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What do patients with unmet medical needs want? A qualitative study of patients’ views and experiences with expanded access to unapproved, investigational treatments in the Netherlands

BACKGROUND: Patients with unmet medical needs sometimes resort to non-standard treatment options, including the use of unapproved, investigational drugs in the context of clinical trials, compassionate use or named-patient programs. The views and experiences of patients with unmet medical needs rega...

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Autores principales: Bunnik, Eline M., Aarts, Nikkie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6842468/
https://www.ncbi.nlm.nih.gov/pubmed/31706313
http://dx.doi.org/10.1186/s12910-019-0420-8
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author Bunnik, Eline M.
Aarts, Nikkie
author_facet Bunnik, Eline M.
Aarts, Nikkie
author_sort Bunnik, Eline M.
collection PubMed
description BACKGROUND: Patients with unmet medical needs sometimes resort to non-standard treatment options, including the use of unapproved, investigational drugs in the context of clinical trials, compassionate use or named-patient programs. The views and experiences of patients with unmet medical needs regarding unapproved, investigational drugs have not yet been examined empirically. METHODS: In this qualitative study, exploratory interviews and focus groups were held with patients with chronic or life-threatening diseases (n = 39), about topics related to non-standard treatment options, such as the search for non-standard treatment options, patients’ views of the moral obligations of doctors, and the conditions under which they would or would not wish to use non-standard treatment options, including expanded access to unapproved, investigational drugs. RESULTS: Respondents had very little knowledge about and/or experience with existing opportunities for expanded access to investigational drugs, although some respondents were actively looking for non-standard treatment options. They had high expectations of their treating physicians, assuming them to be aware of non-standard treatment options, including clinical trials elsewhere and expanded access programs, and assuming that they would inform their patients about such options. Respondents carefully weighed the risks and potential benefits of pursuing expanded access, citing concerns related to the scientific evidence of the safety and efficacy of the drug, side effects, drug-drug interactions, and the maintaining of good quality of life. Respondents stressed the importance of education and assertiveness to obtain access to good-quality health care, and were willing to pay out of pocket for investigational drugs. Patients expressed concerns about equal access to new and/or non-standard treatment options. CONCLUSION: When the end of a standard treatment trajectory comes into view, patients may prefer that treating physicians discuss non-standard treatment options with them, including opportunities for expanded access to unapproved, investigational drugs. Although our respondents had varying levels of understanding of expanded access programs, they seemed capable of making well-considered choices with regard to non-standard treatment options and had realistic expectations with regard to the safety and efficacy of such options. Dutch patients might be less likely to fall prey to false hope than often presumed.
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spelling pubmed-68424682019-11-14 What do patients with unmet medical needs want? A qualitative study of patients’ views and experiences with expanded access to unapproved, investigational treatments in the Netherlands Bunnik, Eline M. Aarts, Nikkie BMC Med Ethics Research Article BACKGROUND: Patients with unmet medical needs sometimes resort to non-standard treatment options, including the use of unapproved, investigational drugs in the context of clinical trials, compassionate use or named-patient programs. The views and experiences of patients with unmet medical needs regarding unapproved, investigational drugs have not yet been examined empirically. METHODS: In this qualitative study, exploratory interviews and focus groups were held with patients with chronic or life-threatening diseases (n = 39), about topics related to non-standard treatment options, such as the search for non-standard treatment options, patients’ views of the moral obligations of doctors, and the conditions under which they would or would not wish to use non-standard treatment options, including expanded access to unapproved, investigational drugs. RESULTS: Respondents had very little knowledge about and/or experience with existing opportunities for expanded access to investigational drugs, although some respondents were actively looking for non-standard treatment options. They had high expectations of their treating physicians, assuming them to be aware of non-standard treatment options, including clinical trials elsewhere and expanded access programs, and assuming that they would inform their patients about such options. Respondents carefully weighed the risks and potential benefits of pursuing expanded access, citing concerns related to the scientific evidence of the safety and efficacy of the drug, side effects, drug-drug interactions, and the maintaining of good quality of life. Respondents stressed the importance of education and assertiveness to obtain access to good-quality health care, and were willing to pay out of pocket for investigational drugs. Patients expressed concerns about equal access to new and/or non-standard treatment options. CONCLUSION: When the end of a standard treatment trajectory comes into view, patients may prefer that treating physicians discuss non-standard treatment options with them, including opportunities for expanded access to unapproved, investigational drugs. Although our respondents had varying levels of understanding of expanded access programs, they seemed capable of making well-considered choices with regard to non-standard treatment options and had realistic expectations with regard to the safety and efficacy of such options. Dutch patients might be less likely to fall prey to false hope than often presumed. BioMed Central 2019-11-09 /pmc/articles/PMC6842468/ /pubmed/31706313 http://dx.doi.org/10.1186/s12910-019-0420-8 Text en © The Author(s). 2019 Open AccessThis article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
spellingShingle Research Article
Bunnik, Eline M.
Aarts, Nikkie
What do patients with unmet medical needs want? A qualitative study of patients’ views and experiences with expanded access to unapproved, investigational treatments in the Netherlands
title What do patients with unmet medical needs want? A qualitative study of patients’ views and experiences with expanded access to unapproved, investigational treatments in the Netherlands
title_full What do patients with unmet medical needs want? A qualitative study of patients’ views and experiences with expanded access to unapproved, investigational treatments in the Netherlands
title_fullStr What do patients with unmet medical needs want? A qualitative study of patients’ views and experiences with expanded access to unapproved, investigational treatments in the Netherlands
title_full_unstemmed What do patients with unmet medical needs want? A qualitative study of patients’ views and experiences with expanded access to unapproved, investigational treatments in the Netherlands
title_short What do patients with unmet medical needs want? A qualitative study of patients’ views and experiences with expanded access to unapproved, investigational treatments in the Netherlands
title_sort what do patients with unmet medical needs want? a qualitative study of patients’ views and experiences with expanded access to unapproved, investigational treatments in the netherlands
topic Research Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6842468/
https://www.ncbi.nlm.nih.gov/pubmed/31706313
http://dx.doi.org/10.1186/s12910-019-0420-8
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