Additional information : www.notre-recherche-clinique.fr
A disease is considered "rare" when it affects at most 1 person out of 2,000. They are mostly of genetic origin.
In pediatrics, in more than half of the cases, rare diseases affect young children under 5 years of age and account for 35% of deaths occurring before the age of 1 year. However, other rare diseases may appear in adulthood.
Despite the scarcity of certain diseases, doctors and public and private scientists are involved in research to better understand these diseases and find new treatments.
The PEDSTART network makes it possible to promote research on such pathologies by linking and using centers recognized for their expertise on these diseases throughout France.
In the context of rare diseases, participation in a clinical trial can be an opportunity to benefit from the best developmental treatments available.
For more information on rare diseases :
- Eurordis: European platform of rare diseases
- Orphanet: The portal for rare diseases and orphan drugs
Young People Advisory Group (YPAG)
Participation in the European network of child experts (also called YPAGnet) is in line with the goal of the PEDSTART network to respect the advice of children and their parents in pediatric clinical research development.
YPAGs (Young Patient Advisory Group) are youth advisory groups. They aim to provide input to improve understanding, communication and biomedical research as well as create innovation in pediatrics.
This initiative was born in the United States at the American Academy of Pediatrics in partnership with children's hospitals and schools.
In Europe these groups have existed for more than 10 years.
In France, the first group "KIDS" launched in Lyon in 2015, and there are plans for more to follow.
Young people aged 11 to 18 are trained in clinical research. They meet monthly to have discussions with professionals and attend international conferences.
Some examples of their contribution include consultation by clinical research promoters to review protocols and other documents explaining trials (such as newsletters and informed consent) and to help develop tools for communicating and disseminating rare diseases. Their contributions are rich and particularly relevant for developing projects and understanding the constraints of participating in a study.
Useful links / Associations
Research is essential to progress in health care.
This progress is possible through the improvement of scientific knowledge; through research conducted in order to better understand the mechanisms of occurrence and development of diseases.
Medicine does not yet know how to cure certain diseases, researchers must find new strategies (new drugs or new combinations of drugs, new methods of care) to better care for patients. Medical progress also concerns the prevention and diagnosis of diseases, the equipment used for care and the tools to assist in the interpretation of data collected in patients.
In order to offer these innovations to all those who can benefit from them, they must first be evaluated in humans by clinical research studies
Medications, when they are intended for children, can not only be evaluated in adults.
It is therefore necessary to conduct specific studies in children or adolescents. In fact, the body and organs (liver, kidney, stomach) of a child do not react in the same way as those of an adult. We must ensure that these drugs do not severely affect the child’s growth and development.
In addition, some diseases only affect children.
Drug doses must be adapted to the age and weight of the child.
It is necessary to have formulations adapted to children who often, before 6 years, have difficulty swallowing tablets or capsules. Liquid forms, in powder, or small grains are necessary, with a consideration for taste which is important and which does not exist when it comes to tablets.
For all these reasons, we cannot be satisfied with the results obtained in adults
If you are offered the opportunity to participate in a clinical trial, it is to improve the management of the pathology that your child is suffering from.
Participating in a clinical trial provides early access to an innovative and promising treatment (drug, medical device, new therapeutic strategy etc.). Early access requires more frequent and necessary follow-up by the medical team with additional consultations and examinations for safety and efficacy, compared to the usual treatment of the pathology. This can add constraints for the patient.
In all cases, the investigator undertakes to propose the best diagnosis to allow the best treatment adapted to each case. The investigator must provide the child with the best care possible and can adapt or change the treatments accordingly at any time whether or not it they are treated in a clinical trial.
A clinical study is research performed on humans to evaluate the efficacy and tolerance of a diagnostic method, a treatment or a medical device.
When it comes to the drug, a clinical study (or trial) aims, for example, to establish or verify data on the fate of the drug in the body, or its mechanism of action, or its effectiveness and tolerance.
The clinical trial can be done in either a sick person or a non-sick person (healthy volunteer)..
In an open trial, the investigator and the patient are aware of all the details of treatments being administered.
Open trials may be conducted over a longer period to provide more information on the safety of a drug.
What is a double-blinded trial ?
In a double-blind trial, neither the investigator nor the caregiver nor the patient know which treatment (placebo, reference treatment, evaluated treatment) is being given to the patient.
The double-blind technique is often used to prevent the investigator, the health care team, and the patient from being influenced by an opinion they might have about a particular treatment.
How are two drugs compared for the samed disease ?
In order to know if a drug will be as good or even better than what is already available (especially in terms of efficacy and / or tolerance), it is essential to make comparisons of these different treatments through a clinical trial.
In the clinical trial multiple groups of people are treated in parallel by the drugs that we want to compare.
Qu’est-ce qu’un placebo ?
A placebo is a product that has the same appearance as the product or drug being evaluated, but that does not contain any active substance.
In some new drug trials, when there is no standard of care, the new drug is compared to a placebo. This comparison makes it possible to be sure of an actual effect of the evaluated drug. If during the course of the trial, the health status of the participant requires they receive the alternative treatment to reasonably improve their health outcome, the investigator will prescribe it. Un placebo est un produit qui a la même apparence que celle du produit que l’on évalue, mais qui ne contient pas de substance active.
From an individual point of view, participating in a clinical trial allows you to benefit from an innovative treatment that is not yet available on the market. This is under the safest possible conditions and at an early stage of research.
For healthy volunteers (who are not sick), participating in the trials helps to benefit the greater community by contributing to medical progress.
In all cases, the investigator undertakes to deliver the best possible care.
Before making your decision, it is important to:
- Read the submitted documents carefully
- Do not hesitate to gather more information by questioning your child's doctor (s)
- Talk to your child's primary doctor if the study is offered by another doctor
- Take the time to think about it, if there is no urgency
At any time you and your child (if they are old enough to give their opinion) can refuse to participate in a clinical study.
This will not change the commitment of the medical team, involved in the treatment of your child, in providing you with standard quality of care.
It is possible to leave a trial at any time, without justification and without risk to the medical care your child will receive, because all participation is free and voluntary.
If you wish to end your participation in the trial, or that of your child, you must inform the investigator who will propose a treatment appropriate to the pathology and procedures to follow.
This decision will not affect the relationship you have with the investigator and the healthcare team.
The investigator may also decide to stop your participation, or that of your child, if he or she feels that the treatment is no longer suitable or there is the appearance of significant side effects. The investigator must of course inform you of this change.
The health of the patient or volunteer always has priority over the trial itself.