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. 2022 Mar;269(3):1670-1677.
doi: 10.1007/s00415-021-10836-8. Epub 2021 Oct 9.

Early and unrestricted access to high-efficacy disease-modifying therapies: a consensus to optimize benefits for people living with multiple sclerosis

Massimo Filippi  1   2 , Romano Danesi  3 , Tobias Derfuss  4 , Martin Duddy  5 , Paolo Gallo  6 , Ralf Gold  7 , Eva Kubala Havrdová  8 , Barbara Kornek  9 , Francesco Saccà  10 , Mar Tintoré  11 , Jörg Weber  12 , Maria Trojano  13
Affiliations

Early and unrestricted access to high-efficacy disease-modifying therapies: a consensus to optimize benefits for people living with multiple sclerosis

Massimo Filippi et al. J Neurol. 2022 Mar.

Abstract

Early intervention with high-efficacy disease-modifying therapy (HE DMT) may be the best strategy to delay irreversible neurological damage and progression of multiple sclerosis (MS). In European healthcare systems, however, patient access to HE DMTs in MS is often restricted to later stages of the disease due to restrictions in reimbursement despite broader regulatory labels. Although not every patient should be treated with HE DMTs at the initial stages of the disease, early and unrestricted access to HE DMTs with a positive benefit-risk profile and a reasonable value proposition will provide the freedom of choice for an appropriate treatment based on a shared decision between expert physicians and patients. This will further optimize outcomes and facilitate efficient resource allocation and sustainability in healthcare systems and society.

Keywords: Benefit–risk profile; Healthcare system; High-efficacy disease-modifying therapy; Multiple sclerosis; Pharmacoeconomics; Unrestricted access.

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Conflict of interest statement

MF is Editor-in-Chief of the Journal of Neurology and Associate Editor of Human Brain Mapping; received compensation for consulting services and/or speaking activities from Alexion, Almirall, Bayer, Biogen, Celgene, Eli Lilly, Genzyme, Merck-Serono, Novartis, Roche, Sanofi, Takeda, and Teva Pharmaceutical Industries; and receives research support from Biogen Idec, Merck-Serono, Novartis, Roche, Teva Pharmaceutical Industries, Italian Ministry of Health, Fondazione Italiana Sclerosi Multipla, and ARiSLA (Fondazione Italiana di Ricerca per la SLA). RD serves on scientific advisory board and has consulting relationship with Ipsen, Novartis, Pfizer, Sanofi Genzyme, AstraZeneca, Janssen, Gilead, Lilly, EUSA Pharma; and reports support for travel, accommodation, expenses from Ipsen, Sanofi Genzyme. TD reports support from Novartis during the study; grants/other support from Novartis, Biogen, Merck, Sanofi Genzyme, Roche, MedDay, GeNeuro, Celgene, Mitsubishi Pharma, Alexion outside the submitted work; and his wife is an employee of Novartis and holds shares of Novartis. MD has received personal honoraria for speaking, advisory boards, participation in research, and travel expenses from Bayer, Biogen, Celgene (BMS), Merck, Mylan, Novartis, Roche, Sanofi Genzyme, Teva and TG Therapeutics and for expert testimony from Biogen. PG has been a consultant and member of Advisory Board for Biogen Italy, Sanofi-Genzyme, Merck-Serono, Almirall, Roche, Bristol-Meyer-Squibb and Novartis-Farma; has received funding for travel and speaker honoraria from Merck-Serono, Biogen Italy, Sanofi-Genzyme, Novartis-Pharma, Roche, Almirall; has received research support from Biogen Italy, Merk-Serono, Sanofi-Genzyme, Roche, Novartis. RG reports honoraria from Bayer, Biogen, Merck Serono, Novartis, and Teva Neuroscience; and research support from Bayer, Biogen, Merck Serono, Novartis, and Teva Neuroscience. EH has received honoraria/research support from Biogen, Merck Serono, Novartis, Roche, and Teva; has served as a member of advisory boards for Actelion, Biogen, Celgene, Merck Serono, Novartis, and Sanofi Genzyme; has been supported by the Czech Ministry of Education research, project PROGRES Q27/LF1. BK has received honoraria for speaking and participation in advisory boards from Biogen, Merck, Novartis, BMS-Celgene, Sanofi-Genzyme, Teva, and Roche. FS received honoraria for public speaking from Alexion, Argenx, Biogen, Mylan, Novartis, Roche, Sanofi, Teva; and for participating in advisory boards for Alexion, Almirall, Argenx, Avexis, Biogen, Forward Pharma, Lexeo Therapeutics, Merk, Novartis, Pomona, Roche, Sanofi, Takeda. MT as received compensation for consulting services and speaking honoraria from Almirall, Bayer Schering Pharma, Biogen-Idec, Genzyme, Janssen, Merck-Serono, Novartis, Roche, Sanofi-Aventis, Viela Bio and Teva Pharmaceuticals. MT is co-editor of Multiple Sclerosis Journal-ETC. JRW has nothing to disclose. M Trojano has served on scientific AB for Biogen, Novartis, Roche, Merck and Genzyme; has received speaker honoraria from Biogen, Roche, Sanofi, Merck, Genzyme and Novartis; and has received research grants for her Institution from Biogen, Merck, Novartis and Roche.

Figures

Fig. 1
Fig. 1
Reimbursement status for RMS for ocrelizumab compared with its EMA label (based on national reimbursement status; see supplementary material for the full list of sources). EMA European Medicines Agency, RMS relapsing multiple sclerosis
Fig. 2
Fig. 2
Different factors highlighting the need for early initiation of HE DMTs in PLwMS. EDSS Expanded Disability Status Scale, DMT disease-modifying therapy, HE high efficacy, ME moderate efficacy, MS multiple sclerosis, NEDA no evidence of disease activity, PLwMS people living with MS, SPMS secondary progressive MS
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