Ensuring access to innovative therapies for children, adolescents, and young adults across Canada: The single patient study experience.

Innovative therapeutic approaches are needed to alleviate the burden of life-limiting, rare, and chronic conditions affecting children, adolescents, and young adults (CAYA). This includes a need for improved access to both clinical research and to non-approved or off-label therapies, together with, ultimately, more therapies achieving regulatory approval in Canada. The single patient study (SPS), also known as an open label individual patient (OLIP) study, was introduced by Health Canada to open access to non-marketed drugs where a clinical trial is not readily available, but the drug is considered too investigational to be managed on a standard Special Access Program. SPS is designed for patients who have a serious or life-threatening condition and have exhausted available treatment options. Our report summarizes this relatively new development in the Canadian regulatory environment and highlights the opportunities and challenges as identified by regulators, pharmaceutical representatives, academic researchers, and patient/parent advocates.
Competing Interests: RJD reports payment from Bayer Canada for chairing a virtual symposium. DAM received consulting fees from ymAbs Therapeutics, Clarity Pharmaceuticals and EUSA Pharma, payment/honoraria from HMP Global, Ology Medical Education, WebMD Global and Platform Q Health Education, and is on an advisory board for Clarity Pharmaceuticals and ymAbs Therapeutics. DAM also reports that his institution received a research grant from BMS. JAW reports that his institution received research funding from Novartis, Daiichi Sankyo and Syndax for a clinical trial, received payment from Amgen for an educational event, and participation on advisory boards for Jazz Pharmaceuticals. There are no other disclosures.
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