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Patient reported outcome measures in rare diseases: a narrative review

BACKGROUND: Rare diseases can lead to a significant reduction in quality of life for patients and their families. Ensuring the patients voice is central to clinical decision making is key to delivering, evaluating and understanding the efficacy of therapeutic interventions. Patient reported outcome...

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Autores principales: Slade, Anita, Isa, Fatima, Kyte, Derek, Pankhurst, Tanya, Kerecuk, Larissa, Ferguson, James, Lipkin, Graham, Calvert, Melanie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5914068/
https://www.ncbi.nlm.nih.gov/pubmed/29688860
http://dx.doi.org/10.1186/s13023-018-0810-x
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author Slade, Anita
Isa, Fatima
Kyte, Derek
Pankhurst, Tanya
Kerecuk, Larissa
Ferguson, James
Lipkin, Graham
Calvert, Melanie
author_facet Slade, Anita
Isa, Fatima
Kyte, Derek
Pankhurst, Tanya
Kerecuk, Larissa
Ferguson, James
Lipkin, Graham
Calvert, Melanie
author_sort Slade, Anita
collection PubMed
description BACKGROUND: Rare diseases can lead to a significant reduction in quality of life for patients and their families. Ensuring the patients voice is central to clinical decision making is key to delivering, evaluating and understanding the efficacy of therapeutic interventions. Patient reported outcome measures (PROMs) are used to capture the patient’s views about their health status and facilitate our understanding of the impact of these diseases and their treatments on patient’s quality of life and symptoms. MAIN TEXT: This review explores some of the current issues around the utilisation of PROMs in rare diseases, including small patient populations and dearth of valid PROMs. Difficulties in validating new or current PROMs for use in clinical trials and research are discussed. The review highlights potential solutions for some of the issues outlined in the review and the implementation of PROMs in research and clinical practice are discussed. CONCLUSION: Patient input throughout the development of PROMs including qualitative research is essential to ensure that outcomes that matter to people living with rare disease are appropriately captured. Given the large number of rare diseases, small numbers of patients living with each condition and the cost of instrument development, creative and pragmatic solutions to PROM development and use may be necessary. Solutions include qualitative interviews, modern psychometrics and resources such as item banking and computer adaptive testing. Use of PROMs in rare disease research and clinical practice offers the potential to improve patient care and clinical outcomes.
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spelling pubmed-59140682018-04-30 Patient reported outcome measures in rare diseases: a narrative review Slade, Anita Isa, Fatima Kyte, Derek Pankhurst, Tanya Kerecuk, Larissa Ferguson, James Lipkin, Graham Calvert, Melanie Orphanet J Rare Dis Review BACKGROUND: Rare diseases can lead to a significant reduction in quality of life for patients and their families. Ensuring the patients voice is central to clinical decision making is key to delivering, evaluating and understanding the efficacy of therapeutic interventions. Patient reported outcome measures (PROMs) are used to capture the patient’s views about their health status and facilitate our understanding of the impact of these diseases and their treatments on patient’s quality of life and symptoms. MAIN TEXT: This review explores some of the current issues around the utilisation of PROMs in rare diseases, including small patient populations and dearth of valid PROMs. Difficulties in validating new or current PROMs for use in clinical trials and research are discussed. The review highlights potential solutions for some of the issues outlined in the review and the implementation of PROMs in research and clinical practice are discussed. CONCLUSION: Patient input throughout the development of PROMs including qualitative research is essential to ensure that outcomes that matter to people living with rare disease are appropriately captured. Given the large number of rare diseases, small numbers of patients living with each condition and the cost of instrument development, creative and pragmatic solutions to PROM development and use may be necessary. Solutions include qualitative interviews, modern psychometrics and resources such as item banking and computer adaptive testing. Use of PROMs in rare disease research and clinical practice offers the potential to improve patient care and clinical outcomes. BioMed Central 2018-04-23 /pmc/articles/PMC5914068/ /pubmed/29688860 http://dx.doi.org/10.1186/s13023-018-0810-x Text en © The Author(s). 2018 Open Access This 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 Review
Slade, Anita
Isa, Fatima
Kyte, Derek
Pankhurst, Tanya
Kerecuk, Larissa
Ferguson, James
Lipkin, Graham
Calvert, Melanie
Patient reported outcome measures in rare diseases: a narrative review
title Patient reported outcome measures in rare diseases: a narrative review
title_full Patient reported outcome measures in rare diseases: a narrative review
title_fullStr Patient reported outcome measures in rare diseases: a narrative review
title_full_unstemmed Patient reported outcome measures in rare diseases: a narrative review
title_short Patient reported outcome measures in rare diseases: a narrative review
title_sort patient reported outcome measures in rare diseases: a narrative review
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5914068/
https://www.ncbi.nlm.nih.gov/pubmed/29688860
http://dx.doi.org/10.1186/s13023-018-0810-x
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