FDA Comment Deadline: Evaluating the Clinical Pharmacology of Oligonucleotide Therapeutics

The Food and Drug Administation (FDA) is establishing a public docket to collect comments on evaluating the clinical pharmacology of oligonucleotide therapeutics. There are many unique clinical pharmacology considerations concerning the development of oligonucleotide therapeutics; however, for the purposes of this request, the Agency is specifically interested in comments regarding the characterization of the effects of hepatic and renal impairment, drug-drug interactions, and immunogenicity on the pharmacokinetics of oligonucleotide therapeutics as well as the effects of oligonucleotide therapeutics on cardiac electrophysiology. Public comments will help the Agency develop recommendations for the design and conduct of studies important to the safe and effective use of oligonucleotide therapeutics and facilitate the regulatory assessment of such studies.

Oligonucleotide therapeutics typically are synthetically modified single- or double-stranded ribonucleic acid (RNA) or deoxyribonucleic acid (DNA) that exert pharmacologic effects through a variety of mechanisms (e.g., altered splicing, RNA interference, immunomodulation, microRNA modulation). Compared to small molecule or biological products, oligonucleotide therapeutics have unique characteristics regarding their chemistry, pharmacology, sites of action, pharmacokinetic disposition, and pharmacodynamics. As a result, there may be special considerations for the design and conduct of clinical pharmacology studies to assess oligonucleotide therapeutics, such as those designed to evaluate the effects of organ impairment or drug interactions. Currently, none of FDA's currently published guidance documents on clinical pharmacology assessments contain specific recommendations for oligonucleotide therapeutics.

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

APHIS Webinar: Biotechnology Quality Management Support Program

The Department of Agriculture (USDA), Animal and Plant Health Inspection Service (APHIS), Biotechnology Regulatory Services is hosting a Biotechnology Quality Management Support (BQMS) Program webinar as part of its BQMS Foundations Workshop series.

The workshop will familiarize participants with the BQMS tools to enhance compliance with current biotechnology regulations at 7 CFR part 340 regarding the import, interstate movement, and environmental release of regulated genetically engineered (GE) organisms. The tools are a modular approach featuring user-friendly, web-based templates, guidelines, and checklists. It will assist users large or small in the implementation of a quality management system for conducting field trials of regulated GE organisms.

Members of the public may attend the webinar with advance registration. More information is available at this USDA website.

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

The National Academies of Sciences, Engineering, and Medicine (NASEM) and the Royal Society have convened an international commission to develop a framework for considering technical, scientific, medical, regulatory, and ethical requirements for human germline genome editing, should society conclude such applications are acceptable.

To inform its deliberations, the Commission is engaging with external expertise and invites public comments. The call for evidence includes several questions that invite broad input; others are more technical in nature. It is being distributed to researchers and professional societies for genetic technologies and reproductive medicine, bioethics forums, patient community organizations, and others. Interested members of the public are also encouraged to participate.

Members of the public may submit comments online. More information is available at this Royal Society website.

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.

NIH Meeting: National Human Genome Research Institute, Advisory Council for Human Genome Research

The National Institutes of Health (NIH), National Human Genome Research Institute (NHGRI), will hold a meeting of its National Advisory Council for Human Genome Research. Agenda items include a report from the Director, a presentation titled "Inter-Society Coordinating Committee for Practitioner Education in Genomics (ISCC-PEG): Promoting Collaboration in Genomics Education", and various concept clearances.

The Council advises the Department of Health and Human Services, NIH, and NHGRI on genetics, genomic research, training, and programs related to the human genome initiative.

Members of the public may attend the meeting in person. More information is available at the NIH website.

FDA Workshop: Identification and Use of Biomarkers to Advance Development of Preventive Vaccines

The Food and Drug Administration (FDA), Center for Biologics Evaluation and Research, in partnership with the National Institutes of Health and the Coalition for Epidemic Preparedness Innovations, are announcing the following public workshop entitled "Identification and Use of Biomarkers to Advance Development of Preventive Vaccines." The purpose of the public workshop is to exchange information with stakeholders from industry, academia, and government about the scientific, clinical, and regulatory challenges encountered in the identification, characterization, and qualification of biomarkers for use in the development of preventive vaccines for infectious diseases indications.

FDA’s Biomarker Qualification Program was established to encourage the development of biomarkers for use in drug development, to facilitate an efficient review process, and to make information on qualified biomarkers publicly available. The 21st Century Cures Act of 2016 (Pub. L. 114-255) further highlighted the importance of biomarker qualification in accelerating the availability of safe and effective products.

The public workshop will bring together government agencies, academia, industry, and other stakeholders to discuss the scientific, clinical, and regulatory challenges encountered in the identification,characterization, and qualification of biomarkers for preventive vaccines for infectious diseases indications. The workshop will include discussions on exploring the use of biomarkers to inform the clinical development of preventive vaccines and in regulatory decision making.

Members of the public may attend in person with advance registration. More information is available at this FDA website.