Building a Better Future for Pediatric Cancer Trials: A C17 x ACCESS Partnership

Clinical trials are critical to making available and sufficiently evaluating innovative therapy options in children with cancer.  However, the rarity of childhood cancers adds greater complexity to the conduct of clinical trials, and almost always necessitates multi-centre participation.  Given these complexities, crucial gaps remain in Canada’s infrastructure to develop and conduct pediatric cancer academic trials, as well as to participate in international academic trials. A long-standing partnership between The C17 Council (C17) and ACCESS is poised to change that.

C17 was born out of the implementation of new Health Canada legislation in 2001, which mandated more stringent regulatory requirements for the conduct of academic and consortia-led drug trials. Today, it serves as an authoritative Canadian voice and provides significant expertise and support for academic pediatric cancer clinical trials in Canada. Together, C17 and ACCESS are working to build capacity and improve infrastructure for the development and conduct of academic trials and forge international collaborations to position Canada as a stronger, more connected member in the global pediatric oncology clinical trials ecosystem.

“C17 has been one of ACCESS’ core partners since its inception – they were among the key stakeholder groups that came together to support its successful CIHR application,” says Christine Williams, Executive Director, ACCESS. “We are now well positioned to harness their insight and expertise to navigate both national and international hurdles related to clinical trials, and to collaborate on specific high-impact projects as well.”

Canada’s geography and healthcare system adds additional complexity. With 13 provinces and territories, each managing its own healthcare and ethics frameworks, the system is fragmented. The opening of clinical trials at multiple centres makes them considerably more complex and expensive; however, crossing provincial borders to access trials is often as difficult as crossing national ones. Both Canadian investigators and international academic trial groups require support to navigate these layers of complexity as well as the relevant federal regulations.

“We’re working with regulatory models and healthcare systems built for an adult population in an earlier era, and one that is not well adapted to conducting clinical trials to lead to new treatments in rare or pediatric populations,” says Kathy Brodeur-Robb, Executive Director, C17 and member of the Senior Leadership Committee, ACCESS. “Canada needs to participate in the international academic collaboration developing new clinical trials and treatments and, given the constraints we face, to make sure that Canadian children are not left behind or without options”.

The partnership will specifically support the development of streamlined frameworks that still meet Canadian and international regulatory, legal and ethical clinical trial requirements – both for Canadian investigator-led trials and international consortia trials being conducted across sites in Canada. Three transformative examples of clinical trials currently supported by both C17 and ACCESS that will benefit from these modernizations include:

• OPTIMISE – An international platform trial co-developed and co-led by investigators in Australia and Canada that will develop and test novel therapies for patients identified by multi-omic sequencing through a national cancer sequencing program.
• DECRYPT – A Canadian-led trial to assess the safety and tolerability of a novel radiation-free treatment for patients less than 6 years old diagnosed with high-risk central nervous system tumors across Canada.
• AALL2131 – A US Children’s Oncology Group international pilot study led by a Canadian Study Chair that will investigate the benefit of combining chemotherapy with targeted tyrosine kinase inhibitors and blinatumomab, and will provide patients in Canada access to a next-generation sequencing assay as part of their participation.

 

The C17 and ACCESS partnership also brings critical strategic alignment. Now more than ever, collaboration with international academic consortia is key to enable access to innovative clinical trials for pediatric cancer patients. In addition to continuing collaborations with US-led consortia, the partnership is building bridges to European and Australian consortia to expand opportunities for children in Canada to participate in cutting-edge research developed elsewhere.

“Together, C17 and ACCESS want Canada to be a preferred destination for all international pediatric cancer clinical trials to ensure our kids have access to the most innovative therapies. We’re not just importing ideas—we’re exporting them, too,” Kathy adds. “Canada has developed real expertise in rare disease frameworks and developing plans for remote access to clinical trials, and other countries are learning from us. That kind of mutual sharing is essential for moving the needle in pediatric cancer.”

As therapies become more targeted, expensive, and technologically complex, the barriers to accessing them through clinical trials are growing—not shrinking. “The C17–ACCESS partnership is seizing this moment to strengthen Canada’s pediatric cancer trials ecosystem around collaboration, problem-solving, and innovation. We are building a future in which children in Canada have access to the best treatments in the world, regardless of geography or jurisdiction”, says Christine.

Pediatric oncology in Canada has never lacked passion or expertise. But to meet the evolving needs of children and families so they can access the newest and most promising therapies through clinical trials, we must strengthen the structures and expertise that support the pediatric cancer trials ecosystem. The C17 and ACCESS collaboration is doing just that—breaking down silos, expanding clinical trial infrastructure and capacity, and creating a future in which no child with cancer in Canada is left behind.