Discussion

As RWD is increasingly used to generate evidence to make safety and efficacy decisions, the challenges and lack of clarity associated with incorporating medication use into analyses should be addressed. We offered points to consider when developing medication use definitions, with a focus on electronic health care data sources. These considerations may help researchers understand the strengths and limitations of different conceptual and operational approaches (e.g., selection of data sources, method of identifying medication) and contextualize their findings. These points are intended to complement considerations from existing rubrics, such as STaRT-RWE[6], the ISPE-ISPOR Consensus Document[7], RECORD-PE[8], and the ENCEPP Guide on Methodological Standards[9], which aid investigators in designing, conducting, reporting on, and comparing across studies.
Our proposed points included considerations for investigators who incorporate medication use into their study. We have shown the importance of the considering context under which the medication is being used in the analyses, the volume of medications formulas, dosages, routes of administration, and the often-longitudinal nature of medication use may influence a definition of medication use.
In summary, adopting and reporting on the use of the proposed considerations may enhance the quality of pharmacoepidemiology research and our ability to contextualize and interpret findings.
Funding statement: This activity is a project supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) as part of an award of $570,206 in federal funds (100% of the project).  The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by FDA, HHS, or the U.S. Government. For more information, please visit FDA.gov.
Conflict of interest disclosure: Anand P. Chokkalingam is an employee of and own stock in Gilead Sciences. The rest of the authors have no competing interests to declare that are relevant to the content of this article.
Ethics approval statement: Because this wasn’t a research activity it is exempt from review and the requirement for informed consent.