Key Considerations in the Health Technology Assessment of Advanced Therapy Medicinal Products in Scotland, The Netherlands, and England.

OBJECTIVES: Advanced therapy medicinal products (ATMPs) are highly innovative therapies. Their costs and uncertain value claims have raised concerns among health technology assessment (HTA) bodies and payers. Little is known about how underlying considerations in HTA of ATMPs shape assessment and reimbursement recommendations. We aim to identify and assess key considerations that played a role in HTA of ATMPs underlying reimbursement recommendations. METHODS: A review of HTA reports was conducted of all authorized ATMPs in Scotland, The Netherlands, and England. Considerations were extracted and categorized into EUnetHTA Core Model domains. Per jurisdiction, considerations were aggregated and key considerations identified (defined as occurring in >1/assessment per jurisdiction). A narrative analysis was conducted comparing key considerations between jurisdictions and different reimbursement recommendations. RESULTS: We identified 15 ATMPs and 18 HTA reports. In The Netherlands and England most key considerations were identified in clinical effectiveness (EFF) and cost- and economic effectiveness (ECO) domains. In Scotland, the social aspects domain yielded most key considerations, followed by ECO and EFF. More uncertainty in evidence and assessment outcomes was accepted when orphan or end-of-life criteria were applied. A higher percentage of considerations supporting recommendations were identified for products with positive recommendations compared with restricted and negative recommendations. CONCLUSIONS: This is the first empirical review of HTA's using the EUnetHTA Core Model to identify and structure key considerations retrospectively. It provides insights in supporting and opposing considerations for reimbursement of individual products and differences between jurisdictions. Besides the EFF and ECO domain, the social, ethical, and legal domains seem to bear considerable weight in assessment of ATMPs.

Lives saved and lost in the first six month of the US COVID-19 pandemic: A retrospective cost-benefit analysis.

In the beginning of the COVID-19 US epidemic in March 2020, sweeping lockdowns and other aggressive measures were put in place and retained in many states until end of August of 2020; the ensuing economic downturn has led many to question the wisdom of the early COVID-19 policy measures in the US. This study's objective was to evaluate the cost and benefit of the US COVID-19-mitigating policy intervention during the first six month of the pandemic in terms of COVID-19 mortality potentially averted, versus mortality potentially attributable to the economic downturn. We conducted a synthesis-based retrospective cost-benefit analysis of the full complex of US federal, state, and local COVID-19-mitigating measures, including lockdowns and all other COVID-19-mitigating measures, against the counterfactual scenario involving no public health intervention. We derived parameter estimates from a rapid review and synthesis of recent epidemiologic studies and economic literature on regulation-attributable mortality. According to our estimates, the policy intervention saved 866,350-1,711,150 lives (4,886,214-9,650,886 quality-adjusted life-years), while mortality attributable to the economic downturn was 57,922-245,055 lives (2,093,811-8,858,444 life-years). We conclude that the number of lives saved by the spring-summer lockdowns and other COVID-19-mitigation was greater than the number of lives potentially lost due to the economic downturn. However, the net impact on quality-adjusted life expectancy is ambiguous.

Imaging of Patients Suspected of SLAP Tear: A Cost-Effectiveness Study.

BACKGROUND. Superior labrum anterior and posterior (SLAP) tears are a common shoulder pathology. Although MRI is the imaging reference standard for diagnosis of this pathology, the cost-effectiveness of common MRI strategies is unclear. OBJECTIVE. The primary objective of our study was to determine the cost-effectiveness of the common MRI-based strategies used for the diagnosis of SLAP tears. METHODS. We created decision analytic models from the perspective of the U.S. health care system over a 2-year time horizon for a hypothetical population of 25-year-old patients with a previous diagnosis of SLAP tear. We used the decision models to compare the differences in incremental cost-effectiveness of the common MRI strategies, which included combinations of 1.5-T and 3-T MR arthrography (MRA) and unenhanced MRI protocols, and the resulting treatment applied for these patients. Input data on cost, probability, and utility estimates were obtained through a comprehensive literature search. The primary effectiveness outcome was quality-adjusted life years. Costs were estimated in 2017 U.S. dollars. RESULTS. When all imaging strategies were considered, the unenhanced 3-T MRI-based imaging strategy was the preferred and dominant option over 3-T MRA and 1.5-T imaging (MRI and MRA). When the model was run without 3-T imaging as an option, 1.5-T MRA was the favored option. Probabilistic sensitivity analyses confirmed the same preferred imaging strategy results. CONCLUSION. An unenhanced 3-T MRI-based strategy is the most cost-effective imaging option for patients with suspected SLAP tear. When 3-T imaging is not available, 1.5-T MRA is more cost-effective than 1.5-T MRI. The main driver of these results is the fact that 3-T MRI and 1.5-T MRA are the most specific tests in these respective scenarios, which results in fewer false-positives, prevents unnecessary surgeries, and leads to decreased costs. CLINICAL IMPACT. Our cost-effectiveness model findings complement prior diagnostic accuracy work, helping produce a more comprehensive approach to define imaging utility for radiologists, clinicians, and patients with SLAP tears who have access to various types of MRI options.

A cost-utility analysis of 18F-fluorocholine-positron emission tomography imaging for localizing primary hyperparathyroidism in the United States.

BACKGROUND: Primary hyperparathyroidism historically necessitated bilateral neck exploration to remove abnormal parathyroid tissue. Improved localization allows for focused parathyroidectomy with lower complication risks. Recently, positron emission tomography using radiolabeled 18F-fluorocholine demonstrated high accuracy in detecting these lesions, but its cost-effectiveness has not been studied in the United States. METHODS: A decision tree modeled patients who underwent parathyroidectomy for primary hyperparathyroidism using single preoperative localization modalities: (1) positron emission tomography using radiolabeled 18F-fluorocholine, (2) 4-dimensional computed tomography, (3) ultrasound, and (4) sestamibi single photon emission computed tomography (SPECT). All patients underwent either focused parathyroidectomy versus bilateral neck exploration, with associated cost ($) and clinical outcomes measured in quality-adjusted life-years gained. Model parameters were informed by literature review and Medicare costs. Incremental cost-utility ratios were calculated in US dollars/quality-adjusted life-years gained, with a willingness-to-pay threshold set at $100,000/quality-adjusted life-year. One-way, 2-way, and threshold sensitivity analyses were performed. RESULTS: Positron emission tomography using radiolabeled 18F-fluorocholine gained the most quality-adjusted life-years (23.9) and was the costliest ($2,096), with a total treatment cost of $11,245 or $470/quality-adjusted life-year gained. Sestamibi single photon emission computed tomography and ultrasound were dominated strategies. Compared with 4-dimentional computed tomography, the incremental cost-utility ratio for positron emission tomography using radiolabeled 18F-fluorocholine was $91,066/quality-adjusted life-year gained in our base case analysis, which was below the willingness-to-pay threshold. In 1-way sensitivity analysis, the incremental cost-utility ratio was sensitive to test accuracy, positron emission tomography using radiolabeled 18F-fluorocholine price, postoperative complication probabilities, proportion of bilateral neck exploration patients needing overnight hospitalization, and life expectancy. CONCLUSION: Our model elucidates scenarios in which positron emission tomography using radiolabeled 18F-fluorocholine can potentially be a cost-effective imaging option for primary hyperparathyroidism in the United States. Further investigation is needed to determine the maximal cost-effectiveness for positron emission tomography using radiolabeled 18F-fluorocholine in selected populations.

Procedural and 12-month in-hospital costs of primary infra-popliteal bypass surgery, infrapopliteal best endovascular treatment, and major lower limb amputation for chronic limb threatening ischemia.

OBJECTIVE: Chronic limb-threatening ischemia (CLTI) is a growing global problem due to the widespread use of tobacco and increasing prevalence of diabetes. Although the financial consequences are considerable, few studies have compared the relative cost-effectiveness of different CLTI management strategies. The Bypass vs Angioplasty in Severe Ischaemia of the Leg (BASIL)-2 trial is randomizing patients with CLTI to primary infrapopliteal (IP) vein bypass surgery (BS) or best endovascular treatment (BET) and includes a comprehensive within-trial cost-utility analysis. The aim of this study is to compare over a 12-month time horizon, the costs of primary IP BS, IP best endovascular treatment (BET), and major limb major amputation (MLLA) to inform the BASIL-2 cost-utility analysis. METHODS: We compared procedural human resource (HR) costs and total in-hospital costs for the index admission, and over the following 12-months, in 60 consecutive patients undergoing primary IP BS (n = 20), IP BET (n = 20), or MLLA (10 transfemoral and 10 transtibial) for CLTI within the BASIL prospective cohort study. RESULTS: Procedural HR costs were greatest for BS (BS £2551; 95% confidence interval [CI], £1934-£2807 vs MLLA £1130; 95% CI, £1046-£1297 vs BET £329; 95% CI, £242-£390; P < .001, Kruskal-Wallis) due to longer procedure duration and greater staff requirement. With regard to the index admission, MLLA was the most expensive due to longer hospital stay (MLLA £13,320; 95% CI, £8986-£18,616 vs BS £8714; 95% CI, £6097-£11,973 vs BET £4813; 95% CI, £3529-£6097; P < .001, Kruskal-Wallis). The total cost of the index admission and in-hospital care over the following 12 months remained least for BET (MLLA £26,327; 95% CI, £17,653-£30,458 vs BS £20,401; 95% CI, £12,071-£23,926 vs BET £12,298; 95% CI, £6961-£15,439; P < .001, Kruskal-Wallis). CONCLUSIONS: Over a 12-month time horizon, MLLA and IP BS are more expensive than IP BET in terms of procedural HR costs and total in-hospital costs. These economic data, together with quality of life data from BASIL-2, will inform the calculation of incremental cost-effectiveness ratios for different CLTI management strategies within the BASIL-2 cost-utility analysis.

Ultrasound-Guided Injection Treatments Versus Surgical Neurectomy for Morton Neuroma: A Cost-Effectiveness Analysis.

BACKGROUND. Morton neuroma is a common, painful disorder of the foot with multiple treatment options of varying cost and effectiveness. OBJECTIVE. The aim of this study was to determine the most cost-effective treatment pathway for symptomatic Morton neuromas when conservative management has failed. METHODS. An incremental cost-utility analysis was performed comparing a direct to surgical neurectomy strategy with three selective injection strategies in which one or more ultrasound-guided injection therapies was tried first before surgery for patients who did not respond to treatment. The three selective injection strategies were selective steroid injection, selective alcohol injection, and selective steroid/alcohol injection in which both steroid injections and alcohol sclerosing injections were trialed successively before surgical neurectomy. The direct-to-surgery approach was compared with the three different selective injection strategies and with a no-treatment strategy in a decision-analytic model for a hypothetical group of patients with symptomatic Morton neuroma in whom conservative management had failed. Model parameters, including treatment costs, effectiveness, complication rates, and health utility states, were estimated from the literature, reimbursement databases, and expert opinion. The outcome was cost per quality-adjusted life year (QALY) with a time horizon of 3 years. A societal cost perspective was adopted with a willingness-to-pay threshold of $100,000/QALY. Sensitivity analyses for key model parameters were performed. RESULTS. For the base input values, the steroid/alcohol selective injection strategy was dominant and yielded an incremental cost-effectiveness ratio of $4401.61/QALY compared with no treatment. The probabilistic sensitivity analysis supported this strategy in 74% of 10,000 simulated trials. Results were robust with low sensitivity to most input parameters. However, when the probability of successful alcohol injection treatment dropped below 40%, the steroid selective injection strategy became most cost-effective. CONCLUSION. A trial of ultrasound-guided injection therapies for Morton neuroma is a cost-effective strategy compared with proceeding directly to surgical neurectomy. CLINICAL IMPACT. Ultrasound-guided injection therapies are indicated as first-line treatment of patients with symptomatic Morton neuromas when conservative management fails.

Thrombectomy is a cost-saving procedure up to 24 h after onset.

INTRODUCTION AND AIM: The treatment of ischemic stroke due to large-vessel occlusion has been revolutionized by mechanical thrombectomy (MT), as multiple trials have consistently shown improved functional outcomes compared to standard medical management both in the early and late time windows after symptom onset. However, MT is an interventional procedure that is more costly than best supportive care (BSC). METHODS: We set out to study the cost-utility and budget impact of MT + BSC versus BSC alone for large-vessel occlusion using a combined decision tree and Markov model. The analysis was conducted from a Belgian payer perspective over a lifetime horizon, and health states were defined by the modified Rankin Scale (mRS). The treatment effect of MT + BSC combined clinical outcomes from all published early and late treatment window studies showing improved mRS after 90 days. Resource use and utilities were informed by an observational Belgian study of 569 stroke patients. Long-term mRS transitions were sourced from the Oxford Vascular study. RESULTS: MT + BSC generated 1.31 additional quality-adjusted life years and resulted in cost savings of €10,216 per patient over lifetime. Deterministic sensitivity analyses demonstrated dominance of MT over a wide range of parameter inputs. In a Belgian setting, adding MT to BSC within an early time window for 1575 eligible stroke patients every year produced cost savings between €6.3 million (year 1) and €14.6 million (year 5), or a total cost saving of €56.2 million over 5 years. CONCLUSION: Mechanical thrombectomy is a highly cost-effective treatment for ischemic stroke patients, providing quality-adjusted survival at lower health care cost, both when given in an early time window, as well as in a late time window.

"Now I Feel a Little Bit More Secure": The Impact of SNAP Enrollment on Older Adult SSI Recipients.

In June 2019, California expanded Supplemental Nutrition Assistance Program (SNAP) eligibility to Supplemental Security Income (SSI) beneficiaries for the first time. This research assesses the experience and impact of new SNAP enrollment among older adult SSI recipients, a population characterized by social and economic precarity. We conducted semi-structured, in-depth interviews with 20 SNAP participants to explore their experiences with new SNAP benefits. Following initial coding, member-check groups allowed for participants to provide feedback on preliminary data analysis. Findings demonstrate that SNAP enrollment improved participants' access to nutritious foods of their choice, contributed to overall budgets, eased mental distress resulting from poverty, and reduced labor spent accessing food. For some participants, SNAP benefit amounts were too low to make any noticeable impact. For many participants, SNAP receipt was associated with stigma, which some considered to be a social "cost" of poverty. Increased benefit may be derived from pairing SNAP with other public benefits. Together, the impacts of and barriers to effective use of SNAP benefits gleaned from this study deepen our understanding of individual- and neighborhood-level factors driving health inequities among low-income, disabled people experiencing food insecurity and SNAP recipients.

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.

How to Prevent Vaccines Falling Victim to Their Own Success: Intertemporal Dependency of Incidence Levels on Indirect Effects in Economic Reevaluations.

OBJECTIVES: Incremental cost-effectiveness analyses may inform the optimal choice of healthcare interventions. Nevertheless, for many vaccines, benefits fluctuate with incidence levels over time. Reevaluating a vaccine after it has successfully decreased incidences may eventually cause a disease resurgence if switching to a vaccine with lower indirect benefits. Decisions may successively alternate between vaccines alongside repeated rises and falls in incidence and when indirect effects from historic use are ignored. Our suggested proposal aims to prevent suboptimal decision making. METHODS: We used a conceptual model of demand to illustrate alternating decisions between vaccines because of time-varying levels of indirect effects. Similar to the concept of subsidies, we propose internalizing the indirect effects achievable with vaccines. In a case study over 60 years, we simulated a hypothetical 10-year reevaluation of 2 oncogenic human papillomavirus vaccines, of which only 1 protects additionally against anogenital warts. RESULTS: Our case study showed that the vaccine with additional warts protection is initially valued higher than the vaccine without additional warts protection. After 10 years, this differential decreases because of declines in warts incidence, which supports switching to the nonwarts vaccine that causes a warts resurgence eventually. Instead, pricing the indirect effects separately supports continuing with the warts vaccine. CONCLUSIONS: Ignoring how the observed incidences depend on the indirect effects achieved with a particular vaccine may lead to repeated changes in vaccines at successive reevaluations, with unintended resurgences, economic inefficiencies, and eroding vaccine confidence. We propose internalizing indirect effects to prevent vaccines falling victim to their own success.

Estimating the Theoretical Cost Implications of Funding New Drugs Considered Not to Be Cost-Effective.

This study aims to estimate the theoretical excess expenditure that would be incurred by the Irish state-payer, should drugs be reimbursed at their original asking ("list") price rather than at a price at which the drug is considered cost-effective. In Ireland, all new drugs are evaluated by the National Centre for Pharmacoeconomics. For this study, drugs that were submitted by pharmaceutical companies from 2012 to 2017 and considered not cost-effective at list price were reviewed. A total of 43 such drugs met our inclusion criteria, and their pharmacoeconomic evaluations were further assessed. The price at which the drug could be considered cost-effective (cost-effective price) at the upper cost-effectiveness threshold used in Ireland (€ 45 000/quality adjusted life-year) was estimated for 18 drugs with an available cost-effectiveness model. Then, for each drug, the list price and cost-effective price (both per unit) were both individually applied to 1 year of national real-world drug utilization data. This allowed the estimation of the expected expenditures under the assumptions of list price paid and cost-effective price paid. The resulting theoretical excess expenditure, the expenditure at list price minus the expenditure at the cost-effective price, was estimated to be €108.2 million. This estimate is theoretical because of the confidentiality of actual drug prices. The estimation is calculated using the list price and likely overestimates the actual excess expenditure, which would reduce to zero if cost-effective prices are agreed. Nevertheless, this estimate illustrates the importance of a process to assess the value of new drugs so that potential excess drug expenditure is identified.

Cost-Effectiveness of a Serum Biomarker Test for Risk-Stratified Liver Ultrasound Screening for Hepatocellular Carcinoma.

OBJECTIVES: Risk-stratified ultrasound screening for hepatocellular carcinoma (HCC), informed by a serum biomarker test, enables resources to be targeted to patients at the highest risk of developing cancer. We aimed to investigate the cost-effectiveness of risk-stratified screening for HCC in the Australian healthcare system. METHODS: A Markov cohort model was constructed to test 3 scenarios for patients with compensated cirrhosis: (1) risk-stratified screening for high-risk patients, (2) all-inclusive screening, and (3) no formal screening. Probabilistic sensitivity analyses were undertaken to determine the impact of uncertainty. Scenario analyses were used to assess cost-effectiveness in Australia's Aboriginal and Torres Strait Islander peoples and to determine the impact of including productivity-related costs of mortality. RESULTS: Both risk-stratified screening and all-inclusive screening programs were cost-effective compared with no formal screening, with incremental cost-effectiveness ratios of A$39 045 and A$23 090 per quality-adjusted life-year (QALY), respectively. All-inclusive screening had an incremental cost-effectiveness ratio of A$4453 compared with risk-stratified screening and had the highest probability of being cost-effective at a willingness-to-pay (WTP) threshold of A$50 000 per QALY. Risk-stratified screening had the highest likelihood of cost-effectiveness when the WTP was between A$25 000 and A$35 000 per QALY. Cost-effectiveness results were further strengthened when applied to an Aboriginal and Torres Strait Islander cohort and when productivity costs were included. CONCLUSIONS: Cirrhosis population-wide screening for HCC is likely to be cost-effective in Australia. Risk-stratified screening using a serum biomarker test may be cost-effective at lower WTP thresholds.

Good Days and Bad Days: Measuring Health-Related Quality of Life in People With Epilepsy.

OBJECTIVES: Cost-effectiveness analyses typically require measurement of health-related quality of life (HRQoL) to estimate quality-adjusted life-years. Challenges with measuring HRQoL arise in the context of episodic conditions if patients are less likely-or even unable-to complete surveys when having disease symptoms. This article explored whether HRQoL measured at regular time intervals adequately reflects the HRQoL of people with epilepsy (PWE). METHODS: Follow-up data from the Epilepsy Support Dog Evaluation study on the (cost-)effectiveness of seizure dogs were used in which HRQoL is measured in 25 PWE with the EQ-5D at baseline and every 3 months thereafter. Seizure count is recorded daily using a seizure diary. Regression models were employed to explore whether PWE were more likely to complete the HRQoL survey on a good day (ie, when seizures are absent or low in frequency compared with other days) and to provide an estimate of the impact of reporting HRQoL on a good day on EQ-5D utility scores. RESULTS: A total of 111 HRQoL measurements were included in the analyses. Regression analyses indicated that the day of reporting HRQoL was associated with a lower seizure count (P<.05) and that a lower seizure count was associated with a higher EQ-5D utility score (P<.05). CONCLUSIONS: When HRQoL is measured at regular time intervals, PWE seem more likely to complete these surveys on good days. Consequently, HRQoL might be overestimated in this population. This could lead to underestimation of the effectiveness of treatment and to biased estimates of cost-effectiveness.

Deriving a Preference-Based Measure for People With Duchenne Muscular Dystrophy From the DMD-QoL.

OBJECTIVES: This study generates a preference-based measure for capturing the quality of life of people with Duchenne muscular dystrophy (DMD) from a new measure of quality of life, DMD-QoL. METHODS: A health state classification system was derived from the DMD-QoL based on psychometric performance of items, factor analysis, and item response theory analysis. Preferences for health states described by the classification system were elicited using an online discrete choice experiment survey with life years as an additional attribute, from members of the UK general population (n = 1043). Discrete choice experiment data was modeled using a conditional fixed-effects logit model and utility estimates were directly anchored on the 1 to 0 full health-dead scale. RESULTS: The health state classification system has 8 dimensions: mobility, difficulty using hands, difficulty breathing, pain, tiredness, worry, participation, and feeling good about yourself. The standard model had mostly statistically significant coefficients and reflected the instrument's monotonic structure. However, 2 dimensions had inconsistent coefficients (where utility increased as health worsened) and a consistent model was estimated that merged adjacent inconsistent severity levels. The best state defined by the classification system has a value of 1 and the worst state has a value of -0.559. CONCLUSION: The modeled results enable DMD-QoL-8D utility values to be generated using DMD-QoL or DMD-QoL-8D data to generate QALYs for people with DMD. QALYs can then be used to inform economic models of the cost-effectiveness of interventions in DMD. Future research comparing the psychometric performance of DMD-QoL-8D to existing generic preference-based measures, including EQ-5D-5L, is recommended.

Cost-Effectiveness of Medicinal Cannabis for Management of Refractory Symptoms Associated With Chronic Conditions: A Systematic Review of Economic Evaluations.

OBJECTIVES: Although there is a growing body of evidence suggesting that cannabinoids may relieve symptoms of some illnesses, they are relatively high-cost therapies compared with illicit growth and supply. This article aimed to comprehensively review economic evaluations of medicinal cannabis for alleviating refractory symptoms associated with chronic conditions. METHODS: Seven electronic databases were searched for articles published up to September 6, 2020. The quality of reporting of economic evaluations was assessed using the Consolidated Health Economic Evaluation Reporting Standards checklist. The extracted data were grouped into subcategories according to types of medical conditions, organized into tables, and reported narratively. RESULTS: This review identified 12 cost-utility analyses conducted across a variety of diseases including multiple sclerosis (MS) (N = 8), pediatric drug-resistant epilepsies (N = 2), and chronic pain (N = 2). The incremental cost-effectiveness ratio varied widely from cost saving to more than US$451 800 per quality-adjusted life-year depending on the setting, perspectives, types of medicinal cannabis, and indications. Nabiximols is a cost-effective intervention for MS spasticity in multiple European settings. Cannabidiol was found to be a cost-effective for Dravet syndrome in a Canadian setting whereas a cost-utility analysis conducted in a US setting deemed cannabidiol to be not cost-effective for Lennox-Gastaut syndrome. Overall study quality was good, with publications meeting 70% to 100% (median 83%) of the Consolidated Health Economic Evaluation Reporting Standards checklist criteria. CONCLUSIONS: Medicinal cannabis-based products may be cost-effective treatment options for MS spasticity, Dravet syndrome, and neuropathic pain, although the literature is nascent. Well-designed clinical trials and health economic evaluations are needed to generate adequate clinical and cost-effectiveness evidence to assist in resource allocation.

Evaluation of the Cost-effectiveness of Drug Treatment for Alzheimer Disease in a Simulation Model That Includes Caregiver and Societal Factors.

Importance: The possibility of widespread use of a novel effective therapy for Alzheimer disease (AD) will present important clinical, policy, and financial challenges. Objective: To describe how including different patient, caregiver, and societal treatment-related factors affects estimates of the cost-effectiveness of a hypothetical disease-modifying AD treatment. Design, Setting, and Participants: In this economic evaluation, the Alzheimer Disease Archimedes Condition Event Simulator was used to simulate the prognosis of a hypothetical cohort of patients selected from the Alzheimer Disease Neuroimaging Initiative database who received the diagnosis of mild cognitive impairment (MCI). Scenario analyses that varied costs and quality of life inputs relevant to patients and caregivers were conducted. The analysis was designed and conducted from June 15, 2019, to September 30, 2020. Exposures: A hypothetical drug that would delay progression to dementia in individuals with MCI compared with usual care. Main Outcomes and Measures: Incremental cost-effectiveness ratio (ICER), measured by cost per quality-adjusted life-year (QALY) gained. Results: The model included a simulated cohort of patients who scored between 24 and 30 on the Mini-Mental State Examination and had a global Clinical Dementia Rating scale of 0.5, with a required memory box score of 0.5 or higher, at baseline. Using a health care sector perspective, which included only individual patient health care costs, the ICER for the hypothetical treatment was $192 000 per QALY gained. The result decreased to $183 000 per QALY gained in a traditional societal perspective analysis with the inclusion of patient non-health care costs. The inclusion of estimated caregiver health care costs produced almost no change in the ICER, but the inclusion of QALYs gained by caregivers led to a substantial reduction in the ICER for the hypothetical treatment, to $107 000 per QALY gained in the health sector perspective. In the societal perspective scenario, with the broadest inclusion of patient and caregiver factors, the ICER decreased to $74 000 per added QALY. Conclusions and Relevance: The findings of this economic evaluation suggest that policy makers should be aware that efforts to estimate and include the effects of AD treatments outside those on patients themselves can affect the results of the cost-effectiveness analyses that often underpin assessments of the value of new treatments. Further research and debate on including these factors in assessments that will inform discussions on fair pricing for new treatments are needed.

Comparison of Cap-Assisted vs Conventional Endoscopic Technique for Management of Food Bolus Impaction in the Esophagus: Results of a Multicenter Randomized Controlled Trial.

INTRODUCTION: "Push" or "pull" techniques with the use of snares, forceps, baskets, and grasping devices are conventionally used to manage esophageal food bolus impaction (FBI). A novel cap-assisted technique has recently been advocated to reduce time taken for food bolus (FB) removal. This study aimed to compare the effectiveness of the cap-assisted technique against conventional methods of esophageal FB removal in a randomized controlled trial. METHODS: Consecutive patients with esophageal FBI requiring endoscopic removal, from 3 Australian tertiary hospitals between 2017 and 2019, were randomized to either the cap-assisted technique or the conventional technique. Primary outcomes were technical success and FB retrieval time. Secondary outcomes were technical success rate, en bloc removal rate, procedure-related complication, length of hospital stay, and cost of consumables. RESULTS: Over 24 months, 342 patients with esophageal FBI were randomized to a cap-assisted (n = 171) or conventional (n = 171) technique. Compared with the conventional approach, the cap-assisted technique was associated with (i) shorter FB retrieval time (4.5 ± 0.5 minutes vs 21.7 ± 0.9 minutes, P < 0.001), (ii) shorter total procedure time (23.0 ± 0.6 minutes vs 47.0 ± 1.3 minutes, P < 0.0001), (iii) higher technical success rate (170/171 vs 160/171, P < 0.001), (iv) higher rate of en bloc removal (159/171 vs 48/171, P < 0.001), and (v) lower rate of procedure-related mucosal tear and bleeding (0/171 vs 13/171, P < 0.001). There were no major adverse events or deaths within 30 days in either group. The total cost of consumables was higher in the conventional group (A$19,644.90 vs A$6,239.90). DISCUSSION: This multicenter randomized controlled trial confirmed that the cap-assisted technique is more effective and less costly than the conventional approach and should be first-line treatment for esophageal FBI.

Comparison of Levonorgestrel-releasing Intrauterine System (LNG-IUS) against Laparoscopic Assisted Supracervical Hysterectomy (LASH) for menorrhagia treatment: An economic evaluation.

BACKGROUND: This economic evaluation and literature review was conducted with the primary aim to compare the cost-effectiveness of laparoscopic assisted supracervical hysterectomy (LASH) with NICE's gold-standard treatment of Levonorgestrel-releasing intrauterine system (LNG-IUS) for menorrhagia. MATERIALS AND METHODS: A cost-utility analysis was conducted from an NHS perspective, using data from two European studies to compare the treatments. Individual costs and benefits were assessed within one year of having the intervention. An Incremental Cost-Effectiveness Ratio (ICER) was calculated, followed by sensitivity analysis. Expected Quality Adjusted Life Years (QALYS) and costs to the NHS were calculated alongside health net benefits (HNB) and monetary net benefits (MNB). RESULTS: A QALY gain of 0.069 was seen in use of LNG-IUS compared to LASH. This yielded a MNB between -£44.99 and -£734.99, alongside a HNB between -0.0705 QALYs and -0.106 QALYS. Using a £20,000-£30,000/QALY limit outlined by NICE,this showed the LNG-IUS to be more cost-effective than LASH, with LASH exceeding the upper bound of the £30,000/QALY limit. Sensitivity analysis lowered the ICER below the given threshold. CONCLUSIONS: The ICER demonstrates it would not be cost-effective to replace the current gold-standard LNG-IUS with LASH, when treating menorrhagia in the UK. The ICER's proximity to the threshold and its high sensitivity alludes to the necessity for further research to generate a more reliable cost-effectiveness estimate. However, LASH could be considered as a first line treatment option in women with no desire to have children.

Whole Grain Intakes Are Associated with Healthcare Cost Savings Following Reductions in Risk of Colorectal Cancer and Total Cancer Mortality in Australia: A Cost-of-Illness Model.

Whole grain consumption has been associated with the reduced risk of several chronic diseases with significant healthcare monetary burden, including cancer. Colorectal cancer (CRC) is one of the most common cancers globally, with the highest rates reported in Australia. Three servings of whole grains provide a 15% reduction in total cancer and 17% reduction in CRC risk; however, 70% of Australians fall short of this level of intake. The aim of this study was to assess the potential savings in healthcare costs associated with reductions in the relative risk of CRC and total cancer mortality following the whole grain Daily Target Intake (DTI) of 48 g in Australia. A three-step cost-of-illness analysis was conducted using input parameters from: (1) estimates of current and targeted whole grain intakes among proportions (5%, 15%, 50%, and 100%) of the Australian adult (≥20 years) population; (2) estimates of reductions in relative risk (with 95% confidence intervals) of CRC and total cancer mortality associated with specific whole grain intake from meta-analysis studies; and (3) estimates of annual healthcare costs of CRC and all cancers from disease expenditure national databases. A very pessimistic (5% of population) through to universal (100% of population) adoption of the recommended DTI in Australia were shown to potentially yield savings in annual healthcare costs equal to AUD 1.9 (95% CI 1.2-2.4) to AUD 37.2 (95% CI 24.1-48.1) million for CRC and AUD 20.3 (95% CI 12.2-27.0) to AUD 405.1 (95% CI 243.1-540.1) million for total cancers. As treatment costs for CRC and other cancers are increasing, and dietary measures exchanging whole grains for refined grains are not cost preclusive nor does the approach increase energy intake, there is an opportunity to facilitate cost-savings along with reductions in disease for Australia. These results suggest specific benefits of encouraging Australians to swap refined grains for whole grains, with greater overall adherence to suggestions in dietary guidelines.