Real World Evidence (RWE)

On the international therapeutic agents scene, the use of real-world evidence (RWE) to support medicinal product authorisation applications is of growing importance, including for veterinary medicinal products.

Recent applications for pet (and, less often, livestock) treatments show that specific diseases should be treated in subgroups; however, it is difficult to find a large number of animals and group them into randomised controlled trials (RCTs) in these cases. Sufficiently meaningful RCTs with small target populations are thus a challenge, and the use of real world data (RWD)/RWE may be of interest. Similarly, the use of RWE can provide valuable therapeutic insights into the use of medicinal products in underrepresented groups.

In addition, RWD/RWE has proven to be useful for regulatory decision-making, particularly in connection with rare events, the optimisation of approved therapeutic regimens (e.g. treatments with antimicrobial active substances), monitoring resistance and interpreting safety signals. There are, however, a number of challenges associated with generating RWE from RWD that must be solved at a scientific and regulatory level. Particular attention needs to be paid to the issue of complete source data and the risk of selection bias. There is also the risk of unintentional manipulation of the results by repeated analysis of (part of) the same RWD.

Swissmedic actively monitors international developments with regard to the regulation and application of RWD/RWE (e.g. EMA Big Data). Future experience will show whether – and, if so, which – new standards will be required to ensure that the established use of RWE in medicinal product review is harmonised internationally.