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Assessing Preference-Based Outcome Measures for Overactive Bladder: An Evaluation of Patient-Reported Outcome Data from the BESIDE Clinical Trial

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Abstract

Objectives

The aim of this study was to compare outcomes using two preference-based measures of health status (EQ-5D-5L and OAB-5D) in patients with overactive bladder (OAB) treated with solifenacin plus mirabegron or solifenacin monotherapy in the BESIDE trial.

Methods

Patients with OAB who remained incontinent after 4 weeks’ treatment with solifenacin 5 mg were randomized 1:1:1 to combination treatment (solifenacin 5 mg plus mirabegron [25 mg for the first 4 weeks/50 mg for the last 8 weeks]), solifenacin 5 mg, or solifenacin 10 mg. EQ-5D-5L and OAB-q were administered at baseline, weeks 4, 8, 12, and end of treatment (EoT). OAB-5D scores were derived from OAB-q results. Responder analysis was carried out using several definitions of minimally important difference.

Results

A total of 2054 patients received one or more doses of study treatment (combination, n = 694; solifenacin 5 mg, n = 684; solifenacin 10 mg, n = 676). EQ-5D-5L Index mean score changes (from baseline to EoT) were greater with combination (0.059) than with solifenacin 5 mg (0.040) and 10 mg (0.044) monotherapy, but the differences were not statistically significant. A significantly greater improvement was observed for combination on OAB-5D (0.107 vs 0.085 for 5 mg, and 0.087 for 10 mg; p ≤ 0.01). The dimensions most improved overall were anxiety/depression, pain/discomfort, and usual activities on EQ-5D-5L, and urge, urine loss, and coping on OAB-5D. The proportion of responders was significantly greater with combination compared with monotherapy using OAB-5D only.

Conclusions

Improvements were observed in all study arms on both the EQ-5D-5L and OAB-5D, although only the OAB-5D showed a statistically significant benefit for combination versus solifenacin monotherapy. Combining generic and condition-specific preference-based health status measures allowed for assessment of dimensions that were particularly relevant to this patient population, while permitting comparison with outcomes from other studies, treatments, and populations via EQ-5D.

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Author information

Authors and Affiliations

  1. The Office of Health Economics, Southside, 7th Floor, 105 Victoria Street, London, SW1E 6QT, UK

    Mike Herdman & Nancy Devlin

  2. Astellas Pharma Europe Ltd., Astellas Medical Affairs, EMEA, HEOR, Chertsey, UK

    Jameel Nazir, Emad Siddiqui & Moses Huang

  3. Astellas Pharma Europe B.V., Astellas Medical Affairs, Global, HEOR, Leiden, Netherlands

    Zalmai Hakimi

  4. Data Management Centre, European Foundation for the study of Chronic LIver Failure (EF-CLIF), Barcelona, Spain

    Marco Pavesi

  5. Alliance Urology Specialists, Greensboro, USA

    Scott MacDiarmid

  6. University of Bristol and Bristol Urological Institute, Bristol, UK

    Marcus J. Drake

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  1. Mike Herdman
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  2. Jameel Nazir
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Contributions

ND, JN, SM, MJD, and ZH proposed key elements of the study design and critically reviewed the draft protocol/analysis plan; MP, SM, and MJD acquired data; ND, MHe, MP, JN, SM, MJD, ES, and ZH contributed to the analysis and interpretation of the data. All authors discussed the results, provided key intellectual input and commented on the manuscript, and approved the final version for submission.

Corresponding author

Correspondence to Mike Herdman.

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Funding support

This research was funded by Astellas Pharma Europe Ltd. Medical writing support, provided by Lucy Kanan and Tyrone Daniel from Bioscript Medical, was funded by Astellas Pharma Europe Ltd.

Financial disclosures

JN, ZH, ES, and MHu are full-time employees of Astellas Pharma; MHe and ND are employees of Office of Health Economics, which was contracted by Astellas Pharma to support the conduct of this study, and members of the EuroQoL group; SM has received consultancy fees from Astellas; MJD has received speaker and advisory board fees, and research funding from Allergan, Astellas and Ferring; MP has no conflicts of interest.

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Herdman, M., Nazir, J., Hakimi, Z. et al. Assessing Preference-Based Outcome Measures for Overactive Bladder: An Evaluation of Patient-Reported Outcome Data from the BESIDE Clinical Trial. Patient 10, 677–686 (2017). https://doi.org/10.1007/s40271-017-0262-8

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  • DOI: https://doi.org/10.1007/s40271-017-0262-8

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