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 …

Most rare diseases still lack approved treatments despite major advances in research
providing the tools to understand their molecular basis, as well as legislation providing …

Importance The mean cost of developing a new drug has been the subject of debate, with
recent estimates ranging from $314 million to $2.8 billion. Objective To estimate the …

A new medicine will take an average of 10–15 years and more than US $2 billion before it
can reach the pharmacy shelf. Traditionally, drug discovery relied on natural products as the …

Pricing of pharmaceuticals is becoming a major challenge for health systems | The BMJ
Skip to main content Intended for healthcare professionals Access provided by Google …

Background Over the past decades, US Congress enabled the US Food and Drug
Administration (FDA) to facilitate and expedite drug development for serious conditions …

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 …

The conventional drug discovery pipeline has proven to be unsustainable for rare diseases.
Herein, we discuss recent advances in biomedical knowledge mining applied to discovering …

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

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