January 7, 2020 Re: Docket No. FDA–2019-N-3453: Promoting Effective Drug Development Programs: Opportunities and Priorities for the Food and Drug Administration's Office of New Drugs Dear Sir/Madam: The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments following the FDA public meeting on Promoting Effective Drug Development Programs: Opportunities and Priorities for the Food and Drug Administration's Office of New Drugs. 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’s members develop medical products and technologies to treat patients afflicted with serious diseases, to delay the onset of these diseases, or to prevent them in the first place. BIO appreciates the Agency’s efforts to seek input from stakeholders on actionable policy suggestions that could be implemented in the near-term by the staff of the Center for Drug Evaluation and Research's (CDER's) Office of New Drugs (OND) to promote effective drug development programs without compromising regulatory standards for the assessment of safety and effectiveness. It is through discussions such as these that various stakeholders can work together to identify challenges and barriers as well as potential solutions for making drug development and review more efficient, with the shared goal of bringing safe, effective, and high-quality medicines to patients in a timely manner. BIO has included in this letter several specific recommendations for CDER on how to promote effective drug development programs. We note that the FDA has indicated that  topics such as real world evidence (RWE) and patient focused drug development (PFDD) are out of scope for the purposes of the public meeting and docket response, however, both of these topics have an integral role…

January 4, 2020 Re: Docket No. FDA–2019-N-5157: 2019 Public Meeting on Center for Drug Evaluation and Research Standard Core Sets: Clinical Outcome Assessments and Endpoints Grant Program. Dear Sir/Madam: The Biotechnology Innovation Organization (BIO) thanks Food and Drug Administration (FDA or Agency) for the opportunity to submit comments following the 2019 Public Meeting on Standard Core Sets: Clinical Outcome Assessments (COAs) and Endpoints Grant Program. 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 commends FDA for its work to coordinate a pilot grant program with the goal of developing publicly available standard core set(s) of COAs and their related endpoints for specific disease indications. BIO appreciates that FDA held the public meeting on December 5, 2019 and sought feedback from various stakeholders through the docket following the public meeting.  BIO provides additional comments regarding considerations for rigorously developed, fit-for-purpose core COAs and active engagement of patients throughout core set development; development of COAs and endpoints to assess physical/functional status; transparency and communication among stakeholders; global use of COAs; evaluating exiting tools; maintenance of COAs; and selection of disease areas to develop COAs in the future.