Workshop chair Debra Leonard concluded the workshop by highlighting key questions and topics for further discussion (perhaps in future workshops facilitated by the IOM roundtable) and potential action items identified from the discussions.
Consider how to close the gap between FDA and payers’ evidence requirements. (The FDA, payers, evidence-based review groups, and providers all expressed a willingness to come together with IOM facilitation for further discussion.)
Conduct an analysis of the cost-effectiveness of an “analytic framework” process (constructing a chain of evidence from pieces that together provide adequate supporting data) versus conducting one or two high-quality, prospective randomized controlled clinical trials.
Define what constitutes “adequate evidence.” Perfect evidence is unattainable. What level of certainty will allow the transition of a genomic intervention into clinical practice?
Educate the public on the need for evidence to support clinical tests and clinical practice. Create a public demand for evidence and reduce the demand for tests simply because they are available or new.
Discuss new economic/reimbursement models that place value on tests that can help identify when a particular treatment will not be beneficial and thereby prevent unnecessary costly therapeutic interventions.
Discuss implementation of a system that does not pay for treatment if a prognostic or predictive genomic test is available and the results of that test do not support treatment in the patient.
Consider whether safety and efficacy (as determined by FDA review and approval/clearance) is sufficient to support the clinical use of a new genomic test in the context of medical practice relative to an individual patient’s situation or whether large amounts of population data should be required.