Planning and conducting paediatric clinical trials face many hurdles, which do not always occur in adult trials. As a result, paediatric trials often take longer and fail more frequently than adult trials. Low patient recruitment is one of the major difficulties faced in conducting paediatric trials and often causes trials to stall. Patient recruitment is especially challenging in the rare disease areas, due to the low number of paediatric patients with that specific disease. A global, multi-center approach is therefore a must in paediatric trials but is often inefficient due to the low number of patients per center.
The next hurdle on the list is the fact that insufficient data are available on medicines for children to design clinical trials, particularly for neonates and infants. In addition to safety and efficacy data of new paediatric medicines, the amount of placebo data is rather limited. This is because administering specific therapies to healthy paediatric controls is considered inappropriate. The rule of thumb in paediatric research is that the child should always benefit from a certain therapy. Modelling and simulating of clinical trials based on adult and historical paediatric data therefore play a key role in planning and supporting a paediatric program. Yet, the unavailability of paediatric data hampers this approach.
Additional challenges in paediatric research entail regulatory and ethical aspects. For instance, the regulations concerning informed consents and assents. The documents for trial participation need to be signed by the child’s legally designated representative and the child itself. These documents are not harmonized but are subject to national law. This is especially challenging in global trials, where multiple countries participate. The trial therefore is subject to many different national regulations. Given that many paediatric trials are global trials, the lack of harmonization of national regulations is a challenge that is often faced.
As every child, with a medical need, deserves the best chance possible to enter well-designed studies and to have access to treatment, initiatives across the world were and are being developed and implemented. A key aspect is improving the infrastructure to conduct paediatric trials in an efficient and timely manner. This allows to enhance patient recruitment, data availability and harmonization of the different ways of working across countries. The Institute for Advanced Clinical Trials for children (I-ACT, based in the US) assist in streamlining and improving clinical trial processes to enhance the quality, timeliness and impact of regulatory-quality data, whilst reducing the administrative burden. I-ACT supports and manages a network of pre-qualified trial-ready sites and collaborates with regional and disease-focused networks to ensure they reach children globally. In Europe, the IMI2 conect4children (c4c) project kicked off in May 2018 and aims to build a pan-European network to facilitate the development of new medicines and therapies for the entire paediatric population. Through the collaboration between academic and private sectors across 20 European countries, c4c endeavors to provide a sustainable, integrated platform for an efficient and swift delivery of high-quality clinical trials for children and young adults across all conditions and diseases
I-ACT and c4c are tackling the need for an improved infrastructure to conduct paediatric trials globally. While there is still a lot to do, the paediatric community is collaborating on a global scale for a simple reason: children are our future and they deserve the best. At Modis, we are proud to provide project management services to c4c and by doing so, help to make a difference in the paediatric community. In c4c, Modis assists in managing the large consortium and reaching important project milestones, thereby aligning all stakeholders involved, ranging from academia to large pharma companies and patient advocacy groups. For more information on these initiatives, visit the I-ACT and c4c website!
Project Manager Life Sciences