Personalized medicine and the role of health economics and outcomes research: issues, applications, emerging trends, and future research.

The decade since the completion of the sequencing of the human genome has witnessed significant advances in the incorporation of genomic information in diagnostic, treatment, and reimbursement practices. Indeed, as case in point, there are now several dozen commercially available genomic tests routinely applied across a wide range of disease states in predictive or prognostic applications. Moreover, many involved in the advancement of personalized medicine would view emerging approaches to stratify patients in meaningful ways beyond genomic information as a signal of the progress made. Yet despite these advances, there remains a general sense of dissatisfaction about the progress of personalized medicine in terms of its contribution to the drug development process, to the efficiency and effectiveness of health care delivery, and ultimately to the provision of the right treatment to the right patient at the right time. Academicians, payers, and manufacturers alike are struggling not only with how to embed the new insights that personalized medicine promises but also with the fundamental issues of application in early drug development, implications for health technology assessment, new demands on traditional health economic and outcomes research methods, and implications for reimbursement and access. In fact, seemingly prosaic issues such as the definition and composition of the term "personalized medicine" are still unresolved. Regardless of these issues, practitioners are increasingly compelled to find practical solutions to the challenges and opportunities presented by the evolving face of personalized medicine today. Accordingly, the articles comprising this Special Issue offer applied perspectives geared toward professionals and policymakers in the field grappling with developing, assessing, implementing, and reimbursing personalized medicine approaches. Starting with a framework with which to characterize personalized medicine, this Special Issue proceeds to illuminate issues related to the intersection of personalized medicine and comparative effectiveness; use of personalized medicine approaches in drug development; methodological challenges; and payer approaches to evaluation and reimbursement of pharmacodiagnostics in the United States and Europe. It concludes with a look ahead, underscoring current controversies yet to be resolved along with their implications for further research and policy. It is hoped that these articles will help inform the daily challenges faced by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) community as it collectively addresses what promises to be a new era in drug development and health care delivery.