Utilisation en contexte québécois des inhibiteurs de la tyrosine kinase du récepteur du facteur de croissance épidermique (EGFR) pour le traitement du cancer du poumon / Use of epidermal growth factor receptor (EGFR) tyrosine kinase inhibitors for the treatment of lung cancer in the québec contex

LE POTENTIEL DES DONNÉES CLINICO-ADMINISTRATIVES POUR AMÉLIORER LES PRATIQUES EN CANCÉROLOGIE: L'INESSS a entrepris un vaste chantier en trois volets pour explorer le potentiel des données clinico-administratives comme levier d'amélioration des pratiques en cancérologie. Les présents travaux font référence au deuxième volet, et portent sur l'évaluation de la valeur des thérapies innovantes en contexte réel québécois. ILS ONT POUR OBJECTIFS: de brosser un portrait de l'utilisation des inhibiteurs de la tyrosine kinase du récepteur du facteur de croissance épidermique (EGFR-ITK) au Québec; d'estimer la survie globale des personnes qui reçoivent un EGFR-ITK pour le traitement du cancer du poumon, dans trois indications, et de déterminer si ces résultats sont similaires à ceux rapportés dans les études publiées; d'apprécier l'état des pratiques au Québec et de déterminer des pistes d'amélioration. LES DONNÉES CLINICO-ADMINISTRATIVES: UTILES POUR L'ÉVALUATION DES THÉRAPIES INNOVANTES: Les travaux réalisés montrent qu'il est possible de brosser un portrait de l'utilisation de certaines thérapies innovantes et d'estimer leur valeur en contexte réel québécois à l'aide des banques de données clinico-administratives. Cependant, il convient de retenir les deux points suivants : certains renseignements utiles ne sont pas disponibles, limitant ainsi les types de thérapies et les indications qui peuvent être évaluées, de même que les méthodes d'évaluation qui peuvent être appliquées; un délai relativement important peut être nécessaire pour obtenir des résultats d'une précision raisonnable sur la survie globale médiane. Pour ces travaux, une cohorte globale constituée des personnes qui ont reçu un EGFR-ITK entre le 1er avril 2001 et le 31 mars 2019 a d'abord été créée, puis trois cohortes correspondant aux indications d'intérêt ont été constituées. ACCÉLÉRER L'ACCÈS AUX NOUVELLES THÉRAPIES: Les analyses suggèrent qu'environ cinq ans ont été nécessaires avant la pleine intégration des EGFR-ITK dans la pratique québécoise. Certains cliniciens semblent toutefois avoir été particulièrement proactifs pour intégrer l'osimertinib à leur pratique. Quelques pistes devraient être envisagées pour accélérer l'accès aux nouvelles thérapies, plus particulièrement lorsque les critères d'admissibilité s'appuient sur de nouveaux paradigmes : prévoir des corridors de services pour faciliter l'accès aux tests compagnons dans toutes les régions du Québec, de même que des ressources pour livrer les résultats dans un délai approprié; améliorer la diffusion de l'information sur la disponibilité des nouvelles thérapies auprès des gestionnaires et des cliniciens; favoriser la diffusion de cette même information auprès des patients et associations de patients. ACCÉLÉRER L'ACCÈS AUX NOUVELLES THÉRAPIES: Les analyses suggèrent qu'environ cinq ans ont été nécessaires avant la pleine intégration des EGFR-ITK dans la pratique québécoise. Certains cliniciens semblent toutefois avoir été particulièrement proactifs pour intégrer l'osimertinib à leur pratique. Quelques pistes devraient être envisagées pour accélérer l'accès aux nouvelles thérapies, plus particulièrement lorsque les critères d'admissibilité s'appuient sur de nouveaux paradigmes : prévoir des corridors de services pour faciliter l'accès aux tests compagnons dans toutes les régions du Québec, de même que des ressources pour livrer les résultats dans un délai approprié; améliorer la diffusion de l'information sur la disponibilité des nouvelles thérapies auprès des gestionnaires et des cliniciens; favoriser la diffusion de cette même information auprès des patients et associations de patients. DES PERSPECTIVES PROMETTEUSES: Le troisième volet de ce chantier exploratoire est en cours. À terme, les résultats les plus utiles des trois volets de ce projet pourront être mis à jour sur une base régulière, d'autres questions visant à améliorer les soins pourront être abordées et les méthodes développées pour l'étude du cancer du poumon pourront éventuellement être adaptées à d'autres types de cancer.

Cost-effectiveness of antenatal multiple micronutrients and balanced energy protein supplementation compared to iron and folic acid supplementation in India, Pakistan, Mali, and Tanzania: A dynamic microsimulation study.

BACKGROUND: Malnutrition among women of childbearing age is especially prevalent in Asia and sub-Saharan Africa and can be harmful to the fetus during pregnancy. In the most recently available Demographic and Health Survey (DHS), approximately 10% to 20% of pregnant women in India, Pakistan, Mali, and Tanzania were undernourished (body mass index [BMI] <18.5 kg/m2), and according to the Global Burden of Disease (GBD) 2017 study, approximately 20% of babies were born with low birth weight (LBW; <2,500 g) in India, Pakistan, and Mali and 8% in Tanzania. Supplementing pregnant women with micro and macronutrients during the antenatal period can improve birth outcomes. Recently, the World Health Organization (WHO) recommended antenatal multiple micronutrient supplementation (MMS) that includes iron and folic acid (IFA) in the context of rigorous research. Additionally, WHO recommends balanced energy protein (BEP) for undernourished populations. However, few studies have compared the cost-effectiveness of different supplementation regimens. We compared the cost-effectiveness of MMS and BEP with IFA to quantify their benefits in 4 countries with considerable prevalence of maternal undernutrition. METHODS AND FINDINGS: Using nationally representative estimates from the 2017 GBD study, we conducted an individual-based dynamic microsimulation of population cohorts from birth to 2 years of age in India, Pakistan, Mali, and Tanzania. We modeled the effect of maternal nutritional supplementation on infant birth weight, stunting and wasting using effect sizes from Cochrane systematic reviews and published literature. We used a payer's perspective and obtained costs of supplementation per pregnancy from the published literature. We compared disability-adjusted life years (DALYs) and incremental cost-effectiveness ratios (ICERs) in a baseline scenario with existing antenatal IFA coverage with scenarios where 90% of antenatal care (ANC) attendees receive either universal MMS, universal BEP, or MMS + targeted BEP (women with prepregnancy BMI <18.5 kg/m2 receive BEP containing MMS while women with BMI ≥18.5 kg/m2 receive MMS). We obtained 95% uncertainty intervals (UIs) for all outputs to represent parameter and stochastic uncertainty across 100 iterations of model runs. ICERs for all scenarios were lowest in Pakistan and greatest in Tanzania, in line with the baseline trend in prevalence of and attributable burden to LBW. MMS + targeted BEP averts more DALYs than universal MMS alone while remaining cost-effective. ICERs for universal MMS compared to baseline IFA were $52 (95% UI: $28 to $78) for Pakistan, $72 (95% UI: $37 to $118) for Mali, $70 (95% UI: $43 to $104) for India, and $253 (95% UI: $112 to $481) for Tanzania. ICERs for MMS + targeted BEP compared to baseline IFA were $54 (95% UI: $32 to $77) for Pakistan, $73 (95% UI: $40 to $104) for Mali, $83 (95% UI: $58 to $111) for India, and $245 (95% UI: $127 to $405) for Tanzania. Study limitations include generalizing experimental findings from the literature to our populations of interest and using population-level input parameters that may not reflect the heterogeneity of subpopulations. Additionally, our microsimulation fuses multiple sources of data and may be limited by data quality and availability. CONCLUSIONS: In this study, we observed that MMS + targeted BEP averts more DALYs and remains cost-effective compared to universal MMS. As countries consider using MMS in alignment with recent WHO guidelines, offering targeted BEP is a cost-effective strategy that can be considered concurrently to maximize benefits and synergize program implementation.

Characteristics of Cost-effectiveness Studies for Oncology Drugs Approved in the United States From 2015-2020.

Importance: Increasingly, cost-effectiveness analyses are being done to determine the value of rapidly increasing oncology drugs; however, this assumes that these analyses are unbiased. Objective: To analyze the characteristics of cost-effectiveness studies and to determine characteristics associated with whether an oncology drug is found to be cost-effective. Design, Setting, and Participants: This retrospective cross-sectional study included 254 cost-effectiveness analyses for 116 oncology drugs that were approved by the US Food and Drug Administration from 2015 to 2020. Exposures: Each drug was analyzed for the incremental cost-effectiveness ratio per quality-adjusted life year, the funding of the study, the authors' conflict of interest, the threshold of willingness-to-pay, from what country's perspective the analysis was done, and whether a National Institute for Health and Care Excellence cost-effectiveness analysis had been done. Main Outcomes and Measures: The main outcome was the odds of a study concluding that a drug was cost-effective. Results: There were 116 drug approvals with 254 studies and country perspectives. Of the country perspectives, 132 (52%) were from the US. Forty-seven of 78 drugs with cost-effective studies had been shown to improve overall survival, whereas 15 of 38 of drugs without a cost-effectiveness study had been shown to improve overall survival. Having a study funded by a pharmaceutical company was associated with higher odds of a study concluding that a drug was cost-effective than studies without funding (odds ratio, 41.36; 95% CI, 11.86-262.23). Conclusions and Relevance: In this cross-sectional study, pharmaceutical funding was associated with greater odds that an oncology drug would be found to be cost-effective. These findings suggest that simply disclosing potential conflict of interest is inadequate. We encourage cost-effectiveness analyses by independent groups.

COVID-19 vaccination in Sindh Province, Pakistan: A modelling study of health impact and cost-effectiveness.

BACKGROUND: Multiple Coronavirus Disease 2019 (COVID-19) vaccines appear to be safe and efficacious, but only high-income countries have the resources to procure sufficient vaccine doses for most of their eligible populations. The World Health Organization has published guidelines for vaccine prioritisation, but most vaccine impact projections have focused on high-income countries, and few incorporate economic considerations. To address this evidence gap, we projected the health and economic impact of different vaccination scenarios in Sindh Province, Pakistan (population: 48 million). METHODS AND FINDINGS: We fitted a compartmental transmission model to COVID-19 cases and deaths in Sindh from 30 April to 15 September 2020. We then projected cases, deaths, and hospitalisation outcomes over 10 years under different vaccine scenarios. Finally, we combined these projections with a detailed economic model to estimate incremental costs (from healthcare and partial societal perspectives), disability-adjusted life years (DALYs), and incremental cost-effectiveness ratio (ICER) for each scenario. We project that 1 year of vaccine distribution, at delivery rates consistent with COVAX projections, using an infection-blocking vaccine at $3/dose with 70% efficacy and 2.5-year duration of protection is likely to avert around 0.9 (95% credible interval (CrI): 0.9, 1.0) million cases, 10.1 (95% CrI: 10.1, 10.3) thousand deaths, and 70.1 (95% CrI: 69.9, 70.6) thousand DALYs, with an ICER of $27.9 per DALY averted from the health system perspective. Under a broad range of alternative scenarios, we find that initially prioritising the older (65+) population generally prevents more deaths. However, unprioritised distribution has almost the same cost-effectiveness when considering all outcomes, and both prioritised and unprioritised programmes can be cost-effective for low per-dose costs. High vaccine prices ($10/dose), however, may not be cost-effective, depending on the specifics of vaccine performance, distribution programme, and future pandemic trends. The principal drivers of the health outcomes are the fitted values for the overall transmission scaling parameter and disease natural history parameters from other studies, particularly age-specific probabilities of infection and symptomatic disease, as well as social contact rates. Other parameters are investigated in sensitivity analyses. This study is limited by model approximations, available data, and future uncertainty. Because the model is a single-population compartmental model, detailed impacts of nonpharmaceutical interventions (NPIs) such as household isolation cannot be practically represented or evaluated in combination with vaccine programmes. Similarly, the model cannot consider prioritising groups like healthcare or other essential workers. The model is only fitted to the reported case and death data, which are incomplete and not disaggregated by, e.g., age. Finally, because the future impact and implementation cost of NPIs are uncertain, how these would interact with vaccination remains an open question. CONCLUSIONS: COVID-19 vaccination can have a considerable health impact and is likely to be cost-effective if more optimistic vaccine scenarios apply. Preventing severe disease is an important contributor to this impact. However, the advantage of prioritising older, high-risk populations is smaller in generally younger populations. This reduction is especially true in populations with more past transmission, and if the vaccine is likely to further impede transmission rather than just disease. Those conditions are typical of many low- and middle-income countries.

Utilisation Trend of Long-Acting Insulin Analogues including Biosimilars across Europe: Findings and Implications.

BACKGROUND: Diabetes mellitus rates and associated costs continue to rise across Europe enhancing health authority focus on its management. The risk of complications is enhanced by poor glycaemic control, with long-acting insulin analogues developed to reduce hypoglycaemia and improve patient convenience. There are concerns though with their considerably higher costs, but moderated by reductions in complications and associated costs. Biosimilars can help further reduce costs. However, to date, price reductions for biosimilar insulin glargine appear limited. In addition, the originator company has switched promotional efforts to more concentrated patented formulations to reduce the impact of biosimilars. There are also concerns with different devices between the manufacturers. As a result, there is a need to assess current utilisation rates for insulins, especially long-acting insulin analogues and biosimilars, and the rationale for patterns seen, among multiple European countries to provide future direction. Methodology. Health authority databases are examined to assess utilisation and expenditure patterns for insulins, including biosimilar insulin glargine. Explanations for patterns seen were provided by senior-level personnel. RESULTS: Typically increasing use of long-acting insulin analogues across Europe including both Western and Central and Eastern European countries reflects perceived patient benefits despite higher prices. However, activities by the originator company to switch patients to more concentrated insulin glargine coupled with lowering prices towards biosimilars have limited biosimilar uptake, with biosimilars not currently launched in a minority of European countries. A number of activities were identified to address this. Enhancing the attractiveness of the biosimilar insulin market is essential to encourage other biosimilar manufacturers to enter the market as more long-acting insulin analogues lose their patents to benefit all key stakeholder groups. CONCLUSIONS: There are concerns with the availability and use of insulin glargine biosimilars among European countries despite lower costs. This can be addressed.

Cost-Effectiveness of COVID-19 Policy Measures: A Systematic Review.

OBJECTIVES: The COVID-19 pandemic has had a major impact on our society, with drastic policy restrictions being implemented to contain the spread of the severe acute respiratory syndrome coronavirus 2. This study aimed to provide an overview of the available evidence on the cost-effectiveness of various coronavirus disease 2019 policy measures. METHODS: A systematic literature search was conducted in PubMed, Embase, and Web of Science. Health economic evaluations considering both costs and outcomes were included. Their quality was comprehensively assessed using the Consensus Health Economic Criteria checklist. Next, the quality of the epidemiological models was evaluated. RESULTS: A total of 3688 articles were identified (March 2021), of which 23 were included. The studies were heterogeneous with regard to methodological quality, contextual factors, strategies' content, adopted perspective, applied models, and outcomes used. Overall, testing/screening, social distancing, personal protective equipment, quarantine/isolation, and hygienic measures were found to be cost-effective. Furthermore, the most optimal choice and combination of strategies depended on the reproduction number and context. With a rising reproduction number, extending the testing strategy and early implementation of combined multiple restriction measures are most efficient. CONCLUSIONS: The quality assessment highlighted numerous flaws and limitations in the study approaches; hence, their results should be interpreted with caution because the specific context (country, target group, etc) is a key driver for cost-effectiveness. Finally, including a societal perspective in future evaluations is key because this pandemic has an indirect impact on the onset and treatment of other conditions and on our global economy.

Epidemiological Analysis of 37,424 Carotid Artery Stenosis Intervention Procedures During 11 Years in the Public Health System in Brazil: Stenting has Been More Common Than Endarterectomy.

BACKGROUND: Stroke is a leading cause of death worldwide, with carotid atherosclerosis accounting for 10-20% of cases. In Brazil, the Public Health System provides care for roughly two-thirds of the population. No studies, however, have analysed large-scale results of carotid bifurcation surgery in Brazil. METHODS: This study aimed to describe rates of carotid artery stenting (CAS) and carotid endarterectomy (CEA) performed between 2008 and 2019 in the country through web scraping of publicly available databases. RESULTS: Between 2008 and 2019, 37,424 carotid bifurcation revascularization procedures were performed, of which 22,578 were CAS (60.34%) and 14,846 (39.66%) were CEA. There were 620 in-hospital deaths (1.66%), 336 after CAS (1.48%) and 284 after CEA (1.92%) (P = 0.032). Governmental reimbursement was US$ 77,216,298.85 (79.31% of all reimbursement) for CAS procedures and US$ 20,143,009.63 (20.69%) for CEA procedures. The average cost per procedure for CAS (US$ 3,062.98) was higher than that for CEA (US$ 1,430.33) (P = 0.008). CONCLUSIONS: In Brazil, the frequency of CAS largely surpassed that of CEA. In-hospital mortality rates of CAS were significantly lower than those of CEA, although both had mortality rates within the acceptable rates as dictated by literature. The cost of CAS, however, was significantly higher. This is a pioneering analysis of carotid artery disease management in Brazil that provides, for the first time, preliminary insight into the fact that the low adoption of CEA in the country is in opposition to countries where utilization rates are higher for CEA than for CAS.

When More Is Better: Underused Advanced Imaging Exams That Can Improve Outcomes and Reduce Cost of Care.

Appropriate use of resources is a tenet of care transformation efforts, with a national campaign to reduce low-value imaging. The next level of performance improvement is to bolster evidence-based screening, imaging surveillance, and diagnostic innovation, which can avert more costly, higher-risk elements of unnecessary care like emergent interventions. Clinical scenarios in which underused advanced imaging can improve outcomes and reduce total cost of care are reviewed, including abdominal aortic aneurysm surveillance, coronary artery disease diagnosis, and renal mass characterization. Reliable abdominal aortic aneurysm surveillance imaging reduces emergency surgery and can be driven by radiologists incorporating best practice standardized recommendations in imaging interpretations. Coronary computed tomography angiography in patients with stable and unstable chest pain can reduce downstream resource use while improving outcomes. Preoperative 99mTc-sestamibi single-photon emission computed tomography (SPECT) reliably distinguishes oncocytoma from renal cell carcinoma to obviate unnecessary nephrectomy. As technological advances in diagnostic, molecular, and interventional radiology improve our ability to detect and cure disease, analyses of cost effectiveness will be critical to radiology leadership and sustainability in the transition to a value-based reimbursement model.

Evaluation of Benefit and Cost Utility of Immediate Postanesthesia Care Unit Radiographs to Predict Airway Compromise After Anterior Cervical Discectomy and Fusion.

STUDY DESIGN: Retrospective review. OBJECTIVE: Anterior cervical discectomy and fusion (ACDF) is a commonly performed procedure that may be complicated by airway compromise postoperatively. This life-threatening complication may necessitate reintubation and reoperation. We evaluated the cost utility of conventional postoperative x-ray. SUMMARY OF BACKGROUND DATA: Studies have demonstrated minimal benefit in obtaining an x-ray on postoperative day 1, but there is some utility of postanesthesia care unit (PACU) x-rays for predicting the likelihood of reoperation. METHODS: We retrospectively reviewed the records of consecutive patients who underwent ACDF between September 2013 and February 2017. Patients were dichotomized into those who received PACU x-rays and those who did not (control group). Primary outcomes were reoperation, reintubation, mortality, and health care costs. RESULTS: Eight-hundred and fifteen patients were included in our analysis: 558 had PACU x-rays; 257 did not. In those who received PACU x-rays, mean age was 53.7 ±â€Š11.3 years, mean levels operated on were 2.0 ±â€Š0.79, and mean body mass index (BMI) was 30.3 ±â€Š6.9. In those who did not, mean age was 51.8 ±â€Š10.9 years, mean levels operated on were 1.48 ±â€Š0.65, and mean BMI was 29.9 ±â€Š6.3. Complications in the PACU x-ray group were reintubation-0.4%, reoperation-0.7%, and death-0.3% (due to prevertebral swelling causing airway compromise). Complications in the control group were reintubation-0.4%, reoperation-0.8%, and death-0. There were no differences between groups with respect to reoperation (P = 0.92), reintubation (P = 0.94), or mortality (P = 0.49). The mean per-patient cost was significantly higher (P = 0.009) in those who received PACU x-rays, $1031.76 ±â€Š948.67, versus those in the control group, $700.26 ±â€Š634.48. Mean length of stay was significantly longer in those who had PACU x-rays (P = 0.01). CONCLUSION: Although there were no differences in reoperation, reintubation, or mortality, there was a significantly higher cost for care and hospitalization in those who received PACU x-rays. Further studies are warranted to validate the results of the presented study.Level of Evidence: 3.

Artificial intelligence in colonoscopy - Now on the market. What's next?

Adoption of artificial intelligence (AI) in clinical medicine is revolutionizing daily practice. In the field of colonoscopy, major endoscopy manufacturers have already launched their own AI products on the market with regulatory approval in Europe and Asia. This commercialization is strongly supported by positive evidence that has been recently established through rigorously designed prospective trials and randomized controlled trials. According to some of the trials, AI tools possibly increase the adenoma detection rate by roughly 50% and contribute to a 7-20% reduction of colonoscopy-related costs. Given that reliable evidence is emerging, together with active commercialization, this seems to be a good time for us to review and discuss the current status of AI in colonoscopy from a clinical perspective. In this review, we introduce the advantages and possible drawbacks of AI tools and explore their future potential including the possibility of obtaining reimbursement.

Factors Associated With Progression to Surgical Intervention for Lumbar Disc Herniation in the Military Health System.

STUDY DESIGN: Retrospective cohort. OBJECTIVE: To determine surgery-free survival of patients receiving conservative management of lumbar disc herniation (LDH) in the military healthcare system (MHS) and risk factors for surgical intervention. SUMMARY OF BACKGROUND DATA: Radiculopathy from LDH is a major cause of morbidity and cost. METHODS: The Military Data Repository was queried for all patients diagnosed with LDH from FY2011-2018; the earliest such diagnosis in a military treatment facility (MTF) was kept for each patient as the initial diagnosis. Follow-up time to surgical intervention was defined as the time from diagnosis to first encounter for lumbar microdiscectomy or lumbar decompression in either a MTF or in the civilian sector. The Military Data Repository was also queried for history of tobacco use at any time during MHS care, age at the time of diagnosis, sex, MHS beneficiary category, and diagnosing facility characteristics. Multivariable Cox proportional hazards models were used to evaluate the associations of patient and diagnosing facility characteristics with time to surgical intervention. RESULTS: A total of 84,985 MHS beneficiaries including 62,771 active duty service members were diagnosed with LDH in a MTF during the 8-year study period. A total of 10,532 (12.4%) MHS beneficiaries, including 7650 (10.9%) active duty, failed conservative management onto surgical intervention with lumbar microdiscectomy or lumbar decompression. Median follow-up time of the cohort was 5.2 (interquartile range 2.6, 7.5) years. Among all healthcare beneficiaries, several patient-level (younger age, male sex, and history of tobacco use) and facility-level characteristics (hospital vs. clinic and surgical care vs. primary care clinic) were independently associated with higher risk of surgical intervention. CONCLUSION: LDH compromises military readiness and negatively impacts healthcare costs. MHS beneficiaries with LDH have a good prognosis with approximately 88% of patients successfully completing conservative management. However, strategies to improve outcomes of conservative management in LDH should address risks associated with both patient and facility characteristics.Level of Evidence: 4.

Exploring the Cost Effectiveness of Shared Decision Making for Choosing between Disease-Modifying Drugs for Relapsing-Remitting Multiple Sclerosis in the Netherlands: A State Transition Model.

BACKGROUND: Up to 31% of patients with relapsing-remitting multiple sclerosis (RRMS) discontinue treatment with disease-modifying drug (DMD) within the first year, and of the patients who do continue, about 40% are nonadherent. Shared decision making may decrease nonadherence and discontinuation rates, but evidence in the context of RRMS is limited. Shared decision making may, however, come at additional costs. This study aimed to explore the potential cost-effectiveness of shared decision making for RRMS in comparison with usual care, from a (limited) societal perspective over a lifetime. METHODS: An exploratory economic evaluation was conducted by adapting a previously developed state transition model that evaluates the cost-effectiveness of a range of DMDs for RRMS in comparison with the best supportive care. Three potential effects of shared decision making were explored: 1) a change in the initial DMD chosen, 2) a decrease in the patient's discontinuation in using the DMD, and 3) an increase in adherence to the DMD. One-way and probabilistic sensitivity analyses of a scenario that combined the 3 effects were conducted. RESULTS: Each effect separately and the 3 effects combined resulted in higher quality-adjusted life years (QALYs) and costs due to the increased utilization of DMD. A decrease in discontinuation of DMDs influenced the incremental cost-effectiveness ratio (ICER) most. The combined scenario resulted in an ICER of €17,875 per QALY gained. The ICER was sensitive to changes in several parameters. CONCLUSION: This study suggests that shared decision making for DMDs could potentially be cost-effective, especially if shared decision making would help to decrease treatment discontinuation. Our results, however, may depend on the assumed effects on treatment choice, persistence, and adherence, which are actually largely unknown.

Cost-effectiveness of Population-Wide Genomic Screening for Hereditary Breast and Ovarian Cancer in the United States.

Importance: Genomic screening for hereditary breast and ovarian cancer (HBOC) in unselected women offers an opportunity to prevent cancer morbidity and mortality, but the potential clinical impact and cost-effectiveness of such screening have not been well studied. Objective: To estimate the lifetime incremental incidence of HBOC and the quality-adjusted life-years (QALYs), costs, and cost-effectiveness of HBOC genomic screening in an unselected population vs family history-based testing. Design, Setting, and Participants: In this study conducted from October 27, 2017, to May 3, 2020, a decision analytic Markov model was developed that included health states for precancer, for risk-reducing mastectomy (RRM) and risk-reducing salpingo-oophorectomy (RRSO), for earlier- and later-stage HBOC, after cancer, and for death. A complimentary cascade testing module was also developed to estimate outcomes in first-degree relatives. Age-specific RRM and RRSO uptake probabilities were estimated from the Geisinger MyCode Community Health Initiative and published sources. Parameters including RRM and RRSO effectiveness, variant-specific cancer risk, costs, and utilities were derived from published sources. Sensitivity and scenario analyses were conducted to evaluate model assumptions and uncertainty. Main Outcomes and Measures: Lifetime cancer incidence, QALYs, life-years, and direct medical costs for genomic screening in an unselected population vs family history-based testing only were calculated. The incremental cost-effectiveness ratio (ICER) was calculated as the difference in cost between strategies divided by the difference in QALYs between strategies. Earlier-stage and later-stage cancer cases prevented and total cancer cases prevented were also calculated. Results: The model found that population screening of 30-year-old women was associated with 75 (95% credible range [CR], 60-90) fewer overall cancer cases and 288 QALYs (95% CR, 212-373 QALYs) gained per 100 000 women screened, at an incremental cost of $25 million (95% CR, $21 millon to $30 million) vs family history-based testing; the ICER was $87 700 (78% probability of being cost-effective at a threshold of $100 000 per QALY). In contrast, population screening of 45-year-old women was associated with 24 (95% CR, 18-29) fewer cancer cases and 97 QALYs (95% CR, 66-130 QALYs) gained per 100 000 women screened, at an incremental cost of $26 million (95% CR, $22 million to $30 million); the ICER was $268 200 (0% probability of being cost-effective at a threshold of $100 000 per QALY). A scenario analysis without cascade testing increased the ICER to $92 600 for 30-year-old women and $354 500 for 45-year-old women. A scenario analysis assuming a 5% absolute decrease in mammography screening in women without a variant was associated with the potential for net harm (-90 QALYs per 100 000 women screened; 95% CR, -180 to 10 QALYs). Conclusions and Relevance: The results of this study suggest that population HBOC screening may be cost-effective among younger women but not among older women. Cascade testing of first-degree relatives added a modest improvement in clinical and economic value. The potential for harm conferred by inappropriate reduction in mammography among noncarriers should be quantified.

Systematic review of unintentional injury prevention economic evaluations 2010-2019 and comparison to 1998-2009.

BACKGROUND: Health economic evaluation studies (e.g., cost-effectiveness analysis) can provide insight into which injury prevention interventions maximize available resources to improve health outcomes. A previous systematic review summarized 48 unintentional injury prevention economic evaluations published during 1998-2009, providing a valuable overview of that evidence for researchers and decisionmakers. The aim of this study was to summarize the content and quality of recent (2010-2019) economic evaluations of unintentional injury prevention interventions and compare to the previous publication period (1998-2009). METHODS: Peer-reviewed English-language journal articles describing public health unintentional injury prevention economic evaluations published January 1, 2010 to December 31, 2019 were identified using index terms in multiple databases. Injury causes, interventions, study methods, and results were summarized. Reporting on key methods elements (e.g., economic perspective, time horizon, discounting, currency year, etc.) was assessed. Reporting quality was compared between the recent and previous publication periods. RESULTS: Sixty-eight recent economic evaluation studies were assessed. Consistent with the systematic review on this topic for the previous publication period, falls and motor vehicle traffic injury prevention were the most common study subjects. Just half of studies from the recent publication period reported all key methods elements, although this represents an improvement compared to the previous publication period (25 %). CONCLUSION: Most economic evaluations of unintentional injury prevention interventions address just two injury causes. Better adherence to health economic evaluation reporting standards may enhance comparability across studies and increase the likelihood that this type of evidence is included in decision-making related to unintentional injury prevention.

Implementation science on the margins: How do we demonstrate the value of implementation strategies?

Implementation science (IS) has developed as a field to assess effective ways to implement and disseminate evidence-based practices. Although the size and rigor of the field has improved, the economic evaluation of implementation strategies has lagged behind other areas of IS (Roberts, Healey, & Sevdalis, 2019). Beyond demonstrating the effectiveness of implementation strategies, there needs to be evidence that investments in these strategies are efficient or financially sustainable. In this editorial, we lay out conceptual challenges in applying economic evaluation to IS and the implications for conducting economic analyses in integrated primary care research. (PsycInfo Database Record (c) 2020 APA, all rights reserved).

Endoscopic full-thickness resection and its treatment alternatives in difficult-to-treat lesions of the lower gastrointestinal tract: a cost-effectiveness analysis.

OBJECTIVE: Endoscopic full-thickness resection (EFTR) has shown efficacy and safety in the colorectum. The aim of this analysis was to investigate whether EFTR is cost-effective in comparison with surgical and endoscopic treatment alternatives. DESIGN: Real data from the study cohort of the prospective, single-arm WALL RESECT study were used. A simulated comparison arm was created based on a survey that included suggested treatment alternatives to EFTR of the respective lesions. Treatment costs and reimbursement were calculated in euro according to the coding rules of 2017 and 2019 (EFTR). R0 resection rate was used as a measure of effectiveness. To assess cost-effectiveness, the average cost-effectiveness ratio (ACER) and the incremental cost-effectiveness ratio (ICER) were determined. Calculations were made both from the perspective of the care provider as well as of the payer. RESULTS: The cost per case was €2852.20 for the EFTR group, €1712 for the standard endoscopic resection (SER) group, €8895 for the surgical resection group and €5828 for the pooled alternative treatment to EFTR. From the perspective of the care provider, the ACER (mean cost per R0 resection) was €3708.98 for EFTR, €3115.10 for SER, €8924.05 for surgical treatment and €7169.30 for all pooled and weighted alternatives to EFTR. The ICER (additional cost per R0 resection compared with EFTR) was €5196.47 for SER, €26 533.13 for surgical resection and €67 768.62 for the pooled rate of alternatives. Results from the perspective of the payer were similar. CONCLUSION: EFTR is cost-effective in comparison with surgical and endoscopic treatment alternatives in the colorectum.

Vagus nerve stimulation with tachycardia detection provides additional seizure reduction compared to traditional vagus nerve stimulation.

PURPOSE: This study investigates the clinical and cost effectiveness of switching from traditional vagus nerve stimulation (VNS) to responsive VNS (rVNS), which has an additional ictal tachycardia detection and stimulation (AutoStim) mode. METHODS: Retrospective chart review was used to collect data from patients with medically refractory epilepsy who underwent generator replacements. Patients with confounding factors such as medication changes were excluded. Vagus nerve stimulation parameters, seizure frequency, and healthcare costs were collected for the 1-year period following generator replacement with the rVNS device. RESULTS: Documented seizure frequency was available for twenty-five patients. After implant with rVNS, 28% of patients had an additional ≥50% seizure reduction. There was a significant decrease in the average monthly seizure count (p = 0.039). In patients who were not already free of disabling seizures (n = 17), 41.2% had ≥50% additional seizure reduction. There was no difference in healthcare costs during the 1-year follow-up after the rVNS implant compared with one year prior. CONCLUSIONS: Ictal tachycardia detection and stimulation provided a significant clinical benefit in patients who were not free of disabling seizures with treatment from traditional VNS. There was no additional increase in healthcare costs during the first year after device replacement.

A Computationally Efficient Method for Probabilistic Parameter Threshold Analysis for Health Economic Evaluations.

Background. Threshold analysis is used to determine the threshold value of an input parameter at which a health care strategy becomes cost-effective. Typically, it is performed in a deterministic manner, in which inputs are varied one at a time while the remaining inputs are each fixed at their mean value. This approach will result in incorrect threshold values if the cost-effectiveness model is nonlinear or if inputs are correlated. Objective. To propose a probabilistic method for performing threshold analysis, which accounts for the joint uncertainty in all input parameters and makes no assumption about the linearity of the cost-effectiveness model. Methods. Three methods are compared: 1) deterministic threshold analysis (DTA); 2) a 2-level Monte Carlo approach, which is considered the gold standard; and 3) a regression-based method using a generalized additive model (GAM), which identifies threshold values directly from a probabilistic sensitivity analysis sample. Results. We applied the 3 methods to estimate the minimum probability of hospitalization for typhoid fever at which 3 different vaccination strategies become cost-effective in Uganda. The threshold probability of hospitalization at which routine vaccination at 9 months with catchup campaign to 5 years becomes cost-effective is estimated to be 0.060 and 0.061 (95% confidence interval [CI], 0.058-0.064), respectively, for 2-level and GAM. According to DTA, routine vaccination at 9 months with catchup campaign to 5 years would never become cost-effective. The threshold probability at which routine vaccination at 9 months with catchup campaign to 15 years becomes cost-effective is estimated to be 0.092 (DTA), 0.074 (2-level), and 0.072 (95% CI, 0.069-0.075) (GAM). GAM is 430 times faster than the 2-level approach. Conclusions. When the cost-effectiveness model is nonlinear, GAM provides similar threshold values to the 2-level Monte Carlo approach and is computationally more efficient. DTA provides incorrect results and should not be used.

An Economic Analysis of Intensive Multidisciplinary Interventions for Treating Medicaid-Insured Children with Pediatric Feeding Disorders.

Background. Intensive multidisciplinary intervention (IMI) represents a well-established treatment for pediatric feeding disorders (PFDs), but program availability represents an access care barrier. We develop an economic analysis of IMI for weaning from gastronomy tube (G-tube) treatment for children diagnosed with PFDs from the Medicaid programs' perspective, where Medicaid programs refer to both fee-for-service and managed care programs. Methods. The 2010-2012 Medicaid Analytic eXtract claims provided health care data for children aged 13 to 72 months. An IMI program provided data on average admission costs. We employed a finite-horizon Markov model to simulate PFD treatment progression assuming 2 treatment arms: G-tube only v. IMI targeting G-tube weaning. We compared the expenditure differential between the 2 arms under varying time horizons and treatment effectiveness. Results. Overall Medicaid expenditure per member per month was $6814, $2846, and $1550 for the study population of children with PFDs and G-tube treatment, the control population with PFDs without G-tube treatment, and the no-PFD control population, respectively. The PFD-diagnosed children with G-tube treatment only had the highest overall expenditures across all health care settings except psychological services. The expenditure at the end of the 8-year time horizon was $405,525 and $208,218 per child for the G-tube treatment only and IMI arms, respectively. Median Medicaid expenditure was between 1.7 and 2.2 times higher for the G-tube treatment arm than for the IMI treatment arm. Limitations. Data quality issues could cause overestimates or underestimates of Medicaid expenditure. Conclusions. This study demonstrated the economic benefits of IMI to treat complex PFDs from the perspective of Medicaid programs, indicating this model of care not only holds benefit in terms of improving overall quality of life but also brings significant expenditure savings in the short and long term.

Changes in Cost-Effectiveness for Chronic Hepatitis C Virus Pharmacotherapy: The Case for Continuous Cost-Effectiveness Analyses.

BACKGROUND: Cost-effectiveness evaluations for hepatitis C virus (HCV) treatments have been published frequently, but new products with significant cost and effectiveness differences make these analyses obsolete. How valuable are economic models for a fixed time period in a dynamic market? OBJECTIVE: To estimate the cost-effectiveness of the best available HCV treatment at different points in time, using the same comparator to demonstrate how rapid innovation in a disease area influences economic outcomes. METHODS: A Markov model was used to calculate the cost-effectiveness of treatment in 2010, 2012, 2014, 2016, and 2018 compared with a standard comparator (no treatment) from the payer perspective. Expected drug costs and treatment effectiveness estimates for sustained virologic response (SVR) were calculated using recommended regimens for each of the 6 HCV genotypes at each time point and distribution of genotypes in the United States. Patients entered the model with different stages of fibrosis. Utility estimates for each health state were used to calculate quality-adjusted life-years (QALYs) earned at each cycle. Incremental cost-effectiveness ratios were reported for each year to compare the "treatment versus no treatment" decision at that time. RESULTS: No HCV treatment resulted in a gain of 11.54 QALYs over a 20-year time horizon at a cost of $42,938. Costs for treated groups were $69,075, $123,267, $125,431, $86,782, and $56,470 for the 2010, 2012, 2014, 2016, and 2018 scenarios, respectively. QALYs gained for treated groups were 12.90, 12.97, 13.34, 13.39, and 13.46 for the 2010, 2012, 2014, 2016, and 2018 scenarios, respectively. The incremental cost-effectiveness ratios in each year compared with no treatment were $19,218 per QALY, $56,104 per QALY, $45,829 per QALY, $23,699 per QALY, and $7,048 per QALY. CONCLUSIONS: Treatment effectiveness for HCV has increased steadily, while treatment costs increased substantially from 2010-2014 before decreasing to its lowest point in 2018. Thus, the dynamic nature of innovation creates the need for iterative cost-effectiveness analyses. DISCLOSURES: No outside funding supported this study. Mattingly reports unrelated consulting from the National Health Council, Bristol Myers Squibb, G&W Laboratories, Allergy and Asthma Foundation of American, and the Massachusetts Health Policy Commission. Love reports an unrelated research grant from the American Cancer Society.