After a six-month training period developed by the project scientific team, KIDS members, as a part of the Scientific Board of Hospital Sant Joan de Déu, cooperated in the following initiatives:
Recommendations guide about the content and format of the informed assent
Well aware of the need of a more understandable and adequate informed assent document for the pediatric population, the KIDS Barcelona group created a guideline of recommendations. The outcome of their work was based on a review of 8 real anonymous models, which were studied for their improvement.
The recommendations guide includes an examination and validation by ICAN and EUPATI. It is available in Spanish and English and it has recently included the instructions to the development of clinical trials with paediatric patients of the Spanish Agency of Medicines and Medical Devices (AEMPS).
Survey to know children and teenagers’ opinion about drugs and its involvement in medical research
The Pediatric Committee (PDCO) is the European Medicines Agency’s (EMA) scientific committee, in which national agencies take part of to ensure the safety, efficiency and adequacy of medicines for children.
This Pediatric Committee prepared an easy survey with eleven questions targeting children and teenagers between 10 and 18 years old, with the goal of knowing their opinion about drugs and their involvement in the medical research (clinical trials with medication). The answering period was from March until May 2015.
The goal of this survey was to obtain information about preferences of this population related with pharmaceutical dosage and types, as well as to ascertain the difficulties that young people face when consuming these medications. In addition, the intention was to know their opinion about their possible involvement in research studies with drugs.
KIDS Barcelona members, in collaboration with their educational institutions, attained more than 500 answers. At the moment, this information is pending for publication in the format of a scientific article.
Rare Commons is a 2.0 platform of clinical research on rare pediatric diseases. Through a methodologic study based on collective intelligence, parents and doctors collaborate to generate scientific knowledge concerning a group of diseases that do not have a complete natural outlined history.
The head of the project is Dr. Mercedes Serrano, neuropediatrician and coordinator of a research project on Lowe Syndrome and Congenital Disorder of Glycosylation (CDG).
Rare Commons was presented by Dr. Serrano in a working meeting with KIDS Barcelona members, with the aim of improving its plan and in order to include some improvement suggested by the Scientific Board.
Among the questions suggested and implemented, are the following:
- Development of a specific section for tips and medical information not related with the pathologies studied in the platform.
- Promote the social media potential to connect families who do not know other cases. This way, although a research project related with their relative's pathology cannot be offered at the moment, isolation and loneliness can be fought.
European regulation about clinical trials
In 2017 the new regulation for clinical trials will be published. KIDS Barcelona members represented by ICAN Youth Committee joined the public consultation period to review and improve the legislation text.
In September 2016, an answer to the public consultation was sent to the European Commission on behalf of all ICAN groups. This included a review of the full text and the information gathered in a survey, which was focused on getting to know the characteristics that young people clinical research summary results should contain.
Feedback about clinical trials addressed to paediatric patients
KIDS Barcelona boys and girls reviewed drafts of clinical trials, submitted by the pharmaceutical industry, in particular about the treatment of:
- Cystic fibrosis.
The matters they worked on and that allowed the improvement of the initial study draft proposal were: palatability, frequency of medical follow-ups and number of medical tests, quality of life data, formulation preferences, information for the patients and families, etc.
At the moment, in collaboration with other groups, we are planning the development of a “white paper” that will allow us to unify working procedures with the pharmaceutical industry and regulators, with the aim of standardizing ways of collaboration.