According to the FDA, real-world evidence (RWE) is clinical evidence regarding the usage, potential benefits, or risks of a medical product derived from an analysis of real-world data. RWE trials are crucial for further understanding newly approved medicinal products in the real-world setting. These trials provide clinical evidence of the long term safety and efficacy of these drugs from real-world data where patients are treated using these drugs. RWE trials mainly involve observational data outside the traditional involvement of randomized controlled trials. Using real-world data is an integral part of RWE trials as it enables researchers to monitor and assess patient outcomes and treatment methods. It also allows for the effects of a medicinal product to be investigated over a long period of time. Information for RWE trials may be sourced from clinical trial registries, patient-generated data, electronic health records, wearable technology, and mobile devices, among others.

Currently, the number of real-world evidence trials has increased for 2021. Using the GlobalData Clinical Trials database, it was found that there are currently 194 real-world evidence trials, the highest annual number recorded so far, followed by 2019, which had 160 trials. There has been a consecutive trend for the rise in RWE trials, except for 2020 where there was a small decrease in trial count, as observed in Figure 1. The decrease in value for trials in 2020 could be attributed to the Covid-19 pandemic, as many studies were delayed due to issues with patient recruitment and site closures because of global lockdown measures. Out of the current trials for 2021, 51.5% are ongoing and 47.9% are planned.

Figure 1: RWE Trials by Year. Source: GlobalData, Pharma Intelligence Centre (Accessed November 2021)

Real-world evidence studies are becoming increasingly popular, mainly due to the efficiency and accuracy of real-world data. RWE studies have been shown to aid in clinical planning, participant recruitment, trial design, and post-marketing surveillance. By using real-world data from virtual trials, synthetic control arms can be developed for rare therapy areas/indications. Healthcare costs can also be reduced by patient personalization, early diagnosis, and remote patient monitoring within trials. Regulatory authorities like the FDA and European Medicines Agency (EMA) are actively promoting the use of RWE trials; in December 2018, the FDA released framework for the RWE program, using real-world data and RWE to help approval decision making for drugs and biologics. Additionally, the EMA recently launched framework contracts with academic and research institutions to conduct EMA-funded efficacy or safety research. Since the beginning of the COVID-19 pandemic, real-world data have been used in EU trials to test and monitor potential treatments.