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. 2021 Aug;27(9):1403-1410.
doi: 10.1177/13524585211030207. Epub 2021 Jul 26.

Ethical use of off-label disease-modifying therapies for multiple sclerosis

Joanna Laurson-Doube  1 , Nick Rijke  1 , Anne Helme  1 , Peer Baneke  1 , Brenda Banwell  2 , Shanthi Viswanathan  3 , Bernhard Hemmer  4 , Bassem Yamout  5
Affiliations

Ethical use of off-label disease-modifying therapies for multiple sclerosis

Joanna Laurson-Doube et al. Mult Scler. 2021 Aug.

Abstract

Background: Off-label disease-modifying therapies (DMTs) for multiple sclerosis (MS) are used in at least 89 countries. There is a need for structured and transparent evidence-based guidelines to support clinical decision-making, pharmaceutical policies and reimbursement decisions for off-label DMTs.

Objectives/results: The authors put forward general principles for the ethical use of off-label DMTs for treating MS and a process to assess existing evidence and develop recommendations for their use.

Conclusion: The principles and process are endorsed by the World Federation of Neurology (WFN), American Academy of Neurology (AAN), European Academy of Neurology (EAN), Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS), European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), Middle-East North Africa Committee for Treatment and Research in Multiple Sclerosis (MENACTRIMS) and Pan-Asian Committee for Treatment and Research in Multiple Sclerosis (PACTRIMS), and we have regularly consulted with the Brain Health Unit, Mental Health and Substance Use Department at the World Health Organization (WHO).

Keywords: Disease-modifying therapies; access to treatment; guideline; off-label treatment.

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Conflict of interest statement

Declaration of Conflicting Interests: The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Multiple Sclerosis International Federation (MSIF) is an alliance of national multiple sclerosis (MS) organisations. MSIF receives income from a wide range of sources, including health care and other companies, individuals, member organisations, campaigns, foundations and trusts. Over the last 5 years, MSIF received funding from the following companies: Biogen, Bristol Myers Squibb (formerly Celgene), MedDay, Merck, Mylan, Novartis, Roche, Sanofi Genzyme and Teva. Our independence and all our donations from the health care industry are governed by our policy: http://www.msif.org/wp-content/uploads/2017/09/Policyand-Practices-in-Relationships-with-the-Healthcare-Industry-2017.pdf. Joanna Laurson-Doube, Nick Rijke, Anne Helme and Peer Baneke have no relevant individual conflicts of interest. Brenda Banwell serves on the Board of ACTRIMS (Americas Committee for Treatment and Research in Multiple Sclerosis), Medical and Scientific Board of the MSIF, Medical Advisory Board of the Canadian Multiple Sclerosis Society, and Board of Directors of the American Academy of Neurology. Dr Banwell has received financial compensation from Novartis for efforts as a central magnetic resonance imaging (MRI) reviewer and as a speaker. Dr Banwell serves as a nonremunerated advisor to Novartis, Biogen-IDEC, Sanofi Aventis, Teva Neuroscience, and Celgene. Dr Banwell has received compensation from Medscape and donations for an educational residency fund, in lieu of personal compensation received from the Corpus for general paediatric MS lectures. Bernhard Hemmer has served on scientific advisory boards for Novartis; he has served as DMSC member for AllergyCare, Polpharma and TG therapeutics; he or his institution have received speaker honoraria from Desitin; his institution received research grants from Regeneron for MS research. He holds part of two patents: one for the detection of antibodies against KIR4.1 in a subpopulation of patients with MS and one for genetic determinants of neutralising antibodies to interferon. Shanthi Viswanathan is the Chair of the Malaysian Clinical Practice Guidelines on the Management of Multiple Sclerosis and has no other conflicts of interest with regard to the content of the current paper. Bassem Yamout has served on advisory boards for Sanofi, Bayer, Roche, Merck and Biogen. He received honoraria as speaker from Sanofi, Bayer, Roche, Merck, Biogen, Pfizer and Lundbeck. He received research grants from Novartis and Biogen. He served on steering committees for Merck.

Figures

Figure 1.
Figure 1.
The percentage of countries that do not have access to on-label DMTs for use, stratified for (a) income level and (b) region, as well as (c) the percentage of countries with no access to on-label high-efficacy DMTs according to income level. Total number of countries that supplied data for this question in the Atlas survey in parentheses (n = number of countries). Please note that we lack data from a number of countries around the world and that World Bank income classification comes with limitations of accuracy and heterogeneity within countries.
Figure 2.
Figure 2.
The percentage of countries that report using each DMT for MS. The number of countries for each DMT is in parentheses. Total number of countries that supplied data for this question in the Atlas survey is 102. Please note that mitoxantrone has regulatory approval in the United States and azathioprine in Germany.
See this image and copyright information in PMC

Comment in

References

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