The Biotechnology Industry Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments on the “Expedited Access for Premarket Approval Medical Devices Intended for Unmet Medical Need for Life Threatening or Irreversibly Debilitating Diseases or Conditions; Draft Guidance for Industry and Food and Drug Administration Staff.”
In general, BIO finds the Draft Guidance to be very insightful and well written, providing useful examples and references to illustrate the important principles underlying most sections. In particular, the discussion of clinical, intermediate, composite, and surrogate endpoints, including their utility during development and their respective pre- and post-market data burdens, is helpful to Sponsors developing premarket approval (PMA) medical devices with the potential to address unmet medical needs for life threatening or irreversibly debilitating diseases or conditions.
Indeed, the Center for Devices and Radiological Health (CDRH) and Center for Biologics Evaluation and Research (CBER) are to be commended for crafting a draft guidance document that discusses in detail the rationale behind the Data Development Plan and the approach to benefit-risk for qualified Expedited Access PMA (EAP) devices, rather than simply the eligibility criteria and benefits of the EAP program, itself. There are several areas, however, for which BIO believes additional elaboration by the Agency would add value to the Draft Guidance document and, ultimately, the EAP Program.