FDA’s Plausible Mechanism Pathway differs from traditional expedited programs because it is a “very narrow construct under which specific types of evidence may be considered sufficient under a specified set of circumstances.” By contrast, established programs such as Fast Track, Breakthrough Therapy designation, and Priority Review “address more programmatic considerations,” including agency interactions and review timelines for promising therapies addressing unmet medical needs. Eva also highlighted several unresolved questions surrounding the Plausible Mechanism Pathway, including its implications for postmarketing obligations, regulatory exclusivity, and commercial incentives.
In discussing the FDA’s Platform Technology Designation program, Eva observed that sponsors “are unlikely to see great utility in the program until the second or third submission based on the platform technology,” underscoring the program’s long-term value for companies developing multiple products from a common platform.
