4th School for Clinical Trialists @ ECCO'21
Peter Bossuyt, ClinCom Member
During the upcoming ECCO Virtual Congress, ClinCom is organising the 4th School for Clinical Trialists. One of ClinCom’s aims is to increase the quality of clinical trials in IBD and to provide new insights into the fast-moving field of clinical trial development. Randomised clinical trials (RCTs) are still the gold standard for the development and registration of new treatment options.
However, RCTs are facing a variety of hurdles. Currently (and fortunately for patients with IBD), there is huge competition in the recruitment to clinical trials with new compounds. Multiple trials with IL-23 antagonists, S1P1 agonists and JAK inhibitors are currently ongoing. This, in addition to the continuing COVID-19 pandemic, is causing some delays in the timelines of these trials. Another important issue with RCTs is the difficulty in assessing adequately the safety of a new drug compound. Patients who are enrolled in an RCT do not necessarily represent the overall IBD population. This is why ClinCom has decided to focus on real-world data during the 4th School for Clinical Trialists. Real-world data are not sufficiently valid to allow assessment of the treatment effect of a new compound for registration purposes. However, they do reflect the efficacy and safety of a compound in the overall IBD population.
The first session of the School for Clinical Trialists will focus on efficacy. It is essential that we have a clear view on what we expect from real-world efficacy data. Can we just copy-paste the endpoints from RCTs to real-world studies? We will explore the different viewpoints on how we define efficacy from a patient’s and a physician’s perspective. We will also raise the provocative question of whether RCTs are needed, as these trials are costly and time-consuming. Newer methods, including big data evaluations, can provide answers where RCTs fail. Finally, we will touch on the hot topic of personalised prediction models. A statistician and a methodologist will debate how they would use real-world data in the prediction of efficacy for personalised medicine.
In the second session we will discuss the safety data derived from real-world studies. Currently, most safety data come from post-marketing registries or pharmacovigilance databases. In recent decades this has helped us to detect important safety signals, but is this traditional approach still valid? The majority of safety data are obtained from pharma-driven initiatives, but investigator-driven research has also impacted substantially on knowledge of the safety profiles of IBD drugs, which in the end all have to be communicated to the patient. With this in mind, we will additionally provide tips and tricks in how to translate these safety data to the patients in a balanced and realistic way.
The 4th School for Clinical Trialists is open to all physicians, researchers, IBD Nurses and clinical trial coordinators with an interest in high-quality research in IBD. We hope to meet you online in July.