SYSTEMATIC VERSUS ETIOLOGY-RESTRICTED FABRY DISEASE SCREENING IN MOROCCO'S DIALYSIS POPULATION: A DECISION-TREE COST-UTILITY ANALYSIS BENCHMARKED ON A DIALYSIS-REVEALED THRESHOLD
Author(s)
Omar Maoujoud, PhD, MD1, Amal Yassine, MD, PhD1, Intissar Haddiya, MD, PhD2.
1ISPOR Morocco, Research Team of pharmacoeconomics & pharmacoepidemiology, Faculty of Medicine Mohammed V University, Rabat, Morocco, 2Department of Nephrology, Faculty of Medicine, Mohammed I University, Oujda, Morocco, Oujda, Morocco.
1ISPOR Morocco, Research Team of pharmacoeconomics & pharmacoepidemiology, Faculty of Medicine Mohammed V University, Rabat, Morocco, 2Department of Nephrology, Faculty of Medicine, Mohammed I University, Oujda, Morocco, Oujda, Morocco.
OBJECTIVES: Fabry disease is an X-linked lysosomal disorder, over-represented in unexplained dialysis cohorts and causing progressive kidney failure. We asked whether screening Morocco's prevalent dialysis population for it is cost-effective from the basic health insurance (AMO) payer perspective. Diagnosed cases receive enzyme replacement therapy, and their relatives are tested in cascade. We compared two screening strategies against a locally revealed threshold.
METHODS: We built a decision-tree cost-utility analysis in Moroccan dirham, lifetime horizon, three percent discounting. Two strategies were compared: screening all dialysis patients with a dried blood spot enzyme test, or only those whose cause of kidney failure is undetermined. Positive tests were confirmed by genotyping, then relatives in cascade. Value comes from relatives treated before end-stage disease, not the dialysis index case. The incremental cost-effectiveness ratio (ICER) was compared with three thresholds: opportunity cost, one times gross domestic product per capita, and one revealed by national dialysis spending. Parameters were varied in one-way and probabilistic analyses (ten thousand iterations, fixed seed).
RESULTS: The all-comers strategy screened 45,000 patients, found about 135 index cases and 220 treatable relatives, at 130,600 dirham per relative found. The restricted strategy screened the 9,000 with undetermined cause, found about 108 index cases and 176 treatable relatives, and cut the cost to 39,100 dirham per relative. The ICER was the same under both strategies, about 29 million dirham per quality-adjusted life-year, 152 times the dialysis-revealed threshold, and was never cost-effective probabilistically. Enzyme replacement therapy would need a 97 percent price cut to become cost-effective.
CONCLUSIONS: Restricting screening to patients with undetermined cause roughly triples detection efficiency, but the ICER does not change: the price of enzyme replacement therapy, not the breadth of screening, drives affordability. Against Morocco's own dialysis-revealed threshold, screening is efficient while treatment stays unaffordable without a managed-entry price. The method transfers to other high-cost therapies.
METHODS: We built a decision-tree cost-utility analysis in Moroccan dirham, lifetime horizon, three percent discounting. Two strategies were compared: screening all dialysis patients with a dried blood spot enzyme test, or only those whose cause of kidney failure is undetermined. Positive tests were confirmed by genotyping, then relatives in cascade. Value comes from relatives treated before end-stage disease, not the dialysis index case. The incremental cost-effectiveness ratio (ICER) was compared with three thresholds: opportunity cost, one times gross domestic product per capita, and one revealed by national dialysis spending. Parameters were varied in one-way and probabilistic analyses (ten thousand iterations, fixed seed).
RESULTS: The all-comers strategy screened 45,000 patients, found about 135 index cases and 220 treatable relatives, at 130,600 dirham per relative found. The restricted strategy screened the 9,000 with undetermined cause, found about 108 index cases and 176 treatable relatives, and cut the cost to 39,100 dirham per relative. The ICER was the same under both strategies, about 29 million dirham per quality-adjusted life-year, 152 times the dialysis-revealed threshold, and was never cost-effective probabilistically. Enzyme replacement therapy would need a 97 percent price cut to become cost-effective.
CONCLUSIONS: Restricting screening to patients with undetermined cause roughly triples detection efficiency, but the ICER does not change: the price of enzyme replacement therapy, not the breadth of screening, drives affordability. Against Morocco's own dialysis-revealed threshold, screening is efficient while treatment stays unaffordable without a managed-entry price. The method transfers to other high-cost therapies.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE567
Topic
Economic Evaluation, Epidemiology & Public Health, Methodological & Statistical Research
Topic Subcategory
Thresholds & Opportunity Cost
Disease
Pediatrics, Rare & Orphan Diseases, Urinary/Kidney Disorders