Valuation of the EORTC Quality of Life Utility Core 10 Dimensions (QLU-C10D) in a Multi-ethnic Asian Setting: How Does Having Cancer Matter?

The aim of the study was to develop and compare utility value sets for the EORTC QLU-C10D, a cancer-specific utility instrument based on the EORTC QLQ-C30, using the preferences of the general public and cancer patients in Singapore, and to assess their measurement properties. A total of 600 individuals from the general public were recruited using a multi-stage random sampling, along with 626 cancer patients with clinically confirmed diagnoses from outpatient clinics of the largest tertiary cancer hospital. Each participant valued 16 pairs of EORTC QLU-C10D health states using a discrete choice experiment (DCE). Conditional logit models were used to analyze the DCE responses of the general public and cancer patients separately. Utility values were assessed for known-group validity and responsiveness in the cancer patients by comparing mean values across subgroups of patients and calculating standardized response means using longitudinal EORTC QLQ-C30 data, respectively. Physical functioning and pain had the most impact on utility for both cancer patients and general public groups. Worst health state utility values were -0.821 and -0.463 for the general public and cancer patients, respectively. Cancer patients' values were lower for mild-to-moderate health states but higher for moderately-to-highly impaired states compared with the general public's values. Both value sets discriminated between patients with differing characteristics and responded equally well to improved health status, but the cancer patients' value set was slightly more responsive to deteriorated health. EORTC QLU-C10D value sets based on the preferences of the Singaporean general public and cancer patients exhibited differences in values but similar psychometric properties.

被引量：- 发表：1970

An Evaluation of an Algorithm for the Selection of Flexible Survival Models for Cancer Immunotherapies: Pass or Fail?

被引量：- 发表：1970

Different Models, Same Results: Considerations When Choosing Between Approaches to Model Cost Effectiveness of Chimeric-Antigen Receptor T-Cell Therapy Versus Standard of Care.

Chimeric antigen-receptor T-cell therapy (CAR-T) is characterised by early phase data at the time of registration, high upfront cost and a complex manufacturing and administration process compared with standard therapies. Our objective was to compare the performance of different models to assess the cost effectiveness of CAR-T using a state-transition model (STM), partitioned survival model (PSM) and discrete event simulation (DES). Individual data for tisagenlecleucel for the treatment of young patients with acute lymphoblastic leukaemia (ALL) were used to populate the models. Costs and benefits were measured over a lifetime to generate a cost per quality-adjusted life-year (QALY). Model performance was compared quantitatively on the outcomes generated and a checklist developed summarising the components captured by each model type relevant to assessing cost effectiveness of CAR-T. Models generated similar results with base-case analyses ranging from an incremental cost per QALY of $96,074-$99,625. DES was the only model to specifically capture CAR-T wait time, demonstrating a substantial loss of benefit of CAR-T with increased wait time. Although model type did not meaningfully impact base-case results, the ability to incorporate an outcome-based payment arrangement (OBA) and wait time are important elements to consider when selecting a model for CAR-T. DES provided greater flexibility compared with STM and PSM approaches to deal with the complex manufacturing and administration process that can lead to extended wait times and substantially reduce the benefit of CAR-T. This is an important consideration when selecting a model type for CAR-T, so major drivers of uncertainty are considered in funding decisions.

被引量：- 发表：1970

MPES-R: Multi-Parameter Evidence Synthesis in R for Survival Extrapolation-A Tutorial.

Survival extrapolation often plays an important role in health technology assessment (HTA), and there are a range of different approaches available. Approaches that can leverage external evidence (i.e. data or information collected outside the main data source of interest) may be helpful, given the extent of uncertainty often present when determining a suitable survival extrapolation. One of these methods is the multi-parameter evidence synthesis (MPES) approach, first proposed for use in HTA by Guyot et al., and more recently by Jackson. While MPES has potential benefits over conventional extrapolation approaches (such as simple or flexible parametric models), it is more computationally complex and requires use of specialist software. This tutorial presents an introduction to MPES for HTA, alongside a user-friendly, publicly available operationalisation of Guyot's original MPES that can be executed using the statistical software package R. Through two case studies, both Guyot's and Jackson's MPES approaches are explored, along with sensitivity analyses relevant to HTA. Finally, the discussion section of the tutorial details important considerations for analysts considering use of an MPES approach, along with potential further developments. MPES has not been used often in HTA, and so there are limited examples of how it has been used and perceived. However, this tutorial may aid future research efforts exploring the use of MPES further.

被引量：- 发表：1970

Treatment Effect Waning in Immuno-oncology Health Technology Assessments: A Review of Assumptions and Supporting Evidence with Proposals to Guide Modelling.

Treatment effect waning (TEW) refers to the attenuation of treatment effects over time. Assumptions of a sustained immuno-oncologic treatment effect have been a source of contention in health technology assessment (HTA). We review how TEW has been addressed in HTA and in the wider scientific literature. We analysed company submissions to English language HTA agencies and summarised methods and assumptions used. We subsequently reviewed TEW-related work in the ISPOR Scientific Presentations Database and conducted a targeted literature review (TLR) for evidence of the maintenance of immuno-oncology (IO) treatment effects post-treatment discontinuation. We found no standardised approach adopted by companies in submissions to HTA agencies, with immediate TEW most used in scenario analyses. Independently fitted survival models do however suggest TEW may often be implicitly modelled. Materials in the ISPOR scientific database suggest gradual TEW is more plausible than immediate TEW. The TLR uncovered evidence of durable survival in patients treated with IOs but no evidence that directly addresses the presence or absence of TEW. Our HTA review shows the need for a consistent and appropriate implementation of TEW in oncology appraisals. However, the TLR highlights the absence of direct evidence on TEW in literature, as TEW is defined in terms of relative treatment effects-not absolute survival. We propose a sequence of steps for analysts to use when assessing whether a TEW scenario is necessary and appropriate to present in appraisals of IOs.

被引量：- 发表：1970