Abstract
Multi-Gene Panel Sequencing (MGPS), compared to Single-Marker Genetic Testing (SMGT), can identify more cancer patients who could benefit from targeted therapies, but impact on outcomes and total costs of care is uncertain. We estimated the cost-effectiveness of MGPS vs SMGT among patients with advanced melanoma (aMEL). Treatment patterns and MGPS/SMGT use for aMEL patients (stage IIIB or metastatic) diagnosed between 2011-2016 were identified from the Flatiron Health electronic health record-derived database. For MGPS and SMGT cohorts, we analyzed: percentage of patients receiving targeted treatments; survival; total healthcare costs. SMGT included BRAF testing; MGPS also included NRAS and KIT mutations. Cost data sources were the CMS Fee Schedule and 2017 Average Sales Price (ASP) drug cost. Using decision modeling, we estimated incremental cost-effectiveness (ICER) from a US payer perspective over a lifetime horizon. We identified 6622 aMEL patients receiving MGPS (n=596) or SMGT (n=6026): 37% of patients tested positive for BRAF with MGPS vs 27% with SMGT. Among MGPS-tested patients, 0.2% tested positive for NRAS or KIT. Of MGPS-tested patients, 19% received treatments targeted to these mutations vs 14% with SMGT. Expected survival was 1.13 life years (LYs) for SMGT vs 1.28 LYs for MGPS. Lifetime total costs were $18,775 higher per patient for MGPS, giving an ICER of $127,503/LY gained. Results were sensitive to the probability of receiving a targeted treatment, survival estimates and treatment costs. At a willingness-to-pay-threshold of $150,000/LY saved, MGPT has a ∼55% probability to be cost-effective. Reducing all current decision uncertainty to eliminate the probability of recommending MGPS when in truth SMGT would be the cost-effective option, has a monetary value of ∼$1Billion. Based on real-world data from a nationwide oncology patient database, MGPS may be moderately cost-effective compared to SMGT in aMEL patients. Further (cost-)effectiveness research is likely worthwhile to reduce current decision uncertainty.
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