Healthy living
Medicines and supplements
Drugs on trial
Scientists developing new treatments depend on volunteers to test their safety and effectiveness
When you take your medication, the one that keeps your blood pressure down, controls your asthma or treats any medical condition from cancer to a less than peppy performance in bed, you can be reasonably sure it’s safe. That’s because it’s been tested in laboratories, on animals and on people like you.
Every new drug has to go through a rigorous testing procedure, and doesn’t get anywhere near a prescription pad unless it’s been through a series of clinical trials. These are research studies in which the drugs are tested to make sure that they are safe and help people feel better, and to pick up any side effects. Clinical trials usually compare new or different treatments against the best existing treatment, to find out whether they’re more effective.
"We do a lot of clinical trials to test new drug treatments," explains Janet Darbyshire, Director of the Medical Research Council's Clinical Trials Unit. "But as well as trialling new drugs we also test treatments, such as surgery and radiotherapy, and we test ways of preventing disease - one area we're working on is trialling HIV vaccines. And there's a big trial run from University College, London, at the moment, looking at ways of screening for ovarian cancer."
Clinical trials have produced many findings that have greatly improved health care. "The Heart Protection Study run from Oxford, found that starting patients on statins at lower levels of cholesterol in the blood could reduce the risk of coronary heart disease," says Janet Darbyshire.
"The negative results from trials are as important as the positives. For instance, with HRT, we knew it would help with menopausal symptoms and reducing fractures and there was some evidence that it would reduce the risk of cardiovascular disease, but we found that if anything it increases the risk of cardiovascular disease."
How do clinical trials work?
Trials on new drugs go through several phases.
Phase I
This is designed to test safety and these trials usually only involve a small number of people. They may already be patients (especially in the case of cancer treatments, where the drugs may have a lot of side effects), or they may be healthy volunteers.
Phase II
At this stage the treatment is tested in a larger group of people – often a few hundred – who usually have the disease. The trial will look to see if the drug has any effect and is safe.
Phase III
This compares the new drug with the most effective treatment currently in use, or if there isn’t one, with a placebo (a dummy drug). No one knows which of the participants are being given the drug and which are taking the placebo.
Clinical trials are often randomised, which means that they compare groups of people who are similar except for the treatment they receive. This helps to give a fair comparison between the treatments. Randomised trials are seen as the most reliable way of testing new treatments or comparing existing treatments. Trials can involve thousands of patients, from different countries, and can last for a year, five years, or longer.
Why volunteer?
"People who take part in trials get a very high standard of care and a lot of support and information," says Kate Law, Director of Clinical Trials at Cancer Research UK. "The vast majority of people who’ve been on a trial give us positive feedback. They have specialist nurses, and a number to call day and night.
"We have about 30,000 patients a year on late phase trials. Some of the patients in our cancer trials have had all the chemotherapy and radiation that’s available to them. They have a choice between the best supportive care and trying a new drug. Many also feel that they’re doing good for future generations and for their own family."
While single trials on their own can provide vital healthcare information, the cumulative knowledge that comes from compiling the results from many trials can be even more powerful. "Looking back over the history of trials, a whole series of clinical trials led to effective treatments for Hodgkin’s disease and for childhood leukaemia," says Janet Darbyshire. "It’s important that people continue to take part in trials or we won’t have any more new or better treatments."
Can things go wrong?
When a Phase I clinical trial carried out by a drug company on premises owned by Northwick Park Hospital in 2006 went wrong, it hit the headlines in spectacular fashion. The small group of healthy volunteers involved had a dramatic reaction to the compound being tested – a monoclonal antibody - and rapidly became ill.
"This was the first time the compound had been given to man," explains Janet Darbyshire, who was part of the Expert Scientific Group who reported on the trial. "The compound was a novel compound which acts as a modulator of the body’s defence system. Some drugs of this type have already proved highly effective in the treatment of diseases such as rheumatoid arthritis. But it can be hard to predict what effect they’ll have. This was a very rare occurrence, and was not predicted by the laboratory or animal studies of the effect of the antibody.
"One of the recommendations that came out of the expert group was that every time we do a Phase I trial, and particularly when we test one of these very novel compounds for the first time in man, we should consider carefully whether we should be testing it in patients or healthy volunteers."
If you do decide to volunteer for a trial, make sure you understand what’s involved and ask as many questions as you need to. Remember that you have the right to withdraw from a trial at any time.
Payment
Some clinical trials, especially those carrying out Phase 1 trials in man, offer volunteers payment. Others offer expenses, to cover the cost of travel etc. Drug companies and independent organisations running clinical trials may base a fee on certain circumstances, for instance how much of your time they will be taking up. If you decide that you want to volunteer for a trial, ask whether you will be reimbursed for expenses, or if payment is made for the inconvenience of repeated visits to the clinical trial unit.
You can download Phase 1 guidelines for clinical trials, which includes a section on payment, from the Association of the British Pharmaceutical Industry website.
How to find out more
- Cancer Research UK’s website, cancerhelp.org.uk has details of trials for different types of cancer, and you can speak to a Cancer Information Nurse on 0207 061 8355
- If you are interested in taking part in a clinical trial for a condition you have, ask your GP or doctor treating you, whether there are any suitable trials currently recruiting volunteers. You could also phone or check the website of the charity relevant to your condition, such as the Arthritis Research Campaign
- The Clinical Trials Unit of the Medical Research Council co-ordinates trials in cancer and HIV and carries out research into conditions including rheumatoid arthritis, respiratory disorders, infectious diseases and haematological disease
- Cancer backup’s free booklet ‘Understanding cancer research trials’ can tell you more about what’s involved in clinical trials. Please click here to find out more
- UKCRC’s free booklet “ Understanding Clinical Trials” ( which covers all disease areas) is available at UKCRC.org or UKCRN.org.uk
Useful websites
- NHS library for information about clinical trials
- Website of the Medical Research Council’s Clinical Trials Unit: this will give you more information about clinical trials and useful links
- Cancer Research UK for details of specific clinical trials. Visit Cancer Back Up for details of specific clinical trials
- The James Lind Library gives general information on clinical trials
Useful articles
Information on this site is for interest only and is not a substitute for professional medical advice. You should consult your own doctor about any specific health concerns.
