title: Using patients as their own controls for cost evaluation of phase I clinical trials.
abstract: OBJECTIVE
  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.
  
  
  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 dollars) US dollars 123 v US dollars 133 and US dollars 219 v US dollars 267, respectively. For the 29 POST patients, the median and mean DMCs for the clinical trial versus standard treatment were US dollars 157 v US dollars 152 and US dollars 226 v US dollars 226, respectively. Using a linear mixed model, there was no significant difference between TRIAL and standard treatment DMCs (P =.54).
  
  
  CONCLUSIONS
  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.
authors.affiliation: Department of Medicine, Memorial Sloan-Kettering Cancer Center, New York, NY 10021, USA.
authors.forename: Eric J
authors.initials: EJ
authors.lastname: Sherman
authors.forename: David M
authors.initials: DM
authors.lastname: Rubin
authors.forename: Ennapadam
authors.initials: E
authors.lastname: Venkatraman
authors.forename: Gary K
authors.initials: GK
authors.lastname: Schwartz
authors.forename: Vincent A
authors.initials: VA
authors.lastname: Miller
authors.forename: Mark H
authors.initials: MH
authors.lastname: Radzyner
authors.forename: Hirsch S
authors.initials: HS
authors.lastname: Ruchlin
authors.forename: David
authors.initials: D
authors.lastname: Spriggs
authors.forename: David G
authors.initials: DG
authors.lastname: Pfister
mesh_terms: D000328:Adult; D000368:Aged; D002173:Cancer Care Facilities / Q000191:economics*; D017321:Clinical Trials, Phase I as Topic / Q000191:economics*; D005260:Female; D017048:Health Care Costs; D017721:Hospital Costs; D006801:Humans; D008297:Male; D008875:Middle Aged; D008910:Minnesota; D009369:Neoplasms / Q000191:economics* / Q000628:therapy; D018579:Patient Selection; D010865:Pilot Projects; D012189:Retrospective Studies; D014481:United States