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Patient preferences on rheumatoid arthritis second-line treatment: a discrete choice experiment of Swedish patients
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Public Health and Caring Sciences, Centre for Research Ethics and Bioethics.ORCID iD: 0000-0002-5865-5590
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Public Health and Caring Sciences, Centre for Research Ethics and Bioethics.ORCID iD: 0000-0002-3823-8263
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Public Health and Caring Sciences, Centre for Research Ethics and Bioethics.
Institute of Inflammation and Ageing, University of Birmingham Research Laboratories, Queen Elizabeth Hospital, Birmingham, B15 2WB, UK.
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2020 (English)In: Arthritis Research & Therapy , E-ISSN 1478-6362, Vol. 22, article id 288Article in journal (Refereed) Published
Abstract [en]

Background

Preference assessments of patients with rheumatoid arthritis can support clinical therapeutic decisions for including biologic and targeted synthetic medicines to use. This study assesses patient preferences for attributes of second-line therapies and heterogeneity within these preferences to estimate the relative importance of treatment characteristics and to calculate the minimum benefit levels patients require to accept higher levels of side effects.

Methods

Between November 2018 to August 2019, patients with rheumatoid arthritis were recruited to a survey containing demographic and disease-related questions as well as a discrete choice experiment to measure their preferences for second-line therapies using biologics or Janus kinases inhibitors. Treatment characteristics included were route of administration, frequency of use, probability of mild short-term side effects, probability of side effects changing appearance, probability of psychological side effects, probability of severe side effects and effectiveness of treatment.

Results

A total of 358 patients were included in the analysis. A latent class analysis revealed three preference patterns: (1) treatment effectiveness as the single most important attribute, (2) route of administration as the most important attribute, closely followed by frequency of use and psychological side effects and (3) severe side effects as the most important attribute followed by psychological side effects. In addition, disease duration and mild side effects influenced the patients’ choices.

Conclusion

Respondents found either effectiveness, route of administration or severe side effects as the most important attribute. Patients noting effectiveness as most important were more willing than other patients to accept higher risks of side effects.

Place, publisher, year, edition, pages
2020. Vol. 22, article id 288
Keywords [en]
Discrete choice experiment, Patient preferences, Rheumatoid arthritis, Second-line treatment
National Category
Social and Clinical Pharmacy Rheumatology and Autoimmunity
Identifiers
URN: urn:nbn:se:uu:diva-429228DOI: 10.1186/s13075-020-02391-wISI: 000600277400001PubMedID: 33341117OAI: oai:DiVA.org:uu-429228DiVA, id: diva2:1512024
Projects
IMI-PREFER
Funder
EU, Horizon 2020Available from: 2020-12-21 Created: 2020-12-21 Last updated: 2024-07-04Bibliographically approved
In thesis
1. Getting a Say: Bringing patients’ views on benefit-risk into medical approvals
Open this publication in new window or tab >>Getting a Say: Bringing patients’ views on benefit-risk into medical approvals
2021 (English)Doctoral thesis, comprehensive summary (Other academic)
Abstract [en]

The focus of this thesis is a new quantitative approach to consider patient preferences on benefits and risks in medical approvals. The overall aim of this thesis was to explore how patient preference information may be relevant to regulatory marketing authorisation decisions.

Study I provides an overview of the different decision-processes of industry, regulatory agencies and health technology assessment bodies/reimbursement agencies along the medical product lifecycle. In total, 15 decision points with the potential to include patient preference information were identified. 

Study II was an exploration of the patient perspective regarding the use of patient preference information in regulatory marketing authorisation decisions. Patients emphasised the need to have a say in decisions affecting their health and to be properly informed about potential risks and benefits of medical products. 

Study III assessed patient preferences on benefits and risks of Rheumatoid Arthritis treatments. Results revealed that patients’ preferences differed substantially. The three most important treatment attributes for patients with rheumatoid arthritis were: the probability of severe side effects, treatment effectiveness and route of administration. Those placing relatively more importance on treatment effectiveness were willing to acceptance higher risk levels of side effects. 

Study IV aimed to determine the influence of an educational tool, compared with traditional written information on patient preferences. It was found that those respondents receiving the educational tool focused more on the potential side effects than those receiving written information. 

Patient preference information has the potential to reveal patients’ preferences on benefits and risks with scientific rigour and can therefore be weighed against clinical data. This thesis supports the development of a structured approach to learn about patient preferences on benefits and risks in medical approvals

Place, publisher, year, edition, pages
Uppsala: Acta Universitatis Upsaliensis, 2021. p. 72
Series
Digital Comprehensive Summaries of Uppsala Dissertations from the Faculty of Medicine, ISSN 1651-6206 ; 1732
Keywords
Educational material, Medical product lifecycle, Patient preferences, Regulatory marketing authorisations, Rheumatoid arthritis
National Category
Medical and Health Sciences
Identifiers
urn:nbn:se:uu:diva-437909 (URN)978-91-513-1168-5 (ISBN)
Public defence
2021-05-12, Sal X, Universitetshuset, Biskopsgatan 3, Uppsala, 13:00 (English)
Opponent
Supervisors
Available from: 2021-04-21 Created: 2021-03-18 Last updated: 2021-11-05

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Schölin Bywall, KarinKihlbom, UlrikHansson, MatsBaecklund, Eva

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