BIO supports CMS’s commitment to improving the quality and delivery of care in the MA and Part D programs. We believe it is critically important to ensure that policies in these programs advance patient access to timely and appropriate treatment, particularly for prescription drugs and vaccines. To that end, we provide comments in the following areas:The specialty tier eligibility cost threshold should be increased for 2019 and in future years and the exceptions process expanded to ensure that the specialty tier does not discriminate against vulnerable beneficiaries;  Co-insurance in the Part D non-preferred drug tier can unduly limit access to care for patients with severe and complex diseases;  Increased enforcement of nondiscrimination should be used when evaluating benefit design in the MA and Part D programs;  Access to innovative treatment options for pain and addiction should be prioritized as a part of addressing opioid overutilization in MA Part D plans;  MA and Part D plans should prioritize and increase access to vaccinations for Medicare beneficiaries;  The timeframes and processes for formulary updates should support the inclusion of new therapies;  Drug tier labels should be accurately reflective of the tier’s composition.  The inclusion of prescription drug costs in Medicare advantage uniformity flexibility should provide patient access to the most appropriate treatment;  Additional flexibility in design of maximum out-of-pocket costs should be considered to assist beneficiaries;  The availability of suitable plan offerings should be increased through removal of the meaningful difference requirements;      Additions to the Star Ratings are critical to accurate assessment of patient care quality; and  Efforts to expand coverage for certain subsets of products MA-PD plans should focus across all drugs offered under the Part D program.

BIO submitted comments after the Food and Drug Administration’s (FDA) public hearings on Patient-Focused Drug Development: Guidance 1 – Collecting Comprehensive and Representative Input. BIO commends the FDA’s efforts to facilitate the use of patient experience data (PED) for medical product development and regulatory decision-making. Flexibility and openness to innovative methods and practices will be important as new ideas and approaches emerge every day. While BIO agrees the patient-focused drug development (PFDD) guidance documents should complement the FDA guidance on patient-reported outcomes (PRO), the PRO guidance is too restrictive and has not led to an increase in qualified or validated PRO tools for use in labeling. BIO asks FDA to keep the PFDD guidance documents broadly applicable and flexible. BIO’s comments include answers to the FDA’s questions and suggestions on how to clarify and improve the PFDD guidance.

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases. The Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) provide new pediatric information in labeling for over 600 products. BIO appreciates the FDA’s efforts to clarify and provide notice of a policy change intended to enable the FDA to apply the Orphan Drug Act (ODA) and PREA to non-rare adult indications corresponding to orphan-designated pediatric subpopulations. However, BIO’s comments detail general concerns with the guidance and specific line edits to clarify and improve the guidance.  

As the U.S. EPA begins its work to develop the proposed 2019 Renewable Fuel Standard (RFS) Renewable Volume Obligation (RVO), a group of trade association advocates encourage the agency to take a renewed look at commercially ready cellulosic biofuels.

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Expedited Programs for Regenerative Medicine Therapies for Serious Conditions. This draft guidance provides important information on the expedited programs available to sponsors of regenerative medicine therapies for serious conditions. BIO recommends the FDA provide further guidance to sponsors on which designation (RMAT or Breakthrough Therapy) is most appropriate for different types of programs, along with other comments on how to clarify the guidance.  

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease. BIO welcomes the FDA’s efforts to provide clear, concise guidance on the regulatory and scientific framework for product developers of targeted treatments. This will help sponsors with clinical trial development, including the types/levels of evidence to support a grouping strategy for clinical trial eligibility and benefit-risk evaluation approaches within a disease exhibiting differences in molecular subset. BIO encourages the FDA to continue to gather stakeholder input on the development of targeted therapies to ensure the guidance keeps pace with scientific advances. BIO’s comments include additional suggestions to clarify the guidance, the scope, and FDA’s receptivity to innovative approaches for the development of targeted therapies.