2017 ASA Biopharmaceutical Section Regulatory-Industry Statistics Workshop

Posted by Cytel

Oct 24, 2017 6:01:00 AM

The ASA Biopharmaceutical Section Regulatory-Industry Statistics Workshop is sponsored by the ASA Biopharmaceutical Section in cooperation with the FDA Statistical Association. Each year 800 statistical practitioners come together to absorb new information on statistical practices in all areas regulated by the FDA.

Cytel was honored to be involved in the workshop program, and our subject matter experts added value to the conference by sharing their academic and regulatory experiences.

Don’t worry if you missed the event!

In this blog, we share the full slide set slide from Cytel contributions at the ASA Biopharmaceutical Section Regulatory Industry Statistics Workshop.

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Topics: Sample Size, Promising Zone, Clinical Research Services, Regulation, FDA, Clinical Data, Adaptive Clinical Trials, adaptive sample size re-estimation, adaptive designs, Bayesian

Adaptive Designs: In Conversation with the NEJM

Posted by Cytel

Aug 17, 2016 11:57:43 AM

Following the recent publication of their review article Adaptive Designs for Clinical Trials in the New England Journal of Medicine,  co-authors Cyrus Mehta ( President and Co-Founder of Cytel, and Adjunct Professor of Biostatistics at Harvard University) and Deepak L. Bhatt M.D C M.P.H. (Executive Director of Interventional Cardiovascular Programs, Brigham and Women’s Hospital Heart and Vascular Center) were invited to participate in a live video discussion with the journal. 

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Topics: Cyrus Mehta, Promising Zone, biostatistics, adaptive designs, adaptive trials

Why You Should Not Power for Superiority Upfront: Promising Zone Clinical Trials with "Adaptive Switch"

Posted by Esha Senchaudhuri

Jun 26, 2015 12:00:06 PM

Powering a trial for superiority can be financially risky. In some instances it may also prove unnecessary.

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Topics: Cyrus Mehta, Promising Zone, Cardiovascular, Adaptive Clinical Trials, Adaptive Finance

Statistical and Operational Challenges of the VALOR Trial: Mehta on the Promising Zone

Posted by Esha Senchaudhuri

Mar 26, 2015 4:00:00 PM

Last year Sunesis completed the VALOR trial, the first clinical study to make use of the groundbreaking promising zone design. The promising zone design implements an unblinded sample re-estimation after an interim look, but only if conditional power during the interim look falls within a designated promising zone. Although the VALOR trial did not confirm the efficacy of the new therapeutic, it provided the drug with the best possible chance to prove its efficacy.

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Topics: Promising Zone, Adaptive Clinical Trials, Statistical Innovations in Clinical Development

How to Shorten a Cardiovascular Outcome Trial By Two Years

Posted by Esha Senchaudhuri

Feb 10, 2015 5:28:00 PM

Cardiovascular outcome trials (CVOTs) have earned the reputation of being the untamable behemoths of the clinical world. Needless to say these trials are long and require extremely large sample-sizes. The Contrave LIGHT study required 8900 patients. The SAVOR TIMI trial enrolled 16,492 patients. Even the EXAMINE trial, which benefited from a promising zone design, required 650 patients. 

However, since the explosive controversy over the FDA’s conditional approval of anti-obesity drug Contrave four years ago, there is much we have learned about how to make these trials shorter while also diminishing the financial risks of investing in them. For example, one of our clients managed to shorten the expected study length of an a CVOT by two years using a four point MACE Assessment (see below). 

In this post, we explore some of the lessons we have learned when designing these large-scale clinical trials.  

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Topics: Cyrus Mehta, Promising Zone, Cytel Strategic Consulting, Interim Analyses, Program and Portfolio Optimization, Cardiovascular, Clinical Development Strategy, Adaptive Clinical Trials

5 Scenarios When ‘Keep it Simple’ May Be Bad Advice for Clinical Trial Designers

Posted by Esha Senchaudhuri

Sep 18, 2014 10:44:00 AM

When designing clinical trials, many trial designers are advised to keep the trial simple. Prima facie, the keep it simple principle seems like sound advice. There are various logistical uncertainties that arise when implementing a clinical trial, and the more simple a trial – so conventional wisdom says – the easier it is to respond to these uncertainties.

According to Zoran Antonijevic, a Senior Director at Cytel Consulting, there is reason to doubt such conventional wisdom. After all, flexibility is hardly a virtue of a traditional trial design. Simple designs may seem to make it easier to monitor data and report results. However, a flexible design can better address remaining uncertainties in product development. These uncertainties are related to treatment effect, dose selection, or a sub-population that would experience the best benefit/risk from the treatment.

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Topics: Oncology, Promising Zone, sample size re-estimation, Enrichment, Cytel Consulting, Efficacy, Interim Analyses, forecasting, optimization, Program and Portfolio Optimization, R&D, Adaptive Clinical Trials

FDA-Industry Session on Cardiovascular Outcome Trials: Mehta on EXAMINE Trial’s Promising Zone Design

Posted by Esha Senchaudhuri

Sep 9, 2014 10:41:00 AM


The FDA requires sponsors of new antidiabetic drugs to conduct cardiovascular outcome trials (CVOTs). CVOTs demonstrate that new therapies do not place unacceptable cardiovascular risk on patients suffering from Type 2 diabetes. The average CVOT requires about 5000 patients and takes an average of 5 years to complete. However, a recent white paper by the Cardiac Safety Research Consortium outlines a variety of methods to decrease sample size and study duration, by employing group sequential and adaptive CVOT designs. 

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Topics: Cyrus Mehta, Promising Zone, Trial Design, FDA, Interim Analyses, Cardiovascular, Adaptive Clinical Trials

Impact of Study Design and Development Strategy on Pharmaceutical Programs and Portfolios

Posted by Esha Senchaudhuri

Sep 2, 2014 11:19:00 AM

As more clinical trials make use of adaptive designs, investors have come to realize that high quality trial designs can result in significant improvements to a trial’s financial risk profile. Regardless of a trial’s eventual success or failure, a well-constructed design provides a drug with the highest possible probability of success while mitigating financial risk.

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Topics: Nitin Patel, Cyrus Mehta, Promising Zone, Trial Quality, Cytel Strategic Consulting, Trial Design, Entrepreneurship, Program and Portfolio Optimization, R&D, Adaptive Clinical Trials

Why adaptive sample size re-estimation designs preserve type 1 error

Posted by Esha Senchaudhuri

Aug 28, 2014 8:00:00 AM


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Topics: Promising Zone, type 1 error, Adaptive Clinical Trials

De-Risking Drug Development using Adaptive Design

Posted by Cytel

May 13, 2014 8:31:00 AM

The VALOR trial recently applied a promising zone design to a Phase 3 evaluation of Vosaroxin, a candidate for the treatment of relapsed/refractory acute myeloid leukemia. CMO Dr. Adam Craig reports that there were intial anxieties about financing a trial that required 800 patients. However, a promising interim look raised investors' confidence in the treatment's success, leading to a high-powered trial of 712 patients.


To view a YouTube video of Dr. Adam Craig talking about the Phase 3 VALOR trial, click below.  

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Topics: Oncology, Promising Zone, sample size re-estimation, Movers & Shapers, Adaptive Clinical Trials

The Perils of Poor Recruitment

Posted by Esha Senchaudhuri

May 5, 2014 10:21:00 PM

A new JAMA study on discontinued randomized trials in Switzerland, Germany and Canada, reports that poor recruitment accounts for 101 out of 253 trials that were eventually discontinued (or about 10% of the 1017 trials which participated in the study). When restricted to industry-sponsored trials with non-healthy volunteers, poor recruitment accounted for the discontinuation of 40 trials out of 119 that were discontinued. Across the board, poor recruitment was the foremost cause of trial discontinuity.

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Topics: Nitin Patel, East, East 6.3, Access to Slides PDF, Recruitment, East Recruit, Sample Size, Promising Zone, sample size re-estimation, Adaptive Clinical Trials

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