OPTIMISE: MS study protocol: a pragmatic, prospective observational study to address the need for, and challenges with, real world pharmacovigilance in multiple sclerosis

Abstract

IntroductionThe power of ‘real world’ data to improve our understanding of the clinical aspects of multiple sclerosis (MS) is starting to be realised. Disease modifying therapy (DMT) use across the UK is driven by national prescribing guidelines. As such, the UK provides an ideal country in which to gather MS outcomes data. A rigorously conducted observational study with a focus on pharmacovigilance has the potential to provide important data to inform clinicians and patients while testing the reliability of estimates from pivotal trials when applied to patients in the UK.Methods and analysisThe primary aim of this study is to characterise the incidence and compare the risk of serious adverse events in people with MS treated with DMTs. The OPTIMISE:MS database enables electronic data capture and secure data transfer. Selected clinical data, clinical histories and patient-reported outcomes are collected in a harmonised fashion across sites at the time of routine clinical visits. The first patient was recruited to the study on 24 May 2019. As of January 2021, 1615 individuals have baseline data recorded; follow-up data are being captured and will be reported in due course.Ethics and disseminationThis study has ethical permission (London City and East; Ref 19/LO/0064). Potential concerns around data storage and sharing are mitigated by the separation of identifiable data from all other clinical data, and limiting access to any identifiable data. The results of this study will be disseminated via publication. Participants provide consent for anonymised data to be shared for further research use, further enhancing the value of the study.

Evaluating the feasibility of a real world pharmacovigilance study (OPTIMISE:MS)

“…Study design, recruitment and the core dataset have previously been described in detail (Dobson et al, 2021). Briefly, eligible participants are those people diagnosed with MS who are eligible to receive DMT according to NHS guidelines (NHS England or Scotland).…”

“…Where routine clinical appointments were carried out virtually, these data were also included. The complete dataset collected is described in detail elsewhere (Dobson et al, 2021). Core baseline data includes MS-specific measures including Expanded Disability Status Scale (EDSS), relapses within the 2 years prior to study entry, current and previous DMT, and MRI results where available.…”

“…The OPTIMISE:MS pharmacovigilance study is a prospective, pragmatic observational study, conducted across 13 UK MS centres. This study aims to address the need for real world pharmacovigilance in MS by recruiting 4000 people living with MS, and following their clinical outcomes for up to 5 years (Dobson et al, 2021). It provides data on outcomes in specialist MS centres, where most UK MS care is provided.…”

Challenges and Opportunities of Real-World Data: Statistical Analysis Plan for the Optimise:MS Multicenter Prospective Cohort Pharmacovigilance Study

“…The length of the recruitment window, coupled with the introduction of remote consenting, should ensure that the sample is not heavily skewed toward those attending clinics most frequently. Details of the study design and protocol have already been published ( 3 ). The study is academically initiated and led, but is guided by a public-private partnership between academic clinical investigators and pharmaceutical companies with marketing authorisations for DMTs.…”