Clinical trials Factsheet

Transcription

1 Clinical trials Factsheet This factsheet talks about the main aspects of clinical trials, including what they are, how they are organised and what they may involve.

2 02 Introduction Clinical trials 03 You may have heard about a clinical trial that you would like to take part in, or your doctors may have offered you the opportunity to take part in one if you are eligible. Whatever your situation, deciding what to do can be very difficult, particularly as information about clinical trials can be confusing. You may feel that you already have a lot to take in about your treatment, before even thinking about clinical trials. It can also be hard to absorb extra information at a time when you may already be feeling overwhelmed. This factsheet aims to answer some of your questions and help you discuss your options with your doctors, so that together you can decide what is best for you. What are clinical trials? Clinical trials are research studies that aim to improve different aspects of patient care. They are a routine part of the work of specialist breast cancer centres. The results of previous clinical trials have given doctors the information they need to make decisions about the treatment offered today, and improve upon what was offered in the past. Similarly, the clinical trials carried out today will enable doctors to improve on the treatment available for patients in the future. There are many different types of clinical trials. Some look at methods of trying to prevent breast cancer and others look at diagnosing or treating it. Other trials may look at the effects a particular treatment has on your quality of life, for example by looking at the extent of side effects. Many breast cancer trials look at new treatments, or different ways of giving existing treatment, such as surgical techniques or chemotherapy. Ideas for clinical trials are first tested and researched thoroughly in a laboratory. The plan for how a trial will be carried out has to be approved before it begins, first by a group of independent scientists and then by a research ethics committee. Why are clinical trials necessary? Clinical trials are necessary for the development of new treatments or combinations of treatments. Trials are the only way of comparing the new treatments with the best existing treatment to see which is better. How long does a trial take? The length of time clinical trials take to complete and publish the results varies from a few months to several years depending on what they are looking at. For example, a trial looking at the long term side effects of radiotherapy may collect information from participants over a number of years before it is known what the long term effects are. Who is suitable to take part in a clinical trial? Every clinical trial has strict guidelines (eligibility criteria) about who can be involved. For example a trial might only be for people with a particular type or stage of cancer, or within a particular age range, which means that not everyone is able to take part. If you are not able to take part in a particular trial, you may be disappointed or feel as if there are limited options for the future. However, it is important to remember that your specialist team will continue to support you and offer the best treatment available. If you are interested in taking part in a trial, it is important to talk it through with your cancer specialist, as they will be familiar with your medical history. They will be aware of major breast cancer trials that are currently in progress and which ones are happening in your area. They will be able to advise you according to your individual situation.

3 04 Deciding to take part in a trial Clinical trials 05 Deciding to take part in a trial If you have been asked to take part in a trial, your specialist or a research nurse will discuss with you exactly what is involved. This might include the type of treatment you may receive, what possible benefits or known side effects you might expect or what extra tests you may need. Questions you might like to ask include the following. How many people are on the trial and what have their experiences been? How long will I be on the trial? If I have to have extra tests what are these likely to be? Will I need to donate blood or tissue samples, and if so, is this an optional part of the trial? Will there be a questionnaire or diary to fill in? How might the treatment affect me physically and emotionally? Will I have to have extra hospital visits? If so, will the trial pay for my travel to and from hospital? Is the data collected about me kept confidential? Does anyone else have access to my records? Who will I be able to contact if you (research nurse) are not available? What treatment will I receive if I do not enter the trial? Once the details have been explained, you should be given written information and time to think it over before you make your decision. If you decide to take part in a clinical trial you will be asked to sign a form saying that you agree to take part and understand what is involved (this is called informed consent). However, this does not commit you in any way and you can still withdraw from the trial at any time if you change your mind. Taking part in a clinical trial may not be a suitable option for everyone. If you decide not to take part, you don t have to give a reason. Your doctors and nurses will continue to care for you and offer you the best current treatment available. Once the trial begins it will be overseen by a research ethics committee including breast cancer specialists and statisticians (people who interpret statistics). The committee helps to ensure that the ethical standards and scientific value of research involving patients are appropriate. The committee must protect the rights of research participants, by ensuring that they receive sufficient information, which can be easily understood, and that appropriate strategies are in place to protect them from any potential negative consequences of the research. If at any time during the trial it becomes clear that one treatment is much better or safer than the other, the trial will be stopped and all patients will be offered the most effective treatment. This occasionally happens in phase 3 trials (see page 7). You will usually be given a named person (normally the research nurse) to contact if you have any problems or are concerned about side effects. Your specialist should also tell your GP that you are taking part in the trial. Will I get to know the results?

4 06 How are new drugs tested? Clinical trials 07 How are new drugs tested? Each new drug goes through rigorous testing before it can be given to patients. First it is tested in the laboratory. If it shows promise, it then has to go through three phases of testing on patients in clinical trials before doctors are allowed to use it to treat patients routinely. Stories in the media sometimes describe a new drug as a major breakthrough when it is still at the stage of laboratory testing. In fact, at that stage it may still be several years away from being tested on patients in clinical trials, which can be disappointing and frustrating for people who see a new drug as a source of hope. If this affects you, it can help to talk to someone from your specialist team about how you are feeling. For further sources of support, see page 11. There are four phases of testing. Phase 1 trials If your breast cancer has not responded to standard treatment, you may be asked to take part in a phase 1 trial. This is the first step in testing a new treatment on people, and the drug is given only to small numbers of patients who are monitored very closely. People with different types of cancer can sometimes take part in the same phase 1 trial. Phase 1 trials see how much of the drug can be given safely and how often it can be given. This is called the maximum dose. They will look at the side effects, but not at the overall effectiveness of a treatment. However, there is a small chance that your cancer may respond to the drug. Phase 2 trials Once the maximum dose and side effects of the drug have been established, phase 2 trials can begin. A larger number of patients is needed at this stage. The aim at this point is to fine-tune (adjust) the dose and establish the precise number of patients who respond to see if it is likely to be an improvement on previous treatments. Phase 3 trials Once the new drug has shown promise as an effective treatment in a phase 2 trial, it is ready to be tested on a much larger number of patients in a phase 3 trial. The aim is to compare the new treatment with the best current or standard treatment to see if the new treatment is better. Some phase 3 trials aim to find out whether it is better to give a treatment for a longer or shorter period, or look at giving a treatment at a different point in the treatment plan. People who take part in phase 3 trials are divided into groups, which are usually (but not always), the same size. The groups then have different treatment. It is important that the groups of people are as similar to each other as possible. This is because the trial s organisers need to be sure that if one group does better than the other, it is because of the treatment, and not because the people in the groups are different from each other in some way. The only way to make sure that the groups are as similar as possible is by allocating people to a group at random.

5 08 How are new drugs tested? Clinical trials 09 You cannot choose which treatment you will receive. It is possible that you may not have the new treatment, in which case you will instead be part of the control group, which receives a standard treatment. This is usually the treatment you would receive if you were not in the trial. Being in the control group and not receiving the new treatment can be difficult for some people to accept. However, these patients are vital to the trial and will still be receiving the best known treatment to date. The number of patients taking part in these trials varies. They can involve up to several thousand patients in many cancer centres in many countries. Phase 4 trials These types of trials are sometimes referred to as post marketing trials as they are conducted after a treatment has been shown to work, and if it is a drug, has been licensed for use. Phase 4 trials look at treatments that are already available for doctors to prescribe, rather than new ones that are still being developed. The three main reasons for conducting phase 4 trials are to find out: more about the side effects and safety of the treatment what the long term risks and benefits are how well the treatment works when it s used more widely than in phase 1 3 clinical trials. Placebos and double blind trials In some trials a new drug is compared with a placebo, which has no active ingredients but is made to look exactly like the drug being tested. The patient will not know whether they are receiving the active treatment or the placebo. The doctor does not know either this is known as a double blind trial. You will be informed of this if it is the case. Using a placebo helps to reduce bias and means the trial results should be more accurate. If necessary, doctors can find out whether or not a patient is getting the new treatment, for example if there are unexpected side effects. By using a placebo, the effect of the new drug can be measured much more accurately. This is because sometimes people appear to do better just because they are involved in a trial, even if they are not given the new drug. It is not known why this occurs. It may be because they are monitored more closely or because they or their doctor are optimistic about the benefit of the new treatment, causing them to interpret signs more positively. Research on breast tissue In recent years, researchers have developed a greater understanding of tumour biology, and have recognised the importance of understanding how different treatments might effect how a cancer behaves. To help with this you may be asked to give your consent for tissue removed during a biopsy or surgery to be used for research.

6 10 Possible benefits and drawbacks of being in a clinical trial Clinical trials 11 Possible benefits and drawbacks of being in a clinical trial Benefits You may be one of the first to benefit from a new treatment. You are taking an active role in your treatment plan. You have the opportunity to help others and improve breast cancer treatment in the future. You will probably be seen more frequently by doctors than people not in the trial as extra information about the effects of the treatment may be needed. You may benefit even if you are having the standard treatment as you may have extra tests and therefore be monitored more closely. Drawbacks There may be unexpected side effects from the new treatment. The new treatment may not be any more effective than the standard treatment. The new treatment may be effective for some but it may not work for you. There may be extra hospital visits required which may not be convenient or desirable. The extra tests may make you feel more nervous or preoccupied with your breast cancer. You may not be in the treatment group you would like to be in. Further support Being diagnosed with breast cancer can be very stressful and you may experience many different emotions. Coping with extra decisions about your treatment can add to the stress. Each person responds differently to their situation and has their own way of coping. You may find it difficult to understand why there is a question over which treatment is best for you. Many people find they can gain some sense of control by finding out as much as possible about their condition and its treatment. It is important that you feel able to talk to your specialist or breast care nurse about any questions or concerns you may have. You may find it helpful to share your feelings with someone in a similar situation. You can do this in a support group or one-to-one with a trained Breast Cancer Care volunteer who has personal experience of breast cancer. We may also be able to arrange for you to speak with someone who has taken part in a breast cancer research trial (although it is unlikely to be the same type of trial as one you may be involved in). For information on any of these options, call our helpline. Breast Cancer Care Breast Cancer Care is here to support you. From diagnosis, throughout treatment and beyond, our services are here every step of the way. Our free, confidential helpline is here for anyone who has questions about breast cancer or breast health. Your call will be answered by one of our nurses or trained staff members with experience of breast cancer. Our website gives instant access to information when you need it. It s also home to the largest online breast cancer community in the UK, so you can share your questions or concerns with other people in a similar situation.

7 12 Further support Clinical trials 13 Our One-to-one support service can put you in touch with someone who knows what you re going through. Just tell us what you d like to talk about and we can find someone who s right for you. Through our professionally-hosted discussion forums you can exchange tips on coping with the side effects of treatment, ask questions, share experiences and talk through concerns online. If you re feeling anxious or just need to hear from someone else who s been there, they offer a way to gain support and reassurance from others in a similar situation to you. We host weekly Live chat sessions on our website offering you a private space to discuss your concerns with others getting instant responses to messages and talking about issues that are important to you. Telephone support groups offer the chance to be part of a regular support group which you can join easily by phone. We run Information and support sessions and courses for people living with and beyond breast cancer, bringing people together to share experiences. We also offer practical sessions to help with issues such as hair loss and finding a suitable bra after surgery. We also offer specific, tailored courses to younger women, and people living with secondary breast cancer. Other organisations Macmillan Cancer Support 89 Albert Embankment London SE1 7UQ General enquiries: Helpline: Website: Textphone: or Text Relay Macmillan Cancer Support provides practical, medical, emotional and financial support to people living with cancer and their carers and families. Over the phone, its cancer support specialists can answer questions about cancer types and treatments, provide practical and financial support to help people live with cancer, and are there if someone just wants to talk. Its website features expert, high-quality information on cancer types and treatments, emotional, financial and practical help, and an online community where people can share information and support. Macmillan also funds expert health and social care professionals such as nurses, doctors and benefits advisers. Our free information resources for anyone affected by breast cancer include factsheets, booklets and DVDs. You can order our publications by using our order form, which can be requested from the helpline. All our publications can also be downloaded from our website. To request a free leaflet containing further information about our services please contact your nearest centre (contact details at the back) or visit

8 14 Notes Notes A large print version of this factsheet can be downloaded from our website, It is also available in Braille or on audio CD on request by phoning This factsheet has been produced by Breast Cancer Care s clinical specialists and reviewed by healthcare professionals and people affected by breast cancer. Centres London and the South East of England Telephone src@breastcancercare.org.uk Wales, South West and Central England Telephone cym@breastcancercare.org.uk East Midlands and the North of England Telephone nrc@breastcancercare.org.uk Scotland and Northern Ireland Telephone sco@breastcancercare.org.uk We are able to provide our publications free of charge thanks to the generosity of our supporters. We would be grateful if you would consider making a donation today to help us continue to offer our free services to anyone who needs them. Please send your cheque/po/caf voucher to Breast Cancer Care, FREEPOST RRKZ-ARZY-YCKG, 5 13 Great Suffolk Street, London SE1 0NS Or to make a donation online using a credit or debit card, please visit Visit All rights are reserved. No part of this publication may be reproduced, stored or transmitted, in any form or by any means, without the prior permission of the publishers.

9 Breast Cancer Care is here for anyone affected by breast cancer. We bring people together, provide information and support, and campaign for improved standards of care. We use our understanding of people s experience of breast cancer and our clinical expertise in everything we do. Visit or call our free helpline on (Text Relay 18001). Interpreters are available in any language. Calls may be monitored for training purposes. Confidentiality is maintained between callers and Breast Cancer Care. Central Office Breast Cancer Care 5 13 Great Suffolk Street London SE1 0NS Telephone Fax info@breastcancercare.org.uk Breast Cancer Care, June 2010, BCC24 Registered charity in England and Wales ( ) Registered charity in Scotland (SC038104) Registered company in England ( ) Printed on recycled paper please recycle