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Controlled Human Infections As a Tool to Reduce Uncertainty in Clinical Vaccine Development

Vaccines can be extremely cost-effective public health measures. Unfortunately the research and development (R&D) of novel vaccines is suffering from rising costs and declining success rates. Because many vaccines target low- and middle income markets (LMIC), output needs to be maintained at a c...

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Autores principales: Roestenberg, Meta, Kamerling, Ingrid M. C., de Visser, Saco J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6215823/
https://www.ncbi.nlm.nih.gov/pubmed/30420951
http://dx.doi.org/10.3389/fmed.2018.00297
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author Roestenberg, Meta
Kamerling, Ingrid M. C.
de Visser, Saco J.
author_facet Roestenberg, Meta
Kamerling, Ingrid M. C.
de Visser, Saco J.
author_sort Roestenberg, Meta
collection PubMed
description Vaccines can be extremely cost-effective public health measures. Unfortunately the research and development (R&D) of novel vaccines is suffering from rising costs and declining success rates. Because many vaccines target low- and middle income markets (LMIC), output needs to be maintained at a constrained budget. In addition, scientific neglect and political uncertainty around reimbursement decisions make it an unattractive arena for private investors. The vaccine development pipeline for LMIC thus is in need for a different, sustainable, and cost-effective development model. In conventional vaccine development, objectives for every clinical development phase have been predefined. However, given the scarcity of resources, the most efficient clinical development path should identify vaccine candidates with the highest potential impact as soon as possible. We argue for a custom-made question-based development path based on the scientific questions, success probabilities and investments required. One question can be addressed by several studies and one study can provide partial answers to multiple questions. An example of a question-based approach is the implementation of a controlled human malaria infection model (CHMI). Malaria vaccine R&D faces major scientific challenges and has limited resources. Therefore, early preliminary efficacy data needs to be obtained in order to reallocate resources as efficiently as possible and reduce clinical development costs. To meet this demand, novel malaria vaccines are tested for efficacy in so-called CHMI trials in which small groups of healthy volunteers are vaccinated and subsequently infected with malaria. Early evaluation studies of critical questions, such as CHMI, are highly rewarding, since they prevent expenditures on projects that are unlikely to succeed. Each set of estimated probabilities and costs (combined with market value) will have its own optimal priority sequence of questions to address. Algorithms can be designed to determine the optimal order in which questions should be addressed. Experimental infections of healthy volunteers is an example of how a question-based approach to vaccine development can be implemented and has the potential to change the arena of clinical vaccine development.
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spelling pubmed-62158232018-11-12 Controlled Human Infections As a Tool to Reduce Uncertainty in Clinical Vaccine Development Roestenberg, Meta Kamerling, Ingrid M. C. de Visser, Saco J. Front Med (Lausanne) Medicine Vaccines can be extremely cost-effective public health measures. Unfortunately the research and development (R&D) of novel vaccines is suffering from rising costs and declining success rates. Because many vaccines target low- and middle income markets (LMIC), output needs to be maintained at a constrained budget. In addition, scientific neglect and political uncertainty around reimbursement decisions make it an unattractive arena for private investors. The vaccine development pipeline for LMIC thus is in need for a different, sustainable, and cost-effective development model. In conventional vaccine development, objectives for every clinical development phase have been predefined. However, given the scarcity of resources, the most efficient clinical development path should identify vaccine candidates with the highest potential impact as soon as possible. We argue for a custom-made question-based development path based on the scientific questions, success probabilities and investments required. One question can be addressed by several studies and one study can provide partial answers to multiple questions. An example of a question-based approach is the implementation of a controlled human malaria infection model (CHMI). Malaria vaccine R&D faces major scientific challenges and has limited resources. Therefore, early preliminary efficacy data needs to be obtained in order to reallocate resources as efficiently as possible and reduce clinical development costs. To meet this demand, novel malaria vaccines are tested for efficacy in so-called CHMI trials in which small groups of healthy volunteers are vaccinated and subsequently infected with malaria. Early evaluation studies of critical questions, such as CHMI, are highly rewarding, since they prevent expenditures on projects that are unlikely to succeed. Each set of estimated probabilities and costs (combined with market value) will have its own optimal priority sequence of questions to address. Algorithms can be designed to determine the optimal order in which questions should be addressed. Experimental infections of healthy volunteers is an example of how a question-based approach to vaccine development can be implemented and has the potential to change the arena of clinical vaccine development. Frontiers Media S.A. 2018-10-29 /pmc/articles/PMC6215823/ /pubmed/30420951 http://dx.doi.org/10.3389/fmed.2018.00297 Text en Copyright © 2018 Roestenberg, Kamerling and de Visser. http://creativecommons.org/licenses/by/4.0/ This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Medicine
Roestenberg, Meta
Kamerling, Ingrid M. C.
de Visser, Saco J.
Controlled Human Infections As a Tool to Reduce Uncertainty in Clinical Vaccine Development
title Controlled Human Infections As a Tool to Reduce Uncertainty in Clinical Vaccine Development
title_full Controlled Human Infections As a Tool to Reduce Uncertainty in Clinical Vaccine Development
title_fullStr Controlled Human Infections As a Tool to Reduce Uncertainty in Clinical Vaccine Development
title_full_unstemmed Controlled Human Infections As a Tool to Reduce Uncertainty in Clinical Vaccine Development
title_short Controlled Human Infections As a Tool to Reduce Uncertainty in Clinical Vaccine Development
title_sort controlled human infections as a tool to reduce uncertainty in clinical vaccine development
topic Medicine
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6215823/
https://www.ncbi.nlm.nih.gov/pubmed/30420951
http://dx.doi.org/10.3389/fmed.2018.00297
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