About Clinical trials
Clinical trials are very demanding next the clinical routine in hospitals. However, they also are drivers in translational research and drug development. Commitment and contribution of medical staff and patients is important for steady progress in EB treatment.
As of now, there is no causal systemic, long-lasting therapy for EB available. Until such is achieved, new approaches for symptomatic treatment or local causal therapies are developed. They aim to increase the patient’s quality of life and correct the underlying gene defect locally on highly affected areas, respectively. In order to translate promising results from the lab to the clinics (“Bench-2-Bedside”) clinical trials are conducted. Depending on the trial phase (phase I-IV), the main focus is on safety assurance, dosage finding, efficacy testing and long-term tolerance observations (see figure below).
The aim of a clinical trial is market authorization and therefore availability of effective therapies for patients. To achive this aim high numbers of participants are needed. The patient cohort in an orphan disease such as EB is small, hence this demands for multicentric, international trials. Here EB-CLINET can be of help in facilitating communication and connection of clinicians and expertise.
To be able to handle the increased time, administrative and financial effort during clinical trials, a well-trained team of physicians, nurses and study coordinators is needed. Before inclusion of patients, competent authorities and an ethics committee must release all study material (study protocol, informed consent forms, investigator medicinal product dossier, questionnaires, and information material). Eligible patients (defined by inclusion and exclusion criteria) must be compliant with the study protocol and each intervention and examination has to be documented in detail following Good Clinical Practice. After completion, the trial results are evaluated and statistically analyzed and should be published.