Results 31 to 40 of about 1,419,402 (298)

Estimating the clinical cost of drug development for orphan versus non-orphan drugs

Orphanet Journal of Rare Diseases, 2019
Background High orphan drug prices have gained the attention of payers and policy makers. These prices may reflect the need to recoup the cost of drug development from a small patient pool.
Kavisha Jayasundara, Aidan Hollis, Murray Krahn, Muhammad Mamdani, Jeffrey S. Hoch, Paul Grootendorst   +5 more
doaj   +1 more source

Clinical pharmacology information in regulatory submissions and labeling: A comparative analysis of orphan and non‐orphan drugs approved by the FDA

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, Martina Sahre, Xinning Yang, Rajanikanth Madabushi, Anuradha Ramamoorthy   +4 more
doaj   +1 more source

Modality diversification and best-in-class small-molecule drugs: Recent trend of orphan drug development

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

Gut microbiota–derived short‐chain fatty acids regulate group 3 innate lymphoid cells in HCC

Hepatology, EarlyView., 2022
Abstract Background and Aims Type 3 innate lymphoid cells (ILC3s) are essential for host defense against infection and tissue homeostasis. However, their role in the development of HCC has not been adequately confirmed. In this study, we investigated the immunomodulatory role of short‐chain fatty acids (SCFAs) derived from intestinal microbiota in ILC3
Chupeng Hu, Bingqing Xu, Xiaodong Wang, Wen‐Hua Wan, Jinying Lu, Deyuan Kong, Yu Jin, Wenhua You, Hua Sun, Xiaoxin Mu, Dongju Feng, Yun Chen   +11 more
wiley   +1 more source

Can a regulatory instrument promote research and development into neglected diseases? A Policy Delphi Survey [PDF]

, 2012
Fehr A. Can a regulatory instrument promote research and development into neglected diseases? A Policy Delphi Survey. Bielefeld: University of Bielefeld; 2012.Background Rare diseases and neglected diseases are characterized by deficits in drug research ...
Fehr, Angela
core   +1 more source

Profitability and Market Value of Orphan Drug Companies: A Retrospective, Propensity-Matched Case-Control Study.

PLoS ONE, 2016
BackgroundConcerns about the high cost of orphan drugs has led to questions being asked about the generosity of the incentives for development, and associated company profits.MethodsWe conducted a retrospective, propensity score matched study of publicly-
Dyfrig A Hughes, Jannine Poletti-Hughes
doaj   +1 more source

Data‐Driven Materials Science for Energy‐Sustainable Applications

Advanced Materials, EarlyView.
Data‐driven approaches powered by artificial intelligence are transforming materials discovery for energy sustainability. This review examines how auto‐generated high‐quality materials databases and domain‐specific language models accelerate research in photovoltaics, thermoelectrics, batteries and magnetic materials. Applications involve extraction of
Jacqueline M. Cole
wiley   +1 more source

Engineering Approaches to Modify Immunomodulatory Functions of Mesenchymal Stromal Cells (MSCs): Tissue Regeneration and Clinical Application

Advanced Science, EarlyView.
Mesenchymal stromal cells (MSCs) show promise for treating immune‐related disorders through immunomodulation and tissue regeneration. This review gives a brief overview of current clinical approval of MSC therapies. It also discussed how bioengineering, including genetic modification, biomaterial delivery, extracellular vesicles, and iPSC‐derived MSCs,
Sichen Yang, Kejia Li, Ning Wang, Zhen Li, Zhiyong Zhang, Rocky S. Tuan, Yangzi Jiang   +6 more
wiley   +1 more source

The processes of reform in Victoria’s alcohol and other drug sector, 2011-2014 [PDF]

, 2014
This paper explores issues and concerns related to the 2014 alcohol and other drug sector recommissioning process in Victoria. Overview In mid-2014, the Victorian Alcohol and Drug Association approached the Drug Policy Modelling Program, at the ...
Drug Policy Modelling Program
core  

Analysis of the first ten years of FDA’s rare pediatric disease priority review voucher program: designations, diseases, and drug development

Orphanet Journal of Rare Diseases
Background The Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) Program was enacted in 2012 to support the development of new products for children.
Catherine Mease, Kathleen L. Miller, Lewis J. Fermaglich, Jeanine Best, Gumei Liu, Erika Torjusen   +5 more
doaj   +1 more source