Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries
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DOI: 10.1371/journal.pone.0140002
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- Rosenberg-Yunger, Zahava R.S. & Daar, Abdallah S. & Thorsteinsdóttir, Halla & Martin, Douglas K., 2011. "Priority setting for orphan drugs: An international comparison," Health Policy, Elsevier, vol. 100(1), pages 25-34, April.
- Iskrov, Georgi & Miteva-Katrandzhieva, Tsonka & Stefanov, Rumen, 2012. "Challenges to orphan drugs access in Eastern Europe: The case of Bulgaria," Health Policy, Elsevier, vol. 108(1), pages 10-18.
- Alain Denis & Lut Mergaert & Christel Fostier & Irina Cleemput & Steven Simoens, 2010. "Issues surrounding orphan disease and orphan drug policies in Europe," Applied Health Economics and Health Policy, Springer, vol. 8(5), pages 343-350, September.
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- Wellman-Labadie, Olivier & Zhou, Youwen, 2010. "The US Orphan Drug Act: Rare disease research stimulator or commercial opportunity?," Health Policy, Elsevier, vol. 95(2-3), pages 216-228, May.
- Jorge Mestre-Ferrandiz;Martina Garau, 2009. "Access Mechanisms for Orphan Drugs: A Comparative Study of Selected European Countries," Briefing 000227, Office of Health Economics.
- Picavet, Eline & Cassiman, David & Simoens, Steven, 2012. "Evaluating and improving orphan drug regulations in Europe: A Delphi policy study," Health Policy, Elsevier, vol. 108(1), pages 1-9.
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- Degtiar, Irina, 2017. "A review of international coverage and pricing strategies for personalized medicine and orphan drugs," Health Policy, Elsevier, vol. 121(12), pages 1240-1248.
- Se Hee Lee & Seung-Lai Yoo & Joon Seok Bang & Jong Hyuk Lee, 2020. "Patient Accessibility and Budget Impact of Orphan Drugs in South Korea: Long-Term and Real-World Data Analysis (2007–2019)," IJERPH, MDPI, vol. 17(9), pages 1-8, April.
- Torrent-Farnell, J. & Comellas, M. & Poveda, J.L. & Abaitua, I. & Gutiérrez-Solana, L.G. & Pérez-López, J. & Cruz, J. & Urcelay, J. & Lizán, L., 2018. "The view of experts on initiatives to be undertaken to promote equity in the access to orphan drugs and specialised care for rare diseases in Spain: A Delphi consensus," Health Policy, Elsevier, vol. 122(6), pages 590-598.
- Fontrier, Anna-Maria, 2022. "Market access for medicines treating rare diseases: Association between specialised processes for orphan medicines and funding recommendations," Social Science & Medicine, Elsevier, vol. 306(C).
- Dyfrig A Hughes & Jannine Poletti-Hughes, 2016. "Profitability and Market Value of Orphan Drug Companies: A Retrospective, Propensity-Matched Case-Control Study," PLOS ONE, Public Library of Science, vol. 11(10), pages 1-12, October.
- Ria Christine Siagian & Dumilah Ayuningtyas, 2019. "Gap analysis for drug development policy-making: An attempt to close the gap between policy and its implementation," PLOS ONE, Public Library of Science, vol. 14(8), pages 1-13, August.
- Marialuisa Saviano & Sergio Barile & Francesco Caputo & Mattia Lettieri & Stefania Zanda, 2019. "From Rare to Neglected Diseases: A Sustainable and Inclusive Healthcare Perspective for Reframing the Orphan Drugs Issue," Sustainability, MDPI, vol. 11(5), pages 1-21, March.
- Nicholas Bagley & Benjamin Berger & Amitabh Chandra & Craig Garthwaite & Ariel D. Stern, 2018. "The Orphan Drug Act at 35: Observations and an Outlook for the Twenty-First Century," NBER Chapters, in: Innovation Policy and the Economy, Volume 19, pages 97-137, National Bureau of Economic Research, Inc.
- Jason C Hsu & Huai-Chueh Wu & Wen-Chia Feng & Chih-Ho Chou & Edward Chia-Cheng Lai & Christine Y Lu, 2018. "Disease and economic burden for rare diseases in Taiwan: A longitudinal study using Taiwan’s National Health Insurance Research Database," PLOS ONE, Public Library of Science, vol. 13(9), pages 1-14, September.
- Pejcic, Ana V. & Iskrov, Georgi & Jakovljevic, Mihajlo Michael & Stefanov, Rumen, 2018. "Access to orphan drugs – comparison across Balkan countries," Health Policy, Elsevier, vol. 122(6), pages 583-589.
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- Shiwei Gong & Dehe Li & Dong Dong, 2020. "How Do Patients and Doctors Perceive Medical Services for Rare Diseases Differently in China? Insights from Two National Surveys," IJERPH, MDPI, vol. 17(16), pages 1-14, August.
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