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Permanent link (DOI): https://doi.org/10.7939/R33X83X5W

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Patient Involvement in the Regulatory Process and Rare Disease Patient Perceptions of Treatment Benefits and Harms Open Access

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Other title
Subject/Keyword
Patient Involvement
Benefits and Harms
Regulatory Process
Rare Diseases
Orphan Drugs
Type of item
Thesis
Degree grantor
University of Alberta
Author or creator
Lindsay-Bellows, Mandy C
Supervisor and department
Dr. Dev Menon (Department of Public Health Sciences), University of Alberta
Dr. Jackie Street (School of Population Health), University of Adelaide
Dr. Tania Stafinski (Department of Public Health Sciences), University of Alberta
Examining committee member and department
Dr. Alun Edwards, University of Calgary
Dr. Tania Stafinski (Department of Public Health Sciences), University of Alberta
Dr. Jackie Street (School of Population Health), University of Adelaide
Dr. Dev Menon (Department of Public Health Sciences), University of Alberta
Department
Department of Public Health Sciences
Specialization
Health Policy Research
Date accepted
2015-09-28T15:44:27Z
Graduation date
2015-11
Degree
Master of Science
Degree level
Master's
Abstract
Patient involvement in healthcare decision-making is becoming an essential part of healthcare policy in today’s patient centred environment. It supports responsive and transparent healthcare programs and policies that are informed by patients for patients. While regulatory agencies, the bodies responsible for the approval of new medicines, involve patients in the regulatory process, little is known about the involvement context or the type of patients engaged. The purpose of this thesis was to explore patient involvement within the regulatory process and gain insights into rare disease patient perceptions of treatment benefits and harms. It contains three papers. The first comprises a review of proposed and current regulatory patient involvement using the International Association for Public Participation Spectrum. The second paper presents findings from three fora and surveys conducted in three different Canadian cities. The fora were used to elicit treatment harm and benefit attributes and treatment benefit priorities from rare disease patients and caregivers. Surveys were used to gather patients’ and caregivers’ levels of expectations of treatment benefit. Their input highlighted the need for survey questions to be relevant and meaningful to health contexts of the target population. The third paper, informed by the second paper, provides an understanding of harm acceptance while considering increasing levels of treatment benefit in a specific rare disease. It was found that mucopolysaccharidosis patients accept lower levels of harm than caregivers, where caregivers consistently selected the maximum level of harm for maximum treatment benefit. The findings of all three papers demonstrate that patient input around the acceptability of different benefit-harm trade-offs is needed in order to make regulatory decisions more patient-centred.
Language
English
DOI
doi:10.7939/R33X83X5W
Rights
Permission is hereby granted to the University of Alberta Libraries to reproduce single copies of this thesis and to lend or sell such copies for private, scholarly or scientific research purposes only. The author reserves all other publication and other rights in association with the copyright in the thesis and, except as herein before provided, neither the thesis nor any substantial portion thereof may be printed or otherwise reproduced in any material form whatsoever without the author's prior written permission.
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