Periodic Reporting for period 6 - HARMONY (Healthcare Alliance for Resourceful Medicines Offensive against Neoplasms in HematologY - Sofia ref.: 116026)
Periodo di rendicontazione: 2022-01-01 al 2023-06-30
Over the last 25 years, academic and pharmaceutical research has made significant progress by increasing Hematological Malignancies (HMs) patients’ survival rates and improving quality of life (QoL). However, a substantial proportion of patients with HMs 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’ outcomes 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. This situation caused an unmet need to harmonize outcome measures and endpoint definitions for HMs and their collection at the European level.
After 6.5 years of joint work, the HARMONY Alliance, the largest IMI European PPP has been able to unify and align all European stakeholders. Through a wide variety of collaborative activities, HARMONY has truly demonstrated the power of Big Data to improve the treatment of HMs.
HARMONY has transformed both the sharing and the use of data by developing a Big Data Platform. HARMONY’s unique repository, with over 160,000 patient records identified, permits to represent the diversity of HM diseases in relation to their pathological, medical, epidemiological, healthcare, and societal scope. All data is protected using a unique anonymization and security process and the platform provides the technological and governance requirements to allow these datasets to be shared and queried by medical and non-medical experts involved in the healthcare provision and HMs' management. This platform has permitted to conduct more than 30 groundbreaking research projects, where stakeholders have teamed up to advance the understanding of HMs with the help of advanced customized data analytic services, including AI modelling. Finally, HARMONY has published HM-specific COS aiming to improve future clinical trial design and consistency, thereby strengthening the entire research, drug development, and patient management not only in the field of HMs, but in cancer in general.
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. This situation caused an unmet need to harmonize outcome measures and endpoint definitions for HMs and their collection at the European level.
After 6.5 years of joint work, the HARMONY Alliance, the largest IMI European PPP has been able to unify and align all European stakeholders. Through a wide variety of collaborative activities, HARMONY has truly demonstrated the power of Big Data to improve the treatment of HMs.
HARMONY has transformed both the sharing and the use of data by developing a Big Data Platform. HARMONY’s unique repository, with over 160,000 patient records identified, permits to represent the diversity of HM diseases in relation to their pathological, medical, epidemiological, healthcare, and societal scope. All data is protected using a unique anonymization and security process and the platform provides the technological and governance requirements to allow these datasets to be shared and queried by medical and non-medical experts involved in the healthcare provision and HMs' management. This platform has permitted to conduct more than 30 groundbreaking research projects, where stakeholders have teamed up to advance the understanding of HMs with the help of advanced customized data analytic services, including AI modelling. Finally, HARMONY has published HM-specific COS aiming to improve future clinical trial design and consistency, thereby strengthening the entire research, drug development, and patient management not only in the field of HMs, but in cancer in general.
After 6.5 years of joint work, HARMONY reaped the benefits of building a research infrastructure with the best brains of European blood cancer research. Through a wide variety of collaborative activities, HARMONY has truly demonstrated the power of Big Data to improve the treatment of HMs. By the end of June 2023, we have identified data from more than 160,00 patients with blood cancer and have transferred almost 100,000 datasets into the platform in ALL, AML, CLL, MDS, MM and NHL from both public and private providers across the EU.
In HARMONY research projects, scientists and physicians have teamed up to advance the understanding of HMs. For instance, in AML, our researchers have identified gene combinations that are associated with prognosis in two subtypes of this condition, have presented new insights into the prognosis of these patients and found factors that contribute to the clinical outcome of AML patients treated prior to the approval of novel drugs.
Regarding ALL, they identified a low-risk subgroup of ALL that could be considered for treatment reduction and found a genetic marker for poor treatment response in adults with a subtype of ALL.
In MM a new revision of the ISS has been published. In addition to biomedical research projects, they are running HTA projects and organizing multi-stakeholder activities, including the HARMONY Patient Cluster.
In MDS, a retrospective study with patients with lenalidomide discontinuation has been performed, and a sex-informed approach to improve the personalised decision-making process as well as a Real-World Validation of Molecular IPSS for MDS are under way.
On CLL, a study has shown how recurrent gene mutations originates a different impact in patient’s prognosis.
In addition, HARMONY has developed a COS for each of the HMs studied as well as an overarching COS for all HMs. This COS may improve the interpretation and comparability of clinical trials and form the basis for future improvements in patient management.
More than 100 organizations from 18 European countries already share our mission to personalize treatments for blood cancer patients. Spreading and exchanging knowledge have been boosted by the participation in a variety of international conferences and events (https://www.harmony-alliance.eu/en/meet-us) to increase global awareness of HARMONY’s achievements and its ongoing work, and that knowledge of HARMONY’s work reaches out an international audience.
As a result of the strong commitment and drive of the multi-stakeholder partners and members involved in HARMONY, and to ensure the continuity of the work, a non-profit research foundation is being established. This foundation will build upon the achievements of HARMONY and bring hematology research and patient care to the next level.
In HARMONY research projects, scientists and physicians have teamed up to advance the understanding of HMs. For instance, in AML, our researchers have identified gene combinations that are associated with prognosis in two subtypes of this condition, have presented new insights into the prognosis of these patients and found factors that contribute to the clinical outcome of AML patients treated prior to the approval of novel drugs.
Regarding ALL, they identified a low-risk subgroup of ALL that could be considered for treatment reduction and found a genetic marker for poor treatment response in adults with a subtype of ALL.
In MM a new revision of the ISS has been published. In addition to biomedical research projects, they are running HTA projects and organizing multi-stakeholder activities, including the HARMONY Patient Cluster.
In MDS, a retrospective study with patients with lenalidomide discontinuation has been performed, and a sex-informed approach to improve the personalised decision-making process as well as a Real-World Validation of Molecular IPSS for MDS are under way.
On CLL, a study has shown how recurrent gene mutations originates a different impact in patient’s prognosis.
In addition, HARMONY has developed a COS for each of the HMs studied as well as an overarching COS for all HMs. This COS may improve the interpretation and comparability of clinical trials and form the basis for future improvements in patient management.
More than 100 organizations from 18 European countries already share our mission to personalize treatments for blood cancer patients. Spreading and exchanging knowledge have been boosted by the participation in a variety of international conferences and events (https://www.harmony-alliance.eu/en/meet-us) to increase global awareness of HARMONY’s achievements and its ongoing work, and that knowledge of HARMONY’s work reaches out an international audience.
As a result of the strong commitment and drive of the multi-stakeholder partners and members involved in HARMONY, and to ensure the continuity of the work, a non-profit research foundation is being established. This foundation will build upon the achievements of HARMONY and bring hematology research and patient care to the next level.
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 QoL.
HARMONY will impact on distinct levels:
- The patient and patient advocacy organizations
Patients shall benefit from: i) linking diagnostic and molecular profiling data 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 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 patients’ QoL.
HARMONY will impact on distinct levels:
- The patient and patient advocacy organizations
Patients shall benefit from: i) linking diagnostic and molecular profiling data 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 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 patients’ QoL.