Industry and Academic Perceptions of Additional Sources of Value in Health Technology Assessment in Australia—More Similar Than Different

OBJECTIVES: To investigate the opinions of Australian stakeholders regarding the sources of value considered in reimbursement decisions for medicines for rare diseases, methods to assess cost-effectiveness (CE), and mechanisms to manage uncertainty in health technology assessment (HTA).

METHODS: An online survey was emailed to stakeholders, seeking levels of agreement with statements on HTA methods, sources of value for medicines (generally and for rare diseases), transparency of decision-making processes, and managing uncertainty in decision-making. Responses were summarised using descriptive statistics. Comparisons between groups were made using chi-square statistics.

RESULTS: Forty-four respondents, from academia (N=11) and the private sector (pharmaceutical companies and consultants, N=33) completed the survey between October 2023 and May 2024. Academia and private sector beliefs were consistent that current methods to assess the CE of medicines in Australia for medicines generally and for rare disease are inadequate, and that the current public information about the sources of value considered in reimbursement decisions is insufficient. Respondents agreed that labour productivity, adherence, reducing uncertainty due to a new diagnostic, disease severity, value to caregivers, and equity should be considered in HTA. Likewise, they agreed that outcome based managed entry agreements (MEA), financial risk share arrangements (RSA) and multi criteria decision analysis (MCDA), should be used in making reimbursement decisions. Significantly fewer academic stakeholders (40%) compared with the private sector (77%) believed an increase to the incremental cost effectiveness ratio (ICER) considered acceptable should be used in reimbursement decision making for medicines that treat rare diseases.

CONCLUSIONS: Stakeholder views were similar and did not diverge between medicines generally and for rare disease. Academic and private sector stakeholders favour the consideration of more value elements in HTA than referred to in the PBAC guidelines. This study highlights that further advice in reimbursement guidelines and transparency in public summary documents of the values considered is needed.