There is significant variability in outcomes evaluated in randomized controlled trials (RCTs) of neuromyelitis optica spectrum disorder (NMOSD). To date, several drugs have been studied and approved for use in patients with NMOSD, and the number of available treatments and trials is expected to increase in coming years. Therefore, defining the appropriate outcomes for future NMOSD clinical trials and identifying the best tools to assess these outcomes is a priority in the field. The main objective is to develop the core outcome set that should be used in studies of medical interventions for adult patients with NMOSD.
The methodological design of this project will be guided by the recommendations of the COMET Initiative and the COSMIN Initiative. The study protocol is developed in accordance with the Core Outcome Set-STAndardised Protocol (COS-STAP) statement, and the final study publications will be written based on the Core Outcome Set-STAndards for Reporting (COS-STAR) statement. The target population of the present study is adult patients with NMOSD, independent of the severity or progression of the disease.
Potentially relevant COS: There is no identified or developed COS for NMOSD.
- Cristian Eduardo Navarro, MD MSc PhD(c) from Universidad de Antioquia (principal investigator).
- Iván Darío Flórez, MD MSc PhD from Universidad de Antioquia and McMaster University (supervisor).
- Sarah Gorst, BSc MSc PhD from University of Liverpool (supervisor).
- María Isabel Zuluaga, MD from Medicarte S.A (supervisor).
Disease Category: Neurology
Disease Name: Neuromyelitis optica spectrum disorder
Age Range: 18 - 99
Sex: Either
Nature of Intervention: Drug
- Clinical experts
- Conference participants
- Consumers (caregivers)
- Consumers (patients)
- Economists
- Epidemiologists
- Journal editors
- Methodologists
- Patient/ support group representatives
- Policy makers
- Researchers
- COS for clinical trials or clinical research
- COS for practice
- Consensus meeting
- Delphi process
- Focus group(s)
- Interview
- Literature review
- Survey
- Systematic review
This study will be developed in three stages:
- Stage 1: a systematic review with four aims; 1) to determine the long list of outcomes which will be part of the Delphi; 2) to describe the minimal clinically important difference (MID) for each outcome reported; 3) to identify the measurement instruments applied to evaluate each outcome in RCTs of NMOSD; and 4) to identify the values and preferences of patients with NMOSD published in qualitative studies.
- Stage 2: a qualitative study with a focus group session with NMOSD patients, caregivers, and their representatives. This qualitative study will explore NMOSD patients’ experiences and understand which treatment outcomes they value most. The results will be compared with the results of the systematic review to evaluate whether RCTs consider patient preferences among the outcomes.
- Stage 3: a Delphi consensus to define the outcomes that will compose the COS. Following the COS-STAD recommendations, key stakeholders that will be invited to participate are patients, healthcare professionals, researchers, systematic reviewers, guideline developers, and policymakers. Quantitative data will be generated, by asking to the key stakeholders to score the importance of outcomes during an online Delphi survey and by making a final decision as to what outcomes should be part of the COS during an online consensus meeting, and to define how and when each outcome should be measure in a RCTs of NMOSD.
The dissemination plan of the COS includes scientific publication in relevant journals, conference presentations, briefing notes to key agencies, publications via academic social media, and identifying NMOSD ambassadors not included in the panel Delphi. This process will be subject to periodic review to ensure strategies are effective and to identify new barriers to implementation.