Often, it takes years for new medicines to gain approval simply because there are not enough people to participate in trials. This is an especially big challenge in rare diseases, where the combination of few patients, and even fewer who are willing to take the chance of receiving the control treatment, makes randomised trials difficult to enroll.
Fortunately, we are at the cusp of a data revolution. Clinical records from real patients offer a valuable source of quantitative information on the effectiveness of standard treatments. Roche is exploring ways to use this real-world data (RWD) to help get life-saving treatments to patients, faster.
Real-World Data: Data that can inform on health status, like electronic health records, claims and billing activities, product and disease registries, surveys, and data from mobile apps.
In one example for a rare cancer, it would have taken years to enroll enough patients for both the treatment and control group of a clinical trial. Instead, we worked with Flatiron Health to use RWD to form the control group, reducing the time required to perform the trial. An approach like this has the potential to help accelerate an approval process that often takes years.