Joint recommendations on cost calculation and estimation in paediatric clinical trials.

The conduct of clinical trials in paediatrics is essential to improve drug therapy in children. In Europe, paediatric clinical trials have been supported by the European Paediatric Regulation since 2007, but there is still a great need for high-quality clinical trials. The personnel and time required to conduct clinical trials in accordance with EU Regulations 536/2014 and 745/2017 is considerably higher compared to other studies, such as observational studies. It is important that this additional workload for the trial centre is fully compensated, also taking into account EU state aid rules. In paediatric trials, it is necessary to take into account the special requirements of paediatric and adolescent medicine when calculating the additional costs. Within the framework of the pan-European paediatric study network c4c/GermanNetPaeT, a working group dealt with specific aspects of cost calculation in order to support paediatric study centres in internal cost calculation as well as in the subsequent preparation of financing requirements for industrial sponsors or public funders. In several workshops the working group developed a cost calculation template with the content derived from the "Joint recommendations for a total services account as a factor in simplifying contracts" of the Deutsche Hochschulmedizin (DHM, German University Medicine), the Netzwerk der Koordinierungszentren für Klinische Studien (KKS Network, Network of Coordinating Centres for Clinical Trials) and the Verband Forschender Arzneimittelhersteller (vfa, German Association of Research-Based Pharmaceutical Companies). By estimating the specific time required for measures and investigations as part of a sample study, the background to the increased time required was discussed and a list with aspects to be considered for cost calculation was compiled together with the study centres. The paediatrics-specific aspects mentioned in detail are intended to increase understanding of the particular problem of higher costs for clinical trials involving children and adolescents and the need for correspondingly appropriate remuneration. This transparent and comprehensible presentation of the higher financial requirements for both the study centres and the financial supporters is intended to promote the high-quality conduct of clinical trials in paediatric study centres in the long term.

Research prioritization in paediatric orthopaedics and the impact on funding.

In 2017, the British Society for Children's Orthopaedic Surgery engaged the profession and all relevant stakeholders in two formal research prioritization processes. In this editorial, we describe the impact of this prioritization on funding, and how research in children's orthopaedics, which was until very recently a largely unfunded and under-investigated area, is now flourishing. Establishing research priorities was a crucial step in this process.

Evaluation of non-research and research industry payments to pediatric hematologist/oncologists in the United States between 2013 and 2021.

Financial interactions between healthcare industry and pediatric hematologist/oncologists (PHOs) could be conflicts of interest. Nevertheless, little is known about financial relationships between healthcare industry and PHOs. This cross-sectional analysis of the Open Payments Database examined general and research payments to PHOs from healthcare industry in the United States between 2013 and 2021. Payments to the PHOs were analyzed descriptively. Trends in payments were assessed using generalized estimating equation models. Of 2784 PHOs, 2142 (76.9%) PHOs received payments totaling $187.3 million from the healthcare industry between 2013 and 2021. Approximately, $46.3 million (24.8%) were general payments and $137.7 million (73.5%) were funding for research where PHOs served as principal investigators (associated research funding). Both general payments and associated research funding considerably increased between 2014 and 2019. The number of PHOs receiving general payments and associated research funding annually increased by 2.2% (95% CI: 1.2-3.3%, p < .001) and 5.0% (95% CI: 3.3-6.8%, p < .001) between 2014 and 2019, respectively.

Pediatric neurosurgery through the lens of time-driven activity-based costing: a pilot study.

OBJECTIVE: Time-driven activity-based costing (TDABC) is a method used in cost accounting that has gained traction in health economics to identify value optimization initiatives. It measures time, assigns value to time increments spent on a patient, and integrates the cost of material and human resources utilized in each episode of care. In this study, the authors report the first use of TDABC to evaluate costs in a pediatric neurosurgical practice. METHODS: A clinical pathway was developed with a multifunction team. A time survey among each care team member, including surgeons, medical assistants (MAs), and patient service representatives (PSRs), was carried out prospectively over a 10-week period at a pediatric neurosurgery clinic. Consecutive patient encounters for Chiari malformation (CM), hydrocephalus, or tethered cord syndrome (TCS) were included. Encounters were categorized as new or established. Relative annual personnel costs, using the salary of a PSR as a reference (i.e., 1.0-unit cost), were calculated for all members using departmental financial data after adjustments. The relative capacity cost rates (minute-1) for each personnel, a representation of per capita cost per minute, were then derived, and the relative costs per visit were calculated. RESULTS: A total of 110 visits (24 new, 86 established) were captured, including 40% CM, 41% hydrocephalus, and 19% TCS encounters. Surgeons had the highest relative capacity cost rate (118.4 × 10-6), more than 10-fold higher than that of an MA or PSR (10.65 × 10-6 and 9.259 × 10-6, respectively). Surgeons also logged more time with patients compared with the rest of the care team in nearly all visits (p ≤ 0.002); consequently, the total visit costs were primarily driven by the surgeon cost (p < 0.0001). Overall, surgeon cost constituted the vast majority of the total visit cost (92%-93%), regardless of whether the visits were new or established. Visit costs did not differ by diagnosis. On average, new visits took longer than established visits (p < 0.001). This difference was largely driven by new CM visits (44.3 ± 13.7 minutes), which were significantly longer than established CM visits (29.8 ± 9.2 minutes; p = 0.001). CONCLUSIONS: TDABC may reveal opportunities to maximize value by highlighting instances of variability and high cost in each module of care delivery. Physician leaders in pediatric neurosurgery may be able to use this information to allocate costs and streamline value care pathways.

Evaluation of claims data from a commercial value-based insurance product shows pediatric imaging is not a major driver of overall or pediatric healthcare expenditures.

BACKGROUND: Initiatives to reduce healthcare expenditures often focus on imaging, suggesting that imaging is a major driver of cost. OBJECTIVE: To evaluate medical expenditures and determine if imaging was a major driver in pediatric as compared to adult populations. METHODS: We reviewed all claims data for members in a value-based contract between a commercial insurer and a healthcare system for calendar years 2021 and 2022. For both pediatric (<18 years of age) and adult populations, we analyzed average per member per year (PMPY) medical expenditures related to imaging as well as other categories of large medical expenses. Average PMPY expenditures were compared between adult and pediatric patients. RESULTS: Children made up approximately 20% of members and 21% of member months but only 8-9% of expenditures. Imaging expenditures in pediatric members were 0.2% of the total healthcare spend and 2.9% of total pediatric expenditures. Imaging expenditures per member were seven times greater in adults than children. The rank order of imaging expenditures and imaging modalities was also different in pediatric as compared to adult members. CONCLUSION: Evaluation of claims data from a commercial value-based insurance product shows that pediatric imaging is not a major driver of overall, nor pediatric only, healthcare expenditures.

Cost-effectiveness of running a paediatric oncology unit in Ethiopia.

OBJECTIVE: To estimate the cost-effectiveness of running a paediatric oncology unit in Ethiopia to inform the revision of the Ethiopia Essential Health Service Package (EEHSP), which ranks the treatment of childhood cancers at a low and medium priority. METHODS: We built a decision analytical model-a decision tree-to estimate the cost-effectiveness of running a paediatric oncology unit compared with a do-nothing scenario (no paediatric oncology care) from a healthcare provider perspective. We used the recently (2018-2019) conducted costing estimate for running the paediatric oncology unit at Tikur Anbessa Specialized Hospital (TASH) and employed a mixed costing approach (top-down and bottom-up). We used data on health outcomes from other studies in similar settings to estimate the disability-adjusted life years (DALYs) averted of running a paediatric oncology unit compared with a do-nothing scenario over a lifetime horizon. Both costs and effects were discounted (3%) to the present value. The primary outcome was incremental cost in US dollars (USDs) per DALY averted, and we used a willingness-to-pay (WTP) threshold of 50% of the Ethiopian gross domestic product per capita (USD 477 in 2019). Uncertainty was tested using one-way and probabilistic sensitivity analyses. RESULTS: The incremental cost and DALYs averted per child treated in the paediatric oncology unit at TASH were USD 876 and 2.4, respectively, compared with no paediatric oncology care. The incremental cost-effectiveness ratio of running a paediatric oncology unit was USD 361 per DALY averted, and it was cost-effective in 90% of 100 000 Monte Carlo iterations at a USD 477 WTP threshold. CONCLUSIONS: The provision of paediatric cancer services using a specialised oncology unit is most likely cost-effective in Ethiopia, at least for easily treatable cancer types in centres with minimal to moderate capability. We recommend reassessing the priority-level decision of childhood cancer treatment in the current EEHSP.

Telehealth: Opportunities to Improve Access, Quality, and Cost in Pediatric Care.

The use of telehealth technology to connect with patients has expanded significantly over the past several years, particularly in response to the global coronavirus disease 2019 pandemic. This technical report describes the present state of telehealth and its current and potential applications. Telehealth has the potential to transform the way care is delivered to pediatric patients, expanding access to pediatric care across geographic distances, leveraging the pediatric workforce for care delivery, and improving disparities in access to care. However, implementation will require significant efforts to address the digital divide to ensure that telehealth does not inadvertently exacerbate inequities in care. The medical home model will continue to evolve to use telehealth to provide high-quality care for children, particularly for children and youth with special health care needs, in accordance with current and evolving quality standards. Research and metric development are critical for the development of evidence-based best practices and policies in these new models of care. Finally, as pediatric care transitions from traditional fee-for-service payment to alternative payment methods, telehealth offers unique opportunities to establish value-based population health models that are financed in a sustainable manner.

Acceleration of mobile health for monitoring post-transplant in the COVID-19 era: Applications for pediatric settings.

BACKGROUND: Since the start of the COVID-19 pandemic and consequent lockdowns, the use of telehealth interventions has rapidly increased both in the general population and among transplant recipients. Among pediatric transplant recipients, this most frequently takes the form of interventions on mobile devices, or mHealth, such as remote visits via video chat or phone, phone-based monitoring, and mobile apps. Telehealth interventions may offer the opportunity to provide care that minimizes many of the barriers of in-person care. METHODS: The present review followed the PRISMA guidelines. Sources up until October 2020 were initially identified through searches of PsycInfo® and PubMed® . RESULTS: We identified ten papers that reported findings from adult interventions and five studies based in pediatrics. Eight of the adult publications stemmed from the same two trials; within the pediatric subset, this was the case for two papers. Studies that have looked at mHealth interventions have found high acceptability rates over the short run, but there is a general lack of data on long-term use. CONCLUSIONS: The literature surrounding pediatric trials specifically is sparse with all findings referencing interventions that are in early stages of development, ranging from field tests to small feasibility trials. The lack of research highlights the need for a multi-center RCT that utilizes robust measures of medication adherence and other outcome variables, with longer-term follow-up before telehealth interventions should be fully embraced.

Correlation Between National Institutes of Health Funding for Pediatric Research and Pediatric Disease Burden in the US.

Importance: The US National Institutes of Health (NIH) is the largest government funding source for biomedical research globally. Burden of disease is one of the factors considered by the NIH in making funding allocations, though it is not known how funding patterns are associated with disease burden for pediatric conditions. Objective: To determine the correlation between NIH funding and disease burden across pediatric conditions. Design, Setting, and Participants: This cross-sectional study evaluates NIH grants funding pediatric research from 2015 to 2018 in the US. Pediatric grants were classified according to disease categories studied. Disease burden for each category was determined using measures from the Institute of Health Metrics and Evaluation and hospitalization data from the 2016 Kids' Inpatient Database. Main Outcome and Measure: Correlation between NIH funding and pediatric disease burden using Spearman rank order coefficients and predicted amounts of disease-specific funding based on disease burden estimated from linear regression models. Results: This study analyzed 14 060 disease-specific pediatric grants awarded by the NIH from 2015 to 2018 in the US. Annual funding for disease categories ranged from $0 to $382 849 631. Funding for pediatric research was correlated with pediatric disability-adjusted life-years (DALYs), deaths, years lived with disability, and years of life lost (r, 0.56-0.63; P < 0.001 for all measures). There was also a correlation between funding and hospital-based metrics, including hospital days, number of hospital admissions, and hospital charges (r, 0.67-0.69; P < .001 for all measures). Eight disease categories received greater than $500 million more than predicted levels relative to DALYs, while 5 disease categories were funded more than $50 million less than predicted levels. Based on predicted levels of funding, congenital birth defects; endocrine, metabolic, blood, and immune disorders; and HIV/AIDS were the most overfunded categories relative to DALYs and hospital days. Conditions identified as most underfunded differed depending on use of DALYs or hospital days in estimating predicted funding levels. Conclusions and Relevance: NIH funding for pediatric research was correlated with pediatric disease burden in the US with variable correlation based on the disease metric applied. There was substantial overfunding and underfunding of certain conditions. Ongoing evaluation of pediatric funding patterns using a complementary set of disease measures may help inform and prioritize pediatric research funding.

Simulación de intubación pediátrica con videolaringoscopio de bajo costo en contexto de pandemia por COVID-19 / Simulation of pediatric intubation using a low-cost videolaryngoscope in the setting of the COVID-19 pandemic

En pacientes con infección por SARS-CoV-2 la intubación endotraqueal es un procedimiento con riesgo elevado de contagio. La videolaringoscopia complementa la protección del profesional, pero los videolaringoscopios comerciales son caros y no siempre están disponibles en las terapias intensivas pediátricas argentinas. El objetivo fue describir la práctica de intubación en un modelo de cabeza de simulación de lactante con un videolaringoscopio artesanal de bajo costo.Quince pediatras sin experiencia previa con el dispositivo participaron de una práctica de intubación en una cabeza de simulación con un videolaringoscopio artesanal. El tiempo promedio del primer intento fue de 116,4 segundos (intervalo de confianza del 95 % [IC95 %]: 84,8-148,0) y, el del siguiente fue de 44,2 segundos (IC95 %: 27,7­60,6). El tiempo disminuyó de forma significativa en el segundo intento (p : 0,0001). El dispositivo permitió la intubación exitosa en todos los intentos acortando la duración del procedimiento en la segunda práctica

In patients with SARS-CoV-2 infection, endotracheal intubation is a procedure with a high risk for transmission. A videolaryngoscopy is a supplementary level of health care provider protection, but commercial videolaryngoscopes are expensive and not always available in pediatric intensive care units in Argentina. Our objective was to describe intubation practice using an infant head mannequin with a low-cost, handcrafted videolaryngoscope.Fifteen pediatricians with no prior experience using the device participated in an intubation practice in a head mannequin with a handcrafted videolaryngoscope. The average time for the first attempt was 116.4 seconds (95 % confidence interval [CI]: 84.8-148.0) and, for the second one, 44.2 seconds (95 % CI: 27.7-60.6). Time decreased significantly for the second attempt (p: 0.0001).A successful intubation was achieved with the device in all attempts, and the procedure duration decreased with the second practice

Short-term general, gynecologic, orthopedic, and pediatric surgical mission trips in Nicaragua: A cost-effectiveness analysis.

BACKGROUND: Short-term surgical missions facilitated by non-governmental organizations (NGOs) may be a possible platform for cost-effective international global surgical efforts. The objective of this study is to determine if short-term surgical mission trips provided by the non-governmental organization (NGO) Esperança to Nicaragua from 2016 to 2020 are cost-effective. METHODS: Using a provider perspective, the costs of implementing the surgical trips were collected via Esperança's previous trip reports. The reports and patient data were analyzed to determine disability-adjusted life years averted from each surgical procedure provided in Nicaragua from 2016-2020. Average cost-effectiveness ratios for each surgical trip specialty were calculated to determine the average cost of averting one disability-adjusted life year. RESULTS: Esperança's surgical missions' program in Nicaragua from 2016 to 2020 was found to be cost-effective, with pediatric and gynecology surgical specialties being highly cost-effective and general and orthopedic surgical specialties being moderately cost-effective. These results were echoed in both scenarios of the sensitivity analysis, except for the orthopedic specialty which was found to not be cost-effective when testing an increased discount rate. CONCLUSIONS: The cost-effectiveness of short-term surgical missions provided by NGOs can be cost-effective, but limitations include inconsistent data from a societal perspective and lack of an appropriate counterfactual. Future studies should examine the capacity for NGOs to collect adequate data and conduct rigorous economic evaluations.

Simulation of pediatric intubation using a low-cost videolaryngoscope in the setting of the COVID-19 pandemic. / Simulación de intubación pediátrica con videolaringoscopio de bajo costo en contexto de pandemia por COVID-19.

In patients with SARS-CoV-2 infection, endotracheal intubation is a procedure with a high risk for transmission. A videolaryngoscopy is a supplementary level of health care provider protection, but commercial videolaryngoscopes are expensive and not always available in pediatric intensive care units in Argentina. Our objective was to describe intubation practice using an infant head mannequin with a low-cost, handcrafted videolaryngoscope. Fifteen pediatricians with no prior experience using the device participated in an intubation practice in a head mannequin with a handcrafted videolaryngoscope. The average time for the first attempt was 116.4 seconds (95 % confidence interval [CI]: 84.8- 148.0) and, for the second one, 44.2 seconds (95 % CI: 27.7-60.6). Time decreased significantly for the second attempt (p: 0.0001). A successful intubation was achieved with the device in all attempts, and the procedure duration decreased with the second practice.

En pacientes con infección por SARS-CoV-2 la intubación endotraqueal es un procedimiento con riesgo elevado de contagio. La videolaringoscopia complementa la protección del profesional, pero los videolaringoscopios comerciales son caros y no siempre están disponibles en las terapias intensivas pediátricas argentinas. El objetivo fue describir la práctica de intubación en un modelo de cabeza de simulación de lactante con un videolaringoscopio artesanal de bajo costo. Quince pediatras sin experiencia previa con el dispositivo participaron de una práctica de intubación en una cabeza de simulación con un videolaringoscopio artesanal. El tiempo promedio del primer intento fue de 116,4 segundos (intervalo de confianza del 95 % [IC95 %]: 84,8-148,0) y, el del siguiente fue de 44,2 segundos (IC95 %: 27,7­60,6). El tiempo disminuyó de forma significativa en el segundo intento (p : 0,0001). El dispositivo permitió la intubación exitosa en todos los intentos acortando la duración del procedimiento en la segunda práctica.

National Pediatric Experience With Virtual Interviews: Lessons Learned and Future Recommendations.

The coronavirus disease 2019 pandemic significantly impacted undergraduate and graduate medical education and created challenges that prevented a traditional approach to residency and fellowship recruitment and interviews. Early in the pandemic, the pediatric education community came together to support applicants and training programs and to foster an equitable recruitment process. We describe many of our community's innovations, including the use of virtual cafés to educate programs and highlight best practices for virtual recruitment and the use of regional webinars to highlight residency programs and provide information to applicants. Surveys of applicants and programs suggest that the virtual interview process worked well overall, with applicants and programs saving both time and money and programs maintaining a high rate of filling their positions. On the basis of this experience, we highlight the strengths and weaknesses of 3 potential models for future interview seasons. We close with a series of questions that need further investigation to create an effective and equitable recruitment process for the future.

Differences in Lifetime Earning Potential Between Pediatric and Adult Physicians.

OBJECTIVES: Compare lifetime earning potential between academic pediatric and adult medicine generalists and subspecialists. Evaluate the effect of decreasing the length of training for pediatric subspecialties whose length of training is longer than that for the adult medicine counterpart. METHODS: Using compensation and debt data from national physician surveys for 2019-2020, we estimated and compared the lifetime earning potential for academic pediatric and adult physicians. RESULTS: Lifetime earning potential was higher for adult physicians than for pediatric physicians across all comparable areas of both general and subspecialty academic practice. The lifetime earning potentials for adult physicians averaged 25% more, or $1.2 million higher, than those of the corresponding pediatric physicians. These differences predominantly were not attributable to unequal training length: when we modeled a shortened length of training for pediatric subspecialists, lifetime earning potential for adult subspecialists still averaged 19% more than that for pediatric subspecialists. For both pediatric and adult medicine, the primarily inpatient, procedure-oriented subspecialties had higher lifetime earning potential than the outpatient, less procedure-oriented subspecialties. CONCLUSIONS: Wide differences in lifetime earning potential between pediatric and adult physicians reflected lower compensation in pediatrics, rather than any differences in training length. Inpatient-based, more procedure-oriented subspecialties had higher lifetime earning potential than outpatient-based, less procedure-oriented subspecialties. Interventions that improve the lifetime earning potential of general pediatrics and the pediatric subspecialties, as well as the less procedure-oriented subspecialties across both pediatric and adult medicine, have the potential to impact both clinical practice and access to care.