1. FDA Final Rule. CFR 316. Orphan Drug Regulations 57 FR 62076 December 29, 1992
2. European Commission (2008) Communication from the commission to the European Parliament, the Council, the European Economic and Social Committee and the Committee of the Regions on Rare Diseases: Europe’s challenges.
http://ec.europa.eu/health/ph_threats/non_com/docs/rare_com_en.pdf
. Accessed 2 Dec 2018
3. U.S. Department of Health and Human Services. National Institute of Health. NCATS and rare diseases research (updated 12 Feb 2018).
https://ncats.nih.gov/rdd
. Accessed 2 Dec 2018
4. Report: complex issues in developing drugs and biological products for rare diseases and accelerating the development of therapies for pediatric rare diseases including strategic plan: accelerating the development of therapies for pediatric rare diseases.
https://www.fda.gov/downloads/RegulatoryInformation/LawsEnforcedbyFDA/SignificantAmendmentstotheFDCAct/FDASIA/UCM404104.pdf
. Accessed 2 Dec 2018
5. Augustine EF, Adams HR, Mink JW (2013) Clinical trials in rare disease: challenges and opportunities. J Child Neurol 28(9):1142–1150