Study identification

PURI

https://redirect.ema.europa.eu/resource/106787

EU PAS number

EUPAS105257

Study ID

106787

Official title and acronym

Study of Operating characteristic of Bayesian methods that borrow treatment effects

DARWIN EU® study

No

Study countries

France
United States

Study description

While the appropriateness of Bayesian methods in the paediatric context is largely recognised, most existing methods refer to borrowing control arm data only. From a regulatory perspective, an important issue with Bayesian methods is that they potentially do not control the Type I Error. Many simulation studies have been performed within the last decade in order to evaluate the operating characteristics, e.g. regarding Type I Error inflation and possible gains with respect to power, associated with Bayesian methods. Moreover, these methods are known to be potentially sensitive to small changes in underlying parameters, such as a weight parameter which determines how much information is borrowed from the historical data, and could potentially leads to different conclusions. Considering the limitations associated with these methodologies, an extension towards borrowing treatment effect, instead of historical control data only, is considered. The primary aim of this Tender is to thoroughly evaluate the available Bayesian methods for borrowing information on treatment effects in the context of paediatric evaluations. More specifically, specific objectives of this project include: - Review and evaluate available and published methods for dynamic borrowing via a structured and critical review (not limited to paediatric extrapolation) - Provide a clear mathematical specification of each model identified, articulating the interpretation of underlying parameters, and define the relevant operating characteristics (OCs) and performance metrics to evaluate for a given context - Evaluate and compare (via simulations) performance of such methods along the pre-defined OCs in various common scenarios - Synthesize and disseminate overall conclusions and recommendations

Study status

Ongoing
Research institutions and networks

Institutions

Quinten Health
First published:
01/02/2024
Institution
Daniel Lee New York, USA

Contact details

Billy Amzal

Primary lead investigator
Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:
Actual:

Date of final study report

Planned:
Sources of funding
Other

More details on funding

European Medicines Agency
Regulatory

Was the study required by a regulatory body?

Yes

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

Not applicable