Results 191 to 200 of about 1,027,150 (305)

Early‐stage health technology assessment of a curative gene therapy for multiple sclerosis

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Aims Multiple sclerosis (MS) is associated with significant early morbidity, reduced life expectancy and substantial healthcare and societal costs. The primary objective of this study is to assess the early cost‐effectiveness potential of a novel gene therapy, IMMUTOL, for MS compared with current high‐efficacy treatment sequences.
Attila Imre, Balázs Nagy, Rok Hren
wiley   +1 more source

The Economic Costs of Congenital Hearing Loss in a South African Cohort. [PDF]

open access: yesHealth Serv Insights
Tombe-Mdewa W   +5 more
europepmc   +1 more source

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

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

open access: yesBritish 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

A plan for black American reparations. [PDF]

open access: yesBMJ Glob Health
Mullen AK   +3 more
europepmc   +1 more source

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

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

Safety concerns reported by coroners following fentanyl patch fatalities in England, Wales and Northern Ireland between 1997 and 2024

open access: yesBritish Journal of Clinical Pharmacology, EarlyView.
Abstract Aim To identify safety concerns reported by coroners following fentanyl patch‐related deaths in England, Wales and Northern Ireland, and determine differences in coronial reporting. Methods A systematic case series linking the National Programme on Substance Use Mortality (NPSUM) and the Preventable Deaths Tracker (PDT) (https ...
Eman Mshari   +2 more
wiley   +1 more source

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