Early cost-utility analysis of Ataluren and Eteplirsen in the treatment of Duchenne Muscular Dystrophy In Egypt

• Published in: Value in health regional issues 2023-11, Vol.38, p.109-117
• Main Authors: Shehata, Zahraa, Metry, Andrew, Rabea, Hoda, El Sherif, Rasha, Abdelrahim, Mohamed, Dawoud, Dalia
• Format: Article
• Language: English
• ISSN: 2212-1099; 2212-1102
• DOI: 10.1016/j.vhri.2023.08.004

Ataluren and Eteplirsen are orphan drugs that delay progression of Duchenne muscular dystrophy (DMD) in mutation-specific subgroups. They have yet to be approved in Egypt but are expected to reach the market soon. This study describes two cost-utility models comparing the drugs with the standard of care (SOC). We used a partition-survival model with five states based on the ambulatory status to model a cohort of ambulatory patients at the age of five. Baseline curves were obtained from a published model then the ambulation loss curve was updated using the KM curve of the SOC from a study by McDonald et al. 2022. Other curves were updated by calibration to this curve. Costs and utilities were from a local study. Deterministic and probabilistic sensitivity analyses were conducted. Prices were estimated based on other orphan drugs’ prices. In the base case, Ataluren 1000 mg and Eteplirsen 50 mg/ml resulted in an ICER of 51,745,605 and 69,652,533 EGP/QALY, respectively at their hypothetical prices of EGP 308,600 for Ataluren 30-sachet pack and EGP 62,800 for Eteplirsen 10 ml vial. The ICER was sensitive to health state utilities but not to state costs. At 911,719 EGP/QALY threshold, the value-based prices (VBPs) were EGP 4680 for Ataluren 1000 mg and EGP 733 for Eteplirsen 10 ml vial. Based on these models, there is a huge gap between the prices of orphan drugs and their VBPs which highlights the need for major policy reforms in the assessment and pricing of orphan drugs. ••Studies assessing the cost-utility of novel Duchenne muscular dystrophy (DMD) treatments in the literature are scarce. While in Egypt, this is considered the first published study in the area of a rare disease.••New DMD medications (Ataluren and Eteplirsen) are expected to reach the market with list prices that are not cost-effective for the Egyptian society, according to the results of this study. Based on the current models, the calculated value-based prices of these drugs are very low compared to the expected prices (as estimated from the prices of other orphan drugs in Egypt).••Despite the recent efforts to update the national cost-effectiveness threshold considering disease rarity and severity factors, more policy reforms are still required to allow access to orphan drugs at fair prices for both manufacturers and society.