[Non-guideline use of innovative and expensive drugs in pediatrics: assessing clinical practice].
OBJECTIVE
The objective of this study was to analyze in a pediatric hospital the use of expensive drugs as part of the new activity-based system (T2A) of funding for French public hospitals. We identified and analyzed the therapeutic use of these drugs in indications not included in the expert recommendations issued to accompany this change, with the goal of proposing specific pediatric recommendations.


METHODS
Analysis of prescriptions from May through September 2005 showed that 259 patients received expensive drugs subject to special reimbursement. The computerized prescription system enabled us to monitor and validate prescriptions daily. Indications for these expensive drugs were ranked by relevance.


RESULTS
The prescriptions analyzed covered 26 expensive drugs. Among the 344 "patient-drugs", 80% were expensive drugs for an accepted therapeutic use, 5% for a pertinent therapeutic use (under evaluation), and 15% for "off-label" uses (2% "not approved" and 13% for indications not considered by the recommendations).


CONCLUSIONS
This study showed that some therapeutic uses not approved by the official recommendations are nevertheless justified. Gathering data from other pediatric hospitals is essential to determine the need for pediatric clinical trials.
Legrand
Tiphaine
T
Service de Pharmacie, AP-HP, Hôpital Robert Debré, Paris.
Roy
Sandrine
S
Mille
Frédéric
F
Bourdon
Olivier
O
Boulan
Yves
Y
Yakouben
Karima
K
Brion
Françoise
F
D002648:Child; D017277:Drug Approval; D016527:Drug Costs; D004348:Drug Labeling; D017723:Drug Utilization Review; D005602:France; D006779:Hospitals, Public; D006801:Humans; D010372:Pediatrics