Development of a Value Assessment Framework for Pediatric Health Technologies Using Multicriteria Decision Analysis: Expanding the Value Lens for Funding Decision Making.

OBJECTIVES: A health technology assessment (HTA) does not systematically account for the circumstances and needs of children and youth. To supplement HTA processes, we aimed to develop a child-tailored value assessment framework using a multicriteria decision analysis approach. METHODS: We constructed a multicriteria-decision-analysis-based model in multiple phases to create the Comprehensive Assessment of Technologies for Child Health (CATCH) framework. Using a modified Delphi process with stakeholders having broad disciplinary and geographic variation (N = 23), we refined previously generated criteria and developed rank-based weights. We established a criterion-pertinent scoring rubric for assessing incremental benefits of new drugs. Three clinicians independently assessed comprehension by pilotscoring 9 drugs. We then validated CATCH for 2 childhood cancer therapies through structured deliberation with an expert panel (N = 10), obtaining individual scores, consensus scores, and verbal feedback. Analyses included descriptive statistics, thematic analysis, exploratory disagreement indices, and sensitivity analysis. RESULTS: The modified Delphi process yielded 10 criteria, based on absolute importance/relevance and agreed importance (median disagreement indices = 0.34): Effectiveness, Child-specific Health-related Quality of Life, Disease Severity, Unmet Need, Therapeutic Safety, Equity, Family Impacts, Life-course Development, Rarity, and Fair Share of Life. Pilot scoring resulted in adjusted criteria definitions and more precise score-scaling guidelines. Validation panelists endorsed the framework's key modifiers of value. Modes of their individual prescores aligned closely with deliberative consensus scores. CONCLUSIONS: We iteratively developed a value assessment framework that captures dimensions of child-specific health and nonhealth gains. CATCH could improve the richness and relevance of HTA decision making for children in Canada and comparable health systems.

Precision oncology for children: A primer for paediatricians.

Cancer is the leading cause of disease-related death in children, adolescents, and young adults beyond the newborn period in North America. Improving survival rates for patients with hard-to-cure cancer remains a challenge. One approach that has gained particular traction is 'precision oncology', whereby next-generation sequencing is used to identify genomic or transcriptomic changes that can help clarify the diagnosis, refine prognosis, define an underlying genetic cause, or identify a unique treatment target for a patient's cancer. In this primer, we provide a brief overview of the evolution of precision paediatric oncology, its current application to clinical oncology practice, and its future potential as a foundational approach to paediatric oncology care in Canada and around the world. We also address the many challenges and limitations inherent to the implementation of precision oncology as the standard of care, including ethical and economic considerations.

Access to essential cancer medicines for children: a comparative mixed-methods analysis of availability, price, and health-system determinants in east Africa.

BACKGROUND: Access to essential childhood cancer medicines is a core determinant of childhood cancer outcomes. Available evidence, although scarce, suggests that access to these medicines is highly variable across countries, particularly in low-income and middle-income countries, where the burden of childhood cancer is greatest. To support evidence-informed national and regional policies for improved childhood cancer outcomes, we aimed to analyse access to essential childhood cancer medicines in four east African countries-Kenya, Rwanda, Tanzania, and Uganda-by determining the availability and price of these medicines and the health system determinants of access. METHODS: In this comparative analysis, we used prospective mixed-method analyses to track and analyse the availability and price of essential childhood cancer medicines, investigate contextual determinants of access to childhood cancer medicines within and across included countries, and assess the potential effects of medicine stockouts on treatment. Eight tertiary care hospitals were included, seven were public sites (Kenyatta National Hospital [KNH; Nairobi, Kenya], Jaramogi Oginga Odinga Referral and Teaching Hospital [JOORTH; Kisumu, Kenya], Moi University Teaching and Referral Hospital [MTRH; Eldoret, Kenya], Bugando Medical Centre [BMC; Mwanza, Tanzania], Muhimbili National Hospital [MNH; Dar es Salaam, Tanzania], Butaro Cancer Centre of Excellence [BCCE; Butaro Sector, Rwanda], and Uganda Cancer Institute [UCI; Kampala, Uganda]) and one was a private site (Aga Khan University Hospital [AKU; Nairobi, Kenya]). We catalogued prices and stockouts for 37 essential drugs from each of the eight study siteson the basis of 52 weeks of prospective data that was collected across sites from May 1, 2020, to Jan 31, 2022. We analysed determinants of medicine access using thematic analysis of academic literature, policy documents, and semi-structured interviews from a purposive sample of health system stakeholders. FINDINGS: Recurrent stockouts of a wide range of cytotoxic and supportive care medicines were observed across sites, with highest mean unavailability in Kenya (JOORTH; 48·5%), Rwanda (BCCE; 39·0%), and Tanzania (BMC; 32·2%). Drugs that had frequent stockouts across at least four sites included methotrexate, bleomycin, etoposide, ifosfamide, oral morphine, and allopurinol. Average median price ratio of medicines at each site was within WHO's internationally accepted threshold for efficient procurement (median price ratio ≤1·5). The effect of stockouts on treatment was noted across most sites, with the greatest potential for treatment interruptions in patients with Hodgkin lymphoma, retinoblastoma, and acute lymphocytic leukaemia. Policy prioritisation of childhood cancers, health financing and coverage, medicine procurement and supply chain management, and health system infrastructure emerged as four prominent determinants of access when the stratified purposive sample of key informants (n=64) across all four countries (Kenya n=19, Rwanda n=15, Tanzania n=13, and Uganda n=17) was interviewed. INTERPRETATION: Access to childhood cancer medicines across east Africa is marked by gaps in availability that have implications for effective treatment delivery for a range of childhood cancers. Our findings provide detailed evidence of barriers to access to childhood cancer medicine at multiple points in the pharmaceutical value chain. These data could inform national and regional policy makers to optimise cancer medicine availability and affordability as part of efforts to improve childhood cancer outcomes specific regions and internationally. FUNDING: American Childhood Cancer Organization, Childhood Cancer International, and the Friends of Cancer Patients Ameera Fund.

What's in a Name? Parents' and Healthcare Professionals' Preferred Terminology for Pathogenic Variants in Childhood Cancer Predisposition Genes.

Current literature/guidelines regarding the most appropriate term to communicate a cancer-related disease-causing germline variant in childhood cancer lack consensus. Guidelines also rarely address preferences of patients/families. We aimed to assess preferences of parents of children with cancer, genetics professionals, and pediatric oncologists towards terminology to describe a disease-causing germline variant in childhood cancer. Using semi-structured interviews we asked participants their most/least preferred terms from; 'faulty gene,' 'altered gene,' 'gene change,' and 'genetic variant,' analyzing responses with directed content analysis. Twenty-five parents, 6 genetics professionals, and 29 oncologists participated. An equal number of parents most preferred 'gene change,' 'altered gene,' or 'genetic variant' (n = 8/25). Parents least preferred 'faulty gene' (n = 18/25). Half the genetics professionals most preferred 'faulty gene' (n = 3/6); however this was least preferred by the remaining genetics professionals (n = 3/6). Many oncologists most preferred 'genetic variant' (n = 11/29) and least preferred 'faulty gene' (n = 19/29). Participants across all groups perceived 'faulty gene' as having negative connotations, potentially placing blame/guilt on parents/children. Health professionals described challenges selecting a term that was scientifically accurate, easily understood and not distressing to families. Lack of consensus highlights the need to be guided by families' preferred terminology, while providing accurate explanations regarding implications of genetic findings.

Defining Essential Childhood Cancer Medicines to Inform Prioritization and Access: Results From an International, Cross-Sectional Survey.

PURPOSE: Access to essential cancer medicines is a major determinant of childhood cancer outcomes globally. The degree to which pediatric oncologists deem medicines listed on WHO's Model List of Essential Medicines for Children (EMLc) essential is unknown, as is the extent to which such medicines are accessible on the front lines of clinical care. METHODS: An electronic survey developed was distributed through the International Society of Pediatric Oncology mailing list to members from 87 countries. Respondents were asked to select 10 cancer medicines that would provide the greatest benefit to patients in their context; subsequent questions explored medicine availability and cost. Descriptive and bivariate statistics compared access to medicines between low- and lower-middle-income countries (LMICs), upper-middle-income countries (UMICs), and high-income countries (HICs). RESULTS: Among 159 respondents from 44 countries, 43 (27%) were from LMICs, 79 (50%) from UMICs, and 37 (23%) from HICs. The top five medicines were methotrexate (75%), vincristine (74%), doxorubicin (74%), cyclophosphamide (69%), and cytarabine (65%). Of the priority medicines identified, 87% (27 of 31) are represented on the 2021 EMLc and 77% (24 of 31) were common to the lists generated by LMIC, UMIC, and HIC respondents. The proportion of respondents indicating universal availability for each of the top medicines ranged from 9% to 46% for LMIC, 25% to 89% for UMIC, and 67% to 100% for HIC. Risk of catastrophic expenditure was more common in LMIC (8%-20%), compared with UMIC (0%-28%) and HIC (0%). CONCLUSION: Most medicines that oncologists deem essential for childhood cancer treatment are currently included on the EMLc. Barriers remain in access to these medicines, characterized by gaps in availability and risks of catastrophic expenditure for families that are most pronounced in low-income settings but evident across all income contexts.

Ethical and Social Values for Paediatric Health Technology Assessment and Drug Policy.

BACKGROUND: Public policy approaches to funding paediatric medicines in advanced health systems remain understudied. In particular, the ethical and social values dimensions of health technology assessment (HTA) and drug coverage decisions for children have received almost no attention in research or policy. METHODS: To elicit and understand the social values that influence decision-making for public funding of paediatric drugs, we undertook a series of in-depth, semi-structured interviews with a stratified purposive sample (n = 22) of stakeholders involved with or affected by drug funding decisions for children at the provincial (Ontario) and national levels in Canada. Constructivist grounded theory methodology guided data collection and thematic analysis. RESULTS: Our study provides empirical evidence about the unique ethical and social values dimensions of HTA for children, and describes a novel social values typology for paediatric drug policy decision-making. Three principal categories of values emerged from stakeholder reflections on HTA and drug policy-making for children: procedural values, structural values, and sociocultural values. Key findings include the importance of attention to the procedural legitimacy of HTA for children, with emphasis on the inclusion of child health voices in processes of technology appraisal and policy uptake; a role for HTA institutions to consider the equity impacts of technologies, both in setting review priorities and in assessing the value of technologies for public coverage; and the potential benefits of a distinct national framework to guide drug policy for children. CONCLUSION: Current approaches to HTA are not well designed for the realities of child health and illness, nor the societal priorities regarding children that our study identified. This research generates new knowledge to inform decision-making on paediatric drugs by HTA institutions and government payers in Canada and other publicly-funded health systems, through insights into the relevant social values for child drug funding decisions from varied stakeholder groups.

Childhood cancer drugs in China: An overview and comparison of regulatory approvals in China and the United States.

Different from less developed countries, 80% of children with cancers in the United States are cured. Traditional chemotherapy drugs are the mainstay of therapies; new targeted medications have become available recently. Using publicly available data, we created a database of cancer drugs with paediatric malignancy indications approved by 31 October 2020 in China and the United States. We compared numbers, type, indications and listing on the World Health Organization Model List of Essential Medicines for Children (WHO EMLc) between the two countries, assessed the correlation between paediatric indications and cancer incidences, and described evidence supporting approvals of targeted medications in the two settings. Our study showed that by 31 October 2020, 31 and 39 cancer drugs available in China and the United States were approved for use in children, corresponding to 137 and 102 paediatric cancer indications, respectively. About half of these drugs (17 in China and 18 in the United States) were listed on the WHO EMLc. The correlation between indications and burden of disease was higher in the United States (r = 0.68) than China (r = 0.59). More traditional chemotherapy drugs were approved in China (n = 27) than the United States (n = 19). Of 20 targeted childhood anticancer medicines approved in the United States, mainly on the basis of single arm trials (27/32 indications, 84.4%), only four were approved for paediatric indications in China, at a median of 2.8 years after US Food and Drug Administration approval. A harmonised, evidence-based regulatory framework is needed to ensure approvals of needed, safe and efficacious childhood cancer drugs across the world.

Determinants of access to childhood cancer medicines: a comparative, mixed-methods analysis of four Caribbean countries.

BACKGROUND: Equitable access to essential medicines is a key facet of childhood cancer care, recognised by WHO as vital to improved childhood cancer outcomes globally. In the Caribbean, childhood cancer outcomes are poorer than those in most high-income countries. We aimed to generate in-depth comparative evidence of the current challenges and opportunities related to access to childhood cancer medicines in the Caribbean to identify context-sensitive health systems strategies to improve drug access and inform evidence-based paediatric cancer policies in the region. METHODS: In this convergent, parallel, mixed-methods study, we mapped and analysed the determinants of access to childhood cancer medicines in four Caribbean countries (The Bahamas, Barbados, Jamaica, and Trinidad and Tobago). We analysed contextual determinants of access to medicines within and across study site jurisdictions, alignment of childhood cancer medicine inclusion between each country's national essential medicines list (NEML) and WHO's 2017 Essential Medicines List for Children, and availability and cost of chemotherapeutic agents at five tertiary care hospitals. We used a mixed-effects logistic regression model to analyse the association of medicine price, procurement efficiency (via median price ratio [MPR]), and site with drug availability. The fixed effect evaluated the effect of site and MPR on the probability of stockout in a given month. We assessed determinants of medicine access via thematic analysis of semi-structured qualitative interviews, literature, and policy documents. FINDINGS: We collected and analysed data for 28 childhood cancer medicines from Barbados, 32 from The Bahamas, 30 from Trinidad and Tobago, and 31 from Jamaica. Despite stepwise inclusion of childhood cancer medicines in NEMLs, all four countries had frequent and recurrent stockouts for many cytotoxic medicines, showing no consistent relationship between NEML inclusion and availability. A mean MPR of greater than 3·0 in Trinidad and Tobago, The Bahamas, and Barbados suggests uniformly high procurement inefficiency, resulting in significant effects on drug stockout days. For each one unit increase in MPR the adjusted odds ratio (OR) of stockout increased by 10% (adjusted OR 1·10, 95% CI 1·04-1·16; p<0·01). These challenges in access to childhood cancer medicines stem from health system and policy dynamics at institutional, national, and supranational levels that cause price volatility and erratic medicine availability. Key challenges include disparate policy commitments (eg, among sites), inefficient procurement and supply chain management practices, and local effects of international market pressures. INTERPRETATION: The Caribbean region exemplifies deficiencies in access to childhood cancer medicines that might be overcome by improved regional harmonisation of drug registration, pharmacovigilance, and procurement alongside national forecasting to strengthen global pharmaceutical planning and prioritisation. Focused political attention to address these challenges is required to ensure efficient, reliable, and sustained availability of cancer mediciness. FUNDING: The SickKids-Caribbean Initiative.

Forecasting essential childhood cancer drug need: An innovative model-based approach.

BACKGROUND: Childhood cancer outcomes in low-income and middle-income countries have not kept pace with advances in care and survival in high-income countries. A contributing factor to this survival gap is unreliable access to essential drugs. METHODS: The authors created a tool (FORx ECAST) capable of predicting drug quantity and cost for 18 pediatric cancers. FORx ECAST enables users to estimate the quantity and cost of each drug based on local incidence, stage breakdown, treatment regimen, and price. Two country-specific examples are used to illustrate the capabilities of FORx ECAST to predict drug quantities. RESULTS: On the basis of domestic public-sector price data, the projected annual cost of drugs to treat childhood cancer cases is 0.8 million US dollars in Kenya and 3.0 million US dollars in China, with average median price ratios of 0.9 and 0.1, respectively, compared with costs sourced from the Management Sciences for Health (MSH) International Medical Products Price Guide. According to the cumulative chemotherapy cost, the most expensive disease to treat is acute lymphoblastic lymphoma in Kenya, but a higher relative unit cost of methotrexate makes osteosarcoma the most expensive diagnosis to treat in China. CONCLUSIONS: FORx ECAST enables needs-based estimates of childhood cancer drug volumes to inform health system planning in a wide range of contexts. It is broadly adaptable, allowing decision makers to generate results specific to their needs. The resultant estimates of drug need can help equip policymakers and health governance institutions with evidence-informed data to advance innovative procurement strategies that drive global improvements in childhood cancer drug access.

The Moral Foundations of Child Health and Social Policies: A Critical Interpretive Synthesis.

BACKGROUND: Allusions to the uniqueness and value of childhood abound in academic, lay, and policy discourse. However, little clarity exists on the values that guide child health and social policy-making. We review extant academic literature on the normative dimensions of child health and social policy to provide foundations for the development of child-focused public policies. METHODS: We conducted a critical interpretive synthesis of academic literature on the normative dimensions of child health and social policy-making. We employed a social constructivist lens to interpret emergent themes. Political theory on the social construction of target populations served as a bridge between sociologies of childhood and public policy analysis. RESULTS: Our database searches returned 14,658 unique articles; full text review yielded 72 relevant articles. Purposive sampling of relevant literature complemented our electronic searches, adding 51 original articles, for a total of 123 articles. Our analysis of the literature reveals three central themes: potential, rights, and risk. These themes retain relevance in diverse policy domains. A core set of foundational concepts also cuts across disciplines: well-being, participation, and best interests of the child inform debate on the moral and legal dimensions of a gamut of child social policies. Finally, a meta-theme of embedding encompasses the pervasive issue of a child's place, in the family and in society, which is at the heart of much social theory and applied analysis on children and childhood. CONCLUSIONS: Foundational understanding of the moral language and dominant policy frames applied to children can enrich analyses of social policies for children. Most societies paint children as potent, vulnerable, entitled, and embedded. It is the admixture of these elements in particular policy spheres, across distinct places and times, that often determines the form of a given policy and societal reactions to it. Subsequent work in this area will need to detail the degree and impact of variance in the values mix attached to children across sociocultural contexts and investigate tensions between what are and what ought to be the values that guide social policy development for children.

The cost-effectiveness of treating childhood cancer in 4 centers across sub-Saharan Africa.

BACKGROUND: The treatment of childhood cancer often is assumed to be costly in African settings, thereby limiting advocacy and policy efforts. The authors determined the cost and cost-effectiveness of maintaining childhood cancer centers across 4 hospitals throughout sub-Saharan Africa. METHODS: Within hospitals representing 4 countries (Kenya, Nigeria, Tanzania, and Zimbabwe), cost was determined either retrospectively or prospectively for all inputs related to operating a pediatric cancer unit (eg, laboratory costs, medications, and salaries). Cost-effectiveness was calculated based on the annual number of newly diagnosed patients, survival rates, and life expectancy. RESULTS: Cost per new diagnosis ranged from $2400 to $31,000, attributable to variances with regard to center size, case mix, drug prices, admission practices, and the treatment abandonment rate, which also affected survival. The most expensive cost input was found to be associated with medication in Kenya, and medical personnel in the other 3 centers. The cost per disability-adjusted life-year averted ranged from 0.3 to 3.6 times the per capita gross national income. Childhood cancer treatment therefore was considered to be very cost-effective by World Health Organization standards in 2 countries and cost-effective in 1 additional country. In all centers, abandonment of treatment was common; modeling exercises suggested that public funding of treatment, additional psychosocial personnel, and modifications of inpatient policies would increase survival rates while maintaining or even improving cost-effectiveness. CONCLUSIONS: Across various African countries, childhood cancer treatment units represent cost-effective interventions. Cost-effectiveness can be increased through the control of drug prices, appropriate policy environments, and decreasing the rate of treatment abandonment. These results will inform national childhood cancer strategies across Africa.

What are the volume and budget needs to provide chemotherapy to all children with acute lymphoblastic leukaemia in Thailand? Development and application of an estimation tool.

OBJECTIVE: Insufficient access to anticancer medicines may contribute to the wide survival differences of children with cancers across the globe. We developed a tool to estimate the volume of medicines and budget requirements to provide chemotherapy to children with acute lymphoblastic leukaemia (ALL). DESIGN: Development and application of an estimation tool. SETTING: Paediatric oncology hospital departments in Thailand. PARTICIPANTS: 318 children aged 0-14 years diagnosed with ALL and 215 children with undiagnosed ALL. INTERVENTIONS: Estimates of volume and budget requirements for administering a full course of chemotherapy for ALL and a further course for children who relapse, according to National Treatment Guidelines. PRIMARY AND SECONDARY OUTCOME MEASURES: Primary outcome measures were the volume (mg) and cost (US$) of medicines needed to treat children with ALL. For medicines whose main indication is paediatric ALL (asparaginase and 6-mercaptopurine), we estimated the difference between volume needed and actual sales in 2017 (secondary outcome). RESULTS: Ten anticancer medicines and four chemoprotective agents are needed for the treatment of paediatric ALL according to the Thai treatment guidelines. Of these 14 medicines, 13 are included in the WHO essential medicines list for children. All are available as generics. We estimated that essential chemotherapy and chemoprotective agents to treat all children diagnosed with ALL in Thailand in 2017 would cost US$ 814 952 (US$ 1 365 422 for diagnosed and undiagnosed children), which corresponds to 0.005% (0.008%) of the country's total health expenditure. The volumes of asparaginase and 6-mercaptopurine available on the Thai market in 2017 were more than sufficient (2.3 and 1.5 times the amounts needed, respectively) to treat all children diagnosed with ALL. CONCLUSIONS: Procuring sufficient quantities of essential medicines to treat children with ALL requires relatively modest resources. Medicine cost should not be a major barrier to ALL treatment in similar settings.

Interventions to improve early detection of childhood cancer in low- and middle-income countries: A systematic review.

BACKGROUND: Childhood cancer outcomes in low- and middle-income countries (LMICs) lag behind those in high-income countries (HICs), in part due to late presentation and diagnosis. Though several interventions targeting early detection of childhood cancer have been implemented in LMICs, little is known about their efficacy. METHODS: We conducted a systematic review to identify studies describing such interventions. We searched multiple databases from inception to December 4, 2019. Studies were included if they reported on LMIC interventions focused on: (a) training of health care providers on early recognition of childhood cancer, or (ii) public awareness campaigns. We used preferred reporting items for systematic reviews and meta-analyses (PRISMA) guidelines to conduct our review. The risk of bias in nonrandomized studies of interventions (ROBINS-I) checklist was used to assess quality of studies. RESULTS: Twelve studies met inclusion criteria (n = 5 full text, n = 7 abstract only). Five studies focused on retinoblastoma only, while the others focused on all types of childhood cancer. The majority studied multiple interventions of which early detection was one component, but reported overall outcomes. All identified studies used pre-post evaluative designs to measure efficacy. Five studies reported statistically significant results postintervention: decrease in extraocular spread of retinoblastoma, decrease in rates of refusal/abandonment of treatment, increase in number of new referrals, increase in knowledge, and an absolute increase in median 5-year survival. Other studies reported improvements without tests of statistical significance. Two studies reported no difference in survival postintervention. The ROBINS-I checklist indicated that all studies were at serious risk of bias. CONCLUSION: Though current evidence suggests that LMIC interventions targeting early detection of childhood cancer through health professional training and/or public awareness campaigns may be effective, this evidence is limited and of poor quality. Robust trials or quasi-experimental designs with long-term follow up are needed to identify the most effective interventions. Such studies will facilitate and inform the widespread uptake of early detection interventions across LMIC settings.

Does moral reasoning influence public values for health care priority setting?: A population-based randomized stated preference survey.

OBJECTIVE: Preferences of members of the public are recognized as important inputs into health care priority-setting, though knowledge of such preferences is scant. We sought to generate evidence of public preferences related to healthcare resource allocation among adults and children. METHODS: We conducted an experimental stated preference survey in a national sample of Canadian adults. Preferences were elicited across a range of scenarios and scored on a visual analogue scale. Intervention group participants were randomized to a moral reasoning exercise prior to each choice task. The main outcomes were the differences in mean preference scores by group, scenario, and demographics. RESULTS: Our results demonstrate a consistent preference by participants to allocate scarce health system resources to children. Exposure to the moral reasoning exercise weakened but did not eliminate this preference. Younger respondent age and parenthood were associated with greater preference for children. The top principles guiding participants' allocative decisions were treat equally, relieve suffering, and rescue those at risk of dying. CONCLUSIONS: Our study affirms the relevance of age in public preferences for the allocation of scarce health care resources, demonstrating a significant preference by participants to allocate healthcare resources to children. However, this preference diminishes when challenged by exposure to a range of moral principles, revealing a strong public endorsement of equality of access. Definitions of value in healthcare based on clinical benefit and cost-effectiveness may exclude moral considerations that the public values, such as equality and humanitarianism, highlighting opportunities to enrich healthcare priority-setting through public engagement.

'The problem is small enough, the problem is big enough': a qualitative study of health technology assessment and public policy on drug funding decisions for children.

BACKGROUND: Public policy approaches to funding paediatric medicines in developed public health systems remain understudied. Current approaches to HTA present a variety of conceptual, methodological and practical problems in the context of child health. This study explores the technical and sociopolitical determinants of public funding decisions on paediatric drugs, through the analysis of interviews with stakeholders involved in or impacted by HTA for child health technologies at the provincial and national levels in Canada. METHODS: We undertook in-depth interviews with a purposive sample (n = 22) of stakeholders involved with or affected by drug funding decisions for children at the provincial (Ontario) and national levels in Canada. Grounded theory methods were employed to guide data collection and analysis. Theory on 'technology-as-policy' and the sociopolitics of health technologies served as sensitizing concepts for inductive data coding and analysis. Emergent themes informed the development of conceptual and practical insights on social values and system dynamics related to child HTA, of relevance to public policymaking on the coverage of health technologies for children in Canada. RESULTS: Participant reflection on the normative and systems dimensions of drug funding for children formed two broad categories: HTA paradigms and sociopolitical context. Our analysis revealed notable differences of context and substance related to child health technology production, evaluation and use. These differences spanned the major phases of HTA (from assembly to assessment to integration) and the surrounding sociopolitical milieu (from markets to governance to politics). Careful analysis of these differences sets in relief a number of substantive and procedural shortcomings of current HTA paradigms in respect of child health. Our findings suggest a need to rethink how HTA is structured and operationalized for child health technologies. CONCLUSIONS: Current approaches to health technology assessment are not well calibrated to the realities of child health and illness. Our study presents a nuanced and contextually grounded analysis of concepts instrumental to drug funding decisions for children. The insights generated are directly applicable to the Canadian and Ontario contexts, but also yield fundamental knowledge about HTA for children that are germane to drug policy in other health systems.

Political priority and pathways to scale-up of childhood cancer care in five nations.

BACKGROUND: Despite increasing global attention to non-communicable diseases (NCDs) and their incorporation into universal health coverage (UHC), the factors that determine whether and how NCDs are prioritized in national health agendas and integrated into health systems remain poorly understood. Childhood cancer is a leading non-communicable cause of death in children aged 0-14 years worldwide. We investigated the political, social, and economic factors that influence health system priority-setting on childhood cancer care in a range of low- and middle-income countries (LMIC). METHODS AND FINDINGS: Based on in-depth qualitative case studies, we analyzed the determinants of priority-setting for childhood cancer care in El Salvador, Guatemala, Ghana, India, and the Philippines using a conceptual framework that considers four principal influences on political prioritization: political contexts, actor power, ideas, and issue characteristics. Data for the analysis derived from in-depth interviews (n = 68) with key informants involved in or impacted by childhood cancer policies and programs in participating countries, supplemented by published academic literature and available policy documents. Political priority for childhood cancer varies widely across the countries studied and is most influenced by political context and actor power dynamics. Ghana has placed relatively little national priority on childhood cancer, largely due to competing priorities and a lack of cohesion among stakeholders. In both El Salvador and Guatemala, actor power has played a central role in generating national priority for childhood cancer, where well-organized and -resourced civil society organizations have disrupted legacies of fragmented governance and financing to create priority for childhood cancer care. In India, the role of a uniquely empowered private actor was instrumental in creating political priority and establishing sustained channels of financing for childhood cancer care. In the Philippines, the childhood cancer community has capitalized on a window of opportunity to expand access and reduce disparities in childhood cancer care through the political prioritization of UHC and NCDs in current health system reforms. CONCLUSIONS: The importance of key health system actors in determining the relative political priority for childhood cancer in the countries studied points to actor power as a critical enabler of prioritization in other LMIC. Responsiveness to political contexts-in particular, rhetorical and policy priority placed on NCDs and UHC-will be crucial to efforts to place childhood cancer firmly on national health agendas. National governments must be convinced of the potential for foundational health system strengthening through attention to childhood cancer care, and the presence and capability of networked actors primed to amplify public sector investments and catalyze change on the ground.

The cost effectiveness of treating Burkitt lymphoma in Uganda.

BACKGROUND: Perceptions of high cost and resource intensity remain political barriers to the prioritization of childhood cancer treatment programs in many low- and middle-income countries (LMICs). Little knowledge exists of the actual cost and cost-effectiveness of such programs. To improve outcomes for children with Burkitt lymphoma (BL), the most common childhood cancer in Africa, the Uganda Cancer Institute implemented a comprehensive BL treatment program in 2012. We undertook an economic evaluation of the program to ascertain the cost-effectiveness of BL therapy in a specific LIC setting. METHODS: We compared the treatment of BL to usual care in a cohort of 122 patients treated between 2012 and 2014. Costs included variable, fixed, and family costs. Our primary measure of effectiveness was overall survival (OS). Patient outcomes were determined through prospective capture and retrospective chart abstraction. The cost per disability-adjusted life-year (DALY) averted was calculated using the World Health Organization's Choosing Interventions That Are Cost-Effective (WHO-CHOICE) methodology. RESULTS: The 2-year OS with treatment was 55% (95% CI, 45% to 64%). The cost per DALY averted in the treatment group was US$97 (Int$301). Cumulative estimate of national DALYs averted through treatment was 8607 years, and the total national annual cost of treatment was US$834,879 (Int$2,590,845). The cost of BL treatment fell well within WHO-CHOICE cost-effectiveness thresholds. The ratio of cost per DALY averted to per capita gross domestic product was 0.14, reflecting a very cost-effective intervention. CONCLUSION: This study demonstrates that treating BL with locally tailored protocols is very cost-effective by international standards. Studies of this kind will furnish crucial evidence to help policymakers prioritize the allocation of LMIC health system resources among noncommunicable diseases, including childhood cancer.