References
- 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).
- Hollak CE, Aerts JM, Ayme S, Manuel J. Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders. Orphanet. J. Rare Dis. 6, 16 (2011).
- Picavet E, Dooms M, Cassiman D, Simoens S. Orphan drugs for rare diseases: grounds for special status. Drug Develop. Res. 73(3), 115–119 (2012).
- Pinxten W, Denier Y, Dooms M, Cassiman JJ, Dierickx K. A fair share for the orphans: ethical guidelines for a fair distribution of resources within the bounds of the 10-year-old European Orphan Drug Regulation. J. Med. Ethics 38(3), 148–153 (2011).
- McCabe C. Balancing economic, ethical and equity concerns in orphan drugs and rare diseases. Eur. J. Hospital Pharmacy Practice. 16(4), 22–25 (2010).
- Pypops U. Patient perspective on CT involvement: are they listening to my needs? Orphanet. J. Rare Dis. 7( Suppl. 7), A39 (2012).
Websites
- Fondation Brocher. www.brocher.ch
- Final CAVOD – Creation of process for the exchange of knowledge between Member States and European authorities on the scientific assessment of the clinical added value for orphan medicines 2011. http://ec.europa.eu/eahc/documents/news/CAVOD_Final%20Report_vf_corrected_281011.pdf