Phase 3 oncology clinical trials have had success rates of 41% (Djulbegovic, Arch Int Med 2008). Against this backdrop, context is needed on the magnitude of benefit to expect from future successful trials, and the interplay of cost. Recognizing the limitations of comparing results across trials and therapies, we assessed recent FDA solid tumor approvals showing overall survival (OS) benefit in a randomized trial. The objective of this analysis is to define the magnitude of benefit and cost of solid tumor therapies approved by FDA from 2009-2013 having phase 3 OS benefit. A systematic review of CenterWatch FDA-Approved Drugs and FDA sNDA/sBLA databases was conducted. Inclusion criteria required FDA approval or efficacy supplement, with OS data published 2009-2013. Hazard ratio (HR) and absolute and relative gain in median OS were assessed. First month drug costs were determined for 4Q2013, using methods previously described by Bach (NEJM 2009). Agents intended to be administered for £ 6 weeks were excluded from the cost analysis. Limitations include omission of many agents that represent important advances yet did not demonstrate statistically significant OS benefit relative to a control arm. 18 FDA approved agents across 25 indications for 9 tumor types were assessed. The mean HR (range) and the mean relative gain in median OS vs trial comparator was 0.702 (0.410-0.817) and 27.91% (11.9%-67.2%), respectively. Medians for each measure were 0.725 and 23.6%. Mean first month treatment costs were $12,601 ($5,881-$50,025) with median costs of $9,282. Over the last 5 years, for FDA-approved agents for treatment of solid tumors, the magnitude of survival benefit, measured either as Hazard Ratio or relative gain in median OS, was 24-30%. This benefit, and first month costs of $9 - 13,000, may represent midpoints of expected levels for future solid tumor agents.
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