Purpose: In 2015, both ASCO and the European Society for Medical Oncology (ESMO) proposed frameworks to quantify the benefit of antineoplastic drugs in the face of rising costs. We applied these frameworks to drugs approved by the US Food and Drug Administration over the past 12 years and examined relationships between costs and benefits.
Methods: We searched FDA.gov for drugs that received initial approval for solid tumors from 2004 to 2015 and calculated the ASCO Net Health Benefit version 2016 (NHB16) and 2015 (NHB15) and the ESMO Magnitude of Clinical Benefit Scale scores for each drug. We calculated descriptive statistics and explored correlations and associations among benefit scores, cost, and independent variables.
Results: We identified 55 drug approvals supported by phase II (18.2%) and III (81.8%) trials, with primary outcomes of overall survival (36.4%), progression-free survival (43.6%), or response rate (20.0%). No significant association was found between NHB16 and year of approval ( P = .81), organ system ( P = .20), or trial comparator arm ( P = .17), but trials with progression-free survival outcomes were associated with higher scores ( P = .007). Both NHB15 and Magnitude of Clinical Benefit Scale scores were approximately normally distributed, but only a moderate correlation existed between them ( r = 0.40, P = .006). No correlation between benefit score and cost (NHB16, r = 0.19; ESMO, r = -0.07) was found. Before 2010, two (15.3%) of 13 approved drugs exceeded $500/NHB point × month compared with 10 (25.0%) of 40 drugs subsequently approved.
Conclusion: Our analysis of the ASCO and ESMO value frameworks illuminates the heterogeneous benefit of new medications and highlights challenges in constructing a unified concept of drug value. Drug benefit does not correlate with cost, and the number of high cost/benefit outliers has increased.