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 determine 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. 

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

The initiative involves broad, multi-stakeholder representation; individuals from the following organizations are participating:

  • 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
  • Late Summer – Early Fall 2017: Delphi rounds 1 and 2
  • Fall 2017: Draft briefing document 
  • November 14-15th 2017: In-person Consensus meeting
  • Late November 2017: Final Delphi round
  • Winter 2017: Release interim reports
  • 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.