One area where the guideline is of particular relevance to BIO, and is thus appreciated, is in establishing both clarity and flexibility around the need and value of studies conducted in nonhuman primates. The language in Section 3.3 that acknowledges the limited value of dedicated studies in postweaning juvenile NHP is supported, as is the final paragraph in Section 4 which specifically addresses the situation of “pediatric first” clinical development in neonates.

Section 182 of the Trade Act of 1974, known as the “Special 301” provisions, requires the United States Trade Representative to identify countries that deny adequate and effective intellectual property protections or fair and equitable market access to U.S. persons who rely on IP protection. As part of the 2019 Special 301 Review process conducted by USTR, Justin Pine prepared BIO’s formal submission and presented oral testimony to USTR and the interagency Special 301 subcommittee highlighting the IP issues facing our members in key markets around the world. The principal concerns highlighted in BIO’s submission are: i) global compulsory licensing trends; ii) lack of adequate or any regulatory data protection; iii) restrictive patentability frameworks; and iv) market access concerns associated with drug pricing controls. 

The Biotechnology Innovation Organization (BIO) responded to the SEC’s request for comments on the nature and timing of disclosures that reporting companies are required to provide in their quarterly reports. BIO believes that the current quarterly reporting framework places an unhealthy emphasis on meeting or exceeding short-term forecasts, which engenders an inefficient outlook on short-term results. Due to the lengthy timeline for potentially life-saving drug discovery, which averages 10-15 years, biotech companies and their investors would be better served by a less frequent (e.g. semiannual) reporting regime that prioritizes long-term value creation. Biotech companies have unique business models and would benefit from scaled disclosure whereby smaller reporting companies (SRCs) and emerging growth companies (EGCs) report on a less frequent (e.g. semiannual) basis while preserving the flexibility to adopt more frequent (e.g. quarterly) reporting as they advance toward commercial stage.

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data. In the comments, BIO thanks FDA for promoting partnership between patients, industry, and the FDA by providing patient groups and other stakeholders with a pathway to submit proposed draft guidance and other resources related to patient experience data. BIO encourages FDA to continue to facilitate the open exchange on patient experience data with all stakeholders, which will lead to more meaningful outcomes. BIO believes strong trilateral communication between the FDA, patients/patient organizations, and Sponsors will help better incorporate patient experience data throughout the product lifecycle, including drug development and review. BIO provides recommendations on how the FDA can better support this trilateral communication, such as the process by which FDA will communicate and provide feedback to stakeholders who submit draft guidance or other data, when dockets are open for stakeholder comments, and how FDA staff will engage with stakeholders around patient experience data.

On January 25, BIO submitted comments in response to the Centers for Medicare and Medicaid Services' (CMS') Modernizing Part D and Medicare Advantage to Lower Drug Prices and Reduce Out-of-Pocket Expenses Proposed Rule. BIO is extremely concerned to see the Agency continue to focus on policy changes that erode the value of the Medicare benefit for seniors, placing patient access at risk. This Proposed Rule is yet another in a series of proposals that prioritize cost containment policies over patient healthcare needs. BIO respectfully requests that the Agency and Administration instead focus on holistic solutions that balance the financial sustainability of the Medicare program, while addressing patient out-of-pocket cost and access to appropriate treatment. Our concerns, detailed further in the balance of this letter, focus on the following: I. CMS should not allow plans to restrict access in the Medicare Part D six protected classes, as plans already have flexibility to manage these drugs in a clinically appropriate manner. II. CMS must go further in protecting beneficiaries enrolled in Medicare Advantage who may be subject to step therapy for their Part B drugs. III. CMS should not move forward with the inclusion of drug pricing information in the explanation of benefits, and instead focus on the updates to e-Prescribing standard to appropriately share cost information. IV. CMS must continue to work to ensure that patients see the benefit of negotiated prices reflected in their out-of-pocket (OOP) costs. V. CMS should move forward with the proposed requirements prohibiting gag clauses.

BIO supports CMS’ 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 is an outdated approach that presents risks for vulnerable beneficiaries and should be re-evaluated; Coinsurance 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; 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; CMS must go further in protecting beneficiaries enrolled in Medicare Advantage who may be subject to step therapy; CMS should mitigate the impact of the increase in the out-of-pocket (OOP) threshold for CY 2020; MA plans should have the flexibility to determine what constitutes a chronic condition for purposes of providing special supplemental benefits for the chronically ill; Additional flexibility in design of maximum OOP costs should be considered to assist beneficiaries; Drug tier labels should accurately reflect the tier’s composition; Inclusion of additional measures in the Star Ratings are critical to accurate assessment of patient care and quality; Access to innovative treatment options for pain and addiction should be prioritized as a part of addressing opioid overutilization in MA and Part D plans.