HTx will create a framework for next generation Health Technology Assessment (HTA) that supports patient-centred, societally oriented, and real-time decision-making for integrated healthcare throughout Europe. HTx will focus on therapeutic areas with high unmet need for which HTA information has to be provided on complex and personalised combinations of health technologies. Based on a select number of relevant case studies, HTx will enhance methods for integrating evidence from RCTs and real-world data (RWD). HTx will also augment statistical and econometric methods for generating robust estimates of effectiveness and cost-effectiveness in order to support relevant HTA decision-making for these complex and personalised combinations of health technologies. HTx will also contribute to improving methods to support personalised treatment advice fitted for sharing with patients and their physicians. This includes the development of statistical and econometric approaches and artificial intelligence/machine learning methods for forecasting treatment effects in specific groups of patients. Simultaneously, in close collaboration with the European Network for HTA (EUnetHTA), HTx will improve synergies between regulatory agencies, HTA bodies and clinical guideline developers. This will include the translation of HTx methods into already existing European guidelines, most prominently those developed by EUnetHTA. HTx will also support initial efforts to discuss reimbursement and funding models that facilitate controlled access to and the pricing of these complex health technologies. Finally, we will evaluate the transferability of HTx results into all EU Member Countries especially in Central and Eastern European (CEE) Countries and promote the dissemination of HTx results to the different European stakeholders with a special focus on the patient community.

The GetReal Initiative brings together partners from the IMI GetReal project to drive the adoption of tools, methodologies and best practices from IMI GetReal and increase the quality of real-world evidence (RWE) generation in medicines development and regulatory/HTA processes across Europe. We will establish, in Work Package (WP) 1, a Think Tank, a number of Task Forces and a RWE Research Community. The Think Tank will consist of international thought leaders and will discuss, assesses and give recommendations on the opportunities and barriers to the generation, use and acceptability of RWE. They will act as ambassadors for the use of RWE during the project and beyond, enagaging with key stakeholder groups to drive policy debate and facilitate the uptake of the outputs of IMI GetReal and the GetReal Initiative. The GetReal Taskforces will drive the focused development of tangible solutions to the key challenges identified in IMI GetReal and the Think Tank. The initial three task forces will be: (i) Pragmatic Trials (design, operational feasibility and analysis/the GetReal PragMagic tool), (ii) network meta-analysis and benefit risk assessment (incl. the GetReal ADDIS tool) and (iii) Statistical Approaches for enriching RCTs with real-world data. The GetReal Research Community will consist of researchers and organisations active in the field of RWE generation, regulators, HTAs, physicians and patients. The Community will review and comment on any GetReal Initiative guidelines, recommendations or white papers ahead of their finalisation, will have access to all the GetReal tools and outputs, receive regular newsletters and receive invitations to attend stakeholder events. The project will also invest in long term sustainability of the GetReal Initiative and the tools on a not-for-profit basis (WP2). The project is supported by professional experienced consortium management (WP3) and an ethics work package (WP4).

Progress in the life sciences and related technologies offer great potential for therapeutic benefits to patients in need. However, major adaptations to current paradigms of bringing medicines to patients are required in order to realize that potential and to address important challenges in the healthcare ecosystem. Against this background, several initiatives are exploring new pathways to market, collectively referred to as Medicines Adaptive Pathways to Patients (MAPPs). The ADAPT-SMART consortium is aligning a limited number of major stakeholders eager to progress towards MAPPs implementation. It will act as a neutral collaborative platform that will engage industry, SMEs, regulators, Health Technology Assessment bodies (HTAs), payers, governments, clinicians and patients. The ADAPT-SMART consortium will contribute to align understanding of the impact of MAPPs, to share learnings between all stakeholders, and to allow the field to actively work towards MAPPs implementation. The impact of the ADAPT-SMART CSA will be a result of the delivery of • actionable advice/recommendations to IMI on how to best leverage results from past/current projects; • concrete proposals for future (IMI) projects; • actionable advice/recommendations and information to other actors in the healthcare environment; • synthesis of learnings from pilot projects and case studies with relevance to MAPPs; • communication of CSA outcomes by way of publications and conference presentations. This CSA will increase the probability of successful implementation of MAPPs and accelerate access to crucial therapies, thus improving the position of both the patients in need of novel treatments and the research-based pharmaceutical industry.