As any seasoned expert in the clinical trials field would affirm, it is not enough to have an innovative therapeutic in the works; designing a clinical trial to prove its promise and then using the right tools to capture the necessary data are crucial.

At two upcoming events—the Clinical Trials in Rare Diseases Europe 2024 and the Clinical Data Management Innovation Europe 2024—experts are set to discuss the latest trends clinical trials operations. Both events are taking place simultaneously 19–20 November in Brussels, Belgium.

As interest in rare disease drug development has grown in recent years, a couple of themes have emerged that are particularly relevant to orphan drug trials.

Unlike conventional studies for common conditions, rare disease trials have to contend with fewer patients who can be difficult to find. Real world evidence then becomes even more essential in showing the efficacy of a new treatment as pharma companies try innovative trial designs that use this data while putting together regulatory packages. On the first day at the rare-diseases-focused event, Vinciane Pirard, Scientific Advocacy and Insights, Medical Affairs Rare Diseases at Sanofi will give a talk on identifying the right kind of evidence needed in a data package for regulators, and payers after a potential approval, especially in the context of rare diseases.

Taking this a step further, a subsequent panel, which includes Pirard and representatives from MSD and Roche, will explore whether clinical trials are even needed for developing rare disease treatments, and how alternatives to placebo arms should be tackled.

Another growing theme in rare diseases has been the importance of giving room to the patient voice. Patient advocates from the Leber's Hereditary Optic Neuropathy Society, Roel Rotterdam & Patient Experience Association, Rare Disease Diversity Coalition will explore how clinical trials can be more inclusive toward participating patients and the role of advisory boards.

At the parallel event, executives from leading pharma companies like Pfizer and AstraZeneca will talk about the ever-increasing integration of artificial intelligence (AI) into clinical data management, and ways to pursue a data-driven approach when it comes to using new technologies. While innovative technologies get introduced each day, a subsequent panel will explore how the ability to do effective scale-up will help decide the most impactful new technology.

On the second day, gathered experts will continue sharing their own experience in navigating logistical questions with clinical trials. Rare disease clinical trials often lack a diverse group of patients partly because most of this research is centred around large academic institutions in select countries, and Elizabeth Thomson, a rare disease clinical trial expert will present the case for why sponsors need to recruit from countries other than the US and the five largest European Union countries. Jenifer Waldrop, Executive Director, Rare Disease Diversity Coalition, will lead a talk on identifying and highlighting underrepresented individuals and ways to support them more fully.

Alongside these sessions, at the data management-focused event, representatives from Eli Lilly, GSK and GlobalData will discuss the implementation of risk-based strategies that can help improve data management for small and mid-sized biotechs.

A detailed agenda for the Clinical Trials in Rare Diseases Europe 2024 can be found here, and the agenda for Clinical Data Management Innovation Europe 2024 can be accessed here.

The conference is hosted by Arena International Events Group, a B2B events company owned by GlobalData, the parent company of Clinical Trials Arena and Pharmaceutical Technology.