Clinical trials

A clinical trial involves research participants. It follows a pre-defined plan or protocol to evaluate the effects of a potential new medicine on health outcomes. By taking part in clinical trials, participants not only play a more active role in their own health care, but they also can access experimental treatments and help others by contributing to medical research.

Clinical trials are usually described based on their Phase: Phase 1, 2 and 3. This classification is used by the Food and Drug Administration (FDA) and the European Medicine Agency (EMA) as well as other regulatory agencies to determine if the drug can be approved for use. 

Each of these clinical phases is described below. 

• A Phase 1 trial tests an experimental treatment on a small group of healthy people (20 to 80) to judge its safety and side effects and to find the correct drug dosage. In the case of some products for severe or life-threatening diseases, such as cancer, the initial human testing is often conducted in patients.
• A Phase 2 trial uses more people (100 to 300). This phase aims to preliminary evaluate the efficacy of an experimental drug in patients who have a certain disease or condition. These trials also continue to study safety, including short-term side effects. This phase can last several years.
• A Phase 3 trial are undertaken to further evaluate safety and clinical efficacy, studying different populations and different dosages, using the drug in combination with other drugs. The number of subjects usually ranges from several hundred to about 3,000 people.  An experimental drug could be approved for clinical use by regulatory agencies based on the results of a Phase 3 trial or in some instances on earlier phase studies.
• Post-approval trials, sometimes referred to as Phase 4 studies, may be conducted after initial marketing approval. These trials are used to gain additional experience from the treatment of patients in the intended therapeutic indication. In certain instances, the applicable regulator may mandate the performance of Phase 4 clinical trials as a condition of approval.

In specific situations, certain phases of development can be merged or even skipped when clear signs of efficacy emerge in the early phases of development and the potential new treatment is designed for patients with major unmet medical needs. However, these deviations from the standard pattern of development must be discussed and approved by health authorities. 

The following list of possible benefits and risks is not exhaustive. The informed consent form of a clinical study would specify the possible benefits and risks of that particular clinical study.

Possible Benefits

• Gaining access to innovative, new, investigational medicines that may not be available outside of the clinical study setting
• Helping to make a difference in the lives of others affected by the same disease or condition

Possible Risks

• Increasing demand for your time due to the potential for more visits to the clinical study site, hospital, or treatment center
• Experiencing side effects from the study medication or study procedures, which may range from minor to serious or life-threatening

As with any decision that affects your health, it’s important to discuss the benefits and risks of clinical study participation with your physician. Although the decision of whether or not to participate is ultimately yours, it’s important to make sure that you understand everything that is involved. Talk with your oncologist, your family, healthcare professionals. Ask questions and discuss your concerns. 

To participate in a clinical trial, patients must meet specific eligibility criteria. These criteria help researchers identify appropriate candidates for participation and are designed to promote safety and accuracy of study results. The criteria may allow or prevent participation.
Patients should discuss this with their physician and oncologist if they wish to be included in a clinical trial.