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The Iterative Development of Medicines Through the European Medicine Agency's Adaptive Pathway Approach

The development of novel regulatory tools such as adaptive clinical trial design and utilization of real-world evidence are topics of high interest. Recently, the European Medicines Agency (EMA) introduced the Adaptive Pathways (AP) that represents an innovative tool in healthcare systems allowing t...

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Autores principales: Nicotera, Giuseppe, Sferrazza, Gianluca, Serafino, Annalucia, Pierimarchi, Pasquale
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6610487/
https://www.ncbi.nlm.nih.gov/pubmed/31316991
http://dx.doi.org/10.3389/fmed.2019.00148
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author Nicotera, Giuseppe
Sferrazza, Gianluca
Serafino, Annalucia
Pierimarchi, Pasquale
author_facet Nicotera, Giuseppe
Sferrazza, Gianluca
Serafino, Annalucia
Pierimarchi, Pasquale
author_sort Nicotera, Giuseppe
collection PubMed
description The development of novel regulatory tools such as adaptive clinical trial design and utilization of real-world evidence are topics of high interest. Recently, the European Medicines Agency (EMA) introduced the Adaptive Pathways (AP) that represents an innovative tool in healthcare systems allowing the early dialogue with multiple stakeholders on promising and innovative medicinal products in areas with an high unmet medical need. The innovative aspect in the AP is the early involvement of several stakeholders such as pharmaceutical industry, the Academia, Health Technology Assessment (HTA) bodies, and patient representatives bringing their real experience with the disease and their expectations about the treatment. AP is not a new licensing tool but an opportunity for a very early discussions, before starting the phase II studies, among all stakeholders, including regulators, companies, HTA bodies, and patient representatives on a new potential medicine in areas of high unmet medical need. The aim of this paper is to describe the evolution of the AP approach from the beginning of the pilot project to date, highlighting major advances, and achievement at European level.
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spelling pubmed-66104872019-07-17 The Iterative Development of Medicines Through the European Medicine Agency's Adaptive Pathway Approach Nicotera, Giuseppe Sferrazza, Gianluca Serafino, Annalucia Pierimarchi, Pasquale Front Med (Lausanne) Medicine The development of novel regulatory tools such as adaptive clinical trial design and utilization of real-world evidence are topics of high interest. Recently, the European Medicines Agency (EMA) introduced the Adaptive Pathways (AP) that represents an innovative tool in healthcare systems allowing the early dialogue with multiple stakeholders on promising and innovative medicinal products in areas with an high unmet medical need. The innovative aspect in the AP is the early involvement of several stakeholders such as pharmaceutical industry, the Academia, Health Technology Assessment (HTA) bodies, and patient representatives bringing their real experience with the disease and their expectations about the treatment. AP is not a new licensing tool but an opportunity for a very early discussions, before starting the phase II studies, among all stakeholders, including regulators, companies, HTA bodies, and patient representatives on a new potential medicine in areas of high unmet medical need. The aim of this paper is to describe the evolution of the AP approach from the beginning of the pilot project to date, highlighting major advances, and achievement at European level. Frontiers Media S.A. 2019-06-27 /pmc/articles/PMC6610487/ /pubmed/31316991 http://dx.doi.org/10.3389/fmed.2019.00148 Text en Copyright © 2019 Nicotera, Sferrazza, Serafino and Pierimarchi. 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
Nicotera, Giuseppe
Sferrazza, Gianluca
Serafino, Annalucia
Pierimarchi, Pasquale
The Iterative Development of Medicines Through the European Medicine Agency's Adaptive Pathway Approach
title The Iterative Development of Medicines Through the European Medicine Agency's Adaptive Pathway Approach
title_full The Iterative Development of Medicines Through the European Medicine Agency's Adaptive Pathway Approach
title_fullStr The Iterative Development of Medicines Through the European Medicine Agency's Adaptive Pathway Approach
title_full_unstemmed The Iterative Development of Medicines Through the European Medicine Agency's Adaptive Pathway Approach
title_short The Iterative Development of Medicines Through the European Medicine Agency's Adaptive Pathway Approach
title_sort iterative development of medicines through the european medicine agency's adaptive pathway approach
topic Medicine
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6610487/
https://www.ncbi.nlm.nih.gov/pubmed/31316991
http://dx.doi.org/10.3389/fmed.2019.00148
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