WP2 is focused on defining the specific questions that are relevant to each hematological malignancy (HM). The coordination of the data that emerges will ensure that results will be generally applicable to patients with blood cancers. WP2 will define what outcomes need to be achieved in terms of each type of blood malignancy, including both clinical outcome measures and the description of which factors are relevant for both patients and caregivers. Check the HARMONY Research Projects >
Read about the Delphi Survey to Develop a Core Outcome Set (COS) for Acute Myeloid Leukemia. A COS constitutes the minimum set of outcomes that should be collected and reported in future clinical trials. Download the Delphi AML Study Protocol document to read more and to have an overview of the full outcome list in the annex.
- Reviewing all the literature that has been published about defining outcomes and how these outcomes have been measured and reported on in the past;
- Identifying overarching questions/aims/goals in order to align core standard outcomes sets under the guidance of key opinion leaders (KOLs) and other stakeholders;
- Aligning core standard outcome sets with the relevant payers, providers, regulatory and health technology assessment (HTAs) agencies in order to coordinate the work with maximum efficiency;
- Contributing to the resolution of legal issues that might arise from data sharing and data storage and from European meta-analyses (in close collaboration with WP3, WP4, WP6, and WP8);
- Facilitating collaboration with other groups, thus enabling all of the work to be aligned and coordinated;
- Defining the most important problems that should be addressed by HARMONY and specifying out expected outcomes.
AEMPS, AIEOP, Amgen, Barts Health, Bayer, Celgene, BfArM, Erasmus MC, ERIC, FISM Onlus, GFM, Goethe University, GPOH, GRAALL, HHU, HULAFE, IECSCYL-IBSAL, IJC, Janssen, LeukaNET, LMU, LYSA, Menarini, MU, Newcastle University, NICE, Novartis, OPBG, SYNAPSE, Takeda, University of Ulm, UNAM, UNIBO, UNITO, University of Cambridge, University of Navarra, University of Rome Tor Vergata, VHIO, VUMC.
WP2 has identified big data sets relevant to HARMONY, contributed to the platform set-up, initiated an AML pilot study and embarked on the definition of standard core outcome sets for hematologic malignancies. WP2 has made considerable progress in assembling and categorizing critical information about HMs:
- Established procedures for governing the workflows, and created an e-consultation tool for stakeholders;
- Besides identifying the data sets related to acute myeloid leukemia (AML) and mapping out the data sources required for a pilot run of the platform => the first two academic data sets were entered into HARMONY and the first EFPIA one will follow by the end of the year;
- WP2 also initiated a number of additional pilot projects (e.g. in MM, CLL and ALL);
- Implemented elements of the Phase 2 reimbursement decisions (products were selected across disease pillars; submission documents were identified; an extraction tool was developed, and pilot methods and tools were created);
- Set up mechanisms to de-identify data and to achieve “de-facto anonymization” and double-brokerage pseudonymization;
- Created descriptions of technical concepts related to handling data, such as “de-facto anonymization” and “hashing”; and
- Drafted a number of associated member engagement framework agreements.
- Scientific, Cross-Indication HM Sectorial sessions have been organized;
- ALL: Information has been collected in on available data sets and have been organized by genetic abnormalities;
- AML: Information, data sets on 5.000 patients, is available and will be used for the pilot test in 2018;
- CLL: 3700 cases (with genetic annotations) have been collected from ERIC/CLL datasets available for HARMONY's pilot project: potentially 8000 additional cases can be added (with a two-year follow-up being planned);
- MDS: Datasets of 1500 cases with molecular data for patients at high risk for MDS have been identified;
- MM: The R-ISS database (European Myeloma Network), including about 4500 patients, is ready to be used in a HARMONY pilot test;
- NHL: dataset for 1523 patients identified for T-NHL;
- Pediatric HMs: An overview of the completed data set has been completed and can be potentially included in HARMONY's work (based on more than 7500 patients).
In 2017, the team of WP2 has mapped the data sources to the pilot run (in collaboration with WP3); reached a consensus on data de-identification using a “de-facto anonymization” or double-brokerage pseudonymization, and outlined the description of technical concepts such as "pseudonymization" and “hashing” (in close interaction with WP8). Finally, in collaboration with WP1, the Associated Member Agreements were put in place.
WP2 will continue to focus on:
- To conduct additional HM pilot studies in relation to, for example, myelodysplastic syndromes (MDS) (evaluation of the impact of novel compounds); and non-Hodgkin’s lymphoma (NHL);
- To define novel projects in all 7 Hematological Malignancies;
- To create and implement a number of horizontal projects linking different disease groups (such as high-risk myelodysplastic syndromes (MDS), childhood AML, and AML);
- To refine, in collaboration with other WPs, the processes involved in data entry, data analysis, and data interpretation;
- To continue the joint WP2/WP6 process of defining “core outcome data sets”.
Download our deliverables for an extended view on WP2:
Work Package Leadership