Enrichment Strategies in Pediatric Drug Development: An Analysis of Trials Submitted to the US Food and Drug Administration.
Clin Pharmacol Ther
; 104(5): 983-988, 2018 11.
Article
em En
| MEDLINE
| ID: mdl-29218740
ABSTRACT
Clinical trial enrichment involves prospectively incorporating trial design elements that increase the probability of detecting a treatment effect. The use of enrichment strategies in pediatric drug development has not been systematically assessed. We analyzed the use of enrichment strategies in pediatric trials submitted to the US Food and Drug Administration from 2012-2016. In all, 112 efficacy studies associated with 76 drug development programs were assessed and their overall success rates were 78% and 75%, respectively. Eighty-eight trials (76.8%) employed at least one enrichment strategy; of these, 66.3% employed multiple enrichment strategies. The highest trial success rates were achieved when all three enrichment strategies (practical, predictive, and prognostic) were used together within a single trial (87.5%), while the lowest success rate was observed when no enrichment strategy was used (65.4%). The use of enrichment strategies in pediatric trials was found to be associated with trial and program success in our analysis.
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Ensaios Clínicos como Assunto
/
Aprovação de Drogas
/
Seleção de Pacientes
/
Sujeitos da Pesquisa
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Desenvolvimento de Medicamentos
Tipo de estudo:
Clinical_trials
/
Prognostic_studies
Limite:
Humans
País como assunto:
America do norte
Idioma:
En
Ano de publicação:
2018
Tipo de documento:
Article