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Preapproval and postapproval evidence on drugs for multiple sclerosis.
Neurology 2018; 90(21):964-973Neur

Abstract

OBJECTIVE

To review the evidence supporting the European Union marketing authorization of drugs for multiple sclerosis (MS) and assess how far postmarketing research addresses information gaps at the time of approval.

METHODS

Through its database, we identified drugs approved by the European Medicines Agency and gathered data on pivotal trials from the European Public Assessment Reports and corresponding publications. We searched Medline, Embase, Cochrane Library, and trial registries for postmarketing randomized controlled trials testing the drugs identified in any form of the disease.

RESULTS

Since approval of interferon and glatiramer up to 2017, the Agency has examined 10 drugs for the treatment of MS, and 8 were included in this study: alemtuzumab, daclizumab, dimethyl fumarate, fampridine, fingolimod, peginterferon-β-1a, natalizumab, and teriflunomide. We analyzed 16 pivotal trials enrolling almost 16,000 participants. Eleven compared new drugs to placebo, 5 to interferon-β-1a. Annualized relapse rate was the primary outcome in two-thirds and coprimary with disability progression in the 2 studies of alemtuzumab. Of the 52 postmarketing trials, 24 reported final results and 28 were ongoing, terminated, or completed but no results were available. None directly compared the approved drugs, thus leaving their respective therapeutic values unknown. Data on the prevention of disease progression were scarce: none of the disease-modifying drugs showed any effect on disability progression.

CONCLUSION

The lack of comparative evidence and data on clinical effectiveness hamper the assessment of therapeutic value and place in therapy of drugs approved for MS.

Authors+Show Affiliations

From IRCCS-Istituto di Ricerche Farmacologiche "Mario Negri" (C.G., V.B., S.G., R.B.); and Neuroimmunology and Neuromuscular Diseases Unit (S.R.), IRCCS-Fondazione Istituto Neurologico Carlo Besta, Milan, Italy. chiara.gerardi@marionegri.it.From IRCCS-Istituto di Ricerche Farmacologiche "Mario Negri" (C.G., V.B., S.G., R.B.); and Neuroimmunology and Neuromuscular Diseases Unit (S.R.), IRCCS-Fondazione Istituto Neurologico Carlo Besta, Milan, Italy.From IRCCS-Istituto di Ricerche Farmacologiche "Mario Negri" (C.G., V.B., S.G., R.B.); and Neuroimmunology and Neuromuscular Diseases Unit (S.R.), IRCCS-Fondazione Istituto Neurologico Carlo Besta, Milan, Italy.From IRCCS-Istituto di Ricerche Farmacologiche "Mario Negri" (C.G., V.B., S.G., R.B.); and Neuroimmunology and Neuromuscular Diseases Unit (S.R.), IRCCS-Fondazione Istituto Neurologico Carlo Besta, Milan, Italy.From IRCCS-Istituto di Ricerche Farmacologiche "Mario Negri" (C.G., V.B., S.G., R.B.); and Neuroimmunology and Neuromuscular Diseases Unit (S.R.), IRCCS-Fondazione Istituto Neurologico Carlo Besta, Milan, Italy.

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't
Review

Language

eng

PubMed ID

29695598

Citation

Gerardi, Chiara, et al. "Preapproval and Postapproval Evidence On Drugs for Multiple Sclerosis." Neurology, vol. 90, no. 21, 2018, pp. 964-973.
Gerardi C, Bertele' V, Rossi S, et al. Preapproval and postapproval evidence on drugs for multiple sclerosis. Neurology. 2018;90(21):964-973.
Gerardi, C., Bertele', V., Rossi, S., Garattini, S., & Banzi, R. (2018). Preapproval and postapproval evidence on drugs for multiple sclerosis. Neurology, 90(21), pp. 964-973. doi:10.1212/WNL.0000000000005561.
Gerardi C, et al. Preapproval and Postapproval Evidence On Drugs for Multiple Sclerosis. Neurology. 2018 05 22;90(21):964-973. PubMed PMID: 29695598.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Preapproval and postapproval evidence on drugs for multiple sclerosis. AU - Gerardi,Chiara, AU - Bertele',Vittorio, AU - Rossi,Silvia, AU - Garattini,Silvio, AU - Banzi,Rita, Y1 - 2018/04/25/ PY - 2017/10/24/received PY - 2018/02/26/accepted PY - 2018/4/27/pubmed PY - 2019/8/14/medline PY - 2018/4/27/entrez SP - 964 EP - 973 JF - Neurology JO - Neurology VL - 90 IS - 21 N2 - OBJECTIVE: To review the evidence supporting the European Union marketing authorization of drugs for multiple sclerosis (MS) and assess how far postmarketing research addresses information gaps at the time of approval. METHODS: Through its database, we identified drugs approved by the European Medicines Agency and gathered data on pivotal trials from the European Public Assessment Reports and corresponding publications. We searched Medline, Embase, Cochrane Library, and trial registries for postmarketing randomized controlled trials testing the drugs identified in any form of the disease. RESULTS: Since approval of interferon and glatiramer up to 2017, the Agency has examined 10 drugs for the treatment of MS, and 8 were included in this study: alemtuzumab, daclizumab, dimethyl fumarate, fampridine, fingolimod, peginterferon-β-1a, natalizumab, and teriflunomide. We analyzed 16 pivotal trials enrolling almost 16,000 participants. Eleven compared new drugs to placebo, 5 to interferon-β-1a. Annualized relapse rate was the primary outcome in two-thirds and coprimary with disability progression in the 2 studies of alemtuzumab. Of the 52 postmarketing trials, 24 reported final results and 28 were ongoing, terminated, or completed but no results were available. None directly compared the approved drugs, thus leaving their respective therapeutic values unknown. Data on the prevention of disease progression were scarce: none of the disease-modifying drugs showed any effect on disability progression. CONCLUSION: The lack of comparative evidence and data on clinical effectiveness hamper the assessment of therapeutic value and place in therapy of drugs approved for MS. SN - 1526-632X UR - https://www.unboundmedicine.com/medline/citation/29695598/Preapproval_and_postapproval_evidence_on_drugs_for_multiple_sclerosis_ L2 - http://www.neurology.org/cgi/pmidlookup?view=long&pmid=29695598 DB - PRIME DP - Unbound Medicine ER -