Alston & Bird Healthcare Week in Review

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 January 30, the Centers for Medicare & Medicaid Services (CMS) announced its Medicaid block grant initiative called the “Healthy Adult Opportunity.” Read more about this proposal and other news below.

I. Regulations, Notices, & Guidance

• On January 27, 2020, the Food and Drug Administration (FDA) issued guidance entitled, Arthroscopy Pump Tubing Sets Intended for Multiple Patient Use—Premarket Notification (510(k)) Submissions. FDA developed this draft guidance document to assist in the preparation of premarket notification submissions (510(k)) for arthroscopy pump tubing sets intended for multiple patient use. This draft guidance outlines the device design considerations, risk mitigation strategies, and testing recommendations for arthroscopy pump tubing sets intended for multiple patient use. This draft guidance document also clarifies the terminology used to describe arthroscopy pump tubing sets intended for multiple patient use.
• On January 29, 2020, FDA issued guidance entitled, Chemistry, Manufacturing, and Control Information for Human Gene Therapy Investigational New Drug Applications. This guidance document provides sponsors of human gene therapy investigational new drug applications (INDs) with recommendations regarding chemistry, manufacturing, and control (CMC) information to be submitted in an IND. The guidance document informs sponsors how to provide sufficient CMC information required to assure product safety, identity, quality, purity, and strength (including potency) of the investigational product. The guidance applies to human gene therapy products and to combination products that contain a human gene therapy in combination with a drug or device.
• On January 29, 2020, FDA issued guidance entitled, Human Gene Therapy for Hemophilia. This guidance document provides recommendations to stakeholders developing human gene therapy (GT) products for the treatment of hemophilia. The guidance provides recommendations on the clinical trial design and related development of coagulation factor VIII (hemophilia A) and IX (hemophilia B) activity assays, including how to address discrepancies in factor VIII and factor IX activity assays. The guidance also includes recommendations regarding preclinical considerations to support development of GT products for the treatment of hemophilia.
• On January 29, 2020, FDA issued guidance entitled, Human Gene Therapy for Rare Diseases. This guidance document provides recommendations to stakeholders developing a human GT product intended to treat a rare disease in adult and/or pediatric patients regarding the manufacturing, preclinical, and clinical trial design issues for all phases of the clinical development program. Such information is intended to assist sponsors in designing clinical development programs for such products, where there may be limited study population size and potential feasibility and safety issues as well as issues relating to the interpretability of bioactivity/efficacy outcomes that may be unique to rare diseases or to the nature of the GT product itself.
• On January 29, 2020, FDA issued guidance entitled, Human Gene Therapy for Retinal Disorders. This guidance provides recommendations to stakeholders developing human GT products for retinal disorders affecting adult and pediatric patients. The guidance focuses on issues specific to GT products for retinal disorders and provides recommendations related to product development, preclinical testing, and clinical trial design for such GT products.
• On January 29, 2020, FDA issued guidance entitled, Interpreting Sameness of Gene Therapy Products under the Orphan Drug Regulations. This draft guidance document provides FDA's current thinking on the criteria to determine sameness of human gene therapy products for the purpose of orphan drug designation and orphan drug exclusivity. The draft guidance is intended to assist stakeholders, including industry and academic sponsors who seek orphan drug designation and orphan drug exclusivity, in the development of gene therapies for rare diseases.
• On January 29, 2020, FDA issued guidance entitled, Long Term Follow-Up After Administration of Human Gene Therapy Products. This guidance document provides sponsors, who are developing a human GT product, recommendations regarding the design of long-term follow-up observational studies for the collection of data on delayed adverse events following administration of a GT product.
• On January 29, 2020, FDA issued guidance entitled, Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up. This guidance provides sponsors of retroviral vector-based human gene therapy products recommendations regarding the testing for replication competent retrovirus during the manufacture of retroviral vector-based gene therapy products, and during follow-up monitoring of patients who have received retroviral vector-based gene therapy products. Recommendations include the identification and amount of material to be tested, and general testing methods. In addition, recommendations are provided on monitoring patients for evidence of retroviral infection after administration of retroviral vector-based gene therapy products.
• On January 30, 2020, FDA issued guidance entitled, Recommendations to Reduce the Possible Risk of Transmission of Creutzfeldt-Jakob Disease and Variant Creutzfeldt Jakob Disease by Blood and Blood Components. This draft guidance provides blood establishments that collect blood and blood components with revised recommendations intended to reduce the possible risk of transmission of Creutzfeldt-Jakob disease (CJD) and variant Creutzfeldt-Jakob disease (vCJD) by blood and blood components. The recommendations in the draft guidance apply to the collection of whole blood and blood components intended for transfusion or for use in further manufacturing, including source plasma.
• On January 31, 2020, the Department of Health and Human Services (HHS) issued a proposed rule entitled, Benefit and Payment Parameters; Notice Requirement for Non-Federal Governmental Plans. This proposed rule sets forth payment parameters and provisions related to the risk adjustment and risk adjustment data validation programs; cost-sharing parameters and cost-sharing reductions; and user fees for federally-facilitated Exchanges and State-based Exchanges on the Federal platform. It also proposes changes related to essential health benefits and would provide states with additional flexibility in the operation and establishment of Exchanges. It includes proposed changes related to cost-sharing for prescription drugs; excepted benefit health reimbursement arrangements offered by non-Federal governmental plan sponsors; the medical loss ratio program; Exchange eligibility and enrollment; exemptions from the requirement to maintain coverage; quality rating information display standards for Exchanges; and other related topics. It also proposes to repeal regulations relating to the Early Retiree Reinsurance Program.

Event Notices         

• February 12, 2020: The Centers for Disease Control and Prevention (CDC) announced a public meeting entitled, Meeting of the Community Preventive Services Task Force (CPSTF). During the afternoon session, the CPSTF will deliberate on systematic reviews of literature related to the following: HIV Prevention: Clinical Decision Support Systems to Increase HIV Screening; Cardiovascular Disease Prevention and Control: Economics of Pharmacy-Based Interventions to Increase Medication Adherence; and one or more proposals for new reviews in the areas of mental health, sleep health, tobacco prevention and control.
• February 13-14, 2020: HHS announced a public meeting entitled, Meeting of the National Vaccine Advisory Committee. During the February 2020 meeting, sessions will focus on vaccine innovation and vaccine confidence with updates from subcommittees and members.
• February 24, 2020: FDA announced a public meeting entitled, Rare Disease Day 2020: Supporting the Future of Rare Disease Product Development. The goal of this meeting is to obtain stakeholders' perspectives on challenges and solutions in rare disease product development and identify commonalities that can support product development across a variety of rare diseases.
• February 26, 2020: FDA announced a public meeting entitled, Oncologic Drugs Advisory Committee. During the morning session, the committee will discuss new drug application 212578 for padeliporfin di-potassium powder for solution for injection, submitted by STEBA Biotech, S.A. The proposed indication (use) for this product is for the treatment of patients with localized prostate cancer. During the afternoon session, the committee will discuss supplemental biologics license application 125477/S-034, for CYRAMZA (ramucirumab) injection for intravenous use, submitted by Eli Lilly and Company. The proposed indication (use) for this product is in combination with erlotinib, for first-line treatment of patients with metastatic non-small cell lung cancer whose tumors have epidermal growth factor receptor exon 19 deletions or exon 21 (L858R) substitution mutations.
• March 2-4, 2020: The Health Resources and Services Administration (HRSA) announced a public meeting entitled, National Advisory Committee on Rural Health and Human Services (NACRHHS). NACRHHS provides advice and recommendations to the Secretary of HHS on policy, program development, and other matters of significance concerning both rural health and rural human services.
• March 4, 2020: FDA announced a public meeting entitled, Vaccines and Related Biological Products Advisory Committee. The committee will discuss and make recommendations on the selection of strains to be included in the influenza virus vaccines for the 2020 to 2021 influenza season. Also, on March 4, 2020, under Topic II, the committee will meet in open session to hear an overview of the research programs in the Laboratory of Respiratory and Special Pathogens, Division of Bacterial, Parasitic, and Allergenic Products, Office of Vaccines Research and Review, CBER.

II. Congressional Hearings

House of Representatives

• On January 28, 2020, the House Committee on Ways and Means held a hearing entitled, Legislative Proposals for Paid Family and Medical Leave. Witnesses present included: Representative Rosa DeLauro (D-CT); Representative Ann Wagner (R-MO); Representative Elise Stefanik (R-NY); Joan Lunden, Journalist and Women’s Health and Wellness Advocate; Kemi Role, Director of Work Equity at the National Employment Law Project; Sharon Terman, Director of Work and Family Policy at Legal Aid at Work of California; Hadley Manning, Director of Policy at Independent Women’s Forum and Independent Women’s Voice; Rebecca Hamilton, Co-CEO of W.S. Badger; and Vicki Shabo, Senior Fellow for Paid Leave Policy and Strategy at New America.
• On January 28, 2020, the House Committee on the Judiciary Subcommittee on Crime, Terrorism, and Homeland Security held a hearing entitled, Fentanyl Analogues: Perspectives on Classwide Scheduling. Witnesses present included: Mr. Kevin Butler, Federal Public Defender, Northern District of Alabama; Dr. Daniel Ciccarone, Professor of Family and Community Medicine, University of California, San Francisco; Dr. Sandra Comer, Professor of Neurobiology, Department of Psychiatry, Columbia University; Dr. Brett Giroir, Assistant Secretary for Health, HHS; Mr. Donald Holman; and Ms. Amanda Liskamm, Director of Opioid Enforcement and Prevention Efforts, U.S. Department of Justice.
• On January 28, 2020, the House Committee on Education and Labor Subcommittee on Health, Employment, Labor, and Pensions held a hearing entitled, Expecting More: Addressing America’s Maternal and Infant Health Crisis. Witnesses present included: Stacey Stewart, President and CEO, March of Dimes; Nikia Sankofa, Executive Director, United States Breastfeeding Committee; and Joia Perry, MD, President, National Birth Equity Collaborative.
• On January 29, 2020, the House Committee on Energy and Commerce Subcommittee on Health held a hearing entitled, Improving Safety and Transparency in America’s Food and Drugs. Witnesses present included: Kao-Ping Chua, M.D., Ph.D., Assistant Professor, Department of Pediatrics, University of Michigan Medical School; Fernando Muzzio, Ph.D., Distinguished Professor, Chemical and Biochemical Engineering, Rutgers, the State University of New Jersey; Richard Kaeser, Vice President, Global Brand Protection, Johnson & Johnson (J&J); Jeff Allen, Ph.D., President and CEO, Friends of Cancer Research; Melanie Benesh, Legislative Attorney, Environmental Working Group; Paul C. DeLeo, Ph.D., Principal, Integral Consulting, Inc.; Tom Balmer, Executive Vice President, National Milk Producers Federation; J. David Carlin, Senior Vice President of Legislative Affairs and Economic Policy, International Dairy Foods Association; Douglas Corey, D.V.M., Past President, American Association of Equine Practitioners; Talia Day, Patient Advocate; Mardi Mountford, President, Infant Nutrition Council of America; Nancy Perry, Senior Vice President, Government Relations, American Society for the Prevention of Cruelty to Animals; and Sara Sorscher, Deputy Director of Regulatory Affairs, Center for Science in the Public Interest.

III. Reports, Studies, & Analyses

• On January 27, 2020, the Government Accountability Office (GAO) published a report entitled, 340B Drug Discount Program: Oversight of the Intersection with the Medicaid Drug Rebate Program Needs Improvement. Among other things, this report examines HHS efforts to ensure entities covered under the 340B Drug Pricing Program do not receive duplicate discounts through the Medicaid Drug Rebate Program. GAO was asked to provide information on the prevention of duplicate discounts and found that limitations in HHS oversight of the 340B and Medicaid Drug Rebate Programs may increase the risk that duplicate discounts occur. Given these limitations in federal oversight, GAO concluded that HHS does not have reasonable assurance that states and covered entities are complying with the prohibition on duplicate discounts. As a result, GAO made several recommendations, which are explained in detail in the report.
• On January 27, 2020, the Kaiser Family Foundation published an issue brief entitled, What You Need to Know About the Medicaid Fiscal Accountability Rule. On November 18, 2019, CMS released a proposed rule called the Medicaid Fiscal Accountability Regulation (MFAR). CMS says that the rule would promote transparency by establishing new reporting requirements related to supplemental payments to Medicaid providers and upper payment limits (UPL) on what providers can be paid by Medicaid programs. In addition to new reporting requirements, the rule makes changes to the rules and review of supplemental payments and UPL arrangements as well as changes to financing the non-federal share of Medicaid with new requirements and reviews of state financing mechanisms. The changes proposed are extremely technical and complex but are likely to have significant implications for provider payment rates and state financing of Medicaid by disrupting current arrangements and restricting the future use of such arrangements. This brief provides some context on Medicaid financing, an overview of current state payment and financing rules, the provisions in the rule, and potential implications for considerations.

IV. Other Health Policy News

• On January 30, 2020, the Centers for Medicare & Medicaid Services (CMS) unveiled guidance that would allow states to receive a block grant for optional Medicaid populations—adults under the age of 65 who would not otherwise be eligible under the traditional Medicaid program. In exchange for accepting a cap on federal funding for beneficiaries in the program, states would have more leeway to decide what benefits to cover and would get a streamlined process to add work requirements and other restrictions. According to CMS, the demonstration initiative, called the Health Adult Opportunity, is “designed to give states unprecedented tools to design innovative health coverage programs tailored to the unique needs of adult beneficiaries, while holding states accountable for results and maintaining strong protections for our most at-risk populations.”
• On January 29, 2020, the U.S. House of Representatives passed S. 3201, the Temporary Reauthorization and Study of the Emergency Scheduling of Fentanyl Analogues Act, by a vote of 320 to 88. The bill, which already passed the Senate, will extend for 15 months Department of Justice (DOJ) temporary power to place fentanyl-like substances on the list of most strictly controlled drugs. DOJ officials have been urging lawmakers for months to extend the powers or make them permanent.

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