Woman’s Weekly GP, Dr Melanie Wynne-Jones investigates what they are, what to expect, whether they’re reliable and whether you should say yes to taking part
Medical advances rely on high-quality research to find new or more effective treatments, or abandon old ones.
More than 600,000 of us took part in clinical NHS research in 2014, helping to build our knowledge of what works, what doesn’t and the potential downside of interventions such as surgery, medicines, vaccines, scans, tests and other physical or psychological therapies.
So if you have a medical condition, it’s likely you may be asked to help.
Why take part?
Your doctors may have tried everything else currently available, or you may hope the new treatment will be better or have fewer side effects.
You may feel reassured that you will be monitored more frequently or have extra tests (travelling expenses are often reimbursed), want to help others or ‘give something back’.
But don’t feel pressured (including by well-meaning but worried loved ones); taking part is always voluntary, and will not otherwise affect your care.
Types of clinical trial
New drugs must be tested on animals, then (often paid) volunteers who may be healthy or have a particular condition (called phase 1 and 2 trials).
They can then be tried on larger numbers of patients (phase 3) to find out more about benefits and side effects.
They may be tested against ‘dummy’ pills (placebos), or one or more similar or completely different drugs (‘head-to-head’ or ‘multi-arm’ trials), often in several hospitals or countries (multi-centres).
Double-blinded random controlled trials aim to eliminate biased assessments by randomly allocating patients to different treatments so neither they, nor their doctors, know who’s taking what.
‘Multi-arm’ trials can be more flexible, cheaper andgive quicker results – especially desirable in fast-moving specialties, such as cancer care, where around one in five patients participates in research.
Clinical trials can also be used to compare different physical treatments (such as operations, implants or acupuncture), talking treatments, lifestyles or health education, or teaching people to become experts in self-monitoring and caring for their condition.
Research may be prospective (deciding what to use and monitor, and seeing what happens) or retrospective (looking back through people’s medical records to see what happened – for example,
after long-term smoking or a head injury).
Ethics and regulation
European rules for medical research are laid down in the Declaration of Helsinki. Safety
is paramount and in the UK all medicines must be tested on animals first.
Researchers must produce a protocol (a detailed description of what is planned, showing that it follows the ‘rules’, and provides full and clear information for patients), which must be passed by the local ethics committee.
This committee may also have to decide what happens if participants can’t give full and informed consent (for example, if they’re unconscious or if they have dementia).
Pharmaceutical and medical product companies are also regulated by the Department
of Health; research details, and in particular, any suspected unexpected serious adverse reactions (SUSARs) in UK-relevant trials must be promptly notified to the Medicines and Healthcare products Regulatory Agency (MHRA).
A recent UK/US review concluded that only 1% of adverse events in phase 1 trials were severe.
The MHRA also publishes codes of practice and carries out inspections.
Trusting the results
Occasionally trials are halted early, because of SUSARs (eg, the so-called ‘elephant man’ reaction in 2006 at Northwick Park), or because one treatment ‘arm’ appears to be significantly safer or more effective.
However this is controversial, as it could mean that longer-term benefits or SUSARs go undetected.
Some believe promising drugs should be ‘fast-tracked’ (eg, an Ebola vaccine), bypassing traditional safeguards, or ‘tried out’ on willing volunteers who already face a shortened life expectancy.
There are also concerns about vested interests; eg, pharmaceutical companies fund many research trials, but a balanced assessment isn’t possible unless unfavourable results are published, too.
How to join a trial
Your hospital doctor may tell you what trials you could enter and many patient information organisations, such as Cancer Research UK, have details too.
You can read more at nhs.uk and cancerresearchuk.org/our-research or listen to people’s stories at healthtalk.org.
The National Institute for Health Research has also set up the UK Clinical Trials Gateway ukctg.nihr.ac.uk to provide easy-to-understand information about current clinical research trials in the UK; you can download the UK Clinical Research Collaboration’s Understanding Clinical Trials booklet (www.ukcrc.org), but you will need a referral from your doctor to participate.
Here’s what you should ask before joining any clinical trial:
✤ What’s the aim of the trial and how will it help people?
✤ Who’s funding the trial?
✤ What treatment will I get if I don’t take part?
✤ How long is the trial expected to last, and how long will I have to participate?
✤ How long will it be before the results of the trial are known?
✤ What will happen if I stop the trial treatment or leave before it ends?
✤ How much of my time will be needed?
✤ What extra tests or appointments will I have?
✤ Will I need to take time off work?
✤ Will I need extra help from family or friends?
✤ Will the costs of my travel to take part in the trial be covered?
✤ If the trial is testing a new drug, will I have to collect it from the hospital, will it be sent to me by post, or will I get it through my doctor?
✤ Will I have to fill out questionnaires or a diary?
✤ What are the possible side effects of treatment?
✤ How could the treatments affect me physically and emotionally?
✤ Who can I contact if I have a problem?
✤ Will someone be available 24 hours a day?
✤ How do I find out the results of the trial?