Drug development

Developing a new drug is an expensive, time consuming and highly regulated process.

It is estimated to take up to fifteen years to develop one new drug from the earliest stages of discovery through preclinical, clinical and regulatory assessment to the time it is widely available for treating patients. The average cost to research and develop each successful drug is estimated to be around £1 billion. However, the attrition rate is high and for every 5,000 – 10,000 compounds that enter the research and development (R&D) pipeline ultimately only one receives approval.

At first, these figures may seem incomprehensible but a deeper understanding of the R&D process can explain why so much time and effort is needed to get a new drug to the clinic.

Target identification

When researchers have gained enough understanding of the underlying cause of the disease, a “target” is selected for a potential new drug. This target is generally a single molecule, such as a gene or protein, which is involved in the disease. Even at this stage it is critical to pick a target that is “druggable” i.e. one that can interact and be affected by a drug molecule.

Target validation

Once a potential target has been chosen, it must be tested to confirm its role in the disease and that it can be acted upon by a drug. This is known as target validation and is an important step in making sure that the correct target is being looked at: improper validation is often cited as the main reason for the failure of drug discovery projects. Researchers demonstrate this through many experiments in cells and in animal models of disease.