BACKGROUND: Coronavirus disease 2019 (COVID-19) disrupted pediatric oncology care globally, increasing demands on health care providers (HCPs) who adapted to continue care. This study sought to characterize the pandemic's impact on pediatric oncology HCPs worldwide. METHODS: A 60-item survey focused on changes to clinical care, resources, and effects on clinicians. A diverse subgroup of institutions was purposefully selected for focus groups that explored teamwork, communication, and changes to care delivery. RESULTS:The survey included 311 responses from 213 institutions representing 79 countries. Sixteen institutions participated in 19 multidisciplinary focus groups in 8 languages. Decreased clinical staff availability was cited by 51% of institutions as a major impact. Staffing modifications included decreased provider availability (66% of institutions), roles or responsibility changes, and transfer outside the specialty. Physical effects included frequent COVID-19 illness; 8% of respondents reported HCP deaths. Fifty percent of providers did not have the necessary personal protective equipment. HCPs also experienced psychological distress and financial concerns. Findings indicated more frequent impact on nurses than other providers. Impacts were described across all hospital resource levels, with staffing modifications more frequent in countries with higher COVID-19 incidence (P < .001) and mortality rate (P = .004). Focus groups revealed negative impacts were stabilized by increased teamwork, communication, contributions outside usual roles, policies aimed at optimizing safety, and feeling that they were contributing. CONCLUSIONS: COVID-19 had a profound impact on the pediatric oncology workforce, creating challenging modifications to staffing and resulting in physical, psychological, and financial distress. Despite these challenges, HCPs caring for children with cancer came together to continue to provide high-quality care.

Adeno-associated virus (AAV)-mediated gene therapy is a novel treatment promising to reduce morbidity associated with hemophilia. While multiple clinical trials continue to evaluate efficacy and safety, limited cost-effectiveness data have been published. This study compared the potential cost-effectiveness of AAV-mediated factor IX(FIX)-Padua gene therapy for severe hemophilia B patients in the United States (US) to on-demand FIX replacement and primary FIX prophylaxis, using either standard or extended half-life FIX products. A microsimulation Markov model was constructed and transition probabilities between health states and utilities were informed by published data. Costs were aggregated using a micro-costing approach. An 18-years-old till death time-horizon from the perspective of a third-party payer in the US was conducted. Gene therapy was more cost-effective than both alternatives considering a $150,000/QALY threshold. The price for gene therapy was assumed $2,000,000 in the base-case scenario, yet one of the one-way sensitivity analyses was conducted using observed manufacturing, administration and five-year follow-up cost of $87,198 for AAV-mediated gene therapy vector as derived from the manufacturing facility and clinical practice at St. Jude Children's Research Hospital. One-way sensitivity analyses showed 10/102 scenarios in which gene therapy was not cost-effective compared to alternative treatments. Notably, gene therapy remained cost-effective in a hypothetical scenario in which we estimated that the discounted factor concentrate price was 20% of the wholesale acquisition cost in the US. Probabilistic sensitivity analysis estimated gene therapy cost-effective at 92% of simulations considering $150,000/QALY threshold. In conclusion, based on detailed simulation inputs and assumptions, gene therapy was more cost-effective than on-demand treatment and prophylaxis for patients with severe hemophilia B.

Historically, qualitative research has complemented quantitative biologic and epidemiologic studies to provide a more complete understanding of pandemics. The COVID-19 pandemic has generated unique and novel challenges for qualitative researchers, who have embraced creative solutions including virtual focus groups and rapid analyses to continue their work. We present our experience conducting a multilingual global qualitative study of healthcare resilience among teams of pediatric oncology professionals during the COVID-19 pandemic. We provide an in-depth description of our methodology and an analysis of factors we believe contributed to our study’s success including our use of technology, engagement of a large multilingual team, global partnerships, and framework-based rapid analysis. We hope these techniques may be useful to qualitative researchers conducting studies during the current pandemic, as well as for all pediatric oncology studies including multiple languages or geographically disparate subjects.

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