New Relevant Evidence in Cholangiocarcinoma Biology and Characterization.

Among solid tumors, cholangiocarcinoma (CCA) emerges as one of the most difficult to eradicate. The silent and asymptomatic nature of this tumor, particularly in its early stages, as well as the high heterogeneity at genomic, epigenetic, and molecular levels delay the diagnosis, significantly compromising the efficacy of current therapeutic options and thus contributing to a dismal prognosis. Extensive research has been conducted on the molecular pathobiology of CCA, and recent advances have been made in the classification and characterization of new molecular targets.

How are patient inputs considered in HTA? A thematic document analysis of NICE ultra-rare disease appraisals.

Patient organisations are increasingly involved in HTA. Given this, it is important to understand what these organisations contribute and how their voices are accounted for in the decision-making process. This study characterises inputs from patient organisations and/or their nominated patient experts in technology appraisals for ultra-rare diseases in England and Wales and seeks to understand how these are considered in NICE final recommendations.

Managing experts' conflicts of interest in the EU Joint Clinical Assessment.

Objective: This article critically evaluates the European Commission's 2024 Implementing Regulation (IR) on conflicts of interest (COIs) management for stakeholders in the European Union (EU) Joint Clinical Assessment (JCA), with a focus on individual experts such as clinicians and patient representatives.

Key Findings: The IR is the first EU-level framework to assess COIs in the context of health technology assessment (HTA). The regulation requires experts involved in the JCA to submit annual declarations of interest for both financial and non-financial interests and presents a matrix on whether these conflicts should disqualify them from participating in the joint work.

Clinical benefit, reimbursement outcomes, and prices of FDA-approved cancer drugs reviewed through Project Orbis in the USA, Canada, England, and Scotland: a retrospective, comparative analysis.

Background: Project Orbis is a global initiative that aims to streamline regulatory review processes across international regulators in the USA, Canada, Australia, UK, Israel, Brazil, Singapore, and Switzerland to bring promising cancer drugs to patients earlier. We explored the clinical benefit, time to regulatory approval and health technology assessment recommendations, reimbursement outcomes, and monthly treatment prices of cancer drugs reviewed through this initiative.

Methods: For this retrospective, comparative analysis, we identified cancer drug approvals reviewed through Project Orbis in the USA, Canada, and the UK between May 1, 2019, and Nov 1, 2023.

Oxidative Stress and Redox-Dependent Pathways in Cholangiocarcinoma.

Cholangiocarcinoma (CCA) is a primary liver tumor that accounts for 2% of all cancer-related deaths worldwide yearly. It can arise from cholangiocytes of biliary tracts, peribiliary glands, and possibly from progenitor cells or even hepatocytes. CCA is characterized by high chemoresistance, aggressiveness, and poor prognosis.