Early research and development relates to the activities of chemists, biologists and pharmacologists who develop and test new active substances in the laboratory.
Historically, new medicines have been sought from among compounds occurring naturally in plants, fungi or marine organisms, and this still happens today. When biological activity is discovered in this way, the molecule causing it is extracted from its natural source, identified and synthesised – usually chemically. Chemists then make many close variations (called analogues), to try and maximise the desired effects and minimise unwanted effects.
We work with our member companies to enhance understanding of preclinical safety and provide a neutral forum to share experiences in what is known as a ‘pre-competitive space’ where information is not proprietary or Intellectual Property (IP) sensitive. ABPI member companies also work together in a number of non-competitive research collaborations. For example, they fund collaboration with the Centre for Drug Safety Science at the University of Liverpool, with the aim of better understanding the safety of developing new medicines; and investigating how to improve the design, tailoring and selection of new medicines to develop for use by patients. If you would like to find out more about this centre visit www.pcweb.liv.ac.uk/drug-safety.
New active substances
The natural world continues to be a source of new medicines, but researchers have also developed many other sophisticated ways of creating new active substances. Increasingly, genomics, proteomics and synthetic chemistry are used in their design. These build on knowledge gained from the study of genes (genomics), a deeper understanding of disease processes; the study of the numerous protein products from specific genes (proteomics) and from the use of computer-aided design.
The development of completely new classes of medicines typically depends on research into the fundamental mechanisms of disease and often involves collaboration with universities and other research institutes.
Much work has to be done to determine whether a potential new medicine is acceptably safe and sufficiently stable before it can be given to humans. The skill of transferring research effectively from preclinical models to early clinical research in man is known as translational medicine. It also works two ways, by applying what is learnt in clinical practice to refer to clinical models.
Early stage clinical trials
Before a new medicine may be given to humans in the UK, a clinical trial application (CTA) has to be made to an executive agency of the Department of Health known as the Medicines and Healthcare products Regulatory Agency (MHRA) for a certificate to conduct clinical trials. The application is reviewed by independent medical and scientific experts, who make their recommendation on whether the trial can start, or whether more information or research is required.
If a CTA is granted, a new medicine will pass through a long and complex process of early and later stage clinical studies before the company can seek marketing authorisation to make the medicine available for doctors to prescribe for widespread use. The conduct and management of clinical trials in the UK is governed by European Union legislation, which sets standards known collectively as Good Clinical Practice (GCP).