50 Years of Pediatric Medical Research
Andrea Rudy, Engagement Lead, Maternal Infant Child & Youth Research Network
Pediatric medical research took a sharp turn 50 years ago—a consequence of nearly 150 years of unregulated investigations that saw great discoveries but also caused significant harm. Medical research involving children was documented as far back as the 18th century, with the 1796 smallpox vaccination study by Dr. Edward Jenner possibly being the first pediatric clinical trial. Using the populations within orphanages and children’s hospitals (and in Canada residential schools), scientists had large subject bases for medical research, a practice that lasted well into the 20th century with little protest from families and the public. Not until the 1964 Declaration of Helsinki were children—a vulnerable population—given a higher standard of protected rights, which was recognized and adopted through a number of global declarations. And thus just over 50 years ago, the pendulum finally swung.
But in the 1970s in Canada, the Canadian Paediatric Society (CPS) recognized the need to make treatments available for children, and was concerned with the lack of adequate prescribing information. While pediatric health research and innovations in Canada were making a number of impressive advances (the licensing of Rh immunoglobulin, virtually eliminating Rh disease; development of the childproof medication cap; the identification of the cystic fibrosis gene; addition of vitamin D in milk to reduce rickets), medications for children were not being properly studied. Other parts of the world were recognizing the ethical issues of not treating children with evidence-based formulas and the need to test medications within their own population, and so began moving forward with investigations. The CPS’s 2011 position statement Drug research and treatment for children in Canada: A challenge, laid out the strategic approach needed to ensure Canadian children benefit from more rigorous pediatric medical research. Over the past 25 years, a number of fundamental requirements have been progressively implemented to ensure ethical pediatric clinical research, which begins with ensuring social value and scientific validity of any trial. Initiatives like the SPOR programs and young persons’ advisory groups (YPAGs) promote the active role of parents and youths in informing study design and research participation, which has helped improve the overall quality of work. Ensuring that the interest and capacity for pediatric clinician scientists remains high in the future, the Canadian Child Health Clinician Scientist Program (CCHCSP) program provides support for next generation of clinician-scientists candidates to develop their requisite knowledge and skills for a career as an independent scientist in child health research.
In recent years, Canadian pediatric medical research projects and initiatives have had a lasting impact on the care and treatment of children. Since 1995, the Pediatric Emergency Research Canada (PERC) network has undertaken numerous multi-centre research projects improving the treatment of children in the ER—work that resulted in a 2011 CIHR-CMAJ Top Achievements in Health Research Award. Established in 1989, the Pediatric Interest Group within the Canadian Critical Care Trials Group (CCCTG) has produced work leading to new therapies for head trauma as well as in transfusion medicine in the PICU. In neonatology, Canada has led several international trials from which results have impacted on the life of thousands of very preterm infants, while the Canadian Neonatal Network, established in 1995, has moved Canada amongst the top countries when it pertains to survival after very preterm birth and short-term outcomes. In many subspecialties, pediatric clinician scientists are making meaningful contributions in precision medicine, rare diseases, and innovative therapies. These include world-leading investigations into cell-based therapies for lung diseases, regenerative medicine and rare disease research, stem cell biology, and Canadian contributions to orpha.net, a global inventory and classification of rare diseases.
More recently formed national networks and initiatives are not only developing new tests and therapies for the treatment of children, they are also engaging families and patients to provide invaluable input into priorities and design, as well as developing effective methods for the translation of new knowledge through training and engagement to help implement clinical and system changes. This attention to social value, ethical design, and dissemination is essential to improving the health of children through pediatric medical research.
Canadian speciality-focused and national networks have become integral in boosting and improving research quality, and with recent global movements towards international pediatric research collaboration, they are highly placed to participate in the effort. It will be the integrated efforts of our academic institutions, research organizations, and hospitals that bring discovery to practice. Improved care results from scientific questions catalyzing studies that produce new therapies for hospitals to implement. But the flow of hospital-collected data to research teams can change the direction of these studies and also lead to new academic questions. This two-way transfer of knowledge and collaboration between the three bodies supporting pediatric medical research is vital to our success.
Moving forward, greater efforts will ensure Canadian children are not left behind other populations in the establishment of better treatments and medical care. Canada now has the momentum of the research community’s attention; the establishment of a number of national research networks; improved family and public awareness and support; and with greater investment from funding bodies, will see increased capacity for multi-jurisdictional studies and evidence sharing.