Work Package 2: Outcomes Definition

WP2 is developing Core Outcome Sets (COS) for Hematological Malignancies (HMs). COS are minimum sets of outcomes to be measured in future clinical trials in HMs, agreed upon by the stakeholders of HARMONY PLUS. These are established using Delphi Surveys. WP2 will further define the COS that were developed during the initial HARMONY action, incorporating novel technological and therapeutic developments. In addition, WP2 will develop COS for the new HMs that are included in HARMONY PLUS. This will guide novel research questions and facilitate patient-centered care and value-based healthcare systems.

Objectives

Developing COS for the HMs that were not yet covered in the initial HARMONY action, including various Myeloproliferative Disorders, Lymphoproliferative Disorders, and other rare HMs;
Defining HM-overarching COS with regard to novel therapeutic strategies such as cellular treatment approaches (e.g., CAR T cell therapies) or targeted therapies (e.g., tyrosine kinase inhibitors and BCL2 inhibitors);
Frequently adjusting COS, accounting for novel technological developments such as deep omics data based on novel digital monitoring tools for improved Patient Reported Outcome Measures, as well as novel improved diagnostic tools;
Aligning the requirements of outcome measures with various stakeholders to enable data-driven decisions by payers and regulators (e.g., reimbursement, value assessment, surrogate endpoints);
Closely collaborating with the existing HARMONY network to allow the creation of both retrospective and prospective studies within HARMONY. Developing a repository of information regarding HM patient samples across different European medical institutions and biobanks that will facilitate their tracking and inclusion in future research proposals and/or create the opportunity to speed up patient recruitment in clinical trials;
Coordinating a “proof-of-principle” for collaboration with other international big data projects and platforms such as the American Society of Hematology (ASH) Data Hub.

View the HARMONY Alliance Research Projects >
View the HARMONY Alliance Delphi Projects >

Work Package Partners

Charite (Lead), IECSCYL, Hospital La Fe, GMV, UNIBO, LeukaNET, UULM, ELN, EMC, UNAV, UNITO, VUMC, UCAM, ERIC, LYSA, LMU, GFM, FISiM-ETS, UNEW, OPBG, EBMT, NICE, AEMPS, BfArM, Celgene, Bayer, Menarini, Pfizer.