Periodic Reporting for period 5 - HARMONY (Healthcare Alliance for Resourceful Medicines Offensive against Neoplasms in HematologY - Sofia ref.: 116026)
Okres sprawozdawczy: 2021-01-01 do 2021-12-31
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 behavior. 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 the largest Innovative Medicines Initiative (IMI) European public-private partnership aiming to improve outcomes of patients with hematological malignancies (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 leukemia (AML), acute lymphoblastic leukemia (ALL), chronic lymphocytic leukemia (CLL), multiple myeloma (MM), myelodysplastic syndrome (MDS), non-Hodgkin lymphoma (NHL) and pediatric 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.
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 behavior. 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 the largest Innovative Medicines Initiative (IMI) European public-private partnership aiming to improve outcomes of patients with hematological malignancies (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 leukemia (AML), acute lymphoblastic leukemia (ALL), chronic lymphocytic leukemia (CLL), multiple myeloma (MM), myelodysplastic syndrome (MDS), non-Hodgkin lymphoma (NHL) and pediatric 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 5 years, the HARMONY Alliance has evolved to become one of the Europe's leading initiatives for Big Data in healthcare. By the end of 2021, data from almost 100,000 patients with blood cancer have been identified and more than 62,770 data have records been shared in diseases like ALL (Acute Lymphoblastic Leukemia), AML (Acute Myeloid Leukemia), CLL (Chronic Lymphocytic Leukemia), MDS (Myelodysplastic Syndromes), MM (Multiple Myeloma), and NHL (Non-Hodgkin Lymphoma) 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.
For instance, in AML, HARMONY has studied the genomic data of thousands of patients, which confirms the co-occurrence of certain mutations, determining which ones occur first and which later in the disease process, as well as the differences between male and female patients. Moreover, the association of mutation patterns with clinical outcomes such as remission, relapse, and survival have been investigated. In addition, for unmutated CLL patients, different prognostic impact of recurrent gene mutations in IGHV-mutated and IGHV-have been studied. 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.
To improve the protocols for clinical trial design and processes, HARMONY have defined 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. COS for each HM have been gathered by launching several Delphi Surveys involving the input from different stakeholders. During 2021, Delphi surveys were successfully completed for AML, CLL, NHL, MDS and MM, while for ALL the first round has also been completed.
HARMONY is continuously in expansion and proposals for collaboration arrive from all over the world. As a consequence, HARMONY is a consolidated community with 49 associated members and 53 partners, being the biggest haematological community in Europe.
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.
For instance, in AML, HARMONY has studied the genomic data of thousands of patients, which confirms the co-occurrence of certain mutations, determining which ones occur first and which later in the disease process, as well as the differences between male and female patients. Moreover, the association of mutation patterns with clinical outcomes such as remission, relapse, and survival have been investigated. In addition, for unmutated CLL patients, different prognostic impact of recurrent gene mutations in IGHV-mutated and IGHV-have been studied. 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.
To improve the protocols for clinical trial design and processes, HARMONY have defined 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. COS for each HM have been gathered by launching several Delphi Surveys involving the input from different stakeholders. During 2021, Delphi surveys were successfully completed for AML, CLL, NHL, MDS and MM, while for ALL the first round has also been completed.
HARMONY is continuously in expansion and proposals for collaboration arrive from all over the world. As a consequence, HARMONY is a consolidated community with 49 associated members and 53 partners, being the biggest haematological community in Europe.
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.
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.