Post-authorisation assessment of orphan drugs

The Lancet

Volumn 386, Issue 10007, 2015, Pages 1940-1941

  Hollak, Carla E M ^a   Biegstraaten, Marieke ^a   Levi, Marcel ^a   Hagendijk, Rob ^b  

^a ACADEMIC MEDICAL CENTER   (Netherlands)

^b UNIVERSITY OF AMSTERDAM   (Netherlands)

Author keywords

[No Author keywords available]

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; GLYCOSPHINGOLIPID; ORPHAN DRUG;

CEREBROVASCULAR ACCIDENT; COST EFFECTIVENESS ANALYSIS; DRUG APPROVAL; DRUG COST; DRUG INDUSTRY; DRUG SURVEILLANCE PROGRAM; ENZYME THERAPY; FABRY DISEASE; FOLLOW UP; HEART FAILURE; HUMAN; KIDNEY FAILURE; LETTER; METABOLIC DISORDER; NEUROPATHY; PRIORITY JOURNAL; RARE DISEASE; DRUG MANUFACTURE; EUROPEAN UNION; ORGANIZATION AND MANAGEMENT; RARE DISEASES;

DRUG APPROVAL; EUROPEAN UNION; FABRY DISEASE; HUMANS; ORPHAN DRUG PRODUCTION; RARE DISEASES;

EID: 84947285240     PISSN: 01406736     EISSN: 1474547X     Source Type: Journal    
DOI: 10.1016/S0140-6736(15)00827-2     Document Type: Letter

References (5)

1. European Commission Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products (consolidated version 7/8/2009) http://ec.europa.eu/health/files/eudralex/vol-1/reg-2000-141-cons-2009-07/reg-2000-141-cons-2009-07-en.pdf (accessed Sept 18, 2015).
2. RJ Desnick, YA Ioannou, CM Eng Alpha-galactosidase A deficiency: Fabry disease CR Scriver AL Beaudet WS Sly D Valle The metabolic and molecular bases of inherited disease 8th edn. 2001 McGraw-Hill New York 3733 3774
3. M Banikazemi, J Bultas, S Waldek et al. Agalsidase-beta therapy for advanced Fabry disease: a randomised trial Ann Intern Med 146 2007 77 86
4. SM Rombach, BE Smid, MG Bouwman, GE Linthorst, MG Dijkgraaf, CE Hollak Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain Orphanet J Rare Dis 8 2013 47
5. European Union Committee of Experts on Rare Diseases Recommendation to the European Commission and Member States on improving informed decisions based on the clinical added value of orphan medicinal products information flow http://www.eucerd.eu/?post-type=document&p=1446 September, 2012 (accessed Sept 18, 2015).