Background Repurposing is a drug development strategy receiving heightened attention
after the Food and Drug Administration granted emergency use authorization of several …

Highlights•β-THAL is one of the most prevalent blood disorders in some regions of the
world.•However, given its prevalence, in the EU and the US it is classified as a rare …

Objective To determine differences in the characteristics of cancer drugs designated as
orphan drugs by the Food and Drug Administration (FDA) and European Medicines Agency …

Highlights•Marketing Authorizations (MA) for Gene Therapy require a complex regulatory
review.•Quality and non-clinical data are generally satisfactorily accepted by …

Abstract Background In the United States (US) and in Europe, cystic fibrosis (CF) qualifies as
a rare disease, thus positioning the field to benefit from regulatory incentives provided by …

Since developments are global in the healthcare arena, more should be done to align EU
and other big markets' regulatory practices for rare disease patients. Notwithstanding efforts …

Sickle cell disease (SCD) is a hereditary red cell disorder with a large disease burden at a
global level. In the United States and Europe, medicines may qualify for orphan designation …

Bearing in mind that it is currently not standard practice to publish non-clinical trial research
protocols in EJHP, we would like to share our thoughts within the EJHP community on …

Introduction The European Medicines Agency (EMA) offers scientific advice to support the
qualification procedure of novel methodologies, such as preclinical and in vitro models …

The delicate balance of funding research and development of treatments for rare disease is
only imperfectly achieved in Europe, and even the current provisional equilibrium is under a …