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Perspectives, Expectations, and Concerns of European Patient Advocates on Advanced Therapy Medicinal Products

This paper presents the results of a qualitative study based on semi-structured interviews of 10 expert patient advocates on several different issues around Advanced Therapy Medicinal Products (ATMPs). The interviews were conducted between February and May 2020 based on a guideline with a list of 8...

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Autores principales: Benvenuti, Stefano, Wang, Chiuhui Mary, Borroni, Simona
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8649896/
https://www.ncbi.nlm.nih.gov/pubmed/34888320
http://dx.doi.org/10.3389/fmed.2021.728529
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author Benvenuti, Stefano
Wang, Chiuhui Mary
Borroni, Simona
author_facet Benvenuti, Stefano
Wang, Chiuhui Mary
Borroni, Simona
author_sort Benvenuti, Stefano
collection PubMed
description This paper presents the results of a qualitative study based on semi-structured interviews of 10 expert patient advocates on several different issues around Advanced Therapy Medicinal Products (ATMPs). The interviews were conducted between February and May 2020 based on a guideline with a list of 8 topics that covered concerns about safety and ethics, access problems and limitations, pricing of ATMPs and educational needs for patient communities. Overall, the interviewees expressed a high degree of convergence of opinions on most of the topics and especially on the identification of the reasons for concern. Conversely, when asked about possible solutions, quite a wide range of solutions were proposed, although with many common points. However, it highlights that the debate is still in its infancy and that there are not yet consolidated positions across the whole community. A general concern emerging from all the interviews is the potential limitation of access to approved ATMPs, both due to the high prices and to the geographical concentration of treatment centers. However, patients recognize the value of a model with a limited number of specialized clinical centers administering these therapies. On the ethical side, patients do not show particular concern as long as ATMPs and the underlying technology is used to treat severe diseases. Finally, patients are asking for both more education on ATMPs as well as for a more continuous involvement of patient representatives in the whole “life-cycle” of a new ATMP, from the development phase to the authorization, from the definition of the reimbursement scheme to the collection of Real Word Data on safety and long-term efficacy of the treatment.
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spelling pubmed-86498962021-12-08 Perspectives, Expectations, and Concerns of European Patient Advocates on Advanced Therapy Medicinal Products Benvenuti, Stefano Wang, Chiuhui Mary Borroni, Simona Front Med (Lausanne) Medicine This paper presents the results of a qualitative study based on semi-structured interviews of 10 expert patient advocates on several different issues around Advanced Therapy Medicinal Products (ATMPs). The interviews were conducted between February and May 2020 based on a guideline with a list of 8 topics that covered concerns about safety and ethics, access problems and limitations, pricing of ATMPs and educational needs for patient communities. Overall, the interviewees expressed a high degree of convergence of opinions on most of the topics and especially on the identification of the reasons for concern. Conversely, when asked about possible solutions, quite a wide range of solutions were proposed, although with many common points. However, it highlights that the debate is still in its infancy and that there are not yet consolidated positions across the whole community. A general concern emerging from all the interviews is the potential limitation of access to approved ATMPs, both due to the high prices and to the geographical concentration of treatment centers. However, patients recognize the value of a model with a limited number of specialized clinical centers administering these therapies. On the ethical side, patients do not show particular concern as long as ATMPs and the underlying technology is used to treat severe diseases. Finally, patients are asking for both more education on ATMPs as well as for a more continuous involvement of patient representatives in the whole “life-cycle” of a new ATMP, from the development phase to the authorization, from the definition of the reimbursement scheme to the collection of Real Word Data on safety and long-term efficacy of the treatment. Frontiers Media S.A. 2021-11-23 /pmc/articles/PMC8649896/ /pubmed/34888320 http://dx.doi.org/10.3389/fmed.2021.728529 Text en Copyright © 2021 Benvenuti, Wang and Borroni. 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 Medicine
Benvenuti, Stefano
Wang, Chiuhui Mary
Borroni, Simona
Perspectives, Expectations, and Concerns of European Patient Advocates on Advanced Therapy Medicinal Products
title Perspectives, Expectations, and Concerns of European Patient Advocates on Advanced Therapy Medicinal Products
title_full Perspectives, Expectations, and Concerns of European Patient Advocates on Advanced Therapy Medicinal Products
title_fullStr Perspectives, Expectations, and Concerns of European Patient Advocates on Advanced Therapy Medicinal Products
title_full_unstemmed Perspectives, Expectations, and Concerns of European Patient Advocates on Advanced Therapy Medicinal Products
title_short Perspectives, Expectations, and Concerns of European Patient Advocates on Advanced Therapy Medicinal Products
title_sort perspectives, expectations, and concerns of european patient advocates on advanced therapy medicinal products
topic Medicine
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8649896/
https://www.ncbi.nlm.nih.gov/pubmed/34888320
http://dx.doi.org/10.3389/fmed.2021.728529
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