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Creating a policy framework for the implementation of precision medicine for pediatric cancers

In recent years, new technologies have enabled researchers to gain a clearer understanding of the genetic mutations that cause cancer in each individual. These discoveries are ushering in a new era in cancer research and care known as precision medicine.

Precision medicine has the potential to provide patients with treatment tailored to their individual cancer. But uncertainty about how effective current precision medicine approaches are for all patients and their often-high costs make it difficult for health care systems to confidently and sustainably invest in them.

“We live in a time of rapid scientific innovation and exciting new therapies, but also mounting pressure on our health systems to deliver expensive new treatments quickly, often before it’s clear how well they work,” says Dr. Avram Denburg, a pediatric oncologist at The Hospital for Sick Children (SickKids) in Toronto. “In this context, it’s important to figure out how to ascribe value to novel precision medicine treatments so we can figure out how to best prioritize scarce health system dollars for these technologies.”

Dr. Denburg hopes his new Terry Fox Investigator Award will help to achieve this. Over the next three years, he will receive a total of $430,000 from the Terry Fox Research Institute and the Canadian Institutes of Health Research to create a policy framework that helps guide health care systems on the adoption of precision medicine approaches for pediatric cancer patients.

This policy framework will help give pediatric precision medicine initiatives such as Terry Fox PROFYLE an avenue to sustainably integrate into provincial health care systems.

“Terry Fox PROFYLE is having a significant impact on the lives of patients, but what we haven't addressed to date is how to move this scientific innovation and knowledge into sustainable health system practice,” says Dr. Denburg. “I'm hopeful that this research can help bridge the gap between evidence and policy.”

As part the New Investigator Award, Dr. Denburg will receive mentorship from the Terry Fox PROFYLE team led by Dr. David Malkin, a pediatric oncologist and senior scientist at The Hospital for Sick Children (SickKids). This mentorship will allow him to collect data being generated across Canada from Terry Fox PROFYLE and root his policy framework in the context of a well-established program with current clinical applications.

“Dr. Denburg’s project is of direct and significant value to the health of children in Canada, and bears considerable potential for real-world impact,” says Dr. Malkin. “We anticipate that this knowledge will translate into improved real-time access to targeted therapies for the patients we serve.”

Read more about all the 2020 Terry Fox New Investigator Award recipients here.

Mentoring program: Terry Fox PROFYLE

Mentor: Dr. David Malkin