Patients are helping researchers, including those at McMaster University, clarify the essential aspects of assessing the value of gene therapy in hemophilia.

The patients were part of the coreHEM initiative, which this week announced the publication of a core set of outcomes sought for clinical trials of gene therapy in hemophilia.

Patients have a central role, said Alfonso Iorio, professor of McMaster Department of Health Research Methods, Evidence and Impact and a co-principal investigator of the project.

“This core set will ensure patient perspectives on critical outcomes are included in important trials, allow fair comparisons between alternative treatments, and more accurate assessments of the value of these therapies,” said Iorio.

The outcomes were developed and agreed upon by a multi-stakeholder group, through an intensive research and formal consensus process.  Besides the patients, other key participating stakeholders included clinicians, payers, health technology assessment groups, regulators, life sciences companies and others.

The coreHEM project is jointly led by McMaster University, the U.S. National Hemophilia Foundation and the Green Park Collaborative, an American non-profit organization dedicated to improving clinical research.

The proposed set of measurements for hemophilia gene therapy clinical trials includes frequency of bleeds; clotting factor activity level; duration of the clotting factor gene; chronic pain; use of the health care system and mental health status.

The final report, “coreHEM: Developing Comparative Effectiveness Outcomes for Gene Therapy in Hemophilia,” details the methods, results, and expected 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 the journal Haemophilia.

The coreHem team will now begin a second phase of the work to develop the required instruments to measure in practice the proposed outcomes.

The coreHEM initiative 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. The sponsoring companies fully participated in the project.