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Vaccine Licensure in the Absence of Human Efficacy Data
Clinical vaccine development and regulatory approval generally occurs in a linear, sequential manner: Phase 1: safety, immunogenicity; Phase 2: immunogenicity, safety, dose ranging, and preliminary efficacy; Phase 3: definitive efficacy, safety, lot consistency; and following regulatory approval, Ph...
Autores principales: | , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8954083/ https://www.ncbi.nlm.nih.gov/pubmed/35335000 http://dx.doi.org/10.3390/vaccines10030368 |
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author | Finch, Courtney L. Martinez, Christian Leffel, Elizabeth Skiadopoulos, Mario H. Hacker, Adam Mwesigwa, Betty Maïga, Diadié Mugisa, Ian Munkwase, Grant Rustomjee, Roxana |
author_facet | Finch, Courtney L. Martinez, Christian Leffel, Elizabeth Skiadopoulos, Mario H. Hacker, Adam Mwesigwa, Betty Maïga, Diadié Mugisa, Ian Munkwase, Grant Rustomjee, Roxana |
author_sort | Finch, Courtney L. |
collection | PubMed |
description | Clinical vaccine development and regulatory approval generally occurs in a linear, sequential manner: Phase 1: safety, immunogenicity; Phase 2: immunogenicity, safety, dose ranging, and preliminary efficacy; Phase 3: definitive efficacy, safety, lot consistency; and following regulatory approval, Phase 4: post-marketing safety and effectiveness. For candidate filovirus vaccines, where correlates of protection have not been identified, and phase 2 and 3 efficacy of disease prevention trials untenable, large and/or protracted, each trial may span decades, with full licensure expected only after several decades of development. Given the urgent unmet need for new Marburg virus and Ebola Sudan virus vaccines, the Sabin Vaccine Institute hosted a key stakeholder virtual meeting in May 2021 to explore the possibility of licensure by use of an “animal rule-like” licensure process, based on a risk/benefit assessment specific to regional needs and informed by epidemiology. This may be appropriate for diseases where there are no or limited treatment options, and those prone to sporadic outbreaks with high rates of transmission, morbidity, and mortality. The discussion focused on two contexts: licensure within the Ugandan regulatory environment, a high burden country where Ebola vaccine trials are ongoing, and licensure by the United States FDA—a well-resourced regulatory agency. |
format | Online Article Text |
id | pubmed-8954083 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-89540832022-03-26 Vaccine Licensure in the Absence of Human Efficacy Data Finch, Courtney L. Martinez, Christian Leffel, Elizabeth Skiadopoulos, Mario H. Hacker, Adam Mwesigwa, Betty Maïga, Diadié Mugisa, Ian Munkwase, Grant Rustomjee, Roxana Vaccines (Basel) Review Clinical vaccine development and regulatory approval generally occurs in a linear, sequential manner: Phase 1: safety, immunogenicity; Phase 2: immunogenicity, safety, dose ranging, and preliminary efficacy; Phase 3: definitive efficacy, safety, lot consistency; and following regulatory approval, Phase 4: post-marketing safety and effectiveness. For candidate filovirus vaccines, where correlates of protection have not been identified, and phase 2 and 3 efficacy of disease prevention trials untenable, large and/or protracted, each trial may span decades, with full licensure expected only after several decades of development. Given the urgent unmet need for new Marburg virus and Ebola Sudan virus vaccines, the Sabin Vaccine Institute hosted a key stakeholder virtual meeting in May 2021 to explore the possibility of licensure by use of an “animal rule-like” licensure process, based on a risk/benefit assessment specific to regional needs and informed by epidemiology. This may be appropriate for diseases where there are no or limited treatment options, and those prone to sporadic outbreaks with high rates of transmission, morbidity, and mortality. The discussion focused on two contexts: licensure within the Ugandan regulatory environment, a high burden country where Ebola vaccine trials are ongoing, and licensure by the United States FDA—a well-resourced regulatory agency. MDPI 2022-02-26 /pmc/articles/PMC8954083/ /pubmed/35335000 http://dx.doi.org/10.3390/vaccines10030368 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Review Finch, Courtney L. Martinez, Christian Leffel, Elizabeth Skiadopoulos, Mario H. Hacker, Adam Mwesigwa, Betty Maïga, Diadié Mugisa, Ian Munkwase, Grant Rustomjee, Roxana Vaccine Licensure in the Absence of Human Efficacy Data |
title | Vaccine Licensure in the Absence of Human Efficacy Data |
title_full | Vaccine Licensure in the Absence of Human Efficacy Data |
title_fullStr | Vaccine Licensure in the Absence of Human Efficacy Data |
title_full_unstemmed | Vaccine Licensure in the Absence of Human Efficacy Data |
title_short | Vaccine Licensure in the Absence of Human Efficacy Data |
title_sort | vaccine licensure in the absence of human efficacy data |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8954083/ https://www.ncbi.nlm.nih.gov/pubmed/35335000 http://dx.doi.org/10.3390/vaccines10030368 |
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