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The Official News & Technical Journal Of The International Society For Pharmacoeconomics And Outcomes Research

Diana Brixner PhD, 2007-2008 ISPOR President and Associate Professor and Chair of the Department of Pharmacotherapy and Executive Director of the Pharmacotherapy Outcomes Research Center at the University of Utah College of Pharmacy, Salt Lake City, UT, USA

Translational Research and the Value Equation

A recent commentary published in the Journal of the American Medical Association addressed the “Meaning of Translational Research and Why it Matters” [1]. In light of ISPOR related topics such as outcomes research, comparative effectiveness, cost-effectiveness, and pharmacoeconomics, it seems timely to consider where these disciplines fit in the continuum of translational research.

As Woolf acknowledges in his article, translational research means different things to different people. The more traditional definition of “bench to bedside” encompasses drug discovery (medicinal chemistry), drug formulation (pharmaceutics), drug testing (pharmacology and clinical development), and patient care (pharmacotherapy). These definitions align particularly well with the Departments in our own College of Pharmacy at the University of Utah, and, most likely, with various other colleges and pharmaceutical companies across the globe. The Institute of Medicine (IOM) Clinical Research Roundtable has labeled this definition as “translational block one”, or T1 [2]. The components of T1 have been traditionally funded by individual National Institutes of Health (NIH) institutes and now collectively through the NIH roadmap initiative [3] and by the launch of the Clinical and Translational Science Award (CTSA) with a goal of $500 million in funding across 60 academic centers by 2012.

An alternative definition of translational research, perhaps more relevant to the disciplines represented by ISPOR and other population-based organizations such as the International Society of Pharmacoepidemiology, Academy Health, Society for Medical Decision Making and others, would be “translating research into practice”. Here the disciplines of epidemiology, evidencebased synthesis, economics, public policy, behavioral science, and biostatistics play a much larger role in understanding how the real world of clinical practice, patient behaviors, and concomitant disease can impact the predictions of the highest quality research produced by randomized clinical trials. The IOM Clinical Research Roundtable labeled this as ”translational block two” or T2 [2]. The challenges of this type of research abound. In specific research on how a new technology can be introduced into practice, one must consider the nuances of a health care system that has previously operated in the absence of such technology. Treatment guidelines are reviewed and revised, systematic reviews are redone, and documentation, such as the Academy of Managed Care Pharmacy (AMCP) Dossier and dossiers for other global region reimbursement agencies, is prepared and updated. The evidence that is created to support these documents is largely conducted in patient populations from primary care physician practices, integrated health care systems, and national administrative claims databases. This “real world” research can include assessment of the impact of therapeutic guidelines on the treatment and outcomes of disease, the impact of disease or medication therapy management intervention programs, and health services research to evaluate the benefit of vaccination or diagnostic screening programs. The practice-based component of this research is primarily conducted in community or ambulatory settings to provide insight on the impact and outcomes of implementing new technologies. The practice-based research was potentially referred to as T3 by Westfall and colleagues [4] to more specifically acknowledge this practice-based approach. Either way the dynamics of funding research in the T-2 T-3 arena is far different than the funding of the more traditional T1 translational research. The adaptation of the NIH towards translational research had dedicated $787 million of a $22.1 billion budget to health services research, and the previously mentioned resources have been directed towards the CTSA, however, with a focus on T1. In fact academic centers across the U.S have struggled with how to incorporate community-based primary care research and pharmacotherapy outcomes research into CTSA grant applications. A more likely source for practice-based research has been the Agency for healthcare Research and Quality (AHRQ) with the charge that research results are widely disseminated and used in healthcare decision making [5]. However, the funding for this mission is sparse with roughly a $300 million budget initially targeted at translational research in practice grants with a shift toward special project research on patient safety and informatics.

A larger concern is that the conversation of defining translational research largely stops here. Although we can study the implementation of technologies into practice, we have neither discussed research that is focused on studying the comparative effectiveness of different technologies nor the associated costs. Yet these two disciplines are the core principles of ISPOR. The benefit and limitations of conducting practice based research have been largely recognized and many of these same limitations apply to real world effectiveness studies; however, there is also acknowledged benefit of this information to the payer. Drugs and/or other technologies should not be expected to act the same way when patient behaviors, disease states, and other factors are not controlled as they are in randomized trials, and these differences can be important in making coverage decisions. The process of rational health care resource allocation should include economics, but how this information is collected and how it is interpreted are important considerations. These ongoing debates are also reflected in the associated funding sources, both for comparative and cost effectiveness, which at this juncture are largely absent outside of those vested in the results.

In summary, there is a continuum of research from bench to bedside to practice to resource allocation where the clarity of definition seems to wane and the funding sources seem to diminish. However, in all likelihood, this is a reflection of the societal changes in how medicines are discovered, applied, and paid for to prevent disease and improve human health. If we include comparative effectiveness and cost-effectiveness as logical next steps in the continuum of translational research, we should be pleased to know there is room for many more transitional states in the future.

1. Woolf SH. The meaning of translational research and why it matters. JAMA 2008;299:211-13.
2. Sung NS, Crowley EFJr., Genel M, et al. Central Challenges facing the national clinical research enterprise. JAMA 2003;289:1278-87.
3. Accessed January 16, 2008.
4. Westfall JM, Mold J, Fagnan L. Practice Based research - “blue highways” on the NIH roadmap. JAMA 2007;297:403-6.
5. Agency for Healthcare Research and Quality. Budget estimates for appropriations committees, fiscal year (FY) 2008: performance budget submission for congressional justification. Performance budget overview 2008 Accessed November 17, 2007


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