Incremental Cost-effectiveness Ratio Threshold Research Articles

Information and Communications Technology-Based Monitoring Service for Tailored Chronic Disease Management in Primary Care: Cost-Effectiveness Analysis Based on ICT-CM Trial Results.

Information and communications technology-based tailored management (TM) intervention is a novel automatic system in which a smartphone app for the management of patients with hypertension and diabetes, the provider web, and Bluetooth devices are linked. However, little evidence exists regarding the cost-effectiveness of the interventions using mobile apps. This study aimed to assess the cost-effectiveness of TM intervention for adult patients with hypertension or diabetes in primary care compared with usual care (UC). Cost-effectiveness analysis using a Markov model was conducted from the Korean health care system perspective. Based on 6-month outcome data from an information and communications technology-based tailored chronic disease management (ICT-CM) trial, effectiveness over a lifetime beyond the trial periods was extrapolated using a cardiovascular disease risk prediction model. Costs were estimated using ICT-CM trial data and national health insurance claims data. Health utility weights were obtained from the Korea National Health and Nutrition Examination Survey. In the base-case analysis, compared with UC, TM was more costly (US $23,157 for TM vs US $22,391 for UC) and more effective (12.006 quality-adjusted life-years [QALYs] for TM vs 11.868 QALYs for UC). The incremental cost-effectiveness ratio was US $5556 per QALY gained. Probabilistic sensitivity analysis showed that the probability of TM being cost-effective compared with UC was approximately 97% at an incremental cost-effectiveness ratio threshold of US $26,515 (KRW 35 million) per QALY gained. Compared with UC, TM intervention is a cost-effective option for patients with hypertension or diabetes in primary care settings. The study results can assist policy makers in making evidence-based decisions when implementing accessible chronic disease management services.

Lifetime Cost-Effectiveness of Structured Education and Exercise Therapy for Knee Osteoarthritis in Australia.

Structured education and exercise therapy programs have been proposed to reduce reliance on total knee replacement (TKR) surgery and improve health care sustainability. The long-term cost-effectiveness of these programs is unclear. To estimate the lifetime cost-effectiveness of implementing a national structured education and exercise therapy program for individuals with knee osteoarthritis with the option for future TKR compared with usual care (TKR for all). This economic evaluation used a life table model in combination with a Markov model to compare costs and health outcomes of a national education and exercise therapy program vs usual care in the Australian health care system. Subgroup, deterministic, and probabilistic sensitivity analyses were completed. A hypothetical cohort of adults aged 45 to 84 years who would undergo TKR was created. Structured education and exercise therapy intervention provided by physiotherapists. The comparator was usual care where all people undergo TKR without accessing the program in the first year. Incremental net monetary benefit (INMB), with an incremental cost-effectiveness ratio threshold of 28 033 Australian dollars (A$) per quality-adjusted life-year (QALY) gained, was calculated from a health care perspective. Transition probabilities, costs, and utilities were estimated from national registries and a randomized clinical trial. The hypothetical cohort included 61 394 individuals (53.9% female; 93.6% aged ≥55 years). Implementation of an education and exercise therapy program resulted in a lifetime cost savings of A$498 307 942 (US $339 922 227), or A$7970 (US $5537) per individual, and resulted in fewer QALYs (0.43 per individual) compared with usual care. At a population level, education and exercise therapy was not cost-effective at the lifetime horizon (INMB, -A$4090 [-US $2841]). Subgroup analysis revealed that the intervention was cost-effective only for the first 9 years and over a lifetime only in individuals with no or mild pain at baseline (INMB, A$11 [US $8]). Results were robust to uncertainty around model inputs. In this economic evaluation of structured education and exercise therapy compared with usual care, the intervention was not cost-effective over the lifetime for all patients but was for the first 9 years and for those with minimal pain. These findings point to opportunities to invest early cost savings in additional care or prevention, including targeted implementation to specific subgroups.

The Economic Impact of Community Paramedics Within Emergency Medical Services: A Systematic Review.

Globally, emergency medical services (EMSs) report that their demand is dominated by non-emergency (such as urgent and primary care) requests. Appropriately managing these is a major challenge for EMSs, with one mechanism employed being specialist community paramedics. This review guides policy by evaluating the economic impact of specialist community paramedic models from a healthcare system perspective. A multidisciplinary team (health economics, emergency care, paramedicine, nursing) was formed, and a protocol registered on PROSPERO (CRD42023397840) and published open access. Eligible studies included experimental and analytical observational study designs of economic evaluation outcomes of patients requesting EMSs via an emergency telephone line ('000', '111', '999', '911' or equivalent) responded to by specialist community paramedics, compared to patients attended by usual care (i.e. standard paramedics). A three-stage systematic search was performed, including Peer Review of Electronic Search Strategies (PRESS) and Preferred Reporting Items for Systematic reviews and Meta-Analyses (PRISMA). Two independent reviewers extracted and verified 51 unique characteristics from 11 studies, costs were inflated and converted, and outcomes were synthesised with comparisons by model, population, education and reliability of findings. Eleven studies (n = 7136 intervention group) met thecriteria. These included one cost-utility analysis (measuring both costs and consequences), four costing studies (measuring cost only) and six cohort studies (measuring consequences only). Quality was measured using Joanna Briggs Institute tools, and was moderate for ten studies, and low for one. Models included autonomous paramedics (six studies, n = 4132 intervention), physician oversight (three studies, n = 932 intervention) and/or special populations (five studies, n = 3004 intervention). Twenty-one outcomes were reported. Models unanimously reduced emergency department (ED) transportation by 14-78% (higher quality studies reduced emergency department transportation by 50-54%, n = 2639 intervention, p < 0.001), and costs were reduced by AU$338-1227 per attendance in four studies (n = 2962). One study performed an economic evaluation (n = 1549), finding both that the costs were reduced by AU$454 per attendance (although not statistically significant), and consequently that the intervention dominated with a > 95% chance of the model being cost effective at the UK incremental cost-effectiveness ratio threshold. Community paramedic roles within EMSs reduced ED transportation by approximately half. However, the rate was highly variable owing to structural (such as local policies) and stochastic (such as the patient's medical condition) factors. As models unanimously reduced ED transportation-a major contributor to costs-they in turn lead to net healthcare system savings, provided there is sufficient demand to outweigh model costs and generate net savings. However, all models shift costs from EDs to EMSs, and therefore appropriate redistribution of benefits may be necessary to incentivise EMS investment. Policymakers for EMSs could consider negotiating with their health department, local ED or insurers to introduce a rebate for successful community paramedic non-ED-transportations. Following this, geographical areas with suitable non-emergency demand could be identified, and community paramedic models introduced and tested with a prospective economic evaluation or, where this is not feasible, with sufficient data collection to enable a post hoc analysis.

Cost-Effectiveness Analysis of Maternal Respiratory Syncytial Virus Vaccine in Protecting Infants from RSV Infection in Japan.

Respiratory syncytial virus (RSV) is one of the major causes of respiratory tract infections among children. Until recently, the monoclonal antibody palivizumab was the only RSV prophylaxis available in Japan. In 2024, the bivalent RSV prefusion F protein-based (RSVpreF) vaccine was approved for the prevention of RSV infection in infants by active immunization of pregnant women. In this study, we assessed the cost-effectiveness of a combined strategy of RSVpreF vaccine and palivizumab in Japanese setting. Using a Markov model, we evaluated prevented cases and deaths of medically attended RSV infections from birth to age 11months for each of the three healthcare settings: inpatient (hospitalization), emergency department visits, and outpatient visits. Incremental cost-effectiveness ratios (ICERs) were calculated from economic outcomes (intervention costs, medication costs, and productivity losses) and quality-adjusted life years (QALYs). Further, we calculated the maximum price of RSVpreF vaccine within which the program would be cost-effective. In comparison with the current prophylaxis (palivizumab alone), a combined prophylaxis of year-round RSVpreF vaccination of pregnant women and palivizumab prescription for premature infants born in < 32weeks gestational age (wGA) and all infants with high risk prevented 14,382 medically attended cases of RSV (hospitalization, 7490 cases; emergency department, 2239 cases; outpatient, 4653 cases) and 7 deaths, respectively. From a healthcare payer perspective, when the price of RSVpreF vaccine was equal to or less than ¥23,948 (US$182), a combination prophylaxis was cost-effective under the ICER threshold of ¥5million per QALY. The other combination prophylaxis of year-round RSVpreF vaccination and palivizumab prescription of premature born in < 32wGA regardless of risk in infants was a dominant strategy (more effective and less costly). A combined prophylaxis of year-round RSVpreF vaccine and palivizumab could be a cost-effective strategy to protect neonates throughout the infant stage (< 1years old) in Japan.

Cost-effectiveness in perioperative therapy in the era of immune checkpoint inhibitors.

8021 Background: The use of immune checkpoint inhibitors (ICIs) in lung cancer treatment, particularly in perioperative therapy, has significantly expanded. In Japan, atezolizumab was approved in 2022 for adjuvant therapy and nivolumab in 2023 for neoadjuvant therapy. Despite the long-term prognosis improvements associated with ICIs, their high costs have escalated healthcare expenses. This study evaluates the cost-effectiveness of perioperative therapies incorporating ICIs in Japan. Methods: A network meta-analysis (NMA) was conducted to derive hazard ratios (HRs) for disease-free survival (DFS) and overall survival (OS) from pivotal phase 3 clinical trials. Additionally, a partitioned survival model was developed to calculate quality-adjusted life years (QALYs) and life years (LYs). Cost data included drug and administration costs based on standard regimens. Results: ICIs with available DFS and OS data included atezolizumab, nivolumab, and pembrolizumab for adjuvant, neoadjuvant, and sandwich therapy, respectively. HRs (DFS/OS) compared to no perioperative therapy were as follows: adjuvant chemotherapy (0.81/0.86), neoadjuvant chemotherapy (0.87/0.80), adjuvant ICI (0.66/0.86), neoadjuvant ICI (0.55/0.49), and sandwich therapy (0.51/0.58). Cost/QALY/LY for each treatment were: no perioperative therapy (¥831,379/2.55/3.57), adjuvant chemotherapy (¥969,600/2.67/3.73), neoadjuvant chemotherapy (¥903,046/2.71/3.82), adjuvant ICI (¥9,209,863/2.70/3.74), neoadjuvant ICI (¥2,760,383/3.02/4.22), and sandwich therapy (¥8,235,047/2.95/4.10). Incremental cost-effectiveness ratios (ICERs) per QALY/LY were ¥425,189/¥295,036 for neoadjuvant chemotherapy and ¥6,152,576/¥4,592,090 for neoadjuvant ICI ($1 = \145). ICERs were not calculated for adjuvant chemotherapy, adjuvant ICI, and sandwich therapy due to extended dominance. Conclusions: In Japan, the general ICER threshold is ¥5,000,000, and ¥7,500,000 for treatments requiring special consideration, such as cancer therapies. The ICER for neoadjuvant chemotherapy was below the general threshold, and neoadjuvant ICIs below the special consideration threshold, making them cost-effective options for NSCLC patients in Japan. Other therapies were dominated, primarily due to 1) adjuvant chemotherapy being marginally more expensive and less effective than neoadjuvant chemotherapy, and 2) the high costs of long-term post operative ICI treatment for adjuvant ICI, and sandwich therapy.

Cost effectiveness analysis of a fixed dose combination pill for primary prevention of cardiovascular disease from an individual participant data meta-analysis

Cardiovascular disease (CVD) continues to impart a large burden on the global population, especially in lower income countries where affordability limits the use of cardiovascular medicines. A fixed dose combination strategy of at least 2 blood pressure lowering medications and a statin with aspirin in a single pill has been shown to reduce the risk of incident CVD by 38% in primary prevention in a recent meta-analysis. We report the in-trial (median follow-up: 5 years) cost-effectiveness of a fixed dose combination (FDC) pill in different income groups based on data from that meta-analysis. Countries were categorized using World Bank economic groups: Lower Middle Income Countries (LMIC), Upper Middle Income Countries (UMIC) and High Income Countries (HIC). Country specific costs were obtained for hospitalized events, procedures, and non-study medications (2020 USD). FDC price was based on the cheapest equivalent substitute (CES) for each component. For the CES-FDC pill versus control the difference in cost was $346 (95% CI: $294-$398) per participant in Lower Middle Income Countries, $838 (95% CI: $781-$895) in Upper Middle Income Countries and $42 (95% CI:-$155 to $239) (cost-neutral) in High Income Countries. During the study period the CES-FDC pill was associated with incremental gain in quality-adjusted life years of 0.06 (95% CI: 0.04-0.08) resulting in an incremental cost-effectiveness ratio (ICER) of $5767 (95% CI: 5735-$5799), $13,937 (95% CI: $13,893-$14,041) and $700 (95% CI: $662-$738) respectively. In subgroups analyses, the highest 10 years CVD risk subgroup had ICERs of $2033, $7322 and-$6000/QALY. A FDC pill produced at CES costs is cost-neutral in HIC. Governments of LMI and UMI countries should assess these results based on the ICER threshold accepted in their own country and own specific health care priorities but should consider prioritizing this strategy for patients with high 10 years CVD risk as a first step. Population Health Research Institute.

A cost-effectiveness analysis of a housing and recovery program for people with severe and persistent mental illnesses and precarious housing

ABSTRACT The Doorway program supports people with Serious and Persistent Mental Illnesses (SPMIs) receiving care within Victoria’s public mental health system and who are precariously housed. This paper reports on its cost-effectiveness. The study design was difference-in-differences involving a Treatment-As-Usual comparison group (TAU) using deidentified client records. The primary outcome measure was the proportion of days of secure housing per participant during the study and pre-study periods. Similarly, health service usage was also extracted from participants’ records. The cost of public mental health service usage was then estimated, including using governmental published data. The program’s Incremental Cost Effectiveness Ratio (ICER) and Cost-effectiveness Acceptability Curve were estimated using the bootstrap method. An ICER threshold of Australian $50,000 per additional person housed (hereafter A$) at 100% was set to determine cost-effectiveness and the probability of cost effectiveness at A$50,000. Mean total costs (secure housing and mental health service usage) were greater for the Doorway group compared to the TAU group (A$22,987). Mean percentage of days securely housed was also greater for the Doorway group (by 45.4%). The ICER value was A$50,613, the probability of this being 83.1%. The Doorway intervention was both more costly and more effective. Its cost-effectiveness approximated the pre-specified ICER threshold.

A Cost-Utility Analysis of Add-On Cannabidiol Versus Usual Care Alone for the Treatment of Seizures Associated with Tuberous Sclerosis Complex in England and Wales.

The aim of this study was to evaluate the cost effectiveness of plant-derived highly purified cannabidiol (Epidyolex® in the UK; 100mg/mL oral solution) as an add-on treatment to usual care for the management of treatment-refractory seizures associated with tuberous sclerosis complex (TSC) in patients aged ≥2 years. A cohort-based model was developed using a National Health Service perspective and lifetime horizon. Health states were based on weekly seizure frequency and seizure-free days, utilizing patient-level data from the GWPCARE6 trial (ClinicalTrials.gov identifier: NCT02544763). Two independent regression models were applied to individual patient-level data to predict seizure-free days and seizure frequency. Healthcare resource utilization data were sourced from a Delphi panel, and patient and caregiver health-related quality of life values were elicited using vignettes valued by the general public. Outcomes relating to TSC-associated neuropsychiatric disorders were modeled with costs and quality-adjusted life-years sourced from published literature. In the base case, compared with usual care alone, 12mg/kg/day cannabidiol was associated with an incremental cost-effectiveness ratio (ICER) of £23,797. The National Institute for Health and Care Excellence disease severity modifier reduced the ICER to £19,831. Probabilities of cost effectiveness at willingness-to-pay thresholds of £20,000 and £30,000 were 30% and 52%, respectively, for the base case and 39% and 66%, respectively, for the disease severity modifier. Results were robust to sensitivity and scenario analyses. At 12mg/kg/day and an ICER threshold of £20,000-£30,000, we provide evidence for the cost effectiveness of add-on cannabidiol treatment for patients with TSC-associated seizures aged ≥2 years who are refractory to current treatment.

Cost-effectiveness study of FIB-4 followed by transient elastography screening strategy for advanced hepatic fibrosis in a NAFLD at-risk population.

The cost-effectiveness to screen hepatic fibrosis in at-risk population as recommended by several professional societies has been limited. This study aimed to investigate the cost-effectiveness of this screening strategy in the expanded at-risk population recently proposed by several societies. A combined model of the decision tree and Markov models was developed to compare expected costs, quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratio (ICER) between screening and no screening groups. The model included liver disease-related health states and cardiovascular disease (CVD) states as a base-case analysis. Screening strategy consisted of fibrosis-4 index (FIB-4) followed by vibration-controlled transient elastography (VCTE) and intensive lifestyle intervention (ILI) as a treatment for diagnosed patients. Cost-effectiveness analysis showed that screening the at-risk population entailed $298 incremental costs and an additional 0.0199 QALY per patient compared to no screening (ICER $14 949/QALY). Screening was cost-effective based on the implicit ICER threshold of $25 000/QALY in Korea. When the effects of ILI on CVD and extrahepatic malignancy were incorporated into the cost-effectiveness model, the ICER decreased by 0.85 times from the base-case analysis (ICER $12 749/QALY). In contrast, when only the effects of liver disease were considered in the model, excluding cardiovascular disease effects, ICER increased from the baseline case analysis to $16 305. Even when replacing with medical costs in Japan and U.S., it remained cost-effective with the estimate below the countries' ICER threshold. Our study provides compelling evidence supporting the cost-effectiveness of FIB-4-based screening the at-risk population for advanced hepatic fibrosis.

Unapređenje procene odnosa troškova i efektivnosti lekova za retke bolesti

Background/Aim. Incremental cost/effectiveness ratio (ICER) of many drugs for rare diseases is often much higher that the accepted cost/effectiveness threshold for reimbursement, primarily due to their extremely high prices, raising the question of their availability. The aim of this article was to review necessary adjustments of methods used for cost/effectiveness analysis of drugs for rare diseases. Methods. This article is a narrative review of methods for adjusting cost/effectiveness analysis of drugs for rare diseases in order to get more realistic estimate of ICER threshold, which is essential information for decision-makers. Results. Inputs in cost/effectiveness analysis of a drug for rare diseases should be adjusted by changing discount rates, estimating utilities in a more precise way, excluding treatment-unrelated costs, calculating local C/E threshold, and most importantly, by negotiating drug price until the C/E threshold is not surpassed. With intensified adjusted cost/effectiveness research within the area, many uncertainties will be ended, and real-life value of many of the drugs for rare diseases will be known, influencing pricing in a sustainable direction. Conclusion. With the adjustments, the true cost/effectiveness of a drug for rare disease will be approached, enabling evidence-based and completely transparent reimbursement decisions.

PP87 Glecaprevir/pibrentasvir (Maviret®) Remains A Cost-effective Treatment For Chronic Hepatitis C Virus Infection After Changes To The Treated Population

IntroductionThe first direct-acting antiviral (DAA) therapies for chronic Hepatitis C virus (HCV) infection were reimbursed via Australia’s Pharmaceutical Benefits Scheme (PBS) in March 2016. This was based on the recommendation from the Pharmaceutical Benefits Advisory Committee (PBAC) that the regimens would be acceptably cost-effective at an incremental cost-effectiveness ratio (ICER) of AUD15,000/quality-adjusted life-year (QALY). Broad access to DAA therapies has been a key strategy in driving a national health goal to eliminate viral hepatitis as a major health threat by 2030. Since the initial PBS listings for DAA therapies and subsequent listings of newer DAA treatments such as Maviret, the demographics and disease characteristics of currently treated patients have markedly changed. The aim of our analysis was to reassess the cost-effectiveness of Maviret, accounting for the changes of the treated population characteristics and retreatment in first-line failures and reinfected individuals.MethodsTo assess the cost-effectiveness six years after initial listing of Maviret, an update was made to the Markov model used to achieve PBS reimbursement for Viekira-Pak® in May 2016. Amendments to the Viekira-Pak model include: changes to baseline age and fibrosis distribution of treated patients, and incorporation of retreatment of first-line failures (those not achieving a sustained virologic response (SVR)) and reinfected individuals. Treatment-related inputs including SVR response rates, adverse events, treatment-related disutility and discontinuations were sourced from pivotal glecaprevir/pibrentasvir clinical trials.ResultsUsing the published price of Maviret, the ICER is above AUD15,000/QALY but well below the commonly used ICER threshold in other chronic diseases (AUD45,000/QALY). When the confidential effective price is used, the ICER is under the AUD15,000/QALY cost-effectiveness threshold set by the PBAC for DAA therapies.ConclusionsDespite substantial changes to the population seeking treatment in Australia since reimbursement in 2016, Maviret remains a cost-effective treatment for chronic HCV infection.

Cost-effectiveness of brentuximab vedotin in Hodgkin lymphoma: a systematic review.

This study aimed to systematically review and critically appraise cost-effectiveness studies on Brentuximab vedotin (BV) in patients with Hodgkin lymphoma (HL). The PubMed, Scopus, Web of Science core collection, and Embase databases were searched until July 3, 2022. We included published full economic evaluation studies on BV for treating patients with HL. The methodological quality of the studies was assessed using the Quality of Health Economic Studies (QHES) checklist. Meanwhile, we used qualitative synthesis to analyze the findings. We converted the incremental cost-effectiveness ratios (ICERs) to the value of the US dollar in 2022. Eight economic evaluations met the study's inclusion criteria. The results of three studies that compared BV plus doxorubicin, vinblastine, and dacarbazine (BV + AVD) front-line therapy with doxorubicin, bleomycin, vincristine, and dacarbazine (ABVD) showed that BV is unlikely to be cost-effective as a front-line treatment in patients advanced stage (III or IV) HL. Four studies investigated the cost-effectiveness of BV in patients with relapsed or refractory (R/R) HL after autologous stem cell transplantation (ASCT). BV was not cost-effective in the reviewed studies at accepted thresholds. In addition, the adjusted ICERs ranged from $65,382 to $374,896 per quality-adjusted life-year (QALY). The key drivers of cost-effectiveness were medication costs, hazard ratio for BV, and utilities. Available economic evaluations show that using BV as front-line treatment or consolidation therapy is not cost-effective based on specific ICER thresholds for patients with HL or R/R HL. To decide on this orphan drug, we should consider other factors such as existence of alternative treatment options, clinical benefits, and disease burden.

Cost-effective analysis of transcatheter aortic valve replacement in patients with severe symptomatic aortic stenosis: A prospective multicenter study

BackgroundTranscatheter aortic valve replacement (TAVR) for severe symptomatic aortic stenosis (AS) does not benefit all patients. We performed a prospective multicenter study to investigate the cost-effectiveness of TAVR in a Japanese cohort. Methods and resultsWe prospectively enrolled 110 symptomatic patients with severe AS who underwent TAVR from five institutions. The quality of life measurement (QOL) was performed for each patient before and at 6 months after TAVR. Patients without an improvement in QOL at 6 months after TAVR were defined as non-responders. Pre-TAVR higher QOL, higher clinical frailty scale predicted the non-responders. Three models, 1) conservative treatment for all patients strategy, 2) TAVR for all patients strategy, and 3) TAVR for a selected patient strategy who is expected to be a responder, were simulated. Lifetime cost-effectiveness was estimated using incremental cost-effectiveness ratio (ICER) and cost per quality-adjusted life-year (QALY) gained. In comparison to conservative therapy for all patients, ICER was estimated to be 5,765,800 yen/QALY for TAVR for all patients and 2,342,175 yen/QALY for TAVR for selected patient strategy patients, which is less than the commonly accepted ICER threshold of 5,000,000 yen/QALY. ConclusionsTAVR for selected patient strategy model is more cost-effective than TAVR for all patient strategy without reducing QOL in the Japanese healthcare system. TAVR for selected patient strategy has potential benefit for optimizing the TAVR treatment in patients with high frailty and may direct our resources toward beneficial interventions.

Value-based pricing of add-on life-extending medicines based on economic evaluation

Value-based pricing of new medicines, when defined as pricing or reimbursement based on economic evaluation, requires the use of a threshold incremental cost-effectiveness ratio. Value-based pricing can leave little room for setting a price above zero for manufacturers of new life-extending add-on medicines that are used in combination regimens. This study aimed to present and explore different methodological approaches for pricing life-extending add-on medicines in this value-based pricing framework. The analysis demonstrated that excluding the costs of background therapy will create opportunity costs. A proportional division of the price can avoid opportunity costs. In the absence of information on relative health benefits, a counterfactual scenario of a head-to-head trial has suggested halving the price of background therapy in the relevant patient subgroups. Overall, the most plausible approach appears to be a proportional division of the total price of the combination therapy, in proportion to the health benefits of the add-on medicine and the background therapy.

An Industry Survey on Unmet Needs in South Korea's New Drug Listing System.

Since introducing the positive listing system in 2007, the South Korean government has undergone multiple changes in its drug listing system. As there is a lack of studies that evaluate the system from an industry perspective, this paper examined South Korea's new drug listing system from the suppliers' perspective. We surveyed members of the three main pharmaceutical industry associations online. The survey (a 5-point Likert scale) covered their satisfactory levels, demands, and updates on the current new drug listing system, especially pharmacoeconomic evaluation, pharmacoeconomic evaluation exemption, and risk-sharing agreement. A total of 56 respondents participated in the survey. The self-reported satisfaction level for value recognition of new drugs was 1.6 (± 0.7) points (5 points = very satisfied). The most highly demanded reforms for PE, RSA, and PEE were incremental cost-effectiveness ratio threshold (92.9%), reimbursement scope expansion (91.1%), and eligible disease (83.9%). Lastly, they also claimed that the indication-based pricing system must be introduced (83.9%). Pricing and reimbursement policies need to improve in such a way that would enable better access to new drugs while still facilitating their development. Given the nature of the current system, some innovative rare disease treatments and anticancer drugs remain unreimbursed, resulting in low satisfaction levels across the pharmaceutical industry. Hence, pathways to speed up the reimbursement assessment process and expand the range of reimbursable diseases are required. Pharmaceutical companies are also important stakeholders, like in the case of clinicians and patients, and their opinions should also be considered in the process of pricing and reimbursement policy reforms.

Cost-Effectiveness Analysis of REBYOTA™ (Fecal Microbiota, Live-jslm [FMBL]) Versus Standard of Care for the Prevention of Recurrent Clostridioides difficile Infection in the USA.

Recurrent Clostridioides difficile infection (rCDI) is common and associated with considerable clinical and economic consequences. REBYOTA™ (fecal microbiota, live-jslm [FMBL]) is a microbiota-based live biotherapeutic approved for the prevention of rCDI following antibiotic treatment for rCDI. We sought to evaluate cost-effectiveness of FMBL compared to standard of care (SOC) from a US third-party payer perspective among patients with one or more (≥ 1) recurrences. A Markov model with a lifetime time horizon was developed. The model population included adult patients who had ≥ 1 recurrence after a primary CDI episode and had completed ≥ 1 round of antibiotics, or had ≥ 2 severe CDI episodes resulting in hospitalization within the last year. The model consisted of six health states with an 8-week model cycle: rCDI, absence of CDI after recurrence, colectomy, ileostomy, ileostomy reversal, and death. Drug costs and rCDI-related medical costs were estimated in 2022 US dollars and discounted at 3% annually. Deterministic sensitivity analyses were performed. Compared to SOC, FMBL at $9000/course resulted in an incremental cost-effectiveness ratio (ICER) of $18,727 per quality-adjusted life year (QALY) gained. The incremental cost was $5336 (FMBL $79,236, SOC $73,900) and the incremental effectiveness was 0.285QALYs (FMBL 10.346, SOC 10.061). The cumulative drug acquisition and administration costs for the FMBL and SOC arms were $24,245 and $16,876, while rCDI-related medical costs for FMBL and SOC were $54,991 and $57,024, respectively. The ICER in the subgroup of patients at first recurrence was $13,727 per QALY gained. FMBL remained cost-effective across all sensitivity analyses. FMBL was found to be cost-effective compared to SOC for the prevention of rCDI with more benefits among patients at first recurrence, with an ICER far below the payer ICER threshold of $100,000. Patients treated with FMBL experienced higher total QALYs and reduced healthcare resource utilization, including reduced hospitalizations.

What is the Value of a Balanced Total Knee Arthroplasty? Getting It Right the First Time

BackgroundInstability is a leading cause of early failure following total knee arthroplasty (TKA). Enabling technologies can improve accuracy, but their clinical value remains undetermined. The purpose of this study was to determine the value of achieving a balanced knee joint at the time of TKA. MethodsA Markov model was developed to determine the value from reduced revisions and improved outcomes associated with TKA joint balance. Patients were modeled for the first 5 years following TKA. The threshold to determine cost-effectiveness was set at an incremental cost effectiveness ratio of $50,000/quality-adjusted life year (QALY). A sensitivity analysis was performed to evaluate the influence of QALY improvement (ΔQALY) and Revision Rate Reduction on additional value generated compared to a conventional TKA cohort. The impact of each variable was evaluated by iterating over a range of ΔQALY (0 to 0.046) and Revision Rate Reduction (0% to 30%) and calculating the value generated while satisfying the incremental cost effectiveness ratio threshold. Finally, the impact of surgeon volume on these outcomes was analyzed. ResultsThe total value of a balanced knee for the first 5 years was $8,750, $6,575, and $4,417 per case, for low, medium, and high-volume surgeons, respectively. Change in QALY accounted for greater than 90% of the value gain with a reduction in revisions making up the rest in all scenarios. The economic contribution of revision reduction was relatively constant regardless of surgeon volume ($500/case). ConclusionAchieving a balanced knee had the greatest impact on ΔQALY over early revision rate. These results can help assign value to enabling technologies with joint balancing capabilities.

Is Surgical Repair With Nerve Allograft More Cost-Effective Than Non-Surgical Management for Persistent Trigeminal Neuropathy? Initial Assessment With Markov Model

Persistent trigeminal neuropathy (PTN) is associated with high rates of depression, loss of work, and decreased quality of life (QoL). Nerve allograft repair can achieve functional sensory recovery in a predictable manner; however, it bears significant upfront costs. In patients suffering from PTN, is surgical repair with allogeneic nerve graft, when compared to non-surgical therapy, a more cost-effective treatment option? A Markov model was constructed with TreeAge Pro Healthcare 2022 (TreeAge Software, Massachusetts) to estimate the direct and indirect costs for PTN. The model ran for 40years with 1-year-cycles on a 40-year-old model patient with persistent inferior alveolar or lingual nerve injury (S0 to S2+) at 3months without signs of improvement, and without dysesthesia or neuropathic pain (NPP). The 2 treatment arms were surgery with nerve allograft versus non-surgical management. There were 3 disease states, functional sensory recovery (S3 to S4), hypoesthesia/anesthesia (S0 to S2+), and NPP. Direct surgical costs were calculated using the 2022 Medicare Physician Fee Schedule and verified with standard institutional billing practices. Non-surgical treatment direct costs (follow-up, specialist referral, medications, imaging) and indirect costs (QoL, loss of employment) were determined from historical data and the literature. Direct surgical costs for allograft repair were $13,291. State-specific direct costs for hypoesthesia/anesthesia were $2,127.84 per year, and $3,168.24 for NPP per year. State-specific indirect costs included decreased labor force participation, absenteeism, and decreased QoL. Surgical treatment with nerve allograft was more effective and had a lower long-term cost. The incremental cost-effectiveness ratio was -10,751.94, indicating surgical treatment should be utilized based on efficiency and cost. With a willingness-to-pay threshold of $50,000, the net monetary benefits of surgical treatment are $1,158,339 compared to $830,654 for non-surgical treatment. With a standard threshold incremental cost-effectiveness ratio of 50,000, the sensitivity analysis shows that surgical treatment would remain the preferred choice based on efficiency even if surgical costs were doubled. Despite high initial costs of surgical treatment with nerve allograft for PTN, surgical intervention with nerve allograft is a more cost-effective treatment option when compared to non-surgical therapy.

Cost-Effectiveness Analysis of CAR T-Cell Therapies vs Antibody Drug Conjugates for Patients with Advanced Multiple Myeloma.

Among advanced multiple myeloma (MM) patients, B-cell maturation antigen (BCMA) specific targets like Belantamab Mafodotin (belamaf) and CAR T-cell therapies have been shown to improve clinical outcomes, but at significant costs. To compare the expected costs per quality-adjusted life years (QALYs) gained among a hypothetical cohort of triple refractory MM patients treated with one of three BCMA-directed therapies: (1) idecabtagene vicleucel (ide-cel), (2) ciltacabtagene autoleucel (cilta-cel), and (3) belamaf for up to 20months. In this cost-effectiveness analysis, we built a Monte Carlo Markov Chain microsimulation model using estimates and parameters from the evidence on MM treatment for 10000 hypothetical patients between the ages for 40 and 80. We assigned expected years of life remaining and made varying assumptions about survival beyond 5years. We predicted total cost of treatment for CAR-T therapy to be six times greater than for belamaf, but the QALYs gained from treatment are 6 to 8 times greater. Ide-cel was weakly dominated by cilta-cel and our base-case incremental cost effectiveness ratio (ICER) comparing cilta-cel with belamaf was $109,497 per QALY gained, averaging $123,618 in probabilistic sensitivity analyses. These findings hinge on the assumption of longer-term survival but suggest that the use of CAR-T therapy is approaching standard ICER thresholds.

Cost-effectiveness of broadly neutralizing antibody prophylaxis for HIV-exposed infants in sub-Saharan African settings.

Infant HIV prophylaxis with broadly neutralizing anti-HIV antibodies (bNAbs) could provide long-acting protection against vertical transmission. We sought to estimate the potential clinical impact and cost-effectiveness of hypothetical bNAb prophylaxis programmes for children known to be HIV exposed at birth in three sub-Saharan African settings. We conducted a cost-effectiveness analysis using the CEPAC-Pediatric model, simulating cohorts of infants from birth through death in Côte d'Ivoire, South Africa and Zimbabwe. These settings were selected to reflect a broad range of HIV care cascade characteristics, antenatal HIV prevalence and budgetary constraints. We modelled strategies targeting bNAbs to only WHO-designated "high-risk" HIV-exposed infants (HR-HIVE) or to all HIV-exposed infants (HIVE). We compared four prophylaxis approaches within each target population: standard of care oral antiretroviral prophylaxis (SOC), and SOC plus bNAbs at birth (1-dose), at birth and 3 months (2-doses), or every 3 months throughout breastfeeding (Extended). Base-case model inputs included bNAb efficacy (60%/dose), effect duration (3 months/dose) and costs ($60/dose), based on published literature. Outcomes included paediatric HIV incidence and incremental cost-effectiveness ratios (ICERs) calculated from discounted life expectancy and lifetime HIV-related costs. The model projects that bNAbs would reduce absolute infant HIV incidence by 0.3-2.2% (9.6-34.9% relative reduction), varying by country, prophylaxis approach and target population. In all three settings, HR-HIVE-1-dose would be cost-saving compared to SOC. Using a 50% GDP per capita ICER threshold, HIVE-Extended would be cost-effective in all three settings with ICERs of $497/YLS in Côte d'Ivoire, $464/YLS in South Africa and $455/YLS in Zimbabwe. In all three settings, bNAb strategies would remain cost-effective at costs up to $200/dose if efficacy is ≥30%. If the bNAb effect duration were reduced to 1 month, the cost-effective strategy would become HR-HIVE-1-dose in Côte d'Ivoire and Zimbabwe and HR-HIVE-2-doses in South Africa. Findings regarding the cost-effectiveness of bNAb implementation strategies remained robust in sensitivity analyses regarding breastfeeding duration, maternal engagement in postpartum care, early infant diagnosis uptake and antiretroviral treatment costs. At current efficacy and cost estimates, bNAb prophylaxis for HIV-exposed children in sub-Saharan African settings would be a cost-effective intervention to reduce vertical HIV transmission.