Oral Disease-Modifying Treatments for Relapsing Multiple Sclerosis: A Likelihood to Achieve No Evidence of Disease Activity or Harm Analysis
The likelihood to help or harm (LHH) is an absolute measure of the benefit versus risk profile of a medication, which can be used to assess the potential for benefit versus harm of different disease-modifying treatments (DMTs) for relapsing multiple sclerosis (R-MS) and facilitate clinical decision-making.
The objective of this study was to assess absolute differences in benefit:risk ratios of oral DMTs for R-MS, using LHH analysis with no evidence of disease activity (NEDA) as beneficial outcome.
The number needed to treat for a paient to achieve NEDA (NNTBNEDA) was used as an effect size metric of efficacy and the number needed to treat for a patient to experience an adverse event (NNTHAE), a serious adverse event (NNTHSAE), or treatment discontinuation due to an adverse event (NNTHAE-D) were used as measures of risk. The LHH—which is the ratio of NNTH:NNTB—values were calculated from published phase III trial data for oral DMTs.
The values for likelihood to achieve NEDA than experience any AE ratio (LHH(AE/NEDA)) were 3.9, 6.8, 12.5 and 3.7, the likelihood to achieve NEDA than experience a SAE ratio (LHH(SAE/NEDA)) values were 3.5, 15, 23.5 and 2.8, and the likelihood to achieve NEDA versus discontinue treatment (LHH(AE-D/NEDA)) values were 20.3, 4.3, 3.9 and 3.1 for cladribine, dimethyl-fumarate, fingolimod, and teriflunomide, respectively.
With all of the oral DMTs examined, R-MS patients are more likely to achieve NEDA than experience any adverse event.
The authors thank Dr S.J. Kedikoglou, MD, MPH, PhD and Mrs. E. Delicha, MSc for their expert statistical advice.
Compliance with Ethical Standards
Ethical Approval and Consent
No ethics approval or patient consent was obtained because all data used in this study were collected from previously published peer-reviewed articles.
Conflict of interest
DP has received consulting, speaking fees, and travel grants from Bayer, Genesis Pharma, Merck, Novartis, Roche, Sanofi-Aventis, Specifar, and Teva. DDM has received consulting, speaking fees, and travel grants from Allergan, Amgen, Cefaly, ElectroCore, Genesis Pharma, Eli Lily, Merck-Serono, Novartis, Roche, Sanofi-Genzyme, Speficar, and Teva.
No funding source had a role in the conception and preparation of this article.
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