The Cytel blog keeps you up to speed with the latest developments in biostatistics and clinical biometrics.

Statistical Approaches to Overcome Challenges in Rare Disease Development

February 28, 2019


In honor of Rare Disease Day 2019 we share a new Cytel podcast featuring Cytel Strategic Consultant Ursula Garczarek discussing how innovative statistical approaches can overcome challenges in rare disease development. Below, you can access the podcast and a summary of some of Ursula's key insights from working in rare diseases and interacting with regulatory agencies for complex and innovative designs.

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Interview: Insight into the Coordination of Rare Diseases at Sanford (CoRDS) registry 

February 28, 2018

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, Senior Research Associate at CoRDS to learn more about the registry and hopes for the future.

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Case studies:Learning from less-well understood adaptive designs

September 29, 2016

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 and operational characteristics, as well as related regulatory interactions.  

To read an abstract and details of the full publication click here. 

 In this blog we'll take a look at some of the case studies under discussion. 

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Adaptive SSR for Small Sample Sizes?

April 21, 2015

“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].

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Bayesian-Bandit Adaptive Designs for Rare Disease Drug Development

October 2, 2014

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 Bandit" is an agent who tries to acquire new knowledge while trying to capitalize on existing knowledge. Clinical studies can therefore utilize bandit designs to recruit patients whose primary goal in participating in a trial is to improve their health outcomes.

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