Results 71 to 80 of about 37,349 (258)

Cost‐utility analysis of nusinersen–risdiplam switch in patients with spinal muscular atrophy in Croatia: A discrete event simulation model

British Journal of Clinical Pharmacology, EarlyView.
Introduction In recent years, the treatment of spinal muscular atrophy (SMA), a rare disease, has significantly progressed, improving patients' survival and overall quality of life. However, current SMA treatments are expensive, and some (nusinersen) are very inconvenient for patients.
Andrej Belančić, Ivana Stević, Elvira Meni Maria Gkrinia, Dinko Vitezić, Slobodan Janković   +4 more
wiley   +1 more source

How the HTAR will contribute to a value‐based decision‐making for medicinal products across the EU

British Journal of Clinical Pharmacology, EarlyView.
The European Union Health Technology Regulation 2021/2282 (HTAR) introduces joint assessment of health technologies (including medicinal products and medical devices) across EU Member States. It was signed into law in 2021 and came into full force in January 2025.
Roisin Adams, Michal Stanak
wiley   +1 more source

Impact of reimbursement systems on patient care – a systematic review of systematic reviews

Health Economics Review
Background There is not yet sufficient scientific evidence to answer the question of the extent to which different reimbursement systems influence patient care and treatment quality. Due to the asymmetry of information between physicians, health insurers
Eva Wagenschieber, Dominik Blunck
doaj   +1 more source

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

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

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

Precision medicine in paediatrics: Progress and priorities

British Journal of Clinical Pharmacology, EarlyView.
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain, Claire Booth, Joseph F. Standing, Charlotte I. S. Barker   +3 more
wiley   +1 more source

Implementation of a billable transitional care model for stroke patients: the COMPASS study

BMC Health Services Research, 2019
Background The COMprehensive Post-Acute Stroke Services (COMPASS) pragmatic trial compared the effectiveness of comprehensive transitional care (COMPASS-TC) versus usual care among stroke and transient ischemic attack (TIA) patients discharged home from ...
Sabina B. Gesell, Cheryl D. Bushnell, Sara B. Jones, Sylvia W. Coleman, Samantha M. Levy, James G. Xenakis, Barbara J. Lutz, Janet Prvu Bettger, Janet Freburger, Jacqueline R. Halladay, Anna M. Johnson, Anna M. Kucharska-Newton, Laurie H. Mettam, Amy M. Pastva, Matthew A. Psioda, Meghan D. Radman, Wayne D. Rosamond, Mysha E. Sissine, Joanne Halls, Pamela W. Duncan   +19 more
doaj   +1 more source

Hair analysis of formoterol as a novel tool to assess long‐term inhalation medication adherence: Data from the TRICOLON trial

British Journal of Clinical Pharmacology, EarlyView.
Aim The aim of this paper is to examine inhaled formoterol concentrations in hair and assess the potential of hair analysis as a tool for measuring long‐term adherence, by validation with objective data from digital inhalers. Methods Hair samples and digital inhaler data were collected from patients with COPD in 10 hospitals in the Netherlands as part ...
Liz J. A. Cuperus, A. Mireille A. Wessels, Huib A. M. Kerstjens, Daan J. Touw, Erik W. M. A. Bischoff, Job van der Palen, Arnoud Aldenkamp, Astrid van Huisstede, Folkert Brijker, Stephan Dik, Lidwien Graat‐Verboom, Martijn Goosens, Peter Th. W. van Hal, Jolanda C. Kuijvenhoven, Lisette I. Z. Kunz, Renske van der Meer, Mariette H. A. Scholma‐Bronsema, Job F. M. van Boven, Johannes C. C. M. in't Veen, the TRICOLON study group, Marjo van der Poel, Irini Piso, Marijke Amelink, Anke Rol, Jennifer de Graaf, Petra Hirmann, Fleur van Tour, Elly Jordens, Lydia Alfing, Gerda Lenderink, Truus Rietveld, Jasmijn van Campen, Jantine de Bruijn, Janice van ter Burg, Liselore Baarssmit, Walter van Litsenburg, Len Knoops, Margot Eggermont‐Schilt, Manon de Waard‐Heijligers, Ilonka Paalvast‐Schouten, Mariette Scholma, Sandra Brookman‐Mozes, Inge Paas, Lidwien Verboom, Karin van Mierlo‐Snoeijs   +44 more
wiley   +1 more source

Standardization of measurement and reporting of medication adherence using the TEN‐SPIDERS tool for claims data

British Journal of Clinical Pharmacology, EarlyView.
This study provides the first open‐source tool to implement the complete TEN‐SPIDERS framework in linked datasets for the determination of the PDC for medication adherence. The TEN‐SPIDERS tool enables transparent, reproducible medication adherence research through automated reporting of all calculation parameters and flexible customization options ...
Adam C. Livori, Tzu‐Yung Kuo, Jenni Ilomäki, Monique F. Kilkenny, Stella Talic, Zanfina Ademi, J. Simon Bell, Jedidiah I. Morton, Lachlan L. Dalli   +8 more
wiley   +1 more source