One of the features of the ICER stakeholder involvement in the development of ICER evidence reports is the ability for public comment. Unfortunately, and this may just a miscommunication, the replies from ICER to public comments frequently miss the point or fail to provide backup for their claims. The purpose of this commentary is to review ICER's responses to public comments by the author on the just released final evidence report on cystic fibrosis. The message is quite simple: the ICER value assessment framework lacks credibility. It fails to meet the standards of normal science. This is seen in ICERs apparent ignorance or rejection of the axioms of fundamental measurement which point quite clearly to the mathematical impossibility of creating QALYs from generic multiattribute utility scores. The ICER report also fails standards by creating a model from prior assumptions; there is no logical basis for constructing a value assessment claim. Either ICER should withdraw its value claims or admit the dubious basis on which the model is built, as a duty to its readership.
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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8051921 | PMC |
http://dx.doi.org/10.24926/iip.v11i2.3248 | DOI Listing |
Lancet
January 2025
Centre for Inflammatory Disease, Department of Immunology and Inflammation, Imperial College London, London, UK; Imperial College Renal and Transplant Centre, Imperial College Healthcare NHS Trust, Hammersmith Hospital, London, UK. Electronic address:
Background: In the UK, booster COVID-19 vaccinations have been recommended biannually to people considered immune vulnerable. We investigated, at a population level, whether the absence of detectable anti-SARS-CoV-2 spike protein IgG antibody (anti-S Ab) following three or more vaccinations in immunosuppressed individuals was associated with greater risks of infection and severity of infection.
Methods: In this prospective cohort study using UK national disease registers, we recruited participants with solid organ transplants (SOTs), rare autoimmune rheumatic diseases (RAIRDs), and lymphoid malignancies.
J Biol Chem
January 2025
Department of Pharmaceutical Chemistry, University of California, San Francisco, San Francisco, CA 94158, USA; Department of Cellular and Molecular Pharmacology, University of California, San Francisco, San Francisco, CA 94158, USA. Electronic address:
Carboxyl-terminus of Hsp70-Interacting Protein (CHIP) is an E3 ubiquitin ligase that marks misfolded substrates for degradation. Hyper-activation of CHIP has been implicated in multiple diseases, including cystic fibrosis and cancer, suggesting that it may be a potential drug target. However, there are few tools available for exploring this possibility.
View Article and Find Full Text PDFInt J Pharm
January 2025
CIDETEC, Basque Research and Technology Alliance (BRTA), Parque Científico y Tecnológico de Gipuzkoa, Donostia-San Sebastián, Spain; Kusudama Therapeutics SA, Parque Científico y Tecnológico de Gipuzkoa, Donostia-San Sebastián, Spain; Biogipuzkoa Health Research Institute, Group of Innovation, 20014 San Sebastian, Spain.
Cystic fibrosis (CF) is characterized by abnormal mucus hydration due to a defective CF Transmembrane Regulator (CFTR) protein, leading to the production of difficult-to-clear mucus. This causes airflow obstruction, recurrent infections, and respiratory complications. Chronic lung infections are the leading cause of death for CF patients and inhaled tobramycin is the first-in-line antibiotic treatment against these infections, mainly caused by Pseudomonas aeruginosa in adult patients.
View Article and Find Full Text PDFPharmaceutics
January 2025
School of Pharmacy, Queen's University Belfast, Belfast BT9 7BL, UK.
This paper presents a comprehensive review of the current literature, clinical trials, and products approved for the delivery of antibiotics to the lungs. While there are many literature reports describing potential delivery systems, few of these have translated into marketed products. Key challenges remaining are the high doses required and, for powder formulations, the ability of the inhaler and powder combination to deliver the dose to the correct portion of the respiratory tract for maximum effect.
View Article and Find Full Text PDFInt J Mol Sci
January 2025
Department of Pediatrics, National Jewish Health, Denver, CO 80206, USA.
The cystic fibrosis transmembrane conductance regulator (CFTR) is an anion channel that is dysfunctional in individuals with cystic fibrosis (CF). The permeability of CFTR can be experimentally manipulated though different mechanisms, including activation via inducing the phosphorylation of residues in the regulatory domain as well as altering the gating/open probability of the channel. Phosphorylation/activation of the channel is achieved by exposure to compounds that increase intracellular cAMP, with forskolin and IBMX commonly used for this purpose.
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