Department of Health and Human Services January 30, 2020 – Federal Register Recent Federal Regulation Documents

The CDC within the HHS announces the next meeting of the CPSTF on February 12, 2020.

The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance entitled ``Long Term Follow-Up After Administration of Human Gene Therapy Products.'' The guidance document 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. This guidance finalizes the draft guidance of the same title dated July 2018 and supersedes the document entitled ``Guidance for Industry: Gene Therapy Clinical TrialsObserving Subjects for Delayed Adverse Events'' dated November 2006. This guidance also supplements the guidance entitled ``Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus during Product Manufacture and Patient Follow-up; Guidance for Industry.''

The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance entitled ``Human Gene Therapy for Rare Diseases; Guidance for Industry.'' The final 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 The guidance announced in this notice finalizes the draft guidance of the same title dated July 2018.

The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance entitled ``Human Gene Therapy for Hemophilia; Guidance for Industry.'' The guidance document provides recommendations to stakeholders developing human gene therapy (GT) products for the treatment of hemophilia. The 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 guidance also includes recommendations regarding preclinical considerations to support development of GT products for the treatment of hemophilia. The guidance announced in this notice finalizes the draft guidance of the same title dated July 2018.

The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance entitled ``Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up; Guidance for Industry.'' The guidance provides sponsors of retroviral vector-based human gene therapy products recommendations regarding the testing for replication competent retrovirus (RCR) during the manufacture of retroviral vector-based gene therapy products, and during follow-up monitoring of patients who have received retroviral vector-based gene therapy products. Recommendations include the 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 guidance supersedes 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 guidance announced in this notice finalizes the draft guidance of the same title dated July 2018.

The Food and Drug Administration (FDA) is requesting nominations for voting members, excluding consumer representative, to serve on the Patient Engagement Advisory Committee (the Committee) in the Center for Devices and Radiological Health. Nominations will be accepted for current and upcoming vacancies effective with this notice. FDA seeks to include the views of women and men, members of all racial and ethnic groups, and individuals with and without disabilities on its advisory committees and, therefore, encourages nominations of appropriately qualified candidates from these groups.

In August 2019, the Department of Health and Human Services (HHS) published an interim final rule to revise its regulations to update references to the International Classification of Disease (ICD) codes from ICD-9-CM to ICD-10-CM, and remove outdated references to chronic lymphocytic leukemia from Energy Employees Occupational Illness Compensation Program regulations. These technical amendments have no effect on the cancer eligibility requirement under the Program because all cancer types are eligible to receive a dose reconstruction from NIOSH. Thus, no eligible claimant will be adversely impacted by the rulemaking finalized in this document.