BIO appreciates the opportunity to comment on these proposed revisions. Patients living with rare diseases often experience significant unmet medical need due to the lack of knowledge about how these diseases are caused or inherited and their progression. That those suffering from rare diseases are predominately children raises issues around how society prioritizes and develops treatments for these conditions. We believe there are significant challenges in reconciling existing population-level value assessment methodologies with the varied healthcare contexts and deeply personal patient-level treatment decisions faced by patients afflicted with rare diseases, their families, and their clinicians. For a number of reasons, applying a patient-centric lens when considering the value of treatment is especially important when considering rare diseases.

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Format and Content of a Risk Evaluation and Mitigation Strategy Document. The draft guidance provides recommendations to applicants on drafting proposed Risk Evaluation and Mitigation Strategy (REMS) documents and converting an already-approved REMS document to a new standardized format. BIO provides suggestions for how to improve the guidance regarding REMS requirements for programs run or administered through specialty pharmacy healthcare settings. In addition, BIO asks the FDA to specify details on storage and retention of participant documents, monitoring of patients, and the duration of retention of participant materials.

BIO submitted comments on Existing General Regulatory and Information Collection Requirements of the Food and Drug Administration (FDA). BIO is committed to promoting a regulatory environment that continues to support and incentivize the biopharmaceutical ecosystem. Minimizing the regulatory burden on the biopharmaceutical sector will foster small business growth, create good, high-paying jobs, directly and indirectly expand the economy, and expedite the development and delivery of safe and effective therapies to patients. BIO developed a priority list of regulations and guidance documents that, if modified or eliminated, would significantly decrease regulatory costs, advance the fast and safe development and delivery of effective therapies, and drive investment in innovation by fostering an environment that recognizes the value these new medicines bring to patients and the overall healthcare system. These documents fall into three categories: Accelerating Products to Market & Reducing Pre-Market Regulatory Costs, Lowering Post-Market Regulatory Compliance Costs, and Removing Regulatory Barriers that Impede Value-Based Arrangements.

The Biotechnology Innovation Organization ("BIO") welcomes the opportunity to submit  comments on the Food and Drug Administration (FDA) draft guidance titled "Considerations in Demonstrating Interchangeability With a Reference Product" issued on January 18, 2017 ("Draft Guidance").