Only 8% of clinical trials with drugs are focused on pediatrics

Pequeno Príncipe Clinical Research Center contributes to increasing this percentage and to improving medical care for children and adolescents

Children and adolescents are considered “therapeutic orphans”, as only 8% of all clinical research developed in the world involving drugs is aimed at these age groups. “The development of drugs and their respective evidence of efficacy and safety originate from research involving adults, so that the use of these technologies in children results from adaptations of these results, situations that are not ideal and can cause therapeutic failures or even adverse events,” says the Ministry of Health, in a document entitled “Pharmaceutical assistance in pediatrics in Brazil”.

At Pequeno Príncipe Hospital, the Clinical Research Center, which was created in 2010, develops studies in partnership with the pharmaceutical industry to contribute to the development of new drugs aimed at young patients, and also conducts its own research.

“These research projects can provide pediatric patients with a new therapeutic option for diseases in which regulated drug alternatives for use in children are scarce and, depending on the disease, do not even exist yet,” explains the coordinator of the Pequeno Príncipe Clinical Research Center, Fábio de Araújo Motta.

Currently, 25 studies are under development at Pequeno Príncipe, and in 64% of them the institution is the coordinating center. There are 13 principal investigators, who coordinate research in ten specialties: anesthesiology, gastroenterology, bone marrow transplantation, cardiology, pulmonology, infectious disease, oncology, hematology, neurology and vaccines. “These numbers demonstrate the seriousness of our work and the commitment to solid results, which bring real contributions to the advancement of pediatric treatments,” emphasizes the coordinator.

Clinical trials are approved by the National Research Ethics Committee (Conep, abbreviation in Portuguese) and by the Pequeno Príncipe Research Ethics Committee (CEP, also in Portuguese). Families, as well as children over 7 years of age who participate in the studies, are consulted in advance about their desire to participate, signing a consent form. “The child who participates in the research is guaranteed to receive the medication for free until it is included in the list of the Brazilian Public Health System (SUS),” informs the coordinator.

Brazilian and world scenario
According to the 2020 Guide of the Pharmaceutical Research Industry Association (Interfarma), Brazil holds the 25th position in the world ranking of clinical research. Of the 3,170 clinical studies started in the world in 2019, in the area of oncology (adult and pediatric), the country participates in 68, that is, 2.1% of the global total.

Considering clinical research in pediatrics, the challenge is even greater. About 80% of drugs approved in the United States, for example, are not intended for pediatric use and contain incomplete information about the effects they may have on children. In Europe, about 50% of medicines used in children had neither clinical trials nor specific authorization for pediatric use, characterizing what is called off-label use.

The lack of investment in research ends up depriving Brazilian patients of the advances made by science. According to Interfarma, the largest volume of spending on medicines sold in Brazil is related to products with more than 11 years of presence in the national market. The delay in updating the market means that Brazilian patients are without access to the greatest health innovations available in other countries. These are treatments that could give more quality of life or even cure diseases.


Medical residency programs train 130 professionals per year

There are 11 residency programs and seven specialization programs, which contribute to the qualification of professionals dedicated to the care of children and adolescents

Cost of the 10 most used drugs in the Hospital not fully covered by the Brazilian Public Health System exceeds US$ 500,000 per year

The list includes drugs intended for patients who need bone marrow transplants and who are undergoing cancer treatment

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