Results 191 to 200 of about 6,198 (250)

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

Fair priority setting in gene-editing research: the accountability for reasonableness framework. [PDF]

EMBO Mol Med
Smits MJ, van Daal M, Verweij M, Nieuwenhuis E, Fuchs S, Jongsma KR.   +5 more
europepmc   +1 more source

A New Outcomes-Based Payment Plan From a Chinese Company to Improve Patient Affordability of CAR-T Product. [PDF]

Int J Health Policy Manag
Zheng SL, Zhang XQ.
europepmc   +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

Non-Medical Activities in Dementia Care in Germany: Use and Experienced Effects. [PDF]

J Prim Care Community Health
Rodriguez FS, Ziegert N, Ross SD.
europepmc   +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

Determining the feasibility of linked claims and vaccination data for a COVID-19 vaccine pharmacoepidemiological study in Germany-RiCO feasibility study protocol. [PDF]

BMJ Open
Timmesfeld N, Ihle P, Denz R, Meiszl K, Scholz K, Oberle D, Drechsel-Bäuerle U, Keller-Stanislawski B, Diebner HH, Meyer I.   +9 more
europepmc   +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

Regional variations in healthcare in people living with dementia in Germany: protocol for a mixed-methods study. [PDF]

BMJ Open
Scheibner HJ, Gesell D, Hauptmann M, Heinze M, Horenkamp-Sonntag D, Koller D, Kubat D, Marschall U, Meixner J, Michalowsky B, von Peter S, Platen M, Riederer C, Schroth J, Swart E, Weirauch L, Dahling V.   +16 more
europepmc   +1 more source