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 randomised controlled trials. Using real-world data is an integral part of RWE trials as it allows 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. Information for RWE trials may be sourced from clinical trial registries, patient-generated data, electronic health records, wearable technology, and mobile devices, among others.

According to GlobalData’s Clinical Trials Database, 2021 has the highest annual amount of real-world evidence trials trials. For 2024, the current number of RWE trials stands at 331 trials and is set to surpass 336 trials as the year is still ongoing.
According to an analysis of the different trial statuses of RWE trials, 53.9% of trials were completed, 22.1% of trials were ongoing and recruiting, 8.1% of trials were planned, 7.0% of trials were ongoing but not recruiting, and 8.2% of trials were suspended/terminated/withdrawn (S/T/W). When looking at geographies, China had 29.9% of the global total number of RWE trials. This was followed by Italy with 10.1%, Germany with 9.8%, the US with 9.2%, and Japan with 8.3%.

RWE 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 using patient personalisation, early diagnosis, and remote patient monitoring within trials.

Regulatory authorities such as the FDA and EMA are actively promoting the use of RWE trials. In December 2018, the FDA released a framework for the RWE programme, 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 has been used in EU trials to test and monitor potential treatments.