Patient Preferences in Rare Diseases: A Qualitative Study in Neuromuscular Disorders to Inform a Quantitative Preference Study (Feb, 10.1007/s40271-020-00482-z, 2021)

被引:0
|
作者
Jimenez-Moreno, A. Cecilia [1 ,2 ]
van Overbeeke, Eline [3 ]
Pinto, Cathy Anne [4 ]
Smith, Ian [5 ]
Sharpe, Jenny [6 ]
Ormrod, James [7 ]
Whichello, Chiara [8 ,9 ]
de Bekker-Grob, Esther W. [8 ,9 ]
Bullok, Kristin [10 ]
Levitan, Bennett [11 ]
Huys, Isabelle [3 ]
de Wit, G. Ardine [5 ]
Gorman, Grainne [1 ]
机构
[1] Newcastle Univ, Translat & Clin Res Inst, Newcastle Upon Tyne, Tyne & Wear, England
[2] Evidera, London, England
[3] Univ Leuven, Clin Pharmacol & Pharmacotherapy, Leuven, Belgium
[4] Merck & Co Inc, Pharmacoepidemiol Dept, Ctr Observat & Real World Evidence, Rahway, NJ 07065 USA
[5] Univ Utrecht, Univ Med Ctr Utrecht, Julius Ctr Hlth Sci & Primary Care, Utrecht, Netherlands
[6] Muscular Dystrophy UK, London, England
[7] Univ Brighton, Sch Appl Social Sci, Brighton, E Sussex, England
[8] Erasmus Univ, Erasmus Sch Hlth Policy & Management, Rotterdam, Netherlands
[9] Erasmus Univ, Erasmus Choice Modelling Ctr, Rotterdam, Netherlands
[10] Eli Lilly & Co, Global Patient Safety Dept, Indianapolis, IN 46285 USA
[11] Janssen Res & Dev, Titusville, NJ USA
来源
PATIENT-PATIENT CENTERED OUTCOMES RESEARCH | 2021年 / 14卷 / 05期
基金
英国惠康基金; 欧盟地平线“2020”;
关键词
D O I
10.1007/s40271-021-00523-1
中图分类号
R19 [保健组织与事业(卫生事业管理)];
学科分类号
摘要
Introduction: It 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. Methods: Fifteen 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. Results: A 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. Conclusions: This 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. © 2021, The Author(s).
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页码:693 / 693
页数:1
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  • [1] Correction to: Patient Preferences in Rare Diseases: A Qualitative Study in Neuromuscular Disorders to Inform a Quantitative Preference Study
    A. Cecilia Jimenez-Moreno
    Eline van Overbeeke
    Cathy Anne Pinto
    Ian Smith
    Jenny Sharpe
    James Ormrod
    Chiara Whichello
    Esther W. de Bekker-Grob
    Kristin Bullok
    Bennett Levitan
    Isabelle Huys
    G. Ardine de Wit
    Grainne Gorman
    The Patient - Patient-Centered Outcomes Research, 2021, 14 : 693 - 693