References
- European Parliament and Council Regulation (EC) No 141/2000 of 16 December 1999 on orphan medicinal products.
- The 97th USA Congress An Act to amend the Federal Food, Drug, and Cosmetic Act to facilitate the development of drugs for rare diseases and conditions, and for other purposes. Effective January 4, 1983
- Morel T, Lhoir A, Picavet E, et al. Regulatory watch: the orphan drug pipeline in Europe. Nat Rev Drug Discov. 2016 Jun 1;15(6):376.
- [cited 2016 Nov 15]. Available from: www.morningstar.com
- European Union. Treaty of Lisbon Amending the Treaty on European Union and the Treaty Establishing the European Community, 13 December 2007, 2007/C 306/01
- European Medicines Agency, 2015 Annual Report, EMA 2016
- [cited 2019 Dec 6]. Available from: https://ec.europa.eu/eurostat/statistics-explained/index.php/R_%26_D_expenditure
- European Medicines Agency Information on benefit ct, Work package 4 report: Benefit-risk tools and processes EMA/297405/2012
- Alvarez MJ, Martinez F. R Vidal La contribució del Sector salut a l’economia, in Estartègies per Sortir de la crisi, Fulls Econòmics del Sistema Sanitari, Oct 2009 Servei Català de la Salut.
- Panel on the return on investments in health research. Making an impact. A preferred framework and indicators to measure returns on investment in health research. Ottawa, ON (Canada): Canadian Academy of Health Science (CAHS); 2009. Available from: www.cahs-acss.ca/e/pdfs/ROI_FullReport.pdf
- Adam P, Permanyer-Miralda G, Guillamón I, et al. Impact of clinical and health care services research on advancing knowledge. Study applied to the CAHTA‟s research Calls (third edition). Barcelona: Catalan Agency for Health Technology Assessment and Research. Catalan Health Service. Department of Health. Government of Catalonia; 2010.
- Morel T, Simoens S. Market watch: are orphan drug companies the pick of the pharmaceutical industr. Nature rev Drug Discov. 2014;13:10.
- Del Montea A, Papagani E. R&D and the growth of firms: empirical analysis of a panel of Italian firms. Res Policy. 2003;32:1003–7.
- E&Y Biotechnology Report 2016: returning to Earth, E&Y 2016
- Dunne P, Hughes H. Age, size, growth and survival: UK companies in the 1980s. J Ind Econ. 1994 Jun;42(2):115–140.
- Giannuzzi V, Landi A, Bosone E, et al. Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs. BMJ Open. 2017;7:e017358.
- Drummond M, Jönsson B, Rutten F, et al. Reimbursement of pharmaceuticals: reference pricing versus health technology assessment. Eur J Health Econ. 2011;12(3):263–271.
- Hughes DA, Poletti-Hughes J. Profitability and market value of orphan drug companies: a retrospective, propensity-matched case-control study. PLoS ONE. 2016;11(10):e0164681.
- Demirel P, Mazzucato M The evolution of firm growth dynamics in the us pharmaceutical industry: is ‘structure’ in the growth process. Related to size and location dynamics? Innogen working paper no. 66; November 2008
- EvaluatePharma® Orphan Drug Report 2015; Evaluate Oct 2015. Availabe from: www.evaluategroup.com/orphandrug2015