Perspectives on Enquiry & Evidence

Cytel's blog featuring the latest industry insights.

Featured post

How to Ask the Right Questions at an Authority Meeting

As a drug developer, you have to live with the answers and comments you get from regulatory authorities. Therefore, it’s essential that you choose your questions carefully and avoid questions you don’t need answers to. Here we take a closer look at how to approach these meetings.

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Perspectives on Enquiry and Evidence

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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