Why use real-world data?

All clinical trials have guidelines, called eligibility criteria, about who can participate. The criteria ensure that only a selected set of people are participated. When the drugs are released in the market, the findings of the selected participants are generalized to the much bigger public. It is thus not uncommon for drugs to behave differently in the real world than in clinical trials.

During the past 30 years, special regulatory programs are available to the FDA including Fast Track (1988, in statute 1997), Priority Review (1992), Accelerated Approval (1992), and Breakthrough Therapy designation (2012). These programs potentially contribute to the variability of the quantity and quality of evidence supporting recent drug approvals.

All these suggest an ongoing need for continued evaluation of therapeutic safety and efficacy after drug approvals. The 21st Century Cures Act, passed in 2016, places additional focus on the use of real-world data to support regulatory decision making. The FDA (more info) has started to use real-world data to monitor postmarket safety and adverse events, and payers to support value-based payment program.

eHealthMe has been monitoring drugs and supplements since 2008. Results from our real-world drug safety study have been referenced on 600+ medical publications, including The Lancet, Mayo Clinic Proceedings, and Nature.