Center for the Evaluation of Value and Risk in Health, Tufts Medical Center, Boston, MA, USA.
Genentech Inc., South San Francisco, CA, USA.
Appl Health Econ Health Policy. 2024 Nov;22(6):827-832. doi: 10.1007/s40258-024-00910-3. Epub 2024 Sep 3.
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).
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.
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 < 0.05 except for novel mechanism of action (Kolmogorov-Smirnov test) and pediatric indication (both bivariate tests). Multivariable models revealed an independent association between novelty characteristics and QALY gain except for unmet clinical need and indicated for a rare disease.
Drugs with novelty characteristics conferred larger health gains than drugs without these characteristics in bivariate analysis, multivariable models, or both. Future research should examine other aspects of drug novelty, such as patient and health system costs and equitable access.
本研究旨在考察新型药物相对于标准治疗的增量健康收益（以质量调整生命年[QALY]衡量）与药物特征之间的关联。
本研究的分析单位是药物-适应证对。对于 1999 年至 2018 年期间经美国 FDA 批准的药物-适应证对，我们从已发表的文献中量化了增量健康收益，并根据 Health Technology Assessment 机构特别关注的新型药物的六个二元（是/否）特征来描述每一对的新颖性：新作用机制、适应证为罕见病、适应证为儿科人群、治疗严重疾病、提供与现有疗法相比有意义的改善、以及有潜力解决未满足的临床需求。我们分析了双变量关联的度量（Mann-Whitney U 和 Kolmogorov-Smirnov 检验）和多变量回归，同时考虑了多个新颖性特征的影响。
我们的样本包括 146 种药物，占该时期（1999-2018 年）经 FDA 批准的药物的 21%。“新颖”药物-适应证对的中位数和平均 QALY 收益超过了非新颖药物-适应证对的相应 QALY 收益。在大多数比较中，除了新作用机制（Kolmogorov-Smirnov 检验）和儿科适应证（两种双变量检验）外，QALY 收益与新颖性特征之间的双变量关系在 p<0.05 时具有统计学意义。多变量模型显示，新颖性特征与 QALY 收益之间存在独立关联，除了未满足的临床需求和适应证为罕见病之外。
在双变量分析、多变量模型或两者中，具有新颖性特征的药物比没有这些特征的药物具有更大的健康收益。未来的研究应考察药物新颖性的其他方面，如患者和医疗系统成本以及公平获得药物的机会。
