Hematologic malignancies (HMs) are a major source of mortality and morbidity, with approximately 44,000 new cases in Europe per year. In addition, they constitute a major economic burden. New therapies in HMs are being developed at a rapid pace, and their clinical value needs to be compared to existing alternatives in a transparent, reproducible, and systematic way.
Policymakers, payers, and clinicians use value frameworks to make informed decisions about treatment options and to optimize resource allocation in healthcare settings. However, the existing clinical value frameworks are not specifically designed for HMs. In a recent paper in Value in Health, the HARMONY Alliance HTA Taskforce examined the suitability of six existing value frameworks for assessing the clinical and societal impact of new interventions for HMs.
Francesco Cerisoli, European Hematology Association, HARMONY Alliance Partner: “All frameworks that we examined, put strong attention on classical clinical outcomes such as overall survival. They hardly consider real-world evidence as a data source. In addition, patient-reported outcomes and quality of life seem to be of minor significance in decision-making. This makes it challenging to apply these value frameworks to therapies for HMs, especially in the case of innovative novel treatment approaches.”
Title of the paper the HARMONY Alliance Taskforce in the Elsevier ScienceDirect Journal is: "Value in Health: Building a Healthcare Alliance for Resourceful Medicine Offensive Against Neoplasms in HematologY Added Value Framework for Hematologic Malignancies: A Comparative Analysis of Existing Tools".
No comprehensive clinical value framework for HMs has been developed and validated for HMs so far. The existing clinical Value Frameworks mainly focus on clinical outcomes such as overall survival or cost considerations. Furthermore, most of these frameworks have been developed with little to no patient involvement, and they do not allow for the use of registry data or real-world evidence. Leveraging its vast European network and its Big Data Platform with real-world evidence, the HARMONY Alliance is uniquely positioned to address these limitations.
The taskforce concluded that the available frameworks do not meet the HARMONY Alliance ambitions for a tool that can assess the added clinical value of innovative health technologies in a transparent, reproducible, and systematic manner. As a next step, they recommend working on a cross-stakeholder agreement on the definition of ‘value’. A novel value framework should be developed based on the outcomes identified by the HARMONY Alliance taskforce. Finally, the newly developed tool should be validated through field testing, leveraging the vast HARMONY network.
Tamas Bereckzy, Leukanet, HARMONY Alliance Partner, representing the perspective of patients/the HARMONY Alliance Patient Cluster: “A patient’s definition of value does not necessarily match a clinician’s or payer’s definition of value. Therefore, the next step in the development of a suitable value framework is to find a cross-stakeholder agreement on the definition of value. Being a multistakeholder project with the involvement of patient organizations, medical associations, pharmaceutical companies, and health technology assessment/regulatory agencies, the HARMONY Alliance is well-positioned to develop a value framework that considers what “value” means to multiple stakeholders.”
Dalia Dawoud, NICE ((National Institute for Health and Care Excellence), HARMONY Alliance Partner: “The findings of this work support the ongoing efforts of HTA organizations to improve their approaches to using RWE in their value assessment. HARMONY Alliance can have a positive impact by informing these efforts, through its multi-stakeholder input, generally and more specifically for hematological malignancies.”
Lars Bullinger, Charité – Universitätsmedizin Berlin, HARMONY Alliance Partner about the implications of this paper for clinicians and researchers: “It was striking that none of the available frameworks meets all stakeholder needs, and therefore also not the goals of HARMONY. Thus, to come up with a tool that allows us to assess the therapeutic value of innovative health technologies and treatment approaches in HMs not only in a transparent, reproducible, and systematic way, but also in a way that reflects all stakeholders’ needs, all partners will have to work closer together in the future and take real-world evidence into account.”
Farzad Ali, Pfizer, HARMONY Alliance Partner about the implications of this paper for the pharma industry: “This assessment highlights that no existing value framework is perfectly holistic. We need to work with patients first and foremost to understand the value through their lens, and ensure that we bring medicine to meet their needs while also considering real-world evidence as well as the payer and clinician community requirements.”
The term ‘real-world evidence’ refers to clinical evidence derived from data collected in a nonrandomized controlled trial setting, such as patient registries, electronic health records, or mobile health. The European Medicines Agency (EMA) and other stakeholders acknowledge real-world evidence as the way forward for healthcare decision-making, particularly in situations where randomized clinical trials are not feasible such as rare conditions including HMs.
Jesus Maria Hernandez Rivas, IBSAL, HARMONY Alliance Coordinator: “The HARMONY Alliance is uniquely equipped to develop an added value framework for HMs because it is a multistakeholder project involving representatives from patient organizations, medical associations, pharmaceutical companies, bioethics, hospitals and health technology assessment/regulatory agencies. The comprehensive focus of our Alliance is covering all the angles of these serious diseases and providing new tools for the improvement of the quantity and quality of life of our patients”.
#BigDataforbloodcancer | Big Data accelerate better and faster treatment for Patients with Hematologic Malignancies
The HARMONY Alliance is a European Public–Private Partnership for Big Data in Hematology that is capturing and mining Big Data on various Hematologic Malignancies. The HARMONY Alliance, funded by Innovative Medicines Initiative, unites more than 100 organizations such as European medical associations, hospitals, research institutes, patient organizations, pharmaceutical companies, IT companies, and health technology assessment/regulatory agencies. The inclusion of all these stakeholders reflects the HARMONY ambition to develop tools that not only can be adopted by clinicians and patients but also be of interest for regulators, payers, and health technology assessment bodies.
HARMONY uses Big Data analytics to accelerate the development of more effective treatments for blood cancer patients. Data are stored in the HARMONY Big Data Platform, which has already identified over 100,000 anonymized patient records, making it one of the largest databases of its kind. Leading research teams are currently using this wealth of information to answer critical questions about hematologic malignancies that cannot be addressed with other methods. In addition, HARMONY is running projects to develop core outcome sets, as well as Health Technology Assessment projects and multi-stakeholder activities. The HARMONY Alliance is currently running two projects: HARMONY (November 2016-June 2023) and HARMONY PLUS (October 2020-October 2023). Both have work packages dedicated to HTA and multi-stakeholder engagement.
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