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Chemistry
Science
Information gathered from preclinical tests are used to apply for permission to conduct clinical trials. In the UK, applications have to go to the Medicines and Healthcare products Regulatory Agency (MHRA) who assess the preclinical data, including safety data, and decides if the evidence supports allowing clinical trials in volunteers and patients to go ahead; while Research Ethics Committees (RECs), coordinated by the Health Research Authority (HRA) ensure that the rights, safety, dignity and well-being of clinical trial participants are protected. A clinical trial can only start when all the necessary approvals have been granted by these bodies. The organisation conducting a trial (usually known as the Sponsor) is also expected to register the trial on a public, searchable database. This helps to keep track of all the trials being conducted across the world at the same time, and also can help people find out where a trial is being conducted, in case they would like to volunteer (via their doctor) to participate.
After the preclinical studies, there are four phases of medicine development in humans, which in practice often overlap. Clinical trials usually start with healthy volunteers, although some Phase 1 trials can involve patients – either way, this will be the first time the medicine is used in people. In the case of healthy volunteer testing, the next step is to involve patients with the disease to see if and how the medicine works.
Phase 2 involves 100−400 patients with the target disease, whereas Phase 3 typically involves 1000−5000 patients with the target disease (some Phase 3 can be very large - involving 15-20,000 patients).
But, before anyone gets involved with clinical trials they’ll sit down with the team and work out exactly what’s going to happen, any of the risks and whether a clinical trial is the right thing for them. As we get more information the trials grow, with more patients in more hospitals not just in the UK but around the world. Trials can take anywhere from weeks to years depending on the type of disease and the type of medicine that we’re studying. This information is shared with the regulator who works out whether to grant a license or not.
New data and technology is opening up conducting research in new ways, looking into treatments that previously seemed impossible. The NHS is very active in research with 700,000 people taking part in research in disease areas such as cancer, heart disease and diabetes.
Clinical trials remain key to developing medicines of the future, but they can’t be carried out without patients and so the pharmaceutical industry is hugely grateful for everyone that makes them happen.
Some medicines work better in some people than in others. The company developing the medicine may know early on that this is likely to be the case, or they may find out during clinical trials in different populations around the world. This may lead to recommendations on which groups are more likely to benefit from the medicine.
In some cases a test may be available to identify those people for whom the medicine will work best.
Once a new medicine is on the market and doctors can prescribe it, studies will still continue as there is more to learn about the medicine.
Probably earlier trials will not have included young children so, if the disease is one that children also get, the medicine will need to be tested in children to identify the best dose to give and to ensure the medicine is safe for them to take.
Also many people will take several medicines at once, sometimes these can interfere with each other. For instance if two medicines are both broken down by the liver, they may be broken down more slowly which may mean that lower doses of one, or both, should be given. This will affect the dose that should be used. The most common medicines that are likely to be taken at the same time as the new medicine will have been considered during earlier trials, but not all possible combinations will have been tested.
There may be other diseases that the medicine might be able to treat, these will also be investigated through further studies.
They may include studies that look at how the new medicine improves clinical practice (eg shorter time in hospital clinics) or patient outcome (eg shorter stays in hospital) which help the NHS make cost savings.
In phase 2-4 clinical trials patients receive medicines which are expected to treat their illness, but there are other types of clinical studies.
Often these involve groups of people being observed to see if they develop a disease, or patients who have a disease are studied to see if past exposure to something might have led to them getting the disease. These are called observational clinical studies and are important in finding out more about what factors are linked to a disease such as heart disease or diabetes.
Trials may also compare the effectiveness of educating patients about their condition, or about how to improve their illness through lifestyle changes. For example one group of patients may be given a leaflet about how healthier eating could help them lose weight; another group might attend sessions where they were shown how to prepare healthy food. These two groups could be compared to see which method has been more effective. Although these trials cannot be double blind, allocation of patients to the different treatment groups would still have to be random to remove any bias.
Clinical trials are really just the beginning of a medicines journey: they must be monitored and regulated for the rest of their lifetime.
A medicine’s license is the green light for it to be given to patients and it covers everything from what it should be used for, who it should be given to and what dose is appropriate. However, new medicines must undergo two separate approval processes before they are available to patients on the NHS.
The UK medicines regulator (the Medicines and Healthcare products Regulatory Agency (MHRA)) assesses its quality, safety and efficacy to determine whether the medicine should be licensed for use. This allows it to be sold and supplied in the UK.
Separately, the medicine must undergo a cost-effectiveness assessment to determine whether it can be provided on the NHS – this is often referred to as reimbursement. This cost-effectiveness assessment is conducted by the National Institute for Health and Care Excellence (NICE) for England, Wales, and Northern Ireland, and by the Scottish Medicines Consortium (SMC) for Scotland. In addition, the All Wales Medicine Strategy Group (AWMSG) can conduct its own assessment of new medicines that do not have a NICE assessment.
A treatment must pass both approval processes to be fully accessible to patients on the NHS. Once a medicine is made available, the pharmaceutical company has a duty to monitor it and make sure it performs safely for as long as it’s on the market.
This process is called pharmacovigilance and it helps inform regulators about previously unknown side effects – it can also help collect real world data that can be fed back to improve future research.
More data means a better understanding of what a medicine can do – often meaning scope to explore using it to treat other conditions or used in other sets of patients.