Real-world evidence derived from real-world data (RWD) has a promising role to inform regulatory decision-making. Based on highly relevant use cases from regulatory practice and across the product lifecycle Real4Reg develops AI-based data-driven methods and tools for the assessment of medicinal products. Findings will inform training activities on good practice examples and will be implemented in existing and emerging guidelines for both health regulatory authorities and health technology assessment (HTA) bodies across Europe. There is urgent need to enable the use and establish the value of the application of RWD across the spectrum of regulatory use cases. The use of RWD is established in regulatory processes such as safety monitoring, but evidentiary value for further use cases, especially in the pre-authorisation and evaluation phase of medicinal products, is rudimentary. The use of RWD in post-authorisation steps is constrained by data variability and by challenges in analysing data from different settings and sources. Thus, the development of new and optimised methods for RWD analyses is essential. Real4Reg addresses the challenges and opportunities of RWD analyses across different health care systems by involving multiple stakeholders to work together in a collaborative approach, also outreaching to already established European initiatives. Our consortium assembles partners with outstanding excellence in the field of RWD analyses, including experts from regulatory agencies/ HTA (BfArM, DKMA, Infarmed), academia (Fraunhofer, UEF, CSC, AU, DZNE) and patient organisations (EUpALS, EIWH). In an advisory board stakeholders provide input and guidance to the project, including patients, industry, payers, HTA bodies and healthcare professionals. The structure and approach of our project facilitates the successful implementation of the effective use of RWD in regulatory decision-making and HTA, and ultimately supports the application of better medicines for patients.

Pharmacogenomics is the study of genetic variability affecting an individual’s response to a drug. Its use allows personalized medicine and reduction in ‘trial and error’ prescribing leading to more efficacious, safer and cost-effective drug therapy. The U-PGx consortium will investigate a pre-emptive genotyping approach (that is: multiple pharmacogenomic variants are collected prospectively and embedded into the patients’ electronic record) of a panel of important pharmacogenomic variants as a new model of personalised medicine. To meet this goal we combine existing pharmacogenomics guidelines and novel health IT solutions. Implementation will be conducted at a large scale in seven existing European health care environments and accounts for the diversity in health system organisations and settings. Feasibility, health outcome and cost-effectiveness will be investigated. We will formulate European strategies for improving clinical implementation of pharmacogenomics based on the findings of this project.

Lack of specific relevant know-how in regulatory science delays the development of new treatment strategies or limits the chances that promising innovations will reach patients. STARS aims to improve the direct regulatory impact of results obtained in medical research. Seventeen European countries are represented in the consortium through their national competent authorities, alongside academic and industry representatives, and associations with relevant experience. The work plan includes the development of a Comprehensive Inventory of existing support activities based on a detailed analysis of the currently established programmes. This analysis is also the basis for development of a Common Strategy to strengthen regulatory sciences and two curricula, the Core Curriculum specifying essential knowledge for the professional training of clinical scientists and the Comprehensive Curriculum defining relevant knowledge for specific post-graduate programmes. Three pilot projects aim (i) to transfer an identified best practice example for training programmes to other EEA countries, (ii) to implement a new support activity addressing a gap in regulatory knowledge of significant relevance and (iii) to implement the Comprehensive Curriculum. STARS will deliver consensual recommendations ensuring sustainable support of academic research and will propose additional support mechanisms based on a comprehensive analysis of needs. STARS has the objective and the potential to complement, coordinate and harmonise regulatory efforts among Member States and at European level to support academic health research for the benefit of patients. The aim is to reach academic researchers very early in the planning of relevant grant applications. A further aim is to strengthen regulatory knowledge in general by reaching clinical scientists during professional training and qualification.