title: The PANGAEA study design - a prospective, multicenter, non-interventional, long-term study on fingolimod for the treatment of multiple sclerosis in daily practice.
abstract: BACKGROUND
  Fingolimod (Gilenya) is an oral medication for patients with highly active relapsing-remitting Multiple Sclerosis (RRMS). Clinical trials and post-marketing experience on more than 114,000 patients have established a detailed safety profile. Total patient exposure now exceeds 195,000 patient-years as stated in the last financial report (Dec 2014) of the Novartis Pharma AG, Basel, Switzerland. However, less is known about the safety of long-term fingolimod use in daily practice. Here, we describe the study design of PANGAEA (Post-Authorization Non-interventional German sAfety of GilEnyA in RRMS patients), a prospective, multicenter, non-interventional, long-term study to collect safety, efficacy, and pharmacoeconomic data on RRMS patients treated with fingolimod (0.5 mg/daily) under real-world conditions in Germany.
  
  
  METHODS
  PANGAEA is striving to assess a real-world safety and efficacy profile of fingolimod, based on data from 4,000 RRMS patients, obtained during a 60-month observational phase. A pharmacoeconomic sub-study of 800 RRMS patients further collects patient-reported outcome measures of disability, quality of life, compliance, treatment satisfaction, and usage of resources during a 24-month observational phase. Descriptive statistical analyses of the safety set as well as of stratified subgroups such as patients with concomitant diabetes mellitus and pretreated patients (e.g., natalizumab) will be conducted.
  
  
  CONCLUSIONS
  PANGAEA seeks to confirm the current safety profile of fingolimod obtained in phase I-III clinical trials. The study design presented here will additionally provide guidance on the therapeutic use of fingolimod in clinical practice and possibly assists physicians in making evidence-based decisions.
authors.affiliation: Zentrum für klinische Neurowissenschaften, Klinik und Poliklinik für Neurologie, Universitätsklinikum Carl Gustav Carus Dresden, Technische Universität Dresden, Fetscherstr. 43, D-01307, Dresden, Germany. tjalf.ziemssen@uniklinikum-dresden.de.
authors.forename: Tjalf
authors.initials: T
authors.lastname: Ziemssen
authors.affiliation: Zentrum für klinische Neurowissenschaften, Klinik und Poliklinik für Neurologie, Universitätsklinikum Carl Gustav Carus Dresden, Technische Universität Dresden, Fetscherstr. 43, D-01307, Dresden, Germany. Raimar.Kern@uniklinikum-dresden.de.
authors.forename: Raimar
authors.initials: R
authors.lastname: Kern
authors.affiliation: Novartis Pharma GmbH, Roonstr. 25, D-90429, Nuernberg, Germany. christian.cornelissen@novartis.com.
authors.forename: Christian
authors.initials: C
authors.lastname: Cornelissen
mesh_terms: D004345:Drug Industry; D005260:Female; D000068876:Fingolimod Hydrochloride / Q000191:economics / Q000627:therapeutic use*; D005858:Germany; D006801:Humans; D007166:Immunosuppressive Agents / Q000191:economics / Q000627:therapeutic use*; D008297:Male; D020529:Multiple Sclerosis, Relapsing-Remitting / Q000188:drug therapy*; D061214:Patient Safety; D011358:Product Surveillance, Postmarketing; D011409:Propylene Glycols / Q000627:therapeutic use; D011446:Prospective Studies; D011788:Quality of Life; D012107:Research Design
chemical_list: D007166:Immunosuppressive Agents; D011409:Propylene Glycols; D000068876:Fingolimod Hydrochloride