Periodic Reporting for period 1 - More-EUROPA (More Effectively Using Registries to suppOrt PAtient-centered Regulatory and HTA decision-making)
Reporting period: 2023-01-01 to 2024-06-30
Medicines regulation and health technology assessment (HTA) decision-making is a dynamic process that is informed by different data at various stages of the drug lifecycle. While randomised controlled trials (RCTs) remain the mainstay in drug development, approval, and reimbursement processes, the value of real-world data (RWD) to contribute to the characterisation of drug effects and their public health and economic impact is increasingly acknowledged and promoted [Arlett et al., 2021]. Evidence based on RWD – real-world evidence (RWE) – can transform, accelerate and de-risk decision making across all phases of (clinical) drug development and the drug lifecycle. Use of RWD can improve efficiency in design and conduct of clinical trial programs, and increase public health value in general.
Objectives
To establish the value of registry-based RWD in augmenting RCT data and to enable the more effective and ethical use of registry data to support patient-centred regulatory and HTA decision-making (MoreEUROPA), More-EUROPA has the following specific objectives:
1 Expand knowledge on drug efficacy and safety using RWD augmenting RCT data (establish value) using specific case studies in WP2 that are supported by novel analytical tools developed in WP1, and by creating a protocol for a registry-based RCT in WP5;
2 Develop a methodological framework (establish value) including analytical tools to integrate evidence derived from RCTs and (multiple) RWD sources (WP1);
3 Develop and standardize methods to increase usability of RWD across different registries (enable use) (WP1, WP2);
4 Develop a screening tool to timely identify suitable registries and RWD (enable use; WP3);
5 Develop an ethical framework describing practice-oriented ethical requirements (enable use) for generating and using patient-relevant RWD to support decision-making (WP4);
6 Create an integrated More-EUROPA framework, incorporating methodological tools and ethical considerations, to favour adoption and use of RWE across the drug lifecycle in regulatory & HTA guidelines and decision-making (WP5);
7 Increase the skills and competencies of HTA staff and regulators towards the use of RWD (favour adoption and use) through interactive multi-stakeholder training sessions (WP6);
8 Create an integrated multi-stakeholder platform for RWD/RWE engaging and aligning with other European programs and initiatives (favour adoption and use) (WP6).
Objectives
To establish the value of registry-based RWD in augmenting RCT data and to enable the more effective and ethical use of registry data to support patient-centred regulatory and HTA decision-making (MoreEUROPA), More-EUROPA has the following specific objectives:
1 Expand knowledge on drug efficacy and safety using RWD augmenting RCT data (establish value) using specific case studies in WP2 that are supported by novel analytical tools developed in WP1, and by creating a protocol for a registry-based RCT in WP5;
2 Develop a methodological framework (establish value) including analytical tools to integrate evidence derived from RCTs and (multiple) RWD sources (WP1);
3 Develop and standardize methods to increase usability of RWD across different registries (enable use) (WP1, WP2);
4 Develop a screening tool to timely identify suitable registries and RWD (enable use; WP3);
5 Develop an ethical framework describing practice-oriented ethical requirements (enable use) for generating and using patient-relevant RWD to support decision-making (WP4);
6 Create an integrated More-EUROPA framework, incorporating methodological tools and ethical considerations, to favour adoption and use of RWE across the drug lifecycle in regulatory & HTA guidelines and decision-making (WP5);
7 Increase the skills and competencies of HTA staff and regulators towards the use of RWD (favour adoption and use) through interactive multi-stakeholder training sessions (WP6);
8 Create an integrated multi-stakeholder platform for RWD/RWE engaging and aligning with other European programs and initiatives (favour adoption and use) (WP6).
At this stage of the project, we have achieved already some of the main outcomes of the project as outlined in the Grant Agreement. In particular, we have organised webinars and engaged in other training activities aimed at training regulators, HTA, patient representatives, industry and other stakeholders on proper use of registry data to support decision-making around medicinal products (WP5). We have developed an initial version of an AI tool to identify relevant registry-based RWD suitable to answer regulatory and HTA questions (WP3). Further, building blocks for the More-EUROPA comprehensive framework for generating registry-based RWE are being created, e.g. through surveys of perceptions of RWD/Registry use (WP1&5), a draft checklist about ethical aspects (WP4), and preparatory work to allow analyses in multiple registry datasets (WP1&2) and the development of basic training material for patient organisations. A detailed description per WP is provided below.
More-EUROPA will develop a comprehensive methodological framework, which includes analytical and screening tools, and that builds on an ethical and patient perspective on what data-to-collect and how data may be shared. This framework will enable regulators and HTA staff to utilise registry-based RWD with more confidence to answer drug-related efficacy, (cost-)effectiveness and safety questions. Stakeholders from all European countries will be targeted and able to attend education and training activities of More-EUROPA. The case studies with input from the patient organisations the European Organisation for Rare Diseases (EURORDIS) and the European Multiple Sclerosis Platform (EMSP) will inform specific regulatory and HTA guidance (on registry-based studies including registry-based RCTs and machine learning/artificial intelligence methods) as well as contribute to and appraise work performed within the European Health Data Space. Interactive two-way training and communication activities will increase RWD/RWE analytical skills of European regulators and HTA staff and their feedback used to improve the comprehensive methodological framework. More-EUROPA researchers have an extensive track record in the use of registry data and are intrinsically connected with the European regulatory (including EMA) and HTA network. In addition, the best practices will be shared widely through different channels across Europe.