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Assessing EB patients’ willingness to participate in clinical studies

New research article of the EB House study team published in the scientific journal Orphanet Journal of Rare Diseases

Conducting clinical research is essential for proving the efficacy and safety of a new treatment. However, trials for rare diseases such as EB are especially challenging due to the small number of patients affected. Additionally, every individual presents its own singular symptoms and challenges. Thus, recruitment of the right patients in a sufficient number for significant statistical analysis, but also patient motivation and retention for clinical trials remain the main hurdles.
In order to improve clinical research in EB, doctors and researchers of the EB House conducted a study to assess the patient’s attitudes towards clinical trial participation. They designed a questionnaire in lay language with 53 queries, which should determine what factors motivate and discourage EB patients in participating in clinical studies. The questions were defined based on the vast experience of the EB House study team as well as literature on trial burden, and addressed the following main topics: general health and quality of life, demographic data, knowledge and interest to participate in clinical studies, pros and cons for participation, and the acceptable extent of individual expenses for trial participation.
The questionnaire was handed over to patients of all EB types registered in the EB House Austria, of whom 36 participated. Statistical evaluation revealed that a high percentage of patients wish to increase their knowledge (66.7%), and is motivated to participate in clinical studies (57.2%). The major driving factor to participate was the hope for better treatments for other EB patients in the future, followed by the hope to relieve their own symptoms. The most important arguments against trial participation were travel distance to the study site and concerns about possible side effects and unknown risks. Especially for severely affected and younger patients, the frequent study visits, blood takes, skin biopsies, inpatient stays, but also financial expenditures and incompatibility with daily life obligations were reasons against trial enrollment.
The outcome of this study provides guidance to optimize study protocols and clinical trial design for EB and other rare diseases, in order to overcome the main obstacles to participating in clinical trials.

To access the article please click here

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