The Cytel blog keeps you up to speed with the latest developments in biostatistics and clinical biometrics.

Cytel and Ingress Health at Virtual ISPOR Europe 2020

November 24, 2020

Virtual ISPOR 2020, held November 16 to 19, presented new opportunities for scientific interaction amongst HEOR community. Cytel and Ingress Health, now a Cytel company, contributed to a range of events including interactive workshops, issue panels, on demand podium presentations and virtual poster presentations.

Continue reading for discussions on tracking COVID-19 trials, reflecting on the successes, opportunities and failures of real world solutions, and bridging the gap between real world data and clinical development.

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Data Standards and Submission Highlights from PHUSE EU CONNECT 2020

November 23, 2020

The Virtual PHUSE-EU CONNECT Conference was held from November 8 to 13 and the event was a great success, despite all of us missing the face-to-face contact.

The conference kicked-off on Sunday night with a Social Virtual event with a “Numerologist Show”. Of course this could not replace and compete with the usual “toast” we were used to do live, so we did it virtually (check out my LinkedIn post where I offer some cocktails recommendations and share the recipe of my favorite cocktail “The Negroni” with a bit of history. But please don’t do it before Friday night, you will need the weekend to recover).

Like every year, Cytel significantly contributed to the event as one of the official sponsors, running a workshop (“Predictive Analytics Using R”), chairing and co-chairing two streams (Machine Learning & Connected Health and Scripts and Macros), preparing four on demand presentations (in Application and Development, Coding and Tricks and Data Standards and Governance streams) and two posters.

In this blog, I focus on presentations related to data standards and data submission to agency, in general.

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10 Key Qualifications for Independent Statisticians Reporting to the DMC

November 19, 2020

Data Monitoring Committees (DMCs) are groups of independent experts who periodically receive (by-arm) reports created by an independent Statistical Data Analysis Center (SDAC) using interim data from ongoing studies. The role of the DMC is to make recommendations about the continuation of the studies based on their best judgment and sometimes specified guidelines.

The DMC typically includes at least one statistician who votes on the decision to recommend stopping, modifying, or continuing a study. The SDAC typically is represented by at least one independent statistician and these statisticians are intermediaries between the sponsor and the DMC. The SDAC independent statisticians facilitate the efforts of the DMC by preparing and presenting summary data, taking care of meeting logistics, etc. These SDAC statisticians need ‘hard skills’ such as expertise in biostatistics, experience with clinical trial data, and knowledge of the study protocol. But it is essential that these SDAC statisticians also have the ‘soft skills’ for this role. In this blog, we highlight 10 key qualifications for these SDAC independent statisticians that are less technical, but no less essential.

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We can design over 100,000 clinical trials in less than an hour. How do we choose from them?

November 18, 2020

The current state of the clinical trials industry faces a challenge that was only hypothetical three or four years ago. Thanks to the advent of cloud-computing and advances in simulation technology, sponsors can now design hundreds of thousands of clinical trials in less than an hour. Yet how do we choose amongst all of these myriad options in a way that optimizes commercial prospects? Cytel’s Chief Scientific Officer sits down with us to discuss the Re-imagined Clinical Trial.

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Role of Prediction and Causal Inference in Clinical Research

November 17, 2020

 

As a part of Cytel’s "New Horizons Webinar Series", Alind Gupta, Senior Data Scientist, presents case studies from his research on applying machine learning for predictive analysis and evidence generation.

The biopharmaceutical and healthcare industries now collect more data than ever before due to advances in the variety of information sources combined with the ability to store vast quantities of diverse data. Sophisticated machine learning (ML) and AI techniques allow us to access and analyze any combination of a multitude of data sources. The way that traditional controlled sources are viewed is being adapted in light of new evidence that emerges from real-world data. In his presentation, Alind introduces us to the concept of ML and Causal Inference and discusses case studies from randomized clinical trials and real-world data.

Click the button to register for the on demand webinar.

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Bayesian Methods for Multiple Cohort Expansion (MuCE) designs

November 16, 2020

MUCE is a Bayesian solution for cohort expansion trials where multiple dose(s) and multiple indication(s) are tested in parallel. Such methods are particularly important for areas like oncology where several doses and several indications must be tested for successful completion of early phase trials, and optimal choice of dose and population to move on from early phase to a reasonable dosage for Phase 3.

Note that for these situations the number of comparator arms for a trial can increase rather rapidly. Testing three doses with three indications essentially requires 9 different trials. An efficient way to test a higher number of trials is therefore necessary for accelerated clinical development.

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Join Cytel and Ingress Health at Virtual ISPOR Europe 2020

November 12, 2020

Cytel and Ingress Health (now a Cytel company) will be contributing to a range of events at Virtual ISPOR EUROPE 2020, on November 16th – November 19th. Our Real-World analytics teams will be collaborating to deliver a number of interactive workshops, issue panels, posters and podiums to showcase their work and share innovative insights in HEOR, evidence generation, knowledge synthesis and decision analysis.

Click below to download our full list of sessions at ISPOR EUROPE and feel free to share this brochure with any colleagues who may find our sessions insightful.

download the  sessions schedule

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Interview with Yannis Jemiai: Advanced Design Framework

November 11, 2020

The widespread use of cloud-computing has altered the clinical trial design process. Whereas three or four years ago, it would take a statistician perhaps two or three days to design five clinical trial designs, a well-resourced statistician can now simulate and model well over 100,000 designs in less than 30 minutes. How does this affect the process of designing clinical trials

According to Yannis Jemiai, Chief Scientific Officer at Cytel, a combination of technology and process changes can establish the foundation for significant increases in productivity. Yannis argues that uncertainty should not be viewed as a challenge but an opportunity. Using statisticians strategically as well as tactically throughout the design process can help R&D teams drive commercial value for greater speed, savings and success.

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Key Design Considerations for Platform Trials

November 10, 2020

Platform trials are a new type of clinical trials where multiple interventions can be evaluated simultaneously against a common control group within a single master protocol. Platform trial designs are an extension of adaptive trial designs that are sometimes referred to as a multi-arm, multi-stage (MAMS) design, as multiple interventions (‘‘multi-arm’’) undergoing multiple interim evaluations (‘‘multi-stage’’) are part of the design features.

This autumn Cytel has been holding a number of webinars on Platform Trials, ranging from a discussion with Cyrus Mehta on statistical innovations to incentivize more sponsors to consider platform trials, to next week's event with Jason Connor (Confluence) on the use of Bayesian methods for these innovative trial designs. 

In a recent webinar Jay Park, Director of Trials Research for Cytel in Canada, presented a webinar to review the concept of platform trials and discuss important design considerations for platform trials. Jay is the author of several leading papers on Platform Trials, including one in CA: A Cancer Journal for Clinicians, the journal with the world's highest impact factor. He has also produced a complimentary primer on the subject which you can download here.

Continue reading this blog to get a summary of his talk. Click the button to access the on demand webinar.

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COVID-19 Trial Tracker Updates (November 9)

November 9, 2020

As the evolving COVID-19 pandemic continues, the Cytel COVID-19 Trial Tracker continues to bring you an up to the minute, real time dashboard about COVID-19 trials around the world. In this blog, we bring you the updates from this week.

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Role of RWA in Transforming Oncological Research

November 5, 2020

In oncology, many manufacturers go into niche indications, where there are very specific tumors, and then they opt for a single arm trial. This potentially works for regulatory purposes (EMA/FDA). However, if they go to the local HTA authorities (NICE/CADTH), they will have to answer the question on the relative effectiveness of the new product compared to the standard of care in that country, and then its cost-effectiveness. Hence, typically, a manufacturer will identify a publication on a trial or real-world evidence on standard of care, or perhaps collect individual patient level data in clinical practice. Subsequently, it will conduct comparative effectiveness analyses for HTA purposes, a naïve comparison or an unanchored MAIC/STC/PSM of the single arm trial compared with the control arm.

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Cytel Introduces Advanced Design Framework: Part 3 - Communication Techniques to Ensure Alignment on Data-Driven Clinical Trial Designs

November 4, 2020

Cytel has recently revealed its Advanced Design Framework, a method developed by Cytel’s thought leaders that draws on decades of experience increasing clinical development productivity. The Framework illustrates how advances in design processes and technology can help development teams deliver greater business results, unifying statistics and strategy in the era of cloud computing and making strategic use of well-resourced statisticians.

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Staffing Needs for RWE Delivery

November 3, 2020

When an expert statistician is paired with an experienced set of data managers, opportunities to capitalize on quantitative strategy are spotted more quickly. Statisticians can determine whether datasets can strengthen study findings by being presented in a way that uses the available data in a scientifically objective way that is at the same time in line with the clients’ strategic objectives.

The practice of combining statistical needs with the processes of data management and other related services for real world evidence, we will henceforth call RWE-Delivery. There are several models for RWE-delivery that can similarly vary with the needs of a study. Questions about process, management and timelines are just as key for this choice of delivery model, as the objectives of the delivery. Therefore, it is important to work closely with delivery teams to determine the possible needs for study completion.

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Value of Detailed Clinical Trial Simulations for Rare Diseases

November 2, 2020

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?

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Cytel Introduces Advanced Design Framework: Part 2- The Need for A Quantitative Evaluation Approach for Deciding Together

October 29, 2020

Cytel has recently revealed its Advanced Design Framework, a method developed by Cytel’s thought leaders that draws on decades of experience increasing clinical development productivity. The Framework illustrates how advances in design processes and technology can help development teams deliver greater business results, unifying statistics and strategy in the era of cloud computing, and making strategic use of well-resourced statisticians.

The framework consists of three parts: Thoroughly Explore, Decide Together, and Communicate Trade-Offs. This week we take a deeper look into the second part of this Framework, revealing how to effectively incorporate varied perspectives to efficiently design innovative clinical trials. Opportunities for quantitative evaluation criteria and design without bias help R&D teams sift through the thousands of trial designs options to optimize for speed, success, and savings.

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The Missing Link: Risking your Traceability (and “Credibility”) when your ADaM package is not traceable back to SDTM

October 28, 2020

About three years ago, Cytel was helping a sponsor on a project where I had to conduct surveillance of some CRO deliverables, mainly for SDTM and ADaM packages. At first, I was involved in the review cycle of SDTM, and began by reviewing some initial mapping specifications including a draft SDTM Annotated CRF. The CRO in charge was quite experienced and there was nothing major to spot in all the different versions I had to review.

Surprisingly, it was not the case some months later, when I had to provide the same review support for the Biostatistics deliverables, specific to the ADaM package. The ADaM datasets overall were well designed, and there were no major open non-conformance issues. However, it was clear from the very beginning that there was something missing - a missing link between SDTM and ADaM.

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Bayesian Dose-Finding Designs – An Overview

October 27, 2020

 

Cytel recently conducted a webinar on Bayesian Dose-finding Designs for Modern Drug Development, presented by Dr. Yuan Ji.

Dr. Ji is a Professor of Biostatistics at The University of Chicago and a well-known name in the industry. In his presentation, he introduces representative Bayesian designs for dose-finding trials. The webinar offers insights on topics including classical DLT-based dose-finding designs, designs with delayed toxicity using time-to-event endpoints, and designs for combination dose-finding trial. Watch the on demand webinar to see the illustration of Bayesian modeling and inference for dose-finding designs that utilize the concept of probability intervals and related methods for clinical development and decision making.
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Need for Technology Solutions to Support Computationally Intensive Bayesian Designs

October 26, 2020

Pharmaceutical and biotech companies are under pressure to deliver more and deliver faster with fewer resources. The cost of drug development, failure rate and human cost associated with prolonged participation in a trial turn out to be steep in case of an ineffective trial. As the industry seeks new levels of clinical trial efficiency and probability of success, more companies are looking to use advanced, innovative and computationally intensive designs like Bayesian methods.

Bayesian methods are of growing interest to the drug development industry, as they allow clinical investigators to leverage historical trial data as well as learnings from new data as it accrues throughout a trial. The result is better-informed decision making, greater program flexibility, and the ability to run smaller, more resource-efficient trials.

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Yuan Ji on U-Design: An All New Efficacy and Toxicity Dose-Finding Module

October 23, 2020

Cytel’s New Horizons Webinar Series introduces you to the latest innovations in statistical trial design. This webinar from the series is presented by Dr. Yuan Ji, a consultant for Cytel. Yuan is the founder of Laiya Consulting and currently is the Professor of Biostatistics at The University of Chicago. In his presentation, Professor Ji introduces the U-Design version 1.4, which mainly consists of a new module of dose-finding trial designs with joint efficacy and toxicity outcomes.

Click the button to register for the next webinar in this series, presented by Cytel's Ursula Garczarek. Ursula will be presenting a case study on the value of detailed clinical trial simulations for rare diseases.

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An Interview with Bart Heeg (Part 2): New Trends in HEOR

October 22, 2020

In this two-part blog series, we interview Bart Heeg, Vice President HEOR and Founder at Ingress Health (A Cytel company). Bart provides us insights on the trends in HEOR and explains why Bayesian methods are also important for Health Economics. Read Part 1 here.

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Cytel Introduces Advanced Design Framework: Part 1 - Methods for Thorough Exploration of Design Space

October 21, 2020

Cytel has recently revealed its Advanced Design Framework, a method developed by Cytel’s thought-leaders after a decade of fine-tuning clinical development processes. The framework consists of three parts: Thoroughly Explore, Decide Together, and Communicate Trade-Offs.

The Framework demonstrates how to unify statistics and strategy in the era of cloud-computing, by making strategic use of well-resourced statisticians. This week, we take a deeper look into the first part of this Framework, revealing how to explore hundreds of thousands of designs available to sponsors, rapidly and in real-time, to improve the chances of identifying the design that optimizes for speed, success, and savings.

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Interview with Thomas Wilke: Health Economics and Real-World Evidence Studies

October 20, 2020

In this interview with Thomas Wilke, Principal Scientist at Ingress-Health (a Cytel company), we talk to him about his background and experience in Health Economics, understand the important considerations of real-world evidence studies and the impact of COVID-19 pandemic on the work of the health economics outcomes researchers who work at Ingress and Cytel. We also cover important HEOR topics such as its benefits for market access studies and real-world analytics (RWA) for regulatory submission.

Cytel and Ingress-Health will be contributing to a range of events at Virtual ISPOR EU 2020, on November 16th – November 19th. Our Real-World analytics teams will be collaborating to deliver a number of interactive workshops, issue panels, posters and podiums to showcase their work and share innovative insights in HEOR, evidence generation, knowledge synthesis and decision analysis.

Click below to download our full list of sessions at ISPOR EU

download the  sessions schedule

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Upcoming Discussions: The Uniqueness of COVID-19 Real-World Data Challenges & The COVID-19 trial tracker

October 19, 2020

COVID-19 has created extreme uncertainties -- a dearth of historical information combined with the need for safety, statistical rigor, and speed has prompted the rapid surge in the generation of clinical data. However, this information is scattered across multiple platforms, making it challenging to measure comparative treatment effects across trials. Consequently, we are seeing a high frequency of failures, that diminishes the public’s confidence in research and slows the path from scientific results to action.

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An Advanced Design Framework for Clinical Development in the Era of Cloud-Computing

October 15, 2020

For over a decade, advanced trial design techniques have promised efficient trials with accelerated timelines, reflecting the ability to quantify uncertainty and de-risk trials using adaptive tools. Despite the emergence of these complex innovative designs, the success of Phase 3 trials has continued to hover at 33% while the average time to market remains about 6 years.

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An Interview with Bart Heeg (Part 1):The Increasing Importance of Health Economics

October 14, 2020

A credible evidence base is needed to support and document the economic value of new technologies and therapeutic approaches. Companies need careful cost-effectiveness analyses for successful reimbursement submissions. In this two-part blog series, we interview Bart Heeg, Vice President HEOR and Founder at Ingress Health (A Cytel company). Bart talks about his background in HEOR, founding Ingress Health and its recent acquisition by Cytel. He also talks about the benefits of turning to an HEOR specialist and provides a sneak peek into Cytel’s presentations at the upcoming ISPOR EU 2020 event.

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Introducing Observational Studies – Three Trends for Statisticians

October 13, 2020

The combination of greater access to electronic health records, bigger electronic claims datasets, and the need for more clinical insight in ensuring patient safety, has made observational studies an important new tool in trial design. Observational studies typically take non-randomized data from outside of a trial and use quantitative and modeling techniques to draw conclusions from big datasets. While typically used for HEOR and market access, augmenting regulatory submissions with observational studies is gaining prominence. As with all data analyses, there is an implicit rule of ‘garbage in-garbage out,’ where data that is not up to the standard required for the formation of sound scientific judgment, should not be used. Sponsors should rely on the most sophisticated tools and advanced analytics to make the most rigorous use of available data.

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Bayesian Statistics and FDA Regulatory Acceptability

October 12, 2020

 

Cytel and Novartis are together hosting a complimentary Bayesian Virtual Symposium and an Interactive 7-part workshop. This series will expose you to cutting edge topics from industry renowned leaders in Bayesian statistics. The introductory webinar “Bayesian Statistics and FDA Regulatory Acceptability” is presented by Greg Campbell, PhD, Former Director of Biostatistics, U.S. Food and Drug Administration.

In the United States Bayesian statistics has been used in regulatory submissions to the Food and Drug Administration (FDA) for confirmatory clinical trials medical devices for more than fifteen years. In this webinar, Dr. Campbell reviews the Bayesian history and accomplishments for medical devices. He talks about the status and opportunities of Bayesian statistics for pharmaceutical drugs and biologicals. We also learn about the challenges and the future of Bayesian statistics in the regulatory environment. You can access the on demand webinar and register for the rest of the series by clicking the button.

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Cyrus Mehta on Increasing the Power of Platform Trials

October 9, 2020

Even before the era of COVID-19, significant attention was channeled to the overwhelming potential of adaptive MAMS designs. Short for multi-arm multi-stage designs, these trials enable numerous therapies to be tested on a single platform with a single comparator arm. When patients are too few or there are several therapies in competition with each other to enroll, adaptive MAMS designs expedite the discovery of new drugs.

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RWE Needs for Natural History Studies

October 7, 2020

With the rise in digital technologies, there has been an explosion in the volume and type of data sources. We can obtain information about individual health from social media data and mobile apps, to wearable sensors and electronic health records. Corporations and governments even use insurance claims data as sources of data for analyses.

This data could yield a more robust and complete picture of diseases, the patient journey, and the effectiveness of interventions in the real world. This in turn is often used by life sciences leaders to make better drug development, reimbursement, and clinical decisions. However, apart from accessing and curating this data, we also need to harness advanced analytical techniques to generate evidence, including the sophisticated use of statistical methods. The RWE data sciences team therefore must be chosen carefully to take on the challenges of these novel uses of data.

The delivery of RWE-analyses requires more than simply statistical knowledge. The variety of RWE methodologies reflect the range of opportunities sponsors have, to cast their assets in the best light. In this blog we outline the RWE design and staffing needs of a specific kind of observational study, namely natural history studies, as regulators are increasing demand for these explanatory assessments of the biochemistry of disease progression.

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The Meta-Analytic-Predictive Priors Generation and Comparisons

October 5, 2020

Staying abreast of the rapid pace of clinical development means adopting innovative or computationally intensive designs like Bayesian methods. These methods allow for the incorporation of prior knowledge, in terms of either expert opinion from clinicians or historical data, in statistical inference. Thus, they have the additional advantage of being able to work with real-world data (generally, real-world data has a lot of missing data) without the need to impute missing values. These kinds of models are also flexible enough to work with temporal data. This helps ease the reliance on large sample approximations that are often required for frequentist methods and generally results in greater efficiency in study design.

In this edition of The Informative Bayesian by Pantelis Vlachos, we learn about information borrowing to form a prior distribution. In a Bayesian framework, borrowing from historical data is equivalent to considering informative priors. These priors can be derived as meta-analytic predictive (MAP) priors or using patient-level data.

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Improve Trial Design with Group Sequential Designs and Sample Size Re-estimation

October 1, 2020

Methods involving Group Sequential Designs is one of the earliest deviations from a traditional two-arm clinical trial with no interim looks at the data. They add incredible value to trials through their abilities to safeguard patients, reach positive conclusions early and keep trial designs simple and streamlined.

Sequential trials also help reduce costs and the number of patients involved, but finding a positive conclusion earlier is quite important too. In the drug development process, where patent lifetime is limited, reaching a decision six months or a year earlier is a big advantage. Sample Size Re-estimation is another key tool in the modern trial designer’s toolkit that proves to be useful. Continue reading this blog to learn how to use these methods and understand how they can improve trial design.

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Innovations in Bayesian Trials: A Virtual Event Brought to you by Cytel and Novartis

September 30, 2020

 

Today, there is a need for advanced quantitative techniques to combine all available information for better decision making in health care. Bayesian statistics allow us to make probabilistic inferences on the parameter of interest, which is missing in a traditional frequentist approach. Apart from the philosophical issues, Bayesian analysis provides a practical and intuitive tool for interpretation of study results and risk evaluation of clinical hypotheses.

Cytel and Novartis are together hosting a complimentary Bayesian Virtual Symposium and an Interactive 7-part workshop. This series will expose you to cutting edge topics from industry renowned leaders in Bayesian statistics. Click the button to learn more and register.

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The New Horizons Series: Adaptive Multi-arm Multi-stage Clinical Trials

September 29, 2020

Innovation in trial designs are offering new routes forward for organizations of any size. They are now also aligned with the overarching goals of improved clinical development, better pre-planning, greater patient safety, less medical waste, and/or increased knowledge.

Cytel is hosting "New Horizons Webinar Series" that will introduce biostatisticians to the latest innovations in statistical trial design. The first webinar in the series on Adaptive Multi-arm Multi-stage Clinical Trials is going to be presented by Cytel's President & Co-Founder, Cyrus Mehta. Click on the button to register.

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TOGETHER trials, and the advantages of adaptive platform designs for investigating COVID-19 therapies

September 28, 2020

Cytel has recently designed and implemented the TOGETHER Trials, funded by the Bill & Melinda Gates Foundation to generate knowledge to help fight COVID-19, particularly in low and middle-income countries. The trials, with sites in Brazil and South Africa, test three existing interventions as possible treatments for COVID-19 in high-risk adults who do not require hospitalization, compared to a placebo.

The TOGETHER trials use an adaptive platform design. This type of design is particularly useful for contexts such as COVID-19 response, where there are many unknowns and a need for accelerated and resource-efficient answers, for 5 reasons.

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Use of External Controls in Clinical Development – Download Audiobook

September 25, 2020

Regulators in both the United States and Europe have responded positively to the use of synthetic control arms (SCA)s in clinical development. The desire to speed up and lower the cost of drug development, coupled with increased availability of rich real-world data, contributed to the increased willingness towards using SCAs as supplementary evidence to accompany regulatory submissions using single arm trial data only.

As with any sophisticated statistical method, deciding on the optimal SCA approach is a necessary condition to ensure robustness of findings. Cytel’s new audiobook "Demystifying synthetic control arms", explains the concept of synthetic controls and offers insights on some common quantitative strategies for trial design and regulatory submission. Continue reading this blog to learn more and get access to the audiobook.

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LIBERTY claim analysis manuscript by Thomas Wilke and Sabrina Mueller

September 22, 2020

Research Scientists, Thomas Wilke and Sabrina Mueller recently published a manuscript on “Diabetes-Related Effectiveness and Cost of Liraglutide or Insulin in German Patients with Type 2 Diabetes: A 5-Year Retrospective Claims Analysis”.

As insulin and liraglutide are both treatment options for type 2 diabetes mellitus (T2DM), it was important to understand their long-term real-world outcomes. A retrospective study was conducted using administrative claims from a German health fund (AOK PLUS) and clinical data; the clinical data was collected in a disease management program. This claims data analysis, in adult patients with T2DM, investigated diabetes-related effectiveness and costs with long-term (up to five years) treatment with liraglutide or any insulin, in a real-world setting.

Continue reading this blog to learn about the unique insights that were gained during this project and get access to the publication.

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The TOGETHER Trial: Cytel Designs and Implements Novel Adaptive Platform Trial for COVID-19 Therapies

September 21, 2020

Cytel has designed and implemented a novel adaptive platform trial for early stage COVID-19. The severity of the coronavirus emerges in five stages, with the majority of clinical trials focusing on therapies for the final stage of the disease. According to the Cytel Clinical Trial Tracker, only 6 of 2000 trials are focused on early stages; staggering given that only 5% of coronavirus cases are considered severe.

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East Alloy: Accelerating the pace of innovation

September 17, 2020

Keeping up with the rapid pace of clinical development means that we need to adopt the innovative or computationally intensive designs like Bayesian methods. Yet, cutting edge technology can sometimes be difficult to assess or can introduce risk. Cytel’s new web-native extension of East, East AlloyTM, makes it practical and sustainable to adopt innovative and computationally intensive designs. Continue reading this blog to learn more.

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New Audiobook on Synthetic Control Arms

September 16, 2020

Synthetic control arms (SCA) are virtual trial arms that use historical claims data and observational data to simulate the control arm of a study. When enrollment targets are low and large amounts of data already exist about the performance of a control, then in many cases using quantitative techniques to simulate a control arm of a trial will expedite timelines and serve as a more optimal use of resources.

Cytel’s new audiobook "Demystifying synthetic control arms", provides insights on synthetic controls, suitable conditions for their use, and some common quantitative strategies for trial design and regulatory submission. Click the button download the audiobook.

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Accurately analyze small, skewed or sparse data with StatXact

September 15, 2020

In clinical trials with small or sparse data, statistical methods meant for large sample sizes may not be helpful to get an accurate interpretation of data. This is where computationally challenging exact methods often come into play. Chris Corcoran, David B. Haight Professor of Analytics in the Huntsman School of Business, is presenting at a Cytel webinar where he will introduce some basic exact statistical procedures provided in Cytel’s StatXact®. The software offers more than 160 tests and procedures for exact inference and power analysis.

Join Chris Corcoran in this example-based discussion where he will illustrate why exact analysis can be crucial in providing accurate results in some very common settings, particularly those involving small or sparse samples. Click on the button to register for the webinar.

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From Before to After: Preparing and Concluding your FDA Data Submission

September 14, 2020

“A good start is half the battle” (the Before) when submitting data to the FDA and there are a couple of cherries to put on top (the After) when your regulatory group has finally submitted the eCTD to the FDA[1] . A good start is to have early discussions with the agency by regularly meeting them and sharing the status of your clinical data standards. While, the cherry on the top is the continuous support you need to guarantee to your submission project to promptly react when the reviewers come back with questions and additional requests during the review process.

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Does your Trial need a Synthetic Control Arm?

September 10, 2020

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.

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Importance of Designing Clinical Trials from a Program Perspective

September 9, 2020

Cytel’s co-founder, Nitin Patel, conducted a webinar on designing clinical trials from a program-level perspective. His presentation helps us understand the value of designing clinical trials considering downstream consequences. Watch the on demand webinar to get insights on the role of simulation in optimizing clinical trials' performance from a program perspective and understanding the hybrid Bayesian-frequentist approach to clinical trial design.

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We also had the opportunity to interview Nitin about his journey since he co-founded Cytel and got his views on implementing a program-wide strategy for pharma and biotech companies. Read the blog here.

Continue reading this blog for key highlights from the webinar.

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Cytel Co-Founder Cyrus Mehta Presents at the Heart Failure Collaboratory, a Public-Private Partnership with FDA

September 4, 2020

On Friday September 11, Cyrus Mehta, co-founder of Cytel, will be delivering a talk to the Heart Failure Collaboratory, on how adaptive designs can be utilized to salvage trials disrupted by COVID-19. The Heart Failure Collaboratory is a public-private consortium with FDA, and will be hosting a day long symposium online, on the application of innovative methods for drug and device studies in the age of COVID-19.


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FSP Navigator – Building the Team that Brings Success

September 1, 2020

In this blog, Cytel's SVP Corey Dunham’s talks about our Functional Services teams and the qualities we seek in new hires. As our FSP division continues to grow, we are looking to hire programmers and biostatisticians. Cytel's recruitment and FSP teams are hosting a one hour long Virtual Careers Open Day on Thursday, September 10th at 11:00AM EDT. Join us to learn more about our expanding FSP team, open career opportunities and what it means to be part of Cytel. Click on the button to register for the virtual event.

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Adopt innovative and computationally intensive designs with East Alloy

August 31, 2020

Pantelis Vlachos, Principal, Strategic Consultant at Cytel, conducted a webinar to introduce the capabilities of East AlloyTM. East Alloy is a new East environment that enables rapid access to innovation with the trust and support you have come to expect from Cytel. The cloud-native software makes it practical to apply computationally intensive Bayesian methods. Download the brochure to learn more.

Download Brochure

This blog is a part of the new blog series on technology and Bayesian decision-making by Pantelis. Continue reading to learn about the methods and capabilities, such as, Bayesian meta-analytic priors, Bayesian MAMS, adaptive dose-finding and others, available to all East Alloy users. 

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Career Perspectives: Interview with Yannis Jemiai, Chief Scientific Officer

August 26, 2020

As Chief Scientific Officer, Dr. Yannis Jemiai plays a pivotal role in maintaining Cytel’s well-established reputation for statistical excellence and our track-record of bringing innovative analytic approaches to the development of medicines for human health. With oversight for the corporate-level Scientific Agenda, Yannis ensures Cytel continues to be known for thought leadership in adaptive designs while expanding its reputation in Bayesian statistics, Complex innovative designs, and the use of Real-World analytics for regulatory and post-approval purposes. Yannis’ research interests include adaptive trial design, causal inference, decision theory, and regulatory affairs.

In this blog we talk to Yannis about his long-standing association with Cytel, his views on adaptive designs and their future; and we get some tips from him for young statisticians, programmers and those interested in pursuing a career in this field.

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Nitin Patel on Designing Clinical Trials from a Program Perspective

August 25, 2020

It is important to take a strategic approach to clinical development in order to minimize the potential for Phase 3 attrition. In our next webinar from the ‘Introduction to Complex Innovative Trial Design’ webinar series, Nitin Patel, co-founder of Cytel, will provide an overview of the concepts of program optimization. As a case study, we will describe a Phase 2 trial design based on program-level optimization. We utilize a hybrid Bayesian-frequentist framework to evaluate the impact of Phase 2 design choices on the probability of Phase 3 success, clinical utility, time to market, trial costs and expected net present value (ENPV) of the product. These factors include Phase 2 sample size, dose selection and go-no go decision rules for Phase 3, and Phase 3 sample size. 

Click the button to register for the webinar on August 26, 2020.

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In this blog, we talk to Nitin about his journey since co-founding Cytel; and gain insights on program optimization, the challenges for both big and small pharmaceutical/biotech companies, and how to go about the planning process.

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Bayesian Borrowing and Real World Data: The Fundamentals

August 21, 2020

As uses of real world data become more familiar for trial design and regulatory submission, sponsors might become more interested in techniques related to Bayesian borrowing. Sophisticated uses of this technique have been applied to COVID-19 vaccines development and complex oncology trials. Simpler versions can be used to optimize across existing datasets, design trials with lower enrollment targets, accelerate the time to regulatory submission, and strengthen the power of a trial. Here we provide an overview of the potential benefits.

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Webinar on Adaptive Designs for Dose Finding: Part 2

August 19, 2020

Bjoern Bornkamp, Statistical Methodologist at Novartis and Jose Pinheiro, Senior Director, Johnson & Johnson provided their insights on adaptive designs for dose finding in Cytel’s latest webinar. The webinar demonstrates how adaptive and Bayesian techniques can be implemented for optimal dose-finding.

This two-part blog series provides a summary of the webinar. Read the first part to get key highlights from the presentation by Jose Pinheiro on the need to conduct dose finding Phase 2 studies, dose selection comparisons and the use of MCP-Mod for dose finding. 

Continue reading this second part to learn about the methods of adaptive dose-finding, presented by Bjoern. Click the button to access the webinar recording and download the presentation slides

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Career Perspectives: Interview with Mrudula Joshi, Associate Director, Statistical Programming Services

August 18, 2020

Mrudula Joshi joined Cytel in July 2005 as a young SAS programmer. Last month, she celebrated her 15th year work anniversary at the company. In this blog we talk to Mrudula about her journey so far, her current role, and achievements; and we get some tips from her for young statisticians, programmers and those interested in pursuing a career in this field.

Cytel’s Biostatistics and Statistical Programming team for Functional Services provides integrated solutions, by blending the expertise of programming and the experience of statistics. As our FSP division continues to grow, we are looking to hire programmers and biostatisticians. Cytel's recruitment and FSP teams are hosting a one hour long Virtual Careers Open Day on Thursday, September 10th at 11:00 AM EDT. Join us to learn more about our expanding FSP team, open career opportunities and what it means to be part of Cytel. Click on the button to register for the virtual event.

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Webinar: Adaptive Designs for Dose Finding

August 13, 2020

Bjoern Bornkamp, Statistical Methodologist at Novartis and Jose Pinheiro, Senior Director, Johnson & Johnson provided their insights on adaptive designs for dose finding in Cytel’s latest webinar. Finding the right dose in Phase 2 gives a potential new therapy its best chance to demonstrate efficacy during Phase 3. A well-executed dose-ranging trial therefore has the potential to alter the course of the entire clinical development program. This webinar demonstrates how adaptive and Bayesian techniques can be implemented for optimal dose-finding.

This two-part blog series will provide a summary of the webinar. In this first part, get key highlights from the presentation by Jose Pinheiro on the need to conduct dose finding Phase 2 studies, dose selection comparisons and the use of MCP-Mod for dose finding. Click the button to access the webinar recording and download the presentation slides

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Virtual Careers Open Day at Cytel

August 12, 2020

Cytel’s Biostatistics and Statistical Programming team provides integrated solutions, by blending the expertise of programming and experience of statistics. Being a notable Functional Services Provider (FSP) in the industry, we take pride in supporting our clients as they navigate the intricacies of 21st century drug development. As our FSP division continues to grow, we are looking to hire programmers and biostatisticians. Cytel's recruitment and FSP teams are hosting a one hour long Virtual Careers Open Day on Thursday, September 10th at 11:00AM EDT. Join us to learn more about our expanding FSP team, open career opportunities and what it means to be part of Cytel. Click on the button to register for the virtual event.

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Making the Most of Your Data II: Optimizing Clinical Information in Trial Design and Implementation Using Bayesian Methods

August 11, 2020

While there is increasing optimism about the discovery of a COVID-19 vaccine, one of the less talked about aspects of such vaccines development are the lessons that can be used in other therapeutic areas. After all, COVID-19 vaccines development has uncovered numerous ways to design and execute trials within shorter time-frames and with less data.

One theme that has emerged consistently is the need to optimize the use of clinical information available, an endeavor well-supported by Bayesian methods.

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Making the Most of Your Observational Data: Creating a Synthetic Control from Your Natural History Study

August 7, 2020

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.

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Head to Head Comparisons Using Real World Data – Design and Data Considerations from Cardiovascular Pilot Investigation

August 5, 2020

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.

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Estimands and their Implications on Clinical Studies

August 4, 2020

Last year, Paul Terrill, Associate Principal of Strategic Consulting at Cytel, presented an engaging webinar on the topic of Estimands. The webinar covered a range of issues from what is an estimand to how to structure early discussions on estimands. On popular demand, Paul will re-run this webinar on August 13, 2020, and add recent developments on this topic. He will share the bottom-line on estimands and discuss their implications for a trial's objectives, design, data collection, statistical analyses and conclusions. Paul will also share his guidance on managing the communication about estimands between multiple internal stakeholders, gaining internal buy-in, and ensuring that a trial’s objectives, design, conduct, analysis and interpretation are in line with the addendum. Register today by clicking on the button.

registerContinue reading this blog to understand the concept of Estimands, their role in clinical trials and the purpose of the final ICH E9(R1) addendum.

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Cytel Scientists Call for a “Statistician-First Workflow” to Optimize Drug Development

July 30, 2020

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.

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Therapeutic Area User Guidance – The hidden Gems

July 28, 2020

CDISC standards have been around for a while with the first SDTM Standard version released in 2004. However, it was only in the last decade that it became “The Standard”, particularly when Health Authorities (HA), such as the US FDA and Japanese PMDA, made it a requirement for data submissions to support most of the regulatory requests for market approval. Additionally, most of the Pharma companies made the CDISC standards a part of their operational data model and consequently, the number of studies using the CDISC standards increased across phases of development.

The benefit of receiving data in standard formats was soon recognized by HA reviewers as they now require lesser time to understand the structure of the data they receive. Integration of data provided by different sponsors, for example on the same indication, for better understanding of safety signals, has become possible with data submitted in standard CDISC format.

However, the HAs such as the US FDA, soon realized that this was not enough, for two main reasons:

  1. sponsors sometimes make bad or different interpretations of the standard
  2. lack of standards or use cases in specific disease areas or indication
The first issue has been addressed by a number of specific HA conformance technical guidance [1,2] and regular public webinars where the HAs presented to the Industry real examples of bad standards implementations [3]. For the second issue, it was soon clear that the gap to be fixed was bigger and required more investment and resources. The CDISC Therapeutic Area User Guidance (TAUGs) are an “attempt” to fill this gap and reduce interpretation, and misinterpretation, of the standards [4].

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Introduction to Population Enrichment by Dr. Thomas Burnett

July 27, 2020

Cytel is conducting a webinar series on complex innovative trial designs. Dr. Thomas Burnett, Senior Research Associate in Medical and Pharmaceutical Statistics at Lancaster University, joined us as the presenter in the latest webinar from this series. In this webinar, “Adaptive Enrichment Designs in Clinical Development”, Dr. Burnett provides us a brief introduction to population enrichment and explains where it fits in clinical trials. He offers his insights on the topics of hypothesis testing and decision making, which is a key component of adaptive designs. You can also learn about a real-world case study (TAPPAS Trial) where this approach was used. Continue reading this blog for highlights from the webinar.

Watch the webinar recording and download the slides by clicking the button.

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Read an interview with Dr. Thomas Burnett on adaptive enrichment.

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Why Consider a Synthetic or External Control Arm?

July 23, 2020

Just as there are numerous adaptations that fall within the umbrella of adaptive designs, there are several different statistical methods that can lead to the construction of a synthetic control arm. Cytel’s ebook, “Demystifying synthetic control arms”, is an effort to explain common strategies for their construction. Download the ebook by clicking on the button.

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Access Sustainable, Verified Innovation with East Alloy

July 22, 2020

Cytel brings to you a new blog series on technology and Bayesian decision-making by Pantelis Vlachos, Principal/Strategic Consultant for Cytel. In his inaugural post Pantelis walks us through the features and benefits of our new offering, East Alloy™. East Alloy™ is a web-based extension of East for clinical trial design that blends the pace of SaaS delivery, the ease of use and robustness of Cytel software, and the velocity of cloud-based computing. Gain some behind-the-scenes insights into the development of this new module and understand how your company can leverage East Alloy to conduct computationally intensive designs with ease, confidence, and speed.

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Webinar: Head to Head Comparisons Using Real World Data

July 21, 2020

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.

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We also had the opportunity to interview Dr. Hernan on head-to-head comparisons. Read the interview here.

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Three Reasons Why Oncology Trials Need Clear Estimands

July 15, 2020

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?

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The FSP Engagement Landscape: Reducing Sponsor Oversight Burden

July 14, 2020

Cytel SVP Corey Dunham’s inaugural post on leading the industry’s largest Biometrics CRO considers the FSP Engagement Landscape, and how the responsibilities of Sponsors and FSP providers are shaped by the needs of different projects.

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Interview with Dr. Thomas Burnett on Adaptive Enrichment

July 13, 2020

Cytel is hosting a complimentary webinar series that introduces biostatisticians and other members of the development team to some of the more commonly used complex innovative trial designs, the benefits of each, and the practical considerations for adoption. You can access the replay of the completed webinars and register for the upcoming ones by clicking on the button.

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In this blog, we interview Dr. Thomas Burnett who is a Senior Research Associate in Medical and Pharmaceutical Statistics at Lancaster University. His main research interests are Adaptive clinical trials and personalized medicine. In the upcoming Cytel webinar on July 15, Thomas will be presenting on the foundational elements of enrichment strategies and adaptive designs.

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COVID-19 Clinical Development Quiz (with Answers)

July 11, 2020

Last week we challenged you to take a COVID-19 Clinical Development Quiz. Many of you used our Trial Tracker to find the right solutions. Here is the quiz again with solutions at the bottom.

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Synthetic control arms demystified: A Cytel Ebook

July 9, 2020

There has been an increased use of synthetic control arms for regulatory submissions in recent years, with three rare disease submissions and 22 medical device submissions approved using these techniques.

Synthetic control arms are virtual trial arms that use historical claims data and observational data to simulate the control arm of a study. When enrollment targets are low and large amounts of data already exist about the performance of a control, then in many cases using quantitative techniques to simulate a control arm of a trial will expedite timelines and serve as a more optimal use of resources.

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Overcoming Clinical Development Challenges in Oncology with Innovative, Adaptive Designs: Complimentary Paper

July 7, 2020

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.

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COVID-19 Clinical Development Quiz

July 2, 2020

There are currently 1570 registered clinical trials for COVID-19 therapies and vaccines. Approximately 20% are registered in the United States and 25% in the European Union. How well do you know the state of COVID-19 clinical development? Take our quiz to find out:

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The Good Data Submission Doctor: CDISC for COVID-19

June 29, 2020

From the time the COVID-19 outbreak was declared a pandemic, the number of studies conducted around the world to either diagnose, prevent or treat the virus literally exploded (1570 as on today, according to the Cytel Global Coronavirus COVID-19 Clinical Trial Tracker1).

Moreover, the pandemic impacted the regular schedule of ongoing clinical trials. Health authorities such as the FDA, promptly provided recommendations in the form of questions and answers on how to handle “disruptions” due to the pandemic2. These disruptions include a range of challenges including skipped assessments or study withdrawal.

“CDISC launched a task force in an effort to support CDISC members and the research community as they work tirelessly to discover critical breakthroughs to treat COVID-19 …. The task force was launched with the goal of developing Interim User Guide and Related material” said David Bobbitt, CDISC CEO, in an interview with Outsourcing-Pharma.com3. On April 21, 2020, the task force released two guidances. In this blog, I provide you with a quick summary of what these guidances address.

The two guidances are:

  • Guidance for Ongoing Studies Disrupted by COVID-19
  • CDISC Interim User Guide for COVID-19

Continue reading to learn more.

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New Whitepaper: Bayesian Methodologies for COVID-19 Drugs, Devices and Biologics

June 27, 2020

Expert statisticians at Cytel have spent the past three and a half months designing and deploying dozens of trials for COVID-19 trials. A new whitepaper describes the critical uses of Bayesian methodologies employed by Cytel statisticians, in their search for effective therapies, prophylactics and vaccines.

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Webinar: Practical Model-based Approaches for Phase I Oncology Trials

June 24, 2020

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.

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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.

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Head to Head Comparisons Using Real World Data: An Interview with Miguel Hernan

June 23, 2020

Supposing two treatments, A and B, need to be compared that have not been compared through a clinical trial. In the absence of such information, those treatments have been compared with each other via a third treatment, C (i.e., A to C and B to C) using indirect treatment comparison approaches.  Recent developments are challenging this status quo.  The increased availability of regulatory-grade RWD helps.  We can also now avoid some of the biases that used to plague the use of observational data.

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Webinar: Synthetic and External Controls in Clinical Trials

June 22, 2020

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.

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Why You Should Construct Primary Endpoints Using Bayesian Methods: Lessons from COVID-19

June 18, 2020

One of the revelations of the COVID-19 pandemic is that the flexibility and potential of Bayesian designs goes far beyond the benefits connected to informed priors. Rather a number of other benefits to Bayesian designs are sometimes under-appreciated. The importance of using Bayesian methods to choose composite endpoints, for example, particularly in longitudinal studies, can be overlooked when considering Bayesian and Frequentist options.

Cytel statisticians reflected on these benefits during a recent panel discussion.

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Optimizing Patient Recruitment: Download Whitepaper

June 18, 2020

A number of trials recently disrupted by the COVID-19 pandemic are now in the process of re-assessing recruitment timelines, and establishing new recruitment targets. Recruitment projections, though, are only as good as the starting assumptions with which a forecast is made and the models subsequently used to calculate timelines.

Overly simplistic assumptions, even familiar ones like, “All patients will arrive and enroll in a strictly linear fashion,” have strong  drawbacks. In order to maximize the potential to achieve recruitment milestones and to avoid misleading trial projections, planning must take careful precautions even when conditions are not this uncertain.

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Significance of Bayesian Model-Based Approaches in Oncology Trials: An Interview with Dr. Satrajit Roychoudhury

June 15, 2020

Cytel conducted a webinar with Dr. Satrajit Roychoudhury, Senior Director, Statistical Research and Data Science Center, Pfizer. Dr. Roychoudhury talked about practical model-based approaches for phase I oncology trials. This webinar is a part of Cytel’s “Introduction to Complex Innovative Trial Designs” webinar series. You can watch the recording by clicking on the button below.

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In this blog, we bring to you an insightful interview with Dr. Satrajit Roychoudhury where he talks to us about his interest in statistics, explains the concept of Bayesian model-based approaches and their importance in oncology trials.

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Weekly Updates from the COVID-19 Trial Tracker (June 12)

June 13, 2020

The Cytel COVID-19 Trial Tracker continues to bring you an up to the minute, real time dashboard about COVID-19 trials around the world. Here are a few updates from this week.

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Adaptive Bayesian Methods: The Secret Weapon in COVID-19 Vaccine Development

June 11, 2020

A recent Cytel panel led by Vice President of Strategic Consulting Natalia Muhlemann evaluated the role that Bayesian methods played in development of a COVID-19 vaccine. The wide-ranging discussion covered the structure and utility of platform trials and the role of master protocols in infectious disease vaccines development, but also addressed the importance of adaptive Bayesian methods in the search for COVID-19 therapies.

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Webinar: Group Sequential Designs and Sample Size Re-estimation

June 10, 2020

Cytel is conducting a webinar series that introduces biostatisticians to some of the more commonly used complex innovative trial designs. Our second webinar from this series was held on June 3, 2020, with Professor Christopher Jennison. Professor Jennison spoke about the modern uses of Group Sequential Designs and Sample Size Re-estimation. Group Sequential Designs were one of the earliest deviations from a traditional two-arm clinical trial with no interim looks at the data. They still add amazing value to trials through their abilities to safeguard patients, reach positive conclusions early and keep trial designs simple and streamlined. Sample Size Re-estimation is another key tool in the modern trial designer’s toolkit.

Cytel also did an interview with Professor Jennison where he spoke to us about these methods, their origin and how they add value to the industry. Continue reading this blog for a summary of the webinar. Click on the button below to access the webinar replay and download the presentation slides.

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Trevor Mundel, President of the Bill & Melinda Gates Foundation, on COVID-19 and the Future of Drug Development in Emerging Economies

June 9, 2020

Trevor Mundel leads the Bill & Melinda Gates Foundation’s efforts to develop high-impact interventions against the leading causes of death and disability in developing countries. During a Cytel panel on the COVID-19 response, Trevor reflects on the complexities of data management, forecasting, dose-finding, recruitment and retention, when responding to a global pandemic.

Hear the entire conversation by clicking the button below or read further to get some of the highlights of this critical discussion.

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New Primer and Ebook on Synthetic Control Arms

June 8, 2020

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

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COVID-19 Weekly Brief

June 4, 2020

This weekly snapshot gives you a quick briefing on the state of COVID-19 therapy and vaccines development. As we head into June, nearly 1500 trials have been registered and over 100,000 patients have been recruited into said trials.

Here is what you need to know:

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Webinar: Innovative Drug Development at a Glance

June 1, 2020

In a recent interview with Cytel, Zoran Antonijevic, longstanding chair and leader of the DIA Adaptive Design Scientific Working Group, spoke about adaptive design methods. He gave us insights on how these methods can help achieve new levels of clinical trial efficiency and probability of success.

Cytel is conducting a webinar series that introduces biostatisticians to some of the more commonly used complex innovative trial designs. On May 20, 2020, Zoran Antonijevic joined us as a speaker at a webinar from this series, “Innovative Drug Development at a Glance - The Concepts, The Vision, & The Factors to Consider”. In the webinar, Zoran defines complex innovative trial designs, summarizes the potential benefits of each, and highlights key factors to consider when applying these techniques. Continue reading this blog for a summary of the webinar. Click the button to get free access to the webinar slides and recording.

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Updates from the Trial Tracker: The State of COVID-19 Development (May 2020)

May 29, 2020

At the close of May 2020, we have about 500 new trials globally but trends in trial design and choice of therapies tested have largely remained the same. The state of Vaccine Trials though has changed in both number and trial design.

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COVID-19 Response: New Opportunities & Implications for the Future of Drug Development in Emerging Economies – Q&A with James Orbinski on Global Health Policy

May 28, 2020

On May 7, Cytel and Certara conducted a virtual panel discussion on new opportunities and implications for the future of drug development in emerging economies. The speakers included highly acclaimed key opinion leaders and industry experts who spoke about new sources of research funding being channeled towards emerging economies and the need to understand its strategic priorities to properly assess future opportunities for growth.

Our first panelist, James Orbinski is a professor at York University's Dahdaleh Institute for Global Health Research. James is an experienced medical doctor, a humanitarian practitioner, a best-selling author and a leading scholar in global health. After extensive field experience with Médecins Sans Frontières / Doctors Without Borders (MSF), Dr. Orbinski was elected MSF’s international president from 1998 to 2001. He launched its Access to Essential Medicines Campaign in 1999, and in that same year accepted the Nobel Peace Prize awarded to MSF. Our moderator from Cytel, Principal Scientist, Edward Mills began the virtual panel discussion by asking James a series of pertinent questions on the issues around the current pandemic. Continue reading this post for the Q&A.

Get access to the virtual panel replay by clicking on the button below.

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Interview with Professor Christopher Jennison: Group Sequential Designs and Sample Size Re-estimation

May 27, 2020

In this blog, we speak with Christopher Jennison, Professor of Statistics at the University of Bath, UK. Professor Jennison provides us insights on group sequential methods, the origin of their implementation and the value they have been adding over the years.

Cytel is hosting a new webinar series that introduces clinical fellows, early career biostatisticians, and others interested in clinical research, to some of the more commonly used complex innovative trial designs. In our previous blog, we spoke with Zoran Antonijevic about adaptive design methods.

Join us for a complimentary webinar on June 3, 2020 where Professor Jennison is going to introduce us to the basics of group sequential designs and sample size re-estimation. Learn how to use these methods and understand how they can improve trial design.

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4 Things You Need to Know about COVID-19 Trial Designs

May 22, 2020

The Cytel Trial Tracker now features summary plots that display trials by country, trial status and study design. This enables us to take a deeper dive into patterns of trial design in COVID-19 studies. This blog post explores the use of single-arm studies, numbers of multiarm studies, and more.

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Interview with Zoran Antonijevic on Adaptive Design Methods

May 18, 2020

In this blog, we speak with Zoran Antonijevic, longstanding chair and leader of the DIA Adaptive Design Scientific Working Group, and former Principal Statistical Consultant at Cytel. We speak about adaptive design methods, their value to the industry, their success and ways to overcome the existing challenges including the hesitation towards its wide adoption.

This summer Cytel is hosting a new webinar series that introduces clinical fellows, early career biostatisticians, and others interested in clinical research, to some of the more commonly used complex innovative trial designs. These webinars will cover the benefits of each design, and the practical considerations for adoption.

On May 20, 2020 Zoran Antonijevic is going to present an introductory overview of all these advanced design methods, in a webinar from this series. This webinar will define complex innovative trial design, briefly introduce the concepts, summarize the potential benefits of each, and highlight key factors to consider when applying these techniques. You can register by clicking on the button below.

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Weekly COVID-19 Trial Tracker Updates

May 14, 2020

This has been an exciting week for COVID-19 studies. We learned that several Cytel clients who have designed new clinical trials using our East software, are about to begin enrolling. Our subject-matter experts are also heavily involved in designing vaccine trials, as well as offering biometrical support and data management for three vaccine trials that have already begun to enroll.

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Innovations in Oncology Trial Design & Development Webinar Series

May 12, 2020

In our previous blog, “Remote Working Arrangement – How to get it right?”, we talked about how the need for social distancing has led most of the employers, across the globe, to make work-from-home arrangements for their employees. As we continue to stay indoors and combat COVID-19, keeping aside some time every day to read and watch useful resources on important industry topics can be very helpful. Cytel's team of oncology trial design and advanced analytics experts have been hosting a series of complimentary webinars covering a range of innovative topics including adaptive design, machine learning, estimands and trial design software. In this post, we offer you a recap of the webinars we conducted in the past few weeks. You can register for the upcoming webinars in our oncology series by clicking on the button below.

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Weekly Insights from the COVID-19 Trial Tracker: Remdesivir, the Oxford Vaccine Study & More

May 7, 2020

There are now over 950 trials registered, which means that 250 new trials were registered in the past week.

Only 540 of these are currently recruiting patients. This would suggest that a number of trials are failing to reach recruitment targets. The most successful trials, though, appear to suggest otherwise. They are recruiting at an unprecedented rate.

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COVID-19: Trials, Designs and Tools for Promising Results - A Virtual Panel Discussion

May 7, 2020

An extraordinary amount of global research is underway as the COVID-19 pandemic continues to evolve and spread throughout the world. There are over 800 registered global clinical trials taking place to develop life-saving treatments and vaccines for patients. The World Health Organization is also facilitating collaboration and accelerated efforts on an unprecedented scale. In these difficult times, sponsors must utilize innovative tools and approaches to design their clinical trials in order to provide promising results for all patient populations as quickly and efficiently as possible. 

A successful virtual panel discussion was conducted by Cytel on the ongoing COVID-19 Trials, on April 15. For the second complimentary virtual panel discussion held on April 23, Cytel partnered with Certara, to present, “COVID-19: Trials, Designs and Tools for Promising Results”. It began with challenges faced by clinicians and drug developers, followed by examples of tools and trial designs currently being used to help sponsors of COVID-19 trials. Continue reading for a summary of the panel discussion.

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Webinar: A Clinician’s Perspective on Cancer Drugs Development

May 5, 2020

Cytel's team of oncology trial design and advanced analytics experts are hosting a series of complimentary webinars covering a range of innovative topics and solutions. On April 28, 2020, Cytel conducted a webinar with Professor Martin Fey, Medical Oncologist, “A Clinician’s Perspective on Cancer Drugs Development”. Our previous blog features an interview with Professor Fey where he talks about his experience of over forty years in medical oncology, the evolution of clinical cancer trials, the difference between clinically meaningful and statistically significant results, the debate around patient perspectives and other important topics around cancer drugs development.

In his webinar, Professor Fey provides us an overview of drug development for cancer treatments, clinician’s perspective on endpoints, importance of patient reported outcomes and patient perspective, and the significance of biomarkers. Continue reading this post for key highlights from the webinar.

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Weekly Insights from the COVID-19 Trial Tracker

May 1, 2020

There are currently 665 trials registered at Cytel’s COVID-19 Trial Tracker, a jump of over 100 trials in the past two weeks. Over 20% of these trials are for chloroquine and hydroxychloroquine, while only 10 of 17 vaccine trials are currently recruiting. Are we collectively prioritizing the correct investigational studies?

Cytel's Weekly Insights takes a closer look into what the data is now telling us. We also examine projected recruitment and completion dates for vaccine trials using two new functionalities: the Cumulative Registration function shows the total number of trials that are ongoing, when they began and when they are projected to end. The Cumulative Recruitment function shows expected enrollment by treatment.

Taken together we get a clearer picture of the journey so far and insight into the complexity of a path toward a vaccine and a cure. 

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Webinar: Transparent Machine Learning in Oncology

April 29, 2020

In our previous blog, we spoke with Alind Gupta, who works as a Machine Learning Researcher at Cytel in Canada. The interview gives you a deep dive into black-box models and transparent machine learning, and how the latter is becoming more important in clinical research today.

On March 21, Cytel conducted a webinar with Alind on, “Transparent Machine Learning in Oncology”. Alind presented our continuing work in immuno-oncology using Bayesian network models for predicting safety and survival outcomes, extrapolating from limited follow-up data and validating with external real-world data for key subgroups. Continue reading for key highlights from the webinar.

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Highlights from the 2020 Virtual CDISC EU Interchange by Angelo Tinazzi - Part 2

April 27, 2020

In the first part of this two-parts blog, I speak about how the European CDISC Committee (E3C) together with CDISC converted our physical event into a virtual one and was held on April 1-2, 2020. I provided a summary of the updates received from the three main health authorities - the US FDA, the Japanese PMDA and the European EMA.

This post offers an overview of the other sessions I attended at the 2020 Virtual CDISC EU Interchange. The agenda was well planned and organized. The distinguished speakers were extremely prepared and answered numerous questions after their presentations. Continue reading for further highlights from the event.

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Highlights from the 2020 Virtual CDISC EU Interchange by Angelo Tinazzi

April 24, 2020

In early March, when countries around the world started implementing lockdowns, the European CDISC Committee (E3C) together with CDISC decided to cancel our physical event in Berlin, planned for April 1-2, 2020. It was a tough decision, but unavoidable and necessary.

We did not let this dampen our spirits and immediately came up with an alternative plan – go virtual with the event! In only two weeks the team managed to pull together a revised program and the registrations were opened on the CDISC website. The scale of the event went from being Europe-only to Global, and around 300 people attended it worldwide. In the end, the event was a hit. Everything worked out very well, with no major technical disruptions and the speakers respecting the allocated time slots.

In this two-part blog post, I share a summary of the sessions I was able to attend, while simultaneously ensuring business continuity for my regular projects.

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A Clinician’s Perspective on Cancer Drugs Development: An Interview with Martin Fey

April 23, 2020

Cytel is hosting a webinar, “A Clinician’s Perspective on Cancer Drugs Development”, on April 28, 2020. Our speaker, Professor Martin Fey, Medical Oncologist from Switzerland, will brief us on treatment evolution and give us a deep dive into clinician perspective on endpoints, PRO and patients perspectives, and importance of biomarkers in oncology.

In this interview, we speak to Professor Fey about his experience of over forty years in medical oncology, the evolution of clinical cancer trials, the difference between clinically meaningful and statistically significant results, the debate around patient perspectives and other important topics around cancer drugs development.

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Weekly Insights from the COVID-19 Trial Tracker

April 23, 2020

Every Week Cytel Brings You Further Insights from the COVID-19 Trial Tracker

From April 8 through April 17, the number of clinical trials testing COVID-19 therapies has risen to 590 world-wide. An estimated 354,000 people will need to be recruited to test these treatments properly, representing a substantial proportion of all patients diagnosed with COVID-19. Last week we looked at recruitment for chloroquine and hydroxychloroquine. This week we take a look at studies for lopinavir, remdesivir, and plasma based therapy.

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Interview with Alind Gupta: Transparent Machine Learning in Oncology

April 20, 2020

Cytel is hosting a webinar on Transparent Machine Learning in Oncology, on April 21, 2020. Our speaker, Alind Gupta, Machine Learning specialist, will provide insights on a particular transparent ML method called Bayesian networks, and how we have been using it for HEOR and other real world applications in oncology trials. As the adoption of machine learning is on the rise, we speak to Alind about the differences between black-box models and transparent machine learning, and how the latter is becoming more important in clinical research today. Alind also speaks about the application of ML on real-world data and how it is going to evolve in the coming years.

Machine learning (ML) aims to discover patterns from data that can be used for prediction, but the use of “black-box” ML models in healthcare research and decision-making has been limited, due to clinical liability and lack of trust from stakeholders. FDA guidelines for ML-based devices mandate transparency to assure continual safety and efficiency as notable recent failures have prompted increasing ML research into bias, fairness and causality. This has ramifications for all therapeutic areas but particularly within oncology.

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Early Insights from Cytel’s New COVID-19 Trial Tracker

April 14, 2020

Last week Cytel launched a COVID-19 Trial Tracker, an Open Access tool to track the global response to the coronavirus pandemic. This central repository of clinical trials will be updated daily to ensure that all new scientific findings can be easily identified on a single website.

While early results are still coming in, there are already important insights we can glean about the current state of research from the COVID-19 Trial Tracker. Delving deeper into these insights results in important new questions about global cooperation for clinical development.

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Career Perspectives: Interview with Marc Lefebvre-Gouy, Statistical Programmer

April 7, 2020

Cytel has industry-leading experts in Statistical Programming with years of SAS® Programming expertise and in-depth knowledge of the specific clinical subject matter. Their insights into strategy, and high competence in project management ensure a new twist on the traditional CRO.

In February 2020 we interviewed Marc Lefebvre-Gouy who is a lead statistical programmer at Cytel in France. Marc made a career transition from genetics to statistical programming and in this interview, he speaks to us about programmers working across therapeutic areas, programming challenges across phases of a trial, and the top technical competencies every programmer must have. He also gives us a sneak peek of his hobby of cooking and his love for wine.

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Remote Working Arrangement – How to get it right?

April 2, 2020

On March 16, the World Health Organization (WHO) Director-General, Dr. Tedros Adhanom Ghebreyesus, in his media briefing on COVID-19, described the coronavirus pandemic as “the defining global health crisis of our time"[1]. The pandemic has affected 176 countries and territories around the world. WHO is urging all governments to scale-up aggressive measures to combat COVID-19 as more cases and deaths are being reported across the globe. Until a vaccine is made available, social distancing is of the utmost importance to reduce transmission and enable health systems to cope.

The U.S. Centers for Disease Control and Prevention (CDC) defines social distancing as “remaining out of congregate settings, avoiding mass gatherings, and maintaining distance (approximately 6 feet or 2 meters) from others when possible” [2]. Governments in several countries have shut down public places such as parks, gyms, malls, schools, restaurants etc., for a minimum of 15 days, to be extended depending upon the situation.

In these circumstances, it is inevitable that companies worldwide also show solidarity by allowing their employees to work remotely, from the safety of their homes. In Seattle, the hub of many of America’s early COVID-19 cases, companies including Amazon, LinkedIn, Microsoft and Google advised workers to stop coming to office in late February [3]. With more employers taking this necessary and precautionary step, the question arises - how do we ensure that it is done right?

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Webinar: Key Design Considerations for Basket Trials and Umbrella Trials by Jay Park

March 31, 2020

 

Since 1953, when the discovery of the structure of DNA was made, we have seen great advancements in genomics. Particularly, in the last few years, the industry has seen a rapid rate of adoption in biomarkers and how they can be used to improve biomedical interventions. Trial investigators have been showing interest in biomarker-guided trials such as basket trials and umbrella trials, developed under the master protocol framework. As a result, we have been seeing a rapid rate of adoption of these innovative trial methods.

In our previous blog, we spoke with Jay Park, Director, Cytel, about the concept of master protocols, their importance and future growth potential. On March 19, Cytel conducted a webinar with Jay on “Key Design Considerations for Basket Trials and Umbrella Trials”. This webinar introduced two master protocol types and explored their extension to design in various contexts from the HIV epidemic in global health to expedited oncology trials. Continue reading for key highlights from the webinar .

Register now to get free access to webinar slides and recording.

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Cytel's Response: EMA Points to consider on implications of Coronavirus disease (COVID-19) on methodological aspects of ongoing clinical trials

March 26, 2020

Further regulatory guidance has been released concerning the implications of the Coronavirus disease (COVID-19) on clinical trials.

 

On March 25th the Biostatistics Working Party (BSWP) of the European Medicines Agency (EMA) Committee for Human Medicinal Products (CHMP) published a draft points to consider guidance document on the actions that sponsors of affected clinical trials should take to help ensure the integrity of their studies, and the interpretation of the study results, while safeguarding the safety of trial participants as a first priority.

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