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FRI0338 Therapeutic conformity to guidelines and drug-approval in adamantiades-behcet's disease: a retrospective analysis of a middle-european cohort
  1. TM Moosmann1,
  2. C Veraar1,
  3. A Frech2,
  4. G Ratzinger3,
  5. B Teuchner4,
  6. G Fraedrich2,
  7. M Schirmer1
  1. 1Internal Medicine VI
  2. 2Vascular Surgery
  3. 3Dermatology
  4. 4Ophthalmology, Lkh - Medizinische Universität Innsbruck, Innsbruck, Austria

Abstract

Background Adamantiades-Behcet's disease (ABD) is a chronic systemic vasculitic disease. Because of multiorgan involvement with diagnostic and therapeutic challenges for physicians, treatment decisions can be very difficult.

Objectives To retrospectively assess the use of medication for ABD-therapy in view of 1.) current guidelines and 2.) approval by authorities in a Middle-European tertiary care center.

Methods Data between 1997 and 2016 from a Middle-European ABD-cohort were retrospectively analysed. First, medical treatment was evaluated for conformity with the EULAR-recommendations for ABD-management and the anti-TNF therapy recommendations by Sfikakis et al. (1,2). Second, medical treatment was evaluated for use according to indications approved by authorities. Therefore official prescribing informations of the Bundesamt für Sicherheit im Gesundheitswesen (BASG)/European Medicines Agency (EMA), exemplary for Europe, and the Food and Drug Administration (FDA), exemplary for the USA, were screened (3–5). The study was approved by the local ethics committee.

Results A total of 174 medical interventions were identified in 76 patients. Until 2008, treatment of ABD was based only on few clinical trials. According to EULAR-recommendations 93,7% were considered as being treated appropriately, including 55.2% of therapeutic approaches exactly matching the recommendations. 88.9% of TNF-inhibitors were indicated according to the anti-TNF-therapy recommendations (n=8/9). Out of 27 used drugs only prednisolone was approved by BASG/EMA- and FDA-authorities for a general indication including ABD, and cyclosporin A had the specific BASG/EMA-indication for ABD-uveitis. Another 12 medications had the indications for different symptoms of ABD, and thirteen medications were not authority-approved for any ABD-treatment.

Conclusions Approvals by BASG/EMA- and FDA-authorities are often missing the indication of ABD. Therefore physicians not only face the complexity of ABD as a rare multiorgan disease, but also have to treat their ABD-patients with unapproved drugs. We propose that medications recommended by international guidelines for the management of rare diseases should be recognized by BASG/EMA- and FDA-authorities, even in case of low evidence.

References

  1. Hatemi G, Silman A, Bang D, et al. EULAR recommendations for the management of Behçet disease. AnnRheumDis. 2008;67:1656.

  2. Sfikakis PP, Markomichelakis N, Alpsoy E et al. Anti-TNF therapy in the management of Behçet's disease-review and basis for recommendations. Rheumatol. 2007;46:736.

  3. BASG. Arzneispezialitätenregister, 20/09/2016 [Internet]. https://aspregister.basg.gv.at/aspregister/faces/aspregister.jspx?_afrLoop=31622307107626619&_afrWindowMode=0&_adf.ctrl-state=kimc6lskg_4.

  4. EMA. European Medicines Agency - Find medicine - European public assessment reports [Internet] http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/landing/epar_search.jsp&mid=WC0b01ac058001d124.

  5. FDA. Drugs@FDA: FDA Approved Drug Products, citated 20.09.2016 [Internet]. Available from: http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm?fuseaction=Search.Search_Drug_Name.

References

Disclosure of Interest None declared

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