Economic outcomes of centralized procurements of gene therapy for patients with orphan diseases: inherited retinal dystrophy
Background. New pathogenetic treatment options, such as gene therapy, are now used to treat previously uncurable diseases. However, price of such treatment is high, especially in the case of orphan diseases, where costs may many-fold exceed the prices for other types of medication. This raises a que...
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| Format: | Article |
| Language: | Russian |
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IRBIS LLC
2022-01-01
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| Series: | Фармакоэкономика |
| Subjects: | |
| Online Access: | https://www.pharmacoeconomics.ru/jour/article/view/623 |
| _version_ | 1797878878380752896 |
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| author | N. А. Avxentyev Yu. V. Makarova V. V. Kadyshev |
| author_facet | N. А. Avxentyev Yu. V. Makarova V. V. Kadyshev |
| author_sort | N. А. Avxentyev |
| collection | DOAJ |
| description | Background. New pathogenetic treatment options, such as gene therapy, are now used to treat previously uncurable diseases. However, price of such treatment is high, especially in the case of orphan diseases, where costs may many-fold exceed the prices for other types of medication. This raises a question of optimal way of financing gene therapy in Russia.Objective: to evaluate economic consequences of centralizing procurement of gene therapy in 2021–2030 in the case of the drug indicated for treatment of biallelic RPE65 mutation-associated retinal dystrophy.Material and methods. Voretigene neparvovec is a new gene therapy that is used to treat RPE65 mutation-associated Leber congenital amaurosis and isolated retinitis pigmentosa. We estimated the number of patients (children and adults) that could be treated with voretigene neparvovec in 2021–2030 in Russia using demographic forecasting method, literature and expert data. Budget costs of treatment were estimated for two scenarios: status-quo, where gene therapy is purchased by regions for higher price, and centralized scenario with federal procurements and lower price for the drug.Results. Up to 100 children and 56 adults could be treated with voretigene neparvovec in 2021–2030 in Russia. Centralizing procurements at the expense of federal budget may save up to 20.7% or 1.8 billion rub. (1.13 billion rub. for children and 0.67 billion rub. for adults), compared to regional procurements.Conclusion. Centralizing procurements of expensive drugs intended for gene therapy of orphan diseases may save budget costs of the Russian Federation, compared to status-quo decentralized purchases. |
| first_indexed | 2024-04-10T02:40:01Z |
| format | Article |
| id | doaj.art-697af79c570746cea190751ae2c2aec4 |
| institution | Directory Open Access Journal |
| issn | 2070-4909 2070-4933 |
| language | Russian |
| last_indexed | 2024-04-10T02:40:01Z |
| publishDate | 2022-01-01 |
| publisher | IRBIS LLC |
| record_format | Article |
| series | Фармакоэкономика |
| spelling | doaj.art-697af79c570746cea190751ae2c2aec42023-03-13T07:48:17ZrusIRBIS LLCФармакоэкономика2070-49092070-49332022-01-0114445146110.17749/2070-4909/farmakoekonomika.2021.116365Economic outcomes of centralized procurements of gene therapy for patients with orphan diseases: inherited retinal dystrophyN. А. Avxentyev0Yu. V. Makarova1V. V. Kadyshev2Financial Research Institute; Institute for Applied Economic Research, Russian Presidential Academy of National Economy and Public AdministrationFinancial Research InstituteBochkov Research Centre for Medical GeneticsBackground. New pathogenetic treatment options, such as gene therapy, are now used to treat previously uncurable diseases. However, price of such treatment is high, especially in the case of orphan diseases, where costs may many-fold exceed the prices for other types of medication. This raises a question of optimal way of financing gene therapy in Russia.Objective: to evaluate economic consequences of centralizing procurement of gene therapy in 2021–2030 in the case of the drug indicated for treatment of biallelic RPE65 mutation-associated retinal dystrophy.Material and methods. Voretigene neparvovec is a new gene therapy that is used to treat RPE65 mutation-associated Leber congenital amaurosis and isolated retinitis pigmentosa. We estimated the number of patients (children and adults) that could be treated with voretigene neparvovec in 2021–2030 in Russia using demographic forecasting method, literature and expert data. Budget costs of treatment were estimated for two scenarios: status-quo, where gene therapy is purchased by regions for higher price, and centralized scenario with federal procurements and lower price for the drug.Results. Up to 100 children and 56 adults could be treated with voretigene neparvovec in 2021–2030 in Russia. Centralizing procurements at the expense of federal budget may save up to 20.7% or 1.8 billion rub. (1.13 billion rub. for children and 0.67 billion rub. for adults), compared to regional procurements.Conclusion. Centralizing procurements of expensive drugs intended for gene therapy of orphan diseases may save budget costs of the Russian Federation, compared to status-quo decentralized purchases.https://www.pharmacoeconomics.ru/jour/article/view/623inherited retinal dystrophyleber congenital amaurosisretinitis pigmentosagene therapybudgetpharmaeconomicsrpe65 gene |
| spellingShingle | N. А. Avxentyev Yu. V. Makarova V. V. Kadyshev Economic outcomes of centralized procurements of gene therapy for patients with orphan diseases: inherited retinal dystrophy Фармакоэкономика inherited retinal dystrophy leber congenital amaurosis retinitis pigmentosa gene therapy budget pharmaeconomics rpe65 gene |
| title | Economic outcomes of centralized procurements of gene therapy for patients with orphan diseases: inherited retinal dystrophy |
| title_full | Economic outcomes of centralized procurements of gene therapy for patients with orphan diseases: inherited retinal dystrophy |
| title_fullStr | Economic outcomes of centralized procurements of gene therapy for patients with orphan diseases: inherited retinal dystrophy |
| title_full_unstemmed | Economic outcomes of centralized procurements of gene therapy for patients with orphan diseases: inherited retinal dystrophy |
| title_short | Economic outcomes of centralized procurements of gene therapy for patients with orphan diseases: inherited retinal dystrophy |
| title_sort | economic outcomes of centralized procurements of gene therapy for patients with orphan diseases inherited retinal dystrophy |
| topic | inherited retinal dystrophy leber congenital amaurosis retinitis pigmentosa gene therapy budget pharmaeconomics rpe65 gene |
| url | https://www.pharmacoeconomics.ru/jour/article/view/623 |
| work_keys_str_mv | AT naavxentyev economicoutcomesofcentralizedprocurementsofgenetherapyforpatientswithorphandiseasesinheritedretinaldystrophy AT yuvmakarova economicoutcomesofcentralizedprocurementsofgenetherapyforpatientswithorphandiseasesinheritedretinaldystrophy AT vvkadyshev economicoutcomesofcentralizedprocurementsofgenetherapyforpatientswithorphandiseasesinheritedretinaldystrophy |