How to advance research methods for studying drugs in children in 2019?

Dr. Lauren E Kelly, Assistant Professor, Department of Pediatrics and Child Health, University of Manitoba; Clinical Trialist, George and Fay Yee Centre for Healthcare Innovation; Scientist, Children’s Hospital Research Institute of Manitoba and the Vaccine and Drug Evaluation Centre, Winnipeg MB

Everybody in the field knows it: child health care providers have to deal with limited safety and dosing information to support pharmacotherapy in children while prescribing drugs to their young patients. In 2018, a team of Star Child Health affiliated scientists published two reviews on pharmacodynamics endpoints (https://www.nature.com/articles/pr201838) and pharmacokinetic studies (https://adc.bmj.com/content/103/7/695.long). Their findings suggest that there are innovative designs, modelling and statistical methods with the potential improve the efficiency of pediatric clinical research studies. While national partnerships and infrastructure have evolved globally to facilitate clinical research studies with children, further challenges exist.

So what is still needed to move evidence-based pharmacotherapy for children forward in 2019?

Consensus on diagnostic measures and data definitions so that small studies can be combined and compared, increasing the strength of clinical recommendations from meta-analyses
Training for clinicians, funders, researchers, ethics board members and the public on the need for and rigor behind innovative study design methods to improve the peer review process for publications and funding competitions
Clinical trials, particularly those applying innovative methods (including Bayesian Decision Analysis), that have open access, prospectively published protocols and statistical analysis plans to help guide the design of future studies
Better integration between statisticians, patients, parents, methodologists, regulators and clinicians in terms of outcome selection with valid measurement tools, study design and data collection
Where applicable, prospectively planned health technology assessment to inform payers/policy makers of the bottom line – how much will it cost/save to implement or de-implement the intervention?
A thorough plan for knowledge translation – actively go beyond an informative publication that may or may not be picked up by the people we do it all for: young patients, families and their care providers. Conducting clinical research to publish new studies in top journals is not a valuable use of public funding. Therefore, engagement with regulators, advocacy groups and policy makers early in the design process should discuss the potential study impact. For example;
How does the present study inform the design of a larger, next phase clinical trial?
How can we efficiently develop a de-implementation plan for a now proven ineffective intervention?
How can we efficiently implement an expensive but safer and more effective intervention in a health care system with fixed cost-spending?