Bibliography
- European regulation on orphan medicinal products: 10 years of experience and future perspectives. Nat Rev Drug Discov 2011;10:341-9
- 2014 Report on the State of the Art of Rare Disease Activities in Europe. EUCERD. 2014
- Letter Executive Director EMA. Fee reductions for designated orphan medicinal products. 2013
- Article 57 (1.n) of Regulation (EC) No 726/2004
- General principles Emea - FDA parallel scientific advice. Letter; London. 2009
- Guideline on compassionate use of medicinal products, pursuant to article 83 of regulation (EC) No 726/2004 London, 19 July 2007
- Guideline on aspects of the application of Article 8(1) and (3) of Regulation (EC) No 141/2000: Assessing similarity of medicinal products versus authorised orphan medicinal products benefiting from market exclusivity and applying derogations from that market exclusivity Brussels, 19.9.2008 C(2008) 4077 final
- Guideline on the scientific application and the practical arrangements necessary to implement Commission Regulation (EC) No 507/2006 on the conditional marketing authorisation for medicinal products for human use falling within the scope of Regulation (EC) No 726/2004
- Guideline on the procedure for accelerated assessment pursuant to article 14 (9) of regulation (EC) No 726/2004
- Guideline on procedures for the granting of a marketing authorisation under exceptional circumstances, pursuant to article 14 (8) of regulation (EC) No 726/2004
- Franco P. Orphan drugs: the regulatory environment. Drug Discov Today 2013;18:163-72