Health care decision makers involved with coverage and payment policies for drugs and devices have traditionally relied on randomized clinical trials (RCTs) for coverage and reimbursement decisions. However, a new report published in the September-October issue of Value in Health documents how decision makers are increasingly seeking evidence on "real-world" outcomes on which to base conclusions. A special task force convened by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) studied these informational needs and concluded that "real-world data are essential for sound coverage and reimbursement decisions."

Though extremely valuable and likely to remain the gold standard, RCTs are conducted under experimental conditions, with rigid protocols for patient selection and treatments. Real-world conditions are different and potentially more relevant as a broader set of patients may receive a wider range of treatments.

The 20-member ISPOR Task Force, co-chaired by Professor Lou Garrison of the University of Washington and Professor Peter Neumann of Tufts-New England Medical Center, examined the pros and cons of using the following types of real-world data: large pragmatic trials, patient registries, administrative claims databases, surveys, and medical records. The task force concluded that each of these types of data can contribute to our understanding of what health outcomes are actually being achieved in the real world. Yet each has methodological limitations and operational challenges that must be considered.

In a commentary accompanying the publication of the report, York University's Professor Mike Drummond, immediate past president of ISPOR, urged that "detailed methodological guidance for the collection of real-world data needs to be produced" and that "reimbursement agencies need to engage in more discussions about the types of data required and the decisions that will result."

These remarks underscore the next steps identified by the task force. In addition, the task force highlighted important related needs, including the needs for modeling, assessment of the costs and benefits of data collection, a transparent process, and ongoing dialog among stakeholders.

Louis P. Garrison, Jr., PhD, is Professor and Associate Director of the Pharmaceutical Outcomes Research and Policy Program in the Department of Pharmacy at the University of Washington. His research interests include policies to encourage appropriate innovation and risk-benefit analyses of new drugs. Professor Garrison can be reached at 206-221-5684 or lgarrisnu.washington.

Peter J. Neumann, ScD, is Director of the Center for the Evaluation of Value and Risk in Health at Tufts-New England Medical Center and Professor of Medicine at Tufts University School of Medicine. His research focuses on the application of cost-effectiveness analysis in health care. Professor Neumann can be reached at pneumanntufts-nemc.

Value in Health (ISSN 1098-3015) publishes papers, concepts, and ideas that advance the field of pharmacoeconomics and outcomes research and help health care leaders to make decisions that are solidly evidence-based. The journal is published bi-monthly and has a regular readership of over 3,000 clinicians, decision-makers, and researchers worldwide.

ISPOR is a nonprofit, international organization that strives to translate pharmacoeconomics and outcomes research into practice to ensure that society allocates scarce health care resources wisely, fairly, and efficiently.

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