Innovation is in our industry’s DNA. It’s the vehicle by which we improve patients’ lives, evolve the way we manage healthcare and benefit wider society. But our innovation gene is just the start, expressing it - discovering and developing treatments that mean alleviating suffering, extending life and improving its quality requires time, great people, significant resources, collaboration across sectors and borders and often-endless experimentation to search out new breakthroughs.
Developing new medicines is a long, complex and risky process with no guarantee of success.
It begins with an idea.
Discovery and pre-clinical research
At the earliest stage, its about understanding a disease and how to influence it, identifying a molecule or compound for development, to see if it has the potential to turn in to a medicines that can change the lives of patients. For every 10,000 molecules identified only one will make it all the way to being approved by the regulatory authority and prescribed for patients by a physician. There are a number of phases in that journey.
That molecule or compound then goes forward to preclinical testing to determine whether it is safe to progress to use in people. These tests are performed either in silico – in computer models, in vitro - testing in a controlled environment outside a living organism, or in vivo - testing in a living organism. Testing of a potentially effective molecule in at least two species of animals is an emotive issue but almost always an essential step to ensure that it can safely go on to the next stage, testing in human subjects.
In order to address further issues of uncertainty surrounding the benefits and risks of a new treatment, it needs to be tested within the framework of a clinical trial. Prior to embarking upon the commonly three-stage clinical trial process itself, a company or academia must submit a protocol for a regulatory assessment, which details what is going to take place in the trial. The protocol is evaluated by an ethics committee, which consists of independent experts and representatives of the lay public. Clinical trial starts with a small trial in healthy volunteers (phase 1) before going in to increasingly large trial in phase 2 and phase 3 which are often multi-centre trials involving sometimes thousands of patients and conducted in multiple countries and regions and having the objective to find out if the new medicine is safe and effective. All trials need to be registered in the publicly available database before start of the trial.
All clinical trials in the European Economic Area must be carried out in strict compliance with guidelines on Good Clinical Practice (GCP), an international ethical and scientific quality standard for designing, recording and reporting trials involving human subjects. Adhering to the GCP guidelines means that the rights of trial subjects are respected and clinical-trial data are reliable. Also, compliance with guidelines on Good Manufacturing Practice (GMP) needs to be ensured so that products are consistently produced and controlled according to quality standards.
During a trial, patients are separated at random into commonly two groups. This randomisation helps to ensure that any outcomes recorded are due to the treatments provided and not the method of patient selection. One group is provided with the study drug or treatment procedure, whereas the other - the control group - is not. The control group may be offered an alternative to the study drug – including the best existing therapy or, where this does not exist, a placebo. Trials should address a wide variety of patients for whom the treatment is intended, including ethnic groups, children and the elderly.
In most of the trials, patients are not aware of whether they are provided with the treatment being investigated or the placebo/other known therapy. This process is known as blinding. Healthcare professionals in the trial may also be prohibited from knowing which patient is in which group and this process, in addition to the former, is known as "double blinding". This helps to protect the trial from any form of bias.
Once the trial has been completed, a team of independent, unbiased researchers analyses its results. A summary of the trial, whether its outcome has been positive or negative, needs to be disclosed on a publicly available database.
The progress in technology for data collection and analysis makes it increasingly possible today to identify the right patient populations, combine more sources of information and monitor the patient on-goingly, which are all advances to improve the quality of clinical research.
The information and results from all the preclinical and clinical studies, together with a description of the medicine's manufacturing process, are compiled and submitted to regulatory agencies in order to demonstrate the safety and effectiveness of the new medicine. This constitutes an application for a marketing authorisation, allowing the medicine to be placed on the market and for patients to have access to it.
Once a medicine has been granted a marketing authorisation it is then subject to a strict monitoring designed to detect any potential safety or quality issues. This process is known as pharmacovigilance and must be undertaken in line with Good Pharmacovigilance Practices (GVP), a set of measures laid down by the EU. The onus is on the company that has been granted the marketing authorisation to collect and collate on a continuous basis, data that relates to the safety, quality and efficacy of the medicine. All this data is then accessible by the regulatory agencies.
Data on medicines use
Data collected from the patients and their use of medicines increase our understanding of diseases and patients and inform the discovery and development of new or optimised treatments.