Identifying best practices for obtaining ethical consent and for data and sample collection in pediatric rheumatic diseases – the role of the EU-wide ethics process in real-life

Abstract

Rare pediatric diseases have incidences as low as 1/million, but the entire sum of all various those rare diseases still affects thousands, if not millions of young patients across Europe. Scientists in every country have made substantial contributions to improve treatment strate-gies with the goal of developing early-onset therapies preventing irreversible organ damage and improving long-term prognosis. International collaboration can foster progress, but still many scientists struggle with the immense variety of ethical requirements, the regulations for data and sample sharing and biobanking structures. A unified, standardized framework applicable for all member states of the European Union is still missing. In order to design recommendations for this urgently needed standardization of the research process, we performed a systematic literature review and several real-life studies with the aim of identifying the best practices of and barriers to transnational pediatric research. An ethics proposal was designed to evaluate the real-life work of research ethics boards across Europe. A study on the investigator perspective was sent to multiple research partners to evaluate their level of experience and ideas to improve the current ways of conducting pediatric re-search. A study on the REB perspective was performed with REBs across Germany to ex-amine their structural and procedural differences. With these practical approaches, accom-panied by a comprehensive literature review, barriers for pediatric research have been iden-tified. Recommendations to overcome these barriers have been drafted, revised, and final-ized with the help of research ethics board members and European experts for ethical and legal aspects of pediatric research. The results of the ethics proposal showed the greatest possible variety. Some research eth-ics boards have denied the proposal while others have approved it. The study on the inves- 136 tigator perspective has shown a great need for more support of pediatricians involved in research projects. The study on the REB perspective supported the wide variety of findings of the ethics proposal with personnel composition and organizational characteristics differ-ing greatly. These findings have been analysed and led to a total of 21 recommendations. The issues that were addressed include 1) general principles, 2) ethics, 3) pediatric princi-ples, 4) consent to pediatric research, 5) pediatric data and biobanks, 6) sharing of data and samples and 7) commercialization and third parties. The process of the evidence synthesis and the resulting recommendations were published in our study: “Recommendations for collaborative paediatric research including biobanking in Europe: a Single Hub and Access point for paediatric Rheumatology in Europe (SHARE) initiative.” 25 These recommendations for collaborative pediatric research on a European scale including data- and sample-biobanking and sharing across borders are the first of their kind and show the urgent need for a unified European legislative framework and evidence-based guidance for its implementation. Children with rheumatic conditions and the many others suffering from rare diseases should no longer be left behind when life-changing research discoveries can be made.