Results 101 to 110 of about 91,140 (235)

Beyond the label: Rethinking off‐label drug use in paediatrics. Towards a scientifically grounded and safer future for paediatric pharmacotherapy

British Journal of Clinical Pharmacology, EarlyView.
Abstract Despite regulatory progress being made in the past two decades, off‐label drug use in paediatrics remains pervasive, with prevalence estimated between 3% and 97% of prescriptions across different clinical settings. Off‐label use—defined as prescribing outside the conditions described in the Summary of Product Characteristics (SmPC)—is often ...
Tjitske M. van der Zanden, Miriam G. Mooij, Matthijs de Hoog, Saskia N. de Wildt   +3 more
wiley   +1 more source

Main factors associated with variability in the assessment of the objective response rate. A re‐analysis of the PHEREXA phase 3 clinical trial

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aim Local investigators (LIs) overestimate the objective response rate (ORR) in comparison to Blinded Independent Central Reviewers (BICR) in oncology. In this study, we re‐analysed data obtained in the PHEREXA trial (NCT01026142) with the following aims: i) to confirm at the single‐patient level the discrepancy observed by analysing ...
Gennaro Daniele, Tommaso Giovagnoli, Pierluigi Navarra, Cinzia Dello Russo   +3 more
wiley   +1 more source

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

Efficacy, safety and cost‐effectiveness of CAR‐T therapy

British Journal of Clinical Pharmacology, EarlyView.
CAR T‐cells demonstrate high efficacy in blood cancers, including ALL, MM and DLBCL. Innovations target solid tumours despite challenges such as antigen escape. Combination therapies enhance the delivery and infiltration of CAR T cells. Toxicity, cost and resistance remain major barriers to clinical use.
Emina Karahmet Sher, Amina Džidić‐Krivić, Emma Pinjic, Nejra Selak, Kanita Omerbasic, Alexandar Chupin, Andrej Belancic, Almir Fajkic   +7 more
wiley   +1 more source

Toward the harmonization of bioequivalence guidelines in Europe: Commentary on the state of the art and future priorities under ICH M13A for immediate‐release oral forms

British Journal of Clinical Pharmacology, EarlyView.
Domenico Nocera, Giusi Forastiero, Andrea Logreco, Alessia Proietti, Antonio Galluccio, Carlotta Lunghi, Elisabetta Poluzzi, Fabrizio De Ponti   +7 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

Comparative efficacy of GLP‐1 RA, tirzepatide and SGLT‐2 inhibitors in metabolic liver disease: A network meta‐analysis

British Journal of Clinical Pharmacology, EarlyView.
Aim Metabolic liver disease, including nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis, is a major cause of chronic liver dysfunction worldwide, creating an urgent need for effective treatments. This systematic literature review (SLR) and network meta‐analysis (NMA) systematically reviews and compares the efficacy and safety ...
Andrej Belančić, Christina Antza, Anastasios Poutachidis, Smaro Palaska, Elvira Meni Maria Gkrinia, Andrea Katrin Faour, Yusuf Ziya Sener, Seher Sener, Rehena Sultana   +8 more
wiley   +1 more source

The quantitative impact of metabolism‐inhibiting drugs on the occurrence of adverse drug reactions—A backward selection approach

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aim The quantitative effect of several inhibitory drugs on the development of adverse drug reactions (ADRs) is currently difficult to estimate. Our aim was to identify metabolic pathways, which, when inhibited, increase the risk for certain ADRs, and to use this system to consider comedication at individual level. Methods Data of a prospective
Judith Berres, Justyna Wozniak, Hannah Fölsch, Anja Knueppel‐Ruppert, Verena Graeff, Andrea Kriegisch‐Stumpf, Harald Dormann, Julia C. Stingl, Katja S. Just   +8 more
wiley   +1 more source

Real-time experience: A systematic review of ecological momentary assessments of everyday functioning in major depressive disorder. [PDF]

Aust Occup Ther J
Volovic-Shushan S, Josman N, Nitzan U, Stern A.   +3 more
europepmc   +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