A method to compare prospective and historical cohorts to evaluate drug effects. Application to the analysis of early treatment effectiveness of intramuscular interferon-β1a in multiple sclerosis patients.

Background: Disease modifying therapy have changed the natural evolution of multiple sclerosis (MS), with efficacy demonstrated in randomized clinical trials. Standard-of-care effectiveness is needed to complement clinical trial data and highlight outcomes in real-world practice, but comparing prospective patients with historical cohorts likely introduces biases. To address these potential biases, assigning a patient with a score that expresses his/her disease prognosis before starting a therapy may make it possible to evaluate the unbiased ability of the therapy to modify disease natural history. Thus, we aimed at analyzing the effectiveness of intramuscular interferon-β1a (im IFN-β1a) matching by BREMSO score (Bayesian Risk Estimate for Multiple Sclerosis at Onset) a prospective real-world cohort of treated patients with a historical cohort of untreated patients.
Material and Methods: We observed 108 newly diagnosed, treatment naïve MS patients over 12 months of treatment with im IFN-β1a. BREMSO score was used to assign a value to each patient, giving the real-world treated patients comparable with the Historical untreated patients, on the basis of the same risk to have unfavorable evolution.
Results: A significantly higher percentage of relapse-free patients is observed in IFN-β1a treated cohort vs. Historical untreated cohort (79.6% vs. 59.3%, p < 0.01). Clinical relapses risk is reduced by 2.2 times in treated patients (p = 0.01).
Conclusions: We propose a promising method to manage observational data in a relatively unbiased way, in order to analyze real-world treatment effectiveness.
Competing Interests: Declaration of Competing Interest Dr. Amato reports personal fees from Bayer, Biogen Idec, Merck Serono, Novartis, Sanofi Genzyme, Teva, Almirall, outside the submitted work; Dr. Bergamaschi reports non-financial support from Biogen, during the conduct of the study; grants and personal fees from Biogen, Mervk-serono, Genzyme, Teva, Novartis, Sanofi aventis, Almirall, Bayer, outside the submitted work; . Dr. Brescia Morra reports personal fees from Novartis, Biogen, Genzyme, Teva, Almirall, Bayer, and Merck, outside the submitted work; Dr. Buccafusca has nothing to disclose. Dr. Ferraro has nothing to disclose. Dr. Mallucci reports non-financial support from Biogen, during the conduct of the study; grants from Biogen, personal fees from Biogen, Genzyme, Merck Serono, outside the submitted work; . Dr. Mirabella reports personal fees from Biogen, Genzyme, Novartis, Merck Serono, Almirall and Teva, outside the submitted work; . Dr. Moiola reports personal fees from Sanofi-Genzyme, Biogen, TEVA, Merck Serono and Novartis, outside the submitted work; . Dr. Moscato has nothing to disclose. Dr. Patti reports personal fees from Bayer Schering, Biogen Idec, Merck Serono, Novartis, and Sanofi Aventis, outside the submitted work; . Dr. Plewnia has nothing to disclose. Dr. Puma is employee of Biogen Italia. Dr. Trojano reports personal fees from Biogen, Merck Serono, Novartis, Sanofi, and Teva, grants from Biogen, Merck Serono, and Novartis, outside the submitted work; Dr. Zaffaroni reports grants and personal fees from Biogen, Genzyme, Novartis, Merck Serono, Sanofi and Teva, outside the submitted work; Dr. Zipoli is employee of Biogen Italia.
(Copyright © 2020. Published by Elsevier B.V.)