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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 1.25 mg or fingolimod 5 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 0.5 mg or fingolimod 1.25 mg 1292 Annualised relapse rate Change in Expanded Disabiity Status Scale (EDSS) score 90%/5% (two-sided)
FREEDOMS [20] Placebo or fingolimod 0.5 mg or fingolimod 1.25 mg 1272 Annualised relapse rate Change in EDSS score Power not published/5% (two-sided)
FREEDOMS II [21] Placebo or fingolimod 0.5 mg or fingolimod 1.25 mg 1083 Annualised relapse rate Change in EDSS score 90%/5% (two-sided)