Results 71 to 80 of about 88,273 (287)

International experience in drug assessment for rare diseases: flexibility of approaches

Реальная клиническая практика: данные и доказательства
Relevance. The regulation of orphan drugs plays a key role in the development of medicines for rare diseases that affect only a small percentage of the population. This specialized regulatory framework is designed to encourage pharmaceutical companies to
K. S. Radaeva, A. S. Kolbin
doaj   +1 more source

Common drug review recommendations for orphan drugs in Canada: basis of recommendations and comparison with similar reviews in Quebec, Australia, Scotland and New Zealand

Orphanet Journal of Rare Diseases, 2018
Background Public payer reimbursement for non-oncology drugs in Canada, including orphan drugs, is based on recommendations by the Common Drug Review (CDR) (with the exception of Quebec).
John I. McCormick, L. Diana Berescu, Nabil Tadros   +2 more
doaj   +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

No difference in between-country variability in use of newly approved orphan and non- orphan medicinal products - a pilot study

Orphanet Journal of Rare Diseases, 2009
Background Regulators and payers have to strike a balance between the needs of the patient and the optimal allocation of resources. Drugs indicated for rare diseases (orphan medicines) are a special group in this context because of their often high per ...
Bloechl-Daum Brigitte, Leufkens Hubert GM, Heemstra Harald E, Stolk Pieter, Heerdink Eibert R   +4 more
doaj   +1 more source

Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act [PDF]

, 2015
Background: Lysosomal storage disorders are a heterogeneous group of approximately 50 monogenically inherited orphan conditions. A defect leads to the storage of complex molecules in the lysosome, and patients develop a complex multisystemic phenotype of
Hoffmann, Georg Friedrich, Mechler, Konstantin, Mountford, William K., Ries, Markus   +3 more
core   +1 more source

Facilitating Expedited Drug Approval– the Past, Present, and Future of Clinical Trials for Pediatric Rheumatic Disease

Arthritis Care &Research, Accepted Article.
Pamela F. Weiss, Hermine I. Brunner
wiley   +1 more source

Nanomedicine Meets Immunotherapy: Advancing Adoptive Cell Therapy with Nanoparticles in the Treatment of Cancer with Sustainability Perspectives

Advanced Science, EarlyView.
This review surveys nanoparticle‐based strategies to enhance adoptive cell therapy, particularly CAR‐T cell approaches, in solid tumor treatment. It describes how nanoparticles can improve tumor immunogenicity and T‐cell infiltration while reducing toxicity, and how they enable in vivo CAR‐T cell generation.
Erica Frostegård, Tatiana Bobrova, Amalie Leinebø, Thibault Leray, Shayamita Ghosh, Lorena García‐Hevia, Chiara Puccinelli, Lorenzo Riccio, Jacopo Sorani, Davide Bonifazi, Julien Caumartin, Emmanuel Donnadieu, Florence Gazeau, Roland Hischier, Else Marit Inderberg, Mónica L. Fanarraga, Maria Loustau, Bernd Nowack, Sébastien Wälchli, Cécilia Ménard‐Moyon   +19 more
wiley   +1 more source

Making rareness count: testing and pricing orphan drugs [PDF]

, 2016
This paper examines the testing and the pricing of orphan drugs, e.g. drugs for patients suffering from rare diseases. Due to the small size of these populations, orphan molecules question established evidentiary practices, namely randomized controlled ...
Doganova, Liliana, Rabeharisoa, Vololona
core   +3 more sources

Toward Predictable Nanomedicine: Current Forecasting Frameworks for Nanoparticle–Biology Interactions

Advanced Intelligent Discovery, EarlyView.
Predictive models successfully screen nanoparticles for toxicity and cellular uptake. Yet, complex biological dynamics and sparse, nonstandardized data limit their accuracy. The field urgently needs integrated artificial intelligence/machine learning, systems biology, and open‐access data protocols to bridge the gap between materials science and safe ...
Mariya L. Ivanova, Michael Nicholls, Nicola Russo, Gueorgui Mihaylov, Konstantin Nikolic   +4 more
wiley   +1 more source

Precision Medicines’ Impact on Orphan Drug Designation

Clinical and Translational Science, 2019
The incentives provided under the Orphan Drug Act (ODA) have been credited for catalyzing the marketing approval of drugs for the treatment of rare diseases by the US Food and Drug Administration.
Christine M. Mueller, Gayatri R. Rao, Katherine I. Miller Needleman   +2 more
doaj   +1 more source