NCT00420212 (DEFINE); NCT00451451 (CONFIRM); NCT00289978 (FREEDOMS); NCT00355134 (FREEDOMS II).

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Two studies provided evidence of a change in patient management following EUS for staging esophageal cancer, a higher level of evidence for a clinical benefit than can be obtained from accuracy studies alone. This evidence contributed to a recommendation for public funding of EUS in staging esophageal cancer in Australia.

Objectives: Delayed-release dimethyl fumarate (DMF; also known as gastroresistant DMF) and fingolimod are oral disease-modifying treatments for relapsing-remitting multiple sclerosis (RRMS). Direct comparisons of these agents are not possible due to a lack of head-to-head trials. In this study, comparative effectiveness research was conducted by indirectly comparing efficacy outcomes at 2 years with DMF or fingolimod treatment of RRMS in Phase 3 studies. MethOds: Individual patient data from the DEFINE and CONFIRM studies of DMF (pooled) and aggregate data from the FREEDOMS and FREEDOMS II studies of fingolimod (pooled using random effects meta-analysis) were utilised. Only results using the approved dosage of DMF (240 mg twice daily) and fingolimod (0.5 mg once daily) are reported. Matching-adjusted indirect comparison was conducted as described in Signorovitch et al (2010). Patients in the pooled DMF trials were weighted such that their average baseline characteristics (age, gender, time from onset of symptoms, Expanded Disability Status Scale score, number of relapses in previous year) matched those reported for patients in pooled fingolimod trials. After matching, weighted efficacy outcomes for patients treated with DMF were compared with summary efficacy outcomes for patients treated with fingolimod. Results: After matching, all baseline characteristics were balanced between the pooled DMF trials and the pooled fingolimod trials. At 2 years, annualised relapse rate ratio (95% confidence interval [CI]) for DMF vs placebo was 0.52 (0.43, 0.62) and for fingolimod vs placebo was 0.48 (0.42, 0.55). Twelve-week confirmed disease progression hazard ratio (95% CI) for DMF vs placebo was 0.70 (0.57, 0.85) and for fingolimod vs placebo was 0.76 (0.61, 0.95). Additional data, including comparison of DMF vs fingolimod, will be presented. cOnclusiOns: In a matching-adjusted indirect comparison, the efficacy of DMF was similar to that of fingolimod on clinical measures of relapse and disability progression.