References
- Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products. OJ num. L 18 of 22.01.2000.
- The Committee for Orphan Medicinal Products and the European Medicines Agency secretariat. European regulation on orphan medicinal products: 10 years of experience and future perspectives. Nat. Rev. Drug Discov. 10(5), 341–349 (2010).
- Buckley BM. Clinical trials of orphan medicines. Lancet 371(9629), 2051–2055 (2008).
- Regnstrom J, Koenig F, Aronsson B et al. Factors associated with success of market authorisation applications for pharmaceutical drugs submitted to the European Medicines Agency. Eur. J. Clin. Pharmacol. 66(1), 39–48 (2010).
- Pariser AR, Slack DJ et al. Characteristics of rare disease marketing applications associated with FDA product approvals 2006–2010. Drug Discov. Today, 17(15–16), 898–904 (2012).
- Putzeist MM, Heemstra HE et al. Determinants for successful marketing authorisation of orphan medicinal products in the EU. Drug Discov. Today 17(7–8), 352–358 (2012).
- Rare diseases. European Parliament legislative resolution of 23 April 2009 on the proposal for a Council recommendation on a European action in the field of rare diseases. (COM(2008)0726 – C6-0455/2008 – 2008/0218(CNS)).
Websites
- European Commission. 1,000th designation of an orphan medicinal product: improving the lives of patients. IP/12/573; 7 June 2012. http://europa.eu/rapid/pressReleasesAction.do?reference=IP/12/573&format=HTML&aged=0&language=EN&guiLanguage=en (Accessed 8 June 2012)
- Ayme S, Rodwell C (Eds). 2011 Report on the state of the art of rare diseases activities in Europa of the European Union Committee of Experts on Rare Diseases – Part I: Overview of rare diseases activities in Europa and key developments in 2012. July 2011. www.eucerd.eu/upload/file/Reports/2011ReportStateofArtRDActivities.pdf (Accessed 6 June 2012)
- EurordisCare2: Survey of diagnostic delays, 8 diseases, Europe. (2004). http://archive.eurordis.org/article.php3?id_article=454 (Accessed 6 June 2012)
- The Project. ERA-Net E-Rare. www.e-rare.eu/content/project (Accessed 6 June 2012)
- Introduction to the International Rare Diseases Research Consortium. http://ec.europa.eu/research/health/medical-research/pdf/rare-diseases/irdirc-introduction_en.pdf (Accessed 4 June 2012)
- Guideline on clinical trials in small populations (CHMP/EWP/83561/2005). www.emea.europa.eu/pdfs/human/ewp/8356105en.pdf (Accessed 6 June 2012)
- Disease Registries in Europe, Orphanet Report Series, Rare Diseases collection. January 2011. www.orpha.net/orphacom/cahiers/docs/GB/Registries.pdf (Accessed 6 June 2012)
- What type of evidence is currently being considered in the development of clinical practice guidelines for rare diseases? – rapid report. IQWiG reports. Commission v01–10. Last updated 28-4-2011. www.iqwig.de/v10-01-what-type-of-evidence-is-currently-being.986.en.html?tid=1313&phlex_override_command=element (Accessed 5 June 2012)