Lessons learned from a new survey about what motivates and retains patients in rare disease trials could have much wider applications in other clinical trials, according to those who conducted the research at Scout Clinical.
The new research indicates that “most sponsors don’t have a comprehensive and systematic approach to trial participation support. This was validated in our research which identified significant gaps in support in all three categories of participation pressures (i.e., financial, physical/logistical, and psychological/emotional),” the report said.
“Sponsors don’t always realize others [in more common trials such as] oncology need” these same supports to help them remain active in a clinical trial, said Jessica McKague, executive director business strategy at Scout Clinical, and a co-author of the report.
Looking at the positive, though, McKague noted, “Covid has increased sponsors focus on the patient perspective, especially when it comes to diversity and inclusion efforts.”
“The key question sponsors should always be asking is ‘how does the clinical trial fit into the patients’ life?’” said report co-author Kimberlee Heidmann, VP Patient Services at Scout Clinical. She advised sponsors and others to “listen to patient advocacy groups. I’d love to see sponsors utilize them more as a resource” when designing a trial and throughout the process, Heidmann said.
“There’s already been a shift toward patient-centricity” in clinical trials, Heidmann noted, but more remains to be done. She urged sponsors to think creatively beyond “stipends and transportation” issue.
While those supports are often very important, clinical trial design that doesn’t focus on participants other needs may flounder regardless, she said. For example, when working with geriatric populations it is often important to address the fact that many may be visually impaired, she said.
The research “raised two issues of relevance to equitable access to clinical trials and increased participation among underserved patient populations,” according to the report. First, whether regulators and ethical review boards must recognize that trial participation exposes individuals affected by rare diseases to incremental financial, physical, and psychological pressures that could be mitigated, and which translate to reduced access or significant incremental burdens for some; and second, whether reducing participation burden would result in fewer negative participant experiences—helping change negative perceptions of clinical research among the general public.
The research was conducted between November 2020 and February 2021 and comprised a series of focus groups with 16 participants and a web-based survey with 126 responses from individuals affected by unique rare diseases.
Author: Michael Causey
