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Developing Comparative Effectiveness Outcomes for Gene Therapy in Hemophilia

Recently, several gene therapy trials for hemophilia, an inherited blood disorder in which the blood does not clot properly, have announced encouraging results demonstrating the prospect of gene therapy to yield a long-term “cure” for those living with the disease. This project aims to develop a set of clearly defined, “core set” of outcomes to measure, demonstrate, and differentiate the effectiveness and value of gene therapy in hemophilia. The inclusion of these measures in clinical development programs will maximize the likelihood that effective, high value therapies will be rapidly available to appropriate patients. 

One benefit of defining and uniformly implementing a set of outcomes that would be included across trials and gene therapy products would be to increase the predictability and consistency of the assessment and appraisal of these treatments by payers and HTA organizations when making coverage and reimbursement decisions. We are including all relevant stakeholders in this initiative, especially patients, to ensure that the resulting outcome sets include benefits that are meaningful to patients receiving this life-changing therapy. 

GPC is teaming with the National Hemophilia Foundation (NHF) and McMaster University of Canada to design and carry out this initiative.  

This initiative will involve a broad, multi-stakeholder participant group. Currently, there are opportunities for participants, sponsors, and steering committee members.

Steering Committee
Composed of stakeholders in policy or key decision-making roles with expertise and knowledge in drug development, coverage and reimbursement, regulation, patient treatment and care, guidelines development, or other roles relevant to hemophilia treatment and gene therapy research. This group will provide guidance on the overall project and approach; participate in the Delphi process, which will identify important outcomes, measurement and reporting tools in hemophilia gene therapy research; assist with the planning of a large in-person stakeholder meeting; and discuss dissemination plans for the final set of recommendations and associated work products.

Project Stakeholders
Individuals with expertise and knowledge in drug development, coverage and reimbursement, regulation, patient treatment and care, guidelines development, clinical trials and methodology relevant to hemophilia treatment and gene therapy research, including patients with hemophilia or parents/caregivers of patients with hemophilia. Participants will participate in the Delphi process which will identify important outcomes, measurement and reporting tools in hemophilia gene therapy research; attend an in-person multi-stakeholder meeting; and will provide feedback on the project report and final set of recommendations prior to wide distribution.

Contact Jennifer Al Naber for further details.

Current sponsors include:

  • Winter 2017: Recruit project participants and sponsors
  • Spring – Early Summer 2017: Background research and key informant interviews
  • Summer 2017: Steering Committee and Project Stakeholder kick-off calls
  • Summer 2017 – Early Fall: Delphi rounds 1 and 2
  • Fall 2017: Draft briefing document 
  • November 2017: In-person meeting
  • Early 2018: Produce white paper and publications

The goals for coreHEM are:

  • To clearly define a “core set” of outcomes to measure, demonstrate and differentiate the effectiveness and value of gene therapy in hemophilia. 
  • To provide patient-important outcomes benchmarks that establish the framework for subsequent payer / HTA assessments.