Clinical Trials: FAQ

The purpose of a clinical trial is to evaluate new treatments for cancer. Basically, before recommending new treatments to all patients involved, we must make certain that they are effective and well tolerated.
Clinical trials (also known as “therapeutic trials” or “clinical studies”) must evaluate the following in particular:

• New drugs or groups of drugs (against the disease or its side-effects)

• New methods of administering them (for instance, by tablets rather than injection)

• New treatment techniques (such as new types of surgical operations or radiotherapy) or diagnosis (such as new biological tests)

Initial Laboratory Tests

When a new drug is discovered, it is first developed in a laboratory, then tested on animals (this is pre-clinical research).

If the results of these tests turn out to be favourable, then it is possible to consider recommending affected patients to participate in its evaluation, with their consent. This evaluation, conducted in collaboration with patients, is called a clinical trial.

The Various Phases of Clinical Trials

In order to guarantee patient safety and scientific rigour, clinical trials involve several steps (or phases) each designed to collect a specific set of information on the new treatment.

The objective of a Phase I trial is to evaluate the tolerance of a completely new drug, and thus also the organism's tolerance to the treatment, with the aim of determining the recommended dosage.
The treatment under study is administered to a small group of patients (10 to 40).

Phase II trials evaluate the effectiveness of a treatment.
These typically require the inclusion of 40 to 80 patients.

Phase III trials are comparative trials. They compare the new treatment with the treatment typically used, known as the “standard treatment”. Patients are randomly assigned to two separate groups (randomisation), which provides uniform, comparable groups (in terms of age, sex, disease characteristics, etc.). One receives the standard treatment and the other the new treatment. Therefore, it is not the doctor who decides which patient gets one treatment and which the other.
These trials require the inclusion of a large number of patients (several hundred or thousands of patients) in order to establish a difference between the treatments.

If the data and findings from these trials come out in favour of the new treatment, it will then be possible to prepare a registration dossier and submit it to the healthcare authorities for a marketing authorisation (MA), which grants approval to put the new treatment onto the market.

When the drug is released for sale, it will be subjected to another thorough testing procedure, known as pharmacovigilance. Thus, in France, any unexpected abnormal signs due to its administration are submitted in a statement to the French Healthcare Product Safety Agency (AFSSAPS). These kinds of studies are called Phase IV trials.

NB: The same patients does not participate in all of the phases in a row but rather is recommended for participation in one trial in one of the four phases, depending on very precise criteria.

Who Takes the Initiative for a Clinical Trial?

The research institution that has announced a plan to conduct a trial is called the “sponsor”. This may be a university, a public or private research institution or a pharmaceutical company. A clinical trial may be implemented within France but they are often conducted at European or even international level.

Two Supervisory Bodies:

• The Personal Protection Committee (CPP)

• The French Healthcare Product Safety Agency (AFSSAPS)

Who conducts the clinical trial?

A clinical trial is conducted by a doctor called the trial investigator. This is the person who recommends patients to participate in the trial, provides them with all the background information they require and monitors them throughout the trial.

Are all patients eligible?

In order to participate in a clinical trial, a patient must meet a specific number of conditions, known as “inclusion criteria”. The inclusion criteria are specific to the particular clinical trial. Because each trial has a specific objective, some patients and not others, will be invited to participate in a particular trial based on their characteristics, such as the size or type of their tumour.

Is participating in a clinical trial useful in advancing research?

Yes, patient participation in clinical trials is a vital contribution to the discovery of new treatments and new strategies which could benefit a large number of people affected by cancer.

Advances in medical science, particularly in cancer treatment, are the fruits of clinical research, thanks to trials conducted in a rigorous, methodical manner which guarantee a certain level of scientific proof. However, there remain numerous situations which demonstrate the uncertainty and inadequacy of current treatments. So there are also numerous advances still to be made, both in researching new treatments and in developing new therapeutic strategies.

It should also be noted that thanks to the act passed on 4 March 2002 on patients' rights and healthcare system quality (called the Kouchner Act), people who have participated in research that was part of a clinical trial can ask to be informed of the overall results at the end of the study.

Am I free to participate in a clinical trial that has been recommended to me? And can I change my mind later once I’ve accepted?

Your participation is free and voluntary. Even after agreeing to participate in a trial, you will be free to quit at any time, in which case your doctor will recommend a different treatment tailored to your disease. This decision will not affect the medical team’s commitment to treating your disease in any way.
Does participating in a clinical trial offer me more opportunities for better treatment?

Participation in a trial does not in any way jeopardise a patient’s chances for treatment administered outside of a clinical trial. But this participation does in certain cases offer the possibility of gaining access to more innovative treatments.
Nevertheless, if you decline to participate in a clinical trial that is offered to you, this decision will not have any consequences on the treatment of your disease, your relationship with your doctor and the healthcare team and on the quality of the care you will receive.

Will I run any personal risks by participating in a clinical trial?

As with any treatment, treatments used in clinical trials can produce side-effects. In most cases these are well known and documented. However, despite conducting extensive laboratory studies prior to conducting these trials on humans, it may occur that certain particularly rare symptoms have not been identified. Moreover, additional testing and examinations to study the unexpected adverse effects may be necessary and would involve some additional constraints on the patient.

All methods of patient treatment and monitoring involved in a clinical trial are precisely defined and subject to numerous regulations (both national and international) and to stringent supervisory bodies.

Throughout the trial, the medical team will be available to provide you with any explanations you find useful regarding the nature and course of the treatment.
However, side-effects vary from patient to patient. In Phase III comparative trials, they are typically comparable with those observed in standard treatments and for the most part are temporary and will go away once the treatment has ended.

What benefits will I receive by participating in a clinical trial?

Participating in a clinical trial offers the possibility of gaining access to an innovative treatment.

It also involves special supervision of treatment and customised, rigorous monitoring, both during and after the end of treatment.

The doctors conducting a clinical trial follow a treatment plan (therapy plan) scrupulously. This plan defines all of the trial’s methodologies (inclusion criteria, duration, treatment schedule, additional examinations, etc.). The therapy plan also provides for patient monitoring throughout the course of the trial in order to verify the effectiveness of and tolerance to the treatment. As a result, all doctors and nurses working on a clinical trial monitor the patient constantly in order to verify that the treatment is producing the expected benefits and to intervene immediately should the slightest side-effect appear.

Therefore, with respect to new treatment, patients taking part in clinical trials enjoy extremely rigorous monitoring throughout the course of the clinical trial as well as after the end of treatment.

Will I be properly informed in order to make my decision freely?

Yes, before participating in a clinical trial, the doctor is obliged to provide the patient with information, first orally, then in writing, covering the list of expected benefits and potential risks. This information may sometimes appear complicated and difficult to understand. Please do not hesitate to contact your doctor for clarification. This way, you can be aware of all the information needed to understand the trial and examine any reasons you may have for participating, while also taking the necessary time to make a well thought-out decision. You will also be informed of possible alternatives if you decide not to join the trial.

Only those patients who give their consent after receiving all the information needed to understand the clinical trial can participate in it. Therefore, it is impossible to be included in a trial without knowing.

Your free, informed consent will be confirmed in writing using a consent form provided by your doctor. This form will confirm your agreement and attest that you have received the information on the study. It will be signed by you and your doctor.

To participate or not to participate: the choice is yours

Please do not hesitate to ask for further information on any points that seem unclear to you. And if after giving the matter some consideration you decide that you are not interested in participating in the trial, you will have every right to decline. This will not have any impact whatsoever on the devotion of your doctor and medical staff to your care.

Freedom to quit the trial whenever you want

If the treatment administered during a clinical trial does not help the patient, the doctor can decide to withdraw the patient from the study and recommend a different treatment. The patient is also free to ask to quit the clinical trial at any time. This decision would not have any consequences on the treatment of the disease, the patient’s relationships with the doctor and healthcare team and the quality of care.

Glossary

Chemotherapy (chemo): a general cancer treatment using drugs. These drugs are intended to destroy the cancer cells or prevent them from multiplying, resulting in their death. Chemotherapy drugs can be administered by injection, IV drip or, on occasion, in tablet form.

Informed consent: The patient is said to have given informed consent when he or she agrees to participate in a clinical trial or study after having received all the information on the aim of the trial and its associated benefits and risks.

Personal Protection Committee (CPP): a board whose job is to verify, before the implementation of a research project, that the Ethical rules are observed and that all measures have been taken to provide maximum protection for future participants. Their participation is only possible if they freely and voluntarily consent after being provided with clear information.

Side-effect: the aim of the treatments is to treat the cancer. Sometimes, they produce undesirable consequences for the patient, which are called side-effects. There are 2 kinds of side-effects: immediate and delayed. While side-effects may be frequent, they do not occur necessarily or automatically. They depend on the treatment received, the dosages administered, the type of cancer and each patient’s individual response to the treatments.

Investigator: an investigator is a doctor as well as an experienced clinical research specialist who prepares a protocol or a treatment plan as part of a clinical trial and implements it on patients.

Therapeutic plan: all the different treatments administered in a specific pre-defined order (for instance, chemotherapy may be preceded by surgery).

Radiotherapy (radiation therapy): “local treatment” of cancer using a device that emits radiation. This radiation is directed at the tumour in order to destroy it. This treatment is administered in a special radiotherapy department. The terms ‘radiation therapy’ and ‘radiation session’ are also used.

Randomisation: Random selection (by computer or using distribution tables) of treatment administered in a “randomised” clinical trial, after the patient has been informed and has given his or her consent. Randomisation results in maximum comparability between groups of patients.

Standard treatment: treatment (or other intervention) commonly used and regarded as effective on the basis of past studies. This is the best known treatment.

For further information

Cancer Info Service, an information and counselling line dedicated to patients and their friends and family:
0810 810 821 (costs the same as a local call)