Inborn errors of immunity: A field without frontiers

G Bucciol, S Delafontaine, I Meyts… - Immunological …, 2024 - Wiley Online Library
The study of primary immunodeficiencies or inborn errors of immunity continues to drive our
knowledge of the function of the human immune system. From the outset, the study of inborn …

[HTML][HTML] ATMP development and pre-GMP environment in academia: a safety net for early cell and gene therapy development and manufacturing

DN Silva, M Chrobok, G Ahlén, P Blomberg… - Immuno-Oncology and …, 2022 - Elsevier
Highlights•Challenges faced during ATMP development in academia.•Experience in
providing support to ATMP development by building and operating a pre-GMP core …

[HTML][HTML] Financing and reimbursement of approved advanced therapies in several European countries

C Iglesias-López, A Agustí, A Vallano, M Obach - Value in Health, 2023 - Elsevier
Objectives The uncertainty in the cost-benefit of advanced therapy medicinal products
(ATMPs) is a current challenge for their reimbursement in health systems. This study aimed …

Cost-effectiveness of a new ACI technique for the treatment of articular cartilage defects of the knee compared to regularly used ACI technique and microfracture

M Snow, V Mandalia, R Custers, PJ Emans… - Journal of Medical …, 2023 - Taylor & Francis
Aims For patients with cartilage defects of the knee, a new biocompatible and in situ cross-
linkable albumin-hyaluronan-based hydrogel has been developed for matrix-associated …

Perspective from developers: Tissue-engineered products for skin wound healing

MM Godoi, EM Reis, J Koepp, J Ferreira - International Journal of …, 2024 - Elsevier
Tissue-engineered products (TEPs) are at the forefront of developmental medicines,
precisely where monoclonal antibodies and recombinant cytokines were 30 years ago …

A comprehensive value-based method for new nuclear medical service pricing: with case study of radium [223Ra] bone metastases treatment

H Wang, H Sun, Y Fu, W Cheng, C Jin, H Shi… - BMC Health Services …, 2024 - Springer
Importance Innovative nuclear medicine services offer substantial clinical value to patients.
However, these advancements often come with high costs. Traditional payment strategies …

In defense of expert knowledge in bioethical discussions on human genome editing

T Rzepiński - The American Journal of Bioethics, 2023 - Taylor & Francis
The development of innovative biomedical technologies that use gene editing tools opens
up new possibilities in the treatment of rare diseases, as well as diseases that were not …

Using automated text classification to explore uncertainty in NICE appraisals for drugs for rare diseases

L Wiedmann, J Blumenau, O Carroll… - International journal of …, 2024 - cambridge.org
ObjectiveThis study examined the application, feasibility, and validity of supervised learning
models for text classification in appraisals for rare disease treatments (RDTs) in relation to …

Methods for Extrapolating Survival Analyses for the Economic Evaluation of Advanced Therapy Medicinal Products

WAS Hardy, DA Hughes - Human Gene Therapy, 2022 - liebertpub.com
There are two significant challenges for analysts conducting economic evaluations of
advanced therapy medicinal products (ATMPs):(1) estimating long-term treatment effects in …

[PDF][PDF] Advanced therapy medicinal products: a comprehensive overview for pharmacy professionals

CMK McKenzie, J Chappell, R Guy… - The …, 2022 - researchportal.bath.ac.uk
Alternative formats If you require this document in an alternative format, please contact:
openaccess@bath.ac.uk Page 1 Citation for published version: McKenzie, CM, Chappell, J …