Profiling trial burden and patients' attitudes to improve clinical research in epidermolysis bullosa.

ABSTRACT:

Background

Epidermolysis bullosa (EB) comprises inherited mechanobullous dermatoses with considerable morbidity and mortality. While current treatments are symptomatic, a growing number of innovative therapeutic compounds are evaluated in clinical trials. Clinical research in rare diseases like EB, however, faces many challenges, including sample size requirements and recruitment failures. The objective of this study was to determine attitudes of EB patients towards clinical research and trial participation as well as the assessment of contextual motivating and discouraging factors in an effort to support patient-centered RD trial designing.

Methods

A 53-items questionnaire was handed over to EB patients (of all types and ages) in contact with the EB House Austria, a designa

SUBMITTER: Prodinger C 

PROVIDER: S-EPMC7350741 | biostudies-literature | 2020 Jul

REPOSITORIES: biostudies-literature