5th Joint triennial congress of the European and Americas Committees
for Treatment and Research in Multiple Sclerosis
Amsterdam, The Netherlands

19.10.2011 - 22.10.2011
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Home - 20.10.2011 - Long-term treatment monitoring 1

Long-term treatment monitoring 1

Thursday, October 20, 2011, 15:30 - 17:00

Study design of a non-interventional registry study to establish long-term safety and pharmaco-economic data on fingolimod (Gilenyaź) in multiple sclerosis patients (PANGAEA)

T. van Lokven, R. Kempcke, T. Ziemssen, M. Meergans (Nuremberg, Dresden, DE)

Background: Fingolimod (Gilenyaź) has been licensed in Germany since April as escalation treatment for multiple sclerosis patients with relapsing-remitting multiple sclerosis (RRMS). Fingolimod is orally available and the first compound of a new substance class, the sphingosine 1-phosphate receptor modulators. An extensive development program confirmed the clinical efficacy and superiority in terms of relapse rate reduction and MRI parameters compared to a standard therapy (interferon beta 1a im.). In order to investigate the safety and efficacy of fingolimod in everyday clinical practice, a national safety study is being conducted (PANGAEA = Post-Authorization Non-interventional German sAfety study of GilEnyA in RRMS patients).
Study design: The aim of this 5-year non-interventional registry study is the prospective compilation of long-term safety and pharmaco-economic data for fingolimod in ca. 4,000 RRMS patients from German neurological clinics and practices.
Over a period of 2 years, pharmaco-economic data will be collected from a sub-population of 800 patients. In addition to compliance and a resource utilization questionnaire, the following instruments will be used: the U.K. Neurological Disability Scale (UK NDS), PRIMUS- A and L, EQ5-D, and the Treatment Satisfaction Questionnaire for Medication 9 (TSQM-9). MSFC testing and SDMT questionnaires, as well as blood lipid levels will be monitored in another subpopulation.
Methods: Together with the Multiple Sclerosis Documentation System (MSDS) Project Development Group in Dresden, the patient management software, MSDS 3D, was adapted to the use of fingolimod. Thanks to its modular structure, physicians are guided through the recommended investigations, which are carried out at study start and quarterly appointments. In addition to the documentation of safety parameters, PANGAEA also fulfils a quality assurance function: the data acquisition reflects the Risk Management Plan of the European Medicines Agency (EMA) for fingolimod.
The measures undertaken in parallel with treatment, such as monitoring the administration of the first dose, ophthalmological examination after 3-4 months and regular laboratory tests, are represented in PANGAEA and integrated into the documentation program.
Summary: Annual interim analyses are planned and are intended to deliver continuous information and transparency with regard to the safety profile of fingolimod in patients with RRMS under real-life conditions.

T. Ziemssen and R. Kempcke have reiceived compensation for serving as consultant and speaker for Novartis Pharma GmbH. Matthias Meergans and Tom van Lokven are employees of Novartis Pharma GmbH.