Co-producing a multi-stakeholder Core Outcome Set for distal Tibia and Ankle fractures (COSTA): a study protocol

Background Ankle fracture is a common injury with a strong evidence base focused on effectiveness of treatments. However, there are no reporting guidelines on distal tibia and ankle fractures. This has led to heterogeneity in outcome reporting and consequently, restricted the contribution of evidence syntheses. Over the past decade, core outcome sets have been developed to address this issue and are available for several common fractures, including those of the hip, distal radius, and open tibial fractures. This protocol describes the process to co-produce—with patient partners and other key stakeholders—a multi-stakeholder derived Core Outcome Set for distal Tibia and Ankle fractures (COSTA). The scope of COSTA will be for clinical trials. Methods The study will have five-stages which will include the following: (i) systematic reviews of existing qualitative studies and outcome reporting in randomised controlled trial studies to inform a developing list of potential outcome domains; (ii) qualitative interviews (including secondary data) and focus groups with patients and healthcare professionals to explore the impact of ankle fracture and the outcomes that really matter; (iii) generation of meaningful outcome statements with the study team, international advisory group and patient partners; (iv) a multi-round, international e-Delphi study to achieve consensus on the core domain set; and (v) an evidence-based consensus on a core measurement set will be achieved through a structured group consensus meeting, recommending best assessment approaches for each of the domains in the core domain set. Discussion Development of COSTA will provide internationally endorsed outcome assessment guidance for clinical trials for distal tibia and ankle fractures. This will enhance comparative reviews of interventions, potentially reducing reporting bias and research waste.

Researchers’ perspectives on methodological challenges and outcomes selection in interventional studies targeting medication adherence in rheumatic diseases: an OMERACT-adherence study

Background Research on adherence interventions in rheumatology is limited by methodological issues, particularly heterogeneous outcomes. We aimed to describe researchers’ experiences with conducting interventional studies targeting medication adherence in rheumatology and their perspectives on establishing core outcomes. Methods Semi-structured interviews using audio conference were conducted with researchers who had conducted an adherence study of any design in the past 10 years. Data collection and thematic analysis were performed iteratively, until saturation. Results We interviewed 13 researchers, most of whom worked in academia and specialized in epidemiology and/or health services research. We identified three themes: 1) improving measurement of adherence (considering all phases of adherence, using appropriate and relevant measures, and establishing clinically meaningful thresholds); 2) challenges in designing and appraising adherence intervention studies (considering the confusion over a plethora of outcomes, difficulties with powering studies to demonstrate meaningful changes, and suboptimal descriptions of adherence interventions in published studies); and 3) advancing outcome assessment in adherence intervention studies (capturing rationale for developing a core domain set as well as recommendations and anticipated challenges by participants). Conclusions Uniquely gathering perspectives from international adherence researchers, our findings led to researcher-informed recommendations for improving adherence research including specifying the targeted adherence phase in designing interventions and studies and providing a glossary of terms to promote consistency in reporting. We also identified recommendations for developing a core domain set for interventional studies targeting medication adherence including involvement of patients, clinicians, and other stakeholders and methodological and practical considerations to establish rigor and support uptake.

THU0565 RESEARCHERS’ PERSPECTIVES ON ADHERENCE INTERVENTION RESEARCH AND OUTCOMES IN RHEUMATOLOGY: AN INTERNATIONAL QUALITATIVE STUDY

Background:Medication non-adherence is a significant problem among patients with rheumatic diseases. Research on adherence interventions in rheumatology is limited and disappointing, with studies using heterogeneous outcomes. Understanding these limitations is needed to inform the design of better interventions and research studies.Objectives:To describe researchers’ perspectives and experiences on adherence intervention research and outcomes in rheumatology.Methods:Semi-structured interviews using video conference were conducted with researchers who had been an investigator on an adherence study of any design in the past 10 years. Interviews were recorded and transcribed verbatim. Participants were asked about their experiences with conducting adherence research and perspectives on introduction of a core domain set of outcomes for adherence intervention trials in rheumatology. Data collection and thematic analysis were conducted iteratively, until saturation.Results:We interviewed 13 researchers from seven countries (Australia, Belgium, Canada, Netherland, Thailand, UK, and USA). A majority worked in academia (75%), specialized in epidemiology and/or health services research (62%) and had led between 2-5 adherence studies in the past five years (62%).Three themes were identified:1) challenges in designing, conducting and evaluating adherence studies;2) current outcomes in adherence intervention studies and their relevance; and3) implementing a core domain set of outcomes for adherence intervention studies.Major challenges in conducting adherence research included inconsistent adherence terminology and measurement. Participants noted a lack of guidance on outcome selection and measurement when evaluating the effectiveness of an adherence intervention and indicated their preference for research to report adherence, intervention-specific, and health-related outcomes. Finally, implementing a core domain set of outcomes was thought to be challenging but valuable in strengthening the evidence (by facilitating meta-analysis), and improving clinical outcomes (by informing clinicians about the effectiveness of interventions).Conclusion:Adherence research in rheumatology has been hindered by lack of standardization and guidance on terminology, measurement and outcome selection. Our findings form the basis for recommendations for improving the design, conduct and evaluation of adherence intervention studies in rheumatology, particularly for developing a core domain set of outcomes to improve consistency and facilitate comparisons.Table 1.Themes and representative quotations.Theme 1: Challenges in designing, conducting and evaluating studies of adherence interventions“…the people you often most want in your sample are the people who are non-adherent and often the people who are non-adherent are the people who are hardest to recruit.”“Long term the issue has been about measurements because people confuse and conflate various aspects of medication adherence.Theme 2: Current outcomes in adherence intervention studies and their relevance“you have a whole range of outcomes…psychological outcomes…there’s measures of health care utilization and things like attendance at hospital, nurse appointments and duration, things like times off work,, and also all the relevant clinical outcomes.”Theme 3: Implementing a core domain set of outcomes for adherence intervention studies“…will make trials more comparable and increase the likelihood that you’d be able to combine efforts internationally”Disclosure of Interests:Shahrzad Salmasi: None declared, Ayano Kelly: None declared, Susan J. Bartlett Consultant of: Pfizer, UCB, Lilly, Novartis, Merck, Janssen, Abbvie, Speakers bureau: Pfizer, UCB, Lilly, Novartis, Merck, Janssen, Abbvie, Maarten de Wit Grant/research support from: Dr. de Wit reports personal fees from Ely Lilly, 2019, personal fees from Celgene, 2019, personal fees from Pfizer, 2019, personal fees from Janssen-Cilag, 2017, outside the submitted work., Consultant of: Dr. de Wit reports personal fees from Ely Lilly, 2019, personal fees from Celgene, 2019, personal fees from Pfizer, 2019, personal fees from Janssen-Cilag, 2017, outside the submitted work., Speakers bureau: Dr. de Wit reports personal fees from Ely Lilly, 2019, personal fees from Celgene, 2019, personal fees from Pfizer, 2019, personal fees from Janssen-Cilag, 2017, outside the submitted work., Lyn March: None declared, Allison Tong: None declared, Peter Tugwell: None declared, Kathleen Tymms: None declared, Suzanne Verstappen Grant/research support from: BMS, Consultant of: Celltrion, Speakers bureau: Pfizer, Mary De Vera: None declared