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How are Child-Specific Utility Instruments Used in Decision Making in Australia? A Review of Pharmaceutical Benefits
Cate Bailey1, Kim Dalziel2, Paula Cronin3
1Health Economics Unit, Melbourne School of Population and Global Health, University of Melbourne, 207 Bouverie St, Carlton 3053, Melbourne, VIC, Australia. cate.bailey@unimelb.edu.au.
Insights
Funding decisions for children's medicines in Australia rarely use child-specific health-related quality of life (HRQOL) measures. Consistent use of these measures is recommended to improve evidence for paediatric drug funding.
Area of Science:
- Health Economics
- Paediatric Medicine
- Health Policy
Background:
- Accurate measurement of health-related quality of life (HRQOL) in children is crucial for informed funding decisions on new interventions.
- The Pharmaceutical Benefits Advisory Committee (PBAC) in Australia reviews medicine funding submissions, impacting public subsidy.
- Child-specific HRQOL measures are essential for evaluating interventions in paediatric populations.
Purpose of the Study:
- To examine the utilization of child-specific HRQOL measures in PBAC funding submissions for medicines used by children in Australia.
- To assess the impact of using adult HRQOL measures versus child-specific measures in decision-making processes.
- To identify gaps and inconsistencies in the application of HRQOL data for paediatric medicines.
Main Methods:
- A systematic review of public summary documents for medicines used by children submitted to the PBAC.
- Identification of documents containing HRQOL information and/or cost-utility analyses relevant to children (under 18 years).
- Extraction and analysis of data on the types of HRQOL measures used (child-specific, adult, or undefined).
Main Results:
- Out of 1889 public summary documents, 62 pertained to children and utilities; only 4 utilized child-specific HRQOL measures.
- In 85% of cases, decision-making uncertainty could have been reduced by employing child-specific HRQOL measures.
- Submissions frequently used adult HRQOL measures, inconsistent approaches, and showed substantial evidence gaps.
Conclusions:
- The use of child-specific HRQOL measures in PBAC submissions for paediatric medicines in Australia is minimal.
- Current practices involve inconsistent methods and reliance on adult measures, leading to potential decision-making uncertainty.
- Consistent implementation of child-specific HRQOL measures is recommended to enhance the evidence base for paediatric medicine funding.
Abstract:
Measuring and valuing health-related quality of life (HRQOL) in children can be challenging but is an important component for providing decision makers with accurate information to fund new interventions, including medicines and vaccines for public subsidy. We review funding submissions of medicines made to the Pharmaceutical Benefits Advisory Committee contained in public summary documents to examine the use of child-specific HRQOL measures in decision making in Australia. A sample frame of medicines used by children was derived from four sources. Public summary documents relating to these medicines were searched in the Pharmaceutical Benefits Advisory Committee web resources for whether they related to children (aged under 18 years) and contained HRQOL information and/or cost-utility analyses. Data about the use of utilities in decision making were extracted and analysed. Of the 1889 public summary documents available, 62 public summary documents (29 medicines) contained information pertaining to children and utilities. Of these, four public summary documents included child-specific HRQOL measures, 16 included adult HRQOL measures, 11 included direct elicitation and the HRQOL source was not defined in 31 documents. Excluding documents using child-specific HRQOL measures, we considered that in 85% of medicines, decision making uncertainty might have been reduced by using child-specific HRQOL measures. Despite the growing literature on economic analysis in paediatric populations, the use of child-specific HRQOL measures in submissions to the Pharmaceutical Benefits Advisory Committee was minimal. Submissions involved inconsistent approaches, use of adult measures and weights, and substantial gaps in evidence. We recommend the consistent use of child-specific measures to improve the evidence base for decisions about medicines for children in Australia.
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