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Background And Objective: Cancer drug costs have increased considerably within healthcare systems, but many drugs lack quality-of-life (QoL) and overall survival (OS) data at the time of reimbursement approval. This study aimed to review the extent of subsequent literature documenting improvements in OS and QoL for cancer drug indications where no such evidence existed at the time of reimbursement approval.
Methods: Drug indications with claims of added therapeutical value but a lack of evidence on OS and QoL that were reimbursed between 2010 and 2020 in Sweden were included for review. Searches were conducted in PubMed and ClinicalTrial.gov for randomized controlled trials examining OS and QoL.
Results: Of the 22 included drug indications, seven were found to have at least one trial with conclusive evidence of improvements in OS or QoL after a mean follow-up of 6.6 years. The remaining 15 drug indications either lacked subsequent randomized controlled trial data on OS or QoL (n = 6) or showed no statistically significant improvements (n = 9). Only one drug demonstrated evidence of improvement in both OS and QoL for its indication.
Conclusions: A considerable share of reimbursed cancer drug indications continue to lack evidence of improvement in both OS and QoL. With limited healthcare resources and an increasing cancer burden, third-party payers have strong incentives to require additional post-reimbursement data to confirm any improvements in OS and QoL.
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http://dx.doi.org/10.1007/s40261-023-01285-4 | DOI Listing |
J Pediatric Infect Dis Soc
September 2025
Infectious Diseases Unit, 3rd Department of Pediatrics, Aristotle University School of Medicine, Hippokration Hospital, Thessaloniki, Greece.
Background: Critically ill pediatric patients admitted to the PICU are highly vulnerable to infections, including invasive fungal diseases and antifungal agents are frequently prescribed. Little is known about antifungal usage in PICUs across Europe.
Methods: A multinational 3-month weekly point-prevalence study for measuring antifungal drug use was organized.
J Comp Eff Res
September 2025
British Heart Foundation, University of Glasgow, Glasgow, UK.
The first paper of this two-part series critically examined the role of composite endpoints in health technology assessments (HTAs) and outlined strategies for determining whether to employ the composite estimate of treatment effect or disaggregate into the component endpoints of the composite and apply separate treatment effects within a modeling framework. In this second paper, we expand the discussion beyond a pivotal trial and consider the way in which additional evidence from the same indication for different drugs in the same class, or the same drug for different indications, could be employed within HTAs. We offer a continuation of the case study of dapagliflozin for the treatment of heart failure with preserved or mildly reduced ejection fraction, where the evidence base was expanded to consider empagliflozin for the same indication, as well as both dapagliflozin and empagliflozin for heart failure with reduced ejection fraction.
View Article and Find Full Text PDFCureus
August 2025
Department of Urology, The Institute of Medical Science, The University of Tokyo, Tokyo, JPN.
In patients with advanced urothelial carcinoma who have progressed after platinum-based chemotherapy, enfortumab vedotin (EV) improves overall survival compared to standard chemotherapy. Additionally, for treatment-naïve patients with locally advanced or metastatic urothelial carcinoma, the combination of pembrolizumab and EV demonstrates superior efficacy over platinum-based chemotherapy. Hence, EV becomes a standard treatment option.
View Article and Find Full Text PDFJ Med Case Rep
September 2025
Department of Anesthesiology, LMU University Hospital Munich LMU, Marchioninistrasse 15, 81377, Munich, Germany.
Background: The treatment of critically ill patients in intensive care units is becoming increasingly complex. For example, organ transplants are regularly carried out, the recipients are seriously ill, and the postoperative course can be complicated. This is why organ replacement and hemadsorption procedures are becoming increasingly important.
View Article and Find Full Text PDFJMIR Res Protoc
September 2025
Service of Clinical Pharmacology, Department of Medicine, Lausanne University Hospital and University of Lausanne, Lausanne, Switzerland.
Background: Janus kinase inhibitors (JAKIs) are small molecules used orally to treat inflammatory and hematological disorders. They have demonstrated impressive efficacy across multiple indications. However, concerns have emerged regarding their safety profile.
View Article and Find Full Text PDF