Periodic Reporting for period 2 - HARMONY PLUS (HEALTHCARE ALLIANCE FOR RESOURCEFUL MEDICINES OFFENSIVE AGAINST NEOPLASMS IN HEMATOLOGY – PLUS)
Période du rapport: 2021-10-01 au 2022-09-30
Haematological malignancies (HM), also known as blood cancers, are a heterogeneous and complex group of multicausal diseases that can’t be easily diagnosed nor treated. Nowadays most treatments are extremely complex, and advances in patient diagnosis and therapies slow due to the low number of patients per centre. The diverse healthcare practice across EU, the rarity of HMs and the spread of patients over different hospitals and different countries makes it extremely challenging to reach a critical mass of data. Thus, there is a need to harmonise, store, and analyse the current HM information to speed-up and support the decision-making process for patients’ access to new therapies. Moreover, in order to make it easier for the patients to receive the right treatment at an appropriate time it is necessary to overcome the different barriers in the drug registration protocol, such as establishing outcome criteria. As a consequence, promising new treatments are delayed or are considered to be too costly to develop. This situation calls for more efficient treatment development and regulatory evaluation. By making use of Big Data technologies, a more efficient drug development can be accelerated as well as regulatory evaluation, access appraisal, and treatment strategies.
HARMONY PLUS takes advantage of the capabilities of the HARMONY Big Data platform to match these unmet needs by expanding its scope to incorporate myeloproliferative neoplasms (MPN), including chronic myeloid leukemia (CML), polycythaemia vera (PV), essential thrombocythaemia (ET), and myelofibrosis (MF); and lymphoproliferative disorders, including Hodgkin’s lymphoma, Waldenström macroglobulinemia and all the other rare HMs not covered by HARMONY. In parallel, HARMONY PLUS will continue to refine and define the Core Outcome Sets (COS), especially for these new HMs to ensure the use by researchers of useful common outcomes relevant to all stakeholders. As previously accomplished in HARMONY, HARMONY PLUS is committed to pursue the maximum ethical and legal requirements, particularly to ensure patient’s right to privacy.
Data-driven research within Europe will be enhanced by converting the current HARMONY platform into an Integrated Services Platform to serve as a valuable tool to support clinical trial design and use of available data as a control arm. This platform, combined with a HaemoDatabank repository with information about HMs patient biological samples across Europe, will facilitate a more efficient research and clinical trial design, and consequently will promote collaborations with recognised databases outside Europe. From the regulatory point of view, HARMONY PLUS will be a valuable technology tool during the evaluation of new treatments and drugs by also considering the patients’ needs.
HARMONY PLUS takes advantage of the capabilities of the HARMONY Big Data platform to match these unmet needs by expanding its scope to incorporate myeloproliferative neoplasms (MPN), including chronic myeloid leukemia (CML), polycythaemia vera (PV), essential thrombocythaemia (ET), and myelofibrosis (MF); and lymphoproliferative disorders, including Hodgkin’s lymphoma, Waldenström macroglobulinemia and all the other rare HMs not covered by HARMONY. In parallel, HARMONY PLUS will continue to refine and define the Core Outcome Sets (COS), especially for these new HMs to ensure the use by researchers of useful common outcomes relevant to all stakeholders. As previously accomplished in HARMONY, HARMONY PLUS is committed to pursue the maximum ethical and legal requirements, particularly to ensure patient’s right to privacy.
Data-driven research within Europe will be enhanced by converting the current HARMONY platform into an Integrated Services Platform to serve as a valuable tool to support clinical trial design and use of available data as a control arm. This platform, combined with a HaemoDatabank repository with information about HMs patient biological samples across Europe, will facilitate a more efficient research and clinical trial design, and consequently will promote collaborations with recognised databases outside Europe. From the regulatory point of view, HARMONY PLUS will be a valuable technology tool during the evaluation of new treatments and drugs by also considering the patients’ needs.
Over the past 6 years, the HARMONY Alliance, the largest IMI European public-private partnership, has become one of Europe´s most unique and mature Big Data Platform for haematological malignancies.
HARMONY PLUS has been applying the lessons learnt in HARMONY and using the scientific knowledge and the technological platform already developed in order to fulfil the new challenges presented.
With the support of almost 50 partnering organizations, almost 20,000 data sets from public and private partners have been identified and are currently under negotiation. These data sets cover different kinds of myeloproliferative neoplasms (MPN), including chronic myeloid leukemia (CML), polycythaemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF), which will be processed in the HARMONY Platform by using Big Data Analytics to generate evidence that can be used to answer critical research questions in haematology.
During the second year of HARMONY PLUS, a new research question has been approved on the “Use of big data to identify optimal treatment pathways leading to successful treatment-free remission in CML”, submitted by the by Karolinska Institute, in collaboration with institutions in the HARMONY network, and others that have joined in HARMONY PLUS. Additionally, the patient cluster has been very active identifying studies containing QoL in CML and has submitted three research questions to the Platform on this particular topic.
Also, during this 2nd year, NICE in close collaboration with Pfizer and BMS has led Task 4.4 to deliver a regulatory/HTA scientific advice procedure on the appropriateness of evidence from the HARMONY platform to be used in regulatory or reimbursement submissions.
Communication and dissemination activities have been aimed to increase global awareness of HARMONY PLUS´ achievements and its ongoing work. The goal is to ensure that knowledge of the project work reaches an international audience, through effective outreach and participation in various global events.
Website: www.HARMONY-alliance.eu
Twitter: https://twitter.com/HARMONYnetEU
LinkedIn: https://www.linkedin.com/company/17987398/
YouTube: https://www.youtube.com/channel/UCTOHQVjSs0Ee1g4cD8FAbwg
Facebook: https://www.facebook.com/HarmonyNetEU/
HARMONY PLUS has been applying the lessons learnt in HARMONY and using the scientific knowledge and the technological platform already developed in order to fulfil the new challenges presented.
With the support of almost 50 partnering organizations, almost 20,000 data sets from public and private partners have been identified and are currently under negotiation. These data sets cover different kinds of myeloproliferative neoplasms (MPN), including chronic myeloid leukemia (CML), polycythaemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF), which will be processed in the HARMONY Platform by using Big Data Analytics to generate evidence that can be used to answer critical research questions in haematology.
During the second year of HARMONY PLUS, a new research question has been approved on the “Use of big data to identify optimal treatment pathways leading to successful treatment-free remission in CML”, submitted by the by Karolinska Institute, in collaboration with institutions in the HARMONY network, and others that have joined in HARMONY PLUS. Additionally, the patient cluster has been very active identifying studies containing QoL in CML and has submitted three research questions to the Platform on this particular topic.
Also, during this 2nd year, NICE in close collaboration with Pfizer and BMS has led Task 4.4 to deliver a regulatory/HTA scientific advice procedure on the appropriateness of evidence from the HARMONY platform to be used in regulatory or reimbursement submissions.
Communication and dissemination activities have been aimed to increase global awareness of HARMONY PLUS´ achievements and its ongoing work. The goal is to ensure that knowledge of the project work reaches an international audience, through effective outreach and participation in various global events.
Website: www.HARMONY-alliance.eu
Twitter: https://twitter.com/HARMONYnetEU
LinkedIn: https://www.linkedin.com/company/17987398/
YouTube: https://www.youtube.com/channel/UCTOHQVjSs0Ee1g4cD8FAbwg
Facebook: https://www.facebook.com/HarmonyNetEU/
HARMONY PLUS will have relevant and realistic scientific and technological impacts on several distinct levels:
- The patients
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
Due to the high genetic and clinical heterogeneity of the haematological malignancies under study, the use of HARMONY’S transnational repository, complemented by an Integrated Services Platform, is the only path to collecting the required amount of patient records and better coordinate all levels of healthcare from the organizational perspective by eliminating the bottlenecks in the information flow.
- Care providers
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.
- Pharmaceutical and healthcare industry
Pharmaceutical and healthcare industry shall benefit from: i) a speed up in patient recruitment; ii) models generating a benefit through a personalized HM approach; iii) a better understanding of patient stratification, and validation of novel endpoints that will increase efficiency in R&D.
- Contribution to the EU’s industrial leadership including small and medium-sized enterprises (SMEs)
HARMONY PLUS will provide a solution that will support the innovation in healthcare, but also the transfer of innovation in several domains, fostering SMEs’ evolvement and creation of novel cost-efficient business models as well as universalize the decision-making capability provided by advanced analysis and data manipulation for large data sets especially for research purposes.
- 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.
- The patients
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
Due to the high genetic and clinical heterogeneity of the haematological malignancies under study, the use of HARMONY’S transnational repository, complemented by an Integrated Services Platform, is the only path to collecting the required amount of patient records and better coordinate all levels of healthcare from the organizational perspective by eliminating the bottlenecks in the information flow.
- Care providers
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.
- Pharmaceutical and healthcare industry
Pharmaceutical and healthcare industry shall benefit from: i) a speed up in patient recruitment; ii) models generating a benefit through a personalized HM approach; iii) a better understanding of patient stratification, and validation of novel endpoints that will increase efficiency in R&D.
- Contribution to the EU’s industrial leadership including small and medium-sized enterprises (SMEs)
HARMONY PLUS will provide a solution that will support the innovation in healthcare, but also the transfer of innovation in several domains, fostering SMEs’ evolvement and creation of novel cost-efficient business models as well as universalize the decision-making capability provided by advanced analysis and data manipulation for large data sets especially for research purposes.
- 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.