{"abstract":"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availability of a draft document entitled \"Long Term Follow-Up After Administration of Human Gene Therapy Products; Draft Guidance for Industry.\" The draft guidance provides sponsors, who are developing a human gene therapy (GT) product, recommendations regarding the design of long term follow-up (LTFU) observational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 The draft guidance, when finalized, is intended to supersede the document entitled \"Guidance for Industry: Gene Therapy Clinical Trials--Observing Participants for Delayed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 This draft guidance, when finalized, is also intended to supplement the guidance entitled \"Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus during Product Manufacture and Patient Follow-up; Draft Guidance for Industry.\"","action":"Notice of availability.","agencies":[{"raw_name":"DEPARTMENT OF HEALTH AND HUMAN SERVICES","name":"Health and Human Services Department","id":221,"url":"https://www.federalregister.gov/agencies/health-and-human-services-department","json_url":"https://www.federalregister.gov/api/v1/agencies/221","parent_id":null,"slug":"health-and-human-services-department"},{"raw_name":"Food and Drug Administration","name":"Food and Drug Administration","id":199,"url":"https://www.federalregister.gov/agencies/food-and-drug-administration","json_url":"https://www.federalregister.gov/api/v1/agencies/199","parent_id":221,"slug":"food-and-drug-administration"}],"body_html_url":"https://www.federalregister.gov/documents/full_text/html/2018/07/12/2018-14867.html","cfr_references":[],"citation":"83 FR 32311","comment_url":null,"comments_close_on":"2018-10-10","correction_of":null,"corrections":[],"dates":"Submit either electronic or written comments on the draft guidance by October 10, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance.","disposition_notes":null,"docket_ids":["Docket No. FDA-2018-D-2173"],"dockets":[{"supporting_documents":[{"title":"Reference 35 Guidance for Industry on Good Pharmacovigilance Practices and Pharmacoepidemiologic Assessment re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0099"},{"title":"Reference 33 - Clonality analysis after retroviral-mediated gene transfer to CD34 cells from the cord blood of ADA-deficient SCID neonates re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0097"},{"title":"Reference 31 - Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1 re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0095"},{"title":"Reference 28 - Guidance for Industry Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0092"},{"title":"Reference 26 - Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo, Nature Biotechnology re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0090"},{"title":"Reference 23 - A high-capacity, capsid-modified hybrid adenovirusadeno-associated virus vector for stable transduction of human hematopoietic cells re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0087"},{"title":"Reference 22 - DHHS NIH Recombinant DNA Advisory Committee Minutes to Meeting 1999 re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0086"},{"title":"Reference 20 - The Sleeping Beauty transposon system a non-viral vector for gene therapy Hum Mol Genet re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0084"},{"title":"Reference 16 - Haematopoietic stem cell transplantation for SCID patients where do we stand re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0080"},{"title":"Reference 12-Gene Therapy for Wiskott-Aldrich Syndrome-Long-Term Efficacy and Genotoxicity re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0076"}],"agency_name":"FDA","documents":[{"comment_count":0,"comment_start_date":"2018-07-12","updated_at":"2023-03-01T03:37:09.940-05:00","comment_url":"https://www.regulations.gov/commenton/FDA-2018-D-2173-0001","allow_late_comments":null,"id":"FDA-2018-D-2173-0001","comment_end_date":"2018-10-11","regulations_dot_gov_open_for_comment":false}],"supporting_documents_count":71,"id":"FDA-2018-D-2173","title":"Observing Subjects for Delayed Adverse Events Following Administration of Human Gene Therapy Products; Availability"}],"document_number":"2018-14867","effective_on":null,"end_page":32312,"executive_order_notes":null,"executive_order_number":null,"explanation":null,"full_text_xml_url":"https://www.federalregister.gov/documents/full_text/xml/2018/07/12/2018-14867.xml","html_url":"https://www.federalregister.gov/documents/2018/07/12/2018-14867/long-term-follow-up-after-administration-of-human-gene-therapy-products-draft-guidance-for-industry","images":{},"images_metadata":{},"json_url":"https://www.federalregister.gov/api/v1/documents/2018-14867?publication_date=2018-07-12","mods_url":"https://www.govinfo.gov/metadata/granule/FR-2018-07-12/2018-14867/mods.xml","not_received_for_publication":null,"page_length":2,"page_views":{"count":736,"last_updated":"2026-04-04 18:15:03 -0400"},"pdf_url":"https://www.govinfo.gov/content/pkg/FR-2018-07-12/pdf/2018-14867.pdf","presidential_document_number":null,"proclamation_number":null,"public_inspection_pdf_url":"https://public-inspection.federalregister.gov/2018-14867.pdf?1531313118","publication_date":"2018-07-12","raw_text_url":"https://www.federalregister.gov/documents/full_text/text/2018/07/12/2018-14867.txt","regulation_id_number_info":{},"regulation_id_numbers":[],"regulations_dot_gov_info":{"supporting_documents":[{"title":"Reference 35 Guidance for Industry on Good Pharmacovigilance Practices and Pharmacoepidemiologic Assessment re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0099"},{"title":"Reference 33 - Clonality analysis after retroviral-mediated gene transfer to CD34 cells from the cord blood of ADA-deficient SCID neonates re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0097"},{"title":"Reference 31 - Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1 re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0095"},{"title":"Reference 28 - Guidance for Industry Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0092"},{"title":"Reference 26 - Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo, Nature Biotechnology re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0090"},{"title":"Reference 23 - A high-capacity, capsid-modified hybrid adenovirusadeno-associated virus vector for stable transduction of human hematopoietic cells re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0087"},{"title":"Reference 22 - DHHS NIH Recombinant DNA Advisory Committee Minutes to Meeting 1999 re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0086"},{"title":"Reference 20 - The Sleeping Beauty transposon system a non-viral vector for gene therapy Hum Mol Genet re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0084"},{"title":"Reference 16 - Haematopoietic stem cell transplantation for SCID patients where do we stand re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0080"},{"title":"Reference 12-Gene Therapy for Wiskott-Aldrich Syndrome-Long-Term Efficacy and Genotoxicity re Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry","document_id":"FDA-2018-D-2173-0076"}],"comments_count":0,"agency_id":"FDA","comments_url":"https://www.regulations.gov/docketBrowser?rpp=50&so=DESC&sb=postedDate&po=0&dct=PS&D=FDA-2018-D-2173","supporting_documents_count":71,"docket_id":"FDA-2018-D-2173","document_id":"FDA-2018-D-2173-0001","regulation_id_number":null,"title":"Observing Subjects for Delayed Adverse Events Following Administration of Human Gene Therapy Products; Availability","checked_regulationsdotgov_at":"2023-03-01T08:37:09Z"},"regulations_dot_gov_url":null,"significant":null,"signing_date":null,"start_page":32311,"subtype":null,"title":"Long Term Follow-Up After Administration of Human Gene Therapy Products; Draft Guidance for Industry; Availability","toc_doc":"Long Term Follow-Up After Administration of Human Gene Therapy Products","toc_subject":"Guidance:","topics":[],"type":"Notice","volume":83}