Do clinical trials for new disease modifying treatments include real world patients with multiple sclerosis?

Abstract

We often see that clinical and demographic characteristics of real-world studies (RWS) do not differ from patients included in randomized controlled trials (RCT).

Objective: to compare clinical and demographic aspects of patients included in RCT and RWS that evaluated new disease modifying treatment in multiple sclerosis (MS).

Methods: a systematic non-language-restricted literature search of RCT and RWS that evaluated new disease modifying treatments (natalizumab, alemtuzumab, ocrelizumab, fingolimod, teriflunomide, dimethyl fumarate and cladribine) from January 2005 to January 2019. Demographic and clinical data were extracted, described and compared.

Results: 18 RCT and 73 RWS were included. We found no differences in clinical and demographic aspects between RCT and RWS except in the frequency of naïve patients included in RCT vs. RWS 65.6% (95%CI 52-74) vs. 36.4% (95%CI 21-46), respectively, (p = 0.013) at study entry, as well as for the inclusion of patients that used previous treatment 34.4% (95%CI 22-41) vs. 63.6% (95%CI 53-74) in RCT and RWS, respectively,(p = 0.007) at study entry.

Conclusion: We did not observe significant differences in most clinical and demographic aspects of included patients in RCT and RWS. Studies that include the full spectrum of MS patients followed in clinical practice are needed.

Keywords: Clinical trials; Multiple sclerosis; Patient´s characteristics; Real world studies.