Enhance your clinical trial design with our strategic consulting services, featuring adaptive trial models and comprehensive regulatory guidance to ensure innovative, compliant, and successful trial outcomes.
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Experience the future of flexible strategic partnerships with Analytics on Demand, providing adaptive and innovative solutions that transcend traditional models to achieve unparalleled operational excellence.
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Maximize efficiency and outcomes with our project-based services, delivering specialized expertise, end-to-end biometrics, and focused solutions for unique projects, ensuring timely completion and exceptional results.
Project-Based Analytical Solutions
Cytel's software platform enables precise trial design and simulation, utilizing adaptive and Bayesian tools to optimize protocols and accelerate drug development with confidence and efficiency.
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Our unique software package streamlines trial implementation with intuitive solutions for protocol development, randomization, and patient management, optimizing operational efficiency and ensuring study success.
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Our innovative preclinical solutions empower your drug development journey with cutting-edge analytics and insights from inception to preclinical stages.
We help you navigate complex trial phases efficiently with data-driven methods & strategies tailored to Phase I-III trials for accelerated drug development.
Maximize your market potential and optimize your commercial strategies with our advanced analytical methods, data science, and tailored commercialization solutions for data-driven commercial success.
Harness the power of real-world data and real-world evidence to gather insights and shape future drug development cycles for enhanced efficacy and regulatory compliance.
Craft optimal trial designs with our advanced analytical methods to enhance efficiency and increase the probability of success throughout your drug’s lifecycle.
Empower your trial delivery by transforming trial designs into actionable strategies with our data-driven approach to ensure seamless delivery and successful outcomes.
Unlock the power of data with our methods for actionable insights to drive informed decisions and optimize clinical trial outcomes.
We offer tailored analytics solutions for your specialized or niche projects enabling you to optimize both efficiency and precision in drug development.
Enhance your clinical trial design with our strategic consulting services, featuring adaptive trial models and comprehensive regulatory guidance to ensure innovative, compliant, and successful trial outcomes.
Experience the future of flexible strategic partnerships with Analytics on Demand, providing adaptive and innovative solutions that transcend traditional models to achieve unparalleled operational excellence.
Maximize efficiency and outcomes with our project-based services, delivering specialized expertise, end-to-end biometrics, and focused solutions for unique projects, ensuring timely completion and exceptional results.
Cytel's software platform enables precise trial design and simulation, utilizing adaptive and Bayesian tools to optimize protocols and accelerate drug development with confidence and efficiency.
Our unique software package streamlines trial implementation with intuitive solutions for protocol development, randomization, and patient management, optimizing operational efficiency and ensuring study success.
Launching a treatment for inflammatory diseases can be a make-or-break endeavor. A robust evidence base and a compelling value story are crucial to success, but current challenges and unexpected bumps in the road will require creative solutions.
Here we describe three case study examples of common issues faced in the inflammatory disease space and how these challenges were overcome using novel solutions, ultimately leading sponsors to submission success.
Evaluating the efficacy and safety of novel therapies in rare indications can be challenging due to the difficulty of recruiting enough patients to conduct a well-powered clinical trial. To address these challenges there has been growing interest in the use of Read more »
To establish treatment efficacy and safety, regulatory and reimbursement decision-makers have traditionally preferred evidence from randomized clinical trials, which, by design, have a low risk of bias. However, single-arm trials (SAT) using an external control arm (ECA) are commonly performed for ethical reasons, due to the difficulties in identifying a suitable comparator arm(s) for head-to-head trials in a rapidly evolving therapeutic landscape and in recruiting patients in the case of rare diseases.
Written by Fei Tang, RWE Senior Research Consultant, and Paul Arora, Assistant Professor (Status), Dalla Lana School of Public Health, University of Toronto
The Food and Drug Administration (FDA) has long been committed to innovative approaches to the regulation of medical device software and other digital health technologies. One such innovation is the use of artificial intelligence (AI) and machine learning (ML) in software, which has the potential to learn from real-world use and experience and improve its performance. The FDA’s vision is that AI/ML-based Software as a Medical Device (SaMD) will deliver safe and effective software functionality that improves the quality of care that patients receive. Read more »
Synthetic control arm (SCA) methods are statistical methods that are seeing rapidly increasing use in comparative effectiveness research. SCA analyses often involve comparing single-arm trials against external control arms constructed from real-world data (RWD) where conducting randomized clinical trials is difficult or infeasible. These benefits are especially evident in rare disease trials where sample sizes are typically substantially smaller, and it is difficult to determine standard-of-care treatments. Rare disease trials conducted with very few patients translates to insufficient statistical power or are performed as single-arm trials that make it difficult to compare against other therapeutic options without SCA methods. Advanced statistical methods are applied to RWD to build the SCA in a way that allows for the appropriate comparison with data gathered during the execution of the single arm trial.
However, each synthetic control project has its own unique challenges with regards to generalizability of results, interpretation and associated statistical methodology. In rare disease indications, the severely limited sample sizes in both single-arm trial and RWD, can present design challenges for SCAs. At ISPOR US 2022, Eric Mackay and Aaron Springford, Research Principal at Cytel, contributed a podium presentation on ‘Power Implications of Estimator Choice in Synthetic Control Arm Analyses’.
The U.S. FDA has recently provided specific guidance[i] on the design and conduct of trials incorporating an external control group, sometimes known as a synthetic control arm. Their guidance represents the culmination of several other topics related to these trial designs and research themes, such as existing guidance on the use real-world data and real-world evidence,[ii] the use of electronic health records and medical claims to support regulatory decision-making,[iii] and guidance on demonstrating substantial evidence of effectiveness for drugs and biological products.[iv]
The evidence is staggering on the unequal health burdens experienced by specific patient groups defined by ethnic, gender, or socioeconomic risk factors and the different health outcomes these groups may have in clinical trials. And while efforts have been made to address these inequalities, they are still falling short. Read more »
Health technology assessment (HTA) submissions require cost effectiveness analyses based on comparative effectiveness studies of survival benefits vs. standard-of-care options in each specific geography. Ideally, these submissions are based on large, randomized control trials (RCTs), however, most new drugs approved in the last five years, specifically in oncology and rare diseases, are being approved based on small clinical trials, often un-controlled or single arm. Often, these trials do not have overall survival as the primary efficacy parameter.
Thus, the dilemma: comparative evidence is still required for HTA submissions, but traditional investigative methods are no longer suitable to support value proposition. So where do we go from here? Read more »
ClinicReal-world data and evidence are increasingly being used in health care decisions and publications. However, there are challenges in identifying suitable data for external control arms, and researchers need to consider solutions to address issues like data quality, bias, and selection bias when using real-world evidence for comparative efficacy analyses.1 Read more »
