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. 2023 May;8(5):e011842.
doi: 10.1136/bmjgh-2023-011842.

Investing in a global pooled-funding mechanism for late-stage clinical trials of poverty-related and neglected diseases: an economic evaluation

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Investing in a global pooled-funding mechanism for late-stage clinical trials of poverty-related and neglected diseases: an economic evaluation

Armand Zimmerman et al. BMJ Glob Health. 2023 May.

Abstract

Introduction: Poverty-related and neglected diseases (PRNDs) cause over three million deaths annually. Despite this burden, there is a large gap between actual funding for PRND research and development (R&D) and the funding needed to launch PRND products from the R&D pipeline. This study provides an economic evaluation of a theoretical global pooled-funding mechanism to finance late-stage clinical trials of PRND products.

Methods: We modelled three pooled-funding design options, each based on a different level of coverage of candidate products for WHO's list of PRNDs: (1) vaccines covering 4 PRNDs, (2) vaccines and therapeutics covering 9 PRNDs and (3) vaccines, therapeutics and diagnostics covering 30 PRNDs. For each option, we constructed a discrete event simulation of the 2019 PRND R&D pipeline to estimate required funding for phase III trials and expected product launches through 2035. For each launch, we estimated global PRND treatment costs averted, deaths averted and disability-adjusted life-years (DALYs) averted. For each design option, we calculated the cost per death averted, cost per DALY averted, the benefit-cost ratio (BCR) and the incremental cost-effectiveness ratio (ICER).

Results: Option 1 averts 18.4 million deaths and 516 million DALYs, has a cost per DALY averted of US$84 and yields a BCR of 5.53. Option 2 averts 22.9 million deaths and 674 million DALYs, has a cost per DALY averted of US$75, an ICER over option 1 of US$49 and yields a BCR of 3.88. Option 3 averts 26.9 million deaths and 1 billion DALYs, has a cost per DALY averted of US$114, an ICER over option 2 of US$186 and yields a BCR of 2.52.

Conclusions: All 3 options for a pooled-funding mechanism-vaccines for 4 PRNDs, vaccines and therapeutics for 9 PRNDs, and vaccines, therapeutics and diagnostics for 30 PRNDs-would generate a large return on investment, avert a substantial proportion of the global burden of morbidity and mortality for diseases of poverty and be cost-effective.

Keywords: Health economics; Health policy; Infections, diseases, disorders, injuries.

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Conflict of interest statement

Competing interests: All authors report grants from the Bill & Melinda Gates Foundation during the conduct of this study.

Figures

Figure 1
Figure 1
Conceptual diagram of modelling approach. The overall model consists of three main components: (1) the DES model that projects the number of product launches and the clinical trial costs, (2) the economic and health benefits model which projects the potential economic and health benefits from product launches and (3) the economic analyses that calculate the BCRs and ICERs of the different design options. BCRs benefit–cost ratio; DES, discrete event simulation; ICERs, incremental cost-effectiveness ratio.

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