CONSANGUINITY-ADJUSTED BUDGET IMPACT OF ORPHAN DRUG ACCESS FOR HEREDITARY TYROSINEMIA TYPE 1 IN MOROCCO: A PAYER-PERSPECTIVE MODEL
Author(s)
Omar Maoujoud, PhD, MD1, Amal Yassine, MD, PhD2.
1ISPOR Morocco, Research Team of pharmacoeconomics & pharmacoepidemiology, Faculty of Medicine Mohammed V University, Rabat, Morocco, 2Moroccan Society for Health Products Economy & ISPOR Morocco, Mohammedia, Morocco.
1ISPOR Morocco, Research Team of pharmacoeconomics & pharmacoepidemiology, Faculty of Medicine Mohammed V University, Rabat, Morocco, 2Moroccan Society for Health Products Economy & ISPOR Morocco, Mohammedia, Morocco.
OBJECTIVES: Morocco has among the region's highest consanguinity levels (mean inbreeding coefficient 0.0065), raising autosomal recessive (AR) disease prevalence, yet most orphan therapies sit outside the basic health insurance (AMO) basket. We quantified the consanguinity-adjusted incidence of hereditary tyrosinemia type 1 (HT1) and the budgetary space AMO coverage of nitisinone would require, payer perspective, over five years.
METHODS: A prevalence-based budget impact model (AMO perspective, five years, Moroccan Dirham [MAD]) adjusted AR incidence for consanguinity (mean coefficient 0.0065). HT1 was anchored on a measured Maghreb incidence, with Tunisia as proxy, versus the global rate. The treated population followed a procurement-anchored ramp under a counterfactual coverage scenario. Nitisinone used the Moroccan public acquisition price from a 2023 hospital tender, applied by weight at 1 mg/kg/day. Probabilistic sensitivity analysis (PSA; 10,000 iterations, fixed seed) propagated uptake, weight, and dose uncertainty.
RESULTS: Measured Maghreb HT1 incidence was 1 in 14,804 (6.8 per 100,000), about sevenfold the global 1 in 100,000; the consanguinity adjustment alone predicted 1 in 32,750. Against roughly 43 expected annual cases, the treated population rose from 15 to 70 over five years, exposing a wide access gap. Annual nitisinone cost was approximately MAD 682,000 per patient. Five-year budget impact was MAD 145.2 million (base case, about MAD 29 million yearly); PSA mean was MAD 192.8 million (95% CI 91.9 to 349.8 million), the wide interval reflecting uptake and weight uncertainty.
CONCLUSIONS: Consanguinity makes treatable AR rare diseases a non-trivial latent commitment for Morocco's 2024-2030 reform, invisible in a reimbursement reference that predates the modern orphan-drug wave and lists a Moroccan price for only a minority of these therapies. Quantifying this space, even under uncertainty, is a prerequisite for a rational orphan-drug pathway, for which HT1 offers a tractable, empirically anchored entry point.
METHODS: A prevalence-based budget impact model (AMO perspective, five years, Moroccan Dirham [MAD]) adjusted AR incidence for consanguinity (mean coefficient 0.0065). HT1 was anchored on a measured Maghreb incidence, with Tunisia as proxy, versus the global rate. The treated population followed a procurement-anchored ramp under a counterfactual coverage scenario. Nitisinone used the Moroccan public acquisition price from a 2023 hospital tender, applied by weight at 1 mg/kg/day. Probabilistic sensitivity analysis (PSA; 10,000 iterations, fixed seed) propagated uptake, weight, and dose uncertainty.
RESULTS: Measured Maghreb HT1 incidence was 1 in 14,804 (6.8 per 100,000), about sevenfold the global 1 in 100,000; the consanguinity adjustment alone predicted 1 in 32,750. Against roughly 43 expected annual cases, the treated population rose from 15 to 70 over five years, exposing a wide access gap. Annual nitisinone cost was approximately MAD 682,000 per patient. Five-year budget impact was MAD 145.2 million (base case, about MAD 29 million yearly); PSA mean was MAD 192.8 million (95% CI 91.9 to 349.8 million), the wide interval reflecting uptake and weight uncertainty.
CONCLUSIONS: Consanguinity makes treatable AR rare diseases a non-trivial latent commitment for Morocco's 2024-2030 reform, invisible in a reimbursement reference that predates the modern orphan-drug wave and lists a Moroccan price for only a minority of these therapies. Quantifying this space, even under uncertainty, is a prerequisite for a rational orphan-drug pathway, for which HT1 offers a tractable, empirically anchored entry point.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE109
Topic
Economic Evaluation, Epidemiology & Public Health, Health Policy & Regulatory
Topic Subcategory
Budget Impact Analysis
Disease
Diabetes/Endocrine/Metabolic Disorders (including obesity), Neurological Disorders, Pediatrics, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)