Paradigms for the development of transformative medicines—lessons from the EGFR story

Successful drug development brings a new therapeutic to the market starting from an idea in the laboratory all the way to making it widely available for patients. Activities span from preclinical research to demonstration that the molecule improves how a patient feels, functions, and survives, culminating in filing with a regulatory agency such as the European Medicines Agency or the US Food and Drug Administration (FDA). In the United States, only ∼1800 new molecular entities (NMEs) have been approved since 1827,1,2 illustrating the challenge of transforming scientific ideas into medicines.