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 ...
Yi H +6 more
europepmc +2 more sources
Clinical pharmacology information in regulatory submissions and labeling: A comparative analysis of orphan and non-orphan drugs approved by the FDA. [PDF]
Clin Transl Sci, 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.
Hsieh J +4 more
europepmc +2 more sources
Orphan drugs' clinical uncertainty and prices: Addressing allocative and technical inefficiencies in orphan drug reimbursement. [PDF]
Front Pharmacol, 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
Eichler HG +3 more
europepmc +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
International comparison of availability for orphan drugs: focused on approved orphan drugs in South Korea. [PDF]
J Pharm Policy PractIntroduction: 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 ...
Shin EY +4 more
europepmc +2 more sources
Orphanet Journal of Rare Diseases, 2022 Background Extremely high prices facilitate drug development for ultra-rare diseases (ultra-orphan drugs). However, various problems arise in terms of healthcare financing and fairness, and the status of ultra-orphan drug pricing remains ambiguous.
Akihiko Kawakami, Ken Masamune
doaj +1 more source
Practice of the Qualification and Recognition for Orphan Drugs in the World and its Inspiration
罕见病研究, 2022 We have analyzed the current status of recognization and qualification of orphan drugs in China and abroad, looking at the aspects of the authority institutions, identification and qualification process, and the number of orphan drugs identified and ...
ZHU Xiaohong +7 more
doaj +1 more source
Expenditure trends of orphan drugs in Serbia: 8-Year analysis of orphan drug market in Serbia [PDF]
Hospital Pharmacology, 2016 Background/Objective: Orphan drugs are medicinal products which treat life-threatening or chronically debilitating diseases which affect small patient populations.
Pejčić Ana V., Iskrov Georgi G.
doaj +1 more source
Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries. [PDF]
PLoS ONE, 2015 OBJECTIVE:To review existing regulations and policies utilised by countries to enable patient access to orphan drugs. METHODS:A review of the literature (1998 to 2014) was performed to identify relevant, peer-reviewed articles. Using content analysis, we
Todd Gammie +2 more
doaj +1 more source
Exploring alternative financing models and early access schemes for orphan drugs: a Belgian case study. [PDF]
Orphanet J Rare Dis, 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.
Abdallah K +5 more
europepmc +2 more sources