BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Pediatric Rare Diseases – A Collaborative Approach for Drug Development Using Gaucher Disease as a Model. BIO appreciates the FDA’s intended goal with the guidance – to reduce the total number of children needed for enrollment in clinical studies – and applauds the agency’s willingness to accept innovative trial designs to limit the number of patients in the control arm and thereby improve trial efficiency. However, BIO has concerns with the guidance, particularly with the FDA’s suggestion for the use of multi-arm, multi-company clinical studies, which have inherent limitations and challenges. In the comments, BIO details these concerns and provides suggestions for clarifying and improving the guidance.

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Grandfathering Policy for Packages and Homogenous Cases of Product Without a Product Identifier. BIO and its member companies are committed to meeting the requirements of the Drug Supply Chain Security Act (DSCSA) and ensuring the U.S. drug supply chain is secure, patients are receiving authentic products, and legitimate medications continue to move without unnecessary delays. The use of the package date for determining whether an un-serialized product would be grandfathered is critical to a smooth transition under DSCSA from un-serialized to serialized inventory. It’s important these products can be sold and distributed until the product has reached its expiration date. BIO thanks the FDA for listening to stakeholder concerns on the implementation of the grandfathering provisions of the DSCSA. This draft guidance will help ensure legitimate product can move through the supply chain and help negate potential issues as stakeholders begin implementing the serialization requirements.

BIO strongly supports CMS’s commitment to improving the quality of the Medicare Advantage (MA) and Part D programs. We consider it especially important to focus on policies that impact access to prescription drugs and biologicals for Medicare beneficiaries in MA and Part D plans. To further improve access to crucial therapies and immunizations for these patients, we urge CMS to consider the following comments, discussed in more detail below: Cost-sharing in the Part D specialty tier can unduly burden patients with severe, complex diseases and is exacerbated by the fact that the dollar-per-month threshold on the specialty tier allows a broad range of therapies to be included. While we appreciated the effort in 2017 toward increasing the threshold, BIO urges CMS to continue to substantially increase the threshold for 2018 and beyond. BIO also urges CMS to review the specialty tiers to ensure they do not discourage enrollment by certain Part D-eligible individuals. CMS should continue to seek information on the Part D tiering exceptions process and ensure that tiering structures do not impede beneficiary access to medically necessary and appropriate treatments. CMS should ensure that the timeframe and process for formulary updates reasonably allows for the addition of new therapies. CMS should work to make Medicare Plan Finder inclusive of the most up to date formulary information to best inform beneficiary prescription drug coverage choice. CMS should finalize the proposal to include a new display measure around the adjudication process and should continue efforts to further increase transparency around this process to provide patients with appropriate access to needed medicines. CMS should evaluate access to specialty pharmacies in considering network adequacy and patient access issues. CMS should continue to encourage increased beneficiary vaccination rates and ensure MA plans deem vaccinations provided by pharmacists, in accordance with state laws, as in-network providers for these services. CMS should finalize the movement of the High Risk Medication (HRM) measure from the Star Ratings to the display measures. CMS should ensure that innovative model testing in the Medicare program works to maintain or improve beneficiary access to appropriate care and treatment. CMS should look to include display measures consistent with the goals of the National Action Plan for Adverse Drug Event Prevention in future years. CMS should finalize the clarification around "reference-based pricing"2 in Part D beneficiary cost-sharing arrangements. CMS should look to further ensure market stabilization for the dually eligible population in Puerto Rico.

BIO supports CMS’s efforts to make changes to the Medicare Advantage, Medicare Fee-for-Service and the Part D Prescription Drug Benefit Programs in a manner that improves overall healthcare quality, while not compromising access to the most appropriate course of treatment. Our comments, detailed further in the balance of this letter, focus on the following areas: Request for Information to Require Pass through of Manufacturer Rebates at the Point of Sale to the Beneficiary Implementation of the Comprehensive Addiction and Recovery Act of 2016 (CARA) Treatment of Follow-On Biological Products as Generics for Non-LIS Catastrophic and LIS Cost-Sharing Revisions to Part D Tiering Exceptions Expedited Substitutions of Certain Generics and Other Midyear Formulary Changes Maximum Out-of-Pocket Limit and Cost-Sharing Limits for Medicare Parts A and B Services Flexibility in the Medicare Advantage Uniformity Requirements Any Willing Pharmacy Standards Terms and Conditions Medicare Advantage and Part D Prescription Drug Program Quality Rating System MA/Part D Artificial Limits