On the feasibility of pediatric dose-finding trials in small samples with information from a preceding trial in adults.
J Biopharm Stat
; 32(5): 652-670, 2022 09 03.
Article
in En
| MEDLINE
| ID: mdl-34962850
ABSTRACT
We consider the case of pediatric dose-finding trials with extremely limited sample size. The operating characteristics of the standard design, the Continual Reassessment Method (CRM), are only well described for sample sizes of about 20 patients or more. In this simulation study, we assume the situation of a pediatric trial with only 10 patients and a preceding dose-finding trial in adults. Based on the adult data, we reduce the set of pediatric doses and formulate (partially) informative prior distributions for the pediatric trial. Our simulations show that such small pediatric dose-finding trials with robustified priors may provide sufficient operating characteristics.
Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Research Design
Type of study:
Diagnostic_studies
/
Prognostic_studies
Limits:
Child
/
Humans
Language:
En
Journal:
J Biopharm Stat
Journal subject:
FARMACOLOGIA
Year:
2022
Document type:
Article