Realizing the Benefits of Practical Clinical TrialsRealizing the Benefits of Practical Clinical Trials

To the Editor: Dr Tunis and colleagues¹ discussed the need for practical clinical trials (PCTs) to better meet the information needs of clinical and health policy decision makers. While there is a growing need for rigorous effectiveness studies, we doubt that such studies alone will be sufficient. First, the conduct of PCTs will not ensure that evidence is translated into clinical practice. The poor translation of evidence into practice is illustrated by numerous examples of suboptimal quality of care despite high-quality evidence.² Second, despite the presence of rigorous economic studies, efficiency and cost-effectiveness are infrequently incorporated into clinical practice guidelines.³ Third, clinical practice guidelines require significant effort for implementation, yet few health care settings have the necessary infrastructure. Rigorous effectiveness trials will be of little benefit until these issues are addressed.

Because manufacturers are among the largest supporters of clinical trials, there must be appropriate incentives for commercial entities to bear the financial and commercial risk in the face of the potential benefits of PCTs. We agree with Tunis et al that demand by purchasers is one incentive, but more important is demonstration of transparent and consistent application of the evidence in decision making. Health care purchasers frequently have disaggregated decision-making processes, with hospital, outpatient care, and pharmacy costs often considered in isolation from one another. The use of cost-effectiveness information to inform Centers for Medicare & Medicaid Services coverage and payment decisions remains ill-defined and, despite the adoption of the Academy of Managed Care Pharmacy formulary dossier, the application for managed care formulary decision making has not been formally evaluated.⁴ Consistent application of the evidence by purchasers and decision makers will be a major stimulus for high-quality effectiveness research.

We believe that there is also a need for methodological standards for the design and conduct of PCTs, cluster randomized trials, and quasi-experimental⁵ studies. This could occur under the guidance of a national task force comprised of government, academic, provider, purchaser, and industry representatives. The most critical steps to ensure that PCTs are conducted is to set national funding priorities, and to demonstrate the consistent application of this evidence by purchasers. The appropriate conduct of these studies would provide the evidence base to improve decision making in an increasingly constrained financial climate in which the incremental costs and benefits of competing technology must be demonstrated.