ASCERTAIN (Affordability and Sustainability improvements through new pricing, Cost- Effectiveness and ReimbursemenT models to Appraise iNnovative health technologies) addresses the need of patients, physicians, payers, regulators, and manufacturers to improve the affordability and accessibility to innovative health technologies (including pharmaceuticals) in Europe. ASCERTAIN aims to enhance current methods of (value-based) pricing, cost-effectiveness modeling, threshold-setting, reimbursement, and payment, to set affordable prices, and to facilitate a cyclic assessment of broader societal benefits including costs and risks. It seeks to find a sustainable balance between access to affordable technologies, the need to stimulate innovation and entrepreneurship, and the need to consider the environmental impact of innovations. Guided by a conceptual framework integrating pricing, health technology assessment and reimbursement/payment, ASCERTAIN will develop open-access, easy to use, policy-supporting tools including (i) pricing models, (ii) value assessment models, and (iii) reimbursement models, which will be adaptable to country-specific conditions. These tools will improve transparency and accountability of decision-making, reduce uncertainty for all stakeholders, reward innovation in areas of high unmet need, accelerate access for patients, and support long-term planning in a sustainable way. Further, these tools will be developed, tested, and validated for three use cases, including precision cancer medicine, cell- and gene therapy, and medical devices (class IIb and III) or in vitro diagnostic (IVD) class D. The multinational, multidisciplinary ASCERTAIN consortium is complemented by an international advisory board consisting of key stakeholders, with whom the policy-supporting tools will be co-created. All tools will be hosted on a cloud-based, publicly accessible, user-friendly platform following open science principles.

Immune cells that are empowered by gene-engineering to seek and destroy cancer cells (engineered T cell therapy) constitute a transformative novel treatment that has the potential to cure cancer. Multiple new versions of this therapy are being developed for distinct types of cancer but their introduction into clinical practice is hampered by a lack of standardized and validated models to predict safety and efficacy, customized manufacturing and monitoring to scale up production and clinical use to industry standard, and strategies for optimal patient conditioning. The T2EVOLVE consortium unites scientists and physicians, regulators and policy makers, SMEs, and patient stakeholders to tackle these challenges in an orchestrated multi-disciplinary multi-stakeholder approach. A core feature of this approach will be the embedding of patient stakeholders as contributing members of the team across all levels of the R&D process. The overall aim is the development of an innovation ecosystem that will accelerate the process of developing engineered T cell therapy in the EU. The project will deliver novel tools for education and for improving the communication between healthcare providers and patients, optimized laboratory models that can help determine how safe and effective new therapies with engineered T cells are, standardized methods in which these therapies are produced and monitored during treatment. The consortium members are innovators and pioneers in this field that are dedicated to bringing the EU to the forefront of the global engineered T cell therapy movement. This effort will ensure that EU citizens will continue to have access to the most innovative and best-available medical care, provide guidance on how to implement this novel treatment into the EU health care system in a sustainable way, and secure a leading role for Europe in this emerging field in medicine and science, the economy and society.

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. 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. 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, including chronic myeloid leukemia, polycythaemia vera, essential thrombocythaemia, and myelofibrosis; and lymphoproliferative disorders, including Hodgkin’s lymphoma, Waldenström macroglobulinemia and all the other rare HMs not covered by HARMONY Project. 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.

Despite significant recent progress in the field of hematological malignancies (HMs), with increasing survival rates and improvement in quality of life, many children and adults with HMs still die from these disorders or experience disabling complications. Therefore, improvement of health care of HMs is an unmet medical need. Thus, it is important to define and align standard and efficient sets of HMs outcomes to measure and evaluate HM data for clinical decisions, long term risk/benefit profile, reimbursement, value analysis, and clinical trials design. Improving outcome measures and endpoint definitions by taking into account “real-life” data and differences in cross-national healthcare practice will undoubtedly result in an optimized, sustainable and effective treatment delivery, as well as in desirable and innovative accelerated pathways for novel drug availability. All these challenges will be addressed within a pan-EU perspective by HARMONY (Healthcare Alliance for Resourceful Medicines Offensive against Neoplasms in HematologY), a comprehensive public-private European consortium of excellence. HARMONY consortium is made up of 51 partners: 44 participants from 10 European countries and 7 pharmaceutical companies from the EFPIA. HARMONY aims to assemble, assess, connect, and analyze heterogeneous HM patient derived Big Data sets to define sets of outcome indicators that can be used for decision-making by key healthcare stakeholders. The consortium will orchestrate leading experts and working cooperative groups in HMs, European study alliances, pharmaceutical market leaders, patient advocacy groups, HTA and regulatory agencies, to: (i) optimize Europe-wide data collection and create a high-quality HM data repository for further explorative studies; (ii) establish a clinical data-sharing platform that empowers clinicians, patients and policy stakeholders to improve decision-making procedures and identify appropriate treatments to patients with HMs