Centre for Research on Health and Social Care Management, Bocconi University, Milano, Lombardia, Italy.
Health Econ. 2022 Sep;31 Suppl 1(Suppl 1):25-43. doi: 10.1002/hec.4562. Epub 2022 Jun 28.
Real-world data are considered a potentially valuable source of evidence for assessing medical technologies in clinical practice, but their widespread use is hampered by numerous challenges. Using the case of coronary stents in Italy, we investigate the potential of administrative databases for estimating costs and health outcomes associated with the use of medical devices in real world conditions. An administrative dataset was created ad hoc by merging hospital records from patients admitted between 2013 and 2019 for stent implantations with ambulatory records, pharmaceutical use data and vital statistics. Health outcomes were multifold: all-cause and cardiac mortality and myocardial infarction, within 30 days, 1, 2, 5 years. Costs were estimated from the National Health System perspective. We used multivariable Cox models and propensity score (PS) methods (PS matching; stratification on PS; inverse probability of treatment weighting using PS; PS adjustment). 257,907 coronary stents were implanted in 113,912 patients. For all health outcomes and follow-up times, and across all methods, patients receiving drug-eluting stents (DES) presented lower risk. For all-cause mortality, the DES patient advantage over bare-metal stent (BMS) patients declined over time but remained significant even at 5 years. For myocardial infarction, results remained quite stable. The DES group presented lower cumulative total costs (ranging from 3264 to 2363 Euros less depending on methods). Our results confirm the consolidated evidence of the benefits of DES compared to BMS. The consistency of results across methods suggests internal validity of the study, while highlighting strengths and limitations of each depending on research context. Administrative data yield great potential to perform comparative effectiveness and cost-effectiveness analysis of medical devices provided certain conditions are met.
真实世界数据被认为是评估临床实践中医疗技术的潜在有价值的证据来源，但由于存在许多挑战，其广泛应用受到阻碍。本文以意大利的冠状动脉支架为例，研究了在真实条件下使用医疗设备的成本和健康结果的估计，探讨了利用行政数据库的潜力。通过合并 2013 年至 2019 年间因支架植入而住院的患者的医院记录与门诊记录、药物使用数据和生命统计数据，创建了一个专门的行政数据集。健康结果是多方面的：30 天内、1 年、2 年、5 年内的全因和心脏死亡率以及心肌梗死。从国家卫生系统的角度估计了成本。我们使用多变量 Cox 模型和倾向评分（PS）方法（PS 匹配；PS 分层；PS 逆概率处理加权；PS 调整）。在 113912 名患者中植入了 257907 个冠状动脉支架。对于所有健康结果和随访时间，以及所有方法，接受药物洗脱支架（DES）的患者风险较低。对于全因死亡率，DES 患者相对于裸金属支架（BMS）患者的优势随时间推移而下降，但即使在 5 年内仍然显著。对于心肌梗死，结果仍然相当稳定。DES 组的累积总成本较低（根据方法的不同，从 3264 欧元到 2363 欧元不等）。我们的结果证实了 DES 相对于 BMS 的获益的一致性证据。方法之间结果的一致性表明研究的内部有效性，同时根据研究背景突出了每种方法的优缺点。行政数据具有很大的潜力，可以在满足某些条件的情况下对医疗设备进行比较有效性和成本效益分析。
