Near-Equivalence: Generating Evidence to Support Alternative Cost-Effective Treatments
Ian F. Tannock, Mark J. Ratain, Daniel A. Goldstein, Allen S. Lichter, Gary L. Rosner, Leonard B. Saltz
Abstract
The cost of cancer drugs is a challenge to patients and healthcare systems. Here, we propose a new paradigm of near-equivalence, which combines various types of evidence to support acceptability of alternative treatments (ALTs) relative to standard-of-care (SOC). Nearequivalence facilitates cost-effective therapies but differs from cost-effectiveness analysis in emphasizing near-equivalent outcomes over cost and includes analysis of pharmacokinetic and pharmacodynamic data to support near-equivalent outcomes. Demonstration of near-equivalence does not require large noninferiority randomized controlled trials (RCTs). Rather, it is supported by the new discipline of interventional pharmacoeconomics. The utilitarian approach of near-equivalence aims to provide the greatest good for the greatest number of patients under real-world constraints; it can increase access to treatment and reduce financial toxicity, with minimal impact on efficacy and often with decreased toxicity. Various approaches to demonstrate near-equivalence, with examples, are described below and in the Table