Study identification

PURI

https://redirect.ema.europa.eu/resource/1000000088

EU PAS number

EUPAS1000000088

Study ID

1000000088

Official title and acronym

DARWIN EU® Comparing direct and indirect methods to estimate prevalence of chronic diseases using real-world data

DARWIN EU® study

Yes

Study countries

Netherlands
Spain
United Kingdom

Study description

In the context of chronic diseases with relatively low prevalence, how do direct and indirect RWD-based estimates of prevalence compare with each other?

The specific objectives of this study are to:
1) Estimate the disease prevalence (direct estimate based on the proportion of individuals with the condition).
2) Estimate the disease incidence rate.
3) Estimate duration of disease using Kaplan-Meier survival curves. Of particular interest is the estimate of median survival as a summary measure of disease duration.
4) Produce an indirect estimation of prevalence as the product of incidence and median survival.
for the following diseases:
- Cystic fibrosis
- Haemophilia (A and/or B)
- Pulmonary arterial hypertension
- Pancreatic cancer
- Sickle cell disease

Results will be provided overall and where possible stratified by age group: paediatrics (0-17 years old) and adults (18 years old and above).

Study status

Finalised
Research institution and networks

Networks

Contact details

Maria de Ridder

Primary lead investigator
Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:
Actual:

Date of final study report

Planned:
Actual:
Sources of funding
EMA
Study protocol
Initial protocol

DARWIN EU_D2.2.3_Protocol_P2-C1-013_Direct and indirect prevalence estimation V2.pdf

English (659 KB - PDF)View document
Regulatory

Was the study required by a regulatory body?

Yes

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

Not applicable