Study Design and First Results of a Non-Interventional Registry Study To Establish Long-Term Safety and Pharmaco-Economic Data on Fingolimod (Gilenya(R)) in Multiple Sclerosis Patients in Germany (PANGAEA) (P04.131)

Abstract

Objective: In order to investigate the safety and efficacy of fingolimod (Gilenya®) in everyday clinical practice, a 4000-patient national safety study is being conducted, in which pharmaco-economic data will also be collected (PANGAEA = P ost- A uthorization N on-interventional G erman s A fety study of Gil E ny A in RRMS patients). Background Fingolimod is licensed in Germany since April 2011 as escalation treatment for patients with relapsing-remitting multiple sclerosis (RRMS). Fingolimod is orally available and the first representative of a new substance class, the sphingosine-1-phosphate receptor modulators. Design/Methods: The aim of this non-interventional registry study is the prospective compilation of long-term data on safety and pharmaco-economic aspects of fingolimod in 4,000 RRMS patients from neurological clinics and practices. Recruitment is planned until end of 2012. The study consists of a 5-year observation period including pharmaco-economic data over 2 years from a sub-sample of 800 patients. In addition to compliance and a resources questionnaire, the following instruments will be used: UK NDS, PRIMUS, EQ5-D and TSQM-9. MSFC and SDMT questionnaires, as well as lipid-values will be monitored in another sub-population of 1000 patients. The Multiple Sclerosis Documentation System (MSDS) Project Development Group in Dresden adapted the patient management software, “MSDS 3D” to the use of fingolimod. In addition to the documentation of safety parameters, PANGAEA also fulfils a quality assurance function. RMP-measures such as monitoring of the first dose, ophthalmological examination and regular laboratory tests, are represented in PANGAEA and integrated into MSDS 3D. Results: Until October 2011, more than 750 patients have been enrolled. For the first time, a summary safety profile of patients documented until April 2012 will be presented. Conclusions: First PANGAEA results will be presented 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. Supported by: Novartis Pharma GmbH. Disclosure: Dr. Ziemssen has received personal compensation for activities with Biogen Idec, Bayer Healthcare, Novartis, Sanofi-Aventis Pharmaceuticals, Inc., Teva Neuroscience, and Synthon. Dr. Ziemssen has received research support for from Bayer Healthcare, Biogen Idec, Novartis, Teva Neuroscience, and Sanofi-Aventis Pharmaceuticals, Inc. Dr. Van Lokven has received personal compensation for activities with Novartis. Dr. Kempke has received personal compensation for activities with Biogen Idec and Novartis as a consultant. Dr. Meergans has received personal compensation for activities with Novartis as an employee.