Economic evaluation of prostate cancer risk assessment methods: A cost-effectiveness analysis using population data.

BACKGROUND: The current prostate cancer (PCa) screening standard of care (SOC) leads to unnecessary biopsies and overtreatment because decisions are guided by prostate-specific antigen (PSA) levels, which have low specificity in the gray zone (3-10 ng/mL). New risk assessment tools (RATs) aim to improve biopsy decision-making. We constructed a modeling framework to assess new RATs in men with gray zone PSA from the British Columbia healthcare system's perspective. METHODS: We evaluated the cost-effectiveness of a new RAT used in biopsy-naïve men aged 50+ with a PSA of 3-10 ng/mL using a time-dependent state-transition model. The model was informed by engaging patient partners and using linked administrative health data, supplemented with published literature. The incremental cost-effectiveness ratio and the probability of the RAT being cost-effective were calculated. Probabilistic analysis was used to assess parameter uncertainty. RESULTS: In the base case, a RAT based on an existing biomarker's characteristics was a dominant strategy associated with a cost savings of $44 and a quality-adjusted life years (QALY) gain of 0.00253 over 18 years of follow-up. At a cost-effectiveness threshold of $50,000/QALY, the probability that using a RAT is cost-effective relative to the SOC was 73%. Outcomes were sensitive to RAT costs and accuracy, especially the detection rate of high-grade PCa. Results were also impacted by PCa prevalence and assumptions about undetected PCa survival. CONCLUSIONS: Our findings showed that a more accurate RAT to guide biopsy can be cost-effective. Our proposed general model can be used to analyze the cost-effectiveness of any novel RAT.

Health Care Costs Attributable to Prostate Cancer in British Columbia, Canada: A Population-Based Cohort Study.

We aimed to estimate the total health care costs attributable to prostate cancer (PCa) during care phases by age, cancer stage, tumor grade, and primary treatment in the first year in British Columbia (BC), Canada. Using linked administrative health data, we followed a cohort of men aged ≥ 50 years at diagnosis with PCa between 2010 and 2017 (Cohort 1) from the diagnosis date until the date of death, the last date of observation, or 31 December 2019. Patients who died from PCa after 1 January 2010, were selected for Cohort 2. PCa attributable costs were estimated by comparing costs in patients to matched controls. Cohort 1 (n = 22,672) had a mean age of 69.9 years (SD = 8.9) and a median follow-up time of 5.2 years. Cohort 2 included 6942 patients. Mean PCa attributable costs were the highest during the first year after diagnosis ($14,307.9 [95% CI: $13,970.0, $14,645.8]) and the year before death ($9959.7 [$8738.8, $11,181.0]). Primary treatment with radiation therapy had significantly higher costs each year after diagnosis than a radical prostatectomy or other surgeries in advanced-stage PCa. Androgen deprivation therapy (and/or chemotherapy) had the highest cost for high-grade and early-stage cancer during the three years after diagnosis. No treatment group had the lowest cost. Updated cost estimates could inform economic evaluations and decision-making.

Understanding COVID-19 Vaccine Confidence in People Living with HIV: A pan-Canadian Survey.

Understanding the roots of Covid-19 vaccine hesitancy in at-risk groups, such as persons living with HIV (PLWH), is of utmost importance. We developed a modified Vaccine Hesitancy Scale (VHS) questionnaire using items from the National Advisory Committee on Immunization Acceptability Matrix. To examine factors associated with receiving COVID-19 vaccine and the link between vaccine attitudes and beliefs with vaccine behavior, PLWH were recruited via social media and community-based organizations (February-May 2022). Descriptive statistics were used to summarize results. Total VHS score was generated by adding Likert scale scores and linear regression models used to compare results between participants who received or did not receive COVID-19 vaccines. Logistic regression models were used to identify factors associated with vaccine uptake. A total of 246 PLWH indicated whether they received a COVID-19 vaccine. 89% received ≥ 1 dose. Mean total VHS(SD) for persons having received ≥ 1 COVID-19 vaccine was 17.8(6.2) vs. 35.4(9.4) for participants not having received any COVID-19 vaccine. Persons who received ≥ 1 dose were significantly older than those who had not received any (48.4 ± 13.8 vs. 34.0 ± 7.7 years, p < 0.0001). The majority of participants considered COVID-19 vaccination important for their health(81.3%) and the health of others(84.4%). Multivariate logistic regression revealed the odds of taking ≥ 1dose were increased 2.4-fold [95% CI 1.6, 3.5] with each increase in age of 10 years (p < 0.0001). Sex and ethnicity were not different between groups. In conclusion, PLWH accept COVID-19 vaccines for both altruistic and individual reasons. With evolving recommendations and increasing numbers of booster vaccines, we must re-examine the needs of PLWH regularly.

The economics of precision health: preventing air pollution-induced exacerbation in asthma.

The demonstrable value of precision medicine, in the context of common environmental exposures, has scarcely been explored. This study evaluated the cost effectiveness of a preventive personalised intervention to reduce the adverse effect of air pollution in the context of asthma. A decision-analytic model was used to conduct a cost-utility analysis of prevention interventions in case of acute exposure to air pollution in mild asthma. Three different strategies, as follows, were compared: no preventive intervention; precision health strategy based on information from genotype testing, followed with treating high-risk patients; and prescribing additional medication to all mild asthmatics as a preventive intervention. The costs and quality-adjusted life years (QALYs) in the base case and alternative scenarios were obtained through probabilistic analysis. The results showed that the precision prevention intervention (anticipatory intervention for asthmatics, guided by relevant genetic abnormality, in the face of acute air pollution) is a cost-effective strategy compared with no such intervention, with an incremental cost-effectiveness ratio of CAD 49 555 per QALY. Furthermore, this strategy is a dominant strategy compared with an intervention that prescribes medication indiscriminately to all asthmatics. The incorporation of genomic testing to stratify risk of asthmatics to pollution-driven exacerbations, and then tailoring a preventive intervention accordingly, may be cost effective relative to untailored methods. These results lend plausibility to the use of precision medicine for limiting asthma exacerbation in the context of air pollution and, potentially, other exposures.

Health-care practitioners' preferences for the return of secondary findings from next-generation sequencing: a discrete choice experiment.

PURPOSE: Health-care practitioners' (HCPs) preferences for returning secondary findings (SFs) will influence guideline compliance, shared decision-making, and patient health outcomes. This study aimed to estimate HCPs' preferences and willingness to support the return (WTSR) of SFs in Canada. METHODS: A discrete choice experiment estimated HCPs' preferences for the following attributes: disease risk, clinical utility, health consequences, prior experience, and patient preference. We analyzed responses with an error component mixed logit model and predicted WTSR using scenario analyses. RESULTS: Two hundred fifty participants of 583 completed the questionnaire (completion rate: 42.9%). WTSR was significantly influenced by patient preference and SF outcome characteristics. HCPs' WTSR was 78% (95% confidence interval: 74-81%) when returning SFs with available medical treatment, high penetrance, severe health consequences, and patient's preference for return. Genetics professionals had a higher WTSR than HCPs of other types when returning SFs with clinical utility and patient preference to know. HCPs >55 years of age were more likely to return SFs compared with younger HCPs. CONCLUSION: This study identified factors that influence WTSR of SFs and indicates that HCPs make tradeoffs between patient preference and other outcome characteristics. The results can inform clinical scenarios and models aiming to understand shared decision-making, patient and family opportunity to benefit, and cost-effectiveness.

A Hierarchical Bayes Approach to Modeling Heterogeneity in Discrete Choice Experiments: An Application to Public Preferences for Prenatal Screening.

BACKGROUND: Previous studies assessing preferences for prenatal screening have focused on preferences of the affected population and have largely assumed homogeneous preferences. We aimed to estimate public preferences and willingness to pay for prenatal screening and diagnosis from a Canadian general population sample, and to model preferences at the individual level. METHODS: A discrete choice experiment was used to elicit preferences for different aspects of prenatal screening and diagnostic strategies. Strategies differed in five attributes: timing of the results, false-negative rate, false-positive rate, risk of miscarriage, and out-of-pocket cost. Respondents made forced and unforced choices using a dual response approach. Hierarchical Bayes analysis was applied to estimate individual-level part-worth utilities. Individual probability and expected uptake of prenatal screening under different scenarios were also assessed. Subgroup analyses were conducted using individual-level preferences. RESULTS: The final analyses were based on a sample of 4601 respondents. Results showed that the two most important attributes were false-negative rate and miscarriage risk. There was significant heterogeneity in preferences among respondents. Individuals' perception of the risk of pregnancy with chromosomal abnormalities affected their preferences for screening. The relatively high uptake of safe and accurate screening among all groups of respondents indicated people's desire for information about the health of their unborn baby regardless of their decision to continue the pregnancy. CONCLUSION: Our findings are consistent with previous studies based on affected-population preferences. This concordance should be reassuring from a policy perspective and can inform the design of publicly funded prenatal screening programs.

Cost-effectiveness of prenatal screening and diagnostic strategies for Down syndrome: A microsimulation modeling analysis.

OBJECTIVES: Down syndrome (DS) is the most frequently occurring fetal chromosomal abnormality and different prenatal screening strategies are used for determining risk of DS worldwide. New non-invasive prenatal testing (NIPT), which uses cell-free fetal DNA in maternal blood can provide benefits due to its higher sensitivity and specificity in comparison to conventional screening tests. This study aimed to assess the cost-effectiveness of using population-level NIPT in fetal aneuploidy screening for DS. METHODS: We developed a microsimulation decision-analytic model to perform a probabilistic cost-effectiveness analysis (CEA) of prenatal screening and diagnostic strategies for DS. The model followed individual simulated pregnant women through the pregnancy pathway. The comparators were serum-only screening, contingent NIPT (i.e., NIPT as a second-tier screening test) and universal NIPT (i.e., NIPT as a first-tier screening test). To address uncertainty around the model parameters, the expected values of costs and quality-adjusted life-years (QALYs) in the base case and all scenario analyses were obtained through probabilistic analysis from a Monte Carlo simulation. RESULTS: Base case and scenario analyses were conducted by repeating the micro-simulation 1,000 times for a sample of 45,605 pregnant women per the population of British Columbia, Canada (N = 4.8 million). Preliminary results of the sequential CEAs showed that contingent NIPT was a dominant strategy compared to serum-only screening. Compared with contingent NIPT, universal NIPT at the current test price was not cost-effective with an incremental cost-effectiveness ratio over $100,000/QALY. Contingent NIPT also had the lowest cost per DS case detected among these three strategies. CONCLUSION: Including NIPT in existing prenatal screening for DS is shown to be beneficial over conventional testing. However, at current prices, implementation of NIPT as a second-tier screening test is more cost-effective than deploying it as a universal test.

Cost-effectiveness of faecal calprotectin used in primary care in the diagnosis of inflammatory bowel disease.

OBJECTIVE: Inflammatory bowel disease (IBD) is a chronic, autoimmune, gastrointestinal disorder. Canada has one of the highest prevalence and incidence rates of IBD in the world. Diagnosis is challenging due to the similarity of symptoms to functional gastrointestinal disorders. Faecalcalprotectin (FC) is a biomarker for active mucosal inflammation and has proven effective in the diagnosis of IBD. Our study objective was to assess the cost-effectiveness of adding an FC test compared with standard practice (blood test) in primary care among adult patients presenting with gastrointestinal symptoms. DESIGN: We constructed a decision analytic tree with a 1-year time horizon. The cut-off level of 100 µg/g was used for FC testing. Probabilistic analyses were conducted for the base case and all scenarios. SETTING: Canadian health sector perspective. POPULATION: A hypothetical cohort of adult patients presenting with gastrointestinal symptoms in the primary care setting. INTERVENTIONS: FC test compared with blood test. MAIN OUTCOME MEASURES: Costs, quality-adjusted life years (QALYs), incremental cost-effectiveness ratio (ICER) of FC test expressed as cost per QALY gained compared with blood test and time to IBD diagnosis. RESULTS: FC testing is expected to cost more ($C295.1 vs $C273.9) than standard practice but yield little higher QALY (0.751vs0.750). The ICER of FC test was $C20 323 per QALY. Probabilistic analysis demonstrated that at a willingness-to-pay threshold of $C50 000 per QALY, there was 81.3% probability of FC test being cost-effective. The use of FC test in primary care reduced the time to IBD diagnosis by 40.0 days (95% CI 16.3 to 65.3 days), compared with blood testing alone. CONCLUSIONS: Based on this analysis of short-term outcomes, screening adult patients in primary care using FC test at a cut-off level of 100 µg/g is expected to be cost-effective in Canada.

Attitudes toward prenatal screening for chromosomal abnormalities: A focus group study.

BACKGROUND: While discrete choice experiments (DCEs) are well established methods to ascertain patient preferences, there is limited literature describing use of qualitative methods in DCE design. AIM: This article provides a case study of the qualitative research process for developing the conceptual attributes for a DCE for prenatal screening and diagnosis. METHODS: Participants were recruited through posters and social media. Four in-depth, semi-structured focus groups with pregnant women and their partners/support people were conducted in Metro Vancouver. FINDINGS: Our analysis indicates that choosing prenatal screening and diagnosis involves four intertwined decisions: whether to undergo screening and testing, which screening test to take, which diagnostic test to take, and what to do with a positive diagnosis. The factors that are important to women and their partners vary depending on the decision and include: time of diagnosis, information on conditions tested, false positives, cost, the invasiveness of the test, and potential harm to woman and baby. DISCUSSION: Findings suggest that certain attributes were more salient for screening versus diagnostic tests. Preferences were often shaped by a woman's perceived ability to care for a child with a genetic anomaly, personal risk factors, parity, views on termination, and perceptions on public or private coverage. Participants valued mental well-being and demonstrated a willingness to trade-off on certain attributes in order to minimize stress or anxiety during pregnancy. CONCLUSION: Study findings will be used to inform DCE attributes, levels, and choice questions. Findings will be important for policy decisions surrounding prenatal testing.

How do parents and providers trade-off between disability and survival? Preferences in the treatment of pediatric medulloblastoma.

OBJECTIVE: The aim of this study was to determine the preferences of parents with children with medulloblastoma and clinicians who manage this condition and their trade-offs between survival and disability using a best-worst scaling (BWS) choice experiment. METHODS: Mixed methods were used to develop a BWS tool. Health professionals involved in the care of medulloblastoma patients were contacted through oncology networks in Canada. Parents of children diagnosed with brain tumors were recruited via two clinics in Vancouver and Toronto. The profile case BWS was used with each participant completing 12 choice profiles with the respondent indicating the best and worst features of each profile. Surveys were stratified into good, moderate, and poor prognosis based on the probability of survival presented. Paired model conditional logit analysis was used to generate quantitative preferences. RESULTS: Fifty-four parents (80% female) and 176 providers (36% female, 79% oncologists) participated in this study. There were many similarities in the parents' and providers' preferences for treatment although the parents tended to value survival higher than disability while providers seemed to value the opposite. Specifically, providers were willing to take more risk of recurrence in a child with good prognosis compared to intermediate and poor prognosis. Also, parents were less willing to take more survival risks than providers when they had to trade-off between mild disability and survival rate. CONCLUSION: This study provides useful insights into the preferences of parents and health care providers, the stakeholders of a collaborative decision for the treatment of pediatric medullo-blastoma, and compares their values and trade-offs between different levels of survival and disability.

Testing the External Validity of a Discrete Choice Experiment Method: An Application to Latent Tuberculosis Infection Treatment.

OBJECTIVES: To explore the external validity and predictive power of stated preferences obtained from a discrete choice experiment (DCE) by comparing the predicted behavior of respondents to their actual choices at an individual level. METHODS: A DCE was performed in patients before being offered treatment for latent tuberculosis infection. A mixed logit model was estimated using hierarchical Bayes. The individual-specific preference coefficients were used to calculate the expected probability of choosing the treatment by each patient. The predicted choice using this probability was compared with their actual decision. We used a receiver-operating characteristic curve and different thresholds to convert probabilities into the predicted choices. The comparability of different distributions for the random parameters was also examined. RESULTS: Our results identified significant heterogeneity in preferences for all attributes among respondents. The best model correctly predicted actual treatment decisions for 83% of the participants. The results from using different thresholds and a receiver-operating characteristic curve also confirmed the compatibility between predicted and actual choices. We showed that individual-specific coefficients reflected respondents' actual choices more closely compared with the aggregate-level estimates. CONCLUSIONS: The results of this study provided support for the external validity of DCEs on the basis of their power to predict actual behavior in this setting. Future investigations are, however, required to establish the external validity of DCEs in different settings.

Societal preferences in the treatment of pediatric medulloblastoma: Balancing risk of death and quality of life.

PURPOSE: Medulloblastoma is the most prevalent childhood brain cancer. Children with medulloblastoma typically receive a combination of surgery, radiation, and chemotherapy. The survival rate is high but survivors often have sequelae from radiotherapy of the entire developing brain and spinal cord. Ongoing genetic studies have suggested that decreasing the dose of radiation might be possible among children with favorable molecular variants; however, this may result in an increased disease recurrence. As such, there is a need to investigate the nature of trade-offs that individuals are willing to make regarding the treatment of medulloblastoma. METHOD: We used best-worst scaling to estimate the importance of attributes affecting the general public's decision making around the treatment of medulloblastoma. After conducting focus groups, we selected three relevant attributes: (1) the accuracy of the genetic test; (2) the probability of serious adverse effects of the treatment(s); and (3) the survival rate. Using the paired method, we applied a conditional logit model to estimate preferences. RESULTS: In total, 3,006 respondents (51.3% female) with an average age of 43 years answered the questionnaires. All coefficients were statistically significantly different from zero and the attribute levels of adverse effects and the survival rate had the most impact on individuals' stated decision making. CONCLUSION: Overall, respondents showed high sensitivity to children experiencing disability particularly in the setting of a good prognosis. However, among children with poor prognostic molecular variants, participants showed tolerance about having a child with mild and partial disability compared to a low rate of survival.

Are patients willing to pay for total shoulder arthroplasty? Evidence from a discrete choice experiment.

BACKGROUND: Total shoulder arthroplasty (TSA) is a common treatment to decrease pain and improve shoulder function in patients with severe osteoarthritis (OA). In Canada, patients requiring this procedure often wait a year or more. Our objective was to determine patient preferences related to accessing TSA, specifically comparing out-of-pocket payments for treatment, travel time to hospital, the surgeon's level of experience and wait times. METHODS: We administered a discrete choice experiment among patients with endstage shoulder OA currently waiting for TSA. Respondents were presented with 14 different choice sets, each with 3 options, and they were asked to choose their preferred scenario. A conditional logit regression model was used to estimate the relative preference and willingness to pay for each attribute. RESULTS: Sixty-two respondents completed the questionnaire. Three of the 4 attributes significantly influenced treatment preferences. Respondents had a strong preference for an experienced surgeon (mean 0.89 ± standard error [SE] 0.11), while reductions in travel time (-0.07 ± 0.04) or wait time (-0.04 ± 0.01) were of less importance. Respondents were found to be strongly averse (-1.44 ± 0.18) to surgical treatment by a less experienced surgeon and to paying out-of-pocket for their surgical treatment (-0.56 ± 0.05). CONCLUSION: Our results suggest that patients waiting for TSA to treat severe shoulder OA have minimal willingness to pay for a reduction in wait time or travel time for surgery, yet will pay higher amounts for treatment by an experienced surgeon.

CONTEXTE: L'arthroplastie totale de l'épaule est un traitement courant visant à atténuer la douleur et à augmenter la fonction de l'épaule chez les patients atteints d'arthrose grave. Au Canada, l'attente est souvent d'un an ou plus pour cette intervention. Notre objectif était de cerner les préférences des patients concernant l'accès à l'arthroplastie, particulièrement en ce qui a trait aux déboursés personnels pour le traitement, à la durée du trajet vers l'hôpital, à l'expérience du chirurgien et au temps d'attente. MÉTHODES: Nous avons mené une expérience avec choix discrets auprès de patients atteints d'arthrose de l'épaule au stade terminal actuellement en attente d'une arthroplastie totale. Les répondants ont reçu 14 ensembles de choix différents, comportant chacun 3 options, et devaient choisir leur scénario préféré. Nous avons utilisé un modèle de régression logit conditionnelle pour estimer la préférence relative et la disposition à payer pour chaque caractéristique. RÉSULTATS: En tout, 62 répondants ont rempli le questionnaire. Trois des 4 caractéristiques ont significativement influencé les préférences de traitement. Les répondants avaient une forte préférence pour un chirurgien expérimenté (moyenne de 0,89 ± écart-type de 0,11), alors que la réduction de la durée du trajet (­0,07 ± 0,04) ou du temps d'attente (­0,04 ± 0,01) était moins importante. Les répondants se sont révélés très réfractaires (­1,44 ± 0,18) à se faire opérer par un chirurgien peu expérimenté et à payer de leur poche leur traitement chirurgical (­0,56 ± 0,05). CONCLUSION: Nos résultats semblent indiquer que les patients en attente d'une arthroplastie totale de l'épaule pour traiter une arthrose grave sont très peu disposés à payer pour réduire le temps d'attente ou la durée du trajet, mais qu'ils sont prêts à desserrer les cordons de leur bourse pour être opérés par un chirurgien chevronné.

Health economic evaluation of plasma oxysterol screening in the diagnosis of Niemann-Pick Type C disease among intellectually disabled using discrete event simulation.

BACKGROUND: Recently a less invasive method of screening and diagnosing Niemann-Pick C (NP-C) disease has emerged. This approach involves the use of a metabolic screening test (oxysterol assay) instead of the current practice of clinical assessment of patients suspected of NP-C (review of medical history, family history and clinical examination for the signs and symptoms). Our objective is to compare costs and outcomes of plasma oxysterol screening versus current practice in diagnosis of NP-C disease among intellectually disabled (ID) patients using decision-analytic methods. METHODS: A discrete event simulation model was conducted to follow ID patients through the diagnosis and treatment of NP-C, forecast the costs and effectiveness for a cohort of ID patients and compare the outcomes and costs in two different arms of the model: plasma oxysterol screening and routine diagnosis procedure (anno 2013) over 5 years of follow up. Data from published sources and clinical trials were used in simulation model. Unit costs and quality-adjusted life-years (QALYs) were discounted at a 3% annual rate in the base case analysis. Deterministic and probabilistic sensitivity analyses were conducted. RESULTS: The outcomes of the base case model showed that using plasma oxysterol screening for diagnosis of NP-C disease among ID patients is a dominant strategy. It would result in lower total cost and would slightly improve patients' quality of life. The average amount of cost saving was $3642 CAD and the incremental QALYs per each individual ID patient in oxysterol screening arm versus current practice of diagnosis NP-C was 0.0022 QALYs. Results of sensitivity analysis demonstrated robustness of the outcomes over the wide range of changes in model inputs. CONCLUSION: Whilst acknowledging the limitations of this study, we conclude that screening ID children and adolescents with oxysterol tests compared to current practice for the diagnosis of NP-C is a dominant strategy with clinical and economic benefits. The less costly, more sensitive and specific oxysterol test has potential to save costs to the healthcare system while improving patients' quality of life and may be considered as a routine tool in the NP-C diagnosis armamentarium for ID. Further research is needed to elucidate its effectiveness in patients presenting characteristics other than ID in childhood and adolescence.