Inability to Enroll Patients Terminates More Than a Quarter of Rare Disease Drug Trials

Rare Daily Staff

More than a quarter of rare diseases trials were terminated between 2016 and 2020 due to low accrual rates, according to research by the data and analytics company GlobalData.

Low accrual rate was the most common reason for trial termination seen in the research by GlobalData, at 26 percent of trials, with other issues such as lack of efficacy (12 percent), business and strategic decisions (6 percent), and product discontinuation (6 percent). The findings were based on a study of 736 clinical trials.

GlobalData said the difficulties with patient recruitment for rare disease clinical trials could be mitigated by the use of virtual trials.

“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,” said Kitty Whitney, director of thematic analysis at GlobalData. “Virtual or decentralized 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 disease 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 more 565 percent.

Clinical trials for rare diseases face numerous challenges that can lead to insufficient trial recruitment and low patient retention. This includes the difficulties in identifying suitable participants due to a small patient pool, the 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,” said Whitney. “More solutions need to be implemented that could essentially bring the trials to the patients.”