Clinical Research & Development

Drug discovery and development is a long, costly and high-risk process that usually takes over 10 years, with an average cost of over £1billion for each new drug brought to market.^3,4

1. Drug discovery

The process begins with drug discovery, where researchers identify and validate potential biological targets (e.g. receptors or genes) that have been implicated in disease pathology.^5,6 High-throughput screening and computational tools (such as artificial intelligence) are used to screen a large number of potential compounds for their ability to interact with these targets.^4-6 Once a promising molecule (a lead compound) is identified, researchers test a series of structural variations to optimise the compound’s ability to bind to the target and exert its effect.⁶

2. Preclinical research

The next stage, preclinical research, involves extensive laboratory and animal studies to assess the safety and biological activity of the optimised lead compound, as well as investigating various formulations.⁷ This includes a thorough assessment of the pharmacokinetic profile (how the drug is absorbed, distributed, metabolised and excreted) as well as the drug’s effects at the active site of the target.⁷ These studies build a picture of possible dosing regimens and potential adverse effects.⁷

3. Clinical trials

If the compound demonstrates a favourable safety and efficacy profile in preclinical testing, it progresses to clinical trials, in which it is tested in humans across several phases to confirm its pharmacological properties, including therapeutic potential and safety for widespread use.^2,7 Because of the rigorous nature of the overall process, it is estimated that approximately 90% of drug candidates fail from the point of entering clinical trials; effective collaboration between academia and industry can help optimise the chances of success in drug development.^4,8

• Healthcare professionals are crucial to the success of clinical trials at every stage, including discussing enrolment with eligible patients, involvement in trial design, administering trial treatments and measuring outcomes^1,11,12
• The results from clinical trials provide critical evidence that informs potential licensing and can also shape clinical guidelines, advance medical knowledge and improve patient care^1,13
• Participation in clinical research not only contributes to the development of innovative therapies, but it also ensures that healthcare practices are grounded in robust scientific evidence^1,13