Recommendations for a minimum data set for monitoring gene therapy in hemophilia: communication from the ISTH SSC Working Group on Gene Therapy.

Independent data collection is crucial in addressing the challenges associated with gene therapy for hemophilia, which is a promising treatment option but requires careful monitoring and management of short-term and potential long-term safety concerns. The International Society on Thrombosis and Haemostasis has identified a minimum efficacy and safety data set included in the World Federation of Hemophilia Gene Therapy Registry that should be collected on a national basis at specific time points for each patient who has been treated with the gene therapy products. This Gene Therapy Minimum Data Set (GT-MDS) was developed to facilitate data collection and to ensure capturing the most relevant data and most known and unknown safety and efficacy parameters recently cited by the European Medicine Agencies. The concept of assembling a minimum data set is not about creating a new data set but rather about identifying a subset of critical and essential topics that should always be included. The GT-MDS is structured into 3 sections and comprises an abridged list of 6 topics during routine gene therapy follow-up, keeping the number of data points low but allowing for rapid and independent data evaluation. The World Federation of Hemophilia Gene Therapy Registry data set, developed by the World Federation of Hemophilia, the International Society on Thrombosis and Haemostasis, and other organizations, including industry partners in 2020, is comprehensive. The GT-MDS reports the minimum relevant information that should not be lost and is mandatory to be collected for all patients who undergo gene therapy. Therefore, the implementation of the gene therapy registry and the minimum data set empowers and enhances data collection at a global level.
Competing Interests: Declaration of competing interests W.M.: Bayer, BioMarin, Biotest, CSL Behring, Chugai, Freeline, LFB, Novo Nordisk, Octapharma, Pfizer, Regeneron, Roche, Sanofi, Sigilon, Sobi, Takeda/Shire, uniQure. B.K.: Grant/research support from CSL Behring, Pfizer, Sanofi, Spark Therapeutics, Takeda, and uniQure; Consultant fees from Be Biopharma, BioMarin, Regeneron, Pfizer, Sanofi; Leadership roles: World Federation for Hemophilia, Foundation for Women and Girls with Blood Disorders. P.C.: Bayer, Boehringer Ingelheim, CSL Behring, Chugai, Freeline, NovoNordisk, Pfizer, Roche, Sanofi, Spark, Sobi, and Takeda. R.K.: Grants from Bayer; consulting fees from BioMarin, Pfizer; honoraria for lectures from Pfizer; participation on a data safety monitoring board or advisory board: BioMarin, Pfizer. F.L.: Grant/research support from CSL Behring, Takeda, uniQure, Sobi; consultant fees from uniQure, Sobi, BioMarin; Research support from CLE Behring, Takeda, Sobi, uniQure. J.M.: Grant/research support from BioMarin, Novartis, Novo Nordisk, Pfizer, F. Hoffmann-La Roche Ltd, Sanofi, Spark Therapeutics, and uniQure; consultant fees for BioMarin, Novo Nordisk, F. Hoffmann-La Roche Ltd, Sanofi, Spark Therapeutics and Takeda; Speaker bureau fees from Novo Nordisk, Pfizer, F. Hoffmann-La Roche Ltd, Sanofi, Takeda, WFH, and ISTH. M.M.: Leadership roles: EAHAD council member 2018-2022. ISTH council member 2018-2022. S.W.P.: Consulting fees from Apcintex, ASC Therapeutics, Bayer, BioMarin, CSL Behring, Equilibra Bioscience, HEMA Biologics, Freeline, LFB, Novo Nordisk, Pfizer, Regeneron/Intellia, Roche/Genentech, Sanofi, Takeda, Spark Therapeutics, and uniQure; Scientific Advisory Board member: Equilibra Bioscience, GeneVentiv. G.P.: consultant to ASC Therapeutics, BioMarin, Pfizer, Regeneron, and Spark Therapeutics and on the scientific advisory boards of Be Bio, Frontera, and Metagenomic. A.S.: speaker: Novo Nordisk, Roche, Sanofi, Takeda, Octapharma, and BioMarin; advisory board: Novo Nordisk, Roche, Sanofi, Takeda, Pfizer, BioMarin, and Spark; research grant: Roche, Novo Nordisk, Sanofi, and Pfizer. J.V.: no personal fees. F.P.: consulting fees from CSL Behring, Biomarin, Roche, Sobi, and Sanofi; payment or honoraria for lectures: Takeda and Spark.
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