Clinical trials. What are the risks?

Before any new medicine can be given to patients detailed information about how it works and how safe it is must be collected.

Clinical trials are the key to getting that information and without volunteers to take part in the trials there would be no new treatments for the serious diseases which blight the lives of so many people.

However, as recent events in France have shown, testing experimental drugs can never be completely risk free.

In this regard it has been reported that one man has died and five others have been hospitalised after taking part in a clinical trial in France of an experimental drug aimed at tacking mood and anxiety issues as well as movement and co-ordination disorders linked to neurological conditions. The medicine involved is a so-called FAAH inhibitor that works by targeting the body’s so-called endocannabinoid system which is also responsible for the human response to cannabis.

It is understood that 90 people have taken part in the trial which has now been suspended.

Fortunately, cases of clinical trials going badly wrong are rare. However, they are not unheard of. For example, in 2006, six healthy volunteers given an experimental anti-inflammatory drug in London ended up in intensive care with organ failure. The worst affected lost his fingers and toes and all the men were subsequently told that they would be likely to develop cancers or auto-immune diseases as a result of their exposure to the drug.

What safeguards are in place?

Before a drug is tested in humans it goes through laboratory and animal testing (which itself can be a controversial issue). Drugs are also tested for toxicity before being given to people. However, inevitably someone always has to be the first person to test a new medicine.

It is estimated that between 50-100 people volunteer each year in the UK to begin the testing of new drugs.

Trials typically have three phases to assess a new medicine for safety and effectiveness: –

Phase I tests for safety. A small number of people, sometimes healthy, and sometimes with a medical condition, are given a tiny dose of the drug under careful supervision, not to test if the drug works, but in order to check for any side effects;

Phase II sees the drug given to people who have a medical condition to see if it does indeed help them;

Phase III trials are only for medicines or devices that have already passed the first two stages, and involve them being compared to existing treatments or a placebo. The trials often last a year or more, involving several thousand patients.

In most cases neither the scientists nor the patients know who has got the real drug so that the results cannot be skewed by expectations.

Once a drug has been through all these stages of testing – which can take up to 10 years – it will then be considered for licensing.

The recent drug trial in France was a Phase I clinical trial.

Whilst the person in France has paid the ultimate price it is worth remembering that thousands of people do safely take part in similar trials each year. Overall, the risks are low but there must inevitably be a ‘jump into the unknown’ whenever new drugs are tried on people for the first time.

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