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Structured benefit–risk evaluation for medicinal products: review of quantitative benefit–risk assessment findings in the literature
A favorable benefit–risk profile remains an essential requirement for marketing authorization of medicinal drugs and devices. Furthermore, prior subjective, implicit and inconsistent ad hoc benefit–risk assessment methods have rightly evolved towards more systematic, explicit or “structured” approac...
Autores principales: | , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
SAGE Publications
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7727082/ https://www.ncbi.nlm.nih.gov/pubmed/33343857 http://dx.doi.org/10.1177/2042098620976951 |
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author | Kürzinger, Marie-Laure Douarin, Ludivine Uzun, Ievgeniia El-Haddad, Chantal Hurst, William Juhaeri, Juhaeri Tcherny-Lessenot, Stéphanie |
author_facet | Kürzinger, Marie-Laure Douarin, Ludivine Uzun, Ievgeniia El-Haddad, Chantal Hurst, William Juhaeri, Juhaeri Tcherny-Lessenot, Stéphanie |
author_sort | Kürzinger, Marie-Laure |
collection | PubMed |
description | A favorable benefit–risk profile remains an essential requirement for marketing authorization of medicinal drugs and devices. Furthermore, prior subjective, implicit and inconsistent ad hoc benefit–risk assessment methods have rightly evolved towards more systematic, explicit or “structured” approaches. Contemporary structured benefit–risk evaluation aims at providing an objective assessment of the benefit–risk profile of medicinal products and a higher transparency for decision making purposes. The use of a descriptive framework should be the preferred starting point for a structured benefit–risk assessment. In support of more precise assessments, quantitative and semi-quantitative methodologies have been developed and utilized to complement descriptive or qualitative frameworks in order to facilitate the structured evaluation of the benefit–risk profile of medicinal products. In addition, quantitative structured benefit–risk analysis allows integration of patient preference data. Collecting patient perspectives throughout the medical product development process has become increasingly important and key to the regulatory decision-making process. Both industry and regulatory authorities increasingly rely on descriptive structured benefit–risk evaluation and frameworks in drug, vaccine and device evaluation and comparison. Although varied qualitative methods are more commonplace, quantitative approaches have recently been emphasized. However, it is unclear how frequently these quantitative frameworks have been used by pharmaceutical companies to support submission dossiers for drug approvals or to respond to the health authorities’ requests. The objective of this study has been to identify and review, for the first time, currently available, published, structured, quantitative benefit–risk evaluations which may have informed health care professionals and/or payor as well as contributed to decision making purposes in the regulatory setting for drug, vaccine and/or device approval. PLAIN LANGUAGE SUMMARY: Quantitative evaluation of the benefit–risk balance for medicinal products The review of the benefits and the risks associated with a medicinal product is called benefit–risk assessment. One of the conditions for a medicinal product to receive marketing authorization is to demonstrate a positive benefit–risk balance in which the benefits outweigh the risks. In order to enhance the transparency and consistency in the assessment of benefit–risk balance, frameworks and quantitative methods have been developed for decision making purposes and regulatory approvals of medicinal products. This article considers published quantitative benefit–risk evaluations which may have informed health care professionals and/or payor as well as contributed to decision making purposes in the regulatory setting for drug, vaccine and/or device approval. |
format | Online Article Text |
id | pubmed-7727082 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2020 |
publisher | SAGE Publications |
record_format | MEDLINE/PubMed |
spelling | pubmed-77270822020-12-18 Structured benefit–risk evaluation for medicinal products: review of quantitative benefit–risk assessment findings in the literature Kürzinger, Marie-Laure Douarin, Ludivine Uzun, Ievgeniia El-Haddad, Chantal Hurst, William Juhaeri, Juhaeri Tcherny-Lessenot, Stéphanie Ther Adv Drug Saf Review A favorable benefit–risk profile remains an essential requirement for marketing authorization of medicinal drugs and devices. Furthermore, prior subjective, implicit and inconsistent ad hoc benefit–risk assessment methods have rightly evolved towards more systematic, explicit or “structured” approaches. Contemporary structured benefit–risk evaluation aims at providing an objective assessment of the benefit–risk profile of medicinal products and a higher transparency for decision making purposes. The use of a descriptive framework should be the preferred starting point for a structured benefit–risk assessment. In support of more precise assessments, quantitative and semi-quantitative methodologies have been developed and utilized to complement descriptive or qualitative frameworks in order to facilitate the structured evaluation of the benefit–risk profile of medicinal products. In addition, quantitative structured benefit–risk analysis allows integration of patient preference data. Collecting patient perspectives throughout the medical product development process has become increasingly important and key to the regulatory decision-making process. Both industry and regulatory authorities increasingly rely on descriptive structured benefit–risk evaluation and frameworks in drug, vaccine and device evaluation and comparison. Although varied qualitative methods are more commonplace, quantitative approaches have recently been emphasized. However, it is unclear how frequently these quantitative frameworks have been used by pharmaceutical companies to support submission dossiers for drug approvals or to respond to the health authorities’ requests. The objective of this study has been to identify and review, for the first time, currently available, published, structured, quantitative benefit–risk evaluations which may have informed health care professionals and/or payor as well as contributed to decision making purposes in the regulatory setting for drug, vaccine and/or device approval. PLAIN LANGUAGE SUMMARY: Quantitative evaluation of the benefit–risk balance for medicinal products The review of the benefits and the risks associated with a medicinal product is called benefit–risk assessment. One of the conditions for a medicinal product to receive marketing authorization is to demonstrate a positive benefit–risk balance in which the benefits outweigh the risks. In order to enhance the transparency and consistency in the assessment of benefit–risk balance, frameworks and quantitative methods have been developed for decision making purposes and regulatory approvals of medicinal products. This article considers published quantitative benefit–risk evaluations which may have informed health care professionals and/or payor as well as contributed to decision making purposes in the regulatory setting for drug, vaccine and/or device approval. SAGE Publications 2020-12-08 /pmc/articles/PMC7727082/ /pubmed/33343857 http://dx.doi.org/10.1177/2042098620976951 Text en © The Author(s), 2020 https://creativecommons.org/licenses/by-nc/4.0/ This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 License (https://creativecommons.org/licenses/by-nc/4.0/) which permits non-commercial use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access page (https://us.sagepub.com/en-us/nam/open-access-at-sage). |
spellingShingle | Review Kürzinger, Marie-Laure Douarin, Ludivine Uzun, Ievgeniia El-Haddad, Chantal Hurst, William Juhaeri, Juhaeri Tcherny-Lessenot, Stéphanie Structured benefit–risk evaluation for medicinal products: review of quantitative benefit–risk assessment findings in the literature |
title | Structured benefit–risk evaluation for medicinal products: review of quantitative benefit–risk assessment findings in the literature |
title_full | Structured benefit–risk evaluation for medicinal products: review of quantitative benefit–risk assessment findings in the literature |
title_fullStr | Structured benefit–risk evaluation for medicinal products: review of quantitative benefit–risk assessment findings in the literature |
title_full_unstemmed | Structured benefit–risk evaluation for medicinal products: review of quantitative benefit–risk assessment findings in the literature |
title_short | Structured benefit–risk evaluation for medicinal products: review of quantitative benefit–risk assessment findings in the literature |
title_sort | structured benefit–risk evaluation for medicinal products: review of quantitative benefit–risk assessment findings in the literature |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7727082/ https://www.ncbi.nlm.nih.gov/pubmed/33343857 http://dx.doi.org/10.1177/2042098620976951 |
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