Humanistic and Economic Burden of Spinal Muscular Atrophy in the Gulf Region.

BACKGROUND: Spinal muscular atrophy (SMA) is a severe, fatal genetic disorder. This study aimed to quantify the humanistic and economic burden of SMA in four Gulf countries, the Kingdom of Saudi Arabia (KSA), the United Arab Emirates (UAE), Oman, and Bahrain, to provide decision-makers with evidence-based strategies for burden mitigation. METHODS: A burden of disease model was developed to quantify SMA's humanistic burden via disability-adjusted life years (DALYs) and its economic burden through direct healthcare costs and indirect productivity losses experienced by patients and caregivers. RESULTS: Type 1 SMA caused the highest burden across all countries, followed by types 2 and 3. Total DALYs were 274,008 in KSA, 35,877 in UAE, 33,459 in Oman, and 8,679 in Bahrain. National direct medical costs totaled 114.9 million USD, 25.0 million USD, 9.5 million USD, and 3.8 million USD in KSA, UAE, Oman, and Bahrain, respectively. Annual indirect costs, driven by productivity losses among patients and caregivers, were estimated at 28.1 million USD in KSA, 5.3 million USD in UAE, 3.9 million USD in Oman, and 0.99 million USD in Bahrain. CONCLUSIONS: The burden of SMA is considerable, particularly in the more severe phenotypes, with a profound humanistic impact and high medical costs. While results vary by population size, the consistent patterns across countries underscore the disease's severity and the urgent need for effective mitigation strategies.