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Identifying the primary outcome for a randomised controlled trial in rheumatoid arthritis: the role of a discrete choice experiment

Identifying the primary outcome for a randomised controlled trial in rheumatoid arthritis: the role of a discrete choice experiment
Identifying the primary outcome for a randomised controlled trial in rheumatoid arthritis: the role of a discrete choice experiment
Background: this study sought to establish the preferences of people with Rheumatoid Arthritis (RA) about the best outcome measure for a health and fitness intervention randomised controlled trial (RCT). The results of this study were used to inform the choice of the trial primary and secondary outcome measure.

Methods: a discrete choice experiment (DCE) was used to assess people’s preferences regarding a number of outcomes (foot and ankle pain, fatigue, mobility, ability to perform daily activities, choice of footwear) as well as different schedules and frequency of delivery for the health and fitness intervention. The outcomes were chosen based on literature review, clinician recommendation and patients’ focus groups. The DCE was constructed in SAS software using the D-efficiency criteria. It compared hypothetical scenarios with varying levels of outcomes severity and intervention schedule. Preference weights were estimated using appropriate econometric models. The partial log-likelihood method was used to assess the attribute importance.

Results: one hundred people with RA completed 18 choice sets. Overall, people selected foot and ankle pain as the most important outcome, with mobility being nearly as important. There was no evidence of differential preference between intervention schedules or frequency of delivery.

Conclusions: foot and ankle pain can be considered the patient choice for primary outcome of an RCT relating to a health and fitness intervention. This study demonstrated that, by using the DCE method, it is possible to incorporate patients’ preferences at the design stage of a RCT. This approach ensures patient involvement at early stages of health care design.
1757-1146
Stamuli, Eugena
e9e2117a-e4aa-4ecb-b9e8-1a933d75f74d
Torgerson, David
3a062e3b-b4e2-40a1-bdf0-7c1541796de2
Northgraves, Matthew
13763629-4b1e-43dd-a311-12737f3b02d5
Ronaldson, Sarah
ee507e59-443e-40e3-a709-552483f787c6
Cherry, Lindsey
95256156-ce8c-4e7c-b04d-b6e459232441
Stamuli, Eugena
e9e2117a-e4aa-4ecb-b9e8-1a933d75f74d
Torgerson, David
3a062e3b-b4e2-40a1-bdf0-7c1541796de2
Northgraves, Matthew
13763629-4b1e-43dd-a311-12737f3b02d5
Ronaldson, Sarah
ee507e59-443e-40e3-a709-552483f787c6
Cherry, Lindsey
95256156-ce8c-4e7c-b04d-b6e459232441

Stamuli, Eugena, Torgerson, David, Northgraves, Matthew, Ronaldson, Sarah and Cherry, Lindsey (2017) Identifying the primary outcome for a randomised controlled trial in rheumatoid arthritis: the role of a discrete choice experiment. Journal of Foot and Ankle Research, 10 (1), [57]. (doi:10.1186/s13047-017-0240-3).

Record type: Article

Abstract

Background: this study sought to establish the preferences of people with Rheumatoid Arthritis (RA) about the best outcome measure for a health and fitness intervention randomised controlled trial (RCT). The results of this study were used to inform the choice of the trial primary and secondary outcome measure.

Methods: a discrete choice experiment (DCE) was used to assess people’s preferences regarding a number of outcomes (foot and ankle pain, fatigue, mobility, ability to perform daily activities, choice of footwear) as well as different schedules and frequency of delivery for the health and fitness intervention. The outcomes were chosen based on literature review, clinician recommendation and patients’ focus groups. The DCE was constructed in SAS software using the D-efficiency criteria. It compared hypothetical scenarios with varying levels of outcomes severity and intervention schedule. Preference weights were estimated using appropriate econometric models. The partial log-likelihood method was used to assess the attribute importance.

Results: one hundred people with RA completed 18 choice sets. Overall, people selected foot and ankle pain as the most important outcome, with mobility being nearly as important. There was no evidence of differential preference between intervention schedules or frequency of delivery.

Conclusions: foot and ankle pain can be considered the patient choice for primary outcome of an RCT relating to a health and fitness intervention. This study demonstrated that, by using the DCE method, it is possible to incorporate patients’ preferences at the design stage of a RCT. This approach ensures patient involvement at early stages of health care design.

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More information

Accepted/In Press date: 28 November 2017
e-pub ahead of print date: 15 December 2017
Published date: 15 December 2017

Identifiers

Local EPrints ID: 435683
URI: http://eprints.soton.ac.uk/id/eprint/435683
ISSN: 1757-1146
PURE UUID: 42f44122-d1ca-4226-b0d8-32350403a09d
ORCID for Lindsey Cherry: ORCID iD orcid.org/0000-0002-3165-1004

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Date deposited: 18 Nov 2019 17:30
Last modified: 13 Nov 2021 02:46

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Contributors

Author: Eugena Stamuli
Author: David Torgerson
Author: Matthew Northgraves
Author: Sarah Ronaldson
Author: Lindsey Cherry ORCID iD

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