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

G.S. Gorman; A.C. Jimenez-Moreno; C.A. Pinto; B. Levitan; C. Whichello; C. Dyer; E. Van Overbeeke; E. de Bekker-Grob; I. Smith; I. Huys; J. Viberg Johansson; K. Adcock; K. Bullock; V. Soekhai; Z. Yuan; H. Lochmuller; A. de Wit

doi:10.12688/wellcomeopenres.16116.1

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

G.S. Gorman, A.C. Jimenez-Moreno, C.A. Pinto, B. Levitan, C. Whichello, C. Dyer, E. Van Overbeeke, E. de Bekker-Grob, I. Smith, I. Huys, J. Viberg Johansson, K. Adcock, K. Bullock, V. Soekhai, Z. Yuan, H. Lochmuller, A. de Wit

Research output: Contribution to Journal › Article › Academic › peer-review

Abstract

© 2020 Jimenez-Moreno AC et al.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.

Original language	English
Article number	253
Journal	Wellcome Open Research
Volume	5
DOIs	https://doi.org/10.12688/wellcomeopenres.16116.1
Publication status	Published - 2020
Externally published	Yes

Funding

We thank all patient organizations supporting this study: MDUK, MDSG; Cure DM CIC; the Lily Foundation for mitochondrial disorders; UMDF, MitoCanada; MDC; MDA; MDF; and, MDNZ. We thank members of the Wellcome Research Centre for Mitochondrial Research that provided input and expertise into the design of this study. We thank the UK Myotonic Dystrophy Patient Registry at Newcastle University for facilitating participants recruitment and supporting with the pilot stages of the study.We thank Newcastle University Ethics Committee for revising the ethical sound of this project.

Funders	Funder number
Cure DM CIC
Lily Foundation
MDNZ
UK Myotonic Dystrophy
United Mitochondrial Disease Foundation
Muscular Dystrophy UK
Newcastle University

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Gorman, G. S., Jimenez-Moreno, A. C., Pinto, C. A., Levitan, B., Whichello, C., Dyer, C., Van Overbeeke, E., de Bekker-Grob, E., Smith, I., Huys, I., Viberg Johansson, J., Adcock, K., Bullock, K., Soekhai, V., Yuan, Z., Lochmuller, H., & de Wit, A. (2020). A study protocol for quantifying patient preferences in neuromuscular disorders: A case study of the IMI PREFER Project. Wellcome Open Research, 5, Article 253. https://doi.org/10.12688/wellcomeopenres.16116.1

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title = "A study protocol for quantifying patient preferences in neuromuscular disorders: A case study of the IMI PREFER Project",

abstract = "{\textcopyright} 2020 Jimenez-Moreno AC et al.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.",

author = "G.S. Gorman and A.C. Jimenez-Moreno and C.A. Pinto and B. Levitan and C. Whichello and C. Dyer and {Van Overbeeke}, E. and {de Bekker-Grob}, E. and I. Smith and I. Huys and {Viberg Johansson}, J. and K. Adcock and K. Bullock and V. Soekhai and Z. Yuan and H. Lochmuller and {de Wit}, A.",

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journal = "Wellcome Open Research",

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Gorman, GS, Jimenez-Moreno, AC, Pinto, CA, Levitan, B, Whichello, C, Dyer, C, Van Overbeeke, E, de Bekker-Grob, E, Smith, I, Huys, I, Viberg Johansson, J, Adcock, K, Bullock, K, Soekhai, V, Yuan, Z, Lochmuller, H & de Wit, A 2020, 'A study protocol for quantifying patient preferences in neuromuscular disorders: A case study of the IMI PREFER Project', Wellcome Open Research, vol. 5, 253. https://doi.org/10.12688/wellcomeopenres.16116.1

TY - JOUR

T1 - A study protocol for quantifying patient preferences in neuromuscular disorders

T2 - A case study of the IMI PREFER Project

AU - Gorman, G.S.

AU - Jimenez-Moreno, A.C.

AU - Pinto, C.A.

AU - Levitan, B.

AU - Whichello, C.

AU - Dyer, C.

AU - Van Overbeeke, E.

AU - de Bekker-Grob, E.

AU - Smith, I.

AU - Huys, I.

AU - Viberg Johansson, J.

AU - Adcock, K.

AU - Bullock, K.

AU - Soekhai, V.

AU - Yuan, Z.

AU - Lochmuller, H.

AU - de Wit, A.

PY - 2020

Y1 - 2020

N2 - © 2020 Jimenez-Moreno AC et al.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.

AB - © 2020 Jimenez-Moreno AC et al.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.

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DO - 10.12688/wellcomeopenres.16116.1

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JO - Wellcome Open Research

JF - Wellcome Open Research

M1 - 253

ER -

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

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