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.
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.
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.
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.
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.
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.
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.
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.
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 (FORxECAST) capable of predicting drug quantity and cost for 18 pediatric cancers. FORxECAST 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 FORxECAST 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
FORxECAST 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.
A pediatric‐specific tool (FORxECAST) was created that estimates drug quantity and cost for 18 cancers. The results enable evidence‐based forecasting of childhood cancer drug quantity and cost to inform health system planning in a wide range of countries.
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.
What's new?
This comprehensive review of the childhood cancer drugs and their paediatric indications approved in China and the United States suggests that key therapeutics for the treatment of children with cancers are approved in China. But more traditional chemotherapy drugs are approved in China than in the United States, and only a few of the targeted childhood anticancer medications approved in the United States, mainly on the basis of single‐arm clinical trials, are also approved in China. A harmonised, evidence‐based regulatory framework is needed to ensure the development and approval of needed, safe, and efficacious childhood cancer drugs across the world.
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.
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.
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.
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.
American Childhood Cancer Organization, Childhood Cancer International, and the Friends of Cancer Patients Ameera Fund.
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.
Childhood cancer treatment is assumed to be too costly for low‐income and middle‐income country settings. Across 4 sub‐Saharan African countries, the authors demonstrate that childhood cancer treatment units can represent very cost‐effective interventions.
IntroductionA major barrier to improving childhood cancer survival is the perception that paediatric oncology services are too costly for low-income and middle-income country (LMIC) health systems. ...We conducted a systematic review to synthesise existing evidence on the costs and cost-effectiveness of treating childhood cancers in LMICs.MethodsWe searched multiple databases from their inception to March 2019. All studies reporting costs or cost-effectiveness of treating any childhood cancer in an LMIC were included. We appraised included articles using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist. Where possible, we extracted or calculated the cost per disability-adjusted life year (DALY) averted using reported survival and country-specific life expectancy. Cost/DALY averted was compared with per capita gross domestic product (GDP) as per WHO-Choosing Interventions that are Cost-Effective guidelines to determine cost-effectiveness.ResultsOf 2802 studies identified, 30 met inclusion criteria. Studies represented 22 countries and nine different malignancies. The most commonly studied cancers were acute lymphoblastic leukaemia (n=10), Burkitt lymphoma (n=4) and Wilms tumour (n=3). The median CHEERS checklist score was 18 of 24. Many studies omitted key cost inputs. Notably, only 11 studies included healthcare worker salaries. Cost/DALY averted was extracted or calculated for 12 studies and ranged from US$22 to US$4475, although the lower-end costs were primarily from studies that omitted key cost components. In all 12, cost/DALY averted through treatment was substantially less than country per capita GDP, and therefore considered very cost-effective.ConclusionMany included studies did not account for key cost inputs, thus underestimating true treatment costs. Costs/DALY averted were nonetheless substantially lower than per capita GDP, suggesting that even if all relevant inputs are included, LMIC childhood cancer treatment is consistently very cost-effective. While additional rigorous economic evaluations are required, our results can inform the development of LMIC national childhood cancer strategies.
There are multiple barriers impeding access to childhood cancer care in the Indian health system. Understanding what the barriers are, how various stakeholders perceive these barriers and what ...influences their perceptions are essential in improving access to care, thereby contributing towards achieving Universal Health Coverage (UHC). This study aims to explore the challenges for accessing childhood cancer care through health care provider perspectives in India.
This study was conducted in 7 tertiary cancer hospitals (3 public, 3 private and 1 charitable trust hospital) across Delhi and Hyderabad. We recruited 27 healthcare providers involved in childhood cancer care. Semi-structured interviews were audio recorded after obtaining informed consent. A thematic and inductive approach to content analysis was conducted and organised using NVivo 11 software.
Participants described a constellation of interconnected barriers to accessing care such as insufficient infrastructure and supportive care, patient knowledge and awareness, sociocultural beliefs, and weak referral pathways. However, these barriers were reflected upon differently based on participant perception through three key influences: 1) the type of hospital setting: public hospitals constituted more barriers such as patient navigation issues and inadequate health workforce, whereas charitable trust and private hospitals were better equipped to provide services. 2) the participant's cadre: the nature of the participant's role meant a different degree of exposure to the challenges families faced, where for example, social workers provided more in-depth accounts of barriers from their day-to-day interactions with families, compared to oncologists. 3) individual perceptions within cadres: regardless of the hospital setting or cadre, participants expressed individual varied opinions of barriers such as acceptance of delay and recognition of stakeholder accountabilities, where governance was a major issue. These influences alluded to not only tangible and structural barriers but also intangible barriers which are part of service provision and stakeholder relationships.
Although participants acknowledged that accessing childhood cancer care in India is limited by several barriers, perceptions of these barriers varied. Our findings illustrate that health care provider perceptions are shaped by their experiences, interests and standpoints, which are useful towards informing policy for childhood cancers within UHC.
Celotno besedilo
Dostopno za:
DOBA, IZUM, KILJ, NUK, PILJ, PNG, SAZU, SIK, UILJ, UKNU, UL, UM, UPUK
Congenital spinal hamartomas are rare benign tumors. They are mostly seen in infants and are typically asymptomatic at presentation. Spinal hamartomas have not been associated with any known cancer ...predisposition syndrome. DICER1 syndrome is a well-characterized cancer predisposition syndrome caused by a germline mutation in the
DICER1
gene, which shows variable expressivity. To our knowledge, spinal hamartoma has never been described in individuals with DICER1 syndrome. Here, we describe a rare association of congenital spinal hamartoma and DICER1 syndrome in a 5-week-old infant, with molecular findings suggestive of the implication of
DICER1
in the pathogenesis of this tumor.
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