OPTIMISE:MS A Prospective, Real World Pharmacovigilance Study in Multiple Sclerosis

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medical condition to be studied:
  Multiple sclerosis

Population studied

• Age groups:
  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  4000

Study design details

• Main study objective:
  To characterize the incidence and compare the risk of serious adverse events in people with MS treated with DMD (comparators will be an untreated cohort, and a cohort treated with first line injectable DMD). A serious adverse event in this context is an adverse event resulting in death, requiring in-patient treatment or prolongation of existing in-patient treatment or hospitalisation
• Outcomes:
  Primary endpoints to be examined according to treatment type, duration and switching include: ● Any SAE (any infection requiring hospitalization, any opportunistic infection, any other SAE, any relapse, death, MeDRA coded), Secondary endpoints for exploratory objectives include: Outcomes associated with sequential therapies with multiple DMT Abnormally low total blood lymphocyte count stratified by grade of lymphopaenia Abnormally increased liver function tests stratified by grade of abnormal liver function Disability progression New MRI lesion activity Occurrence of pregnancy and outcomes of pregnancy
• Data analysis plan:
  The total number of safety events (SAEs) for DMTs (overall and by individual DMTs) will be presented per 100 person years. Both event rates (multiple events per individual) and incidence will be examined. Continuing quality assessment of the database will be performed. Active interrogation of data collected from sites (via regular audit of a proportion of records) will enable early detection of inconsistent and/or erroneous data, allowing corrections to be made prior to any analysis. Inconsistent data will be defined on an individual patient level (where records are selected for audit), e.g. where clinical records are intrinsically inconsistent (e.g. large fluctuations in EDSS recorded with no relapses recorded, large fluctuations in laboratory values without clinical or treatment correlates). In sites where inconsistent data is detected, further records will be interrogated to further evaluate if any systematic error is present.