Introduction: Under the requirements for listing of new drugs on the Australian Pharmaceutical Benefits Scheme, companies are required to submit applications supporting the cost-effectiveness of the product for its proposed indication. Guidelines for these submissions have been available since 1992. There has been a recent change to the Guidelines, so that companies are now encouraged to submit an economic analysis based on trial data, as well as one based on economic modelling.

Objective: (1) To assess the usage of RCTs in economic analysis reviewed by the Pharmaceutical Benefits Advisory Committee (PBAC). (2) To assess the impact of use of RCTs on listing decisions by the PBAC.

Methods: A database was set up that records all submissions to the PBAC since 1992. The database is being analysed for: the number and type of submissions; the proportion using RCTs alone as the basis of the economic analysis; the proportion for which no RCTs are available and so require modelled analysis; those presenting both RCTs and modelled analyses. Differences in cost-effectiveness estimates between the 2 types of analysis have also been recorded where possible.

Results: At least 170 submissions have been reviewed by the PBAC since the introduction of the Guidelines in 1992. The proportion using RCTs is increasing, and the difference between the outcomes of analyses using RCTs and those using modelled economic analyses will be presented.

Discussion: Use of RCTs for making policy and resource allocation decisions is likely to increase. This project describes some of the important differences between trial-based and modelled estimates of the cost-effectiveness of new drug therapies.