Early‐stage health technology assessment of a curative gene therapy for multiple sclerosis
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
Clinical and Economic Trade-Offs in Antifungal Therapy for Invasive Aspergillosis and Mucormycosis: A Markov Model-Based Analysis. [PDF]
Rico Mendoza A +9 more
europepmc +1 more source
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
Economic Evaluation of a Digital Psychobehavioral Intervention for Chronic Inflammatory Skin Disease (MindMySkin): Protocol for a Trial-Based Cost-Effectiveness Analysis. [PDF]
Choi E, Neo SH, Phan P, Petrou S.
europepmc +1 more source
Cost-Effectiveness of Endovascular vs Open Surgery for Chronic Limb-Threatening Ischemia.
Zafari Z +15 more
europepmc +1 more source
Long-Term Cost and Health Impact of a Digital Obesity Intervention in Germany: Economic Modeling Study. [PDF]
Walter E, Kaup M, Althaus AE, Fehring L.
europepmc +1 more source
How the HTAR will contribute to a value‐based decision‐making for medicinal products across the EU
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
Manufacturing cost analysis and exploratory economic evaluation of natural killer cell immunotherapy for paediatric solid tumors. [PDF]
Wahid ST +6 more
europepmc +1 more source
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

