Alston & Bird Health Care Week in Review, September 6, 2019

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.

I. Regulations, Notices, & Guidance

• On September 4, 2019, the Food and Drug Administration (FDA) issued guidance entitled, Evaluation of Internal Standard Responses During Chromatographic Bioanalysis: Questions and Answers Guidance for Industry. This guidance provides recommendations to sponsors, applicants, and contract research organizations regarding internal standard (IS) response variability in chromatographic analytical data submitted in investigational new drug applications, new drug applications, abbreviated new drug applications, biologics license applications, and supplements. Chromatographic analytical methods are commonly used to quantitate analyte concentrations in samples from nonclinical and clinical studies to support regulatory submissions. Depending upon its source, IS response variability may impact the accuracy of analyte concentration measurements. This question and answer (Q&A) document provides the Agency’s current thinking on IS response variability and its potential impact on the accuracy of analyte concentration measurements. This Q&A also suggests approaches to determine whether observed IS response variability is likely to impact the accuracy of the data such that further investigation into the root cause(s) is warranted.
• On September 4, 2019, FDA issued a proposed rule entitled, List of Bulk Drug Substances that Can Be Used to Compound Drug Products in Accordance with Section 503A of the Federal Food, Drug, and Cosmetic Act. FDA has issued a regulation creating a list of bulk drug substances (active pharmaceutical ingredients) that can be used to compound drug products in accordance with certain compounding provisions of the Federal Food, Drug, and Cosmetic Act (FD&C Act), although they are neither the subject of an applicable United States Pharmacopeia (USP) or National Formulary (NF) monograph nor components of FDA-approved drugs. This proposed rule would amend that list by placing five additional bulk drug substances on the list and identifies 26 bulk drug substances that FDA has considered and proposes not to include on the list. Additional substances nominated by the public for inclusion on this list are currently under consideration and will be the subject of a future rulemaking.
• On September 5, 2019, FDA issued guidance entitled, Drugs for Treatment of Partial Onset Seizures: Full Extrapolation of Efficacy From Adults to Pediatric Patients 2 Years of Age and Older. This guidance provides recommendations to sponsors on the clinical development of drugs for the treatment of partial onset seizures (POS) in pediatric patients. Specifically, this guidance addresses FDA’s current thinking regarding clinical development programs that can support extrapolation of the efficacy of drugs approved for the treatment of POS in adults to pediatric patients 2 years of age and older. This guidance finalizes the draft guidance entitled “Drugs for Treatment of Partial Onset Seizures: Full Extrapolation of Efficacy from Adults to Pediatric Patients 4 Years of Age and Older” issued on February 16, 2018.
• On September 5, 2019, FDA issued guidance entitled, Humanitarian Device Exemption Program; Guidance for Industry and Food and Drug Administration Staff. This guidance concerns the humanitarian device exemption (HDE) program as a whole and, among other topics, it explains the criteria FDA considers to determine if "probable benefit" has been demonstrated as part of the Agency's decision-making process regarding marketing authorization for a humanitarian use device (HUD). The guidance also reflects recent amendments to the FD&C Act that affect the HDE program and answers common questions that FDA receives about the program.
• On September 6, 2019, The Centers for Medicare and Medicaid Services (CMS) issued a final rule, entitled, Medicare, Medicaid, and Children's Health Insurance Programs: Program Integrity Enhancements to the Provider Enrollment Process. This final rule implements statutory provisions that require Medicare, Medicaid, and Children's Health Insurance Program (CHIP) providers and suppliers to disclose certain current and previous affiliations with other providers and suppliers. It also provides the agency with additional authority to deny or revoke a provider or supplier's Medicare enrollment in certain specified circumstances. The provisions in this proposed rule enable CMS to take action against unqualified and potentially fraudulent entities and individuals, which in turn could deter other parties from engaging in improper behavior.
• On September 6, 2019, FDA issued guidance entitled, Acceptance Review for De Novo Classification Requests. The purpose of this guidance is to explain the procedures and criteria FDA intends to use in assessing whether a request for an evaluation of automatic class III designation (De Novo classification request or De Novo request) meets a minimum threshold of acceptability and should be accepted for substantive review. This guidance discusses De Novo acceptance review policies and procedures, “Refuse to Accept” principles, and the elements of the De Novo Acceptance Checklist and the Recommended Content Checklist and is being issued to be responsive to an explicit deliverable identified in the Medical Device User Fee Amendments of 2017 (MDUFA IV).

Event Notices       

• September 9-11, 2019: The Health Resources & Services Administration (HRSA) announced a public meeting entitled, National Advisory Committee on Rural Health and Human Services. The National Advisory Committee on Rural Health and Human Services (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. During the September 9, 2019, through September 11, 2019, meeting, NACRHHS will examine the current delivery of health care and human services in rural areas. Agenda items are subject to change as priorities dictate. Refer to the NACRHHS website for any updated information concerning the meeting.
• September 11-12, 2019: FDA announced a public workshop entitled, Food and Drug Administration Science Forum 2019. The purpose of the public workshop is to share with the public the unique scientific research and collaborative efforts of FDA’s 11,000 scientists and researchers, who use novel science and technologies to inform FDA’s regulatory decision-making and drive innovation.
• September 18, 2019: FDA announced a public workshop entitled, Implementing the Food and Drug Administration’s Predictive Toxicology Roadmap. The purpose of this public workshop is to highlight the work FDA has been doing to support and implement FDA’s Predictive Toxicology Roadmap.
• September 24, 2019: HRSA announced a public meeting entitled, Advisory Committee on Heritable Disorders in Newborns and Children. During this meeting, the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) will hear from experts in the fields of public health, medicine, heritable disorders, rare disorders, and newborn screening. Agenda items include a discussion of the role of health information technology within state newborn screening programs and general updates on ACHDNC projects focused on newborn screening.
• September 27, 2019: The Department of Health and Human Services (HHS) announced a public meeting entitled, National Clinical Care Commission. The National Clinical Care Commission (the Commission) is charged to evaluate and make recommendations to HHS Secretary and Congress regarding improvements to the coordination and leveraging of federal programs related to awareness and clinical care for complex metabolic or autoimmune diseases that result from issues related to insulin that represent a significant disease burden in the United States, which may include complications due to such diseases. This virtual meeting will consist of an update on the Data Call to federal agencies and Commission discussion on key topics for secondary research in support of the Report to Congress.
• September 26, 2019: FDA announced a public meeting entitled, Joint Pediatric Advisory Committee and Drug Safety and Risk Management Advisory Committee. The general function of the committees is to provide advice and recommendations to FDA on regulatory issues. The meeting will be open to the public. FDA is establishing a docket for public comment on this document. During the meeting, committee members will discuss the pediatric-focused safety review for OxyContin (oxycodone hydrochloride) extended-release tablets, as mandated by the Food and Drug Administration Safety and Innovation Act (Pub. L. 112-144), and to discuss pediatric data considerations for opioid analgesics labeling and Pediatric Research Equity Act studies for opioids generally, using Opana IR as an example.
• October 11, 2019: HRSA announced a public meeting entitled, Advisory Committee on Interdisciplinary, Community-Based Linkages. The Advisory Committee on Interdisciplinary, Community-Based Linkages (ACICBL) provides advice and recommendations to the Secretary of HHS on policy, program development, and other matters of significance concerning activities under sections 750 – 760, Title VII, Part D of the Public Health Service Act. At this meeting, ACICBL members will discuss issues related to the pending Committee reports.
• October 16-17, 2019: The Centers for Disease Control and Prevention (CDC) announced a public meeting entitled, Meeting of the Community Preventive Services Task Force (CPSTF). The CPSTF is an independent, nonfederal panel whose members are appointed by the CDC Director. CPSTF members represent a broad range of research, practice, and policy expertise in prevention, wellness, health promotion, and public health. Matters proposed for discussion during the upcoming meeting include: Cancer Prevention and Control; Obesity Prevention and Control; Pregnancy Health; and Tobacco Cessation.
• November 6-7, 2019: CDC announced a public meeting entitled, Clinical Laboratory Improvement Advisory Committee. The agenda will include agency updates from CDC, Centers for Medicare and Medicaid Services (CMS), and FDA. Presentations and discussions will focus on an update from the Association of Public Health Opioids Task Force; an update on the clinical laboratory workforce; return of research results to research participants; and improving integration of laboratory information systems with electronic health records. 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 public 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.

II. Congressional Hearings

There were no health-related hearings this week.

III. Reports, Studies, & Analyses

• On September 3, 2019, the Government Accountability Office (GAO) released a report entitled, Prescription Opioids: Patient Options for Safe and Effective Disposal of Unused Opioids. This report examines federally recommended and other available methods patients may use to dispose of unused prescription opioids, and what is known about patients' use of these methods. To do this work, GAO examined peer-reviewed, academic literature on outcomes for prescription opioid disposal; reviewed federal agency documentation; interviewed federal agency officials, independent researchers, and stakeholder group representatives—such as those from the American Medical Association; and analyzed Drug Enforcement Administration data as of April 2019 on permanent drug collection sites. GAO also interviewed representatives of three companies that manufacture commercial in-home disposal products and reviewed publicly available documents about these products.
• On September 4, 2019, RAND Corporation released a report entitled, Displaying Behavioral Health Measurement-Based Care Data: Identifying Key Features from Clinician and Patient Perspectives. Measurement-based care (MBC) in behavioral health is intended to improve the effectiveness of care through routine monitoring of patient symptoms to inform treatment and engage patients. Repeated assessment of patient symptoms, such as depression symptoms, yields multiple scores over time. Little is known about the best methods to display MBC data in a way that is clear and useful to both clinicians and patients. This report documents the findings from qualitative interviews that assessed clinician and patient perspectives on various options for displaying MBC data, with the goal of identifying core features of MBC data displays.

IV. Other Health Policy News

• On September 4, 2019, the U.S. Department of Health and Human Services (HHS) announced more than $1.8 billion in funding to states to continue the Trump administration’s efforts to combat the opioid crisis by expanding access to treatment and supporting near real-time data on the drug overdose crisis. More information about this announcement can be found here.
• On September 4, 2019, U.S. District Judge Richard J. Leon approved the U.S. Department of Justice deal clearing CVS' purchase of Aetna. The deal, which typically would have undergone a judicial merger review, was elevated to an evidentiary procedure after outside groups raised concerns that combining CVS and Aetna will lead to anti-competitive conduct in different health care markets. More information about this decision can be found here.