Sarepta Therapeutics, Inc, Cambridge, MA, USA.
College of Pharmacy, University of Utah, Salt Lake City, UT, USA.
J Med Econ. 2023 Jan-Dec;26(1):594-602. doi: 10.1080/13696998.2023.2200102.
Assessing the value of single or short-term therapies (SSTs) within traditional cost-effectiveness analyses (CEAs) has been a topic of discussion as the number of SSTs increases, particularly regarding the effect of discounting on valuation. To quantify the impact of discounting in economic evaluations, a CEA of a hypothetical SST and equivalent chronic therapy was conducted using standard methods.
A lifetime Markov model was developed for a hypothetical chronic, progressive disease that could be treated with an SST, chronic therapy, or no novel treatment, termed standard of care (SoC). Incremental cost-effectiveness ratios (ICERs) with quality-adjusted life years (QALYs) comparing SST vs. SoC and an equivalent chronic therapy vs. SoC were assessed from a payer perspective. Both treatments had equal benefits and undiscounted lifetime costs; 3% discounting was applied to costs/benefits in the base case, and the impact of discounting was assessed.
In the base case example, both the SST and equivalent chronic therapy vs. SoC had ICERs of $86,000/QALY without discounting. With 3% discounting, the ICER for the SST increased by 116% ($186,000/QALY) while the ICER for the chronic therapy increased by 10% ($95,000/QALY) despite equal clinical benefit. In scenario analyses, the ICER of the SST was consistently higher than the equivalent chronic therapy across a range of assumptions/inputs. Varying the cost/benefit discount rates had a greater impact on the SST. Differences in the ICERs between the therapies increased with increasing life expectancy/time horizon.
The simple model structure may not be reflective of acute or more complex diseases. Also, the scenario of perfect equivalency in efficacy and lifetime costs is hypothetical.
This quantitative assessment showed the extent to which SST CEAs are highly sensitive to discounting, resulting in worse value assessments for SSTs than equivalent chronic therapies.
随着短期疗法(SSTs)数量的增加,评估传统成本效益分析(CEAs)中单次或短期疗法的价值一直是讨论的话题,特别是关于贴现对估值的影响。为了量化经济评估中贴现的影响,使用标准方法对假设的 SST 和等效慢性治疗进行了 CEA。
为一种假设的慢性进行性疾病开发了终生马尔可夫模型,该疾病可以用 SST、慢性治疗或无新型治疗(称为标准治疗)进行治疗。从支付者的角度评估了 SST 与标准治疗和等效慢性治疗与标准治疗相比的增量成本效益比(ICER)与质量调整生命年(QALYs)。两种治疗方法的疗效和无折扣终身成本均相同;在基础情况下,对成本/效益应用 3%的折扣,评估了折扣的影响。
在基础案例中,SST 和等效慢性治疗与标准治疗相比,ICER 均为 86000 美元/QALY,无折扣。贴现 3%时,SST 的 ICER 增加了 116%(186000 美元/QALY),而慢性治疗的 ICER 增加了 10%(95000 美元/QALY),尽管临床疗效相同。在情景分析中,SST 的 ICER 在一系列假设/输入中始终高于等效慢性治疗。成本/效益贴现率的变化对 SST 的影响更大。两种疗法的 ICER 之间的差异随着预期寿命/时间范围的增加而增加。
简单的模型结构可能无法反映急性或更复杂的疾病。此外,疗效和终身成本完全等效的情况是假设的。
这项定量评估表明,SST 的 CEA 对贴现非常敏感,导致 SST 的价值评估比等效慢性治疗差。