Malik Danyal, Cao Xuan, Sanchez Jaron Castillo, Gao Tianshun, Qian Jiang, Montaner Silvia, Sodhi Akrit
Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, Maryland.
Department of Oncology and Diagnostic Sciences, School of Dentistry, University of Maryland, Baltimore.
JAMA Netw Open. 2021 Feb 1;4(2):e2037880. doi: 10.1001/jamanetworkopen.2020.37880.
Ten percent of the Medicare Part B budget is spent on aflibercept, used to treat a myriad of ocular neovascular diseases. A substantial portion of these costs can be attributed to a few hundred ophthalmologists, raising concerns regarding the influence of pharmaceutical companies on the choice of medication by a relatively small group of clinicians. One approach to protect patients' health care interests is to include them in deliberations on the choice of therapy for their eye disease.
To examine factors associated with patients' choice between an effective and less expensive off-label drug or a more effective, but also more expensive, US Food and Drug Administration (FDA)-approved drug.
DESIGN, SETTING, AND PARTICIPANTS: This retrospective cohort analysis used data from the satellite office of a tertiary referral center from August 2, 2013, to April 9, 2018. Insured patients initiating treatment with anti-vascular endothelial growth factor were included in the analysis. Data were analyzed from March 26, 2018, to June 10, 2020.
Patients were asked to choose between bevacizumab (approximately $100 per dose), a chemotherapy that is effective, but not FDA approved, for the treatment of ocular vascular disease, or aflibercept (approximately $2000 per dose), an FDA-approved drug for ocular vascular disease that may be more effective than bevacizumab in some patients. Independent of this choice, patients were separately asked by a study coordinator to participate in an invasive clinical study for which they would not be compensated, there was a small risk for an adverse event, and they would not personally benefit from participating (a surrogate marker for altruism).
Factors associated with patients' choice of medication, including age, sex, ocular disease, race, and participation in an invasive clinical study.
A total of 189 patients were included in the analysis (106 women [56%]; mean [SEM] age, 74.6 [0.8] years). Despite being told that it may not be as effective as aflibercept, 100 patients (53%) selected bevacizumab for their own eye care. An act of altruism (ie, participation in an invasive clinical study) when the patient was making a choice between the 2 drugs was associated with a patient's choice of bevacizumab (odds ratio [OR], 7.03; 95% CI, 2.27-21.80; P < .001); the OR for selecting bevacizumab for patients who never agreed to participate in the clinical study was 0.45 (95% CI, 0.25-0.83; P = .001). Age (OR, 1.00; 95% CI, 0.97-1.03; P = .86), race (OR, 0.70; 95% CI, 0.41-1.22; P = .21), sex (OR, 0.72; 95% CI, 0.39-1.35; P = .31), presence of diabetes (OR, 1.52; 95% CI, 0.59-3.93; P = .39), and type of eye disease (OR, 0.56; 95% CI, 0.30-1.04; P = .07) were not associated with choice of therapy.
These findings suggest that clinicians must consider the ethical implications of the influence of altruism when patients participate in the decision between cost-effective vs the most effective medicines for their own health care.
医疗保险B部分预算的10%用于阿柏西普，该药用于治疗多种眼部新生血管疾病。这些费用的很大一部分可归因于几百名眼科医生，这引发了人们对制药公司对相对少数临床医生用药选择的影响的担忧。保护患者医疗保健利益的一种方法是让他们参与有关其眼病治疗选择的讨论。
研究与患者在选择一种有效但较便宜的非标签药物或一种更有效但也更昂贵的美国食品药品监督管理局（FDA）批准药物之间的选择相关的因素。
设计、设置和参与者：这项回顾性队列分析使用了一家三级转诊中心卫星办公室2013年8月2日至2018年4月9日的数据。纳入分析的是开始使用抗血管内皮生长因子治疗的参保患者。数据于2018年3月26日至2020年6月10日进行分析。
患者被要求在贝伐单抗（每剂约100美元）和阿柏西普（每剂约2000美元）之间做出选择，贝伐单抗是一种对治疗眼部血管疾病有效但未获FDA批准的化疗药物，阿柏西普是一种获FDA批准用于眼部血管疾病的药物，在某些患者中可能比贝伐单抗更有效。独立于这一选择之外，研究协调员分别询问患者是否愿意参加一项他们不会得到补偿、存在小的不良事件风险且他们个人不会从参与中受益的侵入性临床研究（利他主义的一个替代指标）。
与患者用药选择相关的因素，包括年龄、性别、眼病、种族以及参与侵入性临床研究情况。
共有189名患者纳入分析（106名女性[56%]；平均[标准误]年龄为74.6[0.8]岁）。尽管被告知贝伐单抗可能不如阿柏西普有效，但仍有100名患者（53%）选择贝伐单抗用于自己的眼部治疗。当患者在两种药物之间做出选择时，利他行为（即参与侵入性临床研究）与患者选择贝伐单抗相关（优势比[OR]为7.03；95%置信区间为2.27 - 21.80；P <.001）；从未同意参与临床研究的患者选择贝伐单抗的OR为0.45（95%置信区间为0.25 - 0.83；P =.001）。年龄（OR为1.00；95%置信区间为0.97 - 1.03；P =.86）、种族（OR为0.70；95%置信区间为0.41 - 1.22；P =.21）、性别（OR为0.72；95%置信区间为0.39 - 1.35；P =.31）、是否患有糖尿病（OR为1.52；95%置信区间为0.59 - 3.93；P =.39）以及眼病类型（OR为0.56；95%置信区间为0.30 - 1.04；P =.07）均与治疗选择无关。
这些发现表明，当患者参与关于自身医疗保健的性价比高的药物与最有效的药物之间的决策时，临床医生必须考虑利他主义影响所带来的伦理问题。
