The special role of federal sponsorship in the design and conduct of drug or biological agent cancer treatment clinical trials.

94 Background: Industry sponsors are incentivized to conduct trials supporting new drug applications to the FDA, focusing largely on single-agent drug trials. In contrast, federally sponsored trials examine a broader set of research questions. However, the quantitative role of federal sponsorship in drug treatment trials has not been described. These data are crucial for policymakers seeking to prioritize federally sponsored trials in a research landscape dominated by industry. Methods: We analyzed registry data from ClinicalTrials.gov between 2008-2022. U.S.-based trials that included drug or biological agents for the purpose of cancer treatment were included. Studies were categorized by lead sponsor (federal versus industry) and by study phase, intervention, de-escalation, rare cancer, and age. Odds ratios were calculated. Chi-square tests were used. Results: Overall, N=8,561 federal (n=1269, 14.8%) and industry (n=7292, 85.2%) sponsored studies were analyzed. Industry trials were more commonly phase III trials and single-agent drug trials (Table). In contrast, federally sponsored studies were more commonly early stage (Phase I or II) trials (90.5% vs 81.0%, OR=2.25, p<.001) and involved more complex multi-modality drug and biological agent combinations alone (22.7% vs 7.5%, OR=3.63, p<.001) or with radiation (9.5% vs 1.3%, OR=7.75, p<.001), surgery (2.5% vs. 0.2%, OR=15.69, p<.001) or other modality (10.8% vs. 2.2%, OR=5.36, p<.001). Federally sponsored trials were also more commonly dose de-escalation trials and trials conducted in rare cancers and in children. Conclusions: Early-phase, multi-modality, dose-deescalation, rare cancer, and child-focused drug and biological agent cancer clinical trials were more likely to be conducted by federally sponsored research groups. These findings highlight the unique role of federally funded studies in cancer clinical trials. Decreases in federal funding may hinder progress in cancer outcomes. Characteristics of federal- versus industry-sponsored cancer clinical trials. Federal Industry Odds ratio (Fed/Ind) P-value Study Characteristic Category N=1269 % N=7292 % Phase 1 or 2 1149 90.5 5905 81.0 2.25 <.001 3 115 9.1 1319 18.1 0.45 <.001 Intervention Single agent Biological (B) 86 6.8 630 8.6 0.77 .03 Single agent Drug (D) 590 46.5 5842 80.1 0.22 <.001 D & B 288 22.7 545 7.5 3.63 <.001 D/B & Radiation 120 9.5 97 1.3 7.75 <.001 D/B & Surgery 32 2.5 12 0.2 15.69 <.001 D/B, Surgery & Radiation 16 1.3 5 0.1 18.61 <.001 D/B and other modality 1 137 10.8 161 2.2 5.36 <.001 Dose de-escalation Yes 20 1.6 46 0.6 2.52 <.001 Rare cancer 2 Yes 261 20.6 912 12.5 1.81 <.001 Age category Child 3 167 13.2 384 5.3 2.73 <.001 Fed=Federal; Ind=Industry. 1 Including devices or other procedures that are not surgery. 2 Cancers that affect <40,000 U.S. persons/year. 3 Studies that include those <18 years.