
Perspectives on Enquiry & Evidence
Cytel's blog featuring the latest industry insights.

October 2, 2023
Patients’ adherence to the medications or treatment regimens prescribed to them by their clinicians is an important...
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September 8, 2023
Synthetic Control Arms: Leveraging Real-World Data for Comparison in Single-Arm Trials
Single-arm trials, unlike placebo-controlled randomized control trials, forgo the use of a placebo or standard-of-care...
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August 1, 2023
Dynamic Bayesian Borrowing to Bolster Limited Sample Sizes in Rare Indications
Evaluating the efficacy and safety of novel therapies in rare indications can be challenging due to the difficulty of...
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July 28, 2023
Embracing AI and ML in Medical Devices: FDA’s Total Product Lifecycle-Based Regulatory Framework
Written by Fei Tang, RWE Senior Research Consultant, and Paul Arora, Assistant Professor (Status), Dalla Lana School of...
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July 14, 2023
Real-Life Data-Sharing and EU Joint Clinical Assessments: Is Closing this Chasm a Mission Impossible?
Written by Grammati Sarri, David Smalbrugge, Andreas Freitag, and Evie Merinopoulou The vision of a single, centralized...
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May 22, 2023
New CADTH Guidance on RWE Is Now Available, but Critical Aspects Are Still Missing
By Grammati Sarri, Evie Merinopoulou, Vinusha Kalatharan, and Jason Simeone The Canadian Agency for Drugs and...
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May 10, 2023
FDA Increases Calls for Manufacturers to Ensure Trial Diversity, but Does It Fall Short of Addressing Health Inequalities in Product Development?
The evidence is staggering on the unequal health burdens experienced by specific patient groups defined by ethnic,...
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May 3, 2023
Reflections on the RCT DUPLICATE Study and Increasing Confidence in Real-World Evidence
With input by Alind Gupta, Louis Dron, and Jason Simeone. Randomized clinical trials (RCTs) have long been considered...
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April 25, 2023
How Target Trial Emulation Can Take the Guesswork Out of Comparative Effect Estimates in Medicare Drug Price Negotiation
An interview with Miguel Hernán, Harvard University Kolokotrones Professor of Biostatistics and Epidemiology On March...
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April 18, 2023
Navigating Comparative Effectiveness in the Inflation Reduction Act: Methodological Approaches for Healthcare Challenges
The Inflation Reduction Act (IRA), passed in August 2022, marks a significant shift in the US healthcare landscape,...
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April 14, 2023
Data Challenges (and Solutions) for Externally Controlled Trials
Real-world data and evidence are increasingly being used in health care decisions and publications. However, there are...
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March 21, 2023
Retrospective Claims Data Analysis Unlocks Discovery in Multiple Sclerosis Research
One of the lesser-known complications associated with Multiple Sclerosis is a higher risk of serious infections (SIs)....
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March 8, 2023
New Linked Database Expands Potential of RWE: A Unique Opportunity for Multiple Sclerosis Research
Real-world data has been increasingly used to answer questions related to the course, prognosis, and treatment of...
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March 7, 2023
Can RWE Help Restore Decades of Health Inequalities? Yes, and Here’s How
Health inequalities are an enduring issue that can be exacerbated by clinical trial recruitment that does not reflect...
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February 24, 2023
Demystifying Synthetic Control Arms
Last week, Cytel Director & Research Principal Louis Dron discussed new FDA guidance on the design and conduct of...
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February 14, 2023
FDA Guidance on the Design and Conduct of Externally Controlled Trials — What to Watch
The U.S. FDA has recently provided specific guidance[i] on the design and conduct of trials incorporating an external...
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January 31, 2023
Linked Data Studies: Improving the Way We Do Observational Research in Germany
Asthma affects more than 235 million people worldwide, and due to lacking effective implementation of clinical...
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December 29, 2022
Top Perspectives Articles of 2022
Perspectives on Enquiry and Evidence explores a wide variety of topics within clinical trial design and data science in...
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November 3, 2022
Cytel at ISPOR Europe: Two Workshops
Cytel will be represented at over 60 presentations at ISPOR Europe 2022, with more issue panels and workshops than any...
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October 3, 2022
Bayesian Methods for Historical Borrowing: Conjugate Priors
The wider availability of electronic health data, medical registries, and even larger proprietary datasets means that...
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September 28, 2022
Statistical Leaders and the Future of Drug Development
The landscape of drug development has changed dramatically over the last few decades, and effective statistical leaders...
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September 27, 2022
Data Capture and Data Sharing During the COVID-19 Pandemic
On Louis Dron et al., “Data Capture and Sharing in the COVID-19 Pandemic: A Cause for Concern,” The Lancet 4 (10) (2022)
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September 9, 2022
Developing Synthetic Control Arms Using Bayesian Models
A new trend has emerged over the last decade that has changed the way many clinical trials are conducted. Unlike...
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August 25, 2022
Cytel Presents at ICPE 2022
The International Society for Pharmacoepidemiology is hosting its “ICPE 2022: Advancing Pharmacoepidemiology and...
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July 27, 2022
Adaptive Designs Are Re-Defining Drug Development – Learn What's New
Written by Jing Ping Yeo and Charles Warne Adaptive designs are studies that “include a prospectively planned...
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July 26, 2022
7 Ways RWD Is Transforming Clinical Research
To watch this webinar and others from this introductory series, click the link below. The ability to draw on electronic...
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July 7, 2022
Using Quantitative Bias Analysis in Real World Data Strategy
The gold standard for assessing the efficacy for a medicine continues to be RCTs, however, for many reasons (disease...
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May 31, 2022
What it means to be a lead analyst on a Global COVID-19 Trial
The TOGETHER Trial for COVID-19 therapies, designed by clinical trial specialists at Cytel won the Society for Clinical...
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May 12, 2022
Estimator Choice in Synthetic Control Arm Analyses
Synthetic control arm (SCA) methods are statistical methods that are seeing rapidly increasing use in comparative...
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May 5, 2022
Interview with Grammati Sarri on Moderating an Issue Panel 2022
While randomized control trials remain the industry gold-standard for regulatory and reimbursement submissions, there...
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April 29, 2022
An Interview with Gabriel Tremblay on his Poster Contributions to ISPOR US 2022
At ISPOR US 2022, Cytel’s HEOR & RWE experts will be contributing to a range of Issue Panels, In-person Podium...
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April 13, 2022
Join Cytel’s HEOR Consultants at ISPOR US 2022
The Life Sciences landscape has seen an impactful digital evolution in the past two years. The pandemic has accelerated...
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January 20, 2022
Is Borrowing Outcomes from US or China the Future for Rare Conditions?
Limited patient populations resulting in small study sample sizes is a difficulty associated with the development of...
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October 21, 2021
Measuring Non-Adherence and Non-Persistence
A number of methods currently exist to measure non-adherence and non-persistence of medical therapies, for improved...
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October 7, 2021
Non-adherence and Non-persistence
‘Drugs do not work in patients who do not take them,’ said former surgeon general C. Everett Koop. Unfortunately, the...
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August 20, 2021
Using Causal Inference for Decision Making
In clinical research, decision makers need to choose among different courses of action every day. Whether it is...
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August 5, 2021
Establishing the Comparative effectiveness and Safety of New Treatments Using Real World Data
It is important to compare competing interventions to determine value of medicines, both from clinical and societal...
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July 1, 2021
Myths surrounding the use of synthetic control arms in clinical trials: Part 4
Synthetic control arms (SCAs) are best suited for situations when a single arm trial is run in a patient population...
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June 24, 2021
Myths surrounding the use of synthetic control arms in clinical trials: Part 3
Synthetic control arms (SCAs) leverage real world data from various sources or evaluations of historical clinical data...
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June 17, 2021
Myths surrounding the use of synthetic control arms in clinical trials: Part 2
In Part 2 of this four-part blog series, we bust another myth surrounding synthetic control arms (SCAs) used in...
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June 14, 2021
Practicalities of Integrating Electronic Health Records into Data
ACDM’s (Association for Clinical Data Management) eDigital data management expert group (DMEG) focuses on driving...
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June 10, 2021
Myths surrounding the use of synthetic control arms in clinical trials: Part 1
Over the past decade, single arm trials have emerged as an accepted way of assessing a new treatment intervention....
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June 3, 2021
Information Borrowing in Basket Trials
Bayesian methods allow for the incorporation of prior knowledge, in terms of either expert opinion from clinicians or...
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May 20, 2021
Quantitative Bias Analysis to Strengthen Comparative Effectiveness
As more payers and HTA agencies turn to real world data to compare the effectiveness of various treatment effects, two...
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April 21, 2021
Understanding Head-to-Head Clinical Trials
Two therapies are placed in head-to-head clinical trials when they are compared against each other as opposed to a...
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April 7, 2021
Career Perspectives: Interview with Neha Sati
Scientists at Cytel recently published a paper in the Journal of the American Medical Association (JAMA). Among the...
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March 26, 2021
Using External Evidence for Decision-Making for Medical Devices
Former Commissioner of the FDA, Dr. Scott Gottlieb, in several public presentations, would bemoan missed chances to...
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February 12, 2021
Leveraging Synthetic and External Control Arms Using Bayesian Methods
In recent times, Single arm trials are being increasingly used to assess new treatment interventions. They establish...
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February 9, 2021
New Meta-Analysis in JAMA Uses Novel Quantitative Techniques to Demonstrate Baseline Characteristics Informing Response to Common Therapy for Kidney Cancer
Recent years have witnessed improving survival outcomes for those struggling with a range of common kidney cancers....
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January 22, 2021
The Role of Real World Evidence after COVID19
COVID-19 has transformed the pharmaceutical industry in a manner that few could have predicted only a year ago. One of...
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November 24, 2020
Cytel and Ingress Health at Virtual ISPOR Europe 2020
Virtual ISPOR 2020, held November 16 to 19, presented new opportunities for scientific interaction amongst HEOR...
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November 17, 2020
Role of Prediction and Causal Inference in Clinical Research
As a part of Cytel’s "New Horizons Webinar Series", Alind Gupta, Senior Data Scientist, presents case studies from his...
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November 12, 2020
Join Cytel and Ingress Health at Virtual ISPOR Europe 2020
Cytel and Ingress Health (now a Cytel company) will be contributing to a range of events at Virtual ISPOR EUROPE 2020,...
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November 5, 2020
Role of RWA in Transforming Oncological Research
In oncology, many manufacturers go into niche indications, where there are very specific tumors, and then they opt for...
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November 3, 2020
Staffing Needs for RWE Delivery
When an expert statistician is paired with an experienced set of data managers, opportunities to capitalize on...
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October 22, 2020
An Interview with Bart Heeg (Part 2): New Trends in HEOR
In this two-part blog series, we interview Bart Heeg, Vice President HEOR and Founder at Ingress Health (A Cytel...
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October 20, 2020
Interview with Thomas Wilke: Health Economics/World Evidence Studies
In this interview with Thomas Wilke, Principal Scientist at Ingress-Health (a Cytel company), we talk to him about his...
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October 19, 2020
The Uniqueness of COVID-19 Data Challenges; The COVID-19 trial tracker
COVID-19 has created extreme uncertainties -- a dearth of historical information combined with the need for safety,...
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October 14, 2020
The Increasing Importance of Health Economics
A credible evidence base is needed to support and document the economic value of new technologies and therapeutic...
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October 13, 2020
Introducing Observational Studies – Three Trends for Statisticians
The combination of greater access to electronic health records, bigger electronic claims datasets, and the need for...
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October 7, 2020
RWE Needs for Natural History Studies
With the rise in digital technologies, there has been an explosion in the volume and type of data sources. We can...
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September 25, 2020
Use of External Controls in Clinical Development – Download Audiobook
Regulators in both the United States and Europe have responded positively to the use of synthetic control arms (SCA)s...
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September 22, 2020
LIBERTY claim analysis manuscript by Thomas Wilke and Sabrina Mueller
Research Scientists, Thomas Wilke and Sabrina Mueller recently published a manuscript on “Diabetes-Related...
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September 16, 2020
New Audiobook on Synthetic Control Arms
Synthetic control arms (SCA) are virtual trial arms that use historical claims data and observational data to simulate...
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September 10, 2020
Does your Trial need a Synthetic Control Arm?
Single arm trials are emerging as an accepted way of assessing a new treatment intervention. They establish clinical...
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August 7, 2020
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...
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August 5, 2020
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...
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July 23, 2020
Why Consider a Synthetic or External Control Arm?
Just as there are numerous adaptations that fall within the umbrella of adaptive designs, there are several different...
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July 21, 2020
Head to Head Comparisons Using Real World Data
Cytel is conducting a webinar series that focuses on target trial emulation and causal inference approaches using real...
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June 23, 2020
Design and Data Source Considerations from Pilot Investigations in CVD
Supposing two treatments, A and B, need to be compared that have not been compared through a clinical trial. In the...
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May 22, 2020
4 Things You Need to Know about COVID-19 Trial Designs
The Cytel Trial Tracker now features summary plots that display trials by country, trial status and study design. This...
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April 29, 2020
Webinar: Transparent Machine Learning in Oncology
In our previous blog, we spoke with Alind Gupta, who works as a Machine Learning Researcher at Cytel in Canada. The...
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April 20, 2020
Interview with Alind Gupta: Transparent Machine Learning in Oncology
Cytel is hosting a webinar on Transparent Machine Learning in Oncology, on April 21, 2020. Our speaker, Alind Gupta,...
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