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Review
. 2022 Jan;31(1):13-21.
doi: 10.1002/pds.5372. Epub 2021 Oct 29.

A systematic, concept-based method of developing the exposure measure for drug safety and effectiveness studies

Lisa K Kuramoto  1 , Boris G Sobolev  1   2 , Jan Rosner  3   4 , Penelope M A Brasher  1 , Laurent Azoulay  5 , Jacquelyn J Cragg  3   6
Affiliations
Review

A systematic, concept-based method of developing the exposure measure for drug safety and effectiveness studies

Lisa K Kuramoto et al. Pharmacoepidemiol Drug Saf. 2022 Jan.

Abstract

Purpose: In drug safety and effectiveness studies based on secondary data, the choice of an appropriate exposure measure for a given outcome can be challenging. Different measures of exposure can yield different estimates of treatment effect and safety. There is a knowledge gap with respect to developing and refining measures of drug exposure, to ensure that the exposure measure addresses the study question and is suitable for statistical analysis.

Methods: We present a transparent, step-by-step approach to the development of drug exposure measures involving secondary data. This approach would be of interest to students and investigators with initial training in pharmacoepidemiology. We illustrate the approach using a study about Parkinson's disease.

Results: We described the exposure specifications according to the study question. Next, we refined the exposure measure by linking it to knowledge about four major concepts in drug safety and effectiveness studies: drug use patterns, duration, timing, and dose. We then used this knowledge to guide the ultimate choice of exposure measure: time-varying, cumulative 6-month exposure to tamsulosin (a drug used to treat prostate hyperplasia).

Conclusions: The proposed approach links exposure specifications to four major concepts in drug safety and effectiveness studies. Formulating subject-matter knowledge about these major concepts provides an avenue to develop the rationale and specifications for the exposure measure.

Keywords: Parkinson disease; drug evaluation; drug utilization; pharmacoepidemiology.

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References

REFERENCES

    1. Guyatt GH, Haynes RB, Jaeschke RZ, et al. Users' guides to the medical literature: XXV. Evidence-based medicine: principles for applying the Users' guides to patient care. Evidence-Based Medicine Working Group. JAMA. 2000;284(10):1290-1296. doi:10.1001/jama.284.10.1290
    1. Morgan RL, Whaley P, Thayer KA, Schunemann HJ. Identifying the PECO: A framework for formulating good questions to explore the association of environmental and other exposures with health outcomes. Environ Int. 2018;121(1):1027-1031. doi:10.1016/j.envint.2018.07.015
    1. Speckman RA, Friedly JL. Asking structured, answerable clinical questions using the population, intervention/comparator, outcome (PICO) framework. PM R. 2019;11(5):548-553. doi:10.1002/pmrj.12116
    1. Chodick G, Amital H, Shalem Y, et al. Persistence with statins and onset of rheumatoid arthritis: a population-based cohort study. PLoS Med. 2010;7(9):e1000336. doi:10.1371/journal.pmed.1000336
    1. Edmonds A, Yotebieng M, Lusiama J, et al. The effect of highly active antiretroviral therapy on the survival of HIV-infected children in a resource-deprived setting: a cohort study. PLoS Med. 2011;8(6):e1001044. doi:10.1371/journal.pmed.1001044

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