Incorporating pharmacoeconomic and health outcomes into randomized clinical trials

Abstract

Clinical trials represent the most scientifically valid study design for evaluating the safety and efficacy of new medical treatments. However, for pharmacoeconomic outcomes, the usual efficacy clinical trial may not always provide the appropriate vehicle for generating information on the cost–effectiveness of medical treatments. Pragmatic, or naturalistic, clinical trials may be more suited to understanding cost–effectiveness. There are several issues associated with designing clinical trials that include patient-reported outcomes or pharmacoeconomic outcomes. For patient-reported outcomes, it is important to match the domains and instruments with the objectives and characteristics of the target patient population, examine psychometric characteristics (i.e., reliability, validity and responsiveness) when selecting measures, and also consider practical issues related to data collection. Plans need to be put into place to minimize missing patient-reported outcome item level and form data. For pharmacoeconomic outcomes, it is necessary to collect healthcare resource data and to value these resources in some way. This may be challenging when multinational clinical trials are designed and conducted. There are challenges associated with the analysis of cost data and in conducting cost–effectiveness analyses. Design and reporting of prospective, pharmacoeconomic trials will help inform evidence-based medicine and hopefully improve the delivery and outcomes of medical care.

Keywords::

• clinical trial
• safety and efficacy
• PRO
• cost–effectiveness
• randomized controlled trial