February 3, 2020 Dear Sir/Madam: The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA or Agency) for the opportunity to submit comments regarding FDA’s public workshop on Patient-Focused Drug Development Guidance: Incorporating Clinical Outcome Assessments into Endpoints for Regulatory Decision Making. BIO appreciates FDA’s efforts to hold public workshops and develop a series of guidance documents to assist reviewers, drug developers, patient organizations, and other stakeholders with the collection, analysis, and use of patient experience data (PED) for drug development and regulatory decision-making. In general, BIO believes that guidance documents 1-3, and now the discussion guide for Guidance 4, facilitate the advancement and use of systematic approaches to collect meaningful patient and caregiver input that could better inform medical product development and regulatory decision-making. BIO believes that the discussion guide for guidance 4 is clear and concise, includes several instructive examples, and complements and builds upon concepts included in Guidance 3. However, there are several technical topics that would benefit from additional detail and/or case examples in the Discussion Guide for Guidance 4. We have tried to specify what these areas are and where case examples would be helpful. Additionally, across all PFDD guidance and discussion documents there are many connections and interdependencies; BIO believes that the guidance series would greatly benefit from additional cross-referencing between guidance documents. Because this Discussion Guide will inform the 4th guidance in the series, in addition to providing feedback on Discussion Guide for Guidance 4, we have also provided feedback that may not necessarily need to be addressed in Guidance 4 but that we strongly feel should be addressed in the guidance series so as to best encourage the collection and use of PED for drug development and regulatory…

Section 182 of the Trade Act of 1974, known as the “Special 301” provisions, requires the United States Trade Representative to identify countries that deny adequate and effective intellectual property protections or fair and equitable market access to U.S. persons who rely on IP protection. As part of the 2020 Special 301 Review process conducted by USTR, Justin Pine prepared BIO’s formal submission and presented oral testimony to USTR and the interagency Special 301 subcommittee highlighting the IP issues facing our members in key markets around the world.