Home> Multi-Stakeholder Projects> WM: Delphi Survey Project: Towards a Core Outcome Set for Waldenström Macroglobulinaemia (WM)
The HARMONY Alliance Delphi Survey Hackathon for Chronic Myeloid Leukemia (CML), Hodgkin’s Lymphoma (HL), Myeloproliferative Neoplasms (MPN) and Waldenström Macroglobulinaemia (WM) took place on 4 and 18 March 2022. Patients, clinicians, regulators and drug developers were invited to participate. The Delphi Surveys for CML, HL, MPN and WM are now closed. Consensus meetings took place in May 2022.
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At present, several innovative compounds are being tested in clinical trials in order to improve the treatment of WM. However, these trials measure different outcomes, thus questioning their comparability. In addition, the majority of clinical trials do not assess long-term side effects and their influence on the patients’ quality of life. A core outcome set (COS) may assist researchers in selecting outcomes for future trials, thereby promoting harmonization of WM studies and improving clinical management of the disease.
A COS is a minimum set of outcomes to be collected in future clinical trials which, ideally, have been agreed upon by all stakeholders. The COS is intended as an international standard to evaluate ALL treatments in clinical trials and other research settings (e.g., registry and observational studies). Using a COS will improve the comparability of clinical trials, enhance the consistency of reporting, and reduce selective reporting bias. The COS can also be incorporated into clinical guidelines and be used to improve patient management. This project aims to identify a COS for WM that will be accepted by all of HARMONY’s stakeholder groups.
The researchers will use the Delphi method to develop the COS. First, a preliminary list of about 60 outcomes was created, which is based on literature research and expert interviews. Next, a panel of stakeholders will be asked to rate the importance of each outcome on the basis of their personal experiences. Three iterative rounds are planned, using an online survey tool (COMET). After each round, the participants will receive an anonymized summary of the survey results per stakeholder group. This will allow them to revise their answers in the next round. It is expected that the group will converge towards a consensus COS after a number of iterations. The process will be stopped after pre-defined consensus criteria have been achieved. The final version of the COS will be aligned in a meeting with all participants.
Panel participants will be recruited from HARMONY Partners, including clinicians, patients, representatives from the pharmaceutical industry, and health authorities. The aim is to recruit equal participant numbers for all stakeholder groups wherever possible. This project is part of a series of Delphi studies for Hematological Malignancies that are being studied by the HARMONY Alliance. Ultimately, COS will be applicable to all Hematological Malignancies studied by the HARMONY Alliance.
Download the Delphi WM Study Protocol document to inspect the full outcome list >
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Public-private partnership for Big Data in Hematology
HARMONY and HARMONY PLUS are funded through the Innovative Medicines Initiative (IMI), Europe's largest public-private initiative aiming to speed up the development of better and safer medicines for patients. Funding is received from the IMI 2 Joint Undertaking and is listed under grant agreement for HARMONY No. 116026 and grant agreement for HARMONY PLUS No. 945406. This Joint Undertaking receives support from the European Union’s Horizon 2020 Research and Innovation Programme and the European Federation of Pharmaceutical Industries and Associations (EFPIA).
HARMONY Coordination Office
Institute of Biomedical Research of
Salamanca (IBSAL)
Salamanca, Spain
HARMONY Communications
European Hematology Association (EHA)
The Hague, The Netherlands
