Results 181 to 190 of about 2,531 (253)

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

open access: yesBritish 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   +4 more
wiley   +1 more source

From tokenism to youth leadership in child and adolescent mental health research. [PDF]

open access: yesPublic Health Rev
Harrington SM   +7 more
europepmc   +1 more source

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

open access: yesBritish 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   +4 more
wiley   +1 more source

Precision medicine in paediatrics: Progress and priorities

open access: yesBritish 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   +3 more
wiley   +1 more source

Trends in publicly subsidized and private access to GLP‐1 medicines indicated for type 2 diabetes in Australia, 2020–2025

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Aims GLP‐1 medicines provide substantial cardio‐renal benefits for type 2 diabetes (T2D) and are increasingly used for weight loss. Most high‐income countries subsidize access for T2D with restrictions; use outside of these restrictions occurs through private prescriptions. Despite strong consumer demand, the extent of private access to GLP‐1 medicines
Michael O. Falster   +10 more
wiley   +1 more source

Defining a comprehensive set of value assessment criteria for funders of drug repurposing initiatives

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Aims This study aims to develop a structured list of value assessment criteria for drug repurposing (DR) projects designed to support consistent, evidence‐informed decision‐making by funders. The assessment criteria were developed within the REMEDi4ALL project, a multi‐stakeholder Horizon Europe funded initiative to advance DR and were validated ...
Dalma Hosszú   +10 more
wiley   +1 more source

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