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The challenges of access to innovative medicines with limited evidence in the European Union

The European Medicines Agency (EMA) fosters access to innovative medicines through accelerated procedures and flexibility in the authorization requirements for diseases with unmet medical needs, such as many rare diseases as well as oncological diseases. However, the resulting increase of medicines...

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Detalles Bibliográficos
Autores principales: Vallano, Antonio, Pontes, Caridad, Agustí, Antònia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10500193/
https://www.ncbi.nlm.nih.gov/pubmed/37719853
http://dx.doi.org/10.3389/fphar.2023.1215431
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author Vallano, Antonio
Pontes, Caridad
Agustí, Antònia
author_facet Vallano, Antonio
Pontes, Caridad
Agustí, Antònia
author_sort Vallano, Antonio
collection PubMed
description The European Medicines Agency (EMA) fosters access to innovative medicines through accelerated procedures and flexibility in the authorization requirements for diseases with unmet medical needs, such as many rare diseases as well as oncological diseases. However, the resulting increase of medicines being marketed with conditional authorizations and in exceptional circumstances has lead to higher clinical uncertainty about their efficacy and safety than when the standard authorizations are applied. This uncertainty has significant implications for clinical practice and the negotiation of pricing and reimbursement, particularly as high prices are based on assumptions of high value, supported by regulatory prioritization. The burden of clinical development is often shifted towards public healthcare systems, resulting in increased spending budgets and opportunity costs. Effective management of uncertainty, through appropriate testing and evaluation, and fair reflection of costs and risks in prices, is crucial. However, it is important not to sacrifice essential elements of evidence-based healthcare for the sake of access to new treatments. Balancing sensitive and rational access to new treatments, ensuring their safety, efficacy, and affordability to healthcare systems requires thoughtful decision-making. Ultimately, a responsible approach to timely access to innovative medicines that balances the needs of patients with healthcare systems’ concerns is necessary. This approach emphasizes the importance of evidence-based decision-making and fair pricing and reimbursement.
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spelling pubmed-105001932023-09-15 The challenges of access to innovative medicines with limited evidence in the European Union Vallano, Antonio Pontes, Caridad Agustí, Antònia Front Pharmacol Pharmacology The European Medicines Agency (EMA) fosters access to innovative medicines through accelerated procedures and flexibility in the authorization requirements for diseases with unmet medical needs, such as many rare diseases as well as oncological diseases. However, the resulting increase of medicines being marketed with conditional authorizations and in exceptional circumstances has lead to higher clinical uncertainty about their efficacy and safety than when the standard authorizations are applied. This uncertainty has significant implications for clinical practice and the negotiation of pricing and reimbursement, particularly as high prices are based on assumptions of high value, supported by regulatory prioritization. The burden of clinical development is often shifted towards public healthcare systems, resulting in increased spending budgets and opportunity costs. Effective management of uncertainty, through appropriate testing and evaluation, and fair reflection of costs and risks in prices, is crucial. However, it is important not to sacrifice essential elements of evidence-based healthcare for the sake of access to new treatments. Balancing sensitive and rational access to new treatments, ensuring their safety, efficacy, and affordability to healthcare systems requires thoughtful decision-making. Ultimately, a responsible approach to timely access to innovative medicines that balances the needs of patients with healthcare systems’ concerns is necessary. This approach emphasizes the importance of evidence-based decision-making and fair pricing and reimbursement. Frontiers Media S.A. 2023-08-31 /pmc/articles/PMC10500193/ /pubmed/37719853 http://dx.doi.org/10.3389/fphar.2023.1215431 Text en Copyright © 2023 Vallano, Pontes and Agustí. https://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 Pharmacology
Vallano, Antonio
Pontes, Caridad
Agustí, Antònia
The challenges of access to innovative medicines with limited evidence in the European Union
title The challenges of access to innovative medicines with limited evidence in the European Union
title_full The challenges of access to innovative medicines with limited evidence in the European Union
title_fullStr The challenges of access to innovative medicines with limited evidence in the European Union
title_full_unstemmed The challenges of access to innovative medicines with limited evidence in the European Union
title_short The challenges of access to innovative medicines with limited evidence in the European Union
title_sort challenges of access to innovative medicines with limited evidence in the european union
topic Pharmacology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10500193/
https://www.ncbi.nlm.nih.gov/pubmed/37719853
http://dx.doi.org/10.3389/fphar.2023.1215431
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