Genetics/Genomics

DOS Meeting: Implications of Access and Benefit-Sharing Regimes on Global Health and Biomedical Research

The Department of State (DOS) will hold a meeting to discuss the implications of Access and Benefit-Sharing (ABS) regimes on global health and biomedical research. In particular, DOS will focus on the effects of Nagoya Protocol and other ABS implementation on public health systems. This is the seconf of two meetings on this topic; the first meeting will occur on June 25, 2019.

Specific issues of focus include: International research collaborations, international pathogen sample sharing, infectious disease research, including influenza, Zika and other diseases, pandemic and epidemic preparedness and response, medical countermeasure product development efforts, investor interest in vaccine development, and other aspects.

DOS would also welcome views on what the World Health Organization, other United Nations bodies, and non-governmental organizations can do to effectively protect public health equities in the context of the Nagoya Protocol and other national level ABS implementation. DOS also welcomes examples of:

  1. Monetary or non-monetary benefits to the global public health system that are facilitated by international sharing of pathogens, biospecimens, pathogen genetic sequence data, and/or relevant metadata.
  2. Influenza-specific issues and/or examples related to the items described above or other items that could affect global influenza pandemic preparedness and response or efforts to combat seasonal influenza.
  3. Non-ABS challenges and barriers with sharing pathogens internationally or that otherwise have significant implications for global pandemic or epidemic preparedness and response efforts that might merit additional attention or analysis.

Members of the public may attend in person. More information is available at this Federal Register notice and at this DOS website.

FDA Comment Deadline: Development of Antiviral Drugs for the Treatment of Adenoviral Infection in Immunocompromised Patients

The Food and Drug Administration seeks public comments to accompany its August 8, 2019, public workshop titled "Development of Antiviral Drugs for the Treatment of Adenoviral Infection in Immunocompromised Patients." The purpose of the public workshop is to discuss the scientific and clinical trial design considerations for development of antiviral products to treat adenoviral infection.

Discussions are planned around the following topics:

  • Trial design considerations (e.g., trial endpoints, trial populations, treatment strategies, risk/benefit considerations, ethical considerations, virologic testing considerations)
  • Diagnostic assay(s) considerations

Members of the public may submit comments online at Regulations.gov, or via mail. More information is available at this Federal Register notice.

HHS Meeting: All of Us Research Program Tribal Consultation Meeting and Listening Session

The National Institutes of Health (NIH), within the Department of Health and Human Services, hosts the All of Us research program. The All of Us Research Program is an initiative to collect genetic and health information from a diverse set of one million Americans to bolster our understanding of how precision medicine can be used to effectively treat certain diseases. Central to the All of Us mission is to include diversity in the data set.

Therefore, the All of Us Research Program will host a series of five Tribal Consultations to consult on the best practices for engaging with Tribal Nations to facilitate the inclusion of American Indian and Alaska Native populations in this program. The fifth of such meetings will take place on August 21, 2019 in Denver, Colorado.

Members of the public may attend in person. More information is available at the NIH's website and event page.

NASEM Meeting: International Commission on the Clinical Use of Human Germline Genome Editing

The National Academies of Sciences, Engineering, and Medicine (NASEM), in partnership with the Royal Society of the United Kingdom, will hold the first meeting of the International Commission on the Clinical Use of Heritable Human Genome Editing. The Commission aims to develop a framework for scientists, clinicians, and regulatory authorities to consider when assessing potential clinical applications of human germline genome editing. The framework will identify a number of scientific, medical, and ethical requirements that should be considered, and could inform the development of a potential pathway from research to clinical use — if society concludes that heritable human genome editing applications are acceptable.

The Commission is the latest action from the international science community to address issues around human genome editing. It follows the Second International Summit on Human Genome Editing — held last November in Hong Kong by the National Academy of Sciences, the National Academy of Medicine, the Royal Society, and the Academy of Sciences of Hong Kong. The topic became a focus of global attention when a scientist from China revealed at the summit that as a result of his research, twins had been born whose embryonic genomes had been edited. The scientist was widely condemned by the global scientific community for violating long-standing scientific principles and ethical norms.

The US National Academies and the Royal Society are the secretariat of the commission, which includes representatives from 10 nations. Kay Davies, professor of genetics at the MDUK Oxford Neuromuscular Centre at the University of Oxford, England, and Richard Lifton, president of the Rockefeller University in New York City, are co-chairs of the commission. The commission will hold one additional meeting and an international workshop, and will also issue a call for public input to inform their work. A final report from the commission is expected to be issued in the spring of 2020.   

Members of the public may attend the meeting with advance registration either in person or via webcast. More information is available at this NASEM website.

FDA Comment Deadline: Limited Population Pathway for Antibacterial and Antifungal Drugs

The Food and Drug Administration (FDA) is seeking public comments on the draft guidance for industry, “Limited Population Pathway for Antibacterial and Antifungal Drugs,” that was published in the Federal Register on June 13, 2018 (83 FR 27616). FDA is reopening the comment period on this draft guidance for comments to be submitted for consideration before they finish work on the final version of the guidance.

This draft guidance is intended to assist sponsors in the development of certain new antibacterial and antifungal drugs for approval under the limited population pathway for antibacterial and antifungal drugs. This draft guidance also is intended to assist sponsors in developing labeling, including prescribing information, patient labeling, and carton/container labeling, that incorporates certain statements required by section of the Federal Food, Drug, and Cosmetic Act. 

Members of the public may submit comments via mail, fax, or at Regulations.gov. More information is available at this Federal Register notice and on this FDA website

FDA Workshop: Precision Dosing: Defining the Need and Approaches to Deliver Individualized Drug Dosing in the Real-World Setting

The Food and Drug Administration (FDA) will hold a public workshop titled "Precision Dosing: Defining the Need and Approaches to Deliver Individualized Drug Dosing in the Real-World Setting." 

Some clinical trials in drug development are conducted to optimize the dosing regimen of a new drug for the target patient population. Clinical pharmacology trials serve as the major source of evidence for dose individualization (precision dosing) taking into account patient factors that can modify drug response. Despite these efforts prior to a drug’s approval, further dose optimization may be required after drug approval (e.g., accounting for extremes of age, body habitus, organ function and complex multiple characteristics including genotype, drug interactions, and co-morbidities).

The opportunities and challenges for further dose optimization/individualization will be discussed from multiple perspectives including drug developers, regulators, physicians, pharmacists and patients. These relevant groups will also discuss in what contexts precision dosing may be needed, and the challenges of generating evidence to support precision dosing and applying individualized dosing regimen in clinical practice. In addition to evidence created during the new drug development process, real-world data/evidence will be discussed to address some challenges that cannot be addressed by pre- and post-approval clinical trials. The process of translating the knowledge to tools that can be utilized by the medical community for patient care will also be discussed.

Workshop Objectives:

  1. To explore the need for more precise dosing.
  2. To investigate opportunities in the drug development and real-world settings to generate the information needed to support precision drug dosing.
  3. To discuss the translation of the dosing information to electronic drug dosing delivery tools to enable precision dosing in clinical practice.

Members of the public may attend in person with advance registration by August 2, 2019, or by webcast. More information about the meeting, including the agenda, is available at this FDA website.

AHRQ Nomination Deadline: National Advisory Council for Healthcare Research and Quality

The Agency for Healthcare Research and Quality (AHRQ) seeks public nominations to serve on its National Advisory Council for Healthcare Research and Quality. Seven current members' terms will expire in November 2019.

The Council's role is to advise the Secretary of the US Department of Health and Human Services (HHS) and the Director of AHRQ with respect to activities proposed or undertaken to carry out AHRQ's statutory mission. AHRQ produces evidence to make health care safer, higher quality, more accessible, equitable, and affordable, and to work within the HHS and with other partners to make sure that the evidence is understood and used.

To fill the vacancies, AHRQ is seeking individuals who: (1) Are distinguished in the conduct of research, demonstration projects, and evaluations with respect to health care; (2) are distinguished in the fields of health care quality research or health care improvement; (3) are distinguished in the practice of medicine; (4) are distinguished in other health professions; (5) represent the private health care sector (including health plans, providers, and purchasers) or are distinguished as administrators of health care delivery systems; (6) are distinguished in the fields of health care economics, information systems, law, ethics, business, or public policy; and (7) represent the interests of patients and consumers of health care. 42 U.S.C. 299c(c)(2). Individuals are particularly sought with experience and success in these activities. AHRQ will accept nominations to serve on the Council in a representative capacity.

Members of the public may submit nominations by mail or email. More information is available at this Federal Register notice.

FDA Workshop: Development of Antiviral Drugs for the Treatment of Adenoviral Infection in Immunocompromised Patients

The Food and Drug Administration will hold a public workshop titled "Development of Antiviral Drugs for the Treatment of Adenoviral Infection in Immunocompromised Patients." The purpose of the public workshop is to discuss the scientific and clinical trial design considerations for development of antiviral products to treat adenoviral infection.

Discussions are planned around the following topics:

  • Trial design considerations (e.g., trial endpoints, trial populations, treatment strategies, risk/benefit considerations, ethical considerations, virologic testing considerations)
  • Diagnostic assay(s) considerations

Members of the public may attend the workshop in person with advance registration by August 1, 2019, or via webcast. More information is available at this Federal Register notice.

FDA Meeting: Antimicrobial Drugs Advisory Committee

The Food and Drug Administration (FDA) will hold a public meeting of its Antimicrobial Drugs Advisory Committee. The general function of the committee is to provide advice and recommendations to FDA on regulatory issues. The committee will discuss supplemental new drug application (sNDA) 208215, supplement 12, DESCOVY (emtricitabine 200 milligrams (mg) and tenofovir alafenamide 25 mg tablets), submitted by Gilead Sciences, Inc., proposed for pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV-1 infection among individuals who are HIV-negative and at risk for HIV.

Members of the public may attend in person. More information is available at this Federal Register notice.

APHIS Comment Deadline: Movement of Certain Genetically Engineered Organisms

The Animal and Plant Health Inspection Service (APHIS) within the US Department of Agriculture seeks public comment on its proposed rule that would revise the regulations regarding the movement (importation, interstate movement, and environmental release) of certain genetically engineered (GE) organisms in response to advances in genetic engineering and our understanding of the plant pest risk posed by them, thereby reducing regulatory burden for developers of organisms that are unlikely to pose plant pest risks. This proposed rule, which would mark the first comprehensive revision of the regulations since they were established in 1987, would provide a clear, predictable, and efficient regulatory pathway for innovators, facilitating the development of new and novel genetically engineered organisms that are unlikely to pose plant pest risks.

While the current regulations have been effective in ensuring the safe introduction of GE organisms during the past 30 years, advances in genetic engineering have occurred since they were promulgated. APHIS has now accumulated three decades of experience in evaluating GE organisms for plant pest risk. The Agency's evaluations to date have provided evidence that genetically engineering a plant with a plant pest as a vector, vector agent, or donor does not in and of itself result in a GE plant that presents a plant pest risk. Additionally, GE techniques have been developed that do not employ plant pests as donor organisms, recipient organisms, vectors, or vector agents yet may result in GE organisms that pose a plant pest risk. Given these developments, as well as legal and policy issues discussed below, it has become necessary, in our view, to update our regulations accordingly.

Members of the public may submit comments online at Regulations.gov or via mail. More information is available at this Federal Register notice.

Nomination Deadline: Healthcare Infection Control Practices Advisory Committee

The Centers for Disease Control and Prevention (CDC) is seeking nominations for membership on its Healthcare Infection Control Practices Advisory Committee. The Committee consists of 14 experts in fields including but not limited to, infectious diseases, infection prevention, healthcare epidemiology, nursing, clinical microbiology, surgery, hospitalist medicine, internal medicine, epidemiology, health policy, health services research, public health, and related medical fields. Nominations are being sought for individuals who have expertise and qualifications necessary to contribute to the accomplishments of the committee's objectives. Nominees will be selected based on expertise in the fields of infectious diseases, infection prevention, healthcare epidemiology, nursing, environmental and clinical microbiology, surgery, internal medicine, and public health.

Members of the public may submit nominations by mail, email, or fax. More information is available at this Federal Register notice.

HHS Meeting: Advisory Council on Heritable Disorders in Newborns and Children

The Department of Health and Human Services (HHS) will hold a public meeting of its Advisory Council on Heritable Disorders in Newborns and Children (ACHDNC). During the meeting, ACHDNC will hear from experts in the fields of public health, medicine, heritable disorders, rare disorders, and newborn screening. Agenda items include: (1) Review of the Recommended Uniform Screening Panel (RUSP) condition nomination and evidence review process; (2) updates on screening methodologies; (3) rare disease registries; (4) linking data resources; and (5) workgroup updates. Agenda items are subject to changes as priorities dictate.

ACHDNC provides advice and recommendations to the Secretary of HHS on the development of newborn screening activities, technologies, policies, guidelines, and programs for effectively reducing morbidity and mortality in newborns and children having, or at risk for, heritable disorders. ACHDNC's recommendations regarding inclusion of additional conditions for screening, following adoption by the Secretary, are evidence-informed preventive health services provided for in the comprehensive guidelines supported by HRSA through the RUSP pursuant to section 2713 of the Public Health Service Act (42 U.S.C. 300gg-13). Under this provision, non-grandfathered group health plans and health insurance issuers offering group or individual health insurance are required to provide insurance coverage without cost-sharing (a co-payment, co-insurance, or deductible) for preventive services for plan years (i.e., policy years) beginning on or after the date that is one year from the Secretary's adoption of the condition for screening.

Members of the public will have the opportunity to provide comments. In addition to general public comments, the ACHDNC is soliciting specific feedback at this meeting from the public on processes for nominating conditions to the RUSP condition and conducting evidence reviews.

Members of the public may attend the meeting in person with advance registration by July 29, 2019. More information is available at this Federal Register notice and at this HHS website.