Methods for Comparative Effectiveness Research
Effectiveness Guidance Documents (EGDs) provide specific recommendations to product developers and clinical researchers about the design of clinical studies that will produce evidence to inform decision making by patients, clinicians and payers. The goal is to describe clinical studies that would provide these “post-regulatory” decision makers with a reasonable level of confidence that the technology improves health outcomes. In this respect, they are intended to provide technology-specific methodological roadmaps for the design of prospective comparative effectiveness research. CMTP has developed several guidance documents, as listed below. These documents are in various states of completion, with some currently under development. Please select a title for more information.
Pragmatic Clinical Trials
In general, several major characteristics distinguish pragmatic trials and traditional clinical trials. First, PCTs involve the deliberate comparison of clinically relevant alternative treatments, chosen based on the most common decision-making scenarios. Traditional RCTs do not include highly relevant comparisons, leaving decision makers to make comparisons across trials, where assumptions, research methods, patient populations, and other crucial characteristics may be different. Second, PCTs are typically designed to be generalizable to real patient populations by including patients with common co-morbidities and from a variety of demographic backgrounds. Third, PCTs select clinically-relevant outcomes that are intended to address the primary issues and concerns of patients, clinicians and payers. Many RCTs include outcomes that are of primary interest to regulators, and pay less attention to the post-regulatory decision makers that will also use those studies to guide their choices. These clinically-relevant outcomes may include more quality-of-life information and may involve longer follow-up periods than are typical for traditional clinical trials. Importantly, the selection of the most useful and relevant outcomes requires direct consultation with decision makers during study protocol development.
New Indications for Oncology Drugs
Substantial uncertainty exists about the benefits and harms of many off-label uses of oncology drugs, creating an important challenge for clinical and health policy decision makers. The Center for Medical Technology Policy (CMTP) conducted a six-month initiative with the overarching goal of developing recommendations on methods and strategies to improve the validity, relevance and consistency of clinical research designed to assess the comparative effectiveness and value of oncology drugs used for off-label indications. As part of this initiative, CMTP convened a one-day multi-stakeholder New Indications for Oncology Drugs Think Tank to improve the systematic generation of evidence about the effectiveness of oncology drugs for new indications, providing a neutral ground to establish the steps necessary for improved evidence for drug therapy in cancer. A series of semi-structured interviews was also held with approximately twenty-five leading experts and stakeholders.
Devices for Local Treatment of Chronic Wounds
Difficult-to-heal and chronic wounds negatively affect quality of life and incur high health care costs. Although a number of devices directed at local treatment of chronic wounds have received approval by the Food and Drug Administration the evidence that any one device improves wound healing in selected patients over other devices is inconclusive. In order to increase the likelihood that further research will be conducted in the comparative effectiveness of different chronic wound treatments, and that this research will result in information that will aid medical decision makes (physicians, payers, patients, etc.), CMTP recently published an Effectiveness Guidance Document focusing on Devices for Local Treatment of Chronic Wounds. As with all of CMTP's EGDs, we are looking for comments on the content and structure of the document.
Non-invasive Imaging for Coronary Artery Disease
The pace of innovation in the field of non-invasive imaging diagnostics for Coronary Artery Disease (CAD) is has made it difficult to assess the comparative effectiveness and incremental value of these technologies relative to each other and other diagnostic procedures. This has left payers to devise coverage determinations for these technologies with little evidence of their clinical effectiveness and has left patients and clinicians without a clear understanding of the utility of these diagnostics. In order to address this issue and to develop a shared understanding of what type of evidence is minimally sufficient for decision makers (payers, clinicians, patients, and policy makers), CMTP, in collaboration with experts from all relevant stakeholder groups, will develop an Effectiveness Guidance Document on Non-Invasive Imaging for CAD that outlines what type of evidence is sufficient for decision making, and recommends how this evidence can be generated in a timely fashion to guide decisions about the use of the next new cardiac imaging technology.
Gene Expression Profile Tests for Early Stage Breast Cancer
Gene expression profile testing for early stage breast cancer was chosen as a promising molecular diagnostic tool with potential for reducing the incidence of unnecessary chemotherapy for a large number of women.
Methodological Recomendations for Comparative Effectiveness Research on the Treatment of Chronic Wounds
