Results 51 to 60 of about 22,997 (249)

Do drugs approved via expedited approval pathways have therapeutic advantages? A systematic review and meta‐analysis

British Journal of Clinical Pharmacology, EarlyView.
Regulators use expedited approval pathways to speed market approval and patient access to promising new drugs. However, there is uncertainty about whether these pathways are successful in approving drugs with significant therapeutic advantages. This systematic review aims to examine the safety, effectiveness and cost‐effectiveness of drugs approved via
Ashleigh Hooimeyer, Eliza J. McEwin, Zhicheng Wang, Joel Lexchin, Barbara Mintzes   +4 more
wiley   +1 more source

Decoding RNA regulation: Challenges and opportunities for RNA‐based therapies in Europe

British Journal of Clinical Pharmacology, EarlyView.
Abstract RNA‐based medicinal products represent a promising frontier in personalised medicine, offering sequence‐specific disease targeting at various molecular levels, yet their clinical translation in the European Union (EU) may be hindered by regulatory uncertainty around definitions and evidence requirements; this study therefore aims to identify ...
Olivia C. Lewis, Christine Leopold, Joyce M. Hoek, Raymond M. Schiffelers, Marie L. De Bruin   +4 more
wiley   +1 more source

Building local research and development capacity for the prevention and cure of neglected diseases: the case of India

Bulletin of the World Health Organization, 2001
This paper examines the proposal to build research and development (R & D) capabilities for dealing with neglected infectious and tropical diseases in countries where they are endemic, as a potentially cost- and time- effective way to fill the gap ...
Kettler Hannah E., Modi Rajiv
doaj  

Infusion rate adjustment in enzyme replacement therapy with pabinafusp alfa for mucopolysaccharidosis II

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aims Enzyme replacement therapy (ERT) for mucopolysaccharidosis II (MPS II) requires long‐term, weekly intravenous infusions often lasting over 3 h each time, which can burden paediatric patients and caregivers and negatively affect their quality of life and treatment compliance.
Kimitoshi Nakamura, Norio Sakai, Hideaki Hirai, Naoko Takasao, Ryo Ibaraki, Tatsuyoshi Yamamoto, Yuji Sato   +6 more
wiley   +1 more source

Rhodopsin orphan GPCR20 interacts with neuropeptides and directs growth, sexual differentiation, and egg production in female Schistosoma mansoni

Microbiology Spectrum
Schistosomes are parasitic flatworms that cause schistosomiasis, a neglected tropical disease of worldwide importance. Since standard treatment of schistosomiasis relies on a single drug, praziquantel, alternative drugs are needed.
Xuesong Li, Oliver Weth, Martin Haimann, Max F. Möscheid, Theresa S. Huber, Christoph G. Grevelding   +5 more
doaj   +1 more source

Regulatory harmonization: Evolution, globalization and future directions

British Journal of Clinical Pharmacology, EarlyView.
Regulatory harmonization has become an increasingly important and accepted approach to streamline regulatory review processes and expedite access to safe, effective and high‐quality medicines globally. This review explores the evolution and current status of regulatory harmonization, convergence and reliance initiatives.
Orin Chisholm, Elaine Y. L. Blair, Christine Y. Lu   +2 more
wiley   +1 more source

Advance price or purchase commitments to create markets for treatments for diseases of poverty: lessons from three policies

Bulletin of the World Health Organization, 2005
New drugs and vaccines are needed for tackling diseases of poverty in low- and middle-income countries. The lack of effective demand or market for these products translates into insufficient investment being made in research and development to meet the ...
Towse Adrian, Kettler Hannah
doaj  

External Control Arms in Japan: A Systematic Case Analysis of Documented Regulatory Implementation and Uncertainty Management

Clinical Pharmacology &Therapeutics, EarlyView.
External control arms (ECAs) have emerged as important tools to provide benchmark evidence on comparator effectiveness and thereby support regulatory decision making in situations where randomized controlled trials are difficult to conduct. However, how key methodological elements for ECAs are documented and implemented in practice and how residual ...
Suguru Okami, Alexandros Sagkriotis
wiley   +1 more source

The Multiple Criteria Qualitative Value‐Based Pricing Framework “MARIE” for Novel Cell and Gene Therapy

Clinical Pharmacology &Therapeutics, EarlyView.
When setting the price of cell and gene therapies in Japan, the factors considered include the manufacturing and operating costs of the new drug or a comparator, leading to discrepancies between price and values, in particular those specific to cell and gene therapies.
Akina Takami, Ataru Igarashi
wiley   +1 more source

Adverse Reactions Identified after Approval of Novel Medicines: Analysis of 339 New Molecular Entities and Therapeutic Biologics

Clinical Pharmacology &Therapeutics, EarlyView.
Postmarketing safety surveillance data identify safety issues not identified in preapproval clinical trials. We describe the frequency of new safety issues overall and by labeling section and the relationship of the most commonly added safety issues with the cumulative number of prescriptions dispensed via retail pharmacies.
Ellen Pinnow, Brendan Day, Esther H. Zhou, Corinne Woods, Nana Dwumfour, Jenni Y. Lee, Ilynn Bulatao, Sanae Cherkaoui, Manish Kalaria, Sonja Brajovic, Nabila Sadiq, Farideh Sistani, Maggie Lee, Grace Chai, Gerald Dal Pan   +14 more
wiley   +1 more source