Background Debate over the viability of the current commercial research and development
(R&D) model is ongoing. A controversial theme is the cost of bringing a new molecular entity …

Rare diseases are increasingly recognized as a global public health priority. Governments
worldwide currently provide important incentives to stimulate the discovery and development …

This paper describes a comparative analysis of the physicochemical and structural
properties of prodrugs and their corresponding drugs with regard to drug‐likeness rules. The …

Rare diseases are life-threatening or chronically debilitating low-prevalent disorders caused
by pathogenic mutations or particular environmental insults. Due to their high complexity …

Research and development (R&D) costs factor into considerations of the tradeoffs between
prices, intellectual property protection, and incentivizing innovation, all of which can have …

In recent years, regulatory bodies have increasingly recognized the utility of real-world
evidence (RWE) for supplementing and supporting clinical trial data in new drug …

The natural history of most rare diseases is incompletely understood and usually relies on
studies with low level of evidence. Consistent with the goals for future research of rare …

A wide range of complex in vitro models (CIVMs) are being developed for scientific research
and preclinical drug efficacy and safety testing. The hope is that these CIVMs will mimic …

Beschleunigte Zulassungen stellen die Nutzenbewertung und Erstattung von Arzneimitteln
vor besondere Herausforderungen. Der Artikel setzt sich in Form eines narrativen Reviews …

A rare disease, or orphan disease, in the United States is a condition with a national
prevalence of fewer than 200,000 diagnoses. As therapies for rare diseases are developed …