What guides research investments in translational sciences? The case for pharmaceuticals in metastatic cancers

Abstract

Background: Understanding strategies that would optimize the impact of biomedical research is important. Prior research has not distinguished between strategizing investments in basic research versus translational research. Methods: Population-based information on research investments and disease burden in the United States were obtained from publicly available resources such as SEER, National Cancer Statistics Reports and ClinicalTrials.gov. Graphical associations were studied between research investments in translational clinical trials with pharmaceutical drugs for six metastatic cancer sites, approved during 2008 through 2013, and BI-metrics such as 2008 cancer-site specific years-of-life lost (YLL) and historical (2002-2008) and prospective (2008 – 2014) changes in YLL. Associations were explored by study sponsors and by comparative effectiveness trials that include active comparators. Results: Translational research investments were found to be positively associated with anticipated returns based on prospective changes in YLL but negatively associated with the baseline YLL or historical changes in YLL. One exception was non-small cell lung cancer, where the burden was big enough to dominate investments irrespective of returns. Similar patterns in investments were found for both NIH and industry. For trials involving active-comparators or comparative effectiveness trials, positive investment patterns following prospective returns in YLL were found to be more prominent for NIH compared to industry, where incentives for industry are ambiguous. Conclusions and Relevance: Investments in translational research in metastatic cancers, especially in NIH, appeared to follow prospective changes in YLL rather than YLL themselves. This is line with the theories of investment decision-making under uncertainty. Developing and using quantitative measures such as expected value of information metrics can further help guide these decisions and create a more productive dialogue on research investments across stakeholders.