Purpose

To investigate the challenges and potential improvement strategies of cost-effectiveness analyses performed for therapeutics targeting inherited retinal diseases (IRDs).

Design

Perspective.

Methods

A literature review was conducted with discussion of current limitations and improvement recommendations.

Results

Cost-effectiveness analysis (CEA) performed for IRD therapeutics has multiple limitations. First, the available methods used to measure health-related quality of life and health utilities can be inaccurate when used in IRDs. Second, the financial burden to patients and society from vision impairment associated with IRDs has been inadequately studied and includes a variety of expenditures ranging from direct costs of IRD specialty health care to indirect expenses associated with daily living activities. Third, our collective understanding is limited in the areas of IRD natural history and health benefits gained from new IRD treatments (eg, gene therapies). In addition, the therapeutic effect from a patient perspective and its duration of action are not fully understood. Due to the scarcity of data, CEA for newly approved therapies has relied on assumptions and creations of predictive models for both costs and health benefits for these new therapeutics in order to calculate the incremental cost-effectiveness ratio.

Conclusions

CEA studies performed for IRD therapeutics have been limited by the established health utilities in ophthalmology and the lack of disease-specific information. The assumptions and extrapolations in these studies create substantial uncertainty in incremental cost-effectiveness ratio results. An improved framework is required for CEA of IRD therapeutics in order to determine the cost-effectiveness of each therapy brought from clinical trials to clinical practice.

中文翻译：

---

遗传性视网膜疾病新型疗法成本效益分析的挑战

目的

调查针对遗传性视网膜疾病 (IRD) 的疗法进行的成本效益分析的挑战和潜在改进策略。

设计

看法。

方法

进行了文献综述，讨论了当前的局限性和改进建议。

结果

对 IRD 疗法进行的成本效益分析 (CEA) 具有多重局限性。首先，用于衡量与健康相关的生活质量和健康效用的可用方法在 IRD 中使用时可能不准确。其次，与 IRD 相关的视力障碍给患者和社会带来的经济负担研究不足，包括各种支出，从 IRD 专业医疗保健的直接费用到与日常生活活动相关的间接费用。第三，我们对 IRD 自然史和从新的 IRD 治疗（例如基因疗法）获得的健康益处领域的集体理解是有限的。此外，从患者角度来看的治疗效果及其作用持续时间尚不完全清楚。由于资料稀缺，

结论

为 IRD 疗法进行的 CEA 研究受到眼科既定健康实用程序和缺乏特定疾病信息的限制。这些研究中的假设和推断给增量成本效益比结果带来了很大的不确定性。IRD 疗法的 CEA 需要一个改进的框架，以确定从临床试验到临床实践的每种疗法的成本效益。