Core Outcome Measures in Effectiveness Trials

Core outcome set for gene therapy in haemophilia (hemophilia): Results of the coreHEM multistakeholder project

General Information

Abstract:
Background
Gene therapy trial results show potential to cure haemophilia A and haemophilia B. Securing broad access to a cure for a lifelong chronic disease is anticipated to face barriers at the individual and healthcare system levels, which can be partly mitigated by harmonized planning of clinical research studies. The aim of the coreHEM project was to determine the set of outcome measures required to evaluate efficacy, safety, comparative effectiveness and value of gene therapy for haemophilia.

Methods
Modified Delphi consensus process, based on methods adapted from the COMET Initiative.

Results
Forty-nine participants (five patients, five clinicians, five researchers, four regulators, three research agencies, six health technology assessors, nine payers and 12 drug developers) took part in the study, with over 90% participation. The frequency of bleeds, factor activity level, duration of expression, chronic pain, healthcare resource use and mental health were identified as the core outcomes to be measured in addition to regulatory-mandated adverse effects.

Conclusions
For the first time in haemophilia, a core outcome set has been developed, with the involvement of representatives of all relevant stakeholder groups. The core set has been expanded to include outcomes supporting assessment of comparative effectiveness and value, with the goal of streamlining regulatory approval, health technology assessment and market access decisions. Patient involvement ensures that outcomes are meaningful and relevant to those living with haemophilia. Active dialogue among drug developers, regulators and payers throughout the process is expected to facilitate broad uptake of the core outcomes in forthcoming clinical trials.

Authors:
Alfonso Iorio
Mark Skinner
Elizabeth Clearfield: elizabeth.clearfield@cmtpnet.org
D Messner
GF Pierce
M Witkop
S Tunis
coreHEM panel




Publication

Journal:
Haemophilia
Volume:
Issue:
Pages:
-
Year:
2018
DOI:
Further Study Information

Date:
May 2017 - December 2017
Funding source(s):
National Hemophilia Foundation, Bayer, BioMarin Pharmaceutical, Pfizer, Shire, Spark Therapeutics, St. Jude Children’s Research Hospital, uniQure

Health Area

Disease Category
Blood disorders

Disease Name
Haemophilia/hemophilia and other bleeding disorders

Target Population

Age Range
0 - 100

Sex
Either


Nature / type of Intervention
Gene therapy

Method(s)

Consensus meeting
Delphi process
Interview
Literature review

Stakeholders Involved

Clinical experts
Consumers (patients)
Funders
Governmental agencies
Other
Pharmaceutical industry representatives
Regulatory agency representatives
Researchers

Study Type

COS for clinical trials or clinical research

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