Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2025 Mar;43(3):283-296.
doi: 10.1007/s40273-024-01446-z. Epub 2024 Nov 15.

Unravelling the Association Between Uncertainties in Model-based Economic Analysis and Funding Recommendations of Medicines in Australia

Qunfei Chen  1   2 , Martin Hoyle  1 , Varinder Jeet  1 , Yuanyuan Gu  3 , Kompal Sinha  4 , Bonny Parkinson  5
Affiliations

Unravelling the Association Between Uncertainties in Model-based Economic Analysis and Funding Recommendations of Medicines in Australia

Qunfei Chen et al. Pharmacoeconomics. 2025 Mar.

Abstract

Objective: Health technology assessment is used extensively by the Pharmaceutical Benefits Advisory Committee (PBAC) to inform medicine funding recommendations in Australia. The PBAC often does not recommend medicines due to uncertainties in economic modelling that result in delaying access to medicines for patients. The systematic identification of which uncertainties can be reduced with alternative evidence or the collection of additional data can help inform recommendations. This study aims to characterise different types of uncertainty in economic models and empirically assess their association with the PBAC recommendations.

Methods: A framework was developed to characterise four types of uncertainties: methodological, structural, generalisability and parameter uncertainty. The first two types were further subcategorised into parameterisable and unparameterisable uncertainty. Data on uncertainty and other factors were extracted from PBAC's Public Summary Documents of first submissions for 193 medicine (vaccine)-indication pairs including economic modelling between 2014 and 2021. Logistic regression was used to estimate the average marginal effect of each type of uncertainty on the probability of a positive recommendation.

Results: The PBAC more often raised issues regarding parameter uncertainty (95%) and parameterisable structural uncertainty (83%) than generalisability uncertainty (48%) and unparameterisable methodological uncertainty (56%). The logistic regression results suggested that the PBAC was more likely to recommend a medicine without unparameterisable methodological, generalisability, and parameterisable structural uncertainty by 15.0%, 10.2 %, and 17.6%, respectively. Parameterisable methodological, unparameterisable structural and parameter uncertainty were not significantly associated with the PBAC recommendations.

Conclusions: This study identified the uncertainties that had significant associations with PBAC recommendations based on the first submission. This may help improve model quality and reduce resubmissions in the future, thus improving patients' access to medicines.

PubMed Disclaimer

Conflict of interest statement

Declarations. Funding: Open Access funding enabled and organized by CAUL and its Member Institutions. This study is funded by International Macquarie University Research Excellence Scholarship (no. 2018152), Macquarie University Australian Pharmaceutical Scholarship (no. 2019022) and Social Science Projects of Gansu Province of China (no. 21CX1ZA174 and no. GSWSKY2020-76). Five pharmaceutical companies contributed to the Macquarie University Australian Pharmaceutical Scholarship: Amgen Australia, Janssen Australia, MSD Australia, Pfizer Australia, and Roche Australia. No sources of financial assistance were received by M.H., V.J., K.S., Y.G., and B.P. Conflict of Interest: All authors have no conflict of interest. B.P., M.H. and V.J. conduct evaluations of medicines for the Pharmaceutical Benefits Advisory Committee. The views expressed in the paper are not those of the PBAC or the Commonwealth Government of Australia. Data Availability: The data that support the findings of this study were abstracted from the Public Summary Documents ( https://www.pbs.gov.au/pbs/industry/listing/elements/pbac-meetings/psd ). The data are available from the authors upon reasonable request. Code Availability: The code for all analyses reported in the manuscript is available on request. Authorship: Q.C., M.H., V.J., K.S. and Y.G. contributed to the conception of the study. Q.C., B.P., M.H., Y.G., and V.J. formalised the framework. Q.C. extracted the information and V.J. cross-checked the data for a random sample. Q.C. conducted the statistical analyses in consultation with Y.G. All authors contributed to the interpretation of results, drafting and reviewing it critically for the intellectual content.

Figures

Fig. 1
Fig. 1
The characterisation framework of different types of uncertainty. PBS Pharmaceutical Benefits Scheme
Fig. 2
Fig. 2
Levels of uncertainty acknowledged by the PBAC based on results
See this image and copyright information in PMC

Similar articles

See all similar articles

Cited by

References

    1. Taylor RS, Drummond MF, Salkeld G, Sullivan SD. Inclusion of cost effectiveness in licensing requirements of new drugs: the fourth hurdle. BMJ (Clinical research ed). 2004;329(7472):972–5. 10.1136/bmj.329.7472.972. - PMC - PubMed
    1. McCabe C, Claxton K, Culyer AJ. The NICE cost-effectiveness threshold: what it is and what that means. Pharmacoeconomics. 2008;26(9):733–44. 10.2165/00019053-200826090-00004. - PubMed
    1. Ghabri S, Hamers FF, Josselin JM. Exploring uncertainty in economic evaluations of drugs and medical devices: lessons from the first review of manufacturers’ submissions to the French National Authority for Health. Pharmacoeconomics. 2016;34(6):617–24. 10.1007/s40273-016-0381-4. - PubMed
    1. Masucci L, Beca J, Sabharwal M, Hoch JS. Methodological issues in economic evaluations submitted to the pan-Canadian Oncology Drug Review (pCODR). Pharmacoecon Open. 2017;1(4):255–63. 10.1007/s41669-017-0018-3. - PMC - PubMed
    1. Catchpole P, Barrett V. Keeping Pace with pharmaceutical innovation: the importance of the NICE methods review. Pharmacoeconomics. 2020;38(9):901–3. 10.1007/s40273-020-00918-2. - PubMed

LinkOut - more resources