科威特预防孤儿病的投资回报

S. Abuhadida, L. Bastaki, Buthina Bash, Barrak Alhindal
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引用次数: 1

摘要

1型脊髓性肌萎缩症和RPE65突变相关的遗传性视网膜营养不良是两种众所周知的孤儿病,需要昂贵的孤儿药。作为遗传性疾病,它们可以通过一个项目来预防,包括婚前遗传筛查,以检测有缺陷的基因携带者,然后是植入前遗传学诊断,以识别健康的配子和体外受精。我们开发了一个随机财务模型,从科威特卫生部的财务角度评估实施一项预防方案的五年投资回报,以解决这些问题。该预防项目的投资回报率被证明是非常节省成本的,概率平均为9,710,311美元(2,930,727 KWD)。在预防上每花费1美元或金元,可节省1.5美元或金元。与此同时,不实施预防计划可能会使卫生部在同一时期平均损失71,431,037美元(21,555,325千瓦时)。本研究结果有力地支持卫生部从财政角度采用和实施预防方案。
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Return on Investment from the Prevention of Orphan Diseases in Kuwait
Spinal Muscular Atrophy type 1 and RPE65 mutation-associated Inherited Retinal dystrophy are two well-known Orphan diseases for expensive Orphan drugs. Being inheritable disorders, they can be prevented through a program that includes Premarital Genetic Screening to detect the defective gene carriers followed by Preimplantation Genetic Diagnosis to identify healthy gametes and In Vitro Fertilization. We developed a stochastic financial model to assess the Return on Investment over five years of implementing a prevention program to tackle these conditions from the financial perspective of the Ministry of Health in Kuwait. The ROI from the prevention program was shown to be highly cost-saving, with a probabilistic average of 9,710,311 USD (2,930,727 KWD). Every 1 USD or KWD spent on prevention would return 1.5 USD or KWD in savings. Meanwhile, not implementing the prevention program could cost the MOH a probabilistic average of 71,431,037 USD (21,555,325 KWD) within the same period. The findings of this study strongly support the adoption and implementation of the prevention program from the financial perspective of the MOH.
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