Орфанные препараты, принципы их регистрации и применения
Аннотация
Об авторах
Александр Алексеевич Солдатов
Федеральное государственное бюджетное учреждение «Научный центр экспертизы средств медицинского применения» Министерства здравоохранения Российской Федерации
Россия
Россия
Жанна Ильдаровна Авдеева
Федеральное государственное бюджетное учреждение «Научный центр экспертизы средств медицинского применения» Министерства здравоохранения Российской Федерации
Россия
Россия
Наталья Александровна. Алпатова
Федеральное государственное бюджетное учреждение «Научный центр экспертизы средств медицинского применения» Министерства здравоохранения Российской Федерации
Россия
Россия
Николай Васильевич Медуницын
Федеральное государственное бюджетное учреждение «Научный центр экспертизы средств медицинского применения» Министерства здравоохранения Российской Федерации
Россия
Россия
Светлана Леонидовна Лысикова
Федеральное государственное бюджетное учреждение «Научный центр экспертизы средств медицинского применения» Министерства здравоохранения Российской Федерации
Россия
Россия
Список литературы
1. Dionisi-Vici C., Rizzo C., Burlina A.B., Caruso U., Sabetta G., Uziel G., et al. Inborn errors of metabolism in the Italian pediatric population: a national retrospective survey. J Pediatr. 2002; 140: 321-7.
2. Maron B.J., Estes N.A.M. Commotio cordis. New Engl. J Med. 2010; 62(10): 917-27.
3. Orphanet. Undated a. About Orphanet: Mission. Available from: http://www.orpha.net/consor/cgibin/Education_AboutOrphanet.php?lng=EN (accessed 4 August 2010).
4. de Vrueh R.L.A. Why R & D into Rare Diseases Matter. In Rare Diseases in the Age of Health 2.0, Communications in Medical and Care Compunetics 4. Berlin, Heidelberg: Springer-Verlag, 2014.
5. Kataria M.K., Garg M., Anand V., Bilandi A., Kukkar V., Bhandari A. An insight on regulations governing orphan diseases and drugs. RJPBCS 2011; 2(3): 373-85.
6. Rosenberg H., Davis M., James D., Pollock N., Stowell K. Malignant hyperthermia. Orphanet J Rare Dis. 2007; 2(21): 1750-72.
7. Cassidy S.B., Driscoll D.J. Prader-Willi syndrome. Eur J Human Genetics 2009; 17: 3-13.
8. Nussbaum R.L., McInnes R.R., Willard H.F. Thompson & Thompson Genetics in Medicine. Philadelphia, PA: Saunders. 2004.
9. Dietz H.C. New therapeutic approaches for Mendelian disorders. New Engl J Med. 2010; 1: 852-63.
10. Imel E.A., DiMeglio L.A., Hui S.L., Carpenter T.O., Econs M.J. Treatment of X-linked hypophosphatemia with calcitriol and phosphate increases circulating fibroblast growth factor 23 concentrations. J Clin Endocrinol Metabolism 2010; 95(4): 1846-50.
11. Smits P., Bolton A.D., Funari V., Hong M., Boyden E.D., Lu L., et al. Lethal skeletal dysplasia in mice and humans lacking the golgin GMAP-210. New Engl J Med. 2010; 362(3): 206-16.
12. Celgene. Thalomid®: System for Thalomide education and prescribing safety. Available from: http://www.thalomid.com/steps_program.aspx (accessed Aug, 9, 2010).
13. The Orphan Drug Act. United States Public Law № 97-414. Available from: http://www.fda.gov/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/SignificantAmendmentstotheFDCAct/OrphanDrugAct/default.htm.
14. Orphan Drug Regulations 21 CFR Part 316. Federal Register 2011; 76 № 202.
15. Orphan Drug Regulations 21 CFR Part 316. Federal Register 2013; 78 № 113.
16. Orphan Drug Part 316 e-CFR Data is current as of May 5, 2014 http://www.fda.gov/orphan/oda.htm.
17. Developing Products for Rare Diseases & Conditions. Available from: http://www.fda.gov/orphan/oda.htm.
18. Wasserstein JN., Karst KR. Orphan Drug Designation Not Sacrosanct- FDA Revokes Orphan Designation for TheraCLEC for EPI, but Other Exclusivity Issues Remain 2007. Available from: http://www.fdalawblog.net/fda_law_blog_hyman_phelps/2007/07/fda-revokes-alt.html.
19. Field M.J., Boat T.F., eds. Rare Diseases and Orphan Products. Accelerating Research and Development. Washington (DC): National Academies Press (US); 2010. Available from: http://thcc.or.th/ICD-10TM/ge70.htm.
20. Incentives and Regulatory Considerations in Orphan Drug/Medical Device Development - Situation in Japan. MHLW/PMDA, Japan. Speaker: Hiroshi Takeda. Reviewer, Office of New Drug III., PMDA.
21. PMDA Orphan Working Group. Available from: http://www.pmda.go.jp/english/service/projects_am_e.html.
22. Regulation (EC) № 141/2000 of the European parliament and of the council of 16 December 1999 on orphan medicinal products. Official J Eur Union 2000; 18(1): 1-5.
23. Commission Regulation (EC) № 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 J Eur Union 2000; 103(5): 1-4.
24. Communication from the Commission on Regulation (EC) № 141/2000 of the European Parliament and of the Council on orphan medicinal products. Official J Eur Union 2003; 178(2): 1-7.
25. Regulation (EC) № 726/2004 of the European parliament and of the council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency. Official J Eur Union 2004; 136(1): 1-33.
26. Commission Regulation (EC) № 507/2006 of 29 March 2006 on the conditional marketing authorisation for medicinal products for human use falling within the scope of Regulation (EC) № 726/2004 of the European Parliament and of the Council. Official J Eur Union 2006; 92(6): 1-4.
27. Regulation (EC) № 1901/2006 of the European parliament and of the council of 12 December 2006 on medicinal products for pediatric use and amending Regulation (EEC) № 1768/92, Directive 2001/20/EC., Directive 2001/83/EC and Regulation (EC) № 726/2004. Official J Eur Union 2004; 136(1): 1-33.
28. Commission Regulation (EC) № 2049/2005 of 15 December 2005 laying down, pursuant to Regulation (EC) № 726/2004 of the European Parliament and of the Council, rules regarding the payment of fees to, and the receipt of administrative assistance from, the European Medicines Agency by micro, small and medium-sized enterprises. Official J Eur Union 2005; 329(4): 1-4.
29. Communication from the Commission Guideline on aspects of the application of Article 8 (1) and (3) of Regulation (EC) № 141/2000: Assessing similarity of medicinal products versus authorised orphan medicinal products benefiting from market exclusivity and applying derogations from that market exclusivity. C (2008) 4077 final. Brussels; 2008.
30. Review of orphan designation at the time of granting/varying a marketing authorization (SOP/H/3190).
31. Orphan medicinal product designation (SOP/H/3049).
32. Checking-in and electronic filing of documentation for orphan medicinal product designation (SOP/H/3047).
33. Recommendation on elements required to support the medical plausibility and the assumption of significant benefit for an orphan designation (EMA/COMP/15893/2009 Final).
34. Procedure for orphan medicinal product designation. Guidance for sponsors (EMA/710915/2009 Rev. 11. 20 February 2014).
35. Orphan medicines in numbers (EMA/279601/2010).
36. Mitsumoto J., Dorsey E.R., Beck C.A., Kieburtz K., Griggs R.C. Pivotal studies of orphan drugs approved for neurological diseases. Ann Neurology 2009; 66 (2): 184-90.
37. Joppi R., Bertele V., Garattini S. Orphan drug development is progressing too slowly. Br J Clin Pharmacol. 2006; 61(3): 355-60.
38. Joppi R., Bertele V., Garattini S. Orphan drug development is not taking off. Br J Clin Pharmacol. 2009; 67(5): 494-502.
39. Brabers A.E.M., Moors E.H.M., van Weely S., de Vrueh R.L.A. Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe? Orphanet J Rare Dis. 2011; 6: 59-70.
40. Roos J.C., Hyry H.I., Cox T.M. Orphan drug pricing may warrant a competition law investigation. BMJ 2010; 16: 341.
41. Kim S. The Orphan Drug Act: How the FDA Unlawfully Usurped Market Exclusivity. Nw J Tech & Intell Prop 2013; 11 (6 Article 3): 541-59.
42. Tambuyzer E. Rare diseases, orphan drugs and their regulation: questions and misconceptions. Nat Rev Drug Discov 2010; 9(12): 921-9.
Рецензия
Для цитирования:
Солдатов А.А., Авдеева Ж.И., Алпатова Н.А., Медуницын Н.В., Лысикова С.Л. Орфанные препараты, принципы их регистрации и применения. БИОпрепараты. Профилактика, диагностика, лечение. 2015;(3):4-16.
For citation:
Soldatov A.A., Avdeeva Z.I., Alpatova N.A., Medunitsyn N.V., Lysikova S.L. Orphan drugs and the principles of their marketing authorization procedures and clinical use. BIOpreparations. Prevention, Diagnosis, Treatment. 2015;(3):4-16. (In Russ.)
Просмотров: 1181
Послать статью по эл. почте 