The PREFER project will deliver an overview and evaluation of preference elicitation methods to be applied in the entire drug life cycle, i.e. in the early stages of identifying medical needs, in clinical testing, to guide decisions on reimbursement and to make decisions on withdrawal of drugs from the market. A broad array of (combinations of) patient preference methods will be tested prospectively in a large number of case studies. The availability of large patient cohorts will enable to test new methods or deviations from existing methods in a randomized manner, by comparing well-known methods with newer ones. The use of simulation studies will both contribute to smarter design of case studies and to exploring the sensitivity of outcomes of preference studies. Based on discussions with a broad representation of stakeholders e.g. patients, patient organisations, regulatory authorities, HTA bodies and reimbursement agencies, suitable methods will be tested and their contributions to improved decision making will be discussed in recommendations adapted to the needs of all relevant stakeholders. The recommendations from PREFER are expected to lead to changed practices, in that industry will routinely assess whether a preference study would add value at key decision points in the medicinal product life cycle and, if so, implement patient-preference elicitation studies according to the PREFER project recommendations. The PREFER consortium consist of 16 industry partners and 16 academic and SME members including representation from academia, patient organizations, HTA bodies, reimbursement agencies, and project management.

ERA4Health Partnership Phase 2 is aimed to integrate new additional activities to the current ERA4Health Partnership. The main ambition of the partnership it is the contribution to the goal of Europe to be at the forefront of science and innovation in Health Research by 2050 and enhancing the biomedical research to provide a better health for citizens, not only in Europe but at international level. To be able to achieve this aim, ERA4Health Phase 2 will pursue the following specific objectives: SO1. Support relevant medical research including clinical fields and intervention areas (prevention, diagnosis, treatment) SO2. Improve the utilisation of existing health technologies in clinical practice SO3. Build capacity, in particular in conducting IICSs at European scale SO4. Implement and advance the practice of RRI across the breadth of the programme This Phase 2 of the Partnership will continue supporting relevant medical research and new knowledge generation in the research priority areas stablished in the ERA4Health long-term Strategic Research and Innovation Agenda (SRIA), with a particular focus devoted to support and fund multi-country Investigator-Initiated Clinical Studies (IICS). The research priority areas included in the SRIA are: nutrition and life-style related diseases, prevention and public health strategies, cardiovascular diseases, nano and advanced technologies for disease prevention, diagnostic and therapy. ERA4Health will fund non-commercial multi-country IICS to enhance this impactful and added value clinical research. The additional activities in ERA4Health Phase 2 will serve to provide support and building capacity in conducting multi-country IICSs and to overcome the identified challenges and obstacles in these type of IICSs.

Scientific excellence, and acceptance by health authorities of results derived from research on personalised medicine require standards ensuring validity and reproducibility. The objective of PERMIT is to establish, with all relevant stakeholders and invited experts, recommendations ensuring the robustness of personalised medicine trials, which also requires validation of the stratification methods. In a series of workshops participants, partners and selected experts will address the various aspects of methodology, design, data management, analysis and interpretation in personalised medicine research programmes, with the objective to reach consensus and publish recommendations. A mapping of the literature will inventory methodological practice in the various steps of personalised medicine programmes (WP2) and identify needs in terms of standardised methodology. WP3 will address the design of the stratification and validation cohorts, including the issue of the statistical power and the quality of data. WP4 will focus on the use of the stratification algorithms and the robustness the stratification methods. WP5 will address the translational step needed to select treatments to be tested in each of the clusters. WP6 will be dedicated to randomized clinical trials testing treatments in each cluster (including umbrella / basket designs), or comparing the personalised vs. non-personalised approach. Recommendations and publications in scientific journals, a final meeting and webinars will foster adoption and implementation (WP7). The PERMIT consortium is composed of participants: pan-European research infrastructures (ECRIN, EATRIS, ELIXIR-LU/UNILU), funders (DLR), HTAs (KCE, ISCIII), patients (EPF), regulatory (ISS), data protection (TMF) and scientific experts, whereas partners represent stakeholders interested in the quality of evidence generated by personalised medicine research (industry, medicine agencies, ethics committees, funders, journal editors, HTAs, BBMRI).