At a recent Pfizer/ Cytel seminar on rare disease and oncology development, Cytel’s Lingyun Liu presented innovative work on a patient enrichment design. In this blog, we share some design and operational considerations. This approach can help mitigate against underpowering of a clinical trial where there is uncertainty and heterogeneity of treatment effect among subpopulations.
Dec 2, 2016 9:19:10 AM
Oct 5, 2015 4:14:58 PM
One consideration every sponsor of a biomarker-stratified confirmatory trial must take into account, is whether to evaluate the biomarker subpopulation (S) against the rest of the population (S') or against the full population (F).
Mathematically, one would think this makes very little difference as F is partitioned into S and S'. If the null hypothesis is rejected for both S and S' then...
Nov 4, 2014 8:30:00 AM
A key stage of exploratory drug development is implementing a proof-of-concept study to demonstrate the safety of a drug. Given the importance of accurate dose-finding for Phase 3 success, methodological improvements to proof-of-concept studies in Phase 2 can translate into greater likelihood of getting a drug to market.
Sep 18, 2014 10:44:00 AM
When designing clinical trials, many trial designers are advised to keep the trial simple. Prima facie, the keep it simple principle seems like sound advice. There are various logistical uncertainties that arise when implementing a clinical trial, and the more simple a trial – so conventional wisdom says – the easier it is to respond to these uncertainties.
According to Zoran Antonijevic, a...
Topics: Oncology, Promising Zone, sample size re-estimation, Enrichment, Cytel Consulting, Efficacy, Interim Analyses, forecasting, optimization, Program and Portfolio Optimization, R&D, Adaptive Clinical Trials
Jul 10, 2014 1:53:00 PM
Complexities with identifying suitable test populations in oncology studies contribute significantly to the 60% attrition rate in Phase III trials. Cyrus Mehta, (President of Cytel) has recently authored a paper on ‘Biomarker Driven Population Enrichment for Adaptive Oncology Trials,’ (forthcoming in Statistics in Medicine) which provides an innovative method for using two-stage adaptive...
May 16, 2014 2:48:00 PM
Cytel Consulting's Zoran Antonijevic
The key focus of precision medicine is identification of patients who would most benefit from a treatment. Proper enrichment of patient population greatly improves the probability of regulatory approval as well as product differentiation through improved efficacy and safety. Greater product differentiation leads to greater market access, as reimbursement is...