Evidence supporting regulatory-decision making on orphan medicinal products authorisation in Europe: methodological uncertainties

Author:

Pontes CaridadORCID,Fontanet Juan Manuel,Vives Roser,Sancho Aranzazu,Gómez-Valent Mònica,Ríos José,Morros Rosa,Martinalbo Jorge,Posch Martin,Koch Armin,Roes Kit,Oude Rengerink Katrien,Torrent-Farnell Josep,Torres Ferran

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Genetics(clinical),General Medicine

Reference51 articles.

1. European Parliament C of the EU. 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. https://ec.europa.eu/health/sites/health/files/files/eudralex/vol-1/reg_2000_141_cons-2009-07/reg_2000_141_cons-2009-07_en.pdf . Published 2000. Accessed 12 Oct 2018..

2. Jonker A, Mills A, Lau L, et al., eds. Small Population Clinical Trials: Challenges in the Field of Rare Diseases. 2016. http://www.irdirc.org/wp-content/uploads/2017/12/SPCT_Report.pdf . Accessed 12 Oct 2018.

3. Roberts SA, Allen JD, Sigal EV. Despite criticism of the FDA review process, new Cancer drugs reach patients sooner in the United States than in Europe. Health Aff. 2011;30(7):1375–81. https://doi.org/10.1377/hlthaff.2011.0231 .

4. Kesselheim AS, Myers JA, Avorn J. Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer. JAMA. 2011;305(22):2320–6. https://doi.org/10.1001/jama.2011.769 .

5. Jonsson B, Bergh J. Hurdles in anticancer drug development from a regulatory perspective. Nat Rev Clin Oncol. 2012;9(4):236–43. https://doi.org/10.1038/nrclinonc.2012.14 .

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