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Individual Treatment Effects: Implications for Research, Clinical Practice, and Policy

Jennifer S. Graff, PharmD; Thaddeus Grasela, PharmD, PhD; David O. Meltzer, MD, PhD; and Robert W. Dubois, MD, PhD
A framework for researchers, providers, payers, or public health bodies identifies when it is most critical to distinguish between "average" population and individual patient response.
CER will increasingly provide evidence about what treatments work best. For CER to achieve the greatest benefit, we need that evidence for groups of patients as well as for those individuals who may respond differently. We also need to understand the implications of applying population results to particular patients. In some circumstances, population results can and should be applied broadly. In other circumstances, caution is warranted. It is our hope that the framework discussed here begins to address this choice. We hope to encourage discussion about the importance of recognizing when additional research is needed, and about why providers, payers, and policy makers should acknowledge the implications of variation in treatment effect. Doing so should ultimately lead to improvements in health not only for the population as a whole, but also for individual patients like Ms Jones.

Author Affiliations: National Pharmaceutical Council, Washington, DC (JSG, RWD); Cognigen Corporation, Buffalo, NY (TG); Center for Health and the Social Sciences, University of Chicago, Chicago, IL (DOM).

Source of Funding: National Pharmaceutical Council.

Author Disclosures: Drs Graff and DuBois are employed by the Na- tional Pharmaceutical Council, a policy research organization supported by the nation’s major research-based pharmaceutical companies. Dr Grasela is an employee of Cognigen, which provides contract research services in the area of pharmacokinetic and pharmacodynamic modeling. Cognigen also receives consulting fees and fees for public speaking on behalf of client products and projects. Dr Meltzer is a consultant for Patient-Centered Outcomes Research Institute (PCORI) as a member of the methodology committee, and has received research grants for other projects from the National Pharmaceutical Council.

Authorship Information: Concept and design (JSG, RWD, DOM, TG); acquisition of data (JSG, RWD, TG); analysis and interpretation of data (JSG, RWD); drafting of the manuscript (JSG, RWD, DOM, TG); criti- cal revision of the manuscript for important intellectual content (RWD, DOM, TG); obtaining funding (RWD); administrative, technical, or logis- tic support (JSG); supervision (RWD).

Address correspondence to: Jennifer S. Graff, PharmD, National Pharmaceutical Council, 1717 Pennsylvania Ave, Ste 800, Washington, DC 20006. E-mail: jgraff@npcnow.org.
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