Clinical trials are an essential part of advancing knowledge and improving healthcare worldwide. Advances in paediatric research across the globe to improve the lives of babies, children and young people have dramatically reduced rates in morbidity and mortality and increased the quality of life, for example, reducing sudden infant death, increasing the life expectancy for children with thalassemia, cystic fibrosis etc.
Clinical trials within the paediatric population can be conducted as both industry or non-industry trials. But what are the differences between these types of clinical trial?
The main differences between clinical trials sponsored by academia (non-industry) and those sponsored by industry are centred around:
Funding – in non-industry trials the running costs (regulatory, investigators, clinical operations, e-CRF, monitoring, statistics, etc) are covered through grants (governmental, national, EU, charities) whereas in industry trials these costs are paid directly by the company itself.
Investigators – within academia, the principal investigators within the institutions are responsible for the running of their clinical trials and finding partners in other sites. Within industry, the investigators can be either directly employed by the company itself or the company can enlist a contract research organisation (CROs) to run the clinical trial including finding investigators and sites.
Motivation – both types of trial have the ultimate goal of striving to improve healthcare, by providing evidence of the efficacy and safety of a new product in children. The focal points of both do differ though: industry trials tend to focus on trials which will provide evidence to support use in children and licensing by a regulatory authority. Non-industry trials tend to focus on the unmet medical needs.
Type of trial – industry often is researching the efficacy of a new drug or product through clinical trials to support a licence whereas non-industry trials often seek to compare one intervention to another.
Intellectual Property – when clinical trials are sponsored by industry they own the rights to the product, clinical data and publication rights. In non-industry trials these rights are owned by the principal investigator(s) and/or university.
Independent of funding mechanism, the optimal environment for clinical trials involves collaboration between both academia and industry, which is one of the driving forces behind the c4c project.
Paediatric clinical trials and PPI (patient public involvement) within c4c
As part of the development of the c4c network, several Proof of Viability studies (clinical trials) are currently either underway or imminently foreseen. 3 non-industry studies are currently running and 4 industry studies are due to open by the end of 2021. EFCNI is the partner responsible for PPI within TREOCAPA, one of the non-industry studies.
The TREOCAPA trial examines the prophylactic use of paracetamol to close a persistent ductus arteriosus (PDA). The ductus arteriosus is a blood vessel that allows the blood to skip the circulation into the lungs when a baby is in the womb. This blood vessel usually closes in babies shortly after birth but very often does not in extremely preterm born babies (23-28 weeks gestational age). A PDA can cause permanent lung damage as well as feeding and growth problems. This study is a Phase II/III randomised, double-blinded trial carried out in 65 neonatal intensive care units of 17 European countries. To date, 19 sites have been opened and 22 patients recruited. For more information please see: https://www.efcni.org/activities/treocapa/
EFCNI has been involved since the drafting of the TREOCAPA study protocol and has established a dedicated Parent Advisory Board (PAB). Both EFCNI and the PAB provide input into various aspects and materials developed within the framework of the trial. For example, the informed consent forms, patient information summaries, training materials for medical professionals approaching parents, input into follow-up, a video for the general public etc. A dedicated web page to the trial has been set up on EFCNI’s website: https://www.efcni.org/activities/treocapa/ . EFCNI regularly provides updates and promotes the study on their social media channels.