Cost-Effectiveness Analysis (CEA) Studies
Cost-Effectiveness Analysis (CEA) provides useful information to help health care payers manage the use of costly medical technologies in order to maximize the health of their patient populations when facing constrained budgets, and to clinicians and patients to help guide treatment decisions based on CEA's unique endpoints, perspectives (e.g., societal, clinical, or third-party), and time horizon (e.g., within trial or long-term survivorship). To be most useful to decision-makers, CEA of new cancer therapies must have maximal feasibility, be timely, and have high internal validity.
Conducting a CEA alongside a clinical trial can achieve these goals and also offers the benefit of efficiency by utilizing the existing structure of clinical trials to collect additional data for the economic analysis. It is not required that a CEA proposal be included with each clinical trial concept submitted. However, in some instances the addition of CEA may be recommended during evaluation review of the clinical trial concept.
The CEA evaluation criteria are intended to help guide the selection of cancer clinical trials that warrant additional funds for a CEA. The CEA study should be a secondary endpoint of the parent concept. SSCs evaluate CEA proposals paired with clinical trial concepts through their concept evaluation and prioritization process. SSCs will make use of ad hoc CEA expert(s), including resources available at the NCI, to evaluate CEA proposals included in clinical trial concepts.
Criteria for Review of CEA Proposals
Researchers should consider pairing a CEA proposal to phase 3 clinical trials when the following conditions are met:
- The results of a phase 3 clinical trial are expected to substantially influence clinical practice.
- The cost-effectiveness study would be of high impact judged by substantial budget implications for health care systems, either in terms of overall cost savings or added costs to the system.
- It is feasible to conduct a high quality CEA as part of the clinical trial. Specific issues to consider include:
- – The comparator arm should be relevant to current clinical practice.
- – The trial should be of sufficient duration, with respect to follow-up of patient outcomes, that consequences of interest to economic evaluation can be captured either directly or through modeling.
- – There is reasonable statistical power for the key cost-effectiveness analysis.
- Because of high cost, there is a reasonable degree of uncertainty regarding the outcome of the CEA even if the clinical outcome favors the experimental treatment.
CEA proposals included in phase 3 clinical trial concepts should be developed by CGs and CCOP Research Bases. When CGs and CCOP Research Bases choose to submit a CEA proposal, this must be submitted with the phase 3 parent clinical trial concept.
