Most people with epidermolysis bullosa (EB) want to participate in clinical research for potential new therapies, but find travel distances to trial sites and the risk of treatment side effects to be major challenges, a survey in Austria has found.
The study, “Profiling trial burden and patients’ attitudes to improve clinical research in epidermolysis bullosa,” was published in the Orphanet Journal of Rare Diseases.
EB comprises a group of skin disorders characterized by blistering and scarring of the skin and, sometimes, blistering within the body. A growing number of potential new therapies are being tested in clinical trials.
Clinical researchers of rare conditions such as EB often face challenges, including finding a large number of eligible participants within a reasonable timeframe.
In the new study, researchers from the Paracelsus Medical University, in Salzburg, in collaboration with the specialty care center EB House Austria provided a 53-item questionnaire to EB patients and caregivers to determine attitudes toward clinical research and trial participation.
In particular, they sought to understand the factors that most motivated and discouraged patients and caregivers, and to gain insights into how to design trials that are more focused on clinically relevant goals and outcomes.
The analysis comprised responses from 36 participants (mean age of 25.7, age range 5 to 80). From those with available information on country of origin, 17 were from Austria, 10 from Germany, and five from Italy. Fourteen (38.9%) had a mild EB type, and 16 had a more severe disease form. Parents provided responses for six of the 12 participants younger than 18.
Most participants (57%) ranked their desire to participate in clinical trials as high, regardless of the severity of their condition. Age proved to be a deciding factor, with 75% of children expressing high motivation to join in a trial, compared with 44.4% of adults. Respondents who had participated in previous trials also showed more willingness to do so again (59.3% vs 50%).
Slightly more than two-thirds of the respondents indicated a strong desire to increase their personal knowledge about clinical studies in general, and to receive more information about any locally available ones.
Distance to a trial site was the main obstacle to participation for 67.7% of the respondents. The second hurdle was the possibility of adverse reactions, or other unknown risks from experimental treatments (45.5%). In addition, respondents listed personal expenses as another obstacle to enrollment.
Respondents with more severe types of EB also expressed hesitation about invasive procedures. As such, the researchers said that studies should take such concerns into consideration when optimizing their protocols.
Among motivations to participate, altruism appeared to be the strongest. The researchers labeled as altruistic the hope that participation would lead to better treatments for people with EB in the future — 87.5% of respondents cited this hope as an important reason for trial participation — and would add to the body of knowledge concerning EB (68.8%).
The possibility of easing symptoms was also a key motivation for approximately 77% of respondents.
Four individuals — one with mild EB, one severe, and two without indicated severity — viewed a 50% reduction in symptoms as sufficient to consider a clinical trial successful. Two more patients, both with severe EB, viewed 30% as sufficient. However, the low number of responses in this specific analysis warrant caution about drawing conclusions, the team added.
“Despite significant limitations,” the investigators wrote, “this study provides additional indications to better determine and address attitudes towards clinical research among EB patients as well as guidance to optimize clinical trial protocols.”
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