References
- Franco P. Orphan drugs: the regulatory environment. Drug Discov Today. 2013;18(3–4):163–172.
- Gammie T, Lu CY, Baber ZU. Access to orphan drugs: a comprehensive review of legislations, regulations and policies in 35 countries. PLoS One. 2015;10(10):e0140002.
- Minister of Health, Labour and Welfare. Tokyo. Overview of Orphan Drug/Medical Device Designation System. [cited 2019 Jul 26]. Available from: https://www.mhlw.go.jp/english/policy/health-medical/pharmaceuticals/orphan_drug.html
- Health Canada Office of Legislative and Regulatory Modernization, Initial Draft Discussion Document for a Canadian Orphan Drug Regulatory Framework, 2012 Dec 13. Available from: https://www.raredisorders.ca/content/uploads/Proposal-for-Orphan-Drug-Framework_dec-12-2012.pdf
- McCormick JI, Berescu LD, Tadros N. Common drug review recommendations for orphan drugs in Canada: basis of recommendations and comparison with similar reviews in Quebec, Australia, Scotland and New Zealand. Orphanet J Rare Dis. 2018;13(1):27.
- 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.
- Danzon PM. Affordability challenges to value-based pricing: mass diseases, orphan diseases, and cures. Value Health. 2018 Mar;21(3):252–257.
- Berdud M, Drummond M, Towse A Establishing a reasonable price for an orphan drug. Research Paper, Office of Health Economics. 2018; 18/05.
- International Network of Agencies for Health Technology Assessment. Alberta, Canada. [cited 2019 Jul 26]. Available from: http://www.inahta.org/
- Allen N, Walker SR, Liberti L, et al. Health Technology Assessment (HTA) case studies: factors influencing divergent HTA reimbursement recommendations in Australia, Canada, England, and Scotland. Value Health. 2017;20(3):320–328.
- Claxton K, Sculpher M, Drummond M. A rational framework for decision making by the National Institute for Clinical Excellence (NICE). Lancet. 2002;360(9334):711–715.
- Adkins EM, Nicholson L, Floyd D, et al. Oncology drugs for orphan indications: how are HTA processes evolving for this specific drug category? Clinicoecon Outcomes Res. 2017;9:327–342.
- Michel M, Toumi M. Access to orphan drugs in Europe: current and future issues. Expert Rev Pharmacoecon Outcomes Res. 2012;12(1):23–29.
- Walton MJ, O’Connor J, Carroll C, et al. A review of issues affecting the efficiency of decision making in the NICE single technology appraisal process. Pharmacoeconomics Open. 2019 [ cited 2019 Jan 08]. DOI:10.1007/s41669-018-0113-0
- Pearson I, Rothwell B, Olaye A, et al. Economic modeling considerations for rare diseases. Value Health. 2018;21(5):515–524.
- Allen N, Walker SR, Liberti L, et al. Evaluating alignment between Canadian common drug review reimbursement recommendations and provincial drug plan listing decisions: an exploratory study. CMAJ Open. 2016;4(4):E674–8.
- Morgan SG, McMahon M, Mitton C, et al. Centralized drug review processes in Australia, Canada, New Zealand, and the United Kingdom. Health Aff (Millwood). 2006;25(2):337–347.
- Fukuda T, Shiroiwa T. Application of economic evaluation of pharmaceuticals and medical devices in Japan. J Natl Inst Public Health. 2019;68(1):27–33.
- MHLW. Cost-effectiveness evaluation of drugs, medical devices and cellular and tissue-based products [in Japanese]. Tokyo. [cited 2019 Jul 26]. Available from: https://www.mhlw.go.jp/content/000497467.pdf
- Chuikyo. Central Social Insurance Medical Council General Meeting (No. 414) [in Japanese]. [cited 2019 Jul 26]. Available from: https://www.mhlw.go.jp/content/12404000/000508918.pdf
- Sasinowski FJ, Panico EB, Valentine JE. Quantum of effectiveness evidence in FDA’s approval of orphan drugs: update, July 2010 to June 2014. Ther Innov Regul Sci. 2015;49(5):680–697.
- The National Institute for Health and Care Excellence. Technology appraisal data: appraisal recommendations. London. [cited 2019 Jul 26]. Available from: https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance/data/appraisal-recommendations
- FM C, Harris A, Li JJ, et al. Using effectiveness and cost-effectiveness to make drug coverage decisions: a comparison of Britain, Australia, and Canada. JAMA. 2009;302(13):1437–1443.
- Griffiths EA, Hendrich JK, Stoddart SD, et al. Acceptance of health technology assessment submissions with incremental cost-effectiveness ratios above the cost-effectiveness threshold. Clinicoecon Outcomes Res. 2015;31(7):463–476.
- Wonder M, Chin G. What impact does ‘conventional’ economic evaluation have on patient access to new orphan medicines? A comparative study of their reimbursement in Australia (2005-2012). Expert Rev Pharmacoecon Outcomes Res. 2015;15(5):843–850.
- The National Health Service England. Commissioning policies. London. [cited 2019 Jul 26]. Available from: https://www.england.nhs.uk/commissioning/policies/
- The National Institute for Health and Care Excellence. NICE highly specialised technologies guidance. London. [cited 2019 Jul 26]. Available from https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-highly-specialised-technologies-guidance.
- The National Institute for Health and Care Excellence. Cancer Drugs Fund. London. Available from: https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance/cancer-drugs-fund
- Sipp D, Okano H. Japan strengthens regenerative medicine oversight. Cell Stem Cell. 2018;22(2):153–156.
- Kawalec P, Sagan A, Pilc A. The correlation between HTA recommendations and reimbursement status of orphan drugs in Europe. Orphanet J Rare Dis. 2016;11(1):122.
- Tordrup D, Tzouma V, Kanavos P. Orphan drug considerations in health technology assessment in eight European countries. Rare Dis Orphan Drugs. 2014;1(3):86–97.