The need for increased harmonisation of clinical trials and economic evaluations

Abstract

Despite the increasing number of protocol and reporting guidelines available to trialists, there is still little guidance for protocol writers on the incorporation of patient-reported outcomes and economic assessments alongside clinical trials. It is unsurprising, therefore, that trial protocols present disproportionately less information for the economic evaluation component than for clinical outcomes. Costing methodologies, generalisability considerations, methods to address sensitive patient-reported outcome information and missing data are often insufficiently described in the trial protocol. The paper illustrates these shortcomings with specific examples and makes a case for shifting researchers’ attention from the reporting to the design stage of trial-based economic evaluation to promote the validity, generalisability and accountability of trial-based economic evaluations.

Keywords::

• clinical trials
• economic evaluation
• generalisability
• patient-reported outcomes
• protocol
• study design

Financial & competing interests disclosure

D Kyte is supported by a National Institute for Health Research School for Primary Care Research funded PhD studentship. M Calvert is a member of the MRC Midland Hub for Trials Methodology Research, University of Birmingham, United Kingdom (MRC Grant ID G0800808). The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.