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“I would absolutely recommend women to take part in trials if they feel it is right for them. I was very keen to take part in it because I want to try and increase the different drugs that are available for use with ovarian cancer.”

Find out about ovarian cancer clinical trials – what they are, how they work and how you can find out more. 

What are clinical trials?

Clinical trials are research studies that investigate potential new drugs, new ways of giving treatments or different types of treatments and compare them to the current standard treatments.

How do clinical trials work?

Drugs and treatments are usually assessed in three phases before they can be considered as a standard treatment option. Each clinical trial phase is designed to answer a specific set of questions about a new drug or treatment, which means there are strict guidelines about who can participate.

Phase I

The aim of a Phase I trial is to find a safe dose of the treatment and look at the possible side effects. If the treatment is safe it will go into Phase II to see if it has a positive effect on the patient.
 
In many cases, this will be the first time the treatment has been tested outside the laboratory. A small number of people - around 30 or fewer - who have no standard treatment options available, are chosen to participate. 

Phase II

This builds on the findings from Phase I, improving knowledge of the potential side effects of the treatment and the best dose of the treatment to give. 

It usually involves up to 100 people and looks at whether the treatment is having a positive effect on the type of cancer being studied. If tumours respond to the treatment (either by slowing down growth or reducing in size), it moves to Phase III.

Phase III

This phase recruits the largest number of participants with hundreds or even thousands of people taking part, often from hospitals across the country. 
 
It compares the new treatment with a standard treatment and will look to see if the new treatment improves overall survival or progression free survival (how long someone stays free of cancer). 

Phase IV

This happens after a license has been granted, and involves studies to monitor the medicine on an ongoing basis to see if there are any unexpected side effects, or if it causes problems in certain categories of people. 

What are the benefits of joining a clinical trial?

Many women view taking part in a clinical trial as a very positive experience. Some of the benefits include:

  • Getting a new treatment before it is widely available.
  • Being one of the first to benefit if the drug or treatment works.
  • Receiving additional monitoring and care – you will need to attend regular tests and check-ups.
  • Helping advance medical knowledge for the benefit of women now and in the future.

Often studies are randomised, so you will not know whether you are receiving the new or the standard treatment. Some research has shown that taking part in a trial improves long-term survival, even if you do not have the drug/procedure being tested. The reasons for this are not clear, however it has been suggested that hospitals which undertake medical research provide better treatment.

You also need to consider that new treatments are not always better than standard treatments. The new drug or treatment might not work for you and sometimes there are unexpected side effects. 

As trials compare new treatments with standard treatments, you may be selected to receive either the new treatment or the standard treatment. So by agreeing to be in a trial, it does not necessarily mean you will receive the new drug being tested. 

How do I find out about clinical trials?

You may be eligible to take part in a trial. They have strict criteria for joining them to make sure that the results can be relied upon by comparing like with like, and not all treatment centres are involved in trials. Your oncologist should know what is possible, but sometimes you may need to ask specifically about clinical trials. 

You might want to ask:

  • What trials are you eligible for at your treatment centre? 
  • If you’re willing and able to travel, what is available at another centre?
  • What is the aim of the trial? 
  • What is the evidence that this new treatment might be effective?
  • What are the possible risks and benefits of taking part?
  • What taking part would involve compared to not taking part?

You may also be asked to take part in research studies, for example into your wellbeing, which may involve taking part in interviews and surveys. 

Remember, if you are suitable, it is your decision whether or not to join a clinical trial. 

You can also find out about trials across the UK by visiting our Ovarian Cancer Clinical Trials Information Centre.

Having discussed the possibilities for treatment with your oncologist, you may want to seek a second opinion, either at your current hospital, or one which is more involved in research and trials. Your Clinical Nurse Specialist (CNS) should be able to advise you on how to get a second opinion.

Funding and access to drugs across the UK

The approval process for cancer drugs

Licensing

Once a cancer drug has completed clinical trials and there is evidence to show it is effective, it must be granted a licence before it can be widely used in the UK. A licence indicates all the proper checks have been carried out during its development and manufacture and the benefits of a medicine are believed to outweigh the risks. It must also demonstrate that it works for the purpose it is intended for it to be licensed. This licence is also known as a marketing authorisation.

In the UK, licences can be granted by:

  • the Medicines and Healthcare Products Regulatory Agency (MHRA) - which can grant licences for medicines only in the UK
  • the European Medicines Agency (EMA) - which can grant licences for medicines in the European Union (EU)

Funding

Once a drug is licensed it must be approved either by the National Institute for Health and Care Excellence (NICE) for use in England or the Scottish Medicines Consortium (SMC) for use in Scotland. Northern Ireland and Wales have processes in place for approving decisions made by NICE on new treatments and these are typically implemented in each nation.

In England, the manufacturer of the new treatment will apply to NICE for the drug to be approved for use by the NHS. This assessment is called a Technology Appraisal. As part of the assessment process NICE consults with stakeholders, including Target Ovarian Cancer, to ask them about the difference the new treatment would make. The SMC in Scotland follow a similar process. New treatments are assessed on the basis of how effective they are and how much they cost. 

Previously, cancer drugs in England could also be approved for use under the Cancer Drugs Fund (CDF). However, following reforms in 2016 the CDF has changed and now only provides interim funding for new cancer drugs until they are able to complete their full NICE approval. 

Standard drugs and treatment 

Most women with ovarian cancer will be offered standard treatments by their oncologist. This means drugs that are licensed for treating women with ovarian cancer in the UK and approved for use within the NHS on the grounds of clinical and cost effectiveness.

Read more about standard drugs and treatment for ovarian cancer for women recently diagnosed here, and for women if their cancer has come back here.

Non-standard drugs and treatments

Some women may wish to ask about other ways to access different drugs that have not yet been licensed to treat ovarian cancer or not approved for use on the NHS. Sometimes oncologists prescribe drugs to treat women with ovarian cancer (outside the clinical trial setting) that are not yet licensed for ovarian cancer if they believe she may benefit, for example, the drug may have been licensed for use in a different type of cancer. This is referred to as prescribing ‘off licence’ or ‘off-label’. Such drugs are not automatically funded on the NHS in any part of the UK.

An oncologist may also choose to prescribe a drug which is licensed but not yet approved for NHS funding. In either case the oncologist may well have to make a special application for funding for the drug which may or may not be accepted.

Occasionally manufacturers of the drugs in question will run a compassionate access scheme for patients who meet certain criteria, meaning the drug company meet the cost; however approaches to the drug company must be made by your oncologist.

It is important to note it can be quite stressful going through this process at a time when you are unwell. If your oncologist is reluctant or unsure about discussing other drugs, you can always ask for a second opinion. You will always need the support of an oncologist, as they have to make the applications for funding on your behalf.

 Glossary of terms used in clinical trials

There are many terms used in relation to clinical trials which may be difficult to understand, as they are mainly used by researchers and other healthcare professionals. Below is a glossary of some common terms used so that you are able to stay as informed as possible throughout your clinical trial.

Adherence

Keeping to the treatment given in a clinical trial.

Adverse events (AE)

Unwanted effects and symptoms that may or may not be caused by the trial treatment such as nausea, pain, low blood counts. If the symptom or problem is caused by the treatment it is known as a side effect.

Blinding and double blinding

Blinding means that the people having trial treatment do not know which treatment they have been given. Double blinding means that the people on treatment and their doctors also don’t which treatment is being given (until the end of the trial when all the data has been collected).

Clinical trials

These are research studies. They compare different treatments or treatment methods and test and find out whether one treatment is better. All new treatments and drugs must be tested this way to get evidence for the benefits and risks of the new treatment.

Closed to recruitment

This is when no more new people can join the trial. However, existing participants continue to be followed up as part of the trial. When all the trial data needed is collected the whole trial is then closed.

Cross-over trial

A trial where the treatment changes partway through the trial by participants crossing over from one treatment group to the other.

Data monitoring committee

This is an independent committee of experts that oversee the trial’s progress. They may advise changes to the trial or stopping it if they see evidence of unexpected problems for the participants. Also in cases where one treatment is obviously better than another, they can advise that a trial is stopped.

Disease progression

The course of a disease over time.

Eligibility criteria

The clear rules about who can or cannot join the trial, that is whether a person is eligible or not.  The rules are known as inclusion criteria (those that can join) such as having a particular type of disease or stage of disease and exclusion criteria (those that cannot join) such as being over or under a certain age or being pregnant.

Enrolling

The signing up of people to join a clinical trial. This involves matching a potential participant to the eligibility criteria of the trial and going through the informed consent process. (See informed consent.)

Ethics committee

A committee of a mixture of healthcare professionals and lay people who review funded clinical trials. This ensures the trial is run to appropriate ethical standards. The trial must be approved by an ethics committee before people are recruited to the trial.

Evidence base

The best available scientific research available about a disease or health problem. This is used to make decisions about the best treatment to give.

Good Clinical Practice (GCP)

This is an international quality standard for the conduct of clinical studies. Randomised Clinical Trials are required by law to conform to GCP.

Health economics

This is when some trials look at and compare the cost of effective treatments.

Informed consent

This is when a person who is being asked if they want to join a clinical trial, agrees to take part of their own free will. They must be given all the information about the trial, usually both in verbal and in written form. They must also have the chance to ask questions and have time to think about their decision before giving their consent to join the trial. Usually a consent form is signed and dated by both the participant and the trial doctor. A person is still able to withdraw anytime from a trial even after giving informed consent, without giving a reason and without it affecting their healthcare.

Interim analysis

An analysis of trial data which is done at an appropriate time before the end of the trial to check on progress of the trial.

Intervention

This is the treatment or method which is being looked at and tested in the trial, that is hoped to be an improvement on the usual standard treatment,

Meta-analysis

This is a way to combine the numerical results of several trials about the same treatment to provide an ‘average’ estimate of the effects of the treatment.

Open label trials

This means that the people on the trial and their doctors know which treatment they are having.

Open to recruitment

A trial that can still have more people joining.

Outcome Measures

These are measures of health and well-being such as the response to treatment or the recurrence of disease.

Participant

A person that takes part in a clinical trial

Patient Information Sheet (PIS)

This describes all the relevant trial related information in detail. It needs to be read and understood by the potential participant before informed consent is given.

Placebo

This is a dummy treatment. It is designed to have no effect. It looks, smells and tastes like the treatment being tested, in this way the trial participants do not know if they are taking the dummy treatment or real treatment (see blinding).

Prophylaxis

This is a measure to prevent a possible health problem, such as a vaccination. Or taking anti-sickness treatment to prevent feeling sick before the sickness starts.

Protocol

This is the overall plan with the rules and information for the clinical trial. It provides information and background on the disease and the treatments being tested and why they are being tested. The protocol also states the eligibility criteria for participants and the timetable of the trial. Protocols have to be approved by an ethics committee.

Quality of life

This looks at and measures the impact of the trial treatment on people’s quality of life. It may check on tiredness and mood, sense of well-being, activity levels and sleep patterns.

Randomisation

A computer will decide at random which treatment a trial participant will have, so that each person on the trial has the same chance of getting the treatments that are being compared and tested. It ensures that the groups of people being compared in a trial are as similar as possible apart from the treatment they have. This means that any differences in outcomes are more likely to be due to the treatments being compared.

Randomised controlled trial (RCT)

This is a trial that has a treatment group (people getting the treatment being tested) and a control group (people getting either a placebo or the usual standard treatment). People are placed at random into the different groups, which means there is a fair comparison being made between the trial groups.

Serious Adverse Events (SAE)

These are adverse events, which may or may not be caused by the trial treatment, that are regarded as more important and serious. Such as an unexpected stay in hospital or a life threatening problem that happens whilst participating in a trial. All SAEs are reported to the MHRA (Medicine and Healthcare products Regulatory Agency). (See adverse events)

Side effects

If the symptom or problem is caused by the trial treatment, it is known as a side effect.

Sponsor

The organisation responsible for the trial.

Standard treatment

An approved and widely used treatment that is considered to be effective for a particular disease or condition.

Systematic review

A collection of evidence from all the global research studies relevant to a particular research area. It gives a reliable way of reviewing the evidence. When researchers combine the numerical results of these trials and compare them, this is called a meta-analysis. (See meta-analysis)

Trial arm

This is one of the groups within a randomised controlled trial. One or more of the groups are treatment arms and one is usually a control arm. The people in the control arm get either a placebo (dummy treatment) or the usual standard treatment.

Trial Management Group (TMG)

This is a committee of trial researchers responsible for the trial being set-up, the day-to-day running of the trial and the release of any trial results or publications. It includes doctors, nurses, trial and data managers, statisticians and patient and public representatives.

Trial phases

Clinical trials are conducted in several phases:

Phase I: to test treatment safety in a small group of people.

Phase II: looks at the effectiveness of the treatment being tested, and usually has a larger group of people.

Phase III: Compares two or more treatments and checks on side effects. The trial results are used to make recommendations about the licencing and use of the treatment.

Phase IV: This is a post-marketing trial (when the treatment is already in use) and collects wider information on use of the treatment.

 

The information on this page is approved by the Information Standard scheme to ensure that it provides accurate and high-quality information.

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Last reviewed: August 2017
Next review: July 2019