Participating in a clinical study
Clinical studies are dependent on persons who volunteer to take part. As a study participant, you are part of driving research forward.
What is a clinical study, and why is it carried out?
Clinical studies are conducted in order to investigate a group of individuals with a specific disease, to test the efficacy and safety of new medicines, diagnostic tests, products or therapies. Before studies on human subjects begin, tests have already been carried out in several different ways in laboratory trials and animal studies. Clinical studies are necessary in order to answer questions about issues such as effects and side effects. They are also a prerequisite for new medicines, medical devices or therapies to be approved, and to ensure they can start being used within healthcare.
On the website, clinical studies are divided into two subsidiary groups: clinical trials and observational studies.
- Clinical trials are sometimes called intervention studies or experimental studies. A clinical trial is a study that investigates the efficacy of a medicine, a medical device, a diet or a therapy method, for example, on healthy or ill persons.
- Observational studies are sometimes called non-intervention studies, or non-experimental studies. In an observational study, the researchers study the study participants without actively influencing for example the therapy method.
In a clinical study, you investigate for example:
- the problems experienced by people who have or are at risk of a developing a specific disease
- how a new diagnostic test can be used
- how well persons cope with a therapy
- how a therapy can be used in the most effective way
- how a medicine is absorbed, works and breaks down in the body
Why should you participate in a clinical study?
There are several reasons why you might like to participate in a clinical study. It often means a thorough examination, and as a study participant, you also contribute to increased knowledge about diseases or therapies. Depending on how the clinical study is designed, it is not guaranteed that all participants are offered to try a new therapy. Those who do not will be part of a control group that is either prescribed sugar pills, called placebo, or an already approved medicine, which the new therapy is compared to. Even if you may not receive any direct therapy benefit, it can still feel meaningful to contribute to studies that may be of benefit to other people.
How is a clinical study conducted?
Clinical medicine studies can be conducted in many different ways. They last for varying amounts of time, depending on the type of disease the new medicine is intended for. To begin with, small clinical studies are carried out using a few, often healthy study participants, and then you continue with the patient group for whom the medicine is intended. The efficacy and safety of the medicine is then confirmed in major, often very large studies, sometimes involving tens of thousands of patients in several countries.
The different phases of a clinical study
A clinical study is conducted according to a study protocol set in advance. A study may last from a couple of weeks up to one year or more, depending on the disease and the nature of the therapy. The results from clinical studies constitute an important part of the documentation required to get a medicine approved for sale, and are often a requirement in order to get a medical device CE-labelled and marketed. Often, it is also required that companies or researchers carry out studies of the safety of a medicine even following an approval.
The description of the various phases of a clinical study apply for clinical trials with medicines, but the phases are similar also for clinical trials with medical devices.
Phase 1 often uses healthy study participants, around 20–80 individuals who are usually men, to study the safety of the product. A substance is investigated for any side effects, how the body breaks it down and disposes of it, and what the pharmacological characteristics are. Phase 1 starts by testing a very low dose of a substance, and the dose is then gradually increased. Between each increase, a comprehensive safety evaluation is made.
For Phase 2, study participants with the disease or the condition that will be treated with the new therapy are recruited. The number of study participants is still limited, and the patient group should be as homogenous as possible. During Phase 2, a first indication of the efficacy of the therapy on a certain disease, and the optimal dose are obtained. The new therapy is compared to existing therapies or placebo, and further information about the safety of a substance and any side effects is obtained.
During Phase 3, large patient groups of between 200 and 3 000 individuals or more are studied, to confirm the effect and safety of a therapy compared to standard therapy or placebo over a longer period of time. The dose used is the one found most suitable during an earlier phase. The patient groups shall resemble as far as possible the group on which the therapy will be used (for example gender, weight and age). So-called “interaction studies” are also carried out, where the reactions to the substance in question of patients who are simultaneously receiving other therapy are looked at.
If the Phase 3 studies are successful, an application can be made to have the therapy approved by the Medical Products Agency.
Once the medicine is on the market, major studies are carried out to map unusual side effects and to monitor the safety, effectiveness and optimal area of use of the therapy.
To make sure that a clinical study investigates the effect of the medicine, and not the effect of the doctor’s or the study participant’s expectations, studies are often double blind. That means that neither the doctor nor the study participants know what kind of therapy each study participant is receiving. After the completion of the study, it is revealed who has been given the new medicine and who has been given the standard therapy or placebo.
Before a clinical study begins, there must be an ethical approval. This means that anyone who want to conduct a clinical study must first apply for approval from the Ethical Review Authority. To receive approval, the expected benefit of the study must outweigh any risks. The Ethical Review Authority also ensures that the information given to the study participants is clear enough for them to decide whether to participate or not. Clinical trials using medicines and medical devices must also have an approval from the Medical Products Agency.
What does participating in a clinical study mean?
Participating in a clinical study is voluntary. Before accepting, you are entitled to receive both verbal and written information about the planned study. For example, you will get information about:
- the purpose of the research
- the therapy to be given and the additional examinations that need to be done
- how long time it will take
- if there are any restrictions in terms of lifestyle
- how personal data is handled and stored
For clinical trials with medicines, a doctor will give information verbally and it must also be possible to ask a doctor questions during the course of the study.
If you decide to participate, you must give written consent, and at the same time confirm that you have received sufficient information about the study. No clinical trial with medicines may start before this is done. If samples are taken in conjunction with the study, you must also sign a consent according to the Biobanks in Medical Care Act. You may also need to give your consent to study personnel who will have access to the collected study data.
It is important to follow all instructions and to attend the check-ups and planned visits included in the clinical study. Otherwise it might be difficult for the researchers to draw the correct conclusions. If it is difficult for you to follow the instructions for any reason, it is important to tell the personnel conducting the study. The same applies if you start receiving any other therapy while the study is in progress.
Can you end your participation early?
If you no longer want to participate in the study, you are entitled to stop participating at any time – without any explanation. It is important that you inform the study personnel so about your decision. You may then be asked to attend a concluding examination. Any unused medicines and other materials must also be returned. If the decision to end your participation is due to problems that might be a side effect of the therapy, it is important to inform the study personnel about this, as suspected side effects must be reported. After ending your participation in the study, you should return to your former therapy, if applicable.
How do you sign up for a clinical study?
There are various ways to sign upp for a clinical study. Examples are:
- being part of a relevant patient group
- actively looking for studies on the websites of patient organizations or pharmaceutical companies
- searching for information on the websites of the regional nodes
If you are part of a patient group on which researchers want to try out a new medicine or a new method, it may happen that your doctor will ask if you want to take part in a clinical study. If you are interested in becoming a study participant, an examination will be made to make sure that you fulfil the requirements for participants in the study in question before the study therapy starts, so-called “pre-screening”. Taking part is always voluntary.
Apart from being asked directly by healthcare, you can also look for suitable studies to take part in yourself. Sometimes, information about any clinical studies in progress can be found in newspapers or on the websites of patient organizations. Individual pharmaceutical companies and also companies that specialize in conducting pharmaceutical studies on behalf of pharmaceutical companies, known as “CRO companies”, may also inform about clinical studies on their own websites.
Some of the regional nodes that are part of Clinical Studies Sweden have information on their websites about studies in progress. Some of the websites also have forms where you can register your interest in participating in clinical studies.
What happens to the collected data?
In a clinical study, study data is collected and stored in a patient form, which does not include either name or personal identity number. Instead, a code is used, which can be linked to the individual study participant once the study is concluded. Only the clinical investigator responsible and other study personnel have access to the information that can link a particular code to a particular study participant.
In clinical trials, the data in the patient form needs to be checked against the data in the patient’s medical records. A representative of the company that manufactures the medicine, or the clinical investigator responsible if no company is involved in the study, therefore needs to have the right to read the study participants’ medical records. The Medical Products Agency or the corresponding authorities in other countries may also carry out similar checks. As a study participant, you therefore need to give your written consent to these officials reading your medical records if you want to participate in the study.
Data protection legislation regulates if and how personal data can be managed for research purposes. Individuals participating in clinical studies have the right to be informed about if and how their personal data are used, including information about who is responsible for processing the data, the purposes for which the data is processed and how long they are stored. In some situations, the individual whose data is processed is also entitled to get their information corrected or deleted.
In some studies, data is transferred to countries outside the EU or ESS. The General Data Protection Regulation (GDPR) contains provisions that regulate the cases in which such transfers may occur. Transfer of data to a country outside the EU or ESS is, inter alia, permitted if the study participant has explicitly agreed to the transfer after he or she has been informed of the risks involved.
More information about personal data processing can be found in the section on laws and regulations and on the Data Inspection website.
When presenting the scientific results, no data on the patients taking part in the study are included.
Who is responsible for a clinical study?
A clinical trial shall always have a responsible clinical investigator who is a registered doctor or dentist, and a medical expert. As always, they of course have ethical and medical responsibility for their patients.
Many clinical studies are started and conducted by pharmaceutical companies or companies that develop medical devices. However, the studies are always conducted in collaboration with doctors and other healthcare personnel. Doctors can also initiate and conduct studies, for example in order to investigate whether a medicine that is already approved may also be effective as therapy in other diseases.
Good Clinical Practice (GCP) is an international ethical and scientific quality standard for designing, conducting and reporting on clinical studies using medicines or medical devices. The purpose of GCP is to safeguard the rights, safety and well-being of the study participants.
Swedish Research Council’s brochure “Good Research Practice”
The organization or the company that conducts a clinical study, together with any clinics and doctors participating, have considerable obligations and responsibilities. The responsibility covers areas such as:
- ensuring the personnel participating in the study have the competence, time, personnel, equipment and the patients required
- ensuring the personnel have sufficient training in the therapy to be studied and how clinical studies shall be conducted
- ensuring there is approval from the Ethical Review Authority before the study begins
- ensuring all study participants have agreed to participate before any study-related samples are taken and therapy started
- ensuring the study is carried out according to plan, that is according to the study protocol
- ensuring that data collected about the study participants match with their medical records
- trying in a respectful way to find out the reason why a study participant wishes to end participation, although the participant has no obligation to state why
- ensuring that study participants receive correct therapy for any side effects caused by the study therapy, and that all side effects arising during the study are reported to the Medical Products Agency
- ensuring that the study results are presented in a scientific manner and that documentation on the study can be reviewed afterwards
- permitting public agencies to inspect the clinic
- ensuring that there is sufficient insurance cover in the event any loss arises
What protection is available?
The safety and well-being of the study participants has the highest priority, but there is no 100 % guarantee that taking part in a clinical study is risk-free. Although the therapies, including medicines, have been investigated in lots of different ways, including trials on animals, it is not possible to be entirely sure that humans will react in the same way. For example, allergies that humans can suffer from may be difficult to discover in animals. Even though the risk of unexpected events that lead to damage is small, anyone carrying out a clinical study must have an insurance in order to compensate patients who may suffer.