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Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations

Patients with rare diseases often have limited or no options for approved treatments or participation in clinical trials. In such cases, expanded access (or “compassionate use”) provides a potential means of accessing unapproved investigational medicines. It is also possible to capture and analyze c...

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Autores principales: Polak, Tobias B., Cucchi, David G. J., van Rosmalen, Joost, Uyl-de Groot, Carin A., Darrow, Jonathan J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9168458/
https://www.ncbi.nlm.nih.gov/pubmed/35677436
http://dx.doi.org/10.3389/fphar.2022.913567
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author Polak, Tobias B.
Cucchi, David G. J.
van Rosmalen, Joost
Uyl-de Groot, Carin A.
Darrow, Jonathan J.
author_facet Polak, Tobias B.
Cucchi, David G. J.
van Rosmalen, Joost
Uyl-de Groot, Carin A.
Darrow, Jonathan J.
author_sort Polak, Tobias B.
collection PubMed
description Patients with rare diseases often have limited or no options for approved treatments or participation in clinical trials. In such cases, expanded access (or “compassionate use”) provides a potential means of accessing unapproved investigational medicines. It is also possible to capture and analyze clinical data from such use, but doing so is controversial. In this perspective, we offer examples of evidence derived from expanded access programs for rare diseases to illustrate its potential value to the decision-making of regulators and payers in the European Union and the United States. We discuss ethical and regulatory aspects to the use of expanded access data, with a focus on rare disease medicines. The heterogeneous approach to expanded access among countries within the European Union leaves uncertainties to what extent data can be collected and analyzed. We recommend the issuance of new guidance on data collection during expanded access, harmonization of European pathways, and an update of existing European compassionate use guidance. We hereby aim to clarify the supportive role of expanded access in evidence generation. Harmonization across Europe of expanded access regulations could reduce manufacturer burdens, improve patient access, and yield better data. These changes would better balance the need to generate quality evidence with the desire for pre-approval access to investigational medicine.
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spelling pubmed-91684582022-06-07 Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations Polak, Tobias B. Cucchi, David G. J. van Rosmalen, Joost Uyl-de Groot, Carin A. Darrow, Jonathan J. Front Pharmacol Pharmacology Patients with rare diseases often have limited or no options for approved treatments or participation in clinical trials. In such cases, expanded access (or “compassionate use”) provides a potential means of accessing unapproved investigational medicines. It is also possible to capture and analyze clinical data from such use, but doing so is controversial. In this perspective, we offer examples of evidence derived from expanded access programs for rare diseases to illustrate its potential value to the decision-making of regulators and payers in the European Union and the United States. We discuss ethical and regulatory aspects to the use of expanded access data, with a focus on rare disease medicines. The heterogeneous approach to expanded access among countries within the European Union leaves uncertainties to what extent data can be collected and analyzed. We recommend the issuance of new guidance on data collection during expanded access, harmonization of European pathways, and an update of existing European compassionate use guidance. We hereby aim to clarify the supportive role of expanded access in evidence generation. Harmonization across Europe of expanded access regulations could reduce manufacturer burdens, improve patient access, and yield better data. These changes would better balance the need to generate quality evidence with the desire for pre-approval access to investigational medicine. Frontiers Media S.A. 2022-05-23 /pmc/articles/PMC9168458/ /pubmed/35677436 http://dx.doi.org/10.3389/fphar.2022.913567 Text en Copyright © 2022 Polak, Cucchi, van Rosmalen, Uyl-de Groot and Darrow. 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
Polak, Tobias B.
Cucchi, David G. J.
van Rosmalen, Joost
Uyl-de Groot, Carin A.
Darrow, Jonathan J.
Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations
title Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations
title_full Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations
title_fullStr Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations
title_full_unstemmed Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations
title_short Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations
title_sort generating evidence from expanded access use of rare disease medicines: challenges and recommendations
topic Pharmacology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9168458/
https://www.ncbi.nlm.nih.gov/pubmed/35677436
http://dx.doi.org/10.3389/fphar.2022.913567
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