Funding orphan medicinal products beyond price: sustaining an ecosystem

The 97th United States 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) European Parliament and Council Regulation (EC): No 141/2000 of 16 December 1999 on orphan medicinal products Schey, C., Milanova, T., Hutchings, A.: Estimating the budget impact of orphan medicines in Europe: 2010–2020. Orphanet J. Rare Dis. 6, 62 (2011). https://doi.org/10.1186/1750-1172-6-62 Hutchings, A., Schey, C., Dutton, R., Achana, F., Antonov, K.: Estimating the budget impact of orphan drugs in Sweden and France 2013–2020. Orphanet J. Rare Dis. 9, 22 (2014). https://doi.org/10.1186/1750-1172-9-22 Schlander, M., Dintsios, C.M., Gandjour, A.: Budgetary impact and cost drivers of drugs for rare and ultrarare diseases. Value Health 21(5), 525–531 (2018) OECD: Pharmaceutical spending (indicator). https://doi.org/10.1787/998febf6-en. Accessed 16 Jan 2019 (2019) Ford, J.: Drug prices comes under the microscope. Financial Times, 16 August 2015 Richardson, J., Iezzi, A., Maxwell, A.: How important is severity for the evaluation of health services: new evidence using the relative social willingness to pay instrument. Eur. J. Health Econ. 18(6), 671–683 (2016) Richardson, J.: The wish to ‘share’ Health Economics Summer School. University of Heidelberg, Heidelberg (2016) Desser, A.S.: Prioritizing treatment of rare diseases: a survey of preferences of Norwegian doctors. Soc. Sci. Med. 94, 56–62 (2013) Richardson, J., Iezzi, A., Chen, G., Maxwell, A.: Communal sharing and the provision of low-volume high-cost health services: results of a survey. Pharmacoecon. Open. 1(1), 13–23 (2017) De Sola-Morales, O.: The success of EU OMP legislation: an empirical analysis. Value Health 20(9), A701 (2017) 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 https://ec.europa.eu/eurostat/statistics-explained/index.php/R_%26_D_expenditure 16/1/19. Accessed 16 Jan 2019 EU_Investing in Health. https://ec.europa.eu/research/health/...innovation-in-healthcare-overview-report_en.pdf. Accessed 22 Feb 2019 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). http://www.cahs-acss.ca/e/pdfs/ROI_FullReport.pdf (2009). Accessed 22 Feb 2019 Adam, P., Permanyer-Miralda, G., Guillamón, I., Berra, S., Pons, J.M.V.: Impact of clinical and health care services research on advancing knowledge. In: Study Applied to the CAHTA‟s Research CALLS (3rd edn.) Catalan Agency for Health Technology Assessment and Research. Catalan Health Service. Department of Health. Government of Catalonia, Barcelona (2010) EU INVESTING IN HEALTH Commission Staff Working Document Social Investment Package. http://ec.europa.eu/health/strategy/docs/swd_investing_in_health.pdf (2013). Accessed 22 Feb 2019 Morel, T., Lhoir, A., Picavet, E., Mariz, S., Sepodes, B., Llinares, J.: Cassiman D Regulatory watch: the orphan drug pipeline in Europe. Nat. Rev. Drug Discov. 15(6), 376 (2016) http://ec.europa.eu/growth/smes/business-friendly-environment/sme-definition_en. Accessed 15 Jan 2017 Ernst & Young Biotechnology Industry Report 2015: beyond borders Reaching new heights (2015) Shaw, B.: We need to talk about pharma profits; Pharmaboardroom 2019. https://pharmaboardroom.com/articles/we-need-to-talk-about-pharma-profits/