HIDDEN BURDEN OF HEREDITARY TRANSTHYRETIN AMYLOIDOSIS (ATTRV) IN FOUR GULF COUNTRIES: A BURDEN-OF-DISEASE MODELING STUDY
Author(s)
Nada Abaza, BPharm1, Baher Elezbawy, MPH, PhD2, Ahmed Shalaby, MBA3, Abdullah Alsalti, Dr4, Amna Ibrahim Almuaini, MSc, PharmD5, Arif Al Bulushi, Dr6, Dalal Alyouha, MD7, Firas Albadarin, Dr8, Kumayl Al-Lawati, Dr9, Maryam Al-Enezi, Dr10, Mohamed Mahmoud Ibrahim Mahmoud, PhD11, Mohamed Badr, Dr12, Mohamed Abdulrahman AlKhaja, MD13, Omar Sulaiman Abdulaziz, PharmD14, Suha Al Lawati, Dr15, Noha Ali Zaki, BSc16, Rana Raafat Elshazly, B.pharm16, Sherif Attia Abaza, MBA1.
1Syreon Middle East, Cairo, Egypt, 2Health Economist, Syreon Middle East, Alexandria, Egypt, 3Syreon Middle East, Alexandria, Egypt, 4Department of Neurology, Khoula Hospital—Ministry of Health, Muscat, Oman, 5Tawam Hospital, Alain, United Arab Emirates, 6Department of Adult Cardiology, National Heart Center, The Royal Hospital, Ministry of Health, Muscat, Muscat, Oman, 7Ibn Sina Hospital, Ministry of Health, Kuwait City, Kuwait, 8Department of Cardiology, Heart, Vascular and Thoracic Institute, Cleveland Clinic Abu Dhabi, Abu Dhabi, United Arab Emirates, 9Department of Adult Cardiology, National Heart Center, The Royal Hospital, Muscat, Oman, 10Jahra Hospital, Jahra, Kuwait, 11Department of Medicine, Jaber Al-Ahmed Armed Forces Hospital, Kuwait Ministry of Defense., Kuwait, Kuwait, 12GlobeMed Gulf, Dubai, United Arab Emirates, 13King Hamad University Hospital, Al Sayh, Hamad town, Bahrain, 14King Hamad University Hospital, Manama, Bahrain, 15Pharmaceutical Care Department, Ministry of Health, Muscat, Oman, 16AstraZeneca, Dubai, United Arab Emirates.
1Syreon Middle East, Cairo, Egypt, 2Health Economist, Syreon Middle East, Alexandria, Egypt, 3Syreon Middle East, Alexandria, Egypt, 4Department of Neurology, Khoula Hospital—Ministry of Health, Muscat, Oman, 5Tawam Hospital, Alain, United Arab Emirates, 6Department of Adult Cardiology, National Heart Center, The Royal Hospital, Ministry of Health, Muscat, Muscat, Oman, 7Ibn Sina Hospital, Ministry of Health, Kuwait City, Kuwait, 8Department of Cardiology, Heart, Vascular and Thoracic Institute, Cleveland Clinic Abu Dhabi, Abu Dhabi, United Arab Emirates, 9Department of Adult Cardiology, National Heart Center, The Royal Hospital, Muscat, Oman, 10Jahra Hospital, Jahra, Kuwait, 11Department of Medicine, Jaber Al-Ahmed Armed Forces Hospital, Kuwait Ministry of Defense., Kuwait, Kuwait, 12GlobeMed Gulf, Dubai, United Arab Emirates, 13King Hamad University Hospital, Al Sayh, Hamad town, Bahrain, 14King Hamad University Hospital, Manama, Bahrain, 15Pharmaceutical Care Department, Ministry of Health, Muscat, Oman, 16AstraZeneca, Dubai, United Arab Emirates.
OBJECTIVES: Hereditary transthyretin amyloidosis (ATTRv) is a rare, progressive disease associated with substantial morbidity and socioeconomic impact, often underestimated in regions with limited diagnostic awareness. This study aimed to estimate the hidden societal burden of ATTRv in four Gulf countries—the United Arab Emirates (UAE), Oman, Kuwait, and Bahrain—in 2025.
METHODS: A bottom‑up burden‑of‑disease model with a lifetime horizon was developed to quantify the hidden burden of ATTRv. Patients were stratified by clinical phenotype (polyneuropathy [ATTRv‑PN], cardiomyopathy [ATTRv‑CM], and mixed phenotype) and diagnostic status (diagnosed vs undiagnosed/misdiagnosed). Inputs were obtained from published literature and refined through local clinical experts’ interviews. Outcomes included humanistic burden measured in disability‑adjusted life years (DALYs) for patients and caregivers, and indirect costs related to productivity losses. Country‑specific point prevalence estimates were derived from published epidemiological data and adjusted through expert opinion.
RESULTS: Estimated ATTRv patient numbers were 61 in the UAE, 24 in Oman, 26 in Kuwait, and 10 in Bahrain. Mixed phenotype was the most prevalent presentation across all countries. Notably, the model estimated a diagnostic gap of 71-80% of patients being undiagnosed or misdiagnosed. The combined lifetime humanistic burden for patients and caregivers was 1,655 DALYs in the UAE, 273 in Oman, 808 in Kuwait, and 196 in Bahrain. Indirect annual costs were AED8.1M (UAE), OMR0.12M (Oman), KWD0.15M (Kuwait), and BHD0.02M (Bahrain). The annual monetized hidden burden, incorporating humanistic and productivity losses, was estimated at AED17.8M, OMR0.25M, KWD0.37M, and BHD0.12M, respectively, largely driven by mixed phenotype and undiagnosed/misdiagnosed patients.
CONCLUSIONS: ATTRv imposes a substantial hidden societal burden in the Gulf region beyond direct medical costs, driven primarily by underdiagnosis. Addressing this diagnostic gap requires structured early detection strategies, such as multi-modality imaging and timely genetic confirmation. Establishing a prospective Gulf ATTRv registry may strengthen regional real-world evidence and support efficient resource allocation in rare disease management.
METHODS: A bottom‑up burden‑of‑disease model with a lifetime horizon was developed to quantify the hidden burden of ATTRv. Patients were stratified by clinical phenotype (polyneuropathy [ATTRv‑PN], cardiomyopathy [ATTRv‑CM], and mixed phenotype) and diagnostic status (diagnosed vs undiagnosed/misdiagnosed). Inputs were obtained from published literature and refined through local clinical experts’ interviews. Outcomes included humanistic burden measured in disability‑adjusted life years (DALYs) for patients and caregivers, and indirect costs related to productivity losses. Country‑specific point prevalence estimates were derived from published epidemiological data and adjusted through expert opinion.
RESULTS: Estimated ATTRv patient numbers were 61 in the UAE, 24 in Oman, 26 in Kuwait, and 10 in Bahrain. Mixed phenotype was the most prevalent presentation across all countries. Notably, the model estimated a diagnostic gap of 71-80% of patients being undiagnosed or misdiagnosed. The combined lifetime humanistic burden for patients and caregivers was 1,655 DALYs in the UAE, 273 in Oman, 808 in Kuwait, and 196 in Bahrain. Indirect annual costs were AED8.1M (UAE), OMR0.12M (Oman), KWD0.15M (Kuwait), and BHD0.02M (Bahrain). The annual monetized hidden burden, incorporating humanistic and productivity losses, was estimated at AED17.8M, OMR0.25M, KWD0.37M, and BHD0.12M, respectively, largely driven by mixed phenotype and undiagnosed/misdiagnosed patients.
CONCLUSIONS: ATTRv imposes a substantial hidden societal burden in the Gulf region beyond direct medical costs, driven primarily by underdiagnosis. Addressing this diagnostic gap requires structured early detection strategies, such as multi-modality imaging and timely genetic confirmation. Establishing a prospective Gulf ATTRv registry may strengthen regional real-world evidence and support efficient resource allocation in rare disease management.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE156
Topic
Economic Evaluation, Epidemiology & Public Health, Health Policy & Regulatory
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Cardiovascular Disorders (including MI, Stroke, Circulatory), Neurological Disorders