Effectiveness guidance document (EGD) for Chinese medicine trials: a consensus document.

BACKGROUND: There is a need for more Comparative Effectiveness Research (CER) on Chinese medicine (CM) to inform clinical and policy decision-making. This document aims to provide consensus advice for the design of CER trials on CM for researchers. It broadly aims to ensure more adequate design and optimal use of resources in generating evidence for CM to inform stakeholder decision-making. METHODS: The Effectiveness Guidance Document (EGD) development was based on multiple consensus procedures (survey, written Delphi rounds, interactive consensus workshop, international expert review). To balance aspects of internal and external validity, multiple stakeholders, including patients, clinicians, researchers and payers were involved in creating this document. RESULTS: Recommendations were developed for "using available data" and "future clinical studies". The recommendations for future trials focus on randomized trials and cover the following areas: designing CER studies, treatments, expertise and setting, outcomes, study design and statistical analyses, economic evaluation, and publication. CONCLUSION: The present EGD provides the first systematic methodological guidance for future CER trials on CM and can be applied to single or multi-component treatments. While CONSORT statements provide guidelines for reporting studies, EGDs provide recommendations for the design of future studies and can contribute to a more strategic use of limited research resources, as well as greater consistency in trial design.

Compendia and anticancer therapy under Medicare.

In 1993, Congress directed the Medicare program to refer to 3 existing published compendia, American Medical Association Drug Evaluations (AMA-DE), United States Pharmacopoeia Drug Information for the Health Professional (USP-DI), and American Hospital Formulary Service Drug Information (AHFS-DI), to identify unlabeled but medically accepted uses of drugs and biologicals in anticancer chemotherapy regimens. Public discussion during the preceding years had centered on whether to designate unlabeled uses of anticancer treatments as experimental and thus outside the scope of Medicare benefits. American Medical Association Drug Evaluations and USP-DI subsequently ceased publication, and the Medicare program faced increasing calls to revise the list of acceptable compendia, as authorized in the statute. In 2007, the Centers for Medicare & Medicaid Services used its regulatory authority to establish a publicly transparent process to revise the list. The Centers for Medicare & Medicaid Services considered 5 requests in 2008 and added National Comprehensive Cancer Network Drugs and Biologics Compendium, DRUGDEX, and Clinical Pharmacology to the list of compendia. DrugPoints was not added, and AMA-DE was removed. Because of the potential for conflicts of interest to lead to biased judgments, the 2008 Medicare Improvements for Patients and Providers Act has a provision that explicitly prohibits inclusion of compendia that do not have a publicly transparent process for evaluating therapies and identifying potential conflicts of interest.

Preventing tomorrow's sudden cardiac death today: part II: Translating sudden cardiac death risk assessment strategies into practice and policy.

Although current evidence supporting a more precise strategy for identifying patients at highest risk for sudden cardiac death (SCD) is sparse, strategies for translating existing and future evidence into clinical practice and policy are needed today. A great many unanswered questions exist. Examples include the following: At what level of risk for SCD should we pursue further testing or therapy? How should clinical strategies ethically and economically balance alternative outcomes? How can we best translate optimal strategies into clinical practice so as to prevent tomorrow's SCDs? On July 20 and 21, 2006, a group of individuals with expertise in clinical cardiovascular medicine, biostatistics, economics, and health policy was joined by government (Food and Drug Administration; Centers for Medicare and Medicaid Services; National Heart, Lung, and Blood Institute; Agency for Healthcare Research and Quality), professional societies (Heart Rhythm Society), and industry to discuss strategies for risk assessment and prevention of SCD. The meeting was organized by the Duke Center for the Prevention of Sudden Cardiac Death and the Duke Clinical Research Institute. This article, the second of 2 documents, summarizes the policy discussions of that meeting, discusses an analytic framework for evaluating the risks and benefits associated with SCD prevention and risk stratification, and addresses the translation of SCD risk assessment strategies into practice and policy.