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The use of special approval medicines among pediatric patients in a tertiary care hospital: A reality check

BACKGROUND: Special approval medicines (SAMs) are medicines used with approval from the Director General of Health Malaysia when the therapeutic options within regulatory and formulary boundaries appear unsuitable or ineffective to treat the patients. OBJECTIVES: To examine and characterize the use...

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Autores principales: Balan, Shamala, Koo, Kaitian, Muhamad Hamdan, Muhamad Danial, Lee, Su Vin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9587311/
https://www.ncbi.nlm.nih.gov/pubmed/36281358
http://dx.doi.org/10.1016/j.rcsop.2022.100188
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author Balan, Shamala
Koo, Kaitian
Muhamad Hamdan, Muhamad Danial
Lee, Su Vin
author_facet Balan, Shamala
Koo, Kaitian
Muhamad Hamdan, Muhamad Danial
Lee, Su Vin
author_sort Balan, Shamala
collection PubMed
description BACKGROUND: Special approval medicines (SAMs) are medicines used with approval from the Director General of Health Malaysia when the therapeutic options within regulatory and formulary boundaries appear unsuitable or ineffective to treat the patients. OBJECTIVES: To examine and characterize the use of SAMs among children in a Malaysian tertiary care hospital. METHODS: The named-patient basis SAM application forms, cover letter, pharmacist review summary and patient monitoring forms available at the Pharmacy Department between 1st January 2019 and 31st December 2020 were reviewed. Unprocessed, unapproved and stock-basis applications were excluded. The outcome measures were categories, scope, off-label use and cost of SAM. Per-patient data were analyzed descriptively. RESULTS: Overall, 1010 patients (mean age of 8.7 ± 5.6 years) were involved in 328 SAMs applications. The most common SAMs pharmacological groups were nervous system (n = 371, 36.7%) and antineoplastic and immunomodulating agents (n = 332, 32.9%). Top three SAMs were melatonin (11.5%), scopolamine (7.6%) and cholecalciferol (7.1%). A total of 837 (82.9%) and 513 (50.8%) patients were involved in the SAMs applications for non-formulary and unregistered medicines, respectively. Unregistered, non-formulary medicines were applied for 47.3% (n = 478) of the patients. The majority of the scope for SAMs (64.7%) were to substitute the available alternatives in the national formulary which were ineffective or sub-optimal for the patients. Among the 262 patients with repeat applications, 93.8% reported disease or symptom improvement while 1.9% experienced side effects. Up to 17% of SAMs analyzed in this study were used for off-label indications. The total cost of the SAMs was RM8,748,358.38 (USD 2,090,418.86). CONCLUSION: The use of SAMs among children in this hospital involved unregistered, non-formulary medicines used to substitute the available alternatives in the formulary. A concerted effort is warranted in exploring supplementary mechanisms to enhance the medicine registration process and formulary system towards facilitating enhanced provision of treatment for children.
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spelling pubmed-95873112022-10-23 The use of special approval medicines among pediatric patients in a tertiary care hospital: A reality check Balan, Shamala Koo, Kaitian Muhamad Hamdan, Muhamad Danial Lee, Su Vin Explor Res Clin Soc Pharm Article BACKGROUND: Special approval medicines (SAMs) are medicines used with approval from the Director General of Health Malaysia when the therapeutic options within regulatory and formulary boundaries appear unsuitable or ineffective to treat the patients. OBJECTIVES: To examine and characterize the use of SAMs among children in a Malaysian tertiary care hospital. METHODS: The named-patient basis SAM application forms, cover letter, pharmacist review summary and patient monitoring forms available at the Pharmacy Department between 1st January 2019 and 31st December 2020 were reviewed. Unprocessed, unapproved and stock-basis applications were excluded. The outcome measures were categories, scope, off-label use and cost of SAM. Per-patient data were analyzed descriptively. RESULTS: Overall, 1010 patients (mean age of 8.7 ± 5.6 years) were involved in 328 SAMs applications. The most common SAMs pharmacological groups were nervous system (n = 371, 36.7%) and antineoplastic and immunomodulating agents (n = 332, 32.9%). Top three SAMs were melatonin (11.5%), scopolamine (7.6%) and cholecalciferol (7.1%). A total of 837 (82.9%) and 513 (50.8%) patients were involved in the SAMs applications for non-formulary and unregistered medicines, respectively. Unregistered, non-formulary medicines were applied for 47.3% (n = 478) of the patients. The majority of the scope for SAMs (64.7%) were to substitute the available alternatives in the national formulary which were ineffective or sub-optimal for the patients. Among the 262 patients with repeat applications, 93.8% reported disease or symptom improvement while 1.9% experienced side effects. Up to 17% of SAMs analyzed in this study were used for off-label indications. The total cost of the SAMs was RM8,748,358.38 (USD 2,090,418.86). CONCLUSION: The use of SAMs among children in this hospital involved unregistered, non-formulary medicines used to substitute the available alternatives in the formulary. A concerted effort is warranted in exploring supplementary mechanisms to enhance the medicine registration process and formulary system towards facilitating enhanced provision of treatment for children. Elsevier 2022-10-12 /pmc/articles/PMC9587311/ /pubmed/36281358 http://dx.doi.org/10.1016/j.rcsop.2022.100188 Text en © 2022 The Authors https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
spellingShingle Article
Balan, Shamala
Koo, Kaitian
Muhamad Hamdan, Muhamad Danial
Lee, Su Vin
The use of special approval medicines among pediatric patients in a tertiary care hospital: A reality check
title The use of special approval medicines among pediatric patients in a tertiary care hospital: A reality check
title_full The use of special approval medicines among pediatric patients in a tertiary care hospital: A reality check
title_fullStr The use of special approval medicines among pediatric patients in a tertiary care hospital: A reality check
title_full_unstemmed The use of special approval medicines among pediatric patients in a tertiary care hospital: A reality check
title_short The use of special approval medicines among pediatric patients in a tertiary care hospital: A reality check
title_sort use of special approval medicines among pediatric patients in a tertiary care hospital: a reality check
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9587311/
https://www.ncbi.nlm.nih.gov/pubmed/36281358
http://dx.doi.org/10.1016/j.rcsop.2022.100188
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