Incremental Treatment Costs in National Cancer Institute–Sponsored Clinical Trials

CONTEXT Concern about additional costs for direct patient care impedes efforts to enroll patients in clinical trials. But generalizable evidence substantiating these concerns is lacking. OBJECTIVE To assess the additional cost of treating cancer patients in the National Cancer Institute (NCI)–sponsored clinical trials in the United States across a range of trial phases, treatment modalities, and patient care settings. DESIGN Retrospective cost study using a multistage, stratified, random sample of patients enrolled in 1 of 35 active phase 3 trials or phase 1 or any phase 2 trials between October 1, 1998, and December 31, 1999. Unadjusted and adjusted costs were compared and related to trial phase, institution type, and vital status. SETTING AND PARTICIPANTS A representative sample of 932 cancer patients enrolled in nonpediatric, NCI-sponsored clinical trials and 696 nonparticipants with a similar stage of disease not enrolled in a research protocol from 83 cancer clinical research institutions across the United States. MAIN OUTCOME MEASURES Direct treatment costs as measured using a combination of medical records, telephone survey, and Medicare claims data. Administrative and other research costs were excluded. RESULTS The incremental costs of direct care in trials were modest. Over approximately a 2.5-year period, adjusted costs were 6.5% higher for trial participants than nonparticipants ($35 418 vs $33 248; P = .11). Cost differences for phase 3 studies were 3.5% (P = .22), lower than for phase 1 or 2 trials (12.8%; P = .20). Trial participants who died had higher costs than nonparticipants who died (17.9%; $39 420 vs $33 432, respectively; P = .15). CONCLUSIONS Treatment costs for nonpediatric clinical trial participants are on average 6.5% higher than what they would be if patients did not enroll. This implies total incremental treatment costs for NCI–sponsored trials of $16 million in 1999. Incremental costs were higher for patients who died and who were in early phase studies although these findings deserve further scrutiny. Overall, the additional treatment costs of an open reimbursement policy for government-sponsored cancer clinical trials appear minimal.