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Expert Review of Pharmacoeconomics & Outcomes Research Volume 15, 2015 - Issue 1
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Research Report

Budget impact analysis of drugs for ultra-orphan non-oncological diseases in Europe

Michael SchlanderInstitute for Innovation and Valuation in Health Care (InnoVal), An der Ringkirche 4, D-65197 Wiesbaden, Germany;Department of Public Health, Social and Preventive Medicine, Mannheim Medical Faculty, University of Heidelberg, Mannheim, GermanyCorrespondence[email protected] [email protected]
,
Charles Christian AdarkwahDepartment of General Practice, University of Marburg, Marburg, Germany
&
Afschin GandjourFrankfurt School of Finance and Management, Frankfurt, Germany
Pages 171-179 | Published online: 14 Oct 2014

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Domenico Moro, Michael Schlander, Harry Telser, Oriol Sola-Morales, Michael David Clark, Andrew Olaye, Charlotte Camp, Mohit Jain, Thomas Butt & Sumeet Bakshi. (2022) Evaluating discrete choice experiment willingness to pay [DCE-WTP] analysis and relative social willingness to pay [RS-WTP] analysis in a health technology assessment of a treatment for an ultra-rare childhood disease [CLN2]. Expert Review of Pharmacoeconomics & Outcomes Research 22:4, pages 581-598.
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Margit Gombocz & Sabine Vogler. (2020) Public spending on orphan medicines: a review of the literature. Journal of Pharmaceutical Policy and Practice 13:1.
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Daniel Resende Faleiros, Juliana Álvares, Alessandra Maciel Almeida, Vânia Eloisa de Araújo, Eli Iola Gurgel Andrade, Brian B. Godman, Francisco A. Acurcio & Augusto A. Guerra Júnior. (2016) Budget impact analysis of medicines: updated systematic review and implications. Expert Review of Pharmacoeconomics & Outcomes Research 16:2, pages 257-266.
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Articles from other publishers (10)

Barbara Fischer, Harry Telser, Peter Zweifel, Viktor von Wyl, Konstantin Beck & Andreas Weber. (2023) The value of a QALY towards the end of life and its determinants: Experimental evidence. Social Science & Medicine 326, pages 115909.
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Richard Lech, Gideon Chow, Kamalpreet Mann, Patrick Mott, Christine Malmberg & Lindy Forte. (2022) Historical and projected public spending on drugs for rare diseases in Canada between 2010 and 2025. Orphanet Journal of Rare Diseases 17:1.
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Khadidja Abdallah, Isabelle Huys, Kathleen Claes & Steven Simoens. (2021) Methodological Quality Assessment of Budget Impact Analyses for Orphan Drugs: A Systematic Review. Frontiers in Pharmacology 12.
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Tao Ran, Stefan B. Eichmüller, Patrick Schmidt & Michael Schlander. (2020) Cost of decentralized CAR T‐cell production in an academic nonprofit setting . International Journal of Cancer 147:12, pages 3438-3445.
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Olga A. Belousova, Aard J. Groen & Aniek M. Ouendag. (2020) Opportunities and barriers for innovation and entrepreneurship in orphan drug development. Technological Forecasting and Social Change 161, pages 120333.
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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 13:9, pages e0204206.
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Sandra Nestler-Parr, Daria Korchagina, Mondher Toumi, Chris L. Pashos, Christopher Blanchette, Elizabeth Molsen, Thomas Morel, Steven Simoens, Zoltán Kaló, Ruediger Gatermann & William Redekop. (2018) Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group. Value in Health 21:5, pages 493-500.
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Michael Schlander, Charalabos-Markos Dintsios & Afschin Gandjour. (2018) Budgetary Impact and Cost Drivers of Drugs for Rare and Ultrarare Diseases. Value in Health 21:5, pages 525-531.
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Irina Degtiar. (2017) A review of international coverage and pricing strategies for personalized medicine and orphan drugs. Health Policy 121:12, pages 1240-1248.
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Konstantins Logviss, Dainis Krievins & Santa Purvina. (2016) Impact of orphan drugs on Latvian budget. Orphanet Journal of Rare Diseases 11:1.
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