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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...

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Autores principales: Finch, Courtney L., Martinez, Christian, Leffel, Elizabeth, Skiadopoulos, Mario H., Hacker, Adam, Mwesigwa, Betty, Maïga, Diadié, Mugisa, Ian, Munkwase, Grant, Rustomjee, Roxana
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
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.
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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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