Over a quarter of rare diseases trials were terminated between 2016 and 2020* due to low accrual rates, according to research by GlobalData. Difficulties with patient recruitment for rare disease clinical trials could be mitigated by the use of virtual trials.

Low accrual rate was the most common reason for trial termination seen in the research by GlobalData, at 26 per cent of trials, with other issues such as lack of efficacy, business and strategic decisions, and product discontinuation seeing 12 per cent, 6 per cent and 6 per cent respectively.

Kitty Whitney, Director of Thematic Analysis at GlobalData, comments, “While COVID-19 accelerated the use of virtual trials, the shift from traditional models was already underway well before the pandemic. The pharma industry must now take advantage of this wave of innovation and make virtual trials a standard part of clinical development. Virtual or decentralised trials represent a viable and more patient-centric solution to these issues, assisted by ongoing advances in remote monitoring tools, data collection technologies, patient engagement platforms, and wearable devices and digital biomarkers, as well as the roll-out of 5G.”

GlobalData’s Clinical Trial Intelligence database reveals that the number of rare diseases clinical trials has increased significantly over the past 20 years, rising from almost 750 in 2001 to just under 5,000 in 2020 – an increase of over 565 per cent.

Whitney continues, “According to Global Genes, there are thought to be approximately 7,000 rare diseases affecting over 400 million individuals globally. However, 95 per cent of these conditions do not have an FDA-approved treatment, representing a significant unmet need for these patients.”

She adds, “Clinical evaluation of pipeline treatments for rare diseases face numerous challenges leading to insufficient trial recruitment and low patient retention. This includes identifying suitable participants due to a small patient pool, geographic spread of patients, and the physical challenges preventing many individuals from getting to trial sites. It is important to highlight the ongoing struggles that patients with rare conditions face, many of whom are desperate to participate in studies investigating potentially life-saving therapies. More solutions need to be implemented that could essentially bring the trials to the patients.”

* Based on research of 736 trials