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 The draft guidance provides an overview of the types of clinical studies that may be performed and data sources during the product's life cycle.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 The draft guidance uses the traditional framework for the logical progression of drug development in phases (Phase 1-4).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.

September 25, 2019Re: FDA-2019-D-1469: M10 Bioanalytical Method Validation; International Council for Harmonisation; Draft Guidance for IndustryDear Sir/Madam:The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments on the International Council for Harmonisation (ICH) Draft Guidance for Industry “M10 Bioanalytical Method Validation” (Draft Guidance or Guidance).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 believes that the scope of the Guidance is too broad and can be edited for specificity and clarity; please see our detailed line edits for lines 102-111 for our specific suggestion. Additionally, we note the Guidance covers method development for method validation. This raises significant concerns regarding expectations from different regions. We recommend re-wording for clarification or limiting the method development scope to changes to a validated method. Further, BIO believes that if this Guidance also applies to life cycle maintenance of products, the text should be updated to explicitly reference this, see specific comments throughout the accompanying chart.BIO has a few suggestions regarding use of terminology throughout the Guidance. First, BIO suggests avoiding the term “pivotal” throughout the document, which may cause confusion if understood differently across ICH membership or regions. We have noted specific line numbers of its usage in the following chart. Second, BIO suggests that throughout the Guidance either the term “dilution ratio” or “dilution factor” be used consistently, rather than interchangeably.Finally, a section discussing criteria to be…

We are writing on behalf of the Biotechnology Innovation Organization (BIO) to provide comments on the Institute for Clinical and Economic Review’s (ICER’s) solicitation for input on draft revisions to its Value Assessment Framework for the assessment of “single or short-term transformative therapies” (SSTs). ... We appreciate ICER’s recognition that the burgeoning field of transformative and curative therapies requires serious discussion around how these treatments are valued by not just payors, but society at large. These therapies have the potential to fundamentally change how we view the treatment of disease. Yet as we strive to shift our health care system to one that rewards valuable care, BIO is concerned that the science of value assessment for all therapies – not just those that are curative or transformational – is woefully behind where it should be for these tools to be used in a substantive way.

BIO's comments on the EPA’s administration of the of the Renewable Fuel Standard (RFS)  program and how it has put the investments made by BIO Member companies at risk and threatens to prevent growth in advanced and cellulosic biofuels as the RFS intended. EPA’s expansion of granting small refinery exemptions (SRE) retroactively after setting the annual percentage standard ensures that the proposed annual volume obligations will not be met with liquid biofuels, contrary to the statute and congressional intent.

August 25, 2019 Re: Docket No. FDA–2019-N-2012: New Drug Regulatory Program Modernization: Improving Approval Package Documentation and Communication. Dear Sir/Madam: The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments regarding the New Drug Regulatory Modernization: Improving Approval Package Documentation and Communication. 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 goal of providing greater clarity on FDA's application review and decision-making process. We commend FDA’s efforts to modernize the New Drug Regulatory Program through the use of: (1) The Clinical Data Summary Pilot Program (Pilot), through which parts of a Sponsor’s clinical study reports (CSRs) are posted and (2) a new integrated template that will be used to document FDA's review of new drug applications and efficacy supplements. BIO commends the Agency’s efforts to improve transparency in the drug approval process and believes increased transparency will ultimately benefit patients, BIO member companies themselves make strong efforts to support clinical trial transparency by posting protocols and results and sharing data consistent with the Final Rule on Clinical Trial Registration and Results Information Submission. BIO strongly supports the new integrated review process and template and agrees that this format will also clarify and improve communication between FDA and the Sponsor, as well as provide new insights regarding FDA’s regulatory decision making. The FDA has stated it is considering where to focus its efforts to increase transparency around the drug…