Presentation (CTI)

OBJECTIVES: The objective of this study was to analyse characteristics of submissions to the Australian Pharmaceutical Benefits Advisory Committee (PBAC) in which the treatment effect is measured using RWE compared to those which are based on more traditional evidence (specifically randomised controlled clinical trial evidence).
METHODS: A database was created of all submissions to the PBAC including cost-utility analyses (CUAs) from March 2020 to July 2024 (n=341) based on Public Summary Documents, of which 280 submissions had a decision of either a recommendation or rejection. Relevant data was extracted, such as the submission decision and type of evidence to compare with other submission components such as the ICER, rarity of disease and economic model reliability in submissions with randomized evidence vs RWE. Summary statistics were used to analyse and compare recommendations and rejections by the PBAC in submissions that were and were not based on RWE.
RESULTS: Less than 20% (50/280) of submissions were based on RWE and less than half of those (22/50) were recommended by PBAC. Submissions recommended on the basis of RWE were more likely to be for rare conditions (77% versus 41% across all submissions), paediatric conditions (36% versus 14%) or have a smaller budget impact (approximately $14 million versus over $30 million per annum). Submissions based on RWE were more commonly considered to have a high risk of bias (90% compared to 16%). Despite this, RWE submissions were recommended at a higher incremental cost per QALY ratio ($159,000 compared to $75,000 in those recommended based on randomised evidence).
CONCLUSIONS: RWE is more commonly used in submissions for rare or severe conditions where a tolerance for bias in the evidence base and a higher willingness to pay is observed. This research may guide manufacturers on optimizing RWE to enhance HTA submission impact.