Genetics/Genomics

FDA Comment Deadline: Rare Diseases – Early Drug Development and the Role of Pre-Investigational New Drug Application Meetings

The Food and Drug Administration (FDA) is announcing the availability of a draft guidance for industry entitled “Rare Diseases: Early Drug Development and the Role of Pre-Investigational New Drug Application Meetings.” The purpose of this draft guidance is to assist sponsors of drug and biological products for the treatment of rare diseases in planning and conducting more efficient and productive pre-investigational new drug application (pre-IND) meetings.

Drug development for rare diseases has many challenges related to the nature of these diseases. This draft guidance is intended to advance and facilitate the development of drugs and biological products for the treatment of rare diseases.

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

FDA Comment Deadline: Human Gene Therapy for Retinal Disorders

The Food and Drug Administration (FDA) is announcing the availability of a draft document entitled “Human Gene Therapy for Retinal Disorders; Draft Guidance for Industry.” The draft guidance provides recommendations to stakeholders developing human gene therapy (GT) products for retinal disorders affecting adult and pediatric patients. The draft guidance focuses on issues specific to GT products for retinal disorders and provides recommendations related to product development, preclinical testing, and clinical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.

These disorders vary in etiology, prevalence, diagnosis, and management, and include genetic as well as age-related diseases. These disorders manifest with central or peripheral visual impairment and often with progressive visual loss. The draft guidance focuses on issues specific to GT products for retinal disorders and provides recommendations related to product development, preclinical testing, and clinical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.

The original deadline for public comments was October 10, 2018. On September 6, 2018, the FDA announced (83 FR 45249) they were extending the deadline to December 10, 2018.

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

FDA Comment Deadline: Human Gene Therapy for Rare Diseases

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft document entitled “Human Gene Therapy for Rare Diseases; Draft Guidance for Industry.” The draft guidance document provides recommendations to stakeholders developing a human gene therapy (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.

The Orphan Drug Act of 1983 (Pub. L. 97-414) defines a rare disease as a disease or condition that affects fewer than 200,000 persons in the United States. Since most rare diseases have no approved therapies, there is a significant unmet need for effective treatments. However, developing safe and effective products to treat rare diseases can be challenging. For example, it may be more difficult to find and recruit such patients into clinical trials, and many rare diseases exhibit a number of variations or subtypes. Consequently, patients may have highly diverse clinical manifestations and rates of disease progression with unpredictable clinical courses. Despite these challenges, GT-related research and development continue to grow at a rapid rate, with several products advancing in clinical development.

The original deadline for public comments was October 10, 2018. On September 6, 2018, the FDA announced (83 FR 45249) they were extending the deadline to December 10, 2018.

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

FDA Comment Deadline: Human Gene Therapy for Hemophilia

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft document entitled “Human Gene Therapy for Hemophilia; Draft Guidance for Industry.” The draft guidance document provides recommendations to stakeholders developing human gene therapy (GT) products for the treatment of hemophilia. The draft 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 draft guidance also includes recommendations regarding preclinical considerations to support development of GT products for the treatment of hemophilia.

This draft guidance is being issued consistent with FDA's good guidance practices regulation (21 CFR 10.115). The draft guidance, when finalized, will represent the current thinking of FDA on “Human Gene Therapy for Hemophilia.”

The original deadline for public comments was October 10, 2018. On September 6, 2018, the FDA announced (83 FR 45249) they were extending the deadline to December 10, 2018.

Comments can be submitted by mail or electronically at Regulations.govMore information is available at this Federal Register notice.

FDA Comment Deadline: Long Term Follow-Up After Administration of Human Gene Therapy Products

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft document entitled “Long Term Follow-Up After Administration of Human Gene Therapy Products; Draft Guidance for Industry.” The draft guidance provides sponsors, who are developing a human gene therapy (GT) product, recommendations regarding the design of long term follow-up (LTFU) observational studies for the collection of data on delayed adverse events following administration of a GT product.

The draft guidance, when finalized, is intended to supersede the document entitled “Guidance for Industry: Gene Therapy Clinical Trials—Observing Participants for Delayed Adverse Events” dated November 2006. This draft guidance, when finalized, is also intended to supplement the guidance entitled “Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus during Product Manufacture and Patient Follow-up; Draft Guidance for Industry.”

The original deadline for public comments was October 10, 2018. On September 6, 2018, the FDA announced (83 FR 45249) they were extending the deadline to December 10, 2018.

Comments can be submitted by mail or electronically to Regulations.gov. More information can be found at this Federal Register notice.

FDA Comment Deadline: Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up

FDA is announcing the availability of a draft document entitled “Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up; Draft Guidance for Industry.” The draft guidance document provides sponsors of retroviral vector-based human gene therapy products recommendations regarding the testing for RCR during the manufacture of retroviral vector-based products, and during follow-up monitoring of patients who have received retroviral vector-based products. Recommendations are also provided for RCR testing during manufacture, including 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.

The draft guidance, when finalized, is intended to supersede the document entitled “Guidance for Industry: Supplemental Guidance on Testing for Replication Competent Retrovirus in Retroviral Vector Based Gene Therapy Products and During Follow-up of Patients in Clinical Trials Using Retroviral Vectors,” dated November 2006. The draft guidance, when finalized, is also intended to supplement the “Long Term Follow-Up After Administration of Human Gene Therapy Products; Draft Guidance for Industry” and “Chemistry, Manufacturing, and Control Information for Human Gene Therapy Investigational New Drug Applications; Draft Guidance for Industry,” when these draft guidance documents are finalized.

The original deadline for public comments was October 10, 2018. On September 6, 2018, the FDA announced (83 FR 45249) they were extending the deadline to December 10, 2018.

Comments can be submitted by mail or electronically to Regulations.govMore details are available at this Federal Register notice.

FDA Comment Deadline: Chemistry, Manufacturing, and Control Information for Human Gene Therapy Investigational New Drug Applications

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled “Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs); Draft Guidance for Industry.” The draft guidance document provides sponsors of a human gene therapy IND with recommendations regarding CMC information required to assure product safety, identity, quality, purity, and strength (including potency) of the investigational product. The draft guidance applies to human gene therapy products and to combination products that contain a human gene therapy in combination with a drug or device.

The draft guidance, when finalized, is intended to supersede the document entitled “Guidance for FDA Reviewers and Sponsors: Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs),” dated April 2008 (April 2008 guidance).

The original deadline for public comments was October 10, 2018. On September 6, 2018, the FDA announced (83 FR 45249) they were extending the deadline to December 10, 2018.

Comments can be submitted by mail or electronically at Regulations.govMore information is available at this Federal Register notice.

NSF Meeting: National Science Board

The National Science Board (NSB) of the National Science Foundation (NSF) will hold their quarterly meeting to pursue the goals and functions of NSF, as well as recommend and encourage the pursuit of national policies for the promotion of research and education in science and engineering.

The National Science Board has two important roles. First, it establishes the policies of NSF within the framework of applicable national policies set forth by the President and the Congress. In this capacity, the Board identifies issues that are critical to NSF's future, approves NSF's strategic budget directions and the annual budget submission to the Office of Management and Budget, and approves new major programs and awards. The second role of the Board is to serve as an independent body of advisors to both the President and the Congress on policy matters related to science and engineering and education in science and engineering. In addition to major reports, the NSB also publishes occasional policy papers or statements on issues of importance to U.S. science and engineering.

This meeting is open to the public. Public visitors must arrange for a visitor's badge at least 24 hours in advance by emailing your name and organizational affiliation to NSB. Find more information and an agenda on the meeting's event page.

NASEM Meeting: Second International Summit on Human Genome Editing

The National Academies of Sciences (NAS) and Medicine (NAM), along with the Academy of Sciences of Hong Kong (ASHK) and the Royal Society (RS), will convene the Second International Summit on Human Genome Editing.

The summit will bring together a broad range of stakeholders—including researchers, ethicists, policymakers, patient groups, and representatives from science and medical academies and organizations worldwide—to advance the global discussion of the science, application, ethics, and governance of human genome editing. 

Since the first international summit was held in 2015 in Washington, D.C., research on human genome editing has continued to advance rapidly. However, many questions remain. During this summit, stakeholders will explore topics including: 1) potential benefits and risks inherent in conducting genome editing research and in considering clinical applications; 2) ethical and cultural perspectives; 3) legal, regulatory, and policy considerations; and 4) public outreach and engagement.

Members of the public must register online to attend the event before October 31st or until capacity is filled. Find a full agenda and more information online.

FDA Comment Deadline: Evidence-Based Decision in Transplantation – Patient Individualized Treatment

The Food and Drug Administration (FDA) is announcing a public workshop entitled “Evidence-Based Treatment Decision in Transplantation: Patient Individualized Treatment; Choosing the Right Regimen for the Right Patient” and seeking public comments.

This public workshop is intended to discuss potential candidate biomarkers to determine organ transplant patients' immunologic risk for organ rejection or tolerance. The public workshop will include discussion of the biomarker qualification process and how it could be used to develop biomarkers for use in clinical trials in transplantation, to develop new drugs to address unmet needs, and in clinical practice to guide patient treatment selection. Speakers will be patients who will provide perspective on the challenges of living with a transplant, managing immunosuppression and perspectives on tolerability, adherence, and risk that may inform patient-reported outcome and patient-focused drug development.

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

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

The Advisory Committee on Heritable Disorders in Newborns and Children of the Health Resources and Services Administration (HRSA), an agency of the Department of Health and Human Services (HHS), will meet to discuss and advise on the most appropriate application of universal newborn screening tests, technologies, policies, guidelines, and standards.

The mission of the Advisory Committee is to reduce morbidity and mortality in newborns and children who have, or are at risk for, heritable disorders. The Committee advises the Secretary of HHS and makes recommendations concerning funding for projects related to screening for heritable disorders, policies that will help health agencies provide services for children who have or are at risk for these disease, and other related matters. 

Members of the public can attend in-person or online via webcast. Attendees must register online, where they may also submit written comments or request time for an oral comment at the meeting. More information about the meeting, including a full agenda, can be found on the Committee website.

EPA Nomination Deadline: Toxic Substances Control Act Science Advisory Committee on Chemicals

The Environmental Protection Agency (EPA) requests public nominations of scientific experts to be considered for ad hoc participation and possible membership on the Toxic Substances Control Act (TSCA) Science Advisory Committee on Chemicals (SACC). All nominees will be considered for ad hoc participation in the TSCA SACC's peer reviews of the EPA's risk evaluations for the first 10 chemical substances addressed under the TSCA. In addition, all nominees may be considered for TSCA SACC membership to fulfill short term needs when a vacancy occurs on the chartered Committee.

Individuals nominated should have expertise in one or more of the following areas: Women's health; children's health; genetic variability; disproportionately exposed populations; aging; other susceptible populations; biochemistry; chemistry; epidemiology; human health risk assessment; pathology; PBPK modeling; pharmacology; ecological risk assessment; environmental fate; environmental toxicology; occupational, consumer, and general exposure assessment; toxicology; dose response modeling; environmental engineering; biostatistics; computational toxicology; fiber science; inhalation toxicology; volatile organics; and systematic review. 

Members of the public may submit nominations by mail or email. More details regarding these nominations, including submissions, can be found at this Federal Register notice.