Dear Administrator Verma:The Biotechnology Innovation Organization (BIO) appreciates the opportunity to commenton the Center for Medicare and Medicaid Services’ (CMS’s) CY2020 Revisions to PaymentPolicies under the Physician Fee Schedule (PFS) and Other Changes to Part B PaymentPolicies (“Proposed Rule”).BIO is the world’s largest trade association representing biotechnology companies, academicinstitutions, state biotechnology centers and related organizations across the United Statesand in more than 30 other nations. BIO’s members develop medical products andtechnologies to treat patients afflicted with serious diseases, to delay the onset of thesediseases, or to prevent them in the first place. In that way, our members’ noveltherapeutics, vaccines, and diagnostics not only have improved health outcomes, but alsohave reduced healthcare expenditures due to fewer physician office visits, hospitalizations,and surgical interventions. BIO membership includes biologics and vaccine manufacturersand developers who have worked closely with stakeholders across the spectrum, includingthe public health and advocacy communities, to support policies that help ensure access toinnovative and life-saving medicines and vaccines for all individuals.We offer comments on the following areas in the proposed rule, with an emphasis onensuring that Medicare beneficiaries have access to the full range of items and servicesnecessary to their health:• BIO appreciates the opportunity to comment on the agency’s future consideration ofbundled payments under the PFS but urges CMS to prioritize patient access toappropriate therapies and to weigh the results of other demonstrations underwayrather than attempting to swiftly implement bundled payments.• BIO supports the proposal to retain more granular payments for Evaluation andManagement (E/M) services, which we think will support access to appropriate carefor the sickest patients.​

Dear Administrator Verma,The Biotechnology Innovation Organization (BIO) appreciates the opportunity to comment onthe Centers for Medicare & Medicaid Services’ (CMS’) Medicare Hospital OutpatientProspective Payment and Ambulatory Surgical Center Payment Systems Proposed Rule.BIO is the world's largest trade association representing biotechnology companies, academicinstitutions, state biotechnology centers and related organizations across the United Statesand in more than 30 other nations. BIO’s members develop medical products andtechnologies to treat patients afflicted with serious diseases, to delay the onset of thesediseases, or to prevent them in the first place. In that way, our members’ noveltherapeutics, vaccines, and diagnostics not only have improved health outcomes, but alsohave reduced healthcare expenditures due to fewer physician office visits, hospitalizations,and surgical interventions. BIO membership includes biologics and vaccine manufacturersand developers who have worked closely with stakeholders across the spectrum, includingthe public health and advocacy communities, to support policies that help ensure access toinnovative and life-saving medicines and vaccines for all individuals.BIO represents an industry that is devoted to discovering new treatments and ensuringpatient access to them. Accordingly, we closely monitor changes to Medicare’sreimbursement rates and payment policies for their potential impact on innovation andpatient access to drugs and biologicals. Our comments on the Proposed Rule are outlinedbelow

September 30, 2019Re: Docket No. FDA–2014-D-1461: FDA Draft Guidance, Rare Pediatric Priority Review Vouchers.Dear Sir/Madam:The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA or Agency) for the opportunity to submit comments in response to the FDA’s Draft Guidance on Rare Pediatric Disease Priority Review Vouchers.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 people afflicted with serious diseases, to delay the onset of these diseases, or to prevent them in the first place.BIO commends the Agency’s efforts to provide guidance on the implementation of Section 908 of the Food and Drug Administration Safety and Innovation Act (FDASIA), the Rare Pediatric Disease Priority Review Voucher (RPD PRV) Program. The Rare Pediatric Disease Priority Review Voucher Program, along with other incentives, is essential for encouraging investment and research into development for therapies to treat pediatric patients with rare diseases.As FDA works to improve and streamline the implementation of the RPD PRV process, we recommend that the Agency consider a common application for Orphan Designation and the Rare Pediatric Diseases Designation and streamlining the process for transfer and use of the PRV. We believe that our recommendations not only provide clarity regarding some of the elements in the Draft Guidance but also serve to continue to incentivize the development of therapies for rare pediatric diseases, as intended by Congress.PRVs are earned or purchased by a sponsor to be redeemed, along with a PRV fee, for Priority Review of an application. As such, we recommend that a voucher and fee would only be needed in a circumstance where the application would not receive a Priority Review based on its own…

September 30, 2019Re: Docket ID FDA-2019-D-3049: E8(R1) General Considerations for Clinical Studies; International Council for HarmonisationDear Sir/Madam:The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments on E8(R1) General Considerations for Clinical Studies; International Council for Harmonization.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 welcomes this document on internationally accepted principles and practices for the design and conduct of clinical studies of drug and biologic products. The draft guidance provides an overview of the types of clinical studies that may be performed and data sources during the product's life cycle. Although we believe the guidance is well written, we have provided general comments below for FDA’s consideration, as well as detailed comments in the annexed table. The draft guidance uses the traditional framework for the logical progression of drug development in phases (Phase 1-4). BIO recommends that this document resets the framework for how drug development is approached. Scientific methodologies have evolved. There are a multitude of methodologies (e.g., adaptive designs, master protocols, platform trials, observational research, pragmatic research) that can be used to provide evidentiality for drug development. For example, adaptive designs can be used in Phase 1, which is not reflected in the document. BIO recommends that the document lessen the focus on Phase 1-4, which provides appearance of operating in the old paradigm, and instead focus on answering the research questions using appropriate methods at the suitable…

BIO submitted comments to the Food and Drug Administration (FDA) on E19 Optimisation of Safety Data Collection; International Council for Harmonisation. BIO welcomes this document on the optimization of safety data collection and supports the goal of internationally harmonized guidance on when it’s appropriate to use a selective approach to safety data collection in some late-stage pre-marketing or post-marketing studies, and how such an approach would be implemented. BIO provides general and detailed comments for FDA’s consideration.