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Patient Preferences...
Patient Preferences in Rare Diseases: A Qualitative Study in Neuromuscular Disorders to Inform a Quantitative Preference Study
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- Jimenez-Moreno, A. Cecilia (author)
- Translational and Clinical Research Institute, Newcastle University, Newcastle-Upon-Tyne, UK; Evidera, London, UK
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- van Overbeeke, Eline (author)
- Clinical Pharmacology and Pharmacotherapy, University of Leuven, Leuven, Belgium
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- Pinto, Cathy Anne (author)
- Pharmacoepidemiology Department, Center for Observational and Real-world Evidence, Merck & Co, Inc., Rahway, NJ, USA
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- Smith, Ian (author)
- Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands
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- Sharpe, Jenny (author)
- Muscular Dystrophy UK, London, UK
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- Ormrod, James (author)
- School of Applied Social Science, University of Brighton, East Sussex, UK
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- de Bekker-Grob, Esther W. (author)
- Erasmus School of Health Policy and Management, and Erasmus Choice Modelling Centre, Erasmus University, Rotterdam, The Netherlands
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- Bullok, Kristin (author)
- Global Patient Safety Department, Eli Lilly & Co., Indianapolis, IN, USA
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- Levitan, Bennet (author)
- Janssen Research and Development, Titusville, NJ, USA
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- Huys, Isabelle (author)
- Clinical Pharmacology and Pharmacotherapy, University of Leuven, Leuven, Belgium
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- de Wit, G. Ardine (author)
- Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands
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- Gorman, Grainne (author)
- Translational and Clinical Research Institute, Newcastle University, Newcastle-Upon-Tyne, UK
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(creator_code:org_t)
- 2021-02-27
- 2021
- English.
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In: Patient. - : Springer Science and Business Media LLC. - 1178-1653 .- 1178-1661.
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https://doi.org/10.1...
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https://urn.kb.se/re...
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Abstract
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- IntroductionIt has become increasingly important to include patient preference information in decision-making processes for drug development. As neuromuscular disorders represent multisystem, debilitating, and progressive rare diseases with few treatment options, this study aimed to explore unmet health care needs and patient treatment preferences for two neuromuscular disorders, myotonic dystrophy type 1 (DM1) and mitochondrial myopathies (MM) to inform early stages of drug development.MethodsFifteen semi-structured interviews and five focus group discussions (FGDs) were held with DM1 and MM adult patients and caregivers. Topics discussed included (1) reasons for study participation; (2) disease signs/symptoms and their impact on daily lives; (3) top desired benefits; and (4) acceptability of risks and tolerance levels for a hypothetical new treatment. Data were analyzed following a thematic ‘code’ approach.ResultsA total of 52 participants representing a wide range of disease severities participated. ‘Muscle strength’ and ‘energy and endurance’ were the disease-related unmet needs most often mentioned. Additionally, improved ‘balance’, ‘cognition’ and ‘gut function’ were the top desired treatment benefits, while ‘damage to the liver, kidneys or eyes’ was the most concerning risk. Factors influencing their tolerance to risks related to previously having experienced the risk and differentiation between permanent and temporary risks. A few differences were elicited between patients and caregivers.ConclusionsThis qualitative study provided an open forum to elicit treatment-desired benefits and acceptable risks to be established by patients themselves. These findings can inform decisions for developing new treatments and the design of clinical trials for DM1 and MM.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Medicinska och farmaceutiska grundvetenskaper -- Samhällsfarmaci och klinisk farmaci (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Basic Medicine -- Social and Clinical Pharmacy (hsv//eng)
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Jimenez-Moreno, ...
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van Overbeeke, E ...
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Pinto, Cathy Ann ...
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Smith, Ian
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Sharpe, Jenny
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Ormrod, James
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de Bekker-Grob, ...
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Bullok, Kristin
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Levitan, Bennet
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Huys, Isabelle
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de Wit, G. Ardin ...
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Gorman, Grainne
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Uppsala University