Abstract
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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