1. National Organization for Rare Disorders. NORD and Trio Health to provide real-world insights to improve access and quality of care for more than 7,000 rare diseases. April 28, 2016. Available from: https://rarediseases.org/nord-and-trio-health-provide-insights-improve-access-and-quality-care-more-than-7000-rare-diseases/. Accessed May 5, 2017.
2. Pharmaceutical Research and Manufacturers of America. Rare diseases: a report on orphan drugs in the pipeline,2013
3. Evaluate. EvaluatePharma orphan drug report—2017. 4th Edition. Available from: http://info.evaluategroup.com/rs/607-YGS-364/images/EPOD17.pdf. Accessed February 3, 2018.
4. Rare disease terminology and definitions-a systematic global review: Report of the ISPOR Rare Disease Special Interest Group;Richter;Value Health,2015
5. Drugs for exceptionally rare diseases: do they deserve special status for funding?;Hughes;QJM,2005