Off-label use of orphan medicinal products: a Belgian qualitative study

Orphanet Journal of Rare Diseases

Volumn 11, Issue 1, 2016, Pages 1-9

  Dooms, Marc ^a   Cassiman, David ^a   Simoens, Steven ^b  

^a UNIVERSITY HOSPITALS LEUVEN   (Belgium)

^b UNIVERSITY OF LEUVEN   (Belgium)

Author keywords

Efficacy; Off label use; Orphan medicinal products; Rare diseases; Safety; Unlicensed use

Indexed keywords

ORPHAN DRUG;

ARTICLE; BELGIAN; CLINICAL PRACTICE; DRUG SAFETY; DRUG TOLERABILITY; EVIDENCE BASED MEDICINE; FUNDING; HUMAN; OFF LABEL DRUG USE; PATIENT SAFETY; PHARMACY STUDENT; QUALITATIVE RESEARCH; REIMBURSEMENT; BELGIUM; DRUG MANUFACTURE; RARE DISEASES;

BELGIUM; HUMANS; OFF-LABEL USE; ORPHAN DRUG PRODUCTION; RARE DISEASES;

EID: 84992697313     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-016-0507-y     Document Type: Article

References (18)

1. Liang BA, Mackey T. Health care policy. Reforming off-label promotion to enhance orphan disease treatment. Science. 2010;327:273-4.
2. Gupta SK, Nayak RP. Off-label use of medicine: perspective of physicians, patients, pharmaceutical companies and regulatory authorities. J Pharmacol Pharmacother. 2014;5:88-92.
3. European Commission. Regulation (EC) No. 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products. Off J Eur Communities. 2000;L 18:1-5.
4. Orphanet. Orphanet - The portal for rare diseases and orphan drugs. 2015. 2010. Ref Type: Online Source.
5. Medicines for Children. Unlicensed medicines. 2016. 11-4-2016. Ref Type: Online Source.
6. Kesselheim AS, Myers JA, Solomon DH, Winkelmayer WC, Levin R, Avorn J. The prevalence and cost of unapproved uses of top-selling orphan drugs. PLoS One. 2012;7:e31894.
7. Fund Rare Diseases and Orphan Drugs (managed by the King Baudouin Foundation). Recommendations and proposed measures for a Belgian plan for rare diseases - an outline of integrated and comprehensive health and social policy actions for patients with a rare disease. 2011. Ref Type: Online Source.
8. Weynants L, Schoonderbeek C, Weyne C. Off-label use and promotion: risks and potential liabitlity. Bio-Sci Law Rev. 2010;11:115-29.
9. Jellouli Y, Segers T, Vanderlocht S, Van Laer E, Van Lishout N. Research into the off-label use of orphan drugs in the Belgian context. Leuven: KU Leuven; 2014.
10. Article L. 5121-12-1 of the French Public Health Code (Code de la Santé Publique), as last amended by the law no. 2014-892 of 8 August 2014 (Loi de financement rectificative de la sécurité sociale pour 2014 (1)). 2014. Ref Type: Generic.
11. Italian Republic of Law Decree nr. 36 of 20 March 2014, as converted into law (in an amended version) by Law nr. 79 of 16 May 2014 on narcotics and psychotropics and on the use of less costly medicines by the National Health Service. 2014. Ref Type: Generic.
12. Vannieuwenhuysen C, Slegers P, Neyt M, Hulstaert F, Stordeur S, Cleemput I, et al. Towards a better managed off-label use of drugs. Brussels: Belgian Health Care Knowledge Centre (KCE); 2015. p. 252. Ref Type: Report.
13. Pope C, Mays N. Qualitative research in health care. 3rd ed. Oxford: Blackwell Publishing Ltd; 2006.
14. Eguale T, Buckeridge DL, Verma A, Winslade NE, Benedetti A, Hanley JA, et al. Association of Off-label drug use and adverse drug events in an adult population. JAMA Intern Med. 2016;176:55-63.
15. Frattarelli DA, Galinkin JL, Green TP, Johnson TD, Neville KA, Paul IM, et al. Off-label use of drugs in children. Pediatrics. 2014;133:563-7.
16. Janzen RW, Ludwig WD. Off-label therapy: current problems from the perspective of the Pharmaceutical Commission of the German Medical Profession. Z Rheumatol. 2012;71:108.
17. Magalhaes J, Rodrigues AT, Roque F, Figueiras A, Falcao A, Herdeiro MT. Use of off-label and unlicenced drugs in hospitalised paediatric patients: a systematic review. Eur J Clin Pharmacol. 2015;71:1-13.
18. Saiyed MM, Prajapati A, Shah G. Parents' awareness and perspective on off-label medicines use in children. J Pharmacol Pharmacother. 2015;6:88-91.