Seamless Phase IIa/IIb and enhanced dose-finding adaptive design

Jiacheng Yuan*, Herbert Pang, Tiejun TONG, Dong Xi, Wenzhao Guo, Peter Mesenbrink

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

3 Citations (Scopus)

Abstract

In drug development, when the drug class has a relatively well-defined path to regulatory approval and the enrollment is slow with certain patient populations, one may want to consider combining studies of different phases. This article considers combining a proof of concept (POC) study and a dose-finding (DF) study with a control treatment. Conventional DF study designs sometimes are not efficient, or do not have a high probability to find the optimal dose(s) for Phase III trials. This article seeks more efficient DF strategies that allow the economical testing of more doses. Hypothetical examples are simulated to compare the proposed adaptive design vs. the conventional design based on different models of the overall quantitative representation of efficacy, safety, and tolerability. The results show that the proposed adaptive design tests more active doses with higher power and comparable or smaller sample size in a shorter overall study duration for POC and DF, compared with a conventional design.

Original languageEnglish
Pages (from-to)912-923
Number of pages12
JournalJournal of Biopharmaceutical Statistics
Volume26
Issue number5
DOIs
Publication statusPublished - 2 Sep 2016

Scopus Subject Areas

  • Statistics and Probability
  • Pharmacology
  • Pharmacology (medical)

User-Defined Keywords

  • Adaptive design
  • dose finding
  • proof of concept
  • seamless design
  • utility

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