Category Ranking
98%
Total Visits
921
Avg Visit Duration
2 minutes
Citations
20
Article Abstract
Background: In the absence of long-term data, structured expert elicitation gathers expert judgments and associated uncertainties to assess the clinical plausibility of long-term extrapolations.
Objective: The objective of this study was to obtain expert estimates of expected long-term outcomes for advanced renal cell carcinoma treatments to inform cost-effectiveness analysis for National Institute for Health and Care Excellence (NICE)'s pathways pilot.
Methods: Using materials from the structured expert elicitation resources (STEER) repository, aligned with the Medical Research Council (MRC) protocol, the exercise was planned and conducted. Aiming for 5-10 oncologists from diverse UK geographies and settings, experts estimated progression-free survival (PFS) at three landmark timepoints for 21 disease-risk-prior treatment combinations and overall survival for best supportive care. Within an 8-week timeframe, we piloted with one clinician, conducted online training, collected responses via an online survey using the roulette method and mathematically aggregated results through linear opinion pooling.
Results: Nine experts participated (question response rate: 95%). For first-line intermediate/poor-risk patients, clinicians projected similar PFS for three immune oncology/tyrosine kinase inhibitor (TKI) combinations from 5 years onward and comparable PFS for two TKI monotherapies. Nivolumab + ipilimumab was anticipated to achieve the highest PFS amongst first-line therapies. Expert reasoning incorporated treatment class, clinical experience, and awareness of trial data optimism. Expert estimates were generally somewhat optimistic compared with observed UK real-world evidence and pessimistic compared with observed trial data.
Conclusions: Structured expert elicitation is a pragmatic, efficient approach for informing long-term survival extrapolations in the context of a rapidly evolving treatment pathway. We demonstrated that expert elicitation is possible even for complex decision problems in a relatively short timeframe.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1007/s40258-025-01000-8 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Assessing the impact of perfluoroalkyl substances on liver health: a comprehensive study using multi-donor human liver spheroids.
Environ Int
September 2025
Division of Gastrointestinal and Liver Diseases, Department of Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA, United States. Electronic address:
Background: Although per- and polyfluoroalkyl substances (PFAS) have been linked to chronic liver diseases, the specific cellular and molecular mechanisms by which different PFAS contribute to human liver dysfunction remain unclear. This study aims to elucidate those mechanisms.
Methods: We exposed a multi-donor human liver spheroid model composed of multiple cell types to 20 µM of PFHxS, PFOA, PFOS, or PFNA for seven days, followed by single-cell RNA sequencing and lipid staining.
Too vulnerable? Successful practices for conducting research with children and young people who have life-limiting or life-threatening illness.
Palliat Med
September 2025
Florence Nightingale Faculty of Nursing, Midwifery and Palliative Care, Cicely Saunders Institute, King's College London, London, UK.
Background: A dearth of evidence exists on how to include children and young people in palliative care research.
Aim: We aimed to identify successful practices in involvement, recruitment and data collection with children and young people with life-limiting illness in research.
Design: We synthesised methods from five primary studies from three geographical regions in which children with life-limiting conditions were recruited and interviewed.
Understanding lived experiences with KCNQ2 developmental and epileptic encephalopathy.
Epilepsy Behav
September 2025
Biohaven Pharmaceuticals, Inc., New Haven, CT, USA.
Background: KCNQ2 developmental and epileptic encephalopathy (KCNQ2-DEE) is a rare pediatric disorder characterized by seizures and neurodevelopmental impairments. Parent- and healthcare professional (HCP)-reported outcomes regarding the impacts of seizures and neurodevelopmental impairments may guide the design of clinically meaningful KCNQ2-DEE outcome measures.
Methods: Parents of children with KCNQ2-DEE (N = 53) and HCPs with KCNQ2-DEE expertise (N = 2) participated in qualitative interviews exploring signs, symptoms, and impacts of KCNQ2-DEE, and how varying KCNQ2-DEE phenotypes affect child development.
Mapping variation in dual use risk assessments of synthetic biology projects.
Front Bioeng Biotechnol
August 2025
Center for International Security and Cooperation, Stanford University, Stanford, CA, United States.
Introduction: When a life science project is identified as potential dual use research of concern (DURC), United States government policy and biorisk management professionals recommend conducting a risk assessment of the project and using its results to choose strategies to manage any associated risks. However, there is little empirical research on how real-world projects score on DURC assessments, the extent to which reviewers agree or disagree about risks for a given project, or how risk judgments map to recommended risk management strategies. By studying the process of DURC risk assessment, it may be possible to develop methods that are more consistent, accurate, and cost-effective.
View Article and Find Full Text PDFInterpreting immunogenicity in oncology clinical trials.
Cancer Treat Rev
August 2025
Department of Oncology, University Hospital CHUV, Lausanne, Switzerland.
Immunogenicity of a therapeutic protein product may elicit an unintended immune response, and is a critical aspect evaluated in oncology clinical trials. The development of anti-drug antibodies (ADAs) can impact the pharmacokinetics, pharmacodynamics, efficacy, and safety of these therapies. We review the background and nomenclature of immunogenicity assessment in oncology studies and emphasize the complexities in ADA detection arising from assay sensitivity, drug interference, and notably, the frequency of patient sampling for ADA analysis.
View Article and Find Full Text PDF