A study protocol for quantifying patient preferences in neuromuscular disorders: a case study of the IMI PREFER Project

Wellcome Open Research

article

Aura Cecilia Jimenez-Moreno¹  Cathy Anne Pinto³  Bennett Levitan⁴  Chiara Whichello⁵  Christine Dyer¹  Eline Van Overbeeke⁶  Esther de Bekker-Grob⁵  Ian Smith⁷  Isabelle Huys⁶  Jennifer Viberg Johansson⁸  Kate Adcock⁹  Kristin Bullock^1,10  Vikas Soekhai⁵  Zhong Yuan⁴  Hanns Lochmuller^1,11  Ardine de Wit⁷  Grainne S. Gorman¹ 

[1] Wellcome Centre for Mitochondrial Research, Newcastle University;Patient Centered Research;Pharmacoepidemiology Department, Centre for Observational and Realworld Evidence, Merck & Co, Inc.;Department of Epidemiology, Janssen Research & Development;Erasmus School of Health Policy & Management and Erasmus Choice Modelling Centre, Erasmus University Rotterdam;Department of Clinical Pharmacology and Pharmacotherapy, University of Leuven;Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht;Centre for Research Ethics & Bioethics, Uppsala universitet;Muscular Dystrophy UK;Global Patient Safety Department;Brain and Mind Research Institute, University of Ottawa

关键词: Myotonic Dystrophy;    mitochondrial disease;    risk tolerance;    Best Worst Scaling;    Discrete Choice Experiment;    Q-methodology;    patient preferences;    IMI-PREFER;    treatment preferences.;   

DOI  :  10.12688/wellcomeopenres.16116.1

学科分类：内科医学

来源: Wellcome

【 摘 要 】

Objectives: Patient preference studies are increasingly used to inform decision-making during the medical product lifecycle but are rarely used to inform early stages of drug development.  The primary aim of this study is to quantify treatment preferences of patients with neuromuscular disorders, which represent serious and debilitating conditions with limited or no treatment options available.Methods: This quantitative patient preferences study was designed as an online survey, with a cross-over design.  This study will target two different diseases from the neuromuscular disorders disease group, myotonic dystrophy type 1 (DM1) and mitochondrial myopathies (MM). Despite having different physio-pathological pathways both DM1 and MM manifest in a clinically similar manner and may benefit from similar treatment options.  The sample will be stratified into three subgroups: two patient groups differentiated by age of symptom onset and one caregivers group.   Each subgroup will be randomly assigned to complete two of three different preference elicitation methods at two different time points: Q-methodology survey, discrete choice experiment, and best-worst scaling type 2, allowing cross-comparisons of the results across each study time within participants and within elicitation methods. Additional variables such as sociodemographic, clinical and health literacy will be collected to enable analysis of potential heterogeneity.Ethics and Dissemination: This study protocol has undergone ethical review and approval by the Newcastle University R&D Ethics Committee (Ref: 15169/2018). All participants will be invited to give electronic informed consent to take part in the study prior accessing the online survey. All electronic data will be anonymised prior analysis. This study is part of the Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (IMI-PREFER) project, a public-private collaborative research project aiming to develop expert and evidence-based recommendations on how and when patient preferences can be assessed and used to inform medical product decision making.

【 授权许可】

CC BY