title: The clinical and financial impact of a pediatric surgical neuro-oncology clinical trial.
abstract: Pediatric surgical trials are rare and the impact of such trials on the institutions in which they are conducted is unknown. The purpose of this study was to analyze the clinical and financial impact of The Re-MATCH trial, a Phase I clinical trial requiring the biopsy or resection of recurrent medulloblastoma or PNET for enrollment. Inpatient financial and clinical volume information was collected during the 3 years of trial enrollment and the years preceding and following it. The primary endpoints were the difference in direct contribution margin (DCM), or net gain, of study and non-study patients and the difference in surgical volume during the study and non-study periods. The trial enrolled 18 patients; 15 had surgery at the sponsor institution and three had surgery at their home institution, then transferred tumor material to the sponsor institution. There were no differences between the two groups for potentially confounding variables such as neurosurgical procedure work relative value units (P = 0.13) or insurance provider (P = 0.26). There was no difference between the inpatient DCM per case for the institution for non-study patients (mean ± SD, $9039 ± $28,549) and study patients ($14,332 ± $20,231) (P = 0.4819). During the non-study period, there were a mean of 2.78 ± 1.65 pediatric brain tumor resections per month compared to 3.34 ± 1.66 cases per month during the study period, a 17% increase. When the 15 study patients were excluded, there were 2.97 ± 1.64 cases per month, a 7% increase. However, this increase in total case volume including study and non-study patients was not significant (P = 0.121). Phase I investigator-initiated surgically-based clinical trials may increase institutional surgical volume without imposing a financial burden. Finances are unlikely to be a barrier for researchers negotiating for resources to conduct such trials.
authors.affiliation: Department of Neurosurgery, Duke University Medical Center, Box 3272, Durham, NC, 27710, USA. eric.thompson@duke.edu.
authors.forename: Eric M
authors.initials: EM
authors.lastname: Thompson
authors.affiliation: Department of Neurosurgery, University of Florida, Gainesville, FL, USA.
authors.forename: Sridharan
authors.initials: S
authors.lastname: Gururangan
authors.affiliation: Department of Neurosurgery, Stanford University Medical Center, Palo Alto, CA, USA.
authors.forename: Gerald
authors.initials: G
authors.lastname: Grant
authors.affiliation: Department of Neurosurgery, University of Florida, Gainesville, FL, USA.
authors.forename: Duane
authors.initials: D
authors.lastname: Mitchell
authors.affiliation: Department of Neurosurgery, Duke University Medical Center, Box 3272, Durham, NC, 27710, USA.
authors.forename: John H
authors.initials: JH
authors.lastname: Sampson
mesh_terms: D001932:Brain Neoplasms / Q000191:economics* / Q000601:surgery*; D002648:Child; D017321:Clinical Trials, Phase I as Topic / Q000191:economics*; D006801:Humans; D008527:Medulloblastoma / Q000191:economics* / Q000601:surgery*; D018242:Neuroectodermal Tumors, Primitive / Q000191:economics* / Q000601:surgery*; D019635:Neurosurgical Procedures / Q000191:economics
keywords: Clinical trial; Economic; Financial; Medulloblastoma; Neurosurgery; Pediatric