September 22, 2020 Re: Docket No. FDA-2019-N-5553: Annual Summary Reporting Requirements Under the Right to Try Act, Proposed Rule 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 Proposed Rule Annual Summary Reporting Requirements Under the Right to Try Act (Proposed Rule). 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 believes that the Proposed Rule provides important information for Sponsors to follow regarding the reporting requirements under the Right to Try Act. We appreciate FDA’s consideration of implementing the concept of “least burdensome” in its approach. An alternate approach to the separate reporting process as articulated in the Proposed Rule is to align the Right to Try Act reporting with annual reporting for Expanded Access. Creating two separate annual reporting pathways increases the burden on Sponsors and manufacturers responding to these requests. We suggest that in the aligned annual reporting, there can be two sections within the annual report, one for Right to Try and one for Expanded Access for FDA to be able to differentiate between the two. In addition, we suggest that serious adverse event (SAE) reporting should also be aligned with the standard SAE reporting that Sponsors and manufacturers already perform, again, to create a least burdensome approach to these reporting requirements. Further, we believe that the example given in the Proposed Rule of a tabular summary goes beyond the level of information required by the legislation which cites “the…

September 27, 2020 Re: Docket No. FDA-2014-D-1551:  FDA Draft Guidance, Pregnancy, Lactation, and Reproductive Potential:  Labeling for Human Prescription Drug and Biological Products – Content and Format. Dear Sir/Madam: The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA or Agency) for the opportunity to submit comments regarding the Draft Guidance, Pregnancy, Lactation, and Reproductive Potential:  Labeling for Human Prescription Drug and Biological Products – Content and Format. 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 Agency’s inclusion of the pregnancy registry and the risk summary in the label to ensure awareness and encourage Health Care Provider (HCP)-patient conversations.  BIO has two overarching comments on this guidance.  Firstly, it is not clear from the guidance whether sponsors can utilize the pregnancy and lactation data as supportive safety data generated from other drugs that are in the same drug class or possess the same drug molecular structure with representative justification.  It would be helpful if the Agency could provide clarification as to whether this data would be accepted to demonstrate safety for pregnant and lactating women.  Secondly, since pregnancy studies and lactation studies are rarely conducted pre-approval and given the Agency’s prioritization of initiatives focused on use of real world evidence, it would be helpful if the Agency could provide recommendations on utilizing post-marketing real world data to support labeling updates for pregnancy and lactation sections.  BIO has included in this…