New thinking on clinical utility: hard lessons for molecular diagnostics.

OBJECTIVES To describe 5 basic requirements for planning, implementing, and proving clinical utility for diagnostic tests, drawing on recent reimbursement decisions. STUDY DESIGN Review of recent reimbursement decisions by Palmetto GBA's MolDx program, and summary of lessons learned. METHODS Qualitative review of publicly available coverage and reimbursement decisions, plus our industry experience. RESULTS Lack of clinical utility data is the most commonly cited reason for why companies fail to receive favorable coverage and reimbursement decisions in this rapidly growing industry. We summarize 5 strategies to establish clinical utility and secure coverage with reimbursement: 1) understanding that outcomes are hard to capture, but that clinical behavior change is always proximate to outcomes change, 2) starting clinical utility studies early, 3) learning from successes and failures, 4) determining clinical utility with rigorous science, and 5) understanding that clinical utility studies may need to involve private payers and providers from the start. CONCLUSIONS Coverage and reimbursement are shifting from relatively low entry barriers to higher, evidence-based barriers that will require test developers to generate evidence of the net clinical benefits before widespread clinical use will occur. Concerted, early investment in rigorously designed clinical utility studies is necessary.