Drugs for rare diseases: Mixed assessment in Europe

Prescrire International

Volumn 16, Issue 87, 2007, Pages 36-42

[No Author Info available]

Author keywords

[No Author keywords available]

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; ALGLUCERASE; ANAGRELIDE; ARSENIC TRIOXIDE; BLOOD CLOTTING FACTOR 9; BOSENTAN; BUSULFAN; CARBIDOPA PLUS LEVODOPA; CARGLUMIC ACID; CELECOXIB; CLADRIBINE; IBUPROFEN; ILOPROST; IMATINIB; IMIGLUCERASE; LARONIDASE; MERCAPTAMINE; MIGLUSTAT; MILTEFOSINE; MITOTANE; NITISINONE; OMEGA CONOTOXIN MVIIA; ORPHAN DRUG; PEGVISOMANT; PHOTOFRIN; SILDENAFIL; TASONERMIN; UNINDEXED DRUG; ZINC ACETATE;

ACROMEGALY; ADRENAL CANCER; ARTICLE; CLINICAL TRIAL; CYSTIC FIBROSIS; DRUG APPROVAL; DRUG CONTROL; DRUG COST; DRUG EFFICACY; DRUG INDICATION; DRUG INDUSTRY; DRUG LEGISLATION; DRUG MARKETING; DRUG RESEARCH; DRUG SAFETY; DUCHENNE MUSCULAR DYSTROPHY; ERECTILE DYSFUNCTION; EUROPE; FABRY DISEASE; GASTROINTESTINAL STROMAL TUMOR; GAUCHER DISEASE; HEALTH CARE ACCESS; HEALTH CARE AVAILABILITY; HEALTH CARE POLICY; HUMAN; LEUKODYSTROPHY; MEDICAL INFORMATION; MEDICAL SPECIALIST; MYELOID LEUKEMIA; PARKINSON DISEASE; PHARMACEUTICAL CARE; PROGERIA; PROMYELOCYTIC LEUKEMIA; PULMONARY HYPERTENSION; RARE DISEASE; THROMBOCYTHEMIA; VISCERAL LEISHMANIASIS; DRUG MANUFACTURE; ECONOMICS; HEALTH CARE DELIVERY; LEGAL ASPECT; UNITED STATES;

EUROPE; HEALTH SERVICES ACCESSIBILITY; HUMANS; LEGISLATION, DRUG; ORPHAN DRUG PRODUCTION; RARE DISEASES; UNITED STATES;

EID: 33847745042     PISSN: 11677422     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (68)

1. "Regulation (EC) no 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products" Official Journal of the European Communities 22 January 2000: L18/1-L18/5.
2. European Medicines Agency "COMP report to the Commission in relation to article 10 of Regulation 141/2000 on orphan medicinal products" 25 July 2005: 91 pages.
3. Commission of the European communities" Commission staff working document on the experience acquired as a result of the application of Regulation (EC) no 141/2000 on orphan medicinal products and account of the public health benefits obtained" 20 June 2006.Website http://europa.eu.int/comm/enterprise/pharmaceuticals/index_en.htm: 32 pages.
4. Prescrire Rédaction "Médicaments orphelins. L'Europe comble son retard" Rev Prescrire 1998; 18 (190): 868-869.
5. Prescrire Rédaction "Médicaments orphelins. Le règlement européen enfin adopté" Rev Prescrire 2000; 20 (206): 382-383.
6. Orphanet "Les maladies rares" Website http://www.orpha.net accessed 7 July 2006: 4 pages.
7. Orphanet "Prévalence des maladies rares: une enquête bibliographique" June 2006: 14 pages.
8. French Ministère de la santé et de la protection sociale "Plan national maladies rares 2005-2008" 20 November 2004: 37 pages.
9. European organisation for rare diseases "Rare diseases: understanding this public health priority" 2005: 12 pages.
10. European organisation for rare diseases "Eurordis-Care results. Survey results summary" 21 June 2005: 2 pages.
11. Expert group of the rare diseases task force "Overview of current centres of reference on rare diseases in the EU" September 2005: 19 pages.
12. Alcimed "Étude relative aux médicaments orphelins. Phase I. Panorama des conditions de commercialisation des médicaments orphelins en Europe": 38 pages.
13. Académie nationale de pharmacie "Sur la mise à disposition de médicaments orphelins destinés aux traitements de maladies rares et graves" June 2005: 12 pages.
14. Agence française de sécurité sailitaire des produits de santé "Rapport annuel 2005": 142 pages.
15. European Medicines Agency "Status report on the implementation of the European parliament legislation on orphan medicinal products" 30 March 2001: 3 pages.
16. Prescrire Editorial Board "Le point de vue d'un industriel spédalisé" Rev Prescrire 1998; 18 (190):869-870.
17. "Commission Regulation (EC) No 847/2000 of 27 April 2000 laying down the provisions for implementation of the criteria for designation of a medicinal product as an orphan medicinal product and definitions of the concepts 'similar medicinal product' and 'clinical superiority'" Official Journal of the European Communities 28 April 2000: L103/5-L103/ 8.
18. Héron E "Les médicaments orphelins en Europe" Méd Sci 2005; 21 (no spécial): 66-68.
19. European Commission "Draft for public consultation. European Commission guideline on aspects of the application of article 8 of regulation (EC) no 141/2000: Assessment of similarity and/or clinical superiority of orphan medicinal products when assessing marketing authorisation applications and variations" 2004 Website http://europa.eu.int/comm/enterprise/pharmaceuticals/index_en.htm: 20 pages.
20. European organisation forrare diseases "Eurordis survey of orphan drugs availability and pricing in EU member states" 2003: 10 pages.
21. o. Premier traitement substitutif pour la maladie de Fabry" Rev Prescrire 2002; 22 (234): 817 (full version available on www.prescrire.org: 4 pages).
22. Prescrire Editorial Staff "Agalsidase" Prescrire Int 2003; 12 (67): 168-171.
23. Prescrire Editorial Staff "Arsenic trioxyde" Prescrire Int 2004; 13 (72): 135-137.
24. Prescrire Editorial Staff "Bosentan" Prescrire Int 2005; 14 (77): 94-98.
25. Prescrire Editorial Staff "Imatinib in chronic myeloid leukaemia" Prescrire Int 2003; 12 (49): 49-52.
26. Prescrire Editorial Staff "Imatinib in first-line treatment for chronic myeloid leukaemia" Prescrire Int 2003; 12 (68): 216-218.
27. Prescrire Editorial Staff "Imatinib in gastrointestinal stromal tumours" Prescrire Int 2003; 12 (67): 163-164.
28. Prescrire Editorial Staff "Pegvisomant" Prescrire Int 2005; 14 (75): 10-13.
29. Prescrire Editorial Staff "Miglustat" Prescrire Int 2005; 14 (79): 168-170.
30. Prescrire Editorial Staff "Imiglucérase" Prescrire Int 2005; 14 (80): 221.
31. Prescrire Editorial Staff "Carglumic acid" Prescrire Int 2004; 13 (69): 3-4.
32. Prescrire Editorial Staff "Laronidase" Prescrire Int 2004; 13 (74): 217.
33. Prescrire Editorial Staff "Iloprost in pulmonary hypertension" Prescrire Int 2006; 15 (83): 101.
34. Prescrire Editorial Staff "Celecoxib in prevention of colorectal cancer" Prescrire Int 2006; 15 (81): 13-15.
35. Prescrire Editorial Staff "Porfimer in endobrachyesophagus" Prescrire Int 2006; 15 (82): 58-59.
36. o)" Rev Prescrire 2006; 26 (270): 180.
37. o. Un traitement palliatif des cancers surrénaliens" Rev Prescrire 1998; 18 (183): 264-266 + Prescrire Rḑaction "Mitotane: forme phannaceutique différente et nouveau nom" Rev Prescrire 2005; 25 (265): 661.
38. Prescrire Editorial Staff "Ibuprofen in patent ductus arterious" Prescrire Int 2006; 15 (85): 181.
39. Prescrire Editorial Staff "Zinc in Wilson's disease" Prescrire Int 2006; 15 (83): 101.
40. Prescrire Editorial Staff "Anagrelide in essential thrombocythaemia" Prescrire Int 2006; 15 (83): 83-86.
41. o": 35 pages; posted on the EMEA website on 12 September 2006.
42. o": 33 + 10 pages; posted on the EMEA website on 22 June 2006.
43. o). Tyrosinémie de type 1: un médicament efficace" Rev Prescrire 2006; 26 (276): 654 (full version available on www.prescrire.org: 3 pages) (to be translated in Prescrire International - April issue).
44. o 50 mng gélules" January 2005: 6 pages.
45. o. Un autre recours médicamenteux" Rev Prescrire 2006; 26 (277): 735.
46. Prescrire Rédaction "La, maladie de Fabry. Une maladie enzymatique très rare" Rev Prescrire 2002; 22 (234): 817 (full version available on www.prescrire.org: 6 pages).
47. Prescrire Rḑaction "Traitement de la leucémie aiguë promyélocytaire" Rev Prescrire 2004, 24 (248): 218.
48. Prescrire Rédaction "L'hypertension artérielle pulmonaire. Grave quand elle est symptornatique" Rev Prescrire 2004; 24 (256): 843-846.
49. Prescrire Rédaction "La leucémie myéloïde chronique. Des traiternents souventpeu satisfaisants" Rev Prescrire 2002; 22 (234): 840-842.
50. Prescrire Rédaction "Les tumeurs stromales du tube digestif" Rev Prescrire 2003; 23 (239): 379-380.
51. Prescrire Editorial Staff "Acromegaly and its treatment" Prescrire Int 2005; 14 (75): 12-13.
52. Prescrire Editorial Staff "Gaucher's disease" Prescrire Int 1996; 5 (23): 72.
53. Prescrire Rédaction "Les déficits enzymatiques de la synthèse de l'urée, maladies orphelines" Rev Prescrire 2001; 21 (214): 109-110.
54. Prescrire Rédaction "La mucopolysaccharidose de type I" Rev Prescrire 2004, 24 (253): 575-5-575-6.
55. Prescrire Rédaction "Polypose adénomateuse familiale. Surveillance coloscopique et colectomie systématique" Rev Prescrire 2005; 25 (265): 691-695.
56. Prescrire Rédaction "L'enclobrachycesophage, alias cesophage deBarrett" Rev Prescrire 2005; 25 (267): 849-851.
57. Prescrire Rédaction "La leucémie à tricholeucocytes" Rev Prescrire 2006; 26 (270): 209-1-209-2.
58. Prescrire Rédaction "Canal artériel persistant du prématuré" Rev Prescrire 2006; 26 (270): 203-205.
59. Prescrire Rédaction "La maladie de Wilson. Limiterla surcharge en cuivre" Rev Prescrire 2001; 21 (216): 258-259.
60. Prescrire Rédaction "La thrombocytémie essentielle. Cytotoxiques et antiagrégants trap peu évalués" Rev Prescrire 2006; 26 (269): 123-125.
61. Prescrire Rédaction "La tyrosinémie héréditaire de type 1" Rev Prescrire 2006; 26 (276): 697-1-1.
62. Joppi R et al. "Orphan drug development is progressing too slowly" Br J Clin Pharmacol 2006; 61 (3): 355-360.
63. Prescrire Editorial Staff "Comparative advantage of new drugs: French authorities are not sufficiently demanding" Prescrire Int 2005; 14 (76): 75-79.
64. Philipson L "Medical research activities, funding, and creativity in Europe. Comparison with research in the United States" JAMA 2005: 294 (11): 1394-1398.
65. European organisation for rare diseases "01. Introduction" Site Internet) http://www.eurordis.org accessed 10 July 2006: 2 pages.
66. "Accord cadre entre le Comité économique des produits de santé et les entreprises du médicament pour la période 2003-2006" 2003: 12 pages.
67. European Commission "Inventory of community and Member States" incentive measures to aid the research, marketing, development and availability of orphan medicinal products. Revision 2005" 7 September 2006: 28 pages.
68. o - Hypertension artérielle pulmonaire: par voie orale, pas mieux que le bosentan" Rev Prescrire 2006; 26 (274): 492 (full version available on www.prescrire. org: 4 pages).