Amplifying access to early-phase clinical trials for children with cancer

In the world of pediatric oncology, hope often hinges on access – access to clinical trials, cutting-edge treatments, care options, and all the support that makes these possibilities a reality. For families battling childhood cancer, geographical and financial barriers can stand as insurmountable obstacles.

The STEP-1 program, a national initiative supported by ACCESS, aims to dismantle some of these barriers by ensuring that no child in Canada is denied a potentially life-saving clinical trial due to distance or cost. At the heart of this work is a commitment to equity in research and evaluating the impact of such initiatives to improve the quality of work and delivery, ultimately enhancing the quality of life for children with cancer.

Launched in 2024, STEP-1 was born from a pressing need. While early-phase clinical trials investigating novel treatments hold immense promise, they often require children to travel far from home. For families already under tremendous emotional and financial strain, the logistics and costs of travel and accommodation can be overwhelming. Typically, industry-sponsored trials include built-in funding for travel; however, academic phase I and II trials, typically funded by grants or philanthropy, rarely do.

“Families were coming regardless,” explained Dr. Sarah Cohen-Gogo, STEP-1 Project Co-Lead and Pediatric Oncologist, reflecting on the heartbreaking reality. “They would strain their finances to the breaking point or scramble to find support from any available resource. The question became – what if we could take that weight off their shoulders?”

In the spring of 2024, STEP-1 began as a pilot that was supported by the Garron Family Cancer Centre at SickKids in Toronto. The goal was simple but profound: provide upfront financial support for travel and accommodation to families participating in academic early-phase trials. The success of the pilot was immediate and undeniable, sparking interest and enthusiasm from other centres across Canada. Recognizing the potential for national impact, the team applied for support to expand the effort through ACCESS to scale up the program.

“Our ultimate goal is to ensure that any child, from any corner of Canada, can access the trials they need without cost being a barrier,” said Norman Cook, Project Co-Lead and a Person With Lived Experience. “We wanted to replicate the support my daughter experienced when she participated in a clinical trial at the National Cancer Institute in the U.S. – where everything was taken care of – our travel, our accommodation and more.” “Taking the worry away is huge,” said Norman. “For a parent, not having to stress about how to get there or where to stay means you can focus entirely on your child.”

For smaller and medium-sized pediatric oncology centres across Canada, the STEP-1 program is transformative. Centres with limited capacity for early-phase trials can now confidently refer patients to larger centres, knowing that some support is in place. “This national program provides a clear pathway for families and healthcare providers,” noted Dr. Lisa Goodyear, Project Co-Lead and Oncologist. “For smaller centres, where research resources and personnel are limited, having a single point of contact to identify trials and arrange logistics is invaluable, and all the costs are paid for upfront. This levels the playing field for our patients.”

Early-phase clinical trials are demanding – with frequent scans, bloodwork, and specialized expertise often concentrated in a few centres. While efforts are underway within ACCESS to bring trials closer to patients, the reality is that some trials will always require travel. STEP-1 is here to bridge that gap. “There will always be trials that need specific expertise, so bringing every trial to every child is unrealistic,” admitted Dr. Cohen-Gogo. “But through STEP-1, we’re committed to bringing the child to the trial when needed – and making that journey as seamless as possible.”

Strategic partnerships are crucial to this work, and one such collaboration is with Hope Air. “The partnership between ACCESS and Hope Air to support the central coordination of travel for the STEP-1 program will address some existing barriers to clinical trials for children living far from major urban centres or beyond the reach of many who are struggling with the day-to-day cost of living,” said Mark Rubinstein, CEO, Hope Air.

The STEP-1 program is more than a funding mechanism; it is a statement of equity – access for all. By addressing one of the most significant barriers to clinical trial enrolment, it ensures that promising treatments are accessible to all children – not just those living near major centres or with the means to travel. It serves as a reminder that in pediatric oncology, access to research and care is not a privilege – it is a right. Thanks to STEP-1, we are one step closer to ensuring that right for every child with cancer in Canada.

To find information on early phase clinical trials for children and adolescent with cancer within Canada, visit U-Link.

Learn more about ACCESS research themes, groups, and projects here.