Using patients as their own controls for cost evaluation of phase I clinical trials Journal Article


Authors: Sherman, E. J.; Rubin, D. M.; Venkatraman, E.; Schwartz, G. K.; Miller, V. A.; Radzyner, M. H.; Ruchlin, H. S.; Spriggs, D.; Pfister, D. G.
Article Title: Using patients as their own controls for cost evaluation of phase I clinical trials
Abstract: Purpose: Little is known about the cost of phase I trials in cancer patients compared with that of standard treatments, yet the former is often assumed to be greater than the latter. Our objective was to utilize a new approach, using patients as their own controls, to compare in a pilot study the costs of care for patients on phase I trials with those incurred for standard treatment. Patients and Methods: We retrospectively assessed the direct medical costs (DMCs) of 59 patients participating in one of two phase I trials (TRIAL) in solid tumors conducted at Memorial Hospital (MH): (1) perillyl alcohol, and (2) flavopiridol with paclitaxel. Paired-control DMCs were those accrued by the same patient while receiving standard chemotherapy regimens just before (PRE; n = 41) or after (POST; n = 29) the trial at MH, averaged per day. Results: For the 41 PRE patients, the median and mean DMCs per day for the clinical trial versus standard treatment were (US $) $123 v $133 and $219 v $267, respectively. For the 29 POST patients, the median and mean DMCs for the clinical trial versus standard treatment were $157 v $152 and $226 v $226, respectively. Using a linear mixed model, there was no significant difference between TRIAL and standard treatment DMCs (P = .54). Conclusion: Using patients as their own controls represents a new, efficient method for evaluating the cost of phase I trials, and it warrants further study. The results of our pilot study do not suggest that phase I trials always cost payers more than standard treatment. © 2004 by American Society of Clinical Oncology.
Keywords: adult; controlled study; aged; middle aged; retrospective studies; major clinical study; clinical trial; patient selection; solid tumor; united states; paclitaxel; cancer patient; comparative study; chemotherapy; neoplasm; neoplasms; controlled clinical trial; practice guideline; retrospective study; drug cost; health care cost; economics; cancer center; pilot study; pilot projects; cancer care facilities; flavopiridol; phase 1 clinical trial; health care costs; hospital cost; cost benefit analysis; hospital costs; clinical trials, phase i; humans; human; male; female; priority journal; article; perillyl alcohol; minnesota
Journal Title: Journal of Clinical Oncology
Volume: 22
Issue: 7
ISSN: 0732-183X
Publisher: American Society of Clinical Oncology  
Date Published: 2004-04-01
Start Page: 1308
End Page: 1314
Language: English
DOI: 10.1200/jco.2004.06.118
PROVIDER: scopus
PUBMED: 15051779
DOI/URL:
Notes: J. Clin. Oncol. -- Cited By (since 1996):1 -- Export Date: 16 June 2014 -- CODEN: JCOND -- Source: Scopus
Altmetric Score
MSK Authors
  1. Venkatraman Ennapadam Seshan
    285 Seshan
  2. Gary Schwartz
    358 Schwartz
  3. Eric J Sherman
    149 Sherman
  4. Vincent Miller
    262 Miller
  5. David G Pfister
    247 Pfister
  6. David R Spriggs
    312 Spriggs
  7. David M Rubin
    9 Rubin