Validation of real-life asthma research endpoints (Real-life asthma endpoint validation)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/31033
• EU PAS number:
  EUPAS4860
• Study ID:
  31033
• Official title and acronym:
  Validation of real-life asthma research endpoints (Real-life asthma endpoint validation)
• DARWIN EU® study:
  No
• Study countries:
  United Kingdom
• Study description:
  The results of real-life respiratory studies often meet with challenges from peer reviewers who are unfamiliar with the described methods. Particularly problematic are real-life outcomes that refer to “asthma control,” as they differ from the traditional, validated clinical tools. Reviewers often challenge the endpoints, and composite endpoints, used in database studies in terms of their validity – are they truly are accurate reflections of the clinical reality they are attempting to measure. Usually these concerns implicitly assume (that the gold standard in outcome assessment are the measures used in prospective randomized controlled trial (RCTs), and thus request information regarding the association between real-life outcome assessments and RCT-used tools. The aim of this study is to validate a series of objective asthma control measures that have been used in published real-life respiratory research. The outcome measures will be compared to patient-reported outcomes and/or to validated RCT asthma measures (as appropriate). Where possible, their validity, responsiveness and predictive value will also be assessed and a rank order of outcomes (and possibly hierarchical modelling) will be established to aid in appropriate outcome selection for future database studies.
• Study status:
  Finalised

Research institution and networks

Institutions

Research in Real Life

Networks

Respiratory Effectiveness Group (REG)

Belgium

Denmark

France

Germany

Greece

Hungary

Italy

Netherlands

Spain

Sweden

United Kingdom

Study timelines

Date when funding contract was signed

Planned: 15/02/2013

Actual: 30/08/2013

Study start date

Planned: 01/01/2004

Actual: 31/12/2012

Date of final study report

Planned: 30/04/2013

Actual: 01/01/2018

Sources of funding

Other

More details on funding

Respiratory Effectiveness Group, Research in Real Life

Study protocol

Initial protocol

Endpoint validation_REG study proposal_26Feb13_FINAL_ADEPT APPROVED_30Sept13

Regulatory

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