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Healthcare Alliance for Resourceful Medicines Offensive against Neoplasms in HematologY - Sofia ref.: 116026

Periodic Reporting for period 4 - HARMONY (Healthcare Alliance for Resourceful Medicines Offensive against Neoplasms in HematologY - Sofia ref.: 116026)

Reporting period: 2020-01-01 to 2020-12-31

Over the last 25 years, academic and pharmaceutical research has made significant progress, increasing Hematologic Malignancies (HM) patients’ survival rates and improving quality of life. However, a substantial proportion of patients with HM still have a poor prognosis due to relapse and refractory disease. In many cases, potentially life-saving treatment decisions need to be made quickly, hence current decision-making processes need to be speeded up. In addition, the advances in the molecular characterization of HMs achieved in the past years have revealed them to be genetically complex and heterogeneous diseases, translating into highly divergent clinical behaviour. This heterogeneity of HMs makes improving patients’ outcome even more challenging.

Progress along these lines is further complicated by the heterogeneity and diversity among European healthcare systems and oncology practices across the EU. This results in differences in the treatment of patients throughout Europe and limits the direct comparability of the clinical and non-clinical data on HMs that are being collected.

Therefore, there is an unmet need to harmonize outcome measures and endpoint definitions for HMs and their collection at the European level. HARMONY is designed to achieve this by uniting and aligning all European stakeholders.
The HARMONY Alliance is the largest Innovative Medicines Initiative European public-private partnership aiming to improve outcomes of patients with HMs by establishing consensus outcomes among all stakeholders through the analysis of data on a large Big Data platform. HARMONY is composed of 53 partners from 11 countries including the pharmaceutical industry, hospitals, academic, research institutions, regulatory agencies, health technology assessment bodies, and patients’ organizations. HMs within scope are Acute Myeloid Leukaemia (AML), Acute Lymphoblastic Leukaemia (ALL), Chronic Lymphocytic Leukaemia (CLL), Multiple Myeloma (MM), Myelodysplastic Syndrome (MDS), Non-Hodgkin Lymphoma (NHL) and paediatric HMs.

By creating a Big Data platform, the project aims to improve the access to a repository of relevant Data from HM patients. The magnitude of the data in this unique repository will help to represent the diversity of HM diseases in relation to their pathological, medical, epidemiological, healthcare and societal scope. The platform provides the technological requirements and governance to allow these data sets to be shared and queried by medical and non-medical experts involved in the healthcare provision and management of HMs, thus enhancing their value.
Over the past 4 years, HARMONY has evolved to become one of the Europe's leading initiatives for Big Data in healthcare. With the support of 100 organizations, data from more than 60,000 patients with blood cancer have been identified and more than 26,500 data have been shared in diseases like ALL, AML, CLL, MDS, MM, and NHL from both public and private providers across all the EU. The HARMONY Big Data Platform processes this data to generate evidence that can be used to answer critical questions in hematology.

The results of the studies in AML and MM have been presented in international conferences. In AML, HARMONY has analysed genomic data of thousands of patients, confirming the co-occurrence of certain mutations and determining which ones occur first and which later in the disease process. Moreover, HARMONY has studied the association of mutation patterns with clinical outcomes such as remission, relapse, and survival.

Using data from more than 7,000 patients, HARMONY discovered that MM patients who are currently considered as ‘intermediate-risk patients’ constitute a quite diverse group with varying risk of progression or death. About half of them can be classified as low or low-intermediate risk, and the other half as intermediate-high or high risk.

To improve the protocols for clinical trial design and processes, HARMONY aims to define a consensus core outcome set (COS) applicable to all HMs and to define a specific outcome set for each HM, so that all studies in a particular HM could be compared and combined. Thus, we could: i) to get a better understanding of which treatments are best; ii) avoid the problem of some studies only report a selection of the measured outcomes.

To decide which outcomes should be core for each HM, several Delphi Surveys have been launched involving the input of different stakeholders. In 2019 the AML COS Delphi program was launched. A 2nd round was started in 2020 together with Delphis for MDS, MM and NHL. CLL and ALL Delphis will start in 2021.

HARMONY has a wide range of expertise in evidence development strategies to support new treatments and indications. As a result, a new guide (https://cms.harmony-alliance.eu/cgi-bin/itworx/download.cgi?vid=638&uid=-1&dokid=197) has been produced to ease the access to market for innovative HM drugs to improve the current treatments in HMs.

HARMONY is continuously in expansion and proposals for collaboration arrive from all over the world. As a result, the number of Associated Members is continuously raising (from 15 in 2017 to 42 by the end of 2020).

Communications are boosted every year to increase global awareness of HARMONY’S achievements and its ongoing work, to ensure that knowledge of HARMONY’S work reaches out an international audience.
HARMONY aims to establish the efficacy and safety of the early diagnostics and clinical stratification, and the impact that it would have on response to therapy, patient survival and quality of life.
HARMONY will impact on distinct levels:

- The patient and patient advocacy organizations
Patients shall benefit from: i) linking diagnostic and molecular profiling platforms and ongoing trials to facilitate enrolment into specific clinical trials; ii) faster access to innovative diagnostics and treatments; iii) early and precise diagnosis and accurate risk assessment through better-defined outcomes based on patients’ unmet needs.

- Medical and scientific community
Improved management of patients due to the enhanced ability to select the right therapy for each individual on the basis of the disease profile, by applying the HARMONY selected outcomes measures and defined models to improve patient stratification.

- Market access decision-makers: regulatory agencies, HTA
The rational and analytical framework provided by HARMONY supports harmonization on decision-making for regulatory approval and reimbursement, policy and process developments on an international level, and provides tools and techniques to guarantee the efficient collection and analysis of hematologic data.

- European market
Europe shall benefit strengthening its profile as a business location and leading geographical area in the pharmaceutical and medical/healthcare industry.
Pharmaceutical and diagnostics industry shall benefit from: i) models generating a benefit through a personalized HM approach; ii) a better understanding of patient stratification, and validation of novel endpoints that will increase efficiency in R&D.

- Socio-economics
Socio-economic benefits achieved by: i) proving the value of innovative HM therapies for the healthcare system; ii) cost savings through better identifying appropriate patients for treatment; iii) improved decision-making in the selection of therapies which can lead to improved treatment outcomes with a focus on supporting quality of life of patients.
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HARMONY logo with text
HARMONY logo