TL40: Drug approvals for narrow but critical unmet medical needs and diagnostic performance studies: how to harmonize and expedite both
*Deepak B. Khatry, MedImmune 


The Food and Drug Administration (FDA) held a public hearing on February, 2013 to obtain input on a potential new pathway for approving drugs and biologics targeted at serious or life-threatening conditions in smaller subpopulations of patients with serious unmet medical need. The potential new pathway is in response to the 2012 recommendations of the President’s Council of Advisors on Science and Technology (PCAST) to “support the goal of increasing the output of innovative, new medicines for patients with important unmet medical needs, while increasing drug efficacy and safety through industry, academia, and government working together to decrease clinical failure, clinical trial costs, time to market, and regulatory uncertainty.” A few months later in November, 2013 the FDA issued guidance to industry, clinical investigators, Institutional Review Boards (IRBs) and FDA staff on “Design Considerations for Pivotal Clinical Investigations for Medical Devices,” which also includes “diagnostic clinical performance studies.” The new FDA initiative and guidance have overlapping segments with implications for stratified medicines development to enhance healthcare personalization through efficient study sequencing and designs. We will bring together a panel of subject area, study design, and statistical experts from the FDA, academia, and industry to discuss pros and cons for harmonization and expediting of the new initiative within the regulatory guidelines.