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

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

Publication , Journal Article

Zimmerman, A; Diab, MM; Schäferhoff, M; McDade, KK; Yamey, G; Ogbuoji, O

Published in: BMJ global health

May 2023

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.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).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.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.