References Orphandrugs and rare diseases at glance Available at http://www.ema.europa.eu/docs/en_GB/document_library/Other/2010/01/WC500069805.pdf Hoppu, K.: Paediatric clinical pharmacology - at the beginning of new era, Eur J Clin Pharmacol, 2008, 64, p. 201-205 Regulation (EC) No 141/2000 of the European parliament and of the council. Off J Eur Commun, 43, 2000, L18, p. 1-5. Regulation (EC) No 847/2000, Off J EurCommun, 43, 2000, L103, p. 5-8. Regulation (EC) No 1901/2006 of the European Parliament and of the Council;on medicinal products for paediatric use
Medical and scientific knowledge about rare diseases is minimal or lacking, thus making research difficulties for pharmaceutical industry. Orphan drugs in EU are under supervision of European Commission, European medical agency (EMA) and Committee for orphan medicinal products (COMP).
The presentation provides a brief review of all supportive incentives in the field of orphan medicinal products as: the European orphan medicinal product (OMP) regulation, Guideline on Clinical Trials in Small Populations and Commission Regulation (EC) No 2049/2005 / support of small and medium enterprises (SMEs). It also introduces the concept of Clinical added value of orphan medicinal products, as one of the key instruments to increase the availability of orphan medicinal products in the member states. Separately it stresses the necessity of Health technology assessment implementation in whole process of orphan medicinal product development as well as the implementation of the Europlan indicators into the Slovak National plan
Even if organophosphorus (OP) nerve agents were banned entirely, their presence would remain a problem as weapons of terror (like in Syria). Oxime antidotes currently used in medical practice still fall short of their therapeutic purpose, as they fail to fully restore the activity of cholinesterases, the main target for OPs. As orphan drugs, these antidotes are tested too seldom for anybody’s benefit. Over the last few decades, search for improved reactivators has reached new levels, but the translation of data obtained in vitro to in vivo application is still a problem that hinders efficient therapy. In this study, we tested the strengths and weaknesses of extrapolating pyridinium oxime antidotes reactivation efficiency from in vitro to in vivo application. Our results show that this extrapolation is possible with well-determined kinetic constants, but that it also largely depends on oxime circulation time and its tissue-specific distribution. This suggests that pharmacokinetic studies should be planned at the early stages of antidote development. Special attention should also be given to improving oxime distribution throughout the organism to overcome this major constraint in improving overall OP therapy.
(Replagal®) and agalsidase bèta (Fabrazyme®) for Fabry disease. Diemen: CVZ; 2012. http://www.cvz.nl/binaries/live/cvzinternet/hst_content/nl/documenten/rapporten/2012/rpt1211-ziekte-van-fabry.pdf. 4. Letter of Ministry of Health 29 January 2013. The Netherlands. 5. Letter of Ministry of Health 3 October 2013. The Netherlands. 6. National Institute for Health and Care Excellence. Appraising OrphanDrugs. Last updated: 14 April 2008. www.nice.org.uk/aboutnice/whoweare/seniormanagementteam/meetings/2005/12july2005/appraising_orphan_drugs.jsp; www
References COUNCIL RECOMMENDATION of 8 June 2009 on an action in the field of rare diseases (2009/C 151/02) Off J Eur U C. 151:7 - 10. (http://eurlex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:C:2009:151:0007:0010:EN:PDF). Accessed February 20 2013. Foltánová T et al., Orphandrugs used for the treatment in pediatric patients in the Slovak republic. Acta Fac. Pharm. Univ. Comen. 2012, Suppl VI:28 - 34. (http://www.degruyter.com/view/j/afpuc.2012.lix.issue-0/v10219-012-0001-0/v10219-012-0001-0.xml?format=INT) Accessed February 20 2013. Kovács L. et al. National
the last 20 years, fundamental technologies for minimal genome construction have been developed, and in the future researchers will build ideal stable hosts using the least genome for scientific and industrial aims ( 11 ). Genetic circuits can be easily designed by integrating DNA into a minimal genome. This will accelerate the construction of customized industrial genomes for the biologic recovery of environmental toxins, the manufacture of beneficial drugs and chemicals, or the creation of renewable energy sources. The first FDA’s OrphanDrug Act was offered by US
, 2011;6,143-148. WESTERMARK, K. - HOLM, B.B. - SODERHOLM, M. - LLINARES-GARCIA, J. - RIVIERE, F. - AARUM, S. - BUTLEN-DUCUING, F. - TSIGKOS, S. - WILKKACHLICKA, A. - N'DIAMOI, C. - FOLTÁNOVÁ, T. European regulation on orphan medicinal products: 10 years of experience and future perspectives. Nature Rev Drug Discov. 2011;10:341-349. KUŽELOVÁ, M. - KUBAČKOVÁ, K. - PALÁGYI, M. - ŠMÍD, M. Hope for patients with rare diseases - "orphan" drugs. [in slovak]. Čas Lék čes. 2006;145:296-300. VAVŘÍK, M. - MATEJKA, P. - ŠVEC, P. A Survey into Smokers' Interest in Quitting Smoking
in pigs from Slovakia, with evaluation of size, fertility and number of hydatid cysts. Helminthologia, 46 (3): 151 - 158. DOI: 10.2478/s11687-009-0029-4 VUITTON A. D., BRESSON-HADNI, S. (2014): Alveolar echinococcosis: evaluation of therapeutic strategies. Expert Opin. OrphanDrugs, 2 (1): 67 - 86. DOI: 10.1517/21678707.2014.870033 YOLASIGMAZ, A., REITEROVÁ K., TURK, M., REYHAN, E., BOZDAG, A.D., KARABABA, A.O., ALTINTAS NU., ALTINTAS, NA. (2006): Comparison of serological and clinical fi ndings in turkish patients with cystic echinococcosis. Helminthologia, 43 (4
therapeutic approaches in Wilson disease. A long-termfollow up study.Ann Hepatol. 2012 Nov-Dec; 11(6):907-14. PMID: 23109455 15. Ping CC, Hassan Y, Aziz NA, Ghazali R, Awaisu A.Discontinuation of penicillamine in the absence of alternative orphandrugs (trientine-zinc): a case of decompensated liver cirrhosis in Wilson’s disease. J Clin Pharm Ther. 2007 Feb;32(1):101-7. PMID: 17286794 16. Dong QY, Wu ZY. Advance in the pathogenesis and treatment of Wilson disease. Transl Neurodegener. 2012 Nov;1(1):23. doi: 10.1186/2047-9158-1-23 17. Lowette KF, Desmet K, Witters P, Laleman
-08-3105 PMID:15546948 Greten TF, Jaffee EM. Cancer vaccines. J Clin Oncol 1999;17(3):1047-60. PMID:10071300 OrphanDrug Status Awarded for O-Vax Vaccine. Vaccine Weekly web site. http://www.newsrx.com/newsletters/Vaccine-Weekly/2000-10-18/200010183338VW.html Orphan Product Designation. Food and Drug Administration web site. http://www.fda.gov/orphan/DESIGNAT/index.htm Product Pipeline. Avax Technologies web site. http://www.avax-tech.com/tech/product.php Cumulative list of all products that have received Orphan Designation. Food and Drug Administration (FDA) web site