The coreHEM initiative has announced the publication of a core outcome set for clinical trials of gene therapy in hemophilia. The outcomes were developed and agreed upon by a multi-stakeholder group, through an intensive research and formal consensus process. Participating experts and stakeholders included patients, clinicians, payers, health technology assessment groups, regulators, life sciences companies and others. coreHEM has produced the first set of guidelines recommending a specific, minimum set of outcomes to include in hemophilia gene therapy clinical trials. This core set will ensure that patient perspectives on critical outcomes are included in pivotal trials, allow fair comparisons between alternative treatments, and allow more accurate assessments of the value of these therapies. The coreHEM project was jointly led by the Green Park Collaborative (GPC), the National Hemophilia Foundation (NHF), and McMaster University.
The final report, “coreHEM: Developing Comparative Effectiveness Outcomes for Gene Therapy in Hemophilia,” details the methods, results, and impact identified by the initiative, as well as the final core outcome set, and preliminary work on measurements and instruments identified for those outcomes. Research from the coreHEM initiative was published today in Haemophilia. “Core outcome set for gene therapy in haemophilia: Results of the coreHEM multistakeholder project.” (Iorio A, Skinner MW, Clearfield E,et al.; for the coreHEM panel. Core outcome set for gene therapy in haemophilia: Results of the coreHEM multistakeholder project. Haemophilia. 2018;00:1–6. https://doi.org/10.1111/hae.13504)
The recommended core set includes:
- frequency of bleeds,
- clotting factor activity level,
- duration of expression [of clotting factor gene],
- chronic pain,
- utilization of healthcare system (direct costs),
- mental health status.
“The core outcomes set developed through this work has been positively received by all of the participating stakeholders, and we hope that the participating life sciences companies will include these in future trials. We believe that the consensus methods developed for this project may be applicable to a large number of clinical conditions, with similarly positive results,” said Sean R. Tunis MD, one of the three principal investigators for this initiative.
Based on feedback from coreHEM’s participants, and to ensure that the core set is of optimal value to users, the project team will now develop a consensus set of recommended measures and instruments to be used for consistent and comparable assessment of the recommended core outcomes for pivotal and post-approval trials of gene therapies in hemophilia. This second phase of work will continue to be led by GPC, NHF, and McMaster University, and is expected to launch shortly.
coreHEM is a multi-stakeholder partnership run by the Green Park Collaborative, in partnership the National Hemophilia Foundation and McMaster University. coreHEM was funded by a grant from the National Hemophilia Foundation and with support from the following life science industry companies and academic gene therapy groups: Bayer AG, BioMarin Pharmaceutical Inc, Pfizer Inc, Shire Plc, Spark Therapeutics, St. Jude Children’s Research Hospital, and uniQure B.V. These sponsoring companies fully participated in the project. Additional conference support was provided by Alnylam Pharmaceuticals, Novo Nordisk, and Roche Genentech.
About the Green Park Collaborative
The Green Park Collaborative (GPC) is a major initiative of the Center for Medical Technology Policy, an independent 501(c)(3) non-profit organization dedicated to improving the quality, relevance, and efficiency of clinical research. GPC is a multi-stakeholder forum for developing condition- and technology-specific study design recommendations to guide the creation of evidence needed to inform both clinical and payment decisions.
About the National Hemophilia Foundation
The National Hemophilia Foundation (NHF) is a 501(c)(3) non-profit organization dedicated to finding better treatments and cures for inheritable bleeding disorders and to preventing the complications of these disorders through education, advocacy and research. NHF’s programs and initiatives are made possible through the generosity of individuals, corporations and foundations, as well as through a cooperative agreement with the Centers for Disease Control and Prevention (CDC).
About McMaster University
McMaster University in Hamilton, Canada is one of the world's top 100 universities. This medical-doctoral, research-intensive institution has established an international reputation for evidence-based medicine, knowledge-translation research, health informatics, and problem-based learning.