Cytel Blog

In recent times, Single arm trials are being increasingly used to assess new treatment interventions. They establish clinical benefit by demonstrating the effects of a new therapy or treatment, without the need to use placebo or standard of care as a control. Instead, an alternative approach known as external controls or synthetic control arms (SCA) are being used that leverages real world data and historical datasets. Technical knowledge of Bayesian methods is key to being able to design and implement such trials.

Breakthrough treatments in oncology and rare diseases are now commonly approved based on a pivotal single arm trial – however this is not always optimal. Use of single arm trials in oncology or rare diseases requires appropriate comparisons to be developed to document the benefits of the new treatment. Deriving such comparisons from real world or historical trial data is not straightforward and requires data source and methods expertise.

Measuring treatment effect during a clinical trial is often the source of much debate, particularly during rare disease trials that must stimulate investigations using small samples. Unlike statistically significant results, for which there are many tests, meaningful measures of treatment effect are still under development (Kieser 2012). Cytel statistician Ursula Garczarek wonders whether this holds true in the realm of small samples and small target populations. After all, does the summary statistic in such a small trial rely on many assumptions that might not correlate with reality?

Cytel scientists recently published a new eBook on synthetic control arms and a new scientific primer for the more technically advanced. Our new primer focuses on assessing the validity of data, the validity of methodology, and the modes of analysis and interpretation within this burgeoning field. Each of these is a crucial part of understanding how to make the most impact with a synthetic control. Read our blog to learn more about the eBook and the primer, and register to download both.

Cytel also conducted a webinar on Synthetic and External Controls in Clinical Trials with Dr. Kristian Thorlund, a Professor of Biostatistics at McMaster University and Senior Vice President of Real World Evidence at Cytel. In this webinar, Dr. Thorlund introduces synthetic control arms and discusses the validity of data, results and interpretations. He also answers several questions from the audience around sample size calculations, quality of datasets and use of synthetic controls in various kinds of clinical trials. Click on the button to get free access to the recording and the slides from the webinar.

Cytel has recently published a new ebook on synthetic control arms, and a new scientific primer as well.

The Cytel Trial Design Innovations (CTDI) Webinar Series recently hosted a webinar on designing event-based studies. Such studies are essential to designing high-efficiency clinical trials in certain therapeutic areas, but they add a number of challenges to the already complex landscape of adaptive trials.

The webinar was held on January 23rd, featuring Biostatistician and pioneering Bayesian trial-designer Pantelis Vlachos. We had the opportunity to sit down with Dr. Vlachos and speak about innovative trial designs and their benefits, adaptations and interim looks in oncology and cardiovascular, the challenges of designing event-based studies more generally, and how Cytel’s array of software tools, particularly East^®, has enabled trial sponsors to fully consider their options in the design of high-efficiency clinical trials.

With only two weeks left for this fabulous year to end, we would like to thank all our blog subscribers and new readers for following and appreciating the Cytel blog. This year, we collaborated with several experts from both within and outside the company to bring to you a range of interesting topics including real-world evidence, AI, challenges in rare diseases, patient-reported outcomes, data management, and our popular series “The Good Data Submission Doctor” and “Career Perspectives”. In this blog, we share with you the top 5 Cytel blogs that resonated most with our community in 2019.

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Cytel is delighted to have Kannan Natarajan speaking at the “Complex Innovative Trial Design Symposium and East User Training” on November 6 in Boston, MA. We got a chance to sit down with Kannan and talk about his career in statistics, the changing role of statisticians, his views on evolving statistical thinking, estimands and relevance of technology in the context of rare diseases.

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.

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.