Why Use an External Control Arm? | MIT Technology Review & Medidata Solutions

Written in partnership with MIT Technology Review 

It’s easy to see the recruitment problem for investigators studying therapies for cancer and other deadly diseases: Patients with a life-threatening condition need help now. While they might be willing to take a risk on a new treatment, “the last thing they want is to be randomized to a control arm,” says Nicholas Borys, chief medical officer for biotechnology company Celsion. Combine that reluctance with the need to recruit patients who have relatively rare diseases — for example, a form of breast cancer characterized by a specific genetic marker — and the time to recruit enough people can stretch out for months, or even years. Nine out of 10 clinical trials worldwide — not just for cancer but for all types of conditions — can’t recruit enough people within their target time frames. Some trials fail altogether for lack of enough participants. 

What if researchers didn’t need to recruit a control group at all and could offer the experimental treatment to everyone who agreed to be in the study? Celsion is exploring such an approach with Medidata. Medidata has compiled an enormous “big data” resource: Detailed information from more than 24,000 trials and nearly 7 million patients going back about 10 years. 

The idea is to reuse data from patients in past trials to create “external control arms.” These groups serve the same function as traditional control arms, but they can be used in settings where a control group is difficult to recruit: For extremely rare diseases, for example, or conditions such as cancer, which are imminently life-threatening. They can also be used effectively for “single-arm” trials, which make a control group impractical: For example, to measure the effectiveness of an implanted device or a surgical procedure. Perhaps their most valuable immediate use is for doing rapid preliminary trials, to evaluate whether a treatment is worth pursuing to the point of a full clinical trial. 

For mid-sized and emerging biopharma companies, Medidata’s 20+ years of expertise, dedicated support, and continuous learning with customers and trials of all sizes help you stay on the path to success.

Download our white paper with MIT Technology Review, Clinical Trials are Better, Faster, Cheaper with Big Data, for a comprehensive guide to external control arms in clinical trials.