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

Abstract

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.

Access to innovative therapies in pediatric oncology: Report of the nationwide experience in Canada

“…At the time of request, 49% of patients were 0-11 years old, 42% During the study period, requests for cytotoxic chemotherapy, cellular products, and cytokines were inconsistently captured and showed dramatic heterogeneity between centers. Requests reported included tisagenleucel (2), aldesleukin (11), dinutuximab (6), interferon alpha (4) and cytotoxic chemotherapy (clofarabine, treosulfan, temozolomide, pegylated liposomal doxorubicin, nelarabine, cladribine, thiotepa, azacitidine and trabectidine, 45 requests in total). We excluded these from the analysis.…”

“…Pediatric‐friendly dosage forms (e.g., oral liquids, dispersible tablets) still under investigation may be made available for pediatric patients through a similar mechanism. When accessed at an early stage of development, with limited safety data in the pediatric population, or in the context of novel combinations, innovative therapies can be accessed through the single patient study mechanism 11 …”

“…When accessed at an early stage of development, with limited safety data in the pediatric population, or in the context of novel combinations, innovative therapies can be accessed through the single patient study mechanism. 11 The pediatric oncology community have consistently stated that the preferred route of access to innovative therapies is participation in a clinical trial. 12 However, recognition is growing that this is not always feasible and that there are frequent uses of innovative therapies "out of trial" in pediatric oncology.…”

“…When accessed at an early stage of development, with limited safety data in the pediatric population, or in the context of novel combinations, innovative therapies can be accessed through the single patient study mechanism. 11 …”