Below is Alston & Bird’s Healthcare Week in Review, which provides a synopsis of the latest news in healthcare regulations, notices, and guidance; federal legislation and congressional committee action; reports, studies, and analyses; and other health policy news.

Week in Review Highlight of the Week:

On October 17, 2019, three House committees took up H.R. 3, the Lower Drug Costs Now Act of 2019, in hearings and markups. Read more about this Congressional activity below.

I. Regulations, Notices, & Guidance

• On October 15, 2019, the Food and Drug Administration (FDA) issued guidance entitled, Submitting Study Datasets for Vaccines to the Office of Vaccines Research and Review. This technical specification document provides detailed information and specifications for sponsors/applicants regarding the content of datasets submitted to FDA’s Center for Biologics Evaluation and Research (CBER) Office of Vaccines Research and Review (OVRR) and is designed to aid clinical and statistical reviewers in the review of vaccine applications (e.g., biologics license applications). FDA recommends submission of these datasets as part of the applicant’s data tabulation datasets. The submission of standardized datasets facilitates review and analyses of the data and allows for pooling of data, when appropriate. These specifications reflect current CBER OVRR thinking, are built to be consistent with the FDA Study Data Technical Conformance Guide, and are generally consistent with the Therapeutic Area User Guide (TAUG) for Vaccines.
• On October 16, 2019, FDA issued guidance entitled, Prescription Drug User Fee Act Waivers, Reductions, and Refunds for Drug and Biological Products Guidance for Industry. This guidance provides recommendations to applicants planning to request a waiver or reduction in user fees. This revised guidance describes the types of waivers, refunds, and reductions available under the user fee provisions of the FD&C Act; the procedures for requesting waivers, refunds, or reductions; and the process for requesting a reconsideration or appeal of an FDA decision. The guidance also provides clarification on related issues such as user fee exemptions for orphan drugs.
• On October 18, 2019, FDA issued guidance entitled, Drug Master Files Guidance for Industry. Once finalized, this guidance will provide FDA's current thinking on drug master files (DMFs), which are submissions to FDA that may be used to provide confidential, detailed information about facilities, processes, or articles used in the manufacturing, processing, packaging, and storing of human drug products. DMFs are submitted solely at the discretion of their holders and are not required by statute or regulation.

Event Notices         

• October 21, 2019: The Department of Health and Human Services (HHS) announced a public meeting entitled, Advisory Council on Alzheimer’s Research, Care, and Services. During the meeting, the Advisory Council will welcome six new members, and discuss ways to increase access to long-term services and supports that support people with dementia and their caregivers in their homes and the community including innovative state programs. Federal workgroups will also provide updates on work completed in the last quarter, as well as an overview of the 2019 Update to the National Plan to Address Alzheimer’s Disease.
• October 31, 2019: FDA announced a public meeting entitled, International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use Global Meeting on E8(R1) Guideline on General Considerations for Clinical Trials. The purpose of the meeting is to provide information on the draft revised E8(R1) Guideline “General Considerations for Clinical Trials” (ICH E8 Guideline) following the closing of the FDA comment period and closing of the regional consultations conducted in other ICH regions. The ICH E8 Guideline is being revised to provide updated guidance that is both appropriate and flexible enough to address the increasing diversity of clinical trial designs and data sources being employed to support regulatory and other health policy decisions, while retaining the underlying principles of human subject protection and data quality.
• November 4, 2019: FDA announced a public meeting entitled, Health Canada and United States Food and Drug Administration Joint Regional Consultation on the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use. The purpose of the public meeting is to provide information and solicit public input on the current activities of the ICH, as well as the upcoming ICH Assembly Meeting and the Expert Working Group Meetings in Singapore scheduled for November 16 through November 20, 2019.
• November 5-6, 2019: HRSA announced a public meeting entitled, Meeting of the National Advisory Council on Nurse Education and Practice. During meeting, NACNEP will welcome new members to the Council and discuss strategic priorities for nursing education and practice in preparation for the development of the Council’s 17th Report to Congress.
• November 12, 2019: FDA announced a public workshop entitled, Advancing the Development of Pediatric Therapeutics: Pediatric Clinical Trial Endpoints for Rare Diseases with a Focus on Pediatric Patient Perspectives. The purpose of this workshop is to discuss pediatric patient-specific engagement in the development of clinical trial endpoints for rare diseases. In this workshop, FDA will obtain the pediatric patient perspective on their disease/condition and what is most important to consider when designing rare disease trials. There will also be discussion regarding patients’ thoughts on clinical endpoints that are currently being used in clinical trials, potential areas of innovation, and how to create processes that might include pediatric patients and their caregivers as collaborators in endpoint development in early stages of medical product development (e.g., protocol design).
• November 13-14, 2019: FDA announced a public meeting entitled, Immunology Devices Panel of the Medical Devices Advisory Committee. The committee will discuss the topic of immunological responses to metal-containing products regulated as medical devices. The discussion will focus on metal-containing implants as well as dental amalgam.
• November 6-7, 2019: CDC announced a public meeting entitled, Clinical Laboratory Improvement Advisory Committee. The agenda will include agency updates from CDC, CMS, and FDA. Presentations and discussions will focus on a follow up on CLIAC recommendations; an update on the clinical laboratory workforce; improving integration of laboratory information systems with electronic health records; and future CLIAC topics. There will be an extended public comment session focusing on emerging technologies and the clinical laboratory. Agenda items are subject to change as priorities dictate.
• November 7, 2019: FDA announced a public meeting entitled, Promoting Effective Drug Development Programs: Opportunities and Priorities for the Food and Drug Administration's Office of New Drugs. The purpose of the meeting is to solicit specific, actionable policy suggestions that could be implemented in the near-term by the review staff of the Center for Drug Evaluation and Research's Office of New Drugs to promote effective drug development programs without compromising FDA regulatory standards for the assessment of safety and effectiveness.
• November 13, 2019: FDA announced a public meeting entitled, Endocrinologic and Metabolic Drugs Advisory Committee. The committee will discuss supplemental new drug application (sNDA) 204629/S-020 for empagliflozin oral tablet, sponsored by Boehringer Ingelheim Pharmaceuticals, Inc., for the following proposed indication: as an adjunct to insulin therapy to improve glycemic control in adults with type 1 diabetes mellitus.
• November 13-14, 2019: HHS announced a public meeting entitled, Meeting of the CDC/HRSA Advisory Committee on HIV, Viral Hepatitis and STD Prevention and Treatment. The purpose of the meeting is to discuss issues related to the President’s initiative on “Ending the HIV Epidemic: A Plan for America,” building community capacity, strategies for engaging people with HIV who are out of care, models of clinical care, workforce issues, pre-exposure prophylaxis services in community health centers, and pending committee reports.
• November 22, 2019: FDA announced a public meeting entitled, Blood Products Advisory Committee. The committee will meet in open session to discuss scientific considerations for cold stored platelet products intended for transfusion, including product characterization, duration of storage and clinical indications for use. The committee will hear presentations on available characterization and functional studies of cold stored platelets, clinical studies, and the potential role of cold stored platelets in clinical care in military and civilian patient populations. The committee will also discuss the clinical studies needed to support the indications for use of cold stored platelet products stored beyond 3 days.
• December 16-17: CMS announced a town hall meeting entitled, Medicare Program; Town Hall Meeting on the FY 2021 Applications for New Medical Services and Technologies Add-On Payments. CMS is holding this meeting in accordance with section 1886(d)(5)(K)(viii) of the Social Security Act (the Act) to discuss Fiscal Year (FY) 2021 applications for add-on payments for new medical services and technologies under the hospital inpatient prospective payment system . Interested parties are invited to this meeting to present their comments, recommendations, and data regarding whether the FY 2021 new medical services and technologies applications meet the substantial clinical improvement criterion.

II. Congressional Hearings

House of Representatives

• On October 16, 2019, the House Energy and Commerce Subcommittee on Health held a hearing entitled, Legislation to Reverse the Youth Tobacco Epidemic. Witnesses present included: Dorian Fuhrman, Co-founder and parent, Parents Against Vaping e-Cigarettes (PAVe); Phillip Gardiner, Dr.P.H., Senior Program Officer, Tobacco Related Disease Research Program, University of California Office of the President; Matthew Myers, President, Campaign for Tobacco-Free Kids; Michael Siegel, M.D., M.P.H., Professor, Department of Community Health Sciences, Boston University School of Public Health; and Susanne Tanski, M.D., M.P.H., Member, American Academy of Pediatrics (AAP).
• On October 17, 2019, the House Committee on Ways and Means held a hearing entitled, Investing in the U.S. Health System by Lowering Drug Prices, Reducing Out-of-Pocket Costs, and Improving Medicare Benefits. Witnesses present included: Samantha Reid, Patient, Crohn’s Disease; Catherine Alicia Georges EdD, RN, FAAN, National Volunteer President, AARP; Mark E. Miller, PhD, Executive Vice President of Health Care, Arnold Ventures; Judy Feder, PhD, Professor, Georgetown University McCourt School of Public Policy; and Benedic N. Ippolito, PhD, Research Fellow, American Enterprise Institute.
• On October 17, 2019, the House Committee on Education and Labor held a markup of H.R. 3, the Lower Drug Costs Now Act of 2019. H.R. 3, as amended, was favorably forwarded to the House.
• On October 17, 2019, the House Committee on Energy and Commerce held a markup of five health bills. Legislation included: H.R. 3, the Lower Drug Costs Now Act of 2019; H.R. 4665, the Medicare Vision Act of 2019; H.R. 4671, the Help Seniors Afford Health Care Act; H.R. 4618, the Medicare Hearing Act of 2019; and H.R. 4650, the Medicare Dental Act of 2019. All five bills, as amended, were favorably forwarded to the House.

III. Reports, Studies, & Analyses

• On October 17, the Kaiser Family Foundation (KFF) published an issue brief summarizing and comparing the latest policy proposals regarding Medicare drug price negotiations. More specifically, the study begins with a brief description of the statutory prohibition on government drug price negotiations and its history and then describes several legislative proposals introduced in the current Congressional session that would give the HHS Secretary authority to negotiate Medicare drug prices. The brief also reviews analysis from the Congressional Budget Office (CBO) of the potential savings that government negotiations may generate for the Medicare program and its beneficiaries.
• On October 18, KFF published its results from a 50-State Medicaid budget survey for state fiscal years 2019 and 2020. The report provides an in-depth examination of certain policies in place in state Medicaid programs in FY 2019 and policy changes implemented or planned for FY 2020. Key findings from the report are pulled from five sections: eligibility, delivery systems, benefits, long-term services and supports, and provider rates and taxes. Each section highlights key issues to watch for future policy development. The findings are drawn from the 19th annual budget survey of Medicaid officials in all 50 states and the District of Columbia conducted by KFF and Health Management Associates (HMA), in collaboration with the National Association of Medicaid Directors (NAMD).
• On October 18, 2019, the Alston & Bird Health Group released their summary of the CMS final rule entitled, Medicare and Medicaid Programs; Revisions to Requirements for Discharge Planning for Hospitals, Critical Access Hospitals, and Home Health Agencies, and Hospital and Critical Access Hospital Changes to Promote Innovation, Flexibility, and Improvement in Patient Care. The A&B Health Group analyzes the long-awaited final rule on discharge planning, focused on data sharing and patient preferences. The analysis is available here.

IV. Other Health Policy News

• On October 11, the Congressional Budget Office (CBO) released a preliminary estimate of the effects of Title I of H.R. 3, the Lower Drug Costs Now Act of 2019, which would establish a fair price negotiation program. The CBO estimates that the bill would save the federal government around $345 billion over seven years. However, those savings would come at a cost of 8 to 15 fewer drugs being invented over a decade. CBO is continuing to work on analyses of other effects of the bill.

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