Elicitation of health state utilities in neuroendocrine tumours

Abstract

Background:

Neuroendocrine tumours (NETs) are a rare form of neoplasm that can arise in most organs of the body and which share many common pathologic features. Although curative surgery can be conducted for patients with localised disease, once progression occurs and the disease becomes metastatic or un-resectable, treatment aims to extend life and maintain quality-of-life for as long as possible. The aim of the study was to elicit utilities for health state vignettes describing the burdens associated with receiving therapy for advanced NETs.

Methods:

Health state vignettes were developed by reviewing published literature and conducting in-depth interviews with patients and clinical experts. These states described the burden associated with both stable and progressive disease, in addition to the experience of a number of serious toxicities commonly associated with treatments (grade III/IV diarrhoea, hand-foot syndrome, hyperglycaemia, nausea/vomiting, pneumonitis, rash, stomatitis, and thrombocytopenia). One hundred members of the UK general public valued the states using the time trade-off methodology to determine utility values.

Results:

Stable disease had a utility value of 0.77 whilst disease progression was associated with a significant decline in health-related quality-of-life (HRQoL) and a value of 0.61. Toxicities experienced in the context of stable disease exhibited varying degrees of impact, with several being deemed as debilitating as disease progression (such as hand-foot syndrome [0.58] and stomatitis [0.56]).

Conclusion:

Although vignette studies have been criticised for the difficulty in establishing their validity, the collection of health utilities in rare populations is challenging. The findings from this study suggest that advanced NETs is associated with a considerable HRQoL burden, both as a direct result of the disease and the potential of experiencing a number of severe adverse events. These values could assist in future economic evaluation processes.

Keywords::

• Quality-of-life
• Neuroendocrine tumour
• Utility
• Cost-effectiveness

Transparency

Declaration of funding

This study was funded by Novartis.

Declaration of financial/other interests

Paul Swinburn and Andrew Lloyd are employees of Oxford Outcomes, an ICON plc company, who were paid for designing and conducting the study. Jenny Wang and David Chandiwana are employees and shareholders of Novartis, and both authors were involved in the writing of the manuscript, and the decision to submit the paper for publication. Was Mansoor is the recipient of an honorarium from Novartis.

Acknowledgements

The authors acknowledge the contributions made by: Andrea Burgess, Royal Hampshire County Hospital, Winchester, UK; Dr David Farrugia, Cheltenham General Hospital, Cheltenham, UK; Amir Khan, Manor Hospital, Walsall, UK; Dr Denis Talbot, Churchill Hospital, Oxford, UK; and Dr Juan Valle, The Christie, Manchester, UK, for providing their clinical expertise. Honoraria for those contributions were provided by Novartis.