PFDD: BIO Comments on FDA Public Workshop on Patient-Focused Drug Development: Guidance 1- Collecting Comprehensive and Representative Input

Re: Docket No. FDA–2017-N-5896: Public Workshop: Patient-Focused Drug Development: Guidance 1- Collecting Comprehensive and Representative Input

Dear Sir/Madam:

The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments regarding Patient-Focused Drug Development: Guidance 1- Collecting Comprehensive and Representative Input.

BIO is the world's largest trade association representing biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. BIO members are involved in the research and development of innovative healthcare, agricultural, industrial, and environmental biotechnology products.

BIO appreciates the FDA’s initiative to hold the public workshop “Patient-Focused Drug Development: Guidance 1 – Collecting Comprehensive and Representative Input,” on December 18, 2017. We also commend FDA’s efforts to develop and make available the discussion document (and accompanying appendices and glossary) to stakeholders to provide a basis for discussion that will inform the development of guidance to facilitate collection and submission of usable patient experience data (PED) for medical product development and regulatory decision-making. The discussion document, including the appendices and glossary, are informative and serve as a basis for drafting the additional planned guidance documents. We also appreciate FDA’s willingness to be flexible and open to innovative methods and practices, as the science is evolving every day and new ideas and approaches will continue to emerge.

While BIO also agrees that the upcoming guidance documents should be complementary to the FDA guidance on Patient Reported Outcomes (PRO), we believe that the PRO guidance is too restrictive and thus has not led to an increase in qualified or validated PRO tools for use in labeling. To this end, we ask the FDA to consider some of the broad implementation challenges that make the PRO guidance more restrictive, in order to keep the PFDD guidance documents broadly applicable and flexible, and maximize their utility. BIO strongly believes that in order for a broad adoption of PED, the upcoming guidance documents should emphasize the FDA’s willingness to exercise flexibility and acceptance of innovative designs and approaches in collecting such data. We have included additional comments below for the FDA’s consideration as they continue to develop the patient-focused drug development (PFDD) guidance documents.