The Biotechnology Industry Organization (BIO) is pleased to submit the following comments on the draft guidance issued by the Food and Drug Administration (FDA) on April 9, 2014, entitled “Draft Guidance for Industry, Researchers, Patient Groups, and Food and Drug Administration Staff: Meetings with the Office of Orphan Products Development” (the “Draft Guidance”).
We want first to express our continuing appreciation for the specific attention paid to the unique challenges of developing therapies for rare diseases. Recognizing the differences between how drugs and biologics for rare diseases can be developed—given the small patient populations—and drug development for non-rare conditions is crucial to the creation and implementation of innovative strategies to overcome these challenges. The designated Office of Orphan Products Development (OOPD) is an important aspect of this recognition, as it offers manufacturers of rare disease therapies a dedicated interface with the Agency and a central coordinator throughout the regulatory review process.
We commend FDA for establishing concrete expectations for interactions with OOPD and believe the Draft Guidance is important in order to establish a common understanding of the goals and scope of both informal and formal meetings between the Agency and manufacturers. While the Draft Guidance is, on the whole, a thorough and thoughtful document, we offer several suggestions that we believe will further the FDA-stated aim of providing for “consistent procedures to promote well-managed meetings”. We identify three areas below in which we think the Draft Guidance would benefit from greater clarity and specificity.