Study identification

PURI

https://redirect.ema.europa.eu/resource/1000000539

EU PAS number

EUPAS1000000539

Study ID

1000000539

Official title and acronym

Comparative effectiveness and safety studies using the target trial emulation and estimand frameworks (TARGET-EU)

DARWIN EU® study

No

Study countries

Belgium
Denmark
Finland
Italy
Netherlands
Norway
Spain
United Kingdom

Study description

This project, entitled Comparative Effectiveness and Safety Studies Using the Target Trial Emulation and Estimand Frameworks (TARGET EU), aims to advance regulatory use of real-world data (RWD) through application of target trial emulation (TTE) and estimand methodologies.
The overarching goal is to generate evidence that bridges randomized controlled trials (RCTs) and observational studies, supporting regulatory decisions where RCTs are infeasible.
TARGET EU comprises four objectives:
(1) to review and assess the integration of TTE with the estimand framework for evaluating drug effectiveness and safety,
(2) to characterise the suitability of diverse European RWD sources for TTE,
(3) to identify regulatory scenarios—such as PASS, PAES, and trials with external comparators—where TTE provides value, and
(4) to establish good practice guidelines for communicating TTE results to regulators.
To achieve these aims, the consortium will conduct a literature review and regulatory landscape analysis; assess data quality, accessibility, and fitness-for-purpose using a structured framework; and implement ten emulated trials using rich datasets from the Netherlands, Spain, and the UK, with expansion to other EU countries.
These case studies span public health priorities, including COVID-19 vaccine effectiveness and comparative oncology drug safety.

Study status

Planned
Research institutions and networks

Institutions

Teamit Institute
Spain
First published:
12/03/2024
InstitutionOtherENCePP partner
Sint Antonius Hospital

Networks

Contact details

Olaf Klungel

Primary lead investigator
Study timelines

Date when funding contract was signed

Planned:

Study start date

Planned:

Date of final study report

Planned:
Sources of funding
EMA
Regulatory

Was the study required by a regulatory body?

Yes

Is the study required by a Risk Management Plan (RMP)?

Not applicable