The Cytel blog keeps you up to speed with the latest developments in biostatistics and clinical biometrics.
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.
The TOGETHER Trial: Cytel Designs and Implements Novel Adaptive Platform Trial for COVID-19 Therapies
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.
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.
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.
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.
“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 . 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.
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.
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.
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.
Cytel Co-Founder Cyrus Mehta Presents at the Heart Failure Collaboratory, a Public-Private Partnership with FDA
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.
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.
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.
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. Click the button to access the on demand webinar.
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.
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.
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.
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.
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
Career Perspectives: Interview with Mrudula Joshi, Associate Director, Statistical Programming Services
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.
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
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.
Making the Most of Your Data II: Optimizing Clinical Information in Trial Design and Implementation Using Bayesian Methods
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.
Making the Most of Your Observational Data: Creating a Synthetic Control from Your Natural History Study
Recently a biotech approached Cytel for support with a Phase 2 Study in oncology. Regulators had requested a natural history of disease study, which tracks disease progression in the absence of any form intervention. These studies are used to build disease-models that can then inform a range of development opportunities within a drug development program.
A March 2019 FDA Guidance highlighted the importance of such studies for rare diseases, with former FDA Director Scott Gottlieb acknowledging that a lack of knowledge about the natural history of certain diseases is a significant obstacle in rare disease drug development.
Head to Head Comparisons Using Real World Data – Design and Data Considerations from Cardiovascular Pilot Investigation
Cytel is conducting two pilot projects on head-to-head comparisons using real world data. These projects in oncology and cardiovascular will occur in real time and will take place across our latest webinar series on the topic. The aim of this series is to introduce our audience to head to head to comparison using Real World Data (RWD) while focusing on practical application and results from the pilot projects.
The second webinar from this series was held on July 28, 2020 and outlines the design of the cardiovascular pilot investigation. None of the existing randomized trials of recently developed second-line antihyperglycemic agents can provide adequate information on their comparative effectiveness and safety regarding cardiovascular outcomes. Conducting Target Trials to get information of interest would be costly, difficult to perform, and would take many years to complete. As a result, we need to use observational databases to emulate it. This blog provides a brief on the design of the cardiovascular pilot project. Our team of experts also discussed the data requirements and the data source to be used in the pilot investigation, with the primary challenge focusing on how to assess if data are sufficient for the purposes of trial emulation. Continue reading the blog for a summary of the webinar.
Get access to the webinar recording and download the slides by clicking the button.
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.
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.
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:
- sponsors sometimes make bad or different interpretations of the standard
- lack of standards or use cases in specific disease areas or indication
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.
Read an interview with Dr. Thomas Burnett on adaptive enrichment.
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.
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.
Cytel is conducting a webinar series that focuses on target trial emulation and causal inference approaches using real world data. In collaboration with Dr. Miguel Hernán, Professor at Harvard University, Cytel is pioneering two “Head-to-Head Comparisons using Real World Data” studies, one in oncology one and in cardiovascular disease. These projects will occur in real time across this webinar series. Our presenters for the first webinar in this series were Dr. Miguel Hernán and Devon Boyne, Director of Epidemiology at Cytel. Continue reading this blog for a summary of the webinar, “Head to Head Comparisons Using Real World Data - The Time for Causal Inference is Now” conducted on July 7, 2020. Click on the button to access the webinar replay.
We also had the opportunity to interview Dr. Hernan on head-to-head comparisons. Read the interview here.
Unlike many therapeutic areas, oncology benefits from having standardized endpoints like overall survival and progression-free survival, as well as standardized methods of measuring such endpoints. Given that the purpose of an Estimands Framework is ostensibly to streamline scientific questions with specific targets of estimation, what is the benefit of an Estimands Framework for oncology?
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.
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.
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.
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.
Overcoming Clinical Development Challenges in Oncology with Innovative, Adaptive Designs: Complimentary Paper
Having its roots in the seminar rooms of the Dana Farber Cancer Institute, Cytel has a long record of establishing new methods in small samples, adaptive designs, Bayesian designs and multi-arm trials, to align statistical rigor to the goal of accelerating clinical development for oncology trials.
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:
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.
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.
Last week, Cytel conducted its third webinar in the new introductory webinar series on Complex Innovative Trial Designs. Our speaker, Dr. Satrajit Roychoudhury is a Senior Director, Statistical Research and Data Science Center at Pfizer. In this webinar, Dr. Roychoudhury gets into the basics of phase I designs in oncology trials, explains the caveats of frequently used traditional designs and provides insights on how implementing a model-based approach can enable a better statistical inference and decision-making. You can watch the replay of the webinar and access the slides by clicking on the button.
We also had the privilege to interview Dr. Satrajit Roychoudhury. Read our blog where he talks about his interest in statistics, explains the concept of Bayesian model-based approaches and their importance in oncology trials.
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.
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.
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.
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.
Significance of Bayesian Model-Based Approaches in Oncology Trials: An Interview with Dr. Satrajit Roychoudhury
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.
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.
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.
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.
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.
Trevor Mundel, President of the Bill & Melinda Gates Foundation, on COVID-19 and the Future of Drug Development in Emerging Economies
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.
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:
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.
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.
COVID-19 Response: New Opportunities & Implications for the Future of Drug Development in Emerging Economies – Q&A with James Orbinski on Global Health Policy
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.
Interview with Professor Christopher Jennison: Group Sequential Designs and Sample Size Re-estimation
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.
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.
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.
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.
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.
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.
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.
Get free access to COVID-19 Panel slides and recording.
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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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.
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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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.
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.
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.
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.
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.
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.
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.
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". 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” . 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 . With more employers taking this necessary and precautionary step, the question arises - how do we ensure that it is done right?
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 .
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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
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.
Cytel's Response: EMA Guidance on the Management of Clinical Trials During the COVID-19 (Coronavirus) Pandemic
On March 20th the European Commission, the European Medicines Agency (EMA) and the Heads of Medicines Agency (HMA) published new recommendations for sponsors on how to manage the conduct of clinical trials in the context of the COVID-19 pandemic. Extraordinary measures may need to be implemented and trials adjusted due to quarantine, limited access to hospitals, and healthcare professional focus on critical tasks. Here is a review of selected elements of the guidance, interpretations and recommendations.
The FDA issued a guidance yesterday on how the COVID-19 Pandemic may affect the conduct of clinical trials. Below are some key messages from the guidance along with some interpretations and recommendations.
In September 2018, the FDA provided a draft guidance on master protocols reflecting an increased interest in these designs by industry. This came after a 2017 editorial published by the Drs. Woodcock and LaVange from the FDA in the New England Journal of Medicine. In this guidance master protocol is defined as a protocol designed with multiple substudies, which may have different objectives and involves coordinated efforts to evaluate one or more investigational drugs in one or more disease subtypes within the overall trial structure. As the adoption of these innovative trial methods is on the rise, we speak with Jay Park, Director, Cytel, about the concept of master protocols, their importance and future growth potential. We take a closer look at their use in oncology trials where the increase in biomarker driven enrichment and stratification designs, as well as the use of companion diagnostics, ensures that master protocols are often adopted by trial investigators.
Generating high-quality clinical data is a vital but challenging task in modern drug development. Unfortunately, in the current era of ‘big data’ and global clinical operations, spanning multiple sites and digital systems, protecting the quality of clinical data has become harder than ever.
Planning your data strategy is, therefore, crucial to ensure a high-quality evidence package and increase the chances of successful clinical development. However, as we discuss in our new eBook, planning a data strategy is a complex process involving various considerations that require significant amounts of time and expertise to fully address.
Read our eBook for expert insights on planning a data strategy that can help overcome key challenges in clinical development and boost your success.
In this blog, we discuss the many data-related challenges commonly faced in clinical development and how to implement a fail-safe data strategy that can overcome these challenges, bringing effective new therapies to patients.
Over the past decade, a new trend began to emerge, changing the way that clinical trials are conducted. Whereas placebo-controlled randomized control
It is widely acknowledged among drug developers that one of their most important assets is the data generated during clinical trials. Hence, it is no surprise that many companies plan and execute a strategy to protect the quality of the clinical data they produce. It is, however, easy to underestimate just how much time and expertise you need to address the numerous and complex considerations involved in the planning process.
Unlock top tactics and tips on how to plan a rock-solid data strategy to minimize risk and boost clinical success in our latest eBook.
If you are keen to find out how to optimize your clinical data strategy, read on to discover five of the top tips outlined in our eBook from specialists working in the Strategic Consulting, Clinical Research Services, and Data Management teams. Their global reach ensures top insights from every corner of the world.
In clinical development, a high-quality evidence package is a prerequisite for a new therapy to gain approval from regulators and other key decision-makers. As such, the quality of your clinical data is one of the key factors determining whether an effective new therapy reaches patients.
Implementing a data strategy can help to protect the quality of your evidence package. However, many companies start planning their strategy quite late in the development process, which makes it difficult to address (sufficiently address) the complex considerations involved. As we explore in our new eBook, a data strategy planned well in advance of starting Phase 1 and following the industry’s best practices can help you reduce risk, expedite clinical development, and successfully achieve your business objectives.
Download the new eBook, “Are you Harnessing the Power of your Clinical Data?” to find out how to optimize your data strategy to advance clinical development.
In our previous blog, we talked about the value of planning a data strategy for the entire duration of your program (i.e., a ‘program-wide’ strategy). However, it is also important to plan for specific phases of clinical development, because they each have unique challenges. Below we discuss the major challenges commonly encountered in Phase 1 and Phase 2 studies, and the tactics you can use to resolve them. An upcoming article will engage with challenges in Phase 3 and post-market.
The Cytel Trial Design Innovations (CTDI) Webinar Series recently hosted a webinar on designing event-based studies. Such studies are essential to designing high-efficiency clinical trials in certain therapeutic areas, but they add a number of challenges to the already complex landscape of adaptive trials.
The webinar was held on January 23rd, featuring Biostatistician and pioneering Bayesian trial-designer Pantelis Vlachos. We had the opportunity to sit down with Dr. Vlachos and speak about innovative trial designs and their benefits, adaptations and interim looks in oncology and cardiovascular, the challenges of designing event-based studies more generally, and how Cytel’s array of software tools, particularly East®, has enabled trial sponsors to fully consider their options in the design of high-efficiency clinical trials.
In the quest for clinical success, we all strive for evidence packages of the highest quality. If the clinical data is strong, then a promising new therapy is more likely to obtain approval from key stakeholders, such as regulators and payers . As a result, you’ll get the chance to develop a therapy that will help many patients (and you will likely gain returns on your investments). As we discuss in our new eBook on data strategy planning, a carefully planned data strategy can help mitigate risks to your programs and enable you to successfully achieve your goals.
Discover how to plan a data strategy that enhances your clinical programs and enables new therapies to reach patients in our new eBook.
In the high-stakes environment of clinical development, it is never too early to start protecting your valuable data assets with a first-rate strategy. So, keep reading to learn which planning approach to use, who should be involved, when it is best to start, and why it is well worth going to all the effort.
January’s Cytel Trial Design Innovations (CTDI) Webinar Series will feature Biostatistician and pioneering Bayesian trial-designer Pantelis Vlachos. Next week, Dr. Vlachos will speak on high-efficiency trial design for Event-Based Studies, particularly in oncology and cardiovascular trials. In this blog post, we offer a glimpse of Dr. Vlachos’ last CTDI Webinar (February 2019) on high-efficiency trial design using enrichment strategies.
Don't miss next week's webinar "Designing Event-based Studies: Reduce Sample Size and Increase Predictability"! Click on the button below to register.
In clinical development, data is the vital ‘foundation’ that supports your programs. To successfully bring a promising new therapy to patients, the quality of your evidence must be strong enough to gain approval from key decision-makers, including regulators, payers, and health technology assessment (HTA) agencies.
So, how can you strengthen the quality of your clinical evidence package? A key solution is to optimize your data strategy. As we discuss in our new eBook on data strategy planning, making just a few small changes to your planning approach can strengthen your ‘foundation’ and generate various benefits that enhance and expedite clinical development.
Download our free eBook to find out how to optimize your data strategy to boost success in clinical development.
With only two weeks left for this fabulous year to end, we would like to thank all our blog subscribers and new readers for following and appreciating the Cytel blog. This year, we collaborated with several experts from both within and outside the company to bring to you a range of interesting topics including real-world evidence, AI, challenges in rare diseases, patient-reported outcomes, data management, and our popular series “The Good Data Submission Doctor” and “Career Perspectives”. In this blog, we share with you the top 5 Cytel blogs that resonated most with our community in 2019.
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Cytel Inc. and Axio Research joined forces in June 2019, expanding our ability to solve the most complex analytical challenges for the life sciences industry. Cytel offers a full range of clinical data management services delivered by advanced analytics experts with global reach.
In this blog, we talk to Ronald Dumpit, who is based in Bremerton, Washington to find out more about his career path, current role at Axio and his interests outside of work.
PhUSE EU Connect 2019 was held in the beautiful city of Amsterdam between the 10th and 13th of November. This clinical data science conference comprised 19 Streams, including 150 papers, 24 posters and 3 engaging data scientists as keynote speakers. The event was well attended and had several interesting and innovative presentations. Caroline Terrill, Associate Director of Statistical Programming at Cytel UK, conducted a session “No Place Like Home: Managing Remote Programmers Remotely” and stood out as the winner in the Personnel Management category. Based on 5 years' experience of managing remote programmers, Caroline’s paper gives guidance on issues to be considered and traps to be avoided if you are managing people who work remotely.
In this blog, we share the presentations from our Statistical Programmers and summarize some of the sessions that our team members attended.
In association with Statisticians in the Pharmaceutical Industry (PSI) , UCB and Cytel hosted a symposium on September 11, 2019 at UCB’s offices in Slough, Berkshire. The primary agenda was to educate the audience on Artificial Intelligence (AI) approaches and their impact on clinical development.
With recent advances in AI, it is important for quantitative scientists to keep up to date with the most recent methods and be involved in guiding their application to the most pressing analytical challenges. This one-day event covered cutting edge examples of how data science and statistical sciences are intersecting, and its relevance to our attendees.
“Artificial Intelligence and associated methodology is becoming increasingly important to the Pharma Industry and its technical foundation in statistical theory means that PSI is naturally keen to promote good practice through its membership and established Industry links. PSI is proud to have set up a Special Interest Group in this field and is keen to broaden its links and membership.”
- PSI Data Science special interest group
In this blog, we share some of the key takeaways from the symposium. If you are interested in attending similar sessions, you can check Cytel’s list of upcoming events here.
Biotechs and Medtechs, don’t forget your market access strategy (part 4 of 4): How to optimize your market access planning approach
Author: Michael S. Paas, Market Access & Commercialization Expert, Executive at AbbVie and Guest Author at Cytel
In crafting your market access strategy, it can be valuable to follow these five best practices:
- Start planning as early as possible in the drug or device development process, to give yourself enough time to make informed, optimal decisions, and adjust your strategy when needed;
- Plan for global markets and seek early advice from payers and HTA agencies, where possible. There are many different agencies and payers to think about when you launch globally, so it’s vital to ensure you are sufficiently addressing the requirements of the priority markets. Even if you do not plan to commercialize by yourself in countries outside the US, careful, early market access planning with key global markets in mind can maximize the attractiveness of your asset to potential licensors or partners;
Biotechs and Medtechs, don’t forget your market access strategy (part 3 of 4): Harnessing the value of market access planning
Author: Michael S. Paas, Market Access & Commercialization Expert, Executive at AbbVie and Guest Author at Cytel
Addressing the myriad of considerations required for market access planning can be complex and time-consuming, so it is vital to give yourself enough time to make informed decisions. As such, starting to plan your market access strategy at least by early phase II or the proof-of-concept phase of device development can help you inform the design of the clinical program in such a way to ideally generate evidence credible and compelling to payer and HTA stakeholders, as well as regulators, in order to maximize the commercial success of your drug or device.
Planning early also gives you the opportunity to make adjustments along the trajectory of development as you gain new information and insights about the asset itself, as well as about the payer and competitive environment.
At the 2019 Challenges in Rare Diseases Clinical Trials Symposium and East training, Cytel partnered with Alexion to bring together expertise from academia and industry. David Kerr, Director of DMC Services at AXIO, was among the notable speakers and his talk “Data Monitoring Committees – Behind Closed Doors” covered general considerations and options that the DMC has when reviewing the data presentations during their closed sessions. He presented four specific case studies that highlighted the data provided to the DMC from meeting to meeting and discussed how the DMC arrived at their recommendations for each meeting. We had the opportunity to sit down with David and speak about Data Monitoring Committees, understand their proceedings and talk about his presentation at the symposium.
Biotechs and Medtechs, don’t forget your market access strategy (part 2 of 4): The critical role of market access planning in clinical development
Author: Michael S. Paas, Market Access & Commercialization Expert, Executive at AbbVie and Guest Author at Cytel
In my previous post, I established the importance of market access strategy in the clinical development process and why emerging biotechs cannot afford to overlook it.
Click on the button if you missed Part 1 or want to revisit it.
It is imperative to start developing your strategy with market access due diligence and value story creation early in the product development (at least by phase II of drug development or the proof-of-concept phase of a device development). Impactful market access planning involves designing your clinical programs so they sufficiently address the evidence needs of market access stakeholders, patients, and regulators. This means demonstrating the value of your therapy to payers and HTA agencies by generating compelling and robust clinical trial data against appropriate comparators, augmented where possible and helpful with real-world evidence, health economic arguments, economic models and/or relevant patient-reported outcomes.
Biotechs and Medtechs, don’t forget your market access strategy (part 1of 4): Why is market access strategy crucial to succeed?
Market access strategy is an integral part of the clinical development process to ensure success in global healthcare markets and vital access to patients
In clinical development, it’s easy to underestimate the critical role that market access plays in the commercial success of a product. Understandably, organizations instead focus on the regulators, and the steps needed to secure eventual approval. However, ensuring the development plan is aligned with a well-planned market access strategy is essential to help the therapy actually reach the intended patients and realize its true commercial potential following regulatory approval. This is particularly crucial considering that payers and other key stakeholders demand compelling information on a drug’s/device’s value as well as justification of its pricing.
In this four-part blog series, I will address what can be done to optimize your market access planning, and why all that effort is really worth it.
Interview with Kannan Natarajan: Drug Development in Rare Diseases - Need for Innovation in Statistical Thinking
Cytel is delighted to have Kannan Natarajan speaking at the “Complex Innovative Trial Design Symposium and East User Training” on November 6 in Boston, MA. We got a chance to sit down with Kannan and talk about his career in statistics, the changing role of statisticians, his views on evolving statistical thinking, estimands and relevance of technology in the context of rare diseases.
October 3, 2019 was an important day for the ADaM team as it marked the release of the ADaM Implementation Guidance (Ig) 1.2. Download the new guidance from the CDISC website here.
In 2018, several previews of the new Ig were made at CDISC Interchanges all around the world and at PhUSE conferences, it was only a few weeks ago that the final version was released. At first glance, the new Ig does not seem to contain a lot of new concepts and ideas. However, a critical and in-depth review clearly shows the efforts of the entire team to release this new Ig. Creating a new standard or releasing a new version of an existing standard is not an easy job. You need to ensure that you do not introduce anything that contradicts any of the existing CDISC standards. This includes, not only new variables but also any changes in existing sentences or adding entirely new sections that may cause misinterpretations or discrepancies. Moreover, every standard team member comes with their own background, company needs, and specific indication needs. It is not always easy to propose a solution that can satisfy everyone on the team.
A disease is generally considered to be rare if it affects one patient per 200,000 people (1) and most rare diseases affect far fewer than this. However, collectively rare diseases are relatively common, affecting 350 million patients worldwide (2). The path to diagnosis for these patients is often a long, difficult battle and even once the diagnosis is made, it is likely there will be no suitable treatment available. For 90% of rare diseases, there is no approved therapy (2). There is, therefore, a pressing need to develop new, effective therapies that can bring hope to rare disease patients. However, the clinical development environment for life-threatening, rare diseases is fraught with challenges. By their very nature, rare indications have few patients and limited sample size. This scarcity of patients also results in a lack of available information and knowledge about the disease from the best endpoints, to the treatment effect size or the variability of response between subgroups.
"If you went to bed last night as an industrial company, you're going to wake up today as a software and analytics company," CEO of GE, Jeff Immelt
We are living in a new digital world which is evolving every day. Both personally and professionally, we rely on technology for many of our routine activities, and examples of digitization are prevalent across industries. Retail is a big example of how several chains have moved from physical stores to creating an online presence. In some years from now, people perhaps won’t have to learn driving as more self-driven cars will hit the road. Healthcare does not fall far behind in this race towards digitization.
In this blog, we will examine some of the different ways that digitization is set to shift the drug development paradigm.
In place of collecting data from patients recruited for a trial who have been assigned to the control or standard-of-care arm, an external control creates a comparator arm using either real-world data-sets such as electronic health records or previous clinical trials. The external control offers a practical, effective way to leverage real-world evidence and has been applied in regulatory approvals. In this blog, we share an illustrative example of how we can help customers in this emerging area of interest.
This article was originally published as part of a series by pharmaphorum in association with Cytel and is reproduced with their permission.
Ever felt like you ended up somewhere unexpected, not quite knowing what path you took to get there? This is the situation pharma companies can face without the right input from statisticians on clinical trial design – and the results can be very costly. As part of a series of articles in association with Cytel, pharmaphorum spoke with Natasa Rajicic about the vital role statisticians play in improving trial design and preventing expensive mistakes and trial failures.
In this blog, from our career perspectives series, we talk with Jayshree Garade Associate Director, Statistical Programming about her truly global career path at Cytel. Jayshree completed her Masters degree in 2006 and began her professional career with Cytel in Pune India. In the years to follow she supported high profile trials for a number of sponsors, before transferring to our offices in Massachusetts USA. She is currently leading a programming team, working remotely from her North Carolina home. Read on to learn more about life and opportunities at Cytel.
The term biomarker signature describes the behavior of a set of biomarkers that define a signature to maximize the prediction performance. We examine the behavior of specific biomarkers as a set that consistently fluctuate together to maximize the accuracy on predicting the disease-related outcome.
How we apply a biomarker signature depends on the prediction problem. A prognostic biomarker signature is used to predict the disease progression, a risk biomarker signature is used to identify sets of subjects that are likely to develop a disease, and a predictive biomarker signature is used to determine the patients that are likely to respond to a particular treatment. Predictive biomarker signatures are used often in oncology to stratify patients with a specific cancer into sub-populations and develop targeted therapies for the diseased population subtypes defined by the biomarker signature.
In this blog, we share an example project that our data science team has worked on supporting this work. The case study forms part of a new ebook 'Innovative Data Science and Real-World Analytics Approaches in Practice' and we are also delighted to provide the link for download as part of the article.
Health economics and adaptive design methods share common ground in that they both aim to support more efficient and accurate decision making that can enable faster patient access to new health technologies. However, to date, there has been a limited understanding of how, if at all, the two approaches are being used together.
A paper, “A Review of Clinical Trials With an Adaptive Design and Health Economic Analysis,” exploring this important topic was published in the April 2019 issue of Value in Health (1) . In this blog, we catch up with Laura Flight, National Institute for Health Research (NIHR) Doctoral Fellow and the primary author of the paper for a deep dive into the objectives of the publication, key findings and the next steps for promoting better understanding in this area.
Cytel recently hosted a very well-attended and engaging webinar on the topic of “Estimands, not just a statistical issue” presented by Paul Terrill, Associate Principal of Strategic Consulting at Cytel.
The webinar covered a range of issues from what is an estimand to how to structure early discussions on estimands.
In this blog, we are happy to share the replay of the webinar as well a summary of Q&As that arose on this very important topic. For an introduction to the topic, check out our previous blog post 'Estimands 101 with Mouna Akacha'.
By Nicolas Rouillé and Eric Henniger
The right design and the right data ultimately leads to the right decisions, so obtaining fit-for-purpose data, collected based on what your protocol is looking for is vital. However, there are several data pressure points facing oncology drug developers that need specialized expertise and processes to handle. In this blog, we run through some key aspects to consider to smooth your data collection and analysis.
The Cytel team made its annual trip to the PSI (Statisticians in the Pharmaceutical Industry) conference 2nd to 5th June. Taking place in London, UK, the theme of this year's meeting was Data-driven decision-making in medical research. As ever, the discussions both within the official conference agenda and during the networking breaks were engaging and productive.
In this blog, we share some of the particular highlights from the sessions that our team attended. We look forward to participating again in 2020 when the conference will return to Europe.
This article was originally published as part of a series by pharmaphorum in association with Cytel and is reproduced with their permission. Scott Harris, a four-time biotech Chief Medical Officer, and principal at Middleburg Consultants, a pharmaceutical consulting organization, told pharmaphorum’s Richard Staines that using novel adaptive or seamless clinical trial models can help to cut development costs. In doing so they can reduce the risks of trial failure that can spell the end for those biotech companies without the deep pockets of big pharma behind them.
In case you haven’t noticed, the traditional three-phase clinical development process is changing. While big late-stage trials are still pretty common, it’s also no longer a surprise to see sponsors refer to phase 1/2 trials, or phase 2/3, indicating that a smaller trial can be progressed to the next phase if an interim data readout supports further evaluation.
This is known as a “seamless” trial as the boundaries between each development stage have become less defined, and there are other options too.
Middleburg Consultants’ Scott Harris is a proponent of this new way of working and has personal experience of the approach after using it to steer a gastroenterology drug through the approval process.