More-EUROPA

The aim of our project is to develop, implement and establish evidentiary standards and methods to address the data and evidentiary needs of regulatory authorities and health technology assessment (HTA) bodies towards a more efficient use of RWD for the development, registration and assessment of medicinal products in Europe.

 

More-EUROPA has the following specific objectives. This will ultimately decrease the costs associated to drug development/licensing and speed up the accessibility and reimbursement of drugs to European people/patients in need.

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).

 

WP1. Development of novel analytics tools

We will:
 

• assess the expected level of evidence needed by stakeholders across the life cycle of new medicinal products;
• provide a methodological framework and tools to assess/quantify level of evidence and uncertainty to address pre-licensing, licensing decisions and reimbursement questions using registry data to augment clinical trial data; 
• provide a methodological framework and tools for regulatory bodies and HTA agencies to use clinical trial data augmented with registry data to estimate clinical benefit and safety in target populations;
• provide new methodology and tools to enable optimal federated analyses for regulatory questions across multiple registries that can be executed in one pass across the different databases.

WP2. Establishing RWD accessibility and usefulness

We will: 

• provide a framework for regulatory bodies on how to use RWD to assess effectiveness and safety in subgroups of patients who are poorly represented in RCTs;
• extend registry-based RCT evidence supporting the use of rituximab by using RWD from different European multiple sclerosis registries for effectiveness and cost-benefit;
• provide a framework for regulatory bodies on how to use the minimal RWD dataset in their procedures.

WP3.Identifying and screening suitable registries

We will test and set standards for an efficient selection of ‘fit-for-purpose’ registries and data elements, based on leveraging a combination of machine learning tools and expert analysis.
 

WP4. Ethical and patient perspectives and solutions for RWD sharing and use

We will: 

• generate a qualitatively generalizable account of patient and stakeholder perspectives on SWOTs of RWDuse in regulatory/HTA decision making;
• explain to patients how data from other sources than clinical trials can be used to generate additional knowledge on medicines and medical devices: a training module on the analysis of data obtained from other sources than clinical trials, their limits, their potential contribution to scientific and medical knowledge;
• assess the representativeness of the patient population in registries;
• explore patient-relevant information and the current practices regarding PROs and patient-reported outcome measures (PROMs) of the case studies in More-EUROPA;
• share More-EUROPA's developments and findings with medical coordinators of ERNs and European Patient Advisory Groups, in the context of patient and disease registries created by ERNs;
• develop a framework for the “Ethics of RWD decision-making”.

WP5. Dissemination: Guideline development, training and implementation

We will:
 

• develop guidance and a protocol for registry-based RCTs;
• assess changes in appreciation of registry-based RWD among regulators and HTAs;
• train regulators and HTA-staff;
• systematically review the quality of registries in recent EU marketing authorisation and HTA assessment dossiers using EUnetHTA’s REQueST and EMA’s Guideline on registry-based studies;
• amend regulatory and HTA/payer disease-specific guidance with a RWD/registry-based study paragraph;
• submit a request for an EMA, Qualification Procedure – with HTA involvement – for the innovative methods, and the comprehensive framework;
• create a strong link across the project towards adoption, implementation, and use, including a multistakeholder platform.

More EUROPA partners are: Charite Universitaetsmedizin Berlin, Critical Path Institute, Dutch Institute for Clinical Auditing (DICA), European Multiple Sclerosis Platform (EMSP), European Organisation for Rare Disease Association (Eurordis), Faculdade de Farmácia da Universidade de Lisboa, IQVIA, Karolinske Institutet, Quinten, Quinten Health, Radboud University Medical Center, Universita ‘Cattolica Del Sacro Cuore, University Medical Center Groningen, Universite de Paris, Uppsala Universitet. 

E-mail: [email protected]

Lysbeth Bakker, project manager
Peter Mol, project coordinator
Sieta de Vries, project co-lead