A clinical trial is a carefully planned study with human volunteers to find out if a treatment is safe and effective. This kind of scientific study is key to all medical advances, because it helps answer these questions:
Each clinical trial focuses on finding a better way to understand, prevent, diagnose, or treat a disease. All clinical trials are scrutinised by outside experts to make sure the health of the participants is safeguarded. In addition, different phases of trials must be carried out to track safety and efficacy before a new treatment is approved and made widely available.
“Most treatments we use today are available because others have volunteered to take part in them.”
There are two types of clinical trials that people with NETs may be able to participate in: Interventional studies and observational studies. Both of these types of studies are important for increasing the understanding of diseases and their treatment.
The first is called an interventional study, which looks at a specific therapy or way of managing a condition (an intervention). These studies are an important part of figuring out what health effects are specifically caused by the treatment.
The second is called an observational study, which tracks the health of participants in a more natural or real-life setting by studying what people already do or how they are normally treated. These trials give scientists a more realistic idea of how a certain factor (such as a drug treatment) influences health, although observational studies cannot help determine causal relationships.
There are four main steps or phases involved in clinical trials, which usually start by testing the safety of an experimental medicine or procedure in a small group of healthy people or patients before seeing if it works in the group of patients it is intended for.
These trials are only conducted after several years of rigorous assessment in the laboratory and in animal studies, and after appropriate approval from regulatory authorities such as the European Medicines Agency in Europe. If a drug is successful in one phase, researchers may continue to the next phase.
In phase I, researchers are primarily concerned with safety and how drugs are metabolised and excreted by the body.
Phase I trials usually involve a small group of people with no health problems who are given the experimental medicine or undergo the experimental procedure to check that it is not harmful and could potentially work in patients with a specific condition.
In phase II, a slightly larger group of healthy people, patients, or both are given the medicine. This is to again check the safety of the approach and to find out how much of the experimental medicine is needed to have an effect, or to check that the approach is feasible and works.
Phase 2 trials gather preliminary information on the effectiveness of an experimental medicine and it’s short-term safety.
Phase III is an important phase of clinical testing as now the medicine or approach is given to a large number of patients with the disease and compared against a placebo (no treatment) or another standard of care. This is to prove that the treatment works and that it would be safe to give to a wider population of patients.
Phase III trials often study different populations of patients and test different dosages of the experimental medicine.
Phase IV trials are conducted after a medicine or approach has been licensed for use by the regulatory authority of a country.
These trials look at long-term safety and effectiveness, and gather more information on the benefit and risk, and how to optimise the use of a medicine or approach.
Usually, clinical trials aim to show both efficacy (how well a treatment works) and safety (what adverse events occur during the trial and that might be expected with treatment).
Safety is a priority in clinical trials, so researchers closely monitor for unwanted effects that could possibly be attributed to the treatment. These are also known as adverse events.
When it comes to efficacy, researchers can assess whether a treatment seems to affect a specific condition, and how well it works compared to other treatment options.
Almost all treatment decisions are based on the results of clinical trials in mind. When doctors and other healthcare professionals are deciding which treatment to recommend for a patient, they carefully consider the results of trials for the patient’s condition, alongside existing guidelines and treatments available and approved in the country.
This information helps the healthcare professionals to narrow down the treatment choices so they can recommend the treatment most likely to work given the therapies previously tested on similar individuals. Doctors continually update their knowledge by reading the published results of new clinical trials.
The results of clinical trials are often published in scientific journals. Both healthcare professionals and the public can search for the most up-to-date scientific publications using a database called PubMed. This is a free service provided by the US National Library of Medicine® and gives access to articles published in medical, nursing, dental, veterinary and other healthcare journals.
After receiving a diagnosis, many people look up information about their condition to find out about different treatment options. Online, in particular, some sources of information are more reliable than others.
Clinical trials are one of the most objective indicators of how a treatment works, so trial results are the best way to assess the relative merits of different treatments for discussion with healthcare professionals.
People participate in clinical trials for many reasons from simply wanting to help others and contribute to advancing medical knowledge to potentially being able to receive the latest treatment for a disease. Clinical trials are often a way to receive the latest treatments and approaches before they become widely available.
Finding the right patients for a study as efficiently as possible is key to clinical trial success. Volunteers are what makes it possible to test new treatments and make significant medical advances. All of the treatments that are available today, for all diseases, have been tested in clinical trials with volunteers. Without volunteers, there are no studies and there would never be new medicines.
By participating in the tests and treatments outlined in a clinical trial, you may gain a better understanding of different measurements and what those mean for your health in the short-term and long-term.
A clinical trial gives you the opportunity for more interactions with the medical professionals on the research team, allowing you to learn more while actively tracking and discussing your health.
As all new treatments must go through several phases of clinical trials before they become widely available, participating in a trial can be a unique chance to access innovative treatments. As researchers measure your health throughout the trial, they might even discover unexpected benefits of a new treatment.
Clinical trial volunteers allow doctors to understand more about treating people with the same disease in the general population. So you can improve care for other people — which could someday include your friends or loved ones.
Information from clinical trials is used in scientific publications that serve as permanent records of medical advancement. As a clinical trial participant, you are contributing your information to the pool of data that moves science forward.
If you would like to participate in a clinical trial, talk to a member of your healthcare team as they will be able to advise you on whether or not you would be able to do, and if there are trials in your area that you could join.
Your specialist NETs doctor or other member of your multidisciplinary team will also be able to talk you through exactly what would be involved in a clinical trial to make sure you fully understand what would happen if you agreed to participate.
Your national patient support group may also be able to guide you on where clinical trials in NETs are being conducted in your area. Cancer charities such as Cancer Research UK also provide information on clinical trials in cancer.
The European Patients’ Academy website contains publications you can read and download about clinical trial designs, analysing the results of clinical trials and assessing the value of clinical trial results.
The Center for Information and Study on Clinical Research Participation website contains information to help people decide on whether or not to participate in a clinical trial. The site contains frequently asked questions to help you learn about and what to consider before taking part in a trial.
The ClinialTrials.gov website was developed as a place to post details about clinical studies taking place all around the world.
You can find out more about clinical trials on the Ipsen website. You can also search the ClinicalTrials.gov database via the link given above for information about clinical studies that are investigating Ipsen therapies in NETs.
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