A new initiative launched in 2017 will evaluate the comparative effectiveness of gene therapies in hemophilia. Called coreHEM, the endeavour is the result of the collaborative efforts of the Green Park Collaborative—a Center for Medical Technology Policy (CMTP) initiative—the National Hemophilia Foundation (NHF), and McMaster University of Canada.
Gene therapy is a promising new research area for the bleeding disorders community. Recent progresses have included the development of products which have the potential to cure hemophilia A and B. Since hemophilia is a disease which necessitates continued and frequently expensive treatment, the impact of a potentially permanent cure cannot be overstated.
coreHEM was built by an international team made up of patients, clinicians, researchers, product manufacturers, public and private payers, and international government agencies. Its role will be to establish an effectiveness guidance document that will specify a minimum set of essential outcomes for the evaluation of gene therapy efficacy in hemophilia. The initiative will also include an accompanying peer reviewed article providing recommendations for patient-related outcomes for clinical studies.
Ultimately, coreHEM will help all stakeholders make more informed decisions in order to more efficiently move gene therapy research forward. The WFH is pleased to be participating as a key stakeholder and representative of the patient voice for coreHEM.
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