A framework for planning and critiquing medication compliance and persistence research using prospective study designs.

BACKGROUND: Medication compliance and persistence are important determinants of clinical outcomes. With the application of evidence-based therapy, it is increasingly important to ensure that studies that use compliance or persistence as a primary or secondary outcome are designed suitably and employ appropriate analyses to support the inferences made.

OBJECTIVE: The aim of this work was to describe the designs of medication compliance/persistence studies and provide guidance on appropriate analyses, with the ultimate goal of helping health providers and payers of health care understand the impact of compliance and persistence on health outcomes.

METHODS: MEDLINE, CINAHL, EMBASE, and all EBM Reviews databases were searched to locate key research articles about prospective medication compliance and persistence studies. Articles published between 1978 and 2008 were included in the search. Inclusion criteria included a focus on medication compliance and persistence, and prospective research designs. Articles that largely focused on retrospective study designs or were based on opinion rather than evidence were excluded.

RESULTS: A systematic framework was developed that comprised a prospective checklist and a quantitative tool to assess the quality of studies. The key elements of the checklist included the following: title and abstract, introduction or background, objectives, methods and study design, statistical analysis and results, discussion, conclusions, and disclosure of conflicts of interest. For each element, examples are provided to help readers make an informed decision about the design, value, and quality of a particular prospective study.

CONCLUSIONS: The checklist and quantitative tool can be used to provide objective validation of the rigor of prospective research designs. It is anticipated that future research will follow a uniform approach to presentation and evaluation of data, thereby facilitating a clear understanding of the impact of compliance and persistence on health outcomes.