No single country has enough sick children or enthusiastic investigators to complete pediatric studies, particularly in rare diseases.

Canada is regularly involved as a site in multi-national pediatric clinical trials — over 85 percent of industry-sponsored pediatric studies in which Canada participates are multi-national — but that business has declined in recent years.

In addition to the loss of revenue and jobs, Canada’s lack of engagement in these studies means Canadian children are denied the opportunity for early access to new and potentially life-saving therapies. For example, Canadian patients have access to only 50 to 60 percent of rare disease drugs that are available in the United States.

"Canada needs to invest in a coordinated, pan-Canadian approach to pediatric medicines research or we will be left behind."

Canada benefits from having a good health care system and committed professionals, but there are many barriers to conducting drug studies involving participants across the country. Broad input from patients and clinicians is needed to ensure studies are workable at all sites, and available to all potential participants. There is also the need for quality control, to ensure that a study is conducted in the same way at all sites involved, so that the results of a study can be trusted. Given changes in the approach to pediatric studies in other countries, Canada will continue to lose its appeal as a participant in global trials, putting Canadian families at further risk of failing to benefit from emerging scientific breakthroughs.

Developing a global pediatric clinical trials network

Both the U.S. and European Union legislated the requirement for pharmaceutical companies to develop pediatric study plans prior to authorization of drugs for the lucrative adult market. Tied to such legislation is a commitment to reducing the barriers and improving support for pediatric studies. In England, a national commitment catapulted progress from almost no pediatric trials in the country in 2006 to now having over 300 active trials annually. Families and health care providers are actively engaged to make sure that a trial is really needed to fill a therapeutic gap in child health.

With a strong business approach resulting in 82 percent of industry-sponsored trials completed on time and to target, there has been a dramatic rise in the number of industry-sponsored studies involving the country, and new therapies are brought to market more quickly.

Pharmaceutical companies are now actively promoting a plan to develop a Global Pediatric Clinical Trials Network involving around 200 of the best children’s hospitals in the world. They are encouraging countries to adopt England’s “one stop shop” approach, whereby connection to multiple sites within the country is made through a coordinating hub that streamlines administrative processes.

Investing in pediatric research in Canada

Canada needs to invest in a coordinated, pan-Canadian approach to pediatric medicines research or we will be left behind. Such commitment will improve Canada’s standing globally and enable Canada’s pharmaceutical companies to attract business opportunities. Our participation in global pediatric studies will support rapid delivery of studies that Canada cannot deliver alone, bringing medicines for children to market more quickly.

All Canadians should care that our children get early access to new and potentially life-saving therapies, while being assured that the quality of the research means we are getting value for the money invested.