Application of surrogate outcomes for reimbursement decision-making in prostate cancer
- Publication Type:
- Thesis
- Issue Date:
- 2024
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This thesis examines the use of surrogate outcomes to support reimbursement decision-making for patients with localised prostate cancer (LPC). The selection of LPC was motivated by its high prevalence and its natural history necessitating lengthy clinical trials, most often powered on detecting differences in overall survival (OS). The thesis begins by outlining the difficulties of conducting clinical trials due to the extended natural history of LPC. To address this challenge, surrogate outcomes can enable earlier observations before final outcomes become available. Within prostate cancer, the ICECaP study demonstrated that 5-year metastases free survival (MFS) was a strong surrogate for 8-year OS. This implies a potential reduction of three years in the reporting time of LPC trial data, thereby enabling earlier access to new treatments if MFS informs an economic evaluation as the foundation for reimbursement decisions.
A systematic review indicated a lack of robust published evidence supporting the use of surrogate outcomes in economic evaluations of LPC. The objectives of this thesis were threefold: first, to evaluate the viability of applying surrogate outcomes in economic evaluations for LPC; second, to construct an economic model using a surrogacy relationship to assess the societal benefits and costs associated with the use of surrogate outcomes in reimbursement decisions for LPC; and third, to examine the trade-offs between data maturity and time in forming advice for reimbursement considerations.
The nature of the surrogacy relationship impacts the data requirements for the model which in turn influences how can be constructed. Therefore, the model selection was influenced by the available data and the underlying disease pattern of LPC. The model drew upon clinical guidelines and input from clinical experts to reflect practice in Australia. However, its flexibility enables it to be adjusted for use in other settings and clinical contexts.
The analysis of preferences indicated that the availability of data from longer-term studies was a significant factor in positively supporting reimbursement decisions. The results also showed that the potential uncertainty associated with shorter-term trials, perhaps reflected in the use of surrogate outcomes, could be mitigated through interventions with lower (better) incremental cost-effectiveness ratios (ICERs).
The findings of this thesis indicate that constructing a model using a surrogate outcome is feasible and could potentially expedite a reimbursement decision-making process. However, for this approach to ultimately enhance societal welfare hinges on whether the expected benefits materialise in relation to the opportunity cost of making an earlier decision.
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