International comparison of availability for orphan drugs: focused on approved orphan drugs in South Korea [PDF]
Journal of Pharmaceutical Policy and PracticeIntroduction: In this study, we aimed to comparatively analyse the indicators of availability to orphan drugs in South Korea, the United States of America, Europe Union, and Japan.Methods: For 169 drugs designated as orphan drugs in South Korea between ...
Eun Young Shin +4 more
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Impacts of adjustment of National Reimbursement Drug List on orphan drugs volume and spending in China: an interrupted time series analysis [PDF]
BMJ Open, 2023 Objective To evaluate the impacts of the 2017 adjustment of National Reimbursement Drug List (NRDL) on orphan drugs hospital procurement volumes and spending in China.Design We used an interrupted time series design covering the period from 2016 to 2018 ...
Yu Xie +6 more
doaj +2 more sources
Orphan drugs’ clinical uncertainty and prices: Addressing allocative and technical inefficiencies in orphan drug reimbursement [PDF]
Frontiers in Pharmacology, 2023 Legislations incentivising orphan drug development and scientific advances have made orphan drugs pharma’s high-end favourite for the past two decades. Currently, around 50% of new marketing authorizations are for orphan drugs. For third-party healthcare
Hans-Georg Eichler +3 more
doaj +2 more sources
Are payers treating orphan drugs differently?
Journal of Market Access & Health Policy, 2014 Background: Some orphan drugs can cost hundreds of thousands of dollars annually per patient. As a result, payer sensitivity to the cost of orphan drugs is rising, particularly in light of increased numbers of new launches in recent years.
Joshua P. Cohen, Abigail Felix
doaj +2 more sources
Exploring alternative financing models and early access schemes for orphan drugs: a Belgian case study [PDF]
Orphanet Journal of Rare Diseases, 2022 Background Although some jurisdictions have implemented particular adjustments to accommodate often-expensive orphan drugs in their healthcare systems, availability of these drugs remains complex.
Khadidja Abdallah +5 more
doaj +2 more sources
Disentangling the Cost of Orphan Drugs Marketed in the United States [PDF]
Healthcare (Switzerland), 2023 Lawrence Brown +2 more
exaly +2 more sources
Comparison of US and EU Prices for Orphan Drugs in the Perspective of the Considered US Orphan Drugs Act Modifications and Discussed Price-Regulation Mechanisms Adjustments in US and European Union [PDF]
International Journal of Environmental Research and Public Health, 2022 Olga Partyka +2 more
exaly +2 more sources
Medicine in Drug Discovery, 2023 Only 5% of orphan diseases have approved drugs, leading to a high demand for new treatment. As a result, pharmaceutical companies have shifted their focus of drug development to orphan diseases.
Ryo Okuyama
doaj +1 more source
Top Selling (2026) Small Molecule Orphan Drugs: A Journey into Their Chemistry [PDF]
International Journal of Molecular Sciences, 2023 Ornelio Rosati +2 more
exaly +2 more sources