Perspectives on Enquiry & Evidence
Cytel's blog featuring the latest industry insights.
March 11, 2024
In 2023, rare diseases accounted for 30% of product pipeline under development, about half of which comprising...
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February 26, 2024
Sample Size Re-Estimation for Rare Disease Clinical Trials
Written by Boaz N. Adler, MPA, Director, Global Product Engagement, and Valeria Mazzanti, MPH, Associate Director,...
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February 13, 2024
Strategies to Overcome Limited Patient Population Challenges in Rare Disease Studies
Written by Boaz N. Adler, MPA, Director, Global Product Engagement, and Valeria Mazzanti, MPH, Associate Director,...
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January 17, 2024
Late-Stage Clinical Development Strategy: Trade-Offs and Decision-Making in the Confirmatory Setting
Despite accumulating learnings from early phases, several uncertainties remain to be addressed when designing pivotal...
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January 10, 2024
Optimizing Early Clinical Development Strategy
A clinical development strategy is a comprehensive plan designed to establish the safety and efficacy of new...
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May 16, 2022
Cytel & ARCS Collaborate to Strengthen Early Phase Capabilities
A combination of industry and policy forces have recently changed the shape of Australia’s R&D sector, making it a...
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February 17, 2022
A Data Manager’s Role in Supporting a Rare Disease Clinical Study
It is a common perception that the role of a Data Manager is only to perform what we call “Data Cleaning”; making sure...
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January 20, 2022
Is Borrowing Outcomes from US or China the Future for Rare Conditions?
Limited patient populations resulting in small study sample sizes is a difficulty associated with the development of...
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September 29, 2021
How to leverage model-informed drug development for rare diseases
While there is a plethora of rare diseases, some 7000 diseases and counting, one needs to consider the statutory...
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August 10, 2021
CLRPerm: Cytel Scientists and Collaborators Propose a New Method for Meta-analysis for Rare Events
A few weeks ago I wrote about new research conducted by Cytel statisticians, on the challenge of conducting...
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June 25, 2021
Leveraging External Data for Efficient Trial Designs
The main challenge associated with the development of therapies for rare diseases is typically the small study sample...
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February 12, 2021
Leveraging Synthetic and External Control Arms Using Bayesian Methods
In recent times, Single arm trials are being increasingly used to assess new treatment interventions. They establish...
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November 2, 2020
Value of Detailed Clinical Trial Simulations for Rare Diseases
Measuring treatment effect during a clinical trial is often the source of much debate, particularly during rare disease...
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June 22, 2020
Webinar: Synthetic and External Controls in Clinical Trials
Cytel scientists recently published a new eBook on synthetic control arms and a new scientific primer for the more...
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June 8, 2020
New Primer and Ebook on Synthetic Control Arms
Cytel has recently published a new ebook on synthetic control arms, and a new scientific primer as well.
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January 30, 2020
Designing Event-based Studies: Interview with Pantelis Vlachos
The Cytel Trial Design Innovations (CTDI) Webinar Series recently hosted a webinar on designing event-based studies....
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December 18, 2019
Year-End Roundup: Your Favorite Blog Posts of 2019
With only two weeks left for this fabulous year to end, we would like to thank all our blog subscribers and new readers...
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November 5, 2019
Drug Development in Rare Diseases - Innovation in Statistical Thinking
Cytel is delighted to have Kannan Natarajan speaking at the “Complex Innovative Trial Design Symposium and East User...
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February 28, 2019
Statistical Approaches to Overcome Challenges in Rare Disease Development
In honor of Rare Disease Day 2019 we share a new Cytel podcast featuring Cytel Strategic Consultant Ursula Garczarek...
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February 28, 2018
Insight into the Coordination of Rare Diseases at Sanford registry
There is a consensus in the industry that data on rare diseases is limited, incomplete, and difficult to find or...
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September 29, 2016
Case studies:Learning from less-well understood adaptive designs
A paper "Best practices case studies for 'less well-understood' Adaptive designs", has been published by the DIA...
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April 21, 2015
Adaptive SSR for Small Sample Sizes?
“We shouldn’t use an adaptive design, our sample size is too small.” Most clinical trial planners have heard this line...
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October 2, 2014
Bayesian-Bandit Adaptive Designs for Rare Disease Drug Development
Sofia S. Villar is a member of the DART (Design and Analysis of Randomised Trials) group at the MRC Biostatistics Unit...
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