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Objectives Access to early phase trials for children with relapsed, refractory or progressive (RRPD) cancer is limited in Canada. Patients and families face barriers to access trials, which are poorly understood. The aims of this study were to assess availability of early phase trials and examine the impact of distance from home to study centre on trial enrolment among paediatric oncology patients with RRPD. Methods Oncology patients ≤18 years at diagnosis who later had RRPD were identified retrospectively via registry at the only quaternary paediatric oncology centre in British Columbia (BC). We determined if distance to home, as calculated using geocoding software, was predictive of trial offer or enrolment. Results Between January 2015 and July 2021, 266 patients experienced 396 RRPD events. Seventy-five patients (28.2%) were eligible for an early phase trial at least once. At first eligible event, 61 patients (22.9%) were offered trial (median age 11.8 years; 69.0% male; 46.0% with CNS tumour) and thirty patients (11.3%) enrolled. Distance was not associated with odds of offer (OR 1.01, CI 0.98 to 1.05) or enrolment (OR 0.99, CI 0.95 to 1.03) on univariate or multivariable analysis adjusted for sex and disease (OR 0.93, CI 0.86 to 1.00). For offered patients, 2-year event-free survival (EFS) and overall survival (OS) were 39.1% (CI 28.0% to 54.8%) and 51.8% (CI 39.9% to 67.2%), respectively. EFS/OS did not differ with distance or enrolment, but varied by disease (EFS P = 0.002, OS P < 0.0001). Conclusions Children in BC with cancer and RRPD have limited access to early phase trials. Distance was not predictive of enrolment, suggesting that families travel to access therapy. [ABSTRACT FROM AUTHOR]

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. [ABSTRACT FROM AUTHOR]

While paediatric care providers are often the first point of contact for children or youth experiencing mental health challenges, they may lack the resources (e.g. access to a multidisciplinary team) or training to adequately identify or manage such problems. This joint statement describes the key roles and competencies required to assess and address child and youth mental health problems, and the factors that optimize outcomes in this age group. Evidence-informed guidance on screening for and discussing mental health concerns with young people and families is provided. Preventive and therapeutic interventions with demonstrated efficacy in community care settings are discussed. This foundational statement also focuses on the changes to medical education, health systems, and health policy that are needed to improve clinical practice and advocacy efforts in Canada, including appropriate remuneration models, stepped-care approaches, targeted government funding, and professional training and education. [ABSTRACT FROM AUTHOR]

Background: Children with adrenal suppression (AS), a potential side effect of glucocorticoids (GCs) may be asymptomatic, present with nonspecific signs and symptoms or with adrenal crisis. Asymptomatic AS (AAS) can only be diagnosed through screening. Identifying and treating asymptomatic patients before symptoms develop may reduce morbidity. Screening guidelines for AS are lacking. Consequently, screening practices are highly variable. Objective: To assess (1) the screening practices for and recognition of paediatric AAS among clinicians in Canada and (2) the educational impact of a 2-year surveillance program of symptomatic AS cases. Methods: Before and after a 2-year Canadian Paediatric Surveillance Program (CPSP) study of symptomatic AS, participants were surveyed through the CPSP. The prestudy survey was sent to 2,548 participants in March 2010 and the poststudy survey was sent to 2,465 participants in April 2013. Results: Response rates were 32% for the prestudy survey and 21% for the poststudy survey. Between the pre- and poststudy surveys, the percentage of physicians who reported routinely screening patients on GCs for AS increased from 10% to 21% and the percentage who reported having a screening policy in their office/centre increased from 6% to 11%. There was no significant change in the percentage of physicians who had diagnosed a child/youth with AAS in the preceding year. Conclusion: Frequency of screening for AAS increased following the 2-year study but remains low. Development of a clinical practice guideline should increase both awareness of asymptomatic AS among Canadian paediatricians and the identification of AAS, before symptoms develop. [ABSTRACT FROM AUTHOR]