The Cytel blog keeps you up to speed with the latest developments in biostatistics and clinical biometrics.
Single arm trials are emerging as an accepted way of assessing a new treatment intervention. They establish clinical benefit by demonstrating the positive effects of a new therapy or treatment, without the need to use placebo or standard of care as a control. Instead, alternative approaches of establishing the comparison are used; these have become known as external controls or synthetic control arms (SCA) and include approaches leveraging real world data from various sources or evaluations of historical clinical trial data.
Is your Trial a Candidate for a Synthetic Control Arm? Continue reading this blog to learn more.
Making the Most of Your Observational Data: Creating a Synthetic Control from Your Natural History Study
Recently a biotech approached Cytel for support with a Phase 2 Study in oncology. Regulators had requested a natural history of disease study, which tracks disease progression in the absence of any form intervention. These studies are used to build disease-models that can then inform a range of development opportunities within a drug development program.
A March 2019 FDA Guidance highlighted the importance of such studies for rare diseases, with former FDA Director Scott Gottlieb acknowledging that a lack of knowledge about the natural history of certain diseases is a significant obstacle in rare disease drug development.
Head to Head Comparisons Using Real World Data – Design and Data Considerations from Cardiovascular Pilot Investigation
Cytel is conducting two pilot projects on head-to-head comparisons using real world data. These projects in oncology and cardiovascular will occur in real time and will take place across our latest webinar series on the topic. The aim of this series is to introduce our audience to head to head to comparison using Real World Data (RWD) while focusing on practical application and results from the pilot projects.
The second webinar from this series was held on July 28, 2020 and outlines the design of the cardiovascular pilot investigation. None of the existing randomized trials of recently developed second-line antihyperglycemic agents can provide adequate information on their comparative effectiveness and safety regarding cardiovascular outcomes. Conducting Target Trials to get information of interest would be costly, difficult to perform, and would take many years to complete. As a result, we need to use observational databases to emulate it. This blog provides a brief on the design of the cardiovascular pilot project. Our team of experts also discussed the data requirements and the data source to be used in the pilot investigation, with the primary challenge focusing on how to assess if data are sufficient for the purposes of trial emulation. Continue reading the blog for a summary of the webinar.
Get access to the webinar recording and download the slides by clicking the button.
A new peer-reviewed article co-authored by several Cytel scientists re-examines the way in which adaptive trials are designed and implemented within the oncology space. The wide ranging paper spans numerous topics including data considerations, statistical considerations, the commercial and scientific value of flexibility in adaptive designs, and also a “Holistic Approach” to program development, wherein development plans are not created study by study, but across phases.
Cytel is conducting a webinar series that focuses on target trial emulation and causal inference approaches using real world data. In collaboration with Dr. Miguel Hernán, Professor at Harvard University, Cytel is pioneering two “Head-to-Head Comparisons using Real World Data” studies, one in oncology one and in cardiovascular disease. These projects will occur in real time across this webinar series. Our presenters for the first webinar in this series were Dr. Miguel Hernán and Devon Boyne, Director of Epidemiology at Cytel. Continue reading this blog for a summary of the webinar, “Head to Head Comparisons Using Real World Data - The Time for Causal Inference is Now” conducted on July 7, 2020. Click on the button to access the webinar replay.
We also had the opportunity to interview Dr. Hernan on head-to-head comparisons. Read the interview here.
Unlike many therapeutic areas, oncology benefits from having standardized endpoints like overall survival and progression-free survival, as well as standardized methods of measuring such endpoints. Given that the purpose of an Estimands Framework is ostensibly to streamline scientific questions with specific targets of estimation, what is the benefit of an Estimands Framework for oncology?
Overcoming Clinical Development Challenges in Oncology with Innovative, Adaptive Designs: Complimentary Paper
Having its roots in the seminar rooms of the Dana Farber Cancer Institute, Cytel has a long record of establishing new methods in small samples, adaptive designs, Bayesian designs and multi-arm trials, to align statistical rigor to the goal of accelerating clinical development for oncology trials.
Last week, Cytel conducted its third webinar in the new introductory webinar series on Complex Innovative Trial Designs. Our speaker, Dr. Satrajit Roychoudhury is a Senior Director, Statistical Research and Data Science Center at Pfizer. In this webinar, Dr. Roychoudhury gets into the basics of phase I designs in oncology trials, explains the caveats of frequently used traditional designs and provides insights on how implementing a model-based approach can enable a better statistical inference and decision-making. You can watch the replay of the webinar and access the slides by clicking on the button.
We also had the privilege to interview Dr. Satrajit Roychoudhury. Read our blog where he talks about his interest in statistics, explains the concept of Bayesian model-based approaches and their importance in oncology trials.
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.