Migliaccio-Walle Kristen, Caro J Jaime, Möller Jörgen
Caro Research Institute, Concord, MA 01742, USA.
Curr Med Res Opin. 2006 Oct;22(10):2055-63. doi: 10.1185/030079906X132631.
Short bowel syndrome is a rare, life-threatening condition that can result in nutritional malabsorption. Parenteral nutrition provides life-saving support but can lead to complications and affect quality of life. Recombinant human growth hormone, somatropin (rDNA origin), has been shown to significantly reduce dependence on nutritional support (p < 0.05). This study evaluates the economic impact of somatropin use in the management of short bowel syndrome.
A discrete event simulation (DES) model was developed to estimate the benefits and costs associated with somatropin use. Risks of treatment complications and of disease-related events were modeled in identical patient pairs--one receiving parenteral nutrition alone, the other receiving 4 weeks of somatropin--for 2 years following initiation of treatment. Life expectancy was assumed equivalent. Risk functions were estimated from the literature and one randomized clinical trial. Total and component costs associated with each strategy were determined. The distribution of patients reducing parenteral nutrition need and the final parenteral nutrition frequency were also estimated. Sensitivity analyses were completed for key inputs. Direct medical costs are reported in US 2004 dollars.
The model predicted that 96.0% of patients receiving somatropin reduce or eliminate parenteral nutrition within 6 weeks: average use was reduced by 2.8 days and one-third weaned completely. Based on 1.9 L of parenteral nutrition per day, estimated costs were 118,098 dollars in year one and 132,935 dollars in year two. With somatropin, costs dropped to 84,309 dollars in year one--despite the 17,459 dollars cost of somatropin treatment--and 81,250 dollars in year two. Over 2 years savings totaled 85,474 dollars.
Insufficient data required that assumptions be made for some inputs. DES is new in pharmacoeconomics and may be perceived as a limitation.
Somatropin use improves quality of life by reducing the need for parenteral nutrition and results in health care cost savings.
短肠综合征是一种罕见的、危及生命的疾病，可导致营养吸收不良。肠外营养提供了挽救生命的支持，但可能会引发并发症并影响生活质量。重组人生长激素，即生长激素（重组DNA来源），已被证明能显著降低对营养支持的依赖（p < 0.05）。本研究评估生长激素用于短肠综合征管理的经济影响。
建立了一个离散事件模拟（DES）模型，以估计与使用生长激素相关的益处和成本。在治疗开始后的2年里，对相同的患者对进行治疗并发症风险和疾病相关事件风险的建模——一组仅接受肠外营养，另一组接受4周的生长激素治疗。假设预期寿命相同。风险函数根据文献和一项随机临床试验进行估计。确定了与每种策略相关的总成本和分项成本。还估计了减少肠外营养需求的患者分布情况以及最终的肠外营养频率。对关键输入进行了敏感性分析。直接医疗成本以2004年美元报告。
该模型预测，接受生长激素治疗的患者中有96.0%在6周内减少或停止了肠外营养：平均使用天数减少了2.8天，三分之一的患者完全停用。基于每天使用1.9升肠外营养，估计第一年的成本为118,098美元；第二年为132,935美元。使用生长激素后，第一年的成本降至84,309美元——尽管生长激素治疗成本为17,459美元，第二年为81,250美元°两年的总节省为85,474美元。
数据不足要求对一些输入进行假设。DES在药物经济学中是新方法，可能被视为一种局限性。
使用生长激素可减少对肠外营养的需求，从而改善生活质量，并节省医疗保健成本。
