PRICING AN ORPHAN THERAPY WHEN COST-UTILITY ANALYSIS IS INFEASIBLE: A PRICE-EFFICACY FRONTIER FOR PRIMARY HYPEROXALURIA TYPE 1 IN MOROCCO
Author(s)
Omar Maoujoud, MD, PhD1, 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: In primary hyperoxaluria type 1, a recessive disease enriched in consanguineous Morocco, lumasiran, an RNA interference therapy targeting hepatic oxalate, is approved abroad and delays kidney failure but remains unregistered in Morocco and priced far above any payer threshold. Cost-utility analysis is infeasible: no validated model links treatment to renal survival (single-arm evidence; oxalate non-specific). We derived a price-efficacy frontier: the affordable annual price as a function of years of kidney failure delayed, from the mandatory basic-insurance payer perspective.
METHODS: A per-patient lifetime model used 3% continuous discounting in MAD. Treatment runs from diagnosis to kidney failure; duration equals the genotype-specific median onset age (9.9 to 33.9 years) plus delay. Value accrues only during the delay: avoided dialysis (132,600 MAD per year) plus the pre-dialysis-versus-dialysis utility gain. Affordable price equals discounted value over discounted treatment-years at one per-capita gross domestic product (35,000 MAD per quality-adjusted life-year). The delay was swept 10 to 34 years across three genotypes; probabilistic sensitivity analysis used 10,000 seeded iterations.
RESULTS: The frontier rises and plateaus at the avoided-dialysis ceiling, 136,450 MAD per year. For the median missense genotype at a 15-year delay, the affordable price was 48,600 MAD per year (mean 48,500; 95% interval 39,600 to 58,200), 98.6% below the lowest catalogue price. Across every genotype, delay, and threshold (to threefold gross domestic product and a 0.20 utility gain), the required reduction stayed above 96.7%. The dialysis offset dominated; the utility gain contributed under 3%.
CONCLUSIONS: Under assumptions maximally favourable to the manufacturer, the affordable price remains more than 95% below the catalogue price; the gap is structural. Where cost-utility analysis is infeasible, the frontier provides a delay-conditioned reference price applicable to a performance-based managed entry agreement under the 2024-2030 coverage reform, and locates the price reduction required to meet the local payer threshold.
METHODS: A per-patient lifetime model used 3% continuous discounting in MAD. Treatment runs from diagnosis to kidney failure; duration equals the genotype-specific median onset age (9.9 to 33.9 years) plus delay. Value accrues only during the delay: avoided dialysis (132,600 MAD per year) plus the pre-dialysis-versus-dialysis utility gain. Affordable price equals discounted value over discounted treatment-years at one per-capita gross domestic product (35,000 MAD per quality-adjusted life-year). The delay was swept 10 to 34 years across three genotypes; probabilistic sensitivity analysis used 10,000 seeded iterations.
RESULTS: The frontier rises and plateaus at the avoided-dialysis ceiling, 136,450 MAD per year. For the median missense genotype at a 15-year delay, the affordable price was 48,600 MAD per year (mean 48,500; 95% interval 39,600 to 58,200), 98.6% below the lowest catalogue price. Across every genotype, delay, and threshold (to threefold gross domestic product and a 0.20 utility gain), the required reduction stayed above 96.7%. The dialysis offset dominated; the utility gain contributed under 3%.
CONCLUSIONS: Under assumptions maximally favourable to the manufacturer, the affordable price remains more than 95% below the catalogue price; the gap is structural. Where cost-utility analysis is infeasible, the frontier provides a delay-conditioned reference price applicable to a performance-based managed entry agreement under the 2024-2030 coverage reform, and locates the price reduction required to meet the local payer threshold.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE721
Topic
Economic Evaluation, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal), Rare & Orphan Diseases, Urinary/Kidney Disorders