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Fingolimod-Compassionate-use-Programm

Fallstudie zum Konzept einer Therapieoption bei Multipler Sklerose vor Zulassung und Markteinführung

Fingolimod compassionate use program

Case study on the concept of a therapy option for multiple sclerosis prior to marketing approval

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Zusammenfassung

Hintergrund

Um den therapeutischen Bedarf bei Multipler Sklerose (MS) zu erfüllen, sind neue Immuntherapien mit einer anwenderfreundlichen Applikation und höherer Wirksamkeit bei guter Sicherheit erforderlich.

“Compassionate use”

Fingolimod sollte für Patienten mit besonderem Bedarf an einer alternativen Therapie mithilfe eines in der MS bisher einzigartigen Compassionate-use-Programms schon vor Zulassung zur Verfügung stehen. Teilgenommen haben 137 Zentren (75% neurologische Fachpraxen, 25% Kliniken). Während 19 Wochen erhielten 135 Patienten Fingolimod. Kasuistiken zu Krankheitsverläufen, die mit den bisherigen, häufig schlecht vertragenen Therapien unzureichend behandelbar waren, wurden beschrieben. Das Programm bot die Möglichkeit einer verbesserten Kontrolle der Erkrankung. Die unerwünschten Ereignisse lagen im Bereich des Erwarteten.

Schlussfolgerung

Das Fingolimod-Compassionate-use-Programm zeigt den Bedarf an der neuen Therapie auf. Patienten, die bisher nur unzureichend behandelt werden konnten, wurden mit einer Therapie versorgt, die Wirksamkeit, ein gutes Sicherheitsprofil und eine anwenderfreundlichen Darreichungsform bietet.

Summary

Background

In order to meet the needs of therapy of multiple sclerosis (MS) new immune therapies with a user-friendly application and better effectiveness together with good tolerability are necessary.

Compassionate use

With respect to its potential to improve MS therapy, patients with a high medical need were given access to Fingolimod even before marketing approval. Therefore, a compassionate use program unique in the field of MS was initiated. In total 137 centers participated (75 % outpatient neurologists and 25 % hospitals). Within 19 weeks 135 patients were enrolled to receive Fingolimod. The patients in the compassionate use program can be representatively described as showing hardly controllable disease activity and progression with currently available, often poorly tolerated therapy. The compassionate use program for these patients offered better control of the disease with Fingolimod. The adverse events were as expected.

Conclusions

The Fingolimod compassionate use program demonstrated the need for this new therapeutic option. Patients who were not yet sufficiently treated were provided with an effective therapy with a good safety profile and a user-friendly administration form.

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Interessenskonflikt

Der korrespondierende Autor weist für sich und seine Koautoren auf folgende Beziehungen hin: Prof. Dr. Haas, Dr. Linker und Prof. Dr. Hartung sind als Referenten und Studienärzte für die Firma Novartis Pharma GmbH tätig. Dres. Meergans, Ortler und Tracik sind Angestellte der Firma Novartis Pharma GmbH.

Das beschriebene Compassionate-use-Programm wurde von der Firma Novartis Pharma GmbH initiiert. Der vorliegende Originalartikel wurde mit Unterstützung eines bei Novartis angestellten Medical Writers (Dr. Karin Eichele) verfasst.

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Authors and Affiliations

  1. Klinik für Neurologie, Jüdisches Krankenhaus, Heinz-Galinski-Str. 1, 13347, Berlin, Deutschland

    J. Haas

  2. Neurologie, Universitätsklinikum Erlangen, Erlangen, Deutschland

    R.A. Linker

  3. Universitätsklinikum der Heinrich-Heine-Universität Düsseldorf, Düsseldorf, Deutschland

    H.P. Hartung & F. Tracik

  4. Novartis Pharma GmbH Nürnberg, Nürnberg, Deutschland

    M. Meergans, S. Ortler & F. Tracik

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  1. J. Haas
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  2. R.A. Linker
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  3. H.P. Hartung
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  4. M. Meergans
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  5. S. Ortler
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  6. F. Tracik
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Corresponding author

Correspondence to J. Haas.

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Haas, J., Linker, R., Hartung, H. et al. Fingolimod-Compassionate-use-Programm. Nervenarzt 83, 1575–1581 (2012). https://doi.org/10.1007/s00115-012-3612-8

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  • DOI: https://doi.org/10.1007/s00115-012-3612-8

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