More than a quarter of rare disease trials are culled due to low patient rates: report

Rare disease drugs are a gold mine for biopharmas if they are approved, often becoming some of the most expensive medicines available and catering to tiny numbers of people.

Getting them through trials is not always easy given these small patient populations, and a new report out by Global Data, which sifted through more than 700 studies, has found more than one-quarter of rare disease trials were terminated between 2016 and 2020 due to low accrual rates.

This was not the only issue, however; a lack of efficacy, business and strategic decisions, and product discontinuation derailed 12%, 6% and 6% of studies, respectively.

The solution to the recruitment problem? The data analytics and life science consultancy firm says use virtual or siteless trials, a growing new trend that has boomed since the pandemic.

“While COVID-19 accelerated the use of virtual trials, the shift from traditional models was already underway well before the pandemic,” said Kitty Whitney, director of thematic analysis at GlobalData.

“The pharma industry must now take advantage of this wave of innovation and make virtual trials a standard part of clinical development. 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.”

“According to Global Genes, there are thought to be approximately 7,000 rare diseases affecting over 400 million individuals globally,” Whitney added.

“However, 95% of these conditions do not have an FDA-approved treatment, representing a significant unmet need for these patients.

“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.”