Are Drug Novelty Characteristics Associated With Greater Health Benefits?

Objective The aim of this study was to examine the association between characteristics of novel drugs and incremental health gains relative to standard of care, in terms of quality-adjusted life-years (QALYs). Methods This study’s unit of analysis is the drug–indication pair. For pairs approved by the US FDA from 1999 to 2018, we quantified incremental health gains using QALYs from the published literature and characterized each pair’s novelty in terms of a series of six binary (yes/no) characteristics of novel drugs given special consideration by Health Technology Assessment agencies: Novel mechanism of action, Indicated for a rare disease, Indicated for a pediatric population, Treats a serious condition, Offers meaningful improvement over available therapies, and Potential to address unmet clinical needs. We analyzed measures of bivariate association (Mann-Whitney U and Kolmogorov-Smirnov tests) and multivariable regression, accounting for the influence of multiple novelty characteristics simultaneously. Results Our sample of 146 drugs represents 21% of drugs approved the FDA in the time period (1999–2018). Median and mean QALY gains for ‘novel’ drug–indication pairs exceeded corresponding QALY gains for non-novel drug–indication pairs. For most comparisons, the bivariate relationships between QALY gains and novelty characteristics were significant at p