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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
Objectives: the objective of this study was to establish the preferences of patients 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 patients’ preferences regarding a number of outcomes: foot and ankle pain, fatigue, mobility, ability to perform activities of daily living, and choice of footwear. Preferences were also gathered for different schedules and frequency of delivery for the health and fitness intervention. The initial outcomes were chosen based on literature review, clinician recommendation and patients’ focus groups findings. The DCE was constructed using the D-efficiency criteria with SAS software macros. The DCE compared hypothetical scenarios with varying levels of outcomes severity as well as the schedule of the intervention. Preference weights were estimated using a number of econometric models (conditional logit, mixed logit and generalized multinomial logit) which are based on different assumptions, to account for preference and scale heterogeneity. The attribute importance was established by using the partial log-likelihood method.

Results: 100 patients with RA completed 18 choice sets via a web based platform. Overall, patients 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.
1098-3015
A537
Stamuli, E.
5962a60c-9d0a-4882-b7cd-ac85cf9f0314
Torgerson, D.
60ef71a7-dac0-42bd-b6a8-bf3ae1b2e9f5
Northgraves, M.
371f01af-a326-4f29-abfb-8a9e07f363b5
Ronaldson, S.
45a9301c-67fd-449c-807a-91fa5ea65f05
Cherry, Lindsey
95256156-ce8c-4e7c-b04d-b6e459232441
Stamuli, E.
5962a60c-9d0a-4882-b7cd-ac85cf9f0314
Torgerson, D.
60ef71a7-dac0-42bd-b6a8-bf3ae1b2e9f5
Northgraves, M.
371f01af-a326-4f29-abfb-8a9e07f363b5
Ronaldson, S.
45a9301c-67fd-449c-807a-91fa5ea65f05
Cherry, Lindsey
95256156-ce8c-4e7c-b04d-b6e459232441

Stamuli, E., Torgerson, D., Northgraves, M., Ronaldson, S. and Cherry, Lindsey (2017) Identifying the primary outcome for a randomised controlled trial in rheumatoid arthritis: the role of a discrete choice experiment. Value in Health, 20 (9), A537, [PMS63]. (doi:10.1016/j.jval.2017.08.785).

Record type: Meeting abstract

Abstract

Objectives: the objective of this study was to establish the preferences of patients 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 patients’ preferences regarding a number of outcomes: foot and ankle pain, fatigue, mobility, ability to perform activities of daily living, and choice of footwear. Preferences were also gathered for different schedules and frequency of delivery for the health and fitness intervention. The initial outcomes were chosen based on literature review, clinician recommendation and patients’ focus groups findings. The DCE was constructed using the D-efficiency criteria with SAS software macros. The DCE compared hypothetical scenarios with varying levels of outcomes severity as well as the schedule of the intervention. Preference weights were estimated using a number of econometric models (conditional logit, mixed logit and generalized multinomial logit) which are based on different assumptions, to account for preference and scale heterogeneity. The attribute importance was established by using the partial log-likelihood method.

Results: 100 patients with RA completed 18 choice sets via a web based platform. Overall, patients 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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Identifying The Primary Outcome For A Randomised Controlled Trial In Rheumatoid Arthritis: The Role of A Discrete Choice Experiment - Version of Record
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Published date: 20 October 2017

Identifiers

Local EPrints ID: 416219
URI: http://eprints.soton.ac.uk/id/eprint/416219
ISSN: 1098-3015
PURE UUID: 27b7eea7-764b-4d02-9522-dbedcc0419ee
ORCID for Lindsey Cherry: ORCID iD orcid.org/0000-0002-3165-1004

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Date deposited: 08 Dec 2017 17:30
Last modified: 22 Nov 2021 02:55

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Contributors

Author: E. Stamuli
Author: D. Torgerson
Author: M. Northgraves
Author: S. Ronaldson
Author: Lindsey Cherry ORCID iD

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