The CHOICE Institute, School of Pharmacy, University of Washington, Seattle, WA, USA.
Department of Health Services Research, University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Value Health. 2021 Dec;24(12):1746-1753. doi: 10.1016/j.jval.2021.07.004. Epub 2021 Sep 1.
Real option value (ROV) is created when a drug enables a patient to live long enough to benefit from a future innovation. Few studies have quantified ROV in the real world. We aimed to estimate the ex post ROV for ipilimumab in metastatic melanoma using real-world data (RWD).
We developed a framework for calculating ROV using RWD, accounting for the health gain in the standard therapy arm and the uptake of future innovations. A Markov model was developed to estimate the quality-adjusted life-years (QALYs) gained with ipilimumab compared with chemotherapy for patients with or without subsequent cancer immunotherapy (CIT). A nationwide electronic health record-derived, deidentified database was used to estimate survival and uptake of CIT.
The incremental QALYs gained for ipilimumab compared with chemotherapy without subsequent CIT were 1.74. With subsequent CIT, the incremental QALYs compared with chemotherapy increased by 0.92, 0.60, 0.33, 0.18, 0.10, and 0.02 when CIT became available 0, 3, 6, 9, 12, and 24 months after the initiation of first-line treatment, respectively. The results were most sensitive to the survival benefit of ipilimumab, the survival benefit of subsequent CIT, and the uptake of CIT.
This is the first study to estimate ex post ROV using RWD. The ex post ROV was between 1% and 54% of conventional value for patients who received a diagnosis within 2 years before CIT availability. Further studies are needed to understand ROV in other disease areas, particularly those with longer survival times.
当一种药物使患者有足够的寿命获益于未来的创新时，就会产生实物期权价值（ROV）。很少有研究在现实世界中量化 ROV。我们旨在使用真实世界数据（RWD）来估计转移性黑色素瘤中伊匹单抗的事后 ROV。
我们开发了一个使用 RWD 计算 ROV 的框架，该框架考虑了标准治疗组的健康收益和未来创新的采用情况。我们开发了一个马尔可夫模型，以估计与化疗相比，伊匹单抗在接受或不接受后续癌症免疫治疗（CIT）的患者中的质量调整生命年（QALY）。使用全国性的电子健康记录衍生的匿名数据库来估计 CIT 的生存和采用情况。
与没有后续 CIT 的化疗相比，伊匹单抗获得的增量 QALY 为 1.74。有后续 CIT 时，如果 CIT 在一线治疗开始后 0、3、6、9、12 和 24 个月可用，与化疗相比，增量 QALY 分别增加了 0.92、0.60、0.33、0.18、0.10 和 0.02。结果对伊匹单抗的生存获益、后续 CIT 的生存获益和 CIT 的采用最为敏感。
这是第一项使用 RWD 估计事后 ROV 的研究。对于在 CIT 可用前 2 年内接受诊断的患者，事后 ROV 在常规价值的 1%至 54%之间。需要进一步研究以了解其他疾病领域的 ROV，特别是那些生存期较长的疾病领域的 ROV。
