Identifying and addressing patient-centred outcomes to improve medication adherence in rheumatology

Many rheumatic conditions require the long-term use of medications, yet suboptimal adherence remains a major challenge. There are increasing efforts to develop and test strategies to improve medication adherence in rheumatology, however few interventions have been shown to be effective. This may be due to a number of problems. It remains uncertain whether existing interventions to improve adherence address the priorities and concerns of patients with rheumatic conditions. In addition, the outcomes reported in trials targeting adherence are heterogeneous, limiting the ability to assess the comparative effect of interventions. Core domain sets are defined as the minimum set of outcome domains that should be measured and reported in specific clinical trials. They reduce inconsistent outcome reporting and reporting bias. Furthermore, they can help ensure the use of outcomes that are important to patients and decision-makers. Through the Outcome Measures in Rheumatology (OMERACT) initiative, core domain sets have been established for multiple rheumatic conditions. This thesis aims to understand medication adherence from the perspective of patients with diverse rheumatic conditions and their caregivers; and to identify issues in outcome reporting in interventional studies targeting medication adherence in rheumatic conditions as part of OMERACT. Chapter 2 presents a review of qualitative research principles and methodology in the context of rheumatic conditions. An overview of qualitative methods is presented and the key approaches to guide the appraisal of qualitative research are discussed. The next part of this thesis (Chapters 3 to 5) consists of systematic reviews which provide a comprehensive evaluation of outcomes reported in interventional studies in medication adherence in rheumatic conditions and qualitative studies that describe the patients' experience in a variety of rheumatic conditions. The second part of this thesis (Chapter 6) further explores the perspective and priorities of patients with rheumatic conditions and their caregivers using focus groups with nominal group technique. This study addresses patient and caregiver barriers and facilitators to medication adherence. Chapters 7 is a report from a workshop convened at the 2018 OMERACT conference to discuss the challenges with developing a core set of outcomes for interventional studies targeting medication adherence. Despite the challenges of producing a core domain set for this topic, the meeting clarified an approach of how this could be achieved using OMERACT methodology. The thesis provides a greater understanding of outcomes and factors that are important to patients and their caregivers, and the mismatch with currently reported outcomes. It was demonstrated that consistent reporting of outcomes is needed to better inform which interventions are effective. The qualitative studies on patient and caregiver perspectives highlight the need for empathetic care that promotes trust in the doctor. Overall a patient-centred approach to supporting medication adherence is needed in both clinical care and research. This approach will help address the complexity and challenges patients with rheumatic conditions face with their medication-taking and improve outcomes that matter to them