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HARMONY

Healthcare Alliance for Resourceful Medicines Offensive against Neoplasms in HematologY

Abstract

Despite significant recent progress in the field of hematological malignancies (HMs), with increasing survival rates and improvement in quality of life, many children and adults with HMs still die from these disorders or experience disabling complications. Therefore, improvement of health care of HMs is an unmet medical need. Thus, it is important to define and align standard and efficient sets of HMs outcomes to measure and evaluate HM data for clinical decisions, long term risk/benefit profile, reimbursement, value analysis, and clinical trials design. Improving outcome measures and endpoint definitions by taking into account “real-life” data and differences in cross-national healthcare practice will undoubtedly result in an optimized, sustainable and effective treatment delivery, as well as in desirable and innovative accelerated pathways for novel drug availability. All these challenges will be addressed within a pan-EU perspective by HARMONY (Healthcare Alliance for Resourceful Medicines Offensive against Neoplasms in HematologY), a comprehensive public-private European consortium of excellence. HARMONY consortium is made up of 51 partners: 44 participants from 10 European countries and 7 pharmaceutical companies from the EFPIA. HARMONY aims to assemble, assess, connect, and analyze heterogeneous HM patient derived Big Data sets to define sets of outcome indicators that can be used for decision-making by key healthcare stakeholders. The consortium will orchestrate leading experts and working cooperative groups in HMs, European study alliances, pharmaceutical market leaders, patient advocacy groups, HTA and regulatory agencies, to: (i) optimize Europe-wide data collection and create a high-quality HM data repository for further explorative studies; (ii) establish a clinical data-sharing platform that empowers clinicians, patients and policy stakeholders to improve decision-making procedures and identify appropriate treatments to patients with HMs (iii) foster the design of innovative clinical and pre-clinical studies; (iv) define meaningful and harmonized clinical endpoints and outcomes in HMs in order to facilitate clinical decision-making; (v) identify specific biomarkers, which better define outcome parameters (vi) provide novel resources and algorithms to more rapidly advance innovative concepts of patient management in HMs; (vii) involve the patient perspective; (viii) provide a framework for legal, ethical and governance issues. Moreover, the consortium will integrate molecular markers to improve patient outcomes and personalized health management processes. Regarding governance, HARMONY has a multi-level organizational structure, which includes representatives from all stakeholders, and which, ensures efficient and effective management, decision-making, and control of work package (WP) activities. This will be accomplished by creating well-defined management and decision-making structures with clear accountabilities and decision rules. The Consortium Management Bodies will be the Executive Committee, the Steering Committee, and the General Assembly. The management of Big Data is one of the key-features in the consortium. Therefore a Data Quality Supervision Committee will define the quality criteria for “valid records” and perform the review of the quality of the patients’ data provided by different sources (beneficiaries, Associated Members and Co-operative Working Groups). HARMONY is covering seven HMs (multiple myeloma, acute myeloid leukemia, acute lymphoblastic leukemia, chronic lymphocytic leukemia, non-Hodgkin’s lymphoma, myelodysplastic syndromes and pediatric HMs) structured in eight WPs, including: i) the most important key opinion leaders in each disease; ii) several European institutions involved in the study of HMs; iii) the EFPIA lead companies in HMs; iv) institutions experienced in studying “omics” data; v) a large panel of stakeholders (regulatory agencies, HTA, and patients organizations) and; vi) the most active working cooperative groups in HMs. The expected outcomes from HARMONY will be: i) to ensure meaningful patient relevant outcomes are respected and aligned with regulators and health technology assessment bodies to speed up patient access to treatments; ii) to create a comprehensive data-sharing platform that enables data capturing and represents a repository of representative and relevant HM data; iii) to publish defined sets of outcomes, including clinical endpoints and patients’ perception of their quality of life to be recorded for selected HMs; iv) to develop analytical tools (algorithms, methods and software) for complex data sets (e.g. to link molecular and clinical data); v) to harmonize clinical endpoints and methods to implement clinical interventions in a common pan-European database; vi) to integrate “real-life” clinical data into drug development; vii) to develop a set of specific markers (e.g. molecular, imaging, minimal residual disease) to better define outcome parameters. A “Proof-of-Concept” study, based on the analysis of a large series of patients with the diagnosis of acute myeloid leukemia (AML), has been designed. This will be jointly conducted by WPs 2-6 and WP 8 to “define patients with AML who are suitable for intensive therapy and who benefit from stem cell transplantation in first complete remission”. The Key Opinion Leaders will define outcome measures in collaboration with members of WP6, including molecular markers and quality of life. Around 5,000 AML data sets can be identified and anonymized. Access to this novel data repository platform will follow data quality control and subsequent approval from data custodians. This data set will then also provide a good means to test the data analysis algorithms to be used in WP5. Next, KOLs will identify and gather additional data from other clinical trials, other co-operative groups, and “real-life” data to evaluate novel findings in independent cohorts before results are prospectively tested within new and existing trials. Finally, a sustainability plan will ensure a long-lasting impact of HARMONY as it will include: i) the use of the HARMONY database for future analysis by the European Health Systems, scientists, regulators and Industry; ii) that HARMONY is open to include additional HM diseases, which can enhance the goals of the project; iii) the transfer of the HARMONY Proof-of-Concept findings along with the HARMONY tools and algorithms to other non-hematologic malignancies and rare diseases, both within the EU, but also to non-EU countries. Ultimately, HARMONY will offer an unprecedented opportunity to explore HM subgroups and to generate significant epidemiological information that can be further used to distil the impact of the environment, and genetic background on both HM development and treatment response. Thus, the HARMONY network of excellence will form the basis for value-based and outcomes-focused sustainable high quality HM care in Europe.

Project details

Unibo Team Leader: Giovanni Martinelli

Unibo involved Department/s:
Dipartimento di Fisica e Astronomia "Augusto Righi"

Coordinator:
Fundación Instituto de Estudios de Ciencias de la Salud de Castilla y León(Spain)

Other Participants:
Takeda Cambridge Ltd. (United Kingdom)
University of Navarra (Spain)
Josep Carreras Leukaemia Research Institute (IJC)-José Carreras International Foundation (Spain)
GMV Soluciones Globales Internet S.A.U. (Spain)
Agencia Española del Medicamento y Productos Sanitarios (Spain)
Fondacion Para La Investigacion Del Hospital Universitario La Fe De La Comunidad Valenciana (Hulafe) (Spain)
University Of Cambridge (United Kingdom)
Genome Research Limited (United Kingdom)
Vib Nucleomics Core (Belgium)
Leukanet e.V. (Germany)
Gpoh-Gesellschaft Fur Padiatrische Onkologie Und Hamatologie Ggmh (Germany)
European Research Initiative on CLL e.V. (Germany)
ALMA MATER STUDIORUM - Università di Bologna (Italy)
University Of York (United Kingdom)
Eortc Aisbl/Ivzw (Belgium)
MediSapiens Oy (Finland)
Assistance Publique - Hopitaux De Paris (France)
European LeukemiaNet Foundation-ELN-FOUNDATION (Germany)
Università degli Studi di ROMA Tor Vergata (Italy)
National Institute For Health And Care Excellence-Nice (United Kingdom)
Newcastle University (United Kingdom)
Fondazione Italiana Sindromi Mielodisplastiche-FISM onlus (Italy)
Ebmt European Group For Blood And Marrow Transplantation (Netherlands)
Medizinische Universitat Wien (Austria)
Janssen Pharmaceutica Nv (Belgium)
Masarykova Univerzita (Czech Republic)
Heinrich-Heine-Universität Düsseldorf (Germany)
Johann Wolfgang Goethe - Universitaet Frankfurt Am Main (Germany)
A.Menarini - Ind. Farm.Riunite - S.R.L. (Italy)
Ospedale Pediatrico Bambino Gesu' (Italy)
European Hematology Association (Netherlands)
Synapse Research Management Partners Sl (Spain)
Fundacio Privada Institut D'Investigacio Oncologica De Vall-Hebron (Spain)
Novartis Pharma Ag (Switzerland)
Barts Cancer Institute QMUL (United Kingdom)
European Alliance for Personalised Medicine (Belgium)
Groupe Francophone des Myélodysplasies (France)
Ludwig-Maximilians-Universitat Munchen (Lmu) (Germany)
Universitaet Ulm (Germany)
Erasmus Universitair Medisch Centrum Rotterdam (Netherlands)
The Lymphoma Scientific Association (France)
Bayer Ag (Germany)
Bundesinstitut für Arzneimittel und Medizinprodukte (Germany)
Università degli Studi di TORINO (Italy)
Amgen Limited (United Kingdom)
Mll - Munchner Leukamielabor Gmbh (Germany)
Stichting Vu (Netherlands)
Celgene Management Sarl (Switzerland)
Helsingin Yliopisto (Finland)
Group for Research on Adult Acute Lymphoblastic Leukemia-GRAALL (France)

Total Eu Contribution: Euro (EUR) 20.200.000,00
Project Duration in months: 60
Start Date: 01/01/2017
End Date: 31/12/2021

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