COVID-19 Vaccines Cost-Effectiveness Analysis: A Scenario for Iran

COVID-19 vaccines are supposed to be critical measure for ending the pandemic. Governments had to decide on the type of vaccine to provide for their population. In this decision-making process, cost-effectiveness analysis is considered a helpful tool. This study is a cost-effectiveness analysis utilized to calculate the incremental cost per averted disability-adjusted life year (DALY) by vaccination compared to no vaccination for different COVID-19 vaccines. The incremental cost-effectiveness ratio (ICER) for a vaccination with COVID-19 vaccines was estimated at 6.2 to 121.2 USD to avert one DALY and 566.8 to 10,957.7 USD per one death. The lowest and highest ICERs belong to Ad26.COV2.S and CoronaVac, respectively. Considering the scenario of Iran, vaccines that are recommended include ad26.cov2.s, chadox1-S, rAd26-S + rAd5-S, and BNT162b2 in the order of recommendation.

PP452 Impact Of Patient Access Schemes On Health Technology Assessment Agency Guidance For Rare Diseases In England and Scotland

IntroductionPatient access schemes (PAS) are agreements that may enable patients to access drugs or other treatments that may not be cost effective under normal circumstances. The aim of this study was to determine whether the use of PAS by the National Institute for Health and Care Excellence (NICE) and Scottish Medicines Consortium (SMC) for recommended drugs can lead to greater access to medications for rare diseases.MethodsReimbursement data for rare diseases between 2004 and 2021 from health technology assessment (HTA) agencies, namely the SMC (Scotland) and NICE (England), were included. The reviews with positive HTA decisions were considered, while those with negative decisions were excluded. Several observations were made from these data and reported.ResultsAmong the total positive reviews (n = 81), 43 included PAS. The inclusion of PAS in manufacturer submissions was more frequent for NICE than for the SMC (79% and 40% percent, respectively). Most of the drugs with PAS were included in the HTA guidance from both agencies. The positive NICE reviews contingent on PAS consisted of 20 drugs. For the same set of drugs, the SMC recommended 14 with PAS and one without PAS; five drugs were not assessed. Adalimumab was recommended by NICE with a PAS (base-case incremental cost-effectiveness ratio of GBP12,336 [EUR14,256]; GBP13,676 [EUR15,804]) and by the SMC without a PAS (base-case incremental cost-effectiveness ratio of GBP22,519 [EUR26,023]). Hence, without a PAS, the drug was costlier per quality-adjusted life-year for the National Health Service (NHS) Scotland.ConclusionsPAS submissions for rare diseases are more frequent for NICE than for the SMC. With the PAS discounts, the overall cost of the drugs is reduced, resulting in cost effectiveness. The SMC approved some drugs for which NICE required a PAS to improve the economic argument. Hence, the use of PAS for these drugs could lead to potential cost-savings to the NHS Scotland.

Cost-effectiveness analysis of patent foramen ovale closure versus medical therapy alone after cryptogenic stroke.

Background Closure of a patent foramen ovale reduces the risk of recurrent stroke compared with medical therapy alone in young patients with cryptogenic strokes revealed by randomized control trials. Some cost-effectiveness analyses outside Japan have shown that patent foramen ovale closure is cost-effective, but no studies have examined cost-effectiveness in Japan. The objective of this study is to assess cost-effectiveness, from the perspective of a Japanese healthcare payer, of patent foramen ovale closure versus medical therapy alone for the patients with patent foramen ovale related cryptogenic strokes. Methods A cost-effectiveness study was conducted by developing a decision tree and a Markov model. Probabilities and a 5.9-year time horizon followed the RESPECT study. Utilities and costs were based upon published studies and assumptions. The model cycle was one month. All assumptions were assessed by experts, including a cardiologist and a statistical expert. The target population comprised patients with cryptogenic stroke and patent foramen ovale, aged 60 years or younger. Incremental cost-effectiveness ratio was evaluated. Then one-way sensitivity analyses and probabilistic sensitivity analyses were conducted to assess robustness. Results Incremental cost-effectiveness ratio of patent foramen ovale closure compared with medical therapy was estimated at 3,318,152 yen per quality-adjusted life years gained. One-way sensitivity analysis showed that the stable state utility score difference between patent foramen ovale closure and medical therapy had the largest impact on incremental cost-effectiveness ratio. Patent foramen ovale closure is cost-effective at a stable state utility score difference of >0.051, compared with medical therapy. Probabilistic sensitivity analyses demonstrated that patent foramen ovale closure was 50.3% cost-effective with a willingness-to-pay threshold of 5,000,000 yen / quality-adjusted life years. Conclusions From a healthcare payer perspective, patent foramen ovale closure may be cost-effective compared with medical therapy for Japanese patients with cryptogenic stroke who were under 60 years.

Real-world cost-effectiveness of denosumab for the treatment of postmenopausal osteoporosis in Taiwan

Summary This study assessed the cost-effectiveness of continued denosumab treatment, compared with discontinuation of denosumab after one dose, for the treatment of postmenopausal osteoporosis in Taiwan, using real-world fracture reduction effectiveness and cost data. Outcomes indicate that continued denosumab treatment produces an incremental cost-effectiveness ratio of USD $16,743 per QALY. Purpose To evaluate the cost-effectiveness of continued denosumab use versus discontinuation after one dose, for the treatment of postmenopausal osteoporosis in Taiwan, using real-world fracture reduction effectiveness and cost data. Methods A Markov cohort model was used to evaluate the lifetime costs and QALYs associated with continued denosumab treatment versus discontinuation of treatment after one dose. The evaluation was conducted from the perspective of Taiwan’s healthcare system and used a discount rate of 3% per annum. The patient population consisted of postmenopausal women with osteoporosis with a mean age of 77 years who initiated denosumab treatment. Fracture reduction effectiveness data, baseline fracture rates, mortality data, and costs of fracture were informed by Taiwan’s National Health Insurance Research Database. Results Model outcomes showed that continued treatment with denosumab produced an expected gain of 0.042 QALYs and an incremental cost of USD $704, compared with discontinuation of denosumab after one dose. This corresponds to an incremental cost-effectiveness ratio of USD $16,743 per QALY gained. Probabilistic and scenario analysis showed that results are stable to variations in model assumptions and parameters. Conclusion In a real-world setting, at a cost per QALY threshold equivalent to gross domestic product per capita in 2020 in Taiwan (USD $30,038), continued treatment with denosumab in postmenopausal women with osteoporosis is cost-effective compared with treatment discontinuation.

Evaluation of an Alternative Learning System for youths at risk of involvement in urban violence in the Philippines

Background Globally, violence disproportionately affects young people, leading to injury, hospitalisation, death, social dysfunction, and poor mental wellbeing. Moreover, it has far-reaching economic consequences for whole nations, due to loss of productivity. Research suggests that attaining a higher level of education promotes factors that insulate youths from poverty and violence. Purpose In this study, we investigated the outcomes, the cost, and the cost-effectiveness of a non-formal education program with an additional psychosocial component. The short-term outcome measure was an increase in educational attainment, a crucial step for youth empowerment. The program analysed was the Alternative Learning System (ALS) offered by the Balay Rehabiliation Centre in Bagong Silang, an urban slum in Manila, which targeted out of school youth. Methods The cost-effectiveness analysis of ALS compared to a ‘do nothing approach’ was performed from the perspective of the service provider. The study sample comprised 239 learners who were enrolled in the ALS during 2015–2018. For the ‘do nothing’ comparator, a counterfactual scenario was hypothesised. The average cost of the intervention per enrolled learner, and the incremental cost effectiveness ratio (ICER) for passing the Accreditation and Evaluation (A&E) exam at elementary or secondary level, were calculated. Results The ALS intervention studied resulted in 41% (n = 97) of the learners passing the examination over a period of four years (from 2015–2018). The estimated total cost of the intervention was $371,110, corresponding to $1550 per enrolled learner. The incremental cost-effectiveness ratio for a pass in the exam was found to be $3830. Compared to other, international, alternative learning interventions, the ALS intervention as used in Bagong Silang was found to be more cost-effective. Conclusion From the service provider perspective, the ALS for out-of-school young people was found to be a valuable investment to benefit poor young people living in slums in Manila.

Economic Evaluation of Regular Transfusions for Cerebral Infarct Recurrence in the Silent Cerebral Infarct Transfusion Trial

In 2020, the American Society of Hematology published evidence-based guidelines for cerebrovascular disease in individuals with sickle cell anemia (SCA). While guidelines were based on NIH-sponsored randomized controlled trials, no cost-effectiveness analysis was completed for children with SCA and silent cerebral infarcts. We conducted a cost-effectiveness analysis comparing regular blood transfusion versus standard care using Silent Cerebral Infarct Transfusion (SIT) Trial participants. This analysis included a modified societal perspective with direct costs (hospitalization, emergency room visit, transfusion, outpatient care, iron chelation) and indirect costs (special education). Direct medical costs were estimated from hospitalizations from SIT hospitals and unlinked aggregated hospital and outpatient costs from SIT sites using the Pediatric Health Information System. Indirect costs were estimated from published literature. Effectiveness was prevention of infarct recurrence. Incremental cost-effectiveness ratio using a 3-year time horizon (mean SIT trial participant follow-up) compared transfusion versus standard care. A total of 196 participants received transfusions (N=90) or standard care (N=106), with a mean age of 10.0 years. Annual hospitalization costs were reduced by 54% for transfusions than standard care ($4,929 vs. $10,802), but transfusion group outpatient costs added $22,454 to $137,022 per year. Special education costs savings was $2,634 over three years for every infarct prevented. Transfusion therapy had an incremental cost-effectiveness ratio of $22,025 per infarct prevented. Children with pre-existing silent cerebral infarcts receiving blood transfusions have lower hospitalization but higher outpatient costs, primarily associated with oral iron chelator deferasirox. Regular blood transfusion therapy is cost-effective for infarct recurrence in children with SCA. This trial is registered at www.clinicaltrials.gov as NCT00072761.

Cost-Effectiveness of The Healthy Options Group Psychosocial Intervention for Perinatal Women Living with HIV and Depression in Tanzania

BackgroundHealthy Options (HO) is a psychosocial support group intervention facilitated by community-based health workers (CBHWs) to treat depression in perinatal women living with HIV in Tanzania. The objective of this study was to determine incremental cost-effectiveness of HO in treating perinatal depression in HIV positive women in comparison to enhanced standard of mental health care delivered by nurse midwives.MethodsThis study is a cost-effectiveness analysis of HO, a pair-matched cluster-randomized controlled trial conducted in Tanzania. The primary outcome for this study was the level of depressive symptoms. Secondary outcomes included antiretroviral therapy adherence, safe disclosure of HIV status to partner, and food security, among others. This study applied an ingredients approach to cost all resources used in the HO intervention. We estimated total cost, unit cost and incremental cost-effectiveness ratio from a health care provider perspective. We used 3 year time horizon, univariate sensitivity analysis, and adjusted costs to 2017 value.ResultsHO demonstrated effectiveness in reducing depressive symptoms among pregnant women with HIV in Tanzania. Mean reduction in depressive symptoms was observed at 6 weeks and 9 months postpartum with statistical significance. HO costs $849 USD per woman treated (equivalent to $889 in 2017 value). Incremental cost-effectiveness ratio at 6 weeks postpartum is $81,091 per mean decrease in depressive symptoms (equivalent to $84,878 in 2017 value), and at 9 months postpartum is $280,277 per mean decrease in depressive symptoms (equivalent to $293,363 in 2017 value). With sensitivity analysis, results at 6 weeks postpartum ranged from $79,309 to $84,576 (equivalent to $83,012 to $88,525 in 2017 value), and at 9 months ranged from $ 194,001 to $1,139,546 (equivalent to $203,067 to $1,192,753 in 2017 value).ConclusionsBenefits of HO are significantly sustained through 9 months postpartum and intervention unit costs are within range of similar interventions. Human resources composed the largest share of HO total cost. Although uncertainty in the cost-effectiveness ratio of this intervention varied widely, since mental health specialists are scarce in Tanzania, working with CBHWs is likely to offer effective treatment that is more feasible and is a less costly alternative to the usual care.Trial registration: The trial is registered at Clinicaltrials.gov (NCT02039973).

Cost‐Utility of Catheter Ablation for Atrial Fibrillation in Patients with Heart Failure: An Economic Evaluation

Background Recent trials comparing catheter ablation to medical therapy in patients with heart failure (HF) with symptomatic atrial fibrillation despite first‐line management have demonstrated a reduction in adverse outcomes. We performed an economic evaluation to estimate the cost‐utility of catheter ablation as second line therapy in patients with HF with reduced ejection fraction. Methods and Results A Markov model with health states of alive, dead, and alive with amiodarone toxicity was constructed, using the perspective of the Canadian healthcare payer. Patients in the alive states were at risk of HF and non‐HF hospitalizations. Parameters were obtained from randomized trials and Alberta health system data for costs and outcomes. A lifetime time horizon was adopted, with discounting at 3.0% annually. Probabilistic and 1‐way sensitivity analyses were performed. Costs are reported in 2018 Canadian dollars. A patient treated with catheter ablation experienced lifetime costs of $64 960 and 5.63 quality‐adjusted life‐years (QALY), compared with $49 865 and 5.18 QALYs for medical treatment. The incremental cost‐effectiveness ratio was $35 360/QALY (95% CI, $21 518–77 419), with a 90% chance of being cost‐effective at a willingness‐to‐pay threshold of $50 000/QALY. A minimum mortality reduction of 28%, or a minimum duration of benefit of >1 to 2 years was required for catheter ablation to be attractive at this threshold. Conclusions Catheter ablation is likely to be cost‐effective as a second line intervention for patients with HF with symptomatic atrial fibrillation, with incremental cost‐effectiveness ratio $35 360/QALY, as long as over half of the relative mortality benefit observed in extant trials is borne out in future studies.

Clinical and economic impact of current ALK rearrangement testing in Spain compared with a hypothetical no-testing scenario

Background Currently biomarkers play an essential role in diagnosis, treatment, and management of cancer. In non-small cell lung cancer (NSCLC) determination of biomarkers such as ALK, EGFR, ROS1 or PD-L1 is mandatory for an adequate treatment decision. The aim of this study is to determine the clinical and economic impact of current anaplastic lymphoma kinase testing scenario in Spain. Methods A joint model, composed by decision-tree and Markov models, was developed to estimate the long-term health outcomes and costs of NSCLC patients, by comparing the current testing scenario for ALK in Spain vs a hypothetical no-testing. The current distribution of testing strategies for ALK determination and their sensitivity and specificity data were obtained from the literature. Treatment allocation based on the molecular testing result were defined by a panel of Spanish experts. To assess long-term effects of each treatment, 3-states Markov models were developed, where progression-free survival and overall survival curves were extrapolated using exponential models. Medical direct costs (expressed in €, 2019) were included. A lifetime horizon was used and a discount rate of 3% was applied for both costs and health effects. Several sensitivity analyses, both deterministic and probabilistic, were performed in order test the robustness of the analysis. Results We estimated a target population of 7628 NSCLC patients, including those with non-squamous histology and those with squamous carcinomas who were never smokers. Over the lifetime horizon, the current ALK testing scenario produced additional 5060 and 3906 life-years and quality-adjusted life-years (QALY), respectively, compared with the no-testing scenario. Total direct costs were increased up to € 51,319,053 for testing scenario. The incremental cost-effectiveness ratio was 10,142 €/QALY. The sensitivity analyses carried out confirmed the robustness of the base-case results, being the treatment allocation and the test accuracy (sensitivity and specificity data) the key drivers of the model. Conclusions ALK testing in advanced NSCLC patients, non-squamous and never-smoker squamous, provides more than 3000 QALYs in Spain over a lifetime horizon. Comparing this gain in health outcomes with the incremental costs, the resulting incremental cost-effectiveness ratio reinforces that testing non-squamous and never-smoker squamous NSCLC is a cost-effective strategy in Spain.

Cost-Effectiveness Analysis of Dabrafenib Plus Trametinib and Vemurafenib as First-Line Treatment in Patients with BRAF V600 Mutation-Positive Unresectable or Metastatic Melanoma in China

Objective: To evaluate the cost-effectiveness of dabrafenib plus trametinib combination therapy versus vemurafenib as first-line treatment in patients with BRAF V600 mutation-positive unresectable or metastatic melanoma from a healthcare system perspective in China. Methods: This study employed a partitioned survival model with three health states (progression-free survival, post-progression survival and dead) to parameterize the data derived from Combi-v trial and extrapolated to 30 years. Health states’ utilities were measured by EQ-5D-3L, also sourced from the Combi-v trial. Costs including drug acquisition costs, disease management costs and adverse event costs were based on the Chinese Drug Bidding Database and physician survey in China. The primary outcomes of the model were lifetime costs, life-years (LYs), quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratio (ICER). Deterministic and probabilistic sensitivity analyses were conducted, respectively. Result: Dabrafenib plus trametinib is projected to increase a patient’s life expectancy by 0.95 life-years over vemurafenib (3.03 vs. 2.08) and 1.09 QALY gains (2.48 vs. 1.39) with an incremental cost of $3833. The incremental cost-effectiveness ratio (ICER) was $3511 per QALY. In the probabilistic sensitivity analyses, at a threshold of $33,357 per QALY (three times the gross domestic product (GDP) per capita in China in 2020), the probability of dabrafenib plus trametinib being cost-effective was 90%. In the deterministic sensitivity analyses, the results were most sensitive to the dabrafenib plus trametinib drug costs, vemurafenib drug costs and discount rate of cost. Conclusion: Dabrafenib plus trametinib therapy yields more clinical benefits than vemurafenib. Using a threshold of $33,357 per QALY, dabrafenib plus trametinib is very cost-effective as compared with vemurafenib in China.