BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Slowly Progressive, Low-Prevalence Rare Diseases with Substrate Deposition that Results from Single Enzyme Defects: Providing Evidence of Effectiveness for Replacement or Corrective Therapies. It’s important to support and advance drug development for individuals with rare diseases. BIO suggests the FDA eliminate the phrase “low-prevalence rare disease” from the guidance.

BIO submitted comments for the Food and Drug Administration (FDA) public hearing on Facilitating Competition and Innovation in the Biological Products Marketplace, which took place on September 4, 2018. BIO remarked on the need to foster a robust, competitive marketplace for biological products to improve patient access to safe, effective, and affordable therapies. BIO unequivocally believes that safe and effective biosimilars and interchangeable products are good for patients and good for public health. BIO's detailed comments cover topics as they relate to the biosimilars market including interchangeability, analysis of analytical similarity, patented and unpatented uses,  regulatory exclusivity, and the Purple Book.  

Dear Chairman Roberts, Chairman Conaway, Ranking Member Stabenow, and Ranking Member Peterson:As representatives of the U.S. food and agricultural value chain and members of the Coalition for Safe, Affordable Food (the Coalition), we write to express our support for Section 7608 in the House-passed H.R. 2, the Agriculture Improvement Act of 2018, regarding public education on agricultural biotechnology. We believe these provisions will support the U.S. Department of Agriculture (USDA) and other relevant federal agencies in providing consumers with education materials on the National Bioengineered Food Disclosure Law (NBFDL) and its implementing regulations – the National Bioengineered Food Disclosure Standard (NBFDS). Such education materials can assist in explaining how the NBFDS provides consumers with information on the ingredients of the foods they eat, one overriding purpose of the NBFDL...

Re: Docket No. FDA-2018-D-1895: Indications and Usage Section of Labeling for Human Prescription Drug and Biological Products —Content and FormatDear Sir/Madam:The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments to the Draft Guidance titled Indications and Usage Section of Labeling for Human Prescription Drug and Biological Products —Content and Format.

BIO represents an industry that is devoted to discovering new treatments and ensuring patient access to them. Accordingly, we closely monitor changes to Medicare’s reimbursement rates and payment policies for their potential impact on innovation and patient access to drugs and biologicals.As a threshold matter, we raise our concern with the implementation of a policy that can have such impacts in limiting access for beneficiaries in MA via the application of step therapy, as detailed in the Administration’s August 7th memo.2 As detailed in our letter to the Administration, we are particularly concerned with the lack of specificity or detail in this memo around critical patient protections and appropriate implementation, including ensuring: sufficient oversight by CMS; clear clinical criteria for step therapy policies; transparency into, and communication of, step therapy policies to beneficiaries and robust beneficiary protections; timely exceptions and appeals processes; sufficient protections for those on existing therapies; and protection for beneficiaries from higher cost-sharing.

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Patient-Focused Drug Development: Collecting Comprehensive and Representative Input. In the comments, BIO applauds the FDA’s initiative to advance the use of patient-experience data (PED) to inform drug development and regulatory review. BIO sees this first draft guidance, addressing comprehensive and representative input, as an important tool for strengthening our collective understanding of the evidence needed to support the FDA regulatory process with PED. BIO says the guidance presents standards that adhere to established standards for good research practice and provides enough information to gain insight into what is needed to compose an appropriate research study, but BIO strongly believes future guidance documents should emphasize the FDA’s willingness to exercise regulatory flexibility and acceptance of innovative designs and approaches for collecting PED. BIO’s comments provide suggestions for changes to the guidance to encourage broader adoption of PED.