Use this library as a technical resource.
All papers are downloadable in PDF format.
RWE Study Design
Real-world evidence (RWE) derives from data collected outside of highly-controlled randomized clinical trials (patient reported outcomes, registries, databases, surveys, etc). RWE is rapidly gaining importance in healthcare decision making, as reflected in the various guidances that have already been issued or are planned by the EMA and FDA. In this review, we introduce the concept of RWE, describe the main RWE study designs, comment on the most relevant criteria that have to be taken into account, and include the TFS-RWE algorithm to find the most suitable design for you. Finally, we provide an overview of the European Union and United States regulations on RWE.
Epidermolysis Bullosa - Future perspectives in drug development
Epidermolysis bullosa is a heterogeneous group of rare disorders characterized by mechanical fragility of epithelial tissues. Researchers around the globe are investigating new therapies to improve the quality of life of patients with little or even non-available therapeutic options so far, particularly for the more severe variants. New horizons seems could be opened in the coming years in one of the ”Worst Disease You’ve Never Heard Of ”according to DEBRA (Dystrophic Epidermolysis Bullosa Research Association of America).