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“Focus group participants said that motivation is not enough. Identifying where, specifically, trial support services need to improve is the first step in making rare disease trial participation and completion less expensive, ”said Heidmann.

From December 2020 to February 2021, researchers conducted four virtual focus groups with a total of 16 people with a rare disease who had participated in at least one clinical trial. All participants were North American (United States or Canada).

Information from the focus groups was used to develop an online survey (conducted January 26 – February 19) and was open to all rare disease patients and their caregivers, whether or not they participated in a trial. clinical. There were 126 respondents who are affected by a total of 69 rare diseases.

The data collected revealed that although focus group participants were highly motivated to participate in clinical trials, they face various pressures due to factors that negatively impacted their ability to do so. These pressures can be classified into three main types: financial, physical and logistical, and psychological and emotional.

While almost all respondents made a concerted effort to overcome these obstacles and complete the trials, this was not always possible.

“This pragmatic reality contrasts with a general perception within the drug development ecosystem that, due to the high unmet need within rare diseases, motivation is sufficient to ensure a participant’s ability to make it through. or all of the barriers to participating in an essay, “said the paper states.

Focus group participants identified several financial barriers to the success of the trial. In total, 50% of survey respondents indicated that they sometimes or frequently experience financial difficulties. Trial policies may not cover certain participant expenses, such as airline tickets for site visits, wheelchair accessible rental vehicles, and hotel accommodation. Participation in the trials also includes “hidden” costs such as childcare and mileage costs.

Some participants reported barriers to reimbursing expenses covered by the trial. Receipts were not always available and the wait for reimbursement negatively affected their financial situation. Others have reported loss of income due to participation in clinical trials.

“Our research results suggest that current participation support practices leave trial participants with additional financial pressures, which become barriers to joining or completing a trial. This underscores the importance of considering the financial pressures that may be associated with participation as a key to risk assessment of rare disease trials, ”the report says.

Since rare disease clinical trials typically take place in relatively few locations, participants also face significant physical and logistical pressures. Focus group participants reported experiencing physical strain, fatigue, pain, and anxiety while traveling, navigating unfamiliar healthcare environments, and care from unfamiliar providers.

When it comes to psychological and emotional pressures, most focus group participants encountered this type of barrier and a wide range of triggers were reported. Some rare disease patients have missed their families while on trial visits and have become hyper aware of their condition. Some parents have expressed concern over whether participating in an investigational drug trial is the right decision for their child and upset that their child is in pain or distress.

Only one focus group participant received psychological support.

“This is not surprising, as psychological support is generally not offered to trial participants,” the report notes.

Interestingly, about 30% of survey respondents said that receiving emotional support or counseling would make it easier for them to participate in a trial.

When asked what constitutes “good” participation support, focus group members identified three qualities that support strategies should include: being personalized and responsive to patient needs; be comprehensive and transparent; be easily accessible via a single point of contact.

Overall, “over 70% of survey respondents said that if they were not able to receive the participation support services they need, their ability to participate in a trial would definitely or somewhat be affected, ”the researchers wrote.

Taken together, “this information points to several critical success factors for sponsors to reduce the risk of their rare disease clinical trials with an effective participation support strategy,” the report says.

These include “cultivating a mindset shift that recognizes the active role trial participants play in the effective conduct of the trial, engaging with patient communities to conduct burden assessments. participation during the initial trial design and to implement a strategy to support participation in the risk management process of clinical trials ”.