Advisory Committees play an essential role in FDA’s activities to protect and promote public health by providing independent expert advice and recommendation to the Agency on scientific, technical, and policy matters.

Background Information: Advisory Committees play an essential role in FDA’s activities to protect and promote public health by providing independent expert advice and recommendation to the Agency on scientific, technical, and policy matters.  Advisory Committees are panels of outside experts that the FDA sometimes convenes when it requires additional expertise before reaching a decision, such as whether or not to approve a drug or device.  Although Advisory Committees provide recommendations to FDA, FDA makes the final decisions on any matters considered by an Advisory Committee.  General procedures for FDA Advisory Committees are described in 21 CFR Part 14.

In February 2019, the Oncologic Drugs Advisory Committee (ODAC) convened to discuss the NDA for selinexor, submitted by Karyopharm Therapeutics.  Following a series of presentations delivered by representatives from Karyopharm and FDA, and an open public hearing, ODAC discussed whether Phase 2 clinical study data were conclusive to allow for an adequate assessment of safety and efficacy in the proposed patient population.

Following deliberation, ODAC voted on the following question: Should the approval of selinexor be delayed until results of the randomized Phase 3 trial are available?

It voted:         Yes: 8              No: 5                Abstain: 0

Eight members voted “Yes”, indicating that approval should be delayed until results of the Phase 3 trial were available.  These members noted that data from the Phase 2 trial did not meet FDA regulatory standards needed to prove the safety and effectiveness.  The five members who voted “No” noted that patients need options for this indication and that selinexor should be granted accelerated approval.

In July 2019, FDA approved selinexor based on data from the Phase 2 study as well as confidential early data from the Phase 3 trial provided by the study’s independent data safety monitoring board.

Prompt: Imagine you are an FDA staff reviewer in attendance at the Advisory Committee Meeting.  Considering the input of the Advisory Committee