Upcoming US legislation, known as the 21st Century Cures Act, will incorporate more of the patient experience into drug development. There will be a focus on structured collection of data from patients about the personal burden of their disease, as well as the issues that matter most to them as pharmaceutical companies strive for new treatments.
In line with this, while randomized controlled clinical trials will remain the gold standard for assessing the safety and efficacy of investigational new drugs, this new law will give the FDA the ability to review alternative measures of drug efficacy, i.e. Real World Data (RWD). This includes, but is not limited to, electronic health records, pragmatic clinical trials, patient registries, observational studies, Expanded Access Programs (EAPs), surveys, and data collected via social media, smart phones, and wearable devices.
Collecting and utilizing data from normal clinical practices, or from settings that reflect the reality of day-to-day healthcare delivery, is set to become increasingly important in the drug approval process. This is because clinical trial data, alone, often does not accurately reflect how a drug would affect the larger patient population in the real-world setting, due to the strict inclusion and exclusion criteria. For example, elderly patients, patients with an especially severe or rare case of a disease, or patients using concomitant medication are often excluded from trials. RWD can, therefore, be used to understand how these excluded individuals react to a drug, which will provide a much broader insight into the how a product will affect the heterogeneous patient population outside of the clinical trial setting.
This broader insight into the patient population can be beneficial as it leads to:
- Greater understanding of the varied patient journeys, treatment pathways, and levels of drug effectiveness
- More effective drug development programs
- Identification of populations with enhanced benefit/risk
- Minimization of the number of patients exposed to less efficacious therapy
- Expansion of the safety profiles of a drug
- Development of evidence to support expanding the indication for a drug
It remains to be seen how the Trump administration will incorporate the use of real world data into its overall healthcare agenda. But there have been some early signs that the new US president is eager to get new drugs to market quicker, and RWD can definitely be used to help achieve this.
