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.

Strategies to accelerate the elimination of cervical cancer in British Columbia, Canada: a modelling study.

BACKGROUND: To eliminate cervical cancer in Canada by 2040, defined as an annual age-standardized incidence rate (ASIR) lower than 4.0 per 100 000 women, the Canadian Partnership Against Cancer (CPAC) identified 3 priorities for action: increasing human papillomavirus (HPV) vaccine coverage, implementing HPV-based screening and increasing screening participation, and improving follow-up after abnormal screen results. Our objective was to explore the impact of these priorities on the projected time to elimination of cervical cancer in British Columbia. METHODS: We used OncoSim-Cervical, a microsimulation model led and supported by CPAC and developed by Statistics Canada that simulates HPV transmission and the natural history of cervical cancer for the Canadian population. We updated model parameters to reflect BC's historical participation rates and program design. We simulated the transition to HPV-based screening and developed scenarios to explore the additional impact of achieving 90% vaccination coverage, 95% screening recruitment, 90% ontime screening, and 95% follow-up compliance. We projected cervical cancer incidence, ASIR, and year of elimination for the population of BC for 2023-2050. RESULTS: HPV-based screening at current vaccination, participation, and follow-up rates can eliminate cervical cancer by 2034. Increasing on-time screening and follow-up compliance could achieve this target by 2031. Increasing vaccination coverage has a small impact over this time horizon. INTERPRETATION: With the implementation of HPV-based screening, cervical cancer can be eliminated in BC before 2040. Efforts to increase screening participation and follow-up through this transition could potentially accelerate this timeline, but the transition from cytology- to HPV-based screening is fundamental to achieving this goal.

From Treatment to Recovery: Gynecological Survivors' and Caregivers' Perspectives About the Usability of an Educational Resource.

The objective of this study was to understand gynecological cancer (GC) survivors' and their informal caregivers' perceptions about the usability of an educational resource to support their transition from primary cancer treatment into surveillance and/or recovery. After developing an empirical- and experiential-informed educational resource, we used a semi-structured questioning process to understand GC survivors and their caregivers' perceptions about its usability. Data were collected via online focus groups or 1:1 interviews that were audio recorded and transcribed. We used thematic analysis to analyze the data. Ten participants who were survivors or informal caregivers of cervical, ovarian, or uterine/endometrial cancer participated in two rounds of data collection. We grouped qualitative data into two themes: (1) reputable, relevant, and accessible education reduces uncertainty and promotes connection, and (2) individualized delivery of education provided by trusted cancer clinicians. The transition from treatment to surveillance is a challenging time for which reputable, relevant, and accessible educational resources are useful to facilitate an understanding about and self-management of survivorship-related concerns. Survivors and caregivers look to clinicians to provide reputable education to address their needs. This education should be diverse in content and referred to repeatedly throughout the cancer trajectory.

Human papillomavirus-based screening at extended intervals missed fewer cervical precancers than cytology in the HPV For Cervical Cancer (HPV FOCAL) trial.

While cervix screening using cytology is recommended at 2- to 3-year intervals, given the increased sensitivity of human papillomavirus (HPV)-based screening to detect precancer, HPV-based screening is recommended every 4- to 5-years. As organized cervix screening programs transition from cytology to HPV-based screening with extended intervals, there is some concern that cancers will be missed between screens. Participants in HPV FOr CervicAL Cancer (HPV FOCAL) trial received cytology (Cytology Arm) at 24-month intervals or HPV-based screening (HPV Arm) at 48-month intervals; both arms received co-testing (cytology and HPV testing) at exit. We investigated the results of the co-test to identify participants with cervical intraepithelial neoplasia grade 2 or higher (CIN2+) who would not have had their precancer detected if they had only their arm's respective primary screen. In the Cytology Arm, 25/62 (40.3%) identified CIN2+s were missed by primary screen (ie, normal cytology/positive HPV test) and all 25 had normal cytology at the prior 24-month screen. In the HPV arm, three CIN2+s (3/49, 6.1%) were missed by primary screen (ie, negative HPV test/abnormal cytology). One of these three misses had low-grade cytology findings and would also not have been referred to colposcopy outside of the trial. Multiple rounds of cytology did not detect some precancerous lesions detected with one round of HPV-based screening. In our population, cytology missed more CIN2+, even at shorter screening intervals, than HPV-based screening. This assuages concerns about missed detection postimplementation of an extended interval HPV-based screening program. We recommend that policymakers consider a shift from cytology to HPV-based cervix screening.

Impact of TAILORx on chemotherapy prescribing and 21-gene recurrence score-guided treatment costs in a population-based cohort of patients with breast cancer.

BACKGROUND: The trial assigning individualized options for treatment (Rx) (TAILORx) confirmed the predictive value of the 21-gene recurrence score (RS) assay in hormone receptor (HR)-positive, HER2-negative, node-negative breast cancer and established thresholds for chemotherapy benefit in younger and older patients. Real-world chemotherapy use and RS-guided treatment costs in British Columbia post-TAILORx were examined. METHODS: The authors assembled 3 cohorts of HR-positive, HER2-negative, node-negative patients with breast cancer defined by diagnosis: before RS funding (cohort 1 [C1]: January 2013-December 2013), after introduction of public RS funding (cohort 2 [C2]: July 2015-June 2016), and after TAILORx results (cohort 3 [C3]: July 2018-June 2019). Chemotherapy use was compared between cohorts by age and RS. Budgetary impacts of RS testing on chemotherapy costs were evaluated pre- and post-TAILORx. RESULTS: Among the 2066 patients included, chemotherapy use declined by 19% after RS funding was introduced and by an additional 23% after TAILORx publication (P = .001). Reduction in chemotherapy use was significant for RS 11-20 tumors (C3 vs C2, P = .004). There was no significant change in chemotherapy use in patients >50 years old (C2:12% vs C3:10%, P = .22). RS testing was associated with higher cost savings post-TAILORx, except in patients 70 to 80 years old, where testing led to excess costs when adjusting for the low rate of RS-concordant chemotherapy prescribed. CONCLUSIONS: TAILORx has had population-based impacts on chemotherapy prescribing in intermediate RS tumors and patients ≤50 years old. The lower clinical use of RS and increased spending in patients 70-80 years old highlights the importance of careful selection of older candidates for high-cost genomic testing. LAY SUMMARY: The 21-gene recurrence score (RS) test helps predict whether patients with hormone-positive, HER2-negative, lymph node-negative breast cancer are likely to benefit from chemotherapy. The recent trial assigning individualized options for treatment (Rx) (TAILORx) found that patients with intermediate RS tumors did not benefit from chemotherapy. The authors assessed whether TAILORx results translated to real-world changes in chemotherapy prescribing patterns. In this study, chemotherapy use decreased by 23% after TAILORx, with the greatest reductions seen among intermediate RS tumors and younger patients. In contrast, RS testing had lower clinical value and increased treatment costs in elderly patients, which requires further study to ensure optimal care for this age group.

"Bring the Hoses to Where the Fire Is!": Differential Impacts of Marginalization and Socioeconomic Status on COVID-19 Case Counts and Healthcare Costs.

OBJECTIVES: Local health leaders and the Director General of the World Health Organization alike have observed that COVID-19 "does not discriminate." Nevertheless, the disproportionate representation of people of low socioeconomic status among those infected resembles discrimination. This population-based retrospective cohort study examined COVID-19 case counts and publicly funded healthcare costs in Ontario, Canada, with a focus on marginalization. METHODS: Individuals with their first positive severe acute respiratory syndrome coronavirus 2 test from January 1, 2020 to June 30, 2020, were linked to administrative databases and matched to negative/untested controls. Mean net (COVID-19-attributable) costs were estimated for 30 days before and after diagnosis, and differences among strata of age, sex, comorbidity, and measures of marginalization were assessed using analysis of variance tests. RESULTS: We included 28 893 COVID-19 cases (mean age 54 years, 56% female). Most cases remained in the community (20 545, 71.1%) or in long-term care facilities (4478, 15.5%), whereas 944 (3.3%) and 2926 (10.1%) were hospitalized, with and without intensive care unit, respectively. Case counts were skewed across marginalization strata with 2 to 7 times more cases in neighborhoods with low income, high material deprivation, and highest ethnic concentration. Mean net costs after diagnosis were higher for males ($4752 vs $2520 for females) and for cases with higher comorbidity ($1394-$7751) (both P < .001) but were similar across levels of most marginalization dimensions (range $3232-$3737, all P ≥ .19). CONCLUSIONS: This study suggests that allocating resources unequally to marginalized individuals may improve equality in outcomes. It highlights the importance of reducing risk of COVID-19 infection among marginalized individuals to reduce overall costs and increase system capacity.

The FACT-8D, a new cancer-specific utility algorithm based on the Functional Assessment of Cancer Therapies-General (FACT-G): a Canadian valuation study.

INTRODUCTION: Utility instruments are used to assess patients' health-related quality of life for cost-utility analysis (CUA). However, for cancer patients, the dimensions of generic utility instruments may not capture all the information relevant to the impact of cancer. Cancer-specific utilities provide a useful alternative. Under the auspices of the Multi-Attribute Utility in Cancer Consortium, a cancer-specific utility algorithm was derived from the FACT-G. The new FACT-8D contains eight dimensions: pain, fatigue, nausea, sleep, work, support from family/friends, sadness, and worry health will get worse. The aim of the study was to obtain a Canadian value set for the FACT-8D. METHODS: A discrete choice experiment was administered to a Canadian general population online panel, quota sampled by age, sex, and province/territory of residence. Respondents provided responses to 16 choice sets. Each choice set consisted of two health states described by the FACT-8D dimensions plus an attribute representing survival duration. Sample weights were applied and the responses were analyzed using conditional logistic regression, parameterized to fit the quality-adjusted life year framework. The results were converted into utility weights by evaluating the marginal rate of substitution between each level of each FACT-8D dimension with respect to duration. RESULTS: 2228 individuals were recruited. The analysis dataset included n = 1582 individuals, who completed at least one choice set; of which, n = 1501 completed all choice sets. After constraining to ensure monotonicity in the utility function, the largest decrements were for the highest levels of pain (- 0.38), nausea (- 0.30), and problems doing work (- 0.23). The decrements of the remaining dimensions ranged from - 0.08 to - 0.18 for their highest levels. The utility of the worst possible health state was defined as - 0.65, considerably worse than dead. CONCLUSIONS: The largest impacts on utility included three generic dimensions (i.e., pain, support, and work) and nausea, a symptom caused by cancer (e.g., brain tumours, gastrointestinal tumours, malignant bowel obstruction) and by common treatments (e.g., chemotherapy, radiotherapy, opioid analgesics). This may make the FACT-8D more informative for CUA evaluating in many cancer contexts, an assertion that must now be tested empirically in head-to-head comparisons with generic utility measures.

Vaccine nationalism will persist: global public goods need effective engagement of global citizens.

Covid-19 presents a unique opportunity to transform democratic engagement in the governance of global public goods. In this paper, I describe a global public goods framework and how it relates to Covid-19 vaccines, and summarize some of the global responses to Covid-19. I discuss some of the global threats to health and prosperity posed by the inequitable distribution of vaccines, and propose transformative thinking to democratically engage citizens in the governance of global public goods. In recent years, public-private partnerships and philanthropic organizations have successfully stepped in to help international organizations like the UN and WHO provide global public goods, but they are not democratically elected or publicly accountable. Global public goods are critical to addressing Covid-19, future pandemic preparedness, global health policy, health equity, and the unfolding climate crisis. To make us more resistant and resilient to future global health crises we need transformative thinking to democratically engage global citizens. We need to lay the foundations for a 'global social contract' on global public goods.

Direct medical costs of young-onset colorectal cancer: a worldwide systematic review.

BACKGROUND: Given the rising incidence of young-onset colorectal cancer (yCRC) among individuals younger than 50 years old, understanding the economic burden of yCRC is required to inform the delivery of healthcare services. Therefore, we conducted a systematic review of studies assessing the direct medical costs of yCRC, and where relevant average-age onset CRC (aCRC). METHODS: We searched MEDLINE, EMBASE, and Web of Science from inception to May 2022 for original, peer-reviewed studies, that reported direct medical costs (e.g., chemotherapy, radiotherapy, outpatient visits, inpatient care, prescription medications) for yCRC and aCRC. We used a modified version of the Consolidated Health Economic Evaluation Reporting Standards checklist to appraise the studies. Costs were inflation-adjusted to 2020 US dollars. RESULTS: We included 14 studies from 10 countries, including the USA, England, France, Korea, Vietnam, China, Italy, Australia, Canada and Japan. Five studies focused on prevalent disease and reported annualized per-capita cost of prevalent yCRC, ranging from $2,263 to $16,801 and $1,412 to $14,997 among yCRC and aCRC cases, respectively. Nine studies estimated the cost of incident disease. Synthesis of per-capita costs incurred 12 months following colorectal cancer diagnosis ranged from $23,368 to $89,945 for yCRC and $19,929 to $67,195 for aCRC. Five studies used multivariable approaches to compare costs associated with yCRC and aCRC, four showed no differences and one suggested greater costs with yCRC. CONCLUSION: Our synthesis of direct medical costs of yCRC across multiple jurisdictions provide relevant information for healthcare decisions, including on-going considerations for expanding CRC screening strategies to younger adults.

Trajectory of psychosocial symptoms among home care patients with cancer at end-of-life.

PURPOSE: Understanding the end-of-life psychosocial needs of cancer patients at home is a knowledge gap. This study describes the trajectory of psychosocial symptoms in the last 6 months of life among cancer decedents who were receiving home care. METHODS: Observational population-based cohort study of cancer decedents who were receiving home care services between 2007 and 2014. Decedents had to have at least one home care assessment in the last 6 months of life for inclusion. Outcomes were the presence of psychosocial symptoms (i.e., anxiety, loneliness, depression, social decline, caregiver distress, and cognitive decline) at each week before death. RESULTS: Our cohort included 27,295 unique cancer decedents (30,368 assessments), of which 58% died in hospital. Fifty-six percent were older than 74, and 47% were female. The prevalence of all symptoms increased approaching death, except loneliness. Social decline (48%-78%) was the most prevalent psychosocial symptom, though loneliness was reported in less than 10% of the cohort. Caregiver distress rose over time from 15%-27%. A third of the cohort reported issues with cognitive impairment. Multivariate regression showed that physical symptoms such as uncontrolled pain, impairment in independent activities of daily living, and a high level of health instability all significantly worsened the odds of having a psychosocial symptom in the last 3 months of life. CONCLUSION: In this large home care cancer cohort, trajectories of psychosocial symptoms worsened close to death. Physical symptoms, such as uncontrolled pain, were associated with having worse psychosocial symptoms at end of life.