Clinical Research

A clinical trial is a study which investigates the effects of a medicinal product or treatment on people. In the case of the SJD Barcelona Children's Hospital, we focus our trials on children and adolescents. 

In order for a medical product to be authorised and used on people, it must be subjected to clinical trials, which is the most rigorous and reliable way of ascertaining the quantity, efficacy, safety and side effects of drugs.

Normally, in a clinical trial, two treatments or drugs are compared - the one under study and another already known drug, or an inactive substance, also known as placebo. Neither the parents and children nor the investigators themselves know which treatment the patient receives, to avoid skewed results. There is usually an anonymous draw to assign the treatment to the patient.

Regulatory agents linked to drug authorizations, as Food and Drug Administration (FDA) and European Medicines Agency (EMA), determine the design of clinical trials and, therefore, the use or non-use of placebo. In oncology or ultra-rare disease studies, it is common for placebo not to be used and for the study to be conducted directly on the drug.

Children are not simply small adults, drugs and treatments may have different effects than on grown-ups. The majority of treatments administered to paediatric patients have not previously been tested on children, owing to which, it can be difficult to determine the correct dose, the safety thereof, or the side effects which may appear. Moreover, children grow and change very quickly, which means that the doses and administration guidelines for medicines change in each stage of the patient growth (from 0 to 18 years of age). 

For this reason the European Union health authorities resolved to allow an increase in paediatric clinical trials in a regulated setting.

Clinical trials are important because they help researchers and doctors to find new treatments:

• For illnesses which only occur in childhood and adolescence.
• For illnesses which also appear in adults but which manifest themselves differently in children.
• Which help to improve children's quality of life

Clinical trials can also help us to understand the differences caused by growth and development in children, and thus to be more precise when calculating the dose of the drug to be administered to patients in each stage of growth, without giving rise to side effects. Finally, they enable us to create drugs which children can take easily, in different formats (chewing gum, syrups, etc.).

At the SJD Barcelona Children's Hospital, clinical trials are reviewed and approved by the San Juan de Dios Foundation's Independent Ethics Committee and the Spanish Agency of Medicines and Medical Devices (AEMPS).

Additionally, we apply all the legal procedures and control systems stipulated by Spanish legislation for clinical trials on medical products for human use.

En un ensayo clínico de nuestro centro pueden participar todos aquellos niños y niñas, pacientes o no del Hospital, que cumplan los requisitos del estudio clínico que se quiere realizar. Estamos hablando de edad, peso, género, enfermedad a estudiar, tratamientos que recibe actualmente y cualquier otro que se especifique. 

El médico de referencia del paciente puede ayudar a determinar si tu hijo o hija puede ser candidato a participar en uno de nuestros ensayos.

La participación en un ensayo clínico siempre se realiza de forma voluntaria y altruista.

When conducting a clinical trial, it is hoped that the treatment under study will provide benefits, such as being more effective than other treatments, or even that it may cure an illness. Nonetheless, it cannot be guaranteed that the child will receive the study drug, since there is normally a random selection process by means of which each patient is assigned either the drug under study, another already known one or an inactive substance (placebo), making it possible to obtain valid, reliable results. Exceptions exist in clinical trials, for example, in some studies of cancer treatments or ultra-rare diseases.

There is also the possibility of your child suffering from side effects from the administration of the treatment under study, even though clinical trials are specially designed to ensure the minimum possible risk for participating patients.

The first is ascertain whether your child is eligible to participate in a specific clinical trial, depending in the inclusion and exclusion criteria that have been defined (age, weight, current medication, etc.).

If your child is eligible, the doctor will give you all the information on how the clinical trial is going to be conducted, and you will need to sign a document called an informed consent form, in which all the details of the procedures to be carried out during the study (which may include additional tests to those usually performed) will be explained.

It is a document which must be signed by the family and the child (if over 12 years of age) indicating your agreement to participate in a clinical trial.

A number of different aspects are explained in the informed consent form: that your participation is voluntary, the reasons for conducting the clinical trial, which procedures are going to be performed and for how long, the possible risks and benefits of participating in the study and, finally, other treatments available.

Currently, the Hospital participates in clinical trials of more than 30 research areas. We invite you to consult those areas in the Sant Joan de Déu Research Institute website.

A clinical trial is a research conducted on human subjects to test the safety and/or efficacy of a researchal medicinal product: a medicinal product that has not been marketed because it is being tested, or that is marketed but is being tested for a purpose other than that authorized.

Clinical trials are driven by a sponsor, which is a legal entity (company) responsible for conducting the study. This may be a pharmaceutical company or a research centre (such as the Sant Joan de Déu Research Foundation, for example). The sponsor submits a clinical trial protocol, which is the document approved by the ethics committee and the regulatory body which, in Spain, is the Spanish Agency for Medicines and Health Products (AGEMED) and, on the continent, the European Medicines Agency (EMA). 

AGEMED and/or the EMA define the characteristics of the study participants and determine the inclusion and exclusion criteria in each case, as well as the characteristics of the experimental treatment (investigational drug) and the medical procedures to be performed on the patient to determine the efficacy and safety of the treatment. 

To gain access to investigational treatments, the patient must meet the inclusion criteria that have been established in the trial protocol. We can find out if the patient meets these criteria once the patient visits our centre.

In the event that patient recruitment for a clinical trial has been closed or if the patient does not strictly meet the criteria for inclusion in the trial, access to the experimental treatment may be possible if the manufacturer facilitates access to the treatment.

This use of experimental drugs or products outside clinical trials is called "compassionate use" and must be approved by the health authorities, in our case the Ministry of Health.

For both the use of experimental treatments in a clinical trial and for compassionate use, the company that manufactures the experimental treatment funds the product (the drug).

If your child participates in the clinical trial, the study tests and treatment will be funded by the sponsor of the research. Tests that are not included in the protocol, that are part of the clinical follow-up of the patient, or in case of worsening of the clinical situation, should be financed by the patient. 

In the case of access to compassionate use treatment, all medical tests must be financed by the patient.

In our hospital, in the case of international patients, an initial deposit of the amount indicated in the budget is required in order to start the medical care process. This budget is made including all hospital care costs, with the exception of the experimental drug, which is always financed by the sponsoring company.

After diagnostic confirmation and assessment of the extent of the disease, if there is a clinical trial and the patient meets the inclusion criteria, the hospital returns the part of the financial deposit made that corresponds to the medical tests included in the trial protocol, in compliance with the applicable legal requirements.