The PREFERMS Study: Evaluating Real-World Patient Retention on Oral Fingolimod Compared with Injectable Disease-Modifying Therapies in RRMS

Background: The efficacy and safety of fingolimod in relapsing–remitting multiple sclerosis (RRMS) has been well established in controlled clinical trials and the post-marketing setting. However, real-world patient retention on injectable disease-modifying therapies (iDMTs), and related clinical and magnetic resonance imaging (MRI) outcomes have not been evaluated for fingolimod versus iDMTs. Understanding the impact of patient retention in early RRMS could have important implications for treatment decision-making during the initial stages of the disease.

Objectives: To conduct a randomized, prospective, real-world study of patient retention on once-daily oral fingolimod (0.5 mg) compared with retention on first-line iDMTs in the course of RRMS.

Methods: PREFERMS is a 12-month, phase 4, open-label, active-controlled, randomized, multicenter study enrolling ~850 patients, mostly in the early stages of RRMS, who are either treatment-naïve or have been treated with one class of iDMT (interferon or glatiramer acetate) or dimethyl fumarate (< 2 months’ exposure). Patients are in the process of being randomized 1:1 to receive fingolimod or iDMT; those who have previously been treated with iDMT will receive an alternative iDMT class during the study if randomized to iDMT. The primary endpoint is the rate of retention on randomized treatment. Secondary endpoints include reasons for treatment discontinuation, occurrence and persistence of drug-related adverse events, cognitive impairment, percentage change in brain volume and medication satisfaction. Comprehensive MRI, optical coherence tomography, Multiple Sclerosis Functional Composite and quality-of-life assessments are exploratory parameters.

Results: Enrollment started in June 2012; approximately 140 investigational sites across the USA have met more than 60% of the recruitment target as of 28 October 2013. Study results are expected in 2015

Conclusions: PREFERMS will evaluate patient retention on fingolimod compared with retention on iDMT, and the impact this may have on key clinical and MRI outcomes in RRMS. Moreover, this study will provide important real-world clinical evidence and patient-reported outcomes of relevance to early fingolimod therapy.