A deliberative framework to identify the need for real-life evidence building of new cancer drugs after interim funding decision

Abstract

Background

With the rising cost of new oncology treatments, it is no longer sustainable to base initial drug funding decisions primarily on prospective clinical trials as their performance in real-life populations are often difficult to determine. In British Columbia, an approach in evidence building is to retrospectively analyse patient outcomes using observational research on an ad hoc basis.

Methods

The deliberative framework was constructed in three stages: framework design, framework validation and treatment programme characterization, and key informant interview. Framework design was informed through a literature review and analyses of provincial and national decision-making processes. Treatment programmes funded between 2010 and 2013 were used for framework validation. A selection concordance rate of 80% amongst three reviewers was considered to be a validation of the framework. Key informant interviews were conducted to determine the utility of this deliberative framework.

Results

A multi-domain deliberative framework with 15 assessment parameters was developed. A selection concordance rate of 84.2% was achieved for content validation of the framework. Nine treatment programmes from five different tumour groups were selected for retrospective outcomes analysis. Five contributory factors to funding uncertainties were identified. Key informants agreed that the framework is a comprehensive tool that targets the key areas involved in the funding decision-making process.

Conclusions

The oncology-based deliberative framework can be routinely used to assess treatment programmes from the major tumour sites for retrospective outcomes analysis. Key informants indicate this is a value-added tool and will provide insight to the current prospective funding model.