Results 81 to 90 of about 68,610 (290)

Through the lens of marketing authorization holders: experience in use of real‐world data and real‐world evidence in drug development and regulatory submissions in EU

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aim The aim of this study was to assess the general and product‐specific experiences of MAHs use of RWD/RWE in medicines development and in their regulatory submissions, and to explore organizational aspects of MAHs related to RWD/RWE. Methods An electronic survey was conducted, and information collected directly from MAHs.
Sini M. Eskola, Aleksandar Gigov, Hubertus G. M. Leufkens, Andrew Bate, Marie Louise De Bruin, Helga Gardarsdottir   +5 more
wiley   +1 more source

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

The evolving therapeutic landscape of spinal muscular atrophy – A scoping review of investigational agents, emerging delivery technologies and strategic innovations

British Journal of Clinical Pharmacology, EarlyView.
Spinal muscular atrophy (SMA) is a severe neuromuscular disease with emerging therapeutic complexity. This review aims to systematically map the global pipeline of investigational treatments for SMA. Using ClinicalTrials.gov and complementary international registries, we identified 21 planned or ongoing interventional trials from 2020 to 2025 targeting
Andrej Belančić, Patrick Eustaquio, Elvira Meni Maria Gkrinia, Ivana Stević, Eugen Javor, Yun Wah Lam, Slobodan Janković, Dinko Vitezić   +7 more
wiley   +1 more source

Advanced therapy medicinal products (ATMPs) – An analysis of the global regulatory status

British Journal of Clinical Pharmacology, EarlyView.
Abstract Background A rapid progress in the development of advanced therapy medicinal products (ATMPs) has taken place over the past decades. The ATMPs hold great potential but face challenges in the clinical development and the regulatory process.
Allan Cramer, Freja Karuna Hemmingsen Sørup, Marie Lund, Tonny Studsgaard Petersen, Kristian Karstoft   +4 more
wiley   +1 more source

Does Misery Love Company? Evidence from pharmaceutical markets before and after the Orphan Drug Act [PDF]

With substantial fixed costs of drug development, more common conditions can support more products. If additional pharmaceutical products are beneficial, they will attract greater consumption and promote better health, e.g. greater longevity.
Frank R. Lichtenberg, Joel Waldfogel
core  

Cultural Environmentalism and Beyond [PDF]

, 2007
This article is part of a symposium issue entitled Cultural Environmentalism @ 10, occuring on the tenth anniversary of Prof. Boyle\u27s book, Shamans, Software, and Spleens. In this article Prof.
Boyle, James
core   +3 more sources

Nanomaterial‐based immune therapeutic strategies in neurodegenerative diseases

BMEMat, EarlyView.
This review highlights the immunomodulatory potential of nanomaterials (NMs) in treating neurodegenerative diseases (NDs). It focuses on their roles in regulating innate and adaptive immune responses to maintain immune homeostasis. By providing insights into these mechanisms, the review lays the groundwork for innovative NMs therapeutic strategies to ...
Xinru Zhou, Jia Liu, Shulin Lai, Suhan Yin, Bingmin Luo, Longquan Shao, Yiyuan Kang   +6 more
wiley   +1 more source

Perspectives on stimulating industrial research and development for neglected infectious diseases

Bulletin of the World Health Organization, 2001
This paper summarizes recent thinking on stimulating industrial research and development (R & D) for neglected infectious diseases and argues that it is critical to enlarge the value of the market for medicines and vaccines through, for example, global ...
Webber David, Kremer Michael
doaj  

The hope for neglected diseases: R&D incentives [PDF]

Neglected diseases are neglected because they cannot generate enough return on R&D to pharmaceutical firms. This paper analyzes and compares existing proposals for public intervention in R&D for neglected diseases.
Brigitte Granville, Eshref Trushin
core  

Design of experiments‐guided membrane capture facilitates integration of continuous virus inactivation using Phi6 as a surrogate virus in an intensified downstream process

Biotechnology Progress, EarlyView.
Abstract The growing demand for cost‐efficient and flexible biomanufacturing has increased interest in process‐intensified downstream platforms. This study evaluates an intensified monoclonal antibody (mAb) purification sequence, where two unit operations traditionally performed in batch mode—Protein A capture and low pH virus inactivation (VI)—are ...
Mario Grünberg, Lisa Lipski, Gabriel Fisicaro, Thomas Duvignau, Karolina Meyer‐Heinrichs, Diana Carmen Mocsy, Thomas‐Josef Filz, Bastian Quaas, Alexandra Stützer   +8 more
wiley   +1 more source