United States: Alston & Bird Health Care Week In Review, December 10, 2021

Below is Alston & Bird's Health Care 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:

This week, HHS announced several actions to improve maternal health outcomes, including awarding $82 million in ARP emergency home visiting funds. Read more about these actions and other news below.

I. Regulations, Notices & Guidance

• On December 7, 2021, FDA issued draft guidance entitled, Investigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Chemistry, Manufacturing, and Controls Recommendations, Guidance for Sponsor-Investigators. This draft guidance provides FDA's recommendations on the chemistry, manufacturing, and controls (CMC) information needed to support an investigational new drug application (IND) submitted by a sponsor-investigator developing an individualized antisense oligonucleotide (ASO) drug product for a severely debilitating or life-threatening (SDLT) disease caused by a unique genetic variant affecting a small number of individuals (typically one or two).
• On December 7, 2021, FDA issued draft guidance entitled, Investigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Clinical Recommendations; Draft Guidance for Sponsor-Investigators.This draft guidance will provide sponsor investigators who are interested in developing individualized antisense oligonucleotide (ASO) drug products for a rapidly progressive, severely debilitating, or life-threatening (SDLT) genetic disease (caused by a unique genetic variant or variants), with clinical recommendations for submission of investigational new drug applications (INDs).
• On December 8, 2021, FDA issued draft guidance entitled, Considerations for the Use of Real-World Data and Real-World Evidence To Support Regulatory Decision-Making for Drug and Biological Products.This guidance discusses the applicability of FDA's investigational new drug application (IND) regulations to various clinical study designs that utilize real-world data (RWD), and clarifies the Agency's expectations regarding clinical studies using RWD submitted to FDA in support of a regulatory decision regarding the effectiveness or safety of a drug (e.g., as part of a new drug application or a biologics license application) that are not subject to the IND regulations.
• On December 8, 2021, FDA issued final guidance entitled, Tobacco Product User Fees: Responses to Frequently Asked Questions; Guidance for Industry.  This guidance provides information in response to frequently asked questions related to tobacco product user fees assessed and collected under the Federal Food, Drug, and Cosmetic Act (FD&C Act).
• On December 8, 2021, the Centers for Medicare & Medicaid Services (CMS) issued a notice of enforcement discretion entitled, Medicare and Medicaid Programs; Patient Protection and Affordable Care Act; Interoperability and Patient Access for Medicare Advantage Organizations and Medicaid Managed Care Plans, State Medicaid Agencies, CHIP Agencies and CHIP Managed Care Entities, Issuers of Qualified Health Plans on the Federally-Facilitated Exchanges, and Health Care Providers.  This notification is to inform the public that CMS is exercising its discretion in how it enforces the payer-to-payer data exchange provisions. As a matter of enforcement discretion, CMS does not expect to take action to enforce compliance with these specific provisions until CMS is able to address certain implementation challenges.
• On December 9, 2021, FDA issued a notice entitled, Real-World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision-Making for Drug and Biological Products; Draft Guidance for Industry; Reopening of the Comment Period.  FDA is reopening the comment period for the document entitled, Real-World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision-Making for Drug and Biological Products; Draft Guidance of Industry, published in the Federal Register on September 30, 2021.
• On December 9, 2021, FDA issued final guidance entitled, Chemistry, Manufacturing, and Controls Postapproval Manufacturing Changes for Specified Biological Products to Be Documented in Annual Reports.  This guidance provides recommendations to holders of biologics license applications (BLAs) for specified biological products regarding the types of changes to be documented in annual reports. Specifically, the guidance describes chemistry, manufacturing, and controls (CMC) postapproval manufacturing changes that FDA generally considers to have a minimal potential to have an adverse effect on the identity, strength, quality, purity, or potency of the product as they may relate to the safety or effectiveness of the product. Under FDA regulations, such minor changes in the product, production process, quality controls, equipment, facilities, or responsible personnel must be documented by applicants in an annual report.
• On December 9, 2021, FDA issued draft guidance entitled, Chronic Rhinosinusitis with Nasal Polyps: Developing Drugs for Treatment.  The purpose of this draft guidance is to assist sponsors in the clinical development of drugs for the treatment of chronic rhinosinusitis with nasal polyps (CRSwNP). Specifically, this draft guidance addresses FDA's current recommendations regarding trial design, safety, and efficacy considerations for CRSwNP clinical trials.
• On December 9, 2021, FDA issued final guidance entitled, Development of Anti-Infective Drug Products for the Pediatric Population.  The purpose of this guidance is to provide general recommendations on the development of anti-infective drug products for pediatric patients. The guidance addresses enrollment strategies, extrapolation of efficacy, safety database, and other considerations to help facilitate pediatric anti-infective drug product development.
• On December 10, 2021, FDA issued draft guidance entitled, Bowel Cleansing for Colonoscopy: Efficacy and Safety Considerations for Developing New Products.  The purpose of this draft guidance is to provide FDA's current thinking regarding the necessary attributes of patients for enrollment in clinical trials, efficacy assessments, and safety assessments. The draft guidance is intended to serve as a focus for continued discussion among FDA's Division of Gastroenterology, pharmaceutical sponsors, the academic community, and the public.
• On December 10, 2021, FDA issued draft guidance entitled, Cover Letter Attachments for Controlled Correspondences and Abbreviated New Drug Application Submissions.  This guidance is intended to assist prospective applicants, applicants, and holders of abbreviated new drug applications (ANDAs) with optional attachments that can be used when preparing cover letters that accompany controlled correspondence to the Office of Generic Drugs (OGD), as well as original ANDAs, amendments to ANDAs, and supplements to approved ANDAs submitted to FDA.
• On December 10, 2021, FDA issued final guidance entitled, Q3C(R8) Impurities: Guidance for Residual Solvents; International Council for Harmonisation. This guidance provides recommendations for permitted daily exposures (PDEs) for three additional residual solvents: 2-methyltetrahydrofuran, cyclopentyl methyl ether, and tert-butyl alcohol.

Event Notices

• January 19, 2022:  The National Institutes of Health (NIH) announceda public meeting of the National Advisory Council for Biomedical Imaging and Bioengineering (NACBIB). The meeting will feature a report from the Institute Director, Council Members, and other Institute Staff.
• February 4, 2022:  NIH announceda public meeting of the National Institute on Minority Health and Health Disparities. The meeting will feature opening remarks, administrative matters, the Director's Report, presentations, and other business of the Council.
• February 10, 2022:FDA announced a public meeting of the Oncologic Drugs Advisory Committee. The general function of the committee is to provide advice and recommendations to FDA on regulatory issues.
• Various Dates:  The Department of Health and Human Services (HHS) announcedvarious dates for public meetings of the Health Information Technology Advisory Committee (HITAC). The HITAC, among other things, identifies priorities for standards adoption and makes recommendations to the National Coordinator for Health Information Technology (National Coordinator).

II. Congressional Hearings

U.S. House of Representatives

• On December 8, 2021, the House Committee on Energy & Commerce Subcommittee on Health held a hearing entitled, The Future of Biomedicine: Translating Biomedical Research into Personalized Health Care.  Witnesses present included: Dr. Amy Abernethy, President of Clinical Studies Platforms, Verily Life Sciences; Dr. Atul Butte, Distinguished Professor and Director of the Bakar Computational Health Sciences Institute, UCSF, and Chief Data Scientist, UC Health; Mr. Adolph Falcón, Executive Vice President, National Alliance for Hispanic Health; Dr. Leroy Hood, President, Institute for Systems Biology, Affiliate Professor of Immunology, University of Washington; and Dr. Lloyd Minor, Dean, Stanford University School of Medicine.
• On December 10, 2021, the House Committee on Oversight and Reform held a hearing entitled, Unsustainable Drug Prices: Findings from the Committee's Drug Pricing Investigation and the Need for Structural Reforms.  Witnesses present included: Mindy Salango, Patient Advocate with Type 1 Diabetes, Morgantown, West Virginia; Dr. Reshma Ramachandran, Physician-Fellow, Yale National Clinician Scholars Program and Co-Chair, Doctors for America Drug Affordability Action Team; Rena Conti, PhD, Associate Professor, Department of Markets, Public Policy, and Law, Questrom School of Business, Boston University; and David Mitchell, President and Founder, Patients for Affordable Drugs.

III. Reports, Studies & Analyses

• On December 7, 2021, the Government Accountability Office (GAO) published a report entitled, Cybersecurity: NIH Needs to Take Further Actions to Resolve Control Deficiencies and Improve Its Program.  This report is a public version of GAO's June 2021 report on NIH cybersecurity. The agency has taken actions intended to safeguard the confidentiality, integrity, and availability of its systems. However, they found many weaknesses related to identifying risks, protecting systems, and more. GAO made 219 recommendations for improvements. NIH has partially implemented more than half and fully implemented about a third of them.
• On December 8, 2021, GAO published a report entitled, Drug Control Grants: ONDCP Should Document Its Process for Identifying Duplication, Overlap, and Fragmentation.  The Office of National Drug Control Policy (ONDCP) coordinates the nation's drug control policy. ONDCP's responsibilities include tracking and publicizing information about federal grants that fund drug control activities, such as prevention and treatment. ONDCP posted drug control grant information to its website and collected information on barriers for potential grantees. GAO recommends that ONDCP document its process or procedures to identify DOF in drug control grants, and ensure that it includes certain elements, such as how grants are selected for review. ONDCP concurred with the recommendation.
• On December 9, 2021, the RAND Corporation published a report entitled, COVID-19 and the Experiences of Populations at Greater Risk: Description and Top-Line Summary Data - Wave 4, Fall 2021. In this report, the authors summarize descriptive findings from the last of four waves of the COVID-19 and the Experiences of Populations at Greater Risk Survey, with particular focus on populations deemed at risk or underserved, including people of color and those from low- to moderate-income backgrounds.
• On December 10, 2021, HHS Office of Inspector General (OIG) published a report entitled, Medicare Could Have Saved Approximately $993 Million in 2017 and 2018 if It Had Implemented an Inpatient Rehabilitation Facility Transfer Payment Policy for Early Discharges to Home Health Agencies.  OIG's objective was to determine how much Medicare could have saved in calendar years (CYs) 2017 and 2018 if CMS had expanded the IRF transfer payment policy to include early discharges to home health care. OIG found that Medicare could have saved approximately $993 million in CYs 2017 and 2018 if CMS had expanded its IRF transfer payment policy to apply to early discharges to home health care.
• On December 10, 2021, the Kaiser Family Foundation (KFF) published an issue brief entitled, No Surprises Act Implementation: What to Expect in 2022.  The issue brief provides an overview of the No Surprises Act (NSA), which establishes new federal protections against surprise medical bills that take effect in 2022.

IV. Other Health Policy News

• On December 7, 2021, HHS announced that U.S. Surgeon General Dr. Vivek Murthy issued a new Surgeon General's Advisory to highlight the urgent need to address the nation's youth mental health crisis. As the nation continues the work to protect the health and safety of America's youth during this pandemic with the pediatric vaccine push amid concerns of the emerging omicron variant, the U.S. Surgeon General's Advisory on Protecting Youth Mental Health outlines the pandemic's unprecedented impacts on the mental health of America's youth and families, as well as the mental health challenges that existed long before the pandemic. More information on this announcement can be found here.
• On December 8, 2021, the Substance Abuse and Mental Health Services Administration (SAMHSA) announced that it is accepting applications for the first-ever SAMHSA Harm Reduction grant program and expects to issue $30 million in grant awards. This funding, authorized by the American Rescue Plan, will help increase access to a range of community harm reduction services and support harm reduction service providers as they work to help prevent overdose deaths and reduce health risks often associated with drug use. SAMHSA will accept applications from State, local, Tribal, and territorial governments, Tribal organizations, non-profit community-based organizations, and primary and behavioral health organizations. More information on this announcement can be found here.
• On December 8, 2021, HHS and the Department of Housing and Urban Development (HUD) announced the expansion of a partnership established earlier this year to improve access to affordable, accessible housing and the critical services that make community living possible. The partners also announced the launch of a national Housing and Services Resource Center (HSRC) as the hub of this coordinated federal effort. More information on this partnership can be found here.
• On December 9, 2021, CMS released a new monthly National Marketplace Open Enrollment report shows that, so far, nearly 4.6 million Americans have signed up for 2022 health coverage through HealthCare.gov and State-based Marketplaces since the start of the 2022 Open Enrollment Period on November 1. The assistance from the American Rescue Plan continues to drive affordability and accessibility of health coverage with the number of consumers getting coverage for $10 or less per month after tax credits nearly doubling compared to this time last year. Ninety five percent of consumers in HealthCare.gov states are receiving premium tax credits to lower the cost of their monthly premiums. More information on this report can be found here.
• On December 9, 2021, FDA amended the emergency use authorization (EUA) for the Pfizer-BioNTech COVID-19 Vaccine, authorizing the use of a single booster dose for administration to individuals 16 and 17 years of age at least six months after completion of primary vaccination with the Pfizer-BioNTech COVID-19 Vaccine. More information on this action can be found here.
• On December 9, 2021, HHS Secretary Xavier Becerra launched the Biden-Harris Administration's White House Initiative on Asian Americans, Native Hawaiians, and Pacific Islanders (WHIAANHPI) and announced U.S. Trade Representative Ambassador Katherine Tai as his co-chair of both the White House Initiative and the President's Advisory Commission on Asian Americans, Native Hawaiians, and Pacific Islanders. More information on this initiative can be found here.
• On December 10, 2021, HHS announced several actions it had taken this week to improve maternal health outcomes. In particular, on December 7, 2021, the White House hosted the first-ever Maternal Health Day of Action with a nationwide call-to-action to address maternal mortality and morbidity. CMS announced that it plans to establish a national "Birthing-Friendly" hospital designation to drive improvements in perinatal health outcomes and maternal health equity. It also issued guidance on states' implementation of a new state plan option established by the American Rescue Plan (ARP), which will allow states to provide 12 months of extended postpartum coverage to pregnant individuals enrolled in Medicaid and CHIP, beginning April 1, 2022. Finally, HRSA awarded $82 million in ARP emergency home visiting funds to 56 states, jurisdictions, and nonprofit organizations to support children and families affected by the COVID-19 pandemic. This funding builds upon an initial investment of $40 million in ARP funds in May. The Maternal, Infant, and Early Childhood Home Visiting (MIECHV) Program supports the delivery of high quality, voluntary, evidence-based home visiting services to children and families living in communities at risk for poor maternal and child health outcomes. More information on these actions can be found here.

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