Clinical trials information
On this page you will find some useful information about clinical trials, such as what are clinical trials and why are they needed, what are the different types of clinical trial, who conducts clinical trials, and how do I decide whether to participate in a clinical trial.
There is also a really useful section listing some questions that you might like to ask your doctor to help you decide whether or not to take part in a clinical trial.
Please click on the + on the left hand side of each line below to access the information held in the corresponding section.
All new medical treatments, drugs and devices need to be vigorously tested to ensure they are safe and effective for treating medical conditions and diseases in humans. Treatments are tested both in the laboratory and in the clinic. Laboratory tests are done to ensure they are safe for human use. They are then tested in the clinic in medical research studies involving people; there are called clinical trials.
Pharmaceutical companies use the data collected during laboratory testing and clinical trials to form a product license application. This application is submitted to the regulatory authority in each individual country to obtain a license to market and sell the drug. In general, it can take 10-15 years to complete the tests on new medicines and for the medicine to get to the market place.
A clinical trial is a medical research study involving people. Clinical trials test the safety and effectiveness of a new medical treatment, drug, or device. They can also be used to look at:
- The risks and causes of cancer, for example lifestyle factors and genetics
- New and better ways of preventing, screening and diagnosing cancer
- New ways of treating cancer, such as new types of treatment, new combinations of treatments, or different ways of giving treatment
- Side effects to treatments and ways of controlling them.
In the following video interview, Brenda Martone a Nurse Practitioner from Chicago, USA discusses how she approaches patients about participating in a clinical trial. She discusses the definition and types of clinical trials and phases of trials that may impact a patient’s decision about participation. Benda shares insights into her approach of informing patients about what clinical trials are truly about versus some of the myths or preconceived notions that patients may have based upon terminology.
All new treatments are tested first in the laboratory. Only treatments that are likely to work in people are tested in clinical trials. First they are tested in a small group of healthy volunteers before giving them to larger groups of patients in clinical trials.
When a new medical treatment is studied for the first time in people, researchers don’t know exactly how it will work. With any new treatment, there are possible risks as well as benefits. Clinical trials help the researchers discover the answers to these questions:
- Is the treatment safe and does it make you feel better?
- How well does the treatment work?
- Is the treatment better than the treatments already available?
- What are the side effects and risks of the treatment?
Pilot and feasibility studies
Pilot and feasibility studies are usually run before the start of a larger clinical trial to look at:
- The feasibility of running the larger trial. In other words, to see if it is possible to actually carry out the larger trial
- How the main parts of the larger trial will work on a smaller scale (a pilot study).
1. Feasibility studies
Researchers might carry out a feasibility study before starting a much larger clinical trial to see if the larger trial can be done. Feasibility studies look at things such as:
- The patient population, and whether the research team are likely to recruit enough patients for the larger clinical trial
- The design of the clinical trial, and whether people would be willing to be allocated to treatment groups at random (randomisation)
- The assessments and hospital visits needed for the trial, and whether people are able to complete them
- The length of the larger clinical trial, and whether people would be willing to stay in the trial until the end
- Feasibility studies do not help to answer any of the research questions of the main clinical trial.
2. Pilot studies
Unlike feasibility studies, pilot studies may also help answer the research question., and sometimes form the first part of the main clinical trial. The data generated from the pilot study can be used when the results from the main clinical trial are analysed.
Investigational clinical trials
There are 4 phases of clinical trials:
Phase I clinical trials are the first studies in humans. These trials are conducted with small numbers of patients or healthy volunteers (usually male). They are used to investigate the most effective dose of the drug in humans and to determine what side effects are most likely to occur.
Phase II clinical trials investigate the safety and efficacy of the drug in larger numbers of patients (100s) with a focus on the effectiveness of the treatment at different doses, and its safety in the target patient population.
Usually, when phase II is completed the pharmaceutical company will decide whether the safety and efficacy of the drug in the target patient population warrants further development. The drug will only be taken into phase III development if there is a very good chance of the drug meeting the strict safety and efficacy guidelines stipulated by the regulatory authorities.
Phase III clinical trials involve large numbers of patients (1000s), usually on a global scale, and are used to compare the efficacy and safety of the new drug with either the standard existing treatment or placebo (dummy treatment). Phase II clinical trials also study the new treatment in different patient populations and different dosages and combinations of drugs.
Phase III studies are usually randomised and blinded so that the patients (and the doctor in the case of double-blind clinical trials) do not know which treatment they are receiving to avoid a biased interpretation of the results. Data obtained from phase III clinical trials are used to demonstrate the benefits of the new treatment over existing treatment or placebo.
Phase IV clinical trials collect information on the use of the new treatment when used in routine patient care (i.e. after a product license has been granted for marketing). For example, a new treatment may be assessed to see how it is being used in the clinic together with other effective treatments to treat a particular disease or medical condition in a select group of patients.
In an observational study, the researchers do not intervene in the care of the patient, but simply observe and record what happens. During investigational clinical trials, the researchers intervene in the care of the patient in a pre-planned way and record the outcome. Observational studies often use questionnaires and survey to record information about the life styles and care of the people in the studies.
There are two main types of observational study; case-control studies and cohort studies.
Case control studies
These are retrospective studies in which groups of patients with cancer (cases) are compared to groups of patients without cancer (controls). The aim is to see whether exposure to any factor occurs more or less frequently in the cases compared to the control group.
An example of a case control study would be the comparison of smoking habits in people with lung cancer (cases) and people without lung cancer (controls) to determine a link between smoking and lung cancer.
Case control studies are relatively cheap, quick and easy to carry out. There is no loss-to-follow-up and they are suitable for rare events. However, the timing of events cannot be reliably established and it is difficult to assess causality. Also, case control studies rely on people remembering events, and are, therefore, subject to recall bias.
Cohort studies follow a group of people over time to assess the incidence of a disease (or some other event). Cohort studies are used to describe the effect of being exposed to one or more risk factors on the incidence of the disease. Cohort studies can be prospective (planned) or retrospective (historical).
Cohort studies can be used to assess the relationship over time between exposure to certain risk factors and the development of the disease. They can also be used to assess cause and effect, although randomised clinical trials are preferable for this. However, for rare diseases, the cohort may have to be very large with a long follow-up, resulting in an expensive study and many people being lost-to-follow-up.
Screening is done to detect cancer before it has started to cause symptoms i.e. for the early diagnosis of cancer. Screening is used to detect particular cancers, either in the general population (for example, breast cancer screening), or in people with a higher risk of developing cancer (for example, bowel cancer screening in older people).
Prevention trials are used to determine whether a particular treatment can prevent cancer from developing. They are carried out with people who do not have cancer, and can be done with the general population, or a group of people at higher risk of developing cancer.
Clinical trials involve a number of different organisations. Often, clinical trials are designed, planned and sponsored by a pharmaceutical company or a public health agency. These organisations may subcontract the implementation, monitoring and reporting of clinical trials to a contract research organisation (CRO). The design, monitoring, analysis and reporting of clinical trials require specialised scientific expertise contributed by the staff employed by the pharmaceutical company, the health agency, or the CRO.
Clinical trials are conducted in hospital departments, GP surgeries, or specialist phase I units, depending on the phase of the trial and the patient population under investigation. There will be a team of doctors, scientists, nurses and other medical and healthcare professionals running the clinical trial, which will be led by a doctor with a number of years research experience (the principal investigator).
Before a trial can start, it is reviewed by the regulatory body of the country where the trial is taking place to ensure strict regulatory guidelines are being followed in the design and planned conduct of the trial. It is then reviewed by an Ethics Committee (or Ethical Review Board) to ensure that the patient’s or volunteer’s rights are protected, and the highest standards of clinical practice will be observed to ensure the safety of the patient or volunteer.
When deciding whether to take part in a clinical trial, you should be clear about the purpose of the clinical trial and what is expected of you. You need to consider the following fully before you enroll in a clinical trial;
You will be cared for by doctors and nurses with detailed knowledge about the latest treatments for your condition.
You will be monitored very closely during the trial and may benefit from additional testing that would not normally be carried out in routine clinical practice.
You will be given the opportunity to be one of the first patients to benefit from the new treatment under investigation; if you are not responding well to current therapies, participation in a clinical trial may give you access to a treatment that will work better for you.
However, you may experience unexpected side effects to the new treatment and/or it may not be an effective treatment for you. The new drug may prove to be less effective than current available treatments.
Close monitoring of the trial may result in more frequent hospital visits and more testing than would occur if you were not on the clinical trial. This could be disruptive to your life.
You should discuss any concerns or issues you have regarding your health and care during the clinical trial with your doctor before enrolling. It is very important to be fully informed about the purpose of the clinical trial, potential benefits and side effects of taking the new treatment, and what is expected of you in terms of hospital visits and tests during the clinical trial.
Phase III clinical trials usually compare a new treatment with either current treatment or a placebo or dummy treatment. It is important to remember that in these clinical trials treatments are randomly allocated and usually neither you nor your doctor will know which treatment you are on until the end of the trial. You or your doctor will not be able to choose which treatment you receive. At the end of the trial, if you were receiving the least effective treatment you might be given the opportunity to switch to the more effective treatment; your doctor will discuss this with you.
For participation in a clinical trial you will always be asked to sign a consent form to indicate your agreement to take part and that you are fully informed about the treatment under investigation and what is expected of you during the trial. You have the right to withdraw from a clinical trial at any time, and you do not have to give an explanation. Your care at the hospital or clinic will not be affected in any way and you will be offered current, existing treatment for you condition.
All information collected during a clinical trial, including your personal details and case notes, will be kept strictly confidential. The organisation responsible for analysing and reporting the results of the trial will identify your test results and details by a number and your initials only.
In the following video, Clare, a clinical trial participant openly shares her experiences. She shares her thoughts on her decision to participate in a randomised clinical trial, her experiences with the consent form and the issues she considered when joining the trial. She is candid in her discussions. She gives us the opportunity to reflect on the options presented by being a part of a randomised clinical trial.
Questions about the trial
- What is the purpose of the trial?
- How long will I be in the trial?
- How many assessments/hospital visits are there and when do they take place?
- What kinds of tests and treatments are involved?
- How will the doctor know if the treatment is working?
- How will I be told about the trial’s results?
- How long do I have to make up my mind about joining this trial?
- Who can I speak with about questions I have during and after the trial?
- Who will be in charge of my care?
- Is there someone I can talk to who has been in the trial?
Questions about risks and benefits
- What are the possible side effects or risks of the new treatment?
- What are the possible benefits?
- How do the possible risks and benefits of this trial compare to those of the standard treatment?
Questions about your rights
- How will my health information be kept private? And what happens to my health information after the trial?
- What happens if I decide to leave the trial?
- Do I have to give a reason if I decide to leave the trail?
- Will my treatment and care be affected if I decide to leave the trial?
Questions about costs
- Will I have to pay for any of the treatments or tests?
- Will I have to pay for travel to the hospital for trial assessments/hospital visits, including car parking if I travel by car?
- If I don’t have my own transport, can I get help travelling to the hospital for trial assessments/hospital visits?
Questions about daily life
- How could the trial affect my daily life?
- How often will I have to come to the hospital or clinic?
- If I need to go to another hospital for some of the tests, how far away will this be?
- Will I have to stay in the hospital during the clinical trial? If so, how often and for how long?
- Will I have check-ups after the trial? If so, how often are the check-ups?
Questions about other treatments
- What are the other treatments for my kidney cancer, including standard treatments?
- How does the treatment I would receive in this trial compare with the other treatments?
Translational research is research that applies findings from basic science to enhance human health and well-being. In a medical research context, translational research aims to “translate” findings in fundamental research into medical practice and meaningful health outcomes.
Translational research is often referred to as from ‘bench-to-bedside’ research; research from laboratory experiments through clinical trials to administration to the patient. Translational research uses knowledge from the basic sciences to produce new drugs, devices, and treatment options for patients.
Translational research helps turn early innovations into new health products. Research in universities, pharmaceutical companies and charities produces many new discoveries and inventions, which have the potential to improve health; however, turning these ideas into useful products can be extremely difficult.
Researchers need to bring together the right combination of medical, scientific and business expertise to develop the product and position it within the market. To be able to do this, they need to anticipate what the market will be looking for, and how the product will be introduced alongside the competition. Translational research helps to overcome these obstacles, bridging the gap between basic research and a deliverable product.
The following websites contain information about clinical trials and medical research. There are also some links to clinical trial databases that you can use to search for clinical trials. Additional resources, such as videos, online courses and websites to help you get to grips with the clinical trial process can be found on our Clinical Trial Resources page.
Please note, that we cannot be held responsible for the contents of any pages referenced by an external link.
NHS Choices: Clinical trials and medical research
UK Clinical Trials Gateway: About clinical trials
ClinicalTrials.com (American website): ClinicalTrials.com
The following links give information about clinical trials involving kidney cancer patients in the UK:
NHS Choices: Kidney cancer – clinical trials
UK Clinical Trials Gateway: Trials search
Clinicaltrials.gov: Find studies