Compounded drugs as an alternative to the therapeutical gaps of inborn errors of metabolism
Autor(a) principal: | |
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Data de Publicação: | 2023 |
Outros Autores: | , , , , , , , |
Tipo de documento: | Artigo |
Idioma: | eng |
Título da fonte: | Brazilian Journal of Pharmaceutical Sciences |
Texto Completo: | https://www.revistas.usp.br/bjps/article/view/211575 |
Resumo: | Inborn errors of metabolism are rare disorders with few therapeutic options for their treatments, which can make patients suffer with complications. Therefore, compounded drugs might be a promising option given that they have the ability of meeting the patient’s specific needs, (i) identification of the main drugs described in the literature; (ii) proposal of compounding systems and (iii) calculation of the budgetary addition for the inclusion of these drugs into the Brazilian Unified Health System. The research conducted a literature review and used management data as well as data obtained from official Federal District government websites. The study identified 31 drugs for the treatment of inborn errors of metabolism. Fifty eight percent (58%) (18) of the medicines had their current demand identified, which are currently unmet by the local Health System. The estimated budget for the production of compounded drugs was of R$363,16.98 per year for approximately 300 patients. This estimated cost represents a budgetary addition of only 0.17% from the total of expenditures planned for drug acquirement. There is a therapeutic gap for inborn errors of metabolism and compounding pharmacies show potential in ensuring access to medicine therapy with a low-cost investment. |
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Brazilian Journal of Pharmaceutical Sciences |
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Compounded drugs as an alternative to the therapeutical gaps of inborn errors of metabolismRare diseasesMetabolismInborn errorsTherapeutic gapCompounded drugsInborn errors of metabolism are rare disorders with few therapeutic options for their treatments, which can make patients suffer with complications. Therefore, compounded drugs might be a promising option given that they have the ability of meeting the patient’s specific needs, (i) identification of the main drugs described in the literature; (ii) proposal of compounding systems and (iii) calculation of the budgetary addition for the inclusion of these drugs into the Brazilian Unified Health System. The research conducted a literature review and used management data as well as data obtained from official Federal District government websites. The study identified 31 drugs for the treatment of inborn errors of metabolism. Fifty eight percent (58%) (18) of the medicines had their current demand identified, which are currently unmet by the local Health System. The estimated budget for the production of compounded drugs was of R$363,16.98 per year for approximately 300 patients. This estimated cost represents a budgetary addition of only 0.17% from the total of expenditures planned for drug acquirement. There is a therapeutic gap for inborn errors of metabolism and compounding pharmacies show potential in ensuring access to medicine therapy with a low-cost investment.Universidade de São Paulo. Faculdade de Ciências Farmacêuticas2023-04-28info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionapplication/pdfhttps://www.revistas.usp.br/bjps/article/view/21157510.1590/s2175-97902023e21109Brazilian Journal of Pharmaceutical Sciences; Vol. 59 (2023); e21109Brazilian Journal of Pharmaceutical Sciences; v. 59 (2023); e21109Brazilian Journal of Pharmaceutical Sciences; Vol. 59 (2023); e211092175-97901984-8250reponame:Brazilian Journal of Pharmaceutical Sciencesinstname:Universidade de São Paulo (USP)instacron:USPenghttps://www.revistas.usp.br/bjps/article/view/211575/194566https://creativecommons.org/licenses/by/4.0/info:eu-repo/semantics/openAccessAdrielle da Silva FachiniLorena Freitas Barros MalaquiasLuma de Lira NogueiraLudmila Alvim Gomes Pinho GiaconeNoemia Urruth Leão TavaresMaria Teresinha de Oliveira CardosoGuilherme Martins GelfusoDayde Lane Mendonça da SilvaRafael Santos Santana2023-05-29T20:51:32Zoai:revistas.usp.br:article/211575Revistahttps://www.revistas.usp.br/bjps/indexPUBhttps://old.scielo.br/oai/scielo-oai.phpbjps@usp.br||elizabeth.igne@gmail.com2175-97901984-8250opendoar:2023-05-29T20:51:32Brazilian Journal of Pharmaceutical Sciences - Universidade de São Paulo (USP)false |
dc.title.none.fl_str_mv |
Compounded drugs as an alternative to the therapeutical gaps of inborn errors of metabolism |
title |
Compounded drugs as an alternative to the therapeutical gaps of inborn errors of metabolism |
spellingShingle |
Compounded drugs as an alternative to the therapeutical gaps of inborn errors of metabolism Adrielle da Silva Fachini Rare diseases Metabolism Inborn errors Therapeutic gap Compounded drugs |
title_short |
Compounded drugs as an alternative to the therapeutical gaps of inborn errors of metabolism |
title_full |
Compounded drugs as an alternative to the therapeutical gaps of inborn errors of metabolism |
title_fullStr |
Compounded drugs as an alternative to the therapeutical gaps of inborn errors of metabolism |
title_full_unstemmed |
Compounded drugs as an alternative to the therapeutical gaps of inborn errors of metabolism |
title_sort |
Compounded drugs as an alternative to the therapeutical gaps of inborn errors of metabolism |
author |
Adrielle da Silva Fachini |
author_facet |
Adrielle da Silva Fachini Lorena Freitas Barros Malaquias Luma de Lira Nogueira Ludmila Alvim Gomes Pinho Giacone Noemia Urruth Leão Tavares Maria Teresinha de Oliveira Cardoso Guilherme Martins Gelfuso Dayde Lane Mendonça da Silva Rafael Santos Santana |
author_role |
author |
author2 |
Lorena Freitas Barros Malaquias Luma de Lira Nogueira Ludmila Alvim Gomes Pinho Giacone Noemia Urruth Leão Tavares Maria Teresinha de Oliveira Cardoso Guilherme Martins Gelfuso Dayde Lane Mendonça da Silva Rafael Santos Santana |
author2_role |
author author author author author author author author |
dc.contributor.author.fl_str_mv |
Adrielle da Silva Fachini Lorena Freitas Barros Malaquias Luma de Lira Nogueira Ludmila Alvim Gomes Pinho Giacone Noemia Urruth Leão Tavares Maria Teresinha de Oliveira Cardoso Guilherme Martins Gelfuso Dayde Lane Mendonça da Silva Rafael Santos Santana |
dc.subject.por.fl_str_mv |
Rare diseases Metabolism Inborn errors Therapeutic gap Compounded drugs |
topic |
Rare diseases Metabolism Inborn errors Therapeutic gap Compounded drugs |
description |
Inborn errors of metabolism are rare disorders with few therapeutic options for their treatments, which can make patients suffer with complications. Therefore, compounded drugs might be a promising option given that they have the ability of meeting the patient’s specific needs, (i) identification of the main drugs described in the literature; (ii) proposal of compounding systems and (iii) calculation of the budgetary addition for the inclusion of these drugs into the Brazilian Unified Health System. The research conducted a literature review and used management data as well as data obtained from official Federal District government websites. The study identified 31 drugs for the treatment of inborn errors of metabolism. Fifty eight percent (58%) (18) of the medicines had their current demand identified, which are currently unmet by the local Health System. The estimated budget for the production of compounded drugs was of R$363,16.98 per year for approximately 300 patients. This estimated cost represents a budgetary addition of only 0.17% from the total of expenditures planned for drug acquirement. There is a therapeutic gap for inborn errors of metabolism and compounding pharmacies show potential in ensuring access to medicine therapy with a low-cost investment. |
publishDate |
2023 |
dc.date.none.fl_str_mv |
2023-04-28 |
dc.type.driver.fl_str_mv |
info:eu-repo/semantics/article info:eu-repo/semantics/publishedVersion |
format |
article |
status_str |
publishedVersion |
dc.identifier.uri.fl_str_mv |
https://www.revistas.usp.br/bjps/article/view/211575 10.1590/s2175-97902023e21109 |
url |
https://www.revistas.usp.br/bjps/article/view/211575 |
identifier_str_mv |
10.1590/s2175-97902023e21109 |
dc.language.iso.fl_str_mv |
eng |
language |
eng |
dc.relation.none.fl_str_mv |
https://www.revistas.usp.br/bjps/article/view/211575/194566 |
dc.rights.driver.fl_str_mv |
https://creativecommons.org/licenses/by/4.0/ info:eu-repo/semantics/openAccess |
rights_invalid_str_mv |
https://creativecommons.org/licenses/by/4.0/ |
eu_rights_str_mv |
openAccess |
dc.format.none.fl_str_mv |
application/pdf |
dc.publisher.none.fl_str_mv |
Universidade de São Paulo. Faculdade de Ciências Farmacêuticas |
publisher.none.fl_str_mv |
Universidade de São Paulo. Faculdade de Ciências Farmacêuticas |
dc.source.none.fl_str_mv |
Brazilian Journal of Pharmaceutical Sciences; Vol. 59 (2023); e21109 Brazilian Journal of Pharmaceutical Sciences; v. 59 (2023); e21109 Brazilian Journal of Pharmaceutical Sciences; Vol. 59 (2023); e21109 2175-9790 1984-8250 reponame:Brazilian Journal of Pharmaceutical Sciences instname:Universidade de São Paulo (USP) instacron:USP |
instname_str |
Universidade de São Paulo (USP) |
instacron_str |
USP |
institution |
USP |
reponame_str |
Brazilian Journal of Pharmaceutical Sciences |
collection |
Brazilian Journal of Pharmaceutical Sciences |
repository.name.fl_str_mv |
Brazilian Journal of Pharmaceutical Sciences - Universidade de São Paulo (USP) |
repository.mail.fl_str_mv |
bjps@usp.br||elizabeth.igne@gmail.com |
_version_ |
1800222918023577600 |