Feb 28, 2018 9:02:00 AM
There is a consensus in the industry that data on rare diseases is limited, incomplete, and difficult to find or access. Recently we came across the CoRDS patient registry based at Sanford University and learned that the registry is an effective tool used to gather information useful to researchers studying rare diseases.
We sat down with Benjamin Forred, Project Manager, and Austin Letcher,...
Topics: Rare Disease, CRO, clinical research, Adaptive Clinical Trials, Clinical Data, real world evidence
Sep 29, 2016 9:28:14 AM
A paper "Best practices case studies for 'less well-understood' Adaptive designs", has been published by the DIA Scientific Working Group on Adaptive Designs as a twin document to the previously discussed "Challenges and Opportunities of 'Less Well Understood' Adaptive Designs". This publication furthers understanding by reviewing 10 important case studies and sharing details on their design...
Topics: Clinical Development Strategy, adaptive designs, adaptive trials, Adaptive Clinical Trials, adaptive sample size re-estimation, Seamless designs, Rare Disease, Multi-Arm Multi-Stage Studies, Multi-Arm Studies, EAST 6.4
Apr 21, 2015 6:09:28 PM
“We shouldn’t use an adaptive design, our sample size is too small.”
Most clinical trial planners have heard this line of reasoning so often it has come to be taken as true. Never mind the fact that the first product to receive FDA approval using an adaptive sample size re-estimation design, was for a genetic condition affecting fewer than two thousand children worldwide [1].
Topics: Clinical Research Services, Rare Disease, Clinical Development Strategy, Adaptive Clinical Trials
Oct 2, 2014 8:30:00 AM
Sofia S. Villar is a member of the DART (Design and Analysis of Randomised Trials) group at the MRC Biostatistics Unit in Cambridge England. She has recently been awarded the first Biometrika post-doctoral research fellowship.
In the post below Villar offers her position on how challenges in rare disease drug development may be alleviated by Bayesian-Bandit adaptive designs. The "Multi-armed...
Topics: Bayesian Methods, Trial Design, Rare Disease