Precision Health Economics, 11100 Santa Monica Blvd, Ste 500, Los Angeles, CA 90025, USA.
Am J Manag Care. 2012 Nov;18(11 Suppl):S265-71.
To quantify in the context of chronic myeloid leukemia (CML) the additional value patients receive when innovative treatments enable them to survive until the advent of even more effective future treatments (ie, the "option value").
Observational study using data from the Surveillance, Epidemiology and End Results (SEER) cancer registry comprising all US patients with CML diagnosed between 2000 and 2008 (N = 9,760).
We quantified the option value of recent breakthroughs in CML treatment by first conducting retrospective survival analyses on SEER data to assess the effectiveness of TKI treatments, and then forecasting survival from CML and other causes to measure expected future medical progress. We then developed an analytical framework to calculate option value of innovative CML therapies, and used an economic model to value these gains. We calculated the option value created both by future innovations in CML treatment and by medical progress in reducing background mortality.
For a recently diagnosed CML patient, the option value of innovative therapies from future medical innovation amounts to 0.76 life-years. This option value is worth $63,000, equivalent to 9% of the average survival gains from existing treatments. Future innovations in CML treatment jointly account for 96% of this benefit.
The option value of innovative treatments has significance in the context of CML and, more broadly, in disease areas with rapid innovation. Incorporating option value into traditional valuations of medical innovations is both a feasible and a necessary practice in health technology assessment.
在慢性髓性白血病（CML）的背景下量化创新治疗使患者能够存活到更有效未来治疗方法出现时所获得的附加价值（即“期权价值”）。
利用包含所有 2000 年至 2008 年间被诊断为 CML 的美国患者的监测、流行病学和最终结果（SEER）癌症登记处的数据进行观察性研究（N=9760）。
我们首先通过对 SEER 数据进行回顾性生存分析来评估 TKI 治疗的效果，从而量化 CML 治疗最近突破的期权价值，然后预测 CML 和其他原因导致的生存情况，以衡量预期的未来医学进展。然后，我们开发了一个分析框架来计算创新 CML 疗法的期权价值，并使用经济模型来评估这些收益。我们计算了由 CML 治疗的未来创新和降低背景死亡率的医学进展所带来的期权价值。
对于最近诊断的 CML 患者，未来医学创新的创新疗法的期权价值为 0.76 个生命年。这个期权价值价值 63000 美元，相当于现有治疗方法带来的平均生存收益的 9%。CML 治疗的未来创新共同构成了这一收益的 96%。
在 CML 背景下，以及在具有快速创新的疾病领域中，创新治疗的期权价值具有重要意义。将期权价值纳入医疗创新的传统评估中，在卫生技术评估中既可行又必要。
