Cytel President Explains Benefits of Adaptive Trial Design

Published: Aug 09, 2016

Cytel President Explains Benefits of Adaptive Trial Design August 9, 2016
By Alex Keown, Breaking News Staff

CAMBRIDGE, Mass. – Traditionally designed clinical trials can hinder efficiency in drug development. Modern clinical trials could benefit from an adaptive design that would allow for changes to be made during the course of a trial, a new article in the New England Journal of Medicine suggests.

The article, “Adaptive Designs for Clinical Trials,” was co-authored by Deepak L. Bhatt, a professor of medicine at Harvard Medical School and Cyrus Mehta, president of Massachusetts-based Cytel. In their article, the authors argue that adaptive designs are ways for clinicians to address limitations of randomized controlled trials, having the potential to increase efficiency, deliver benefits to trial participants and future patients and improve the likelihood of finding a true benefit of a therapy.”

In an exclusive interview with BioSpace, Mehta said many clinical trials have failed in the past due to poor designs. Describing traditional trial methods as a box, Mehta said clinicians have an initial plan of what to do with a population, sample size and end points. Patients would be enrolled and accruing data would not be examined until it was too late. Using an adaptive trial design, Mehta said accruing data can be examined along the way, providing clinicians the ability to make changes to the future course of the trial—in a controlled manner that won’t allow it to be compromised, he said.

In 2010, the U.S. Food and Drug Administration issued guidance on adaptive trial designs. Since that time Cytel, which specializes in adaptive designs, has run “several hundred” trials.

Cytel began as a design software company in the 1980s, but moved into creating software that helps companies with clinical trials. Cytel’s software specializes in designing adaptive studies for clinical trials. Over the years the company made a name for itself in clinical research and has now become a “go to” organization for companies needing assistance with their clinical research, in part due to the company’s reputation for statistical work and software.

During an hour long conversation, Mehta outlined several instances when adaptive trial designs would be beneficial, including in oncological trials where he said adaptive designs would be help as it would allow researchers to shift strategies during a trial. Such options will allow clinical researchers to end a trial early due to futility, something that would ultimately save companies a lot of money, as well as stopping a trial early due to positive efficacy, Mehta said.

Using such a design means that having the right dosage, population and sample size will allow drug companies to get stronger and more positive outcomes—that is if the drug works, he said.

“If the drug doesn’t work, there’s nothing you can do to save it,” Mehta quipped.

The article in the New England Journal of Medicine uses case studies to show the benefits of adaptive clinical trials and the different kinds of adaptive designs that are available for clinicians. Mehta said the article is written clearly so a non-statistician will be able to grasp the nuances of adaptive trial designs and adopt them into their own research. Mehta said he hopes clinicians who read the article will be influenced to implement adaptive designs into their clinical research, which he said will ultimately benefit patients.

“Anyone can read the article and understand the benefits as well as the operational issues that had to be dealt with (in the examples cited),” Mehta said.

However, Mehta said the adaptive design trial is a strategy that some have balked at using due to lack of understanding and fear of regulatory disapproval. He said many clinicians more familiar with the regulatory side of things have a desire to “play it safe,” but statisticians have a full grasp on the goal. He said companies that use more statisticians are likely to adopt an adaptive trial design.

Since the article was published, Mehta said he has received positive feedback, which he hopes will not only benefit clinical trial designs, but also boost Cytel’s place in adaptive trial designs.

“We are presenting a methodology that will infiltrate into the clinical trial world,” Mehta said.

That methodology isn’t bad for a company that started in Mehta’s apartment more than 27 years ago, but has since then morphed into a company with more than 600 employees. And the company has not finished growing. Mehta said he anticipates hiring another 100 employees before the end of the year.

“It’s really been quite amazing to see,” Mehta said of his company’s growth.

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