Improving clinical paediatric research and learning from COVID-19: recommendations by the Conect4Children expert advice group

Abstract

Background The COVID-19 pandemic has had a devastating impact on multiple aspects of healthcare, but has also triggered new ways of working, stimulated novel approaches in clinical research and reinforced the value of previous innovations. Conect4children (c4c, www.conect4children.org) is a large collaborative European network to facilitate the development of new medicines for paediatric populations, and is made up of 35 academic and 10 industry partners from 20 European countries, more than 50 third parties, and around 500 affiliated partners. Methods We summarise aspects of clinical research in paediatrics stimulated and reinforced by COVID-19 that the Conect4children group recommends regulators, sponsors, and investigators retain for the future, to enhance the efficiency, reduce the cost and burden of medicines and non-interventional studies, and deliver research-equity. Findings We summarise aspects of clinical research in paediatrics stimulated and reinforced by COVID-19 that the Conect4children group recommends regulators, sponsors, and investigators retain for the future, to enhance the efficiency, reduce the cost and burden of medicines and non-interventional studies, and deliver research-equityWe provide examples of research innovation, and follow this with recommendations to improve the efficiency of future trials, drawing on industry perspectives, regulatory considerations, infrastructure requirements and parent–patient–public involvement. We end with a comment on progress made towards greater international harmonisation of paediatric research and how lessons learned from COVID-19 studies might assist in further improvements in this important area.

Newborn Screening in a Pandemic—Lessons Learned

“…The COVID-19 pandemic had an impact on pediatric research, but at the same time, it provided an opportunity to explore novel ways of planning and delivering services, including NBS [ 37 ]. Before including a new condition into existing NBS programs, research and evaluation were required, and practical aspects had to be adapted to comply with the circumstances created by the COVID-19 pandemic.…”

Potential of Microneedle Systems for COVID-19 Vaccination: Current Trends and Challenges

“…To address the limits and present drawbacks of hypodermic needle injection, vaccines can incorporate MAP’s innovation in stability, bioavailability, potency, and less adverse effects [ 74 , 150 , 208 ]. Using the keyword “microneedle vaccination” as a search term, seven studies were discovered on Clinical Trial.gov that used the vaccine MAP.…”

“…On the one hand, this enhanced information is essential for elucidating the many processes of the host immune response involved in viral neutralization and/or the elimination of infected cells [ 147 , 148 , 149 ]. On the other hand, it will also forecast the outcome when used at a large scale in the targeted demographic [ 150 , 151 , 152 ]. As a result, the effect of gender and age on the virus’s ability to modulate host immune response, as well as the impact of SARS-CoV-2 infection on the immunity of patients with chronic diseases such as diabetes, hypertension, and chronic obstructive pulmonary disease (COPD), must be fully addressed [ 153 , 154 ].…”

Can Innovative Trial Designs in Orphan Diseases Drive Advancement of Treatments for Common Neurological Diseases?

“…Such approaches also reduced participant and staff burden. 31 Specific procedures to present and validate DHTs for regulatory feedback are emerging, including the EMA qualification of novel methodologies. 32 , 33 Notably, in 2019, the EMA qualified the use of 95% stride velocity as assessed using a wearable device as a secondary end point for clinical trials in DMD.…”

“…Necessary innovations in development, use, and deployment of DHTs were accelerated by the COVID‐19 pandemic because of the need to reduce person‐to‐person contacts, and to support remote data gathering and study monitoring. Such approaches also reduced participant and staff burden 31 . Specific procedures to present and validate DHTs for regulatory feedback are emerging, including the EMA qualification of novel methodologies 32,33 .…”