Naccache M, Konkle B, Peyvandi F, Miesbach W, O’Mahony B, Pile S, Youttananukorn T, Coffin D, Pierce G. Global Efforts in Uniting all stakeholders in ensuring safety of hemophilia gene therapy patients: the World Federation of Hemophilia Gene Therapy Registry. (2024), POSTER ABSTRACT (MP-021). (2024), Issue Information. Haemophilia, 30: 1-2. https://doi.org/10.1111/hae.15016
Introduction
The World Federation of Haemophilia (WFH) launched the Gene Therapy Registry (GTR) aimed at gathering comprehensive data on all people with haemophilia (PWH) who receive gene therapy worldwide.
Methods
The GTR was designed to standardise and centralise global data collection for the gathering and dissemination of gene therapy data. The GTR is a prospective, observational, and longitudinal registry. Data entry occurs once, either directly from haemophilia treatment centre (HTC) into the GTR or through data transfer from National Registries. The GTR Scientific Advisory Board oversees all data entered into the GTR and its dissemination. Specificde-identified data will be shared with various stakeholders: participating patients will have access to view their own data; HTCs and National Registries will receive aggregated global safety data; industry part-ners will receive product-specific data, and regulatory agencies and health technology assessment organisations can request specific data to inform their decisions.
Results
The WFH is engaged with a broad network of collabora-tors, and the GTR National Registries & HTC Consortium has been established to foster dialogue, obtain feedback from our collaborators, and establish mutually beneficial collaboration. This group includes representatives from Brazil, Ireland, Saudi Arabia and Sweden, and registries from Australia, Canada, France, Germany, Japan, the Netherlands, Spain, the United Kingdom and the United States. The European Medicines Agency (EMA) issued a pivotal letter of support to the WFHGTR. The CHMP endorses the GTR as the worldwide registry for consolidating all international data on PWH who receive gene therapy and encourages collaboration of all HTCs and National Registries, stating that the WFH GTR is of particular value for post approval safety and efficacy studies of gene therapies and recommending its use as a planned data source for mandated Phase IV studies.
Conclusions
The GTR facilitates the accumulation of data in one registry and supports the efficient dissemination of valuable information to all stakeholders, advancing our understanding of gene therapy’s safety, efficacy, and long-term effects. In the current landscape of numerous registries in haemophilia around the world, the success of the GTR depends on collaborative relationships with all stakeholders, including patients, HTCs, National Registries, industry partners and regulatory bodies.
Disclosures
Barbara Konkle: Be Biotherapy, Biomarin, Novo Nordisk, Pfizer, Sanofi; Flora Peyvandi: BioMarin Pharmaceutical Inc., CSL Behring, F. Hoffmann-La Roche Ltd., Grifols, Sanofi, Sobi, Takeda; Wolfgang Miesbach: Bayer, BioMarin, Biotest, Chugai, CSL Behring, Free-line, LFB, Novo Nordisk, Octapharma, Pfizer, Regeneron, Roche, Sanofi, Sigilon, Sobi, Takeda/shire; Brian O’Mahony: Biomarin, CSL Behring, Freeline, Irish Haemophilia Society, Pfizer, Roche; Mike Makris: Grifols, Novo Nordisk, Sanofi, Takeda; Steven Pipe: Apcintex, ASC Therapeutics, Bayer, BioMarin, CSL Behring, HEMA Biologics, Freeline, LFB, Novo Nordisk, Pfizer, Regeneron/Intellia, Genentech/Roche, GeneVentiv and Equilibra Bioscience, Sanofi, Takeda, Spark Therapeutics, uniQure, Siemens; Mark Skinner: Band Therapeutics, Bayer, BioMarin, CSL Behring, Novo Nordisk, Pfizer, Roche/Genentech, Sanofi, Takeda, Vega Therapeutics; Glenn F. Pierce: ASC Therapeutics, Be Bio, BioMarin, Decibel Therapeutics, Frontera, Intellia, Metagenomi, Novo Nordisk, Pfizer, Regeneron, Spark Therapeutics, Third Rock Ventures.
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