Core Outcome Measures in Effectiveness Trials

CoreHEM - Core Outcomes for Evaluating the Comparative Effectiveness of Gene Therapy in Hemophilia (Haemophilia)

General Information

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. The goal of this project is to develop a clearly defined set of core outcomes to measure, demonstrate and differentiate the effectiveness and value of gene therapy in hemophilia.

Center for Medical Technology Policy, Green Park Collaborative (principal supervisors)
[Elizabeth Clearfield:]
National Hemophilia Foundation
Mark Skinner JD, Institute for Policy Advancement Ltd
Alfonso Iorio MD, Health Information Research Unit, McMaster University

Further Study Information

Current Stage:
May 2017 - December 2017
Funding source(s):
National Hemophilia Foundation, Bayer, BioMarin, Freeline Therapeutics, Pfizer, Shire, Spark Pharmaceuticals, uniQure, Others (TBD)

Health Area

Disease Category
Blood disorders

Disease Name
Haemophilia and other bleeding disorders

Target Population

Age Range
0 - 100


Nature / type of Intervention
Gene therapy


Consensus meeting
Delphi process
Literature review

Hemophilia is an inherited bleeding disorder, caused by a deficiency of coagulation factor VIII (hemophilia A) or factor IX (hemophilia B). Persons living with hemophilia (PWH) experience various degrees of bleeding, depending on residual coagulation factor levels. Bleeding occurs most commonly in joints, soft tissue and muscles, causing short-term symptoms (acute bleeding, acute pain) and long-term-complications (chronic pain, hemophilia arthropathy, or disability). Acute and chronic complications result in a major impact on health-related quality of life (HRQoL) of PWH.

The core outcome set will be developed through a consensus process utilizing online Delphi voting and culminating in an in-person consensus meeting. Literature review and key informant interviews will be used to create a list of outcome domains and corresponding outcome measurement instruments; a Steering Committee will review and provide recommendations for additional outcomes to consider. The Delphi process will be used to condense and prioritize the outcome list and facilitate agreement in the measurement and reporting of each selected outcome. The in-person consensus meeting will include a final round of Delphi voting to confirm the recommended core outcome set.

Stakeholders Involved

Clinical experts
Consumers (caregivers)
Consumers (patients)
Governmental agencies
Patient/ support group representatives
Pharmaceutical industry representatives
Regulatory agency representatives

Study Type

COS for clinical trials or clinical research
COS for practice

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