Use cases for development, optimisation and implementation of artificial intelligence methods for real world data analyses in regulatory decision-making and health technology assessment along the product lifecycle (Real4Reg)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/105545
• EU PAS number:
  EUPAS105544
• Study ID:
  105545
• Official title and acronym:
  Use cases for development, optimisation and implementation of artificial intelligence methods for real world data analyses in regulatory decision-making and health technology assessment along the product lifecycle (Real4Reg)
• DARWIN EU® study:
  No
• Study countries:
  Denmark

  Finland

  Germany

  Portugal
• Study description:
  The use of real world data (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. Also, the use of RWD in post-authorisation steps is constrained by data variability and by challenges in analysing data from different settings and sources. Moreover, there are emerging opportunities in the use of artificial intelligence (AI), but there is a lack of knowledge on its appropriate application to heterogeneous RWD sources to increase evidentiary value in the regulatory decision-making and health technology assessment (HTA) context. Thus, the development of new and optimised AI-supported methodologies for RWD analyses is essential. In addition, the four use cases to be investigated in this study contain phenotype-specific open questions of high regulatory interest.
• Study status:
  Ongoing

Research institution and networks

Institutions

Pharmacoepidemiology Research, Federal Institute for Drugs and Medical Devices (BfArM)

Germany

School of Pharmacy/Kuopio Research Center of Geriatric Care, University of Eastern Finland (Gerho)

Finland

Aarhus University & Aarhus University Hospital DEPARTMENT OF CLINICAL EPIDEMIOLOGY

Denmark

Data Analytic Center (DAC), Danish Medicine Agency

Denmark

National Authority of Medicines and Health Products, I.P. (Infarmed) Lisboa, Portugal;
Fraunhofer Society, Fraunhofer Institute for Algorithms and Scientific Computing (SCAI), Research Group AI and Data Science Sankt Augustin, Germany;
IT Centre for Science (CSC) Espoo, Finland;
German Centre for Neurodegenerative Diseases (DZNE), Research Group Pharmacoepidemiology Bonn, Germany;
European Organization for Professionals and Patients with ALS (EUpALS) Leuven, Belgium;
European Institute of Women’s Health (EIWH) Dublin, Ireland.

Networks

Real4Reg

Belgium

Denmark

Finland

Germany

Ireland

Portugal

Study timelines

Date when funding contract was signed

Planned: 05/11/2022

Actual: 11/11/2022

Study start date

Planned: 01/09/2023

Actual: 11/09/2023

Date of final study report

Planned: 31/12/2026

Sources of funding

Other

More details on funding

HORIZON Europe (European Union, European Comission)

Study protocol

Initial protocol

Study_protocols_Real4Reg_2023_June_29

Updated protocol

Study_protocols_Real4Reg_2024_April_11.pdf

Regulatory

• Was the study required by a regulatory body?:
  No
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable