Early Cost-Utility Analysis of Ataluren and Eteplirsen in the Treatment of Duchenne Muscular Dystrophy in Egypt
Copyright © 2023 International Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved..
OBJECTIVES: Ataluren and eteplirsen are orphan drugs that delay progression of Duchenne muscular dystrophy in mutation-specific subgroups. They have yet to be approved in Egypt but are expected to reach the market soon. This study describes 2 cost-utility models comparing the drugs with the standard of care.
METHODS: We used a partition-survival model with 5 states based on the ambulatory status to model a cohort of ambulatory patients at the age of 5 years. Baseline curves were obtained from a published model; then the ambulation loss curve was updated using the Kaplan-Meier curve of the standard of care from a study by McDonald et al. 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.
RESULTS: In the base case, ataluren 1000 mg and eteplirsen 50 mg/mL resulted in an incremental cost-effectiveness ratio of EGP 51 745 605 and EGP 69 652 533/quality-adjusted life-year, respectively, at their hypothetical prices of EGP 308 600 for ataluren 30-sachet pack and EGP 62 800 for eteplirsen 10 mL vial. The incremental cost-effectiveness ratio was sensitive to health state utilities but not to state costs. At EGP 911 719/quality-adjusted life-year threshold, the value-based prices were EGP 4680 for ataluren 1000 mg and EGP 733 for eteplirsen 10 mL vial.
CONCLUSIONS: Based on these models, there is a huge gap between the prices of orphan drugs and their value-based prices, which highlights the need for major policy reforms in the assessment and pricing of orphan drugs.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2023 |
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| Erschienen: |
2023 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:38 |
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| Enthalten in: |
Value in health regional issues - 38(2023) vom: 02. Nov., Seite 109-117 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Shehata, Zahraa [VerfasserIn] |
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| Links: |
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| Themen: |
AIW6036FAS |
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| Anmerkungen: |
Date Completed 14.11.2023 Date Revised 29.11.2023 published: Print-Electronic Citation Status MEDLINE |
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| doi: |
10.1016/j.vhri.2023.08.004 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM363552030 |
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| 245 | 1 | 0 | |a Early Cost-Utility Analysis of Ataluren and Eteplirsen in the Treatment of Duchenne Muscular Dystrophy in Egypt |
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| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a Copyright © 2023 International Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved. | ||
| 520 | |a OBJECTIVES: Ataluren and eteplirsen are orphan drugs that delay progression of Duchenne muscular dystrophy in mutation-specific subgroups. They have yet to be approved in Egypt but are expected to reach the market soon. This study describes 2 cost-utility models comparing the drugs with the standard of care | ||
| 520 | |a METHODS: We used a partition-survival model with 5 states based on the ambulatory status to model a cohort of ambulatory patients at the age of 5 years. Baseline curves were obtained from a published model; then the ambulation loss curve was updated using the Kaplan-Meier curve of the standard of care from a study by McDonald et al. 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 | ||
| 520 | |a RESULTS: In the base case, ataluren 1000 mg and eteplirsen 50 mg/mL resulted in an incremental cost-effectiveness ratio of EGP 51 745 605 and EGP 69 652 533/quality-adjusted life-year, respectively, at their hypothetical prices of EGP 308 600 for ataluren 30-sachet pack and EGP 62 800 for eteplirsen 10 mL vial. The incremental cost-effectiveness ratio was sensitive to health state utilities but not to state costs. At EGP 911 719/quality-adjusted life-year threshold, the value-based prices were EGP 4680 for ataluren 1000 mg and EGP 733 for eteplirsen 10 mL vial | ||
| 520 | |a CONCLUSIONS: Based on these models, there is a huge gap between the prices of orphan drugs and their value-based prices, which highlights the need for major policy reforms in the assessment and pricing of orphan drugs | ||
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| 700 | 1 | |a El Sherif, Rasha |e verfasserin |4 aut | |
| 700 | 1 | |a Abdelrahim, Mohamed |e verfasserin |4 aut | |
| 700 | 1 | |a Dawoud, Dalia |e verfasserin |4 aut | |
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