Table 1 Summary of pivotal trials in the illustrative development plan
From: The inclusion of real world evidence in clinical development planning
Study | Treatments | No. of patients (N) | Primary outcome | Relevant secondary outcomes | Power/significance level |
|---|---|---|---|---|---|
Phase II [17] | Placebo or fingolimod 1.25Â mg or fingolimod 5Â mg | 281 | Hamburg Quality of Life Questionnaire (HAQUAMS) and Beck Depression Inventory second edition (BDI-II) | Not published | Not published |
TRANSFORMS [18] | Interferon beta-1a or fingolimod 0.5Â mg or fingolimod 1.25Â mg | 1292 | Annualised relapse rate | Change in Expanded Disabiity Status Scale (EDSS) score | 90%/5% (two-sided) |
FREEDOMS [20] | Placebo or fingolimod 0.5Â mg or fingolimod 1.25Â mg | 1272 | Annualised relapse rate | Change in EDSS score | Power not published/5% (two-sided) |
FREEDOMS II [21] | Placebo or fingolimod 0.5Â mg or fingolimod 1.25Â mg | 1083 | Annualised relapse rate | Change in EDSS score | 90%/5% (two-sided) |