Implementation of clinical trial, observational studies or in general research projects that aim to collect data collection of the health status of patients requires the primary need to obtain a proper approval of the research study from the ethics committee/institutional review board according to European Union (EU) guidelines and nationals laws. In addition participation of the patients elicit the need to obtain a proper consent from the patient. In case of participation of minors the consent must be obtained from the parents with the addition of an assent for children with an appropriate developmental age to understand its content.
Until few years ago most of the drugs administered in pediatrics were used off-label but this situation changed recently in Europe with the adoption of a pediatric legislation that facilitate the implementation of clinical trials for new drugs. In addition the EU has adopted directives in order to provide guideline for review by ethics committee and for consent/assent procedures.
There is however still a substantial burden to implement clinical studies in children. For example a recent study conducted by the Paediatric Rheumatology International Trials Organisation (PRINTO at www.printo.it) in a rare disease such as juvenile dermatomyositis (JDM) with off label drugs used in current clinical practice worldwide has required activation of 103 clinical centers to enroll 130 patients and the ethical process required by review boards took two years. Of note 97% of the ethical review boards gave their approval without requesting any change at all.
Procedures should be implemented at a European level to facilitate implementation and conduct of studies in children by simplifying the bureaucratic burden that is now placed on research in children.
Disclosure of Interest None Declared
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