What’s Next for External Controls | MIT Technology Review & Medidata Solutions

Written in partnership with MIT Technology Review

External control arms have potential applications after regulatory approval of a product as well, says Ruthie Davi, vice president of data science, Acorn AI at Medidata. For example, once a treatment is on the market, insurance companies may want to compare it with other treatments for the same condition to determine whether it’s cost-effective and whether they will cover it. “The payers may want a comparison to a different study therapy than was used in the randomized controlled trials conducted for regulatory approval. Then the sponsor is faced with having to do a randomized controlled trial against that other therapy,” with all the time and expense associated with such trials, Davi says. The sponsor might accomplish the same objective much more efficiently with an external control arm, mining Medidata’s database for a matched set of patients treated with the other therapy.

Nicholas Borys, chief medical officer for biotechnology company Celsion, points out that historical data will also have diminishing validity as care standards change and older data eventually stops being useful for studying certain things. The performance of a new treatment is generally measured against the current standard of care. The care received by a patient in a historical control arm a few years ago may be substantially different from the care that today’s control patient is getting. While the new treatment may perform well by 2015 standards, it needs to perform well by 2021 standards to represent an improvement. A true breakthrough, such as some cutting-edge immunotherapies, can transform the standard of care very quickly and render older patient data irrelevant for many types of studies. “But if you’re careful about choosing the endpoints, you still might be able to use the data for some things,” Borys adds. And with tens of thousands of clinical trials in progress at any given time, there is always a steady supply of new data.

Beyond the benefits of using external control arms in a clinical trial, Borys also sees potential in being able to track patients after the trial is over: an ability that Medidata can offer. “You can get a better sense of what side effects are really going on in the real world, or how well patients are following up with their treatment, or what are the real costs of treating a patient,” he says. “That kind of information is hard to determine in a clinical trial environment, and it could really help us understand how our drugs would fit into a real-world paradigm.”

But the greatest value Borys sees currently is in helping to point development of all kinds of treatments in the directions most likely to be successful. “Right now, we really don’t know if a drug is showing a benefit until we’ve given it to a few hundred patients,” he says. “If we have an external control arm, we can get a pretty good signal with a fraction of those numbers.”

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