title: [Fingolimod compassionate use program: case study on the concept of a therapy option for multiple sclerosis prior to marketing approval].
abstract: 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.
  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.
authors.affiliation: Klinik für Neurologie, Jüdisches Krankenhaus, Heinz-Galinski-Str. 1, 13347 Berlin, Deutschland. Judith.Haas@jkb-online.de
authors.forename: J
authors.initials: J
authors.lastname: Haas
authors.forename: R A
authors.initials: RA
authors.lastname: Linker
authors.forename: H P
authors.initials: HP
authors.lastname: Hartung
authors.forename: M
authors.initials: M
authors.lastname: Meergans
authors.forename: S
authors.initials: S
authors.lastname: Ortler
authors.forename: F
authors.initials: F
authors.lastname: Tracik
mesh_terms: D000328:Adult; D016022:Case-Control Studies; D015897:Comorbidity; D057176:Compassionate Use Trials; D017277:Drug Approval; D064420:Drug-Related Side Effects and Adverse Reactions / Q000453:epidemiology*; D005060:Europe / Q000453:epidemiology; D000068876:Fingolimod Hydrochloride; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008297:Male; D008875:Middle Aged; D009103:Multiple Sclerosis / Q000175:diagnosis / Q000188:drug therapy* / Q000453:epidemiology*; D015995:Prevalence; D011409:Propylene Glycols / Q000627:therapeutic use*; D013110:Sphingosine / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D007166:Immunosuppressive Agents; D011409:Propylene Glycols; D000068876:Fingolimod Hydrochloride; D013110:Sphingosine