Reliable Cancer Therapies

Phase I clinical trial
If the results of tests of the new treatment in laboratory and on animals are promising, the new treatment will be given for the first time to humans during a phase I clinical trial. For cancer treatments, a phase I clinical trial usually presents the following characteristics:
- The new treatment is given to a small number of patients, usually around 20-30
- The patients invited to participate are usually patients running out of treatment options
- The goal is to find the best way of giving the treatment and the highest dose that can be administered without being harmful. For drugs, ascending doses are given and side-effects are carefully watched
- The risks are important and the benefits are not guaranteed. The goal is not to see if the treatment can work. However, some patients have been helped by receiving new treatments in phase I clinical trials.
Thanks to phase I clinical trials, researchers and doctors will know the best dose, the best way to give it and whether the treatment is safe.

Phase II clinical trial
If the treatment has been shown to be safe in the phase I clinical trial, a phase II clinical trial can be implemented. For cancer treatments, a phase II clinical trial usually presents the following characteristics:
- The new treatment is given to a limited number of cancer patients, usually between a few dozens to 100
- The treatment is usually given to all patients but in some clinical trials, participants are randomly assigned to receive the new treatment or not.
- The goal is to find evidence that the treatment works. This will be evaluated by how the cancer reacts to the treatment. When the tumor is not growing, or even shrinking or disappearing, it is assumed that the treatment works.
- The side effects are also carefully watched to make sure that benefits of the treatment are higher than risks.
Thanks to phase II clinical trials, researchers and doctors know if the treatment works for one or for several types of cancer.

Phase III clinical trial
With the information given by the phase II clinical trial, a phase III clinical trial can be implemented. The new treatment seems to work, but it has to be compared to the current standard treatment to see if it has any added value for patients. A phase III clinical trial presents the following characteristics:
- The study involves a large number of patients, usually hundreds or thousands
- Two treatment groups are usually defined and participants are randomly assigned to one of the 2 groups. In addition to the current standard of care, patients in one group do not receive the new treatment while patients in the other group do.
- When the new treatment is a drug, patients who do not receive the new treatment receive a placebo looking like the new treatment. Whatever the group they belong to, patients do not know if they receive the new treatment or a placebo. Doctors taking care of the participants do not know whether one patient receives the new treatment or a placebo. This is defined as double-blinded, meaning that patients and doctors are blinded regarding the treatment. When patients and their doctors know what treatment they receive, they often are more confident in the chance of success if they receive the new treatment than if they receive the placebo. This can compromise the results of the study.
- To evaluate the efficacy of the new treatment, doctors will look if the tumor is not growing or even shrinking or disappearing. But to conclude on the efficacy of the new treatment, the researchers will look if patients receiving the new treatment live longer than patients not receiving it. Side-effects will also be carefully watched to see if they do not counterbalance the potential benefits of the treatment.

Thanks to phase III clinical trials, researchers and doctors know if the treatment has an added value in comparison to the current standard of care. If it has an added value, the results of the study will be analyzed by authorities in charge of the approval of medicines, mainly the European Medicines Agency or EMA in the European Union and the Food and Drug Administration or FDA in the Unites States of America. The EMA and FDA will decide if the treatment should be available and for which type of cancer. In Europe, each country decides if the new treatment will be covered by health insurance.