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
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Estimating the budget impact of orphan medicines in Europe: 2010 - 2020 [PDF]
Orphanet Journal of Rare Diseases, 2011 Background Orphan drugs are a growing issue of importance to European healthcare policy makers. The success of orphan drug legislation in Europe has resulted in an increasing number of licensed medicines for rare diseases, and many more yet unlicensed ...
Milanova Tsveta +2 more
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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
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Clinical pharmacology information in regulatory submissions and labeling: A comparative analysis of orphan and non‐orphan drugs approved by the FDA [PDF]
Clinical and Translational Science, 2022 Clinical pharmacology is an integral discipline supporting the development, regulatory evaluation, and clinical use of drugs for the treatment of both common and rare diseases.
Julie Hsieh +4 more
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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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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
Societal preferences for funding orphan drugs in China: An application of the discrete choice experiment method [PDF]
Frontiers in Public Health, 2022 ObjectivesTo explore whether a societal preference for orphan drugs exists in Chinese general public and to quantitatively measure the personal trade-off between essential attributes of orphan drugs through a discrete choice experiment.MethodsA labeled ...
Shuoyuan Tan +12 more
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Medicinski pregled, 2013 Introduction. Drugs used for treatment of rare diseases are known worldwide under the term of orphan drugs because pharmaceutical companies have not been interested in ?adopting? them, that is in investing in research, developing and producing these drugs.
Svetlana Golocorbin-Kon +4 more
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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