1. Regulation (EC) Nº 141/2000 of the European Parliament and the Council of 16 December 1999 on orphan medicinal products. In: Union EPatCotE, ed. Vol 141/2000: Official Journal od the European Communities 2000.
2. NICE Appraisals of Rare Diseases;Powell,2019
3. Rare disease terminology and definitions—a systematic global review: report of the ISPOR Rare Disease Special Interest Group;Richter;Value Health,2015
4. Rare Diseases and Orphan Products. Accelerating Research and Development. In: Institute of Medicine of the National Academies; 2010.
5. The UK Strategy for Rare Diseases,2013