Bibliography
- EURORDIS, Rare Diseases Europe website. Available from: http://www.eurordis.org/about-rare-diseases
- Sasinowski FJ. Quantum of Effectiveness Evidence in FDA’s Approval of Orphan Drugs. Chairman of the Board, NORD. Available from: http://nordphysicianguides.org/wp-content/uploads/2012/02/NORD-study-of-FDA-approval-of-orphan-drugs.pdf
- Workshop on Natural History Studies of Rare Diseases: Meeting the Needs of Drug Development and Research. Available from: https://events-support.com/Documents/Summary-NHS.pdf