Drug Costs in Federally Sponsored Cancer Clinical Trials: A Systematic Review
Hong Xiao1, Michael L LeBlanc1, Monica M Bertagnolli2
1Public Health Sciences Division, Fred Hutchinson Cancer Center, Seattle, Washington.
Importance:
Federally sponsored cancer clinical trials (FS-CCTs) address research questions often overlooked by industry (eg, pharmaceutical or biotechnology companies), such as treatment combinations, rare cancers, within-class comparisons, and de-escalation strategies. However, drug costs in these trials have not been comprehensively quantified. Federal agencies typically support trial infrastructure but not drugs, necessitating cosponsorship or in-kind donations from pharmaceutical companies. These arrangements often involve complex contractual processes that may delay trial initiation. Quantifying drug-related costs is important for informing policy and optimizing federal support for cancer research.
Objective:
To estimate the direct purchase costs of drugs in FS-CCTs and examine cost patterns across sponsor types and therapeutic classes.
Design, Setting, And Participants:
This systematic review included a search of the ClinicalTrials.gov registry using a structured query to identify all US-based cancer treatment trials initiated between January 1, 2017, and December 31, 2023, that involved pharmaceutical or biological interventions and received federal sponsorship. Analyses were conducted between June 1 and September 20, 2025.
Exposure:
Drug or biological interventions in FS-CCTs.
Main Outcomes And Measures:
Wholesale acquisition cost-based trial-level total and per-patient drug costs. Between-group comparisons used Mann-Whitney and Kruskal-Wallis tests for 2- and multiple-group comparisons, respectively.
Results:
A total of 1378 US FS-CCTs with 140 066 participants were included in the analysis. The estimated median trial-level drug costs were $7.58 million (IQR, $2.11-$19.88 million), with substantially higher median costs for phase 3 trials ($58.92 million [IQR, $12.25-$127.17 million]) vs early phase trials ($6.90 million [IQR, $2.01-$17.70 million]) (P < .001) and for immunotherapies ($9.25 million [IQR, $3.12-$23.22 million]), targeted therapies ($6.60 million [IQR, $2.22-$18.06 million]), and combined immunotherapy and targeted therapy ($15.08 million [IQR, $6.30-$34.83 million]) compared with chemotherapy ($0.43 million [IQR, $0.11-$2.87 million]) (P < .001). Experimental agents accounted for approximately 80% of total drug costs. The median per-patient drug costs were higher in trials that were solely federally funded ($196 764 [IQR, $83 516-$414 514]) vs cosponsored trials ($149 879 [IQR, $35 329-$390 142]) (P < .001). Among 221 comparative phases 2 to 3 trials, the median experimental arm costs ($12.91 million [IQR, $3.37-$46.96 million]) were 5-fold higher than in control arms ($2.05 million [IQR, $0.16-$11.31 million]).
Conclusions And Relevance:
In this systematic review of FS-CCTs, drug costs were substantial, particularly in late-stage trials and those examining immunotherapies and targeted agents. High costs routinely required federally sponsored groups to seek industry support, which may have limited independence and constrained new treatment discovery. Policy innovations are needed to reduce trial drug cost burden and enable FS-CCT researchers to conduct scientifically and clinically relevant trials.
Insights
Federally sponsored cancer clinical trials (FS-CCTs) incur substantial drug costs, especially for immunotherapies and targeted agents in late-stage studies. These high expenses necessitate industry partnerships, potentially impacting research independence and innovation.
Area of Science:
- Oncology
- Health Economics
- Clinical Trial Management
Background:
- Federally sponsored cancer clinical trials (FS-CCTs) investigate critical research areas not typically addressed by industry sponsors.
- Quantifying drug costs in FS-CCTs is essential for policy development and optimizing federal support for cancer research.
- Federal agencies often do not cover drug costs, requiring cosponsorship or donations, which can complicate trial initiation.
Purpose of the Study:
- To estimate the direct drug purchase costs within FS-CCTs.
- To analyze drug cost patterns based on sponsor type and therapeutic class.
Main Methods:
- Systematic review of US-based cancer treatment trials initiated between 2017 and 2023 from ClinicalTrials.gov.
- Inclusion criteria: pharmaceutical or biological interventions and federal sponsorship.
- Cost estimation based on wholesale acquisition costs; statistical analysis using Mann-Whitney and Kruskal-Wallis tests.
Main Results:
- Median trial-level drug costs were $7.58 million, significantly higher in Phase 3 ($58.92M) vs. early-phase trials ($6.90M).
- Immunotherapies ($9.25M), targeted therapies ($6.60M), and combination therapies ($15.08M) were more costly than chemotherapy ($0.43M).
- Experimental agents comprised ~80% of total drug costs; per-patient costs were higher in solely federally funded trials.
Conclusions:
- Drug costs in FS-CCTs are substantial, particularly for advanced therapies and late-stage trials.
- High drug costs frequently necessitate industry collaboration, potentially limiting trial independence and hindering novel treatment discovery.
- Policy adjustments are needed to alleviate the financial burden of trial drugs and support independent cancer research.
Related Concept Videos
01:16Clinical Trials
There are four phases in a clinical trial. A phase one...
01:11Clinical Trials: Overview
01:21Cancer Survival Analysis
01:12Hazard Ratio
For example, in a clinical trial evaluating a...
02:56Treatment Resistent Cancers
02:49Cancer Therapies
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...