IMPORTANCE Tisagenlecleucel, a chimeric antigen receptor T-cell therapy for relapsed or refractory pediatric acute lymphoblastic leukemia, has been approved for use in multiple jurisdictions. The public list price is US $475 000, or more than CaD $600 000. Assessing the cost-effectiveness of tisagenlecleucel is necessary to inform policy makers on the economic value of this treatment.OBJECTIVE To assess the value for money of tisagenlecleucel compared with current standard care for tisagenlecleucel-eligible pediatric patients with acute lymphoblastic leukemia under unknown long-term effectiveness. DESIGN, SETTING, AND PARTICIPANTSA cost-utility analysis of tisagenlecleucel compared with current standard care using a Canadian population-based registry of pediatric patients with acute lymphoblastic leukemia was performed. Results from 3 pooled single-arm tisagenlecleucel clinical trials and a provincial pediatric cancer registry were combined to create treatment and control arms, respectively. The population-based control arm consisted of patients meeting clinical trial inclusion and exclusion criteria, starting at second relapse. Multistate and individual-level simulation modeling were combined to predict patient lifetime health trajectories by treatment strategy. Tisagenlecleucel efficacy was modeled across long-term cure rates, from 10% to 40%, to account for limited information on its long-term effectiveness. Uncertainty was tested with 1-way and probabilistic sensitivity analysis. Data were collected in September 2017, and analysis began in December 2017.EXPOSURES Tisagenlecleucel compared with current standard care for tisagenlecleucel-eligible patients.MAIN OUTCOMES AND MEASURES Relative health care costs, survival gains, and quality-adjusted life-years (QALYs) between tisagenlecleucel and current standard care. RESULTSThe treatment and control arms were modeled on 192 and 118 patients, respectively. The mean (SD) age of control individuals was 10 (4.25) years, and the mean (SD) age of the pooled clinical trial sample was 11 (6) years. The control individuals had 78 boys (66%), and the pooled clinical trial sample had 102 boys (53%).

Background Adolescent women treated for Hodgkin lymphoma (HL) are at increased risk of breast cancer (BC). We evaluate the cost-utility of eight high-risk BC surveillance strategies for this population, including the Children’s Oncology Group guideline of same-day annual mammography and magnetic resonance imaging (MRI) beginning at age 25 years. Methods A discrete event simulation model was used to simulate the life histories of a cohort of 500 000 25-year-old women treated for HL at age 15 years. We estimated BC incidence and mortality, life expectancy, quality-adjusted life-years (QALYs), health-care costs, and the relative cost-utility (incremental cost-utility ratio [ICUR]) under the eight assessed surveillance strategies. One-way sensitivity analysis enabled modeling of uncertainty evaluation. A publicly funded health-care payer perspective was adopted. Results Costs across the eight screening strategies ranged from $32 643 to $43 739, whereas QALYs ranged from 24.419 to 24.480. In an incremental cost-effectiveness analysis, annual mammography beginning at age 25 years was associated with an ICUR of $43 000/QALY gained, annual MRI beginning at age 25 years with a switch to annual mammography at age 50 years had an ICUR of $148 000/QALY, and annual MRI beginning at age 25 years had an ICUR of $227 222/QALY. Among all assessed surveillance strategies, the differences in life expectancy were small. Conclusions Current high-risk BC surveillance guidelines do not reflect the most cost-effective strategy in survivors of adolescent HL. The results suggest that groups at high risk of BC may require high-risk surveillance guidelines that reflect their specific risk profile.

ADHD diagnoses increase discontinuously by a child's school starting age, with young‐for‐grade students having much higher ADHD diagnostic rates. Whether these higher rates reflect over‐diagnosis or under‐diagnosis remains unknown. To decompose this diagnostic discrepancy, we exploit differences in parent and teacher pre‐diagnostic assessments within a regression discontinuity strategy based on school starting age. We show that being young‐for‐grade or male generates over‐assessment of symptoms specifically from teacher assessment. However, under‐assessments of the oldest students in a grade, especially the oldest females, account for a large part of the observed school starting age assessment gap. We argue that this difference by sex and higher school starting age effects in lower‐income schools may exacerbate known gaps in educational attainment by gender and socioeconomic status. Importantly, we fail to find evidence that teachers who receive special education training make such errors.

Over the 10-year time frame, the immediate loading of dental implants with mandibular fixed prosthesis proved to cost similar to the conventional loading protocol, underscoring the feasibility and reliability of this protocol from the patient's perspective. (REB protocol reference # 33395).