EDRN Clinical Utility Trial Proposal Review Criteria
|
Significance Does it address an unmet clinical need, how big is that unmet need? What is the performance of the current clinical decision rule? How strong is the evidence of performance characteristics (e.g. sensitivity, specificity, PPV/NPV) for the proposed test/decision rule? Has it been validated in Phase 3 study with adequate sample size, strong study design, and appropriate blinding? Has the cutoff/decision rule been locked down in previous validation studies? What are the potential harms and benefits for the proposed intervention? Has this harm/benefit trade-off been quantified by analyses, simulation, or modeling? Will the trial hypothesis if confirmed have high likelihood to change the current clinical practice (acceptance by patients, health care providers, payers, health policy makers)? Will the new test enhance or reduce equity in heterogenous populations? Is the assay clinical grade? How robust the assay is in analytical reproducibility, storage and process requirements, transportability (only can be run in central lab or any CLIA labs)? |
|
Study Design (Approaches) Are the proposed endpoints (e.g., stage shift, mortality, #unnecessary procedures, etc.), particularly the primary study outcome and effect size, well justified? Does preliminary data support it? Does it support the suggested potential clinical utility? Can the primary outcome be adequately ascertained (e.g., biopsy confirmation rate, diagnostic work-up imaging sensitivity, MCED test resolution rate)? Has the challenge been adequately addressed? Do inclusion/exclusion criteria appropriately reflect the target population, and with high chance for successful recruitment? Are choices of control and intervention arms well justified? If it is a single arm intervention study, will the trial findings be convincing enough without control arm to change clinical practice or to lead to Phase 5 trial? If it is an observational study, will it be able to demonstrate potential clinical utility with high likelihood to change clinical practice? Is sample size well justified for controlling type 1 and type 2 error? Is there a safeguard build-in to adjust sample size or make go/no-go decision (e.g., interim analysis, group sequential design, adaptive design)? Are there adequate considerations for various threats to trial success? Are potential harms adequately considered and handled? |
|
Feasibility PI and the study team’s track record in recruitment, managing multi-center studies. The likelihood of acceptance and buy-in from target population and health care providers. Is recruitment plan convincing, outcome event rate projection reasonable? Does the trial team have appropriate infrastructure ready or could be quickly deployed to launch the trial? Will the trial be able to complete within this funding cycle? Is the resource allocation efficient and leveraged on the local existing infrastructure, resource, medical practice, industrial partnership, etc. |