Interest in rare disease trial participation is at an all-time high even as the number of trials to treat rare diseases has ballooned, according to two new studies, but enrollment remains difficult.

The National Organization for Rare Disorders (NORD) reports that 88 percent of people living with a rare disease would use an investigational treatment for their condition. At the same time, data from analysis firm GlobalData show the number of rare disease clinical trials increased to almost 5,000 in 2020, up from almost 750 in 2001 — an increase of more than 565 percent.

Despite the growth in trial participation interest and trial activity, more than a quarter of clinical trials targeting rare diseases between 2016 and 2020 had to be terminated early due to low patient enrollment, GlobalData claims.

GlobalData’s report cited low enrollment as the most common reason for terminating a trial early, with 26 percent of 736 trials included in the firm’s analysis halted for that reason. Other reasons for early termination included lack of efficacy (12 percent), business and strategic decisions (6 percent) and product discontinuation (6 percent).

NORD’s study, which compared rare disease trial data from 2019 to those collected 30 years earlier, shows only 62 percent of respondents willing to participate in a trial in 1988. The number of people participating in clinical trials also has increased, with 16 percent of NORD survey respondents reporting they had already participated in a clinical trial, up from 12 percent in 1988.

The survey also found that 38 percent of respondents were aware of existing patient registries or natural history studies, which collect longitudinal data to help inform the direction for future clinical R&D of new drugs and therapies. Of the 38 percent of respondents familiar with the registries or studies, 78 percent say they have participated in them. Eighty percent of the respondents said they had not yet participated in either option, but more than half (53 percent) said they would participate if such an option became available for their specific rare disease.

Read the latest NORD report here: https://bit.ly/2PVaXql.

Read the GlobalData analysis here: http://bit.ly/2PQvxIh.