Background Targeted cancer drugs (TCDs) have revolutionized oncology but vary in clinical benefit and patient out-out-pocket (OOP) costs. The ASCO Value Framework uses survival, toxicity, and symptom palliation data to quantify the net health benefit (NHB) of cancer drugs. We evaluated associations between NHB, uptake, and spending on oral TCDs. Methods We conducted a retrospective cohort study of patients aged 18-64 years with an incident oral TCD pharmacy claim in 2012-2020 in a nationwide de-identified commercial claims dataset. TCDs were categorized as having high (>60), medium (40-60), and low (<40) NHB scores. We plotted the uptake of TCDs by NHB category and used standard descriptive statistics to evaluate patient OOP and total spending. Generalized linear models evaluated the relationship between spending and TCD NHB, adjusted for cancer indication. Results We included 8,524 patients with incident claims for eight oral TCDs with nine first-line indications in advanced melanoma, breast, lung, and pancreatic cancer. Medium- and high-NHB TCDs accounted for most TCD prescriptions. Median OOP spending was $18.78 for the first 28-day TCD supply (IQR $0.00-$87.57); 45% of patients paid $0 OOP. Median total spending was $10,118.79 (IQR $6,365.95-$10,600.37) for an incident 28-day TCD supply. Total spending increased $1,083.56 for each 10-point increase in NHB score (95% CI $1,050.27-$1,116.84, p < .01 for H0=$0). Conclusion Low-NHB TCDs were prescribed less frequently than medium- and high-NHB TCDs. Total spending on oral TCDs was high and positively associated with NHB. Commercially insured patients were largely shielded from high OOP spending on oral TCDs.

中文翻译：

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口服靶向癌症药物净健康效益、摄取和支出之间的关联

背景 靶向抗癌药物 (TCD) 彻底改变了肿瘤学，但临床效益和患者自付费用 (OOP) 各不相同。 ASCO 价值框架使用生存、毒性和症状缓解数据来量化癌症药物的净健康效益 (NHB)。我们评估了 NHB、口服 TCD 的吸收和支出之间的关联。方法 我们在全国范围内去识别的商业索赔数据集中，对 2012 年至 2020 年期间发生过口服 TCD 药品索赔的 18-64 岁患者进行了回顾性队列研究。 TCD被分类为具有高(＞60)、中(40-60)和低(＜40)NHB分数。我们按 NHB 类别绘制了 TCD 的使用情况，并使用标准描述性统计数据来评估患者 OOP 和总支出。广义线性模型评估了支出与 TCD NHB 之间的关系，并根据癌症适应症进行了调整。结果 我们纳入了 8,524 名患者，他们对 8 种口服 TCD 进行了事件索赔，涉及晚期黑色素瘤、乳腺癌、肺癌和胰腺癌的 9 种一线适应症。中、高 NHB TCD 占 TCD 处方的大部分。第一个 28 天 TCD 供应的 OOP 支出中位数为 18.78 美元（IQR 0.00 美元-87.57 美元）； 45% 的患者支付 0 美元 OOP。事件 28 天 TCD 供应的总支出中位数为 10,118.79 美元（IQR 6,365.95-10,600.37 美元）。 NHB 评分每增加 10 分，总支出就会增加 1,083.56 美元（95% CI 1,050.27-1,116.84 美元，H0=0 美元时 p < .01）。结论 低 NHB TCD 的处方频率低于中和高 NHB TCD。口服 TCD 的总支出很高，并且与 NHB 呈正相关。商业保险患者在很大程度上免受口服 TCD 上高额 OOP 支出的影响。