Cost-utility analysis of emicizumab prophylaxis in haemophilia A patients with factor VIII inhibitors in Korea.

AIMS: Haemophilia A patients with factor VIII inhibitors (HAPI) experience frequent spontaneous bleeding, approximately once a week, and require expensive bypassing agent (BPA) treatments to control bleeding over their lifetime. According to the HAVEN 1 trial, weekly emicizumab (Hemlibra®) prophylaxis injection reduces annualized bleeding rates (ABR) by 87% compared with BPA on-demand treatment (BPA-OD) administered at the time of bleeding. Our study aimed to assess the cost-effectiveness of emicizumab prophylaxis in HAPI in Korea. METHODS: Using a lifetime Markov model with health states of 'alive with bleeds' and 'dead', we simulated the experience of HAPI receiving emicizumab prophylaxis (treatment arm) or BPA-OD (control arm) and estimated expected clinical and economic outcomes under each treatment arm. Model parameters included comparative effectiveness, clinical and epidemiologic characteristics of Korean HAPI, costs of drug treatment and medical events and utility for 'alive with bleeds' state under each treatment. We utilized local data, including National Health Insurance claims data, national statistics, literature and expert surveys with haematologists. RESULTS: Base-case analysis results showed that compared with BPA-OD, lifetime emicizumab prophylaxis prevented 807 bleedings, extended 3.04 quality-adjusted life-years and reduced costs by 2.6 million US dollars. Thus, emicizumab prophylaxis is a dominant treatment option with better effectiveness and lower costs than BPA-OD. A series of one-way sensitivity analyses consistently showed dominant results, confirming that lifetime emicizumab prophylaxis is a cost-saving intervention for HAPI. CONCLUSION: Emicizumab prophylaxis is an excellent treatment choice reducing ABR, improving quality of life and reducing costs.

Cost-utility analysis of pralatrexate for relapsed or refractory peripheral T-cell lymphoma based on a case-matched historical control study along with single arm clinical trial.

BACKGROUND: Patients with relapsed or refractory peripheral T-cell lymphoma (R/R PTCL) treated with pralatrexate have previously shown superior overall survival (OS) compared to those who underwent conventional chemotherapy (CC, 15.4 vs. 4.07 months). We conducted an economic evaluation of pralatrexate from a societal perspective in Korea based on data from the PROPEL phase II study. METHODS: Using a Markov model with a weekly cycle, we simulated the experience of patients with R/R PTCL receiving pralatrexate or CC for 15 years. The model consists of five health states; initial treatment, treatment pause, subsequent treatment, stem cell transplantation (SCT) success, and death. Comparative effectiveness was based on PROPEL phase II single-arm study and its matched historical control analysis. Costs included drug, drug administration, monitoring, adverse event management, and SCT costs. RESULTS: The incremental cost-effectiveness ratio of the base case was $39,153 per quality-adjusted life-year (QALY) gained. The results of one-way sensitivity analysis ranged from $33,949 to $51,846 per QALY gained, which remained within an implicit willingness-to-pay (WTP) threshold of anticancer drugs in Korea. CONCLUSIONS: Pralatrexate is a cost-effective intervention with improved OS and incremental costs within the WTP limit. Pralatrexate could function as a new therapeutic option for patients suffering from life-threatening R/R PTCL.

Prevalence and socio-economic burden of heart failure in an aging society of South Korea.

BACKGROUND: Heart failure (HF) is one of the leading causes of morbidity and mortality in South Korea. With the rapidly aging population in the country, the prevalence of HF and its associated costs are expected to rise continuously. This study was carried out to estimate the prevalence and economic burden of HF in order to understand its impact on our society. METHODS: A prevalence-based, cost-of-illness study was conducted using the 2014 Health Insurance Review and Assessment Service-National Patients Sample (HIRA-NPS) data. Adult HF patients were defined as those aged ≥19 years who had at least one insurance claim record with a primary or secondary diagnosis of HF (ICD-10 codes of I11.0, I13.0, I13.2, and I50.x). The costs consist of direct costs (i.e., medical and non-medical costs) and indirect costs (i.e., productivity loss cost due to morbidity and premature death). Subgroup analyses were conducted by age group, history of HF hospitalization, and type of universal health security program enrolled in. RESULTS: A total of 475,019 adults were identified to have HF in 2014. The estimated prevalence rate of HF was 12.4 persons per 1,000 adults. According to the base cases and the extended definition of the cases, the annual economic burden of HF from a societal perspective ranges from USD 1,414.0 to 1,560.5 for individual patients, and from USD 752.8 million to 1,085.6 million for the country. A high percentage (68.5 %) of this socioeconomic burden consist of medical costs, followed by caregiver's cost (13.2 %), productivity loss costs due to premature death (10.8 %) and morbidity (4.2 %), and transportation costs (3.4 %). The HF patients with prior hospitalization due to HF annually spent 9.7 times more for National-Health-Insurance-covered medical costs compared to HF patients who were not previously hospitalized. CONCLUSIONS: In the present study, HF patients who were older and had a history of prior hospitalization for HF as well as an indigent status were shown at high risk of spending more for healthcare to treat their HF. An effective disease management protocol should be employed to target this patient group.

Effect of Core Balance Training on Muscle Tone and Balance Ability in Adult Men and Women.

(1) Background: The amount of physical activity most adults perform is less than the recommended amount, and the resulting decrease in physical strength makes them vulnerable to various diseases. A decrease in muscle size and strength due to damage caused by disease or aging negatively affects functional strength. Muscle evaluation in adults can yield results that are predictive indicators of aging and unexpected disability. In addition, balance ability is essential to prevent falls and injuries in daily life and maintain functional activities. It is important to develop and strengthen balance in the lower extremities and core muscles to maintain and enhance overall body balance. This study aimed to analyze the effects of core balance training on muscle tone and balance ability in adults. (2) Methods: The participants of this study were 32 adult male and female university students (male: mean age = 21.3 ± 1.9 years, weight = 74.2 ± 12.6 kg, BMI = 23.4 + 2.5, n = 14; female: mean age = 21.0 ± 1.4 years, weight = 64.6 + 1.2 kg, BMI = 22.4 ± 2.4, n =18). Thirty-two adults (training group: 16, control group: 16; male: 16, female: 16) participated in the Myoton PRO (gastrocnemius lateral/medial, tibialis anterior), Pedalo balance system, and Y-balance test. (3) Results: The following results were obtained for muscle elasticity, stiffness, and dynamic/static balance ability after 10 weeks of core balance training. 1. There was no significant difference in muscle elasticity (gastrocnemius lateral/medial, tibialis anterior) (p < 0.05). 2. Muscle stiffness (gastrocnemius lateral/medial, tibialis anterior) significantly increased (p < 0.05). 3. Dynamic/static balance ability significantly increased (p < 0.05). (4) Conclusions: In future, data for the age and sex of various participants, should be accumulated by recruiting participants to study muscle characteristics, such as muscle elasticity and stiffness. Estimating the appropriate injury range and optimal exercise capacity is possible through follow-up studies. The findings can then be used as a basis for predicting injuries or determining and confirming the best time to resume exercise.

Potentially inappropriate prescribing of cardiovascular system and antiplatelet/anticoagulant drugs among elderly patients: a Korean population-based national study.

OBJECTIVES: To investigate the prevalence of potentially inappropriate prescribing (PIP) for cardiovascular system (CVS) and antiplatelet/anticoagulant (AP/AC) drugs among Korean elderly patients, using the Screening Tool of Older Persons' Prescriptions (STOPP) criteria version 2 and to identify the risk factors related to PIP. METHODS: The 2016 National Aged Patient Sample data, comprising National Health Insurance claim records for a random sample of 20% of patients aged ≥ 65 years, were used to calculate PIP prevalence of outpatient prescriptions. For criteria including drug-disease interactions, PIP prevalence per indication was estimated. RESULTS: Among 1,274,148 elderly patients and 27,062,307 outpatient prescription claims, 100,085 patients (7.85%) and 341,664 claims (1.27%) had one or more PIP. The most frequent PIP was "non-steroidal anti-inflammatory drug with concurrent antiplatelet agent (s) without proton-pump inhibitor prophylaxis" in the claim-level (0.97%) and patient-level (6.33%) analyses. "Beta-blocker with bradycardia" (16.47% of claims) and "angiotensin receptor blockers in patients with hyperkalaemia" (23.89% of claims) showed the highest PIP prevalence per indication. Logistic regression analysis revealed that, among the patient and health care provider characteristics, female, older age, more severe comorbidities, polypharmacy, higher level of healthcare organization, and specialty of prescriber were significantly associated with a higher risk of PIP. CONCLUSIONS: Our findings of a high prevalence of PIP for CVS and AP/AC drugs among the elderly suggest that an effective strategy is urgently needed to improve the prescription practices of these drugs.

Socioeconomic Impact of the Rotavirus Vaccine in Korea: Comparing the Epidemiologic and Economic Characteristics of Rotavirus Gastroenteritis Before and After the Introduction of Vaccines.

BACKGROUND: Since the introduction of 2 rotavirus (RV) vaccines in Korea, the vaccination rate has reached over 80% with out-of-pocket spending in the private market. We investigated the socioeconomic impact of RV vaccines in Korea to assess their value and public health contribution. METHODS: By using National Health Insurance Service claims data, we compared the epidemiologic and economic characteristics of rotavirus gastroenteritis (RVGE) before and after the introduction of RV vaccines. For each year of the study period, the annual prevalence and national costs of RVGE were estimated based on children under 5 years with at least 1 National Health Insurance Service claims record with a diagnosis of RVGE. RESULTS: Compared with the prevaccination period, the prevalence of RVGE decreased in the postvaccination period by 48.9% from 2097 per 100,000 children in 2006 to 1072 per 100,000 children in 2015, implying an increase in the vaccination rate and the prevention effect of the vaccines. The highest reduction was observed among those 12 to <24 months of age (-73.4%), presumably due to the benefit of full vaccination, while children under 2 months, ineligible for the RV vaccine, showed an increase (41.7%). The number of hospitalized RVGE cases per year decreased by 69.0%. The national economic burden of RVGE decreased by 28.6%. CONCLUSIONS: The substantial reduction in the socioeconomic burden of RVGE after the introduction of RV vaccines confirms their benefit to society. This study would help health policy makers make empirical decisions on incorporating the vaccination into national immunization programs.

Cost-effectiveness analysis of the implementation of a National Immunization Program for rotavirus vaccination in a country with a low rotavirus gastroenteritis-related mortality: A South Korean study.

Rotavirus is a leading cause of severe gastroenteritis among children younger than 5 years in South Korea. Two rotavirus vaccines (RVs), pentavalent human-bovine reassortant vaccine (Rotateq®; RV5) and attenuated human strain originated monovalent vaccine (Rotarix®; RV1), have been available for voluntary vaccination using out-of-pocket payment since 2007 and 2008, respectively. Yet, RVs are not included in the National Immunization Program (NIP), partly because of the low associated mortality rate. We assessed the cost-effectiveness of RVs to assist the evidence-based decision-making process for NIP implementation in South Korea. Using a transparent age-structured static cohort model, we simulated the experience of ten annual birth cohorts of South Korean children from 2018 to 2027. Model inputs included rotavirus gastroenteritis (RVGE) incidence and mortality rates, RVGE treatment costs, vaccine coverage and timeliness, and vaccine effectiveness and price. The incremental costs of including RVs in the NIP compared to no vaccination were 59,662,738 USD and 152,444,379 USD for RV1 and RV5, respectively. The introduction of RV1 and RV5 can prevent 4799 disability-adjusted life years (DALYs) and 5068 DALYs. From the societal perspective, the incremental cost-effectiveness ratios (ICERs) for adopting RV into the NIP versus no vaccination were 12,432 USD per DALY averted for RV1 and 30,081 USD per DALY averted for RV 5. The weighted average for the ICERs of the two vaccines computed using the market share of each vaccine in the current voluntary use as a weight, was 21,698 USD per DALY averted. The estimated ICER was below 1 × gross domestic product per capita (30,000 USD), which has been a commonly used willingness-to-pay threshold for health care technology assessment in South Korea, suggesting that introducing RVs into the NIP would be cost-effective.

The incremental economic burden of heart failure: A population-based investigation from South Korea.

BACKGROUND: The prevalence of heart failure (HF) and its economic burden are increasing with age of the South Korean population. This study aimed to assess the economic impact of HF from the societal perspective. METHODS: A prevalence-based, incremental cost-of-patient study was performed to estimate the cost ratio between patients with HF and those without HF based on the claims database of the national health insurance in South Korea. We defined adult HF patients as those aged ≥19 years who had at least one insurance claim record with a primary or secondary diagnosis of HF. Age- and gender-matched controls were defined using a 1:4 greedy matching method. Costs were estimated by including medical costs for insurance-covered and non-covered services, transportation costs, caregiver's cost, and time costs of patients. The ratio of costs between patients with HF and those without HF was adjusted for age, gender, and type of universal health security program in the multivariate regression model. RESULTS: The average annual per-capita cost was estimated to be $6,601 for patients with HF (n = 14,252), which is about 3.38 (95% confidence interval [CI]: 3.31-3.46) times higher than that for patients without HF (n = 1,116,882) and 1.64 (95% CI: 1.59-1.70) times higher than that for the age- and gender-matched patients without HF (n = 57,008). In the multivariate regression model, the annual per-capita total costs were 1.98-fold (95% CI: 1.94-2.02) statistically higher for patients with HF than for patients without HF after adjustment for age, gender, and type of universal health security program. CONCLUSIONS: This study demonstrates a significant incremental burden of HF. Given that the prevalence of HF is expected to increase with an increase in the aging population, the national economic burden is expected to be substantial in the future. Thus, greater emphasis on the prevention and treatment of HF is warranted.

Prevalence and predictors of potentially inappropriate prescribing of central nervous system and psychotropic drugs among elderly patients: A national population study in Korea.

OBJECTIVES: To investigate the prevalence of potentially inappropriate prescribing (PIP) of central nervous system and psychotropic (CNS-PS) drugs to the Korean elderly population, and to identify PIP-associated factors. METHODS: Ambulatory care visits were identified from the 2013 National Aged Patient Sample (HIRA-APS-2013) data, composed of 20% random samples of all enrollees in the universal health security program aged ≥65 years. The CNS-PS section of Screening Tool of Older Person's potentially inappropriate Prescriptions (STOPP) criteria version 2 was used to identify PIP at these visits. RESULTS: A total of 24,427,069 prescription claims records and 1,122,080 patients were included in the study; 10.73% of the claims and 53.64% of the patients satisfied at least one STOPP criterion in the prescription of CNS-PS drugs. The highest prevalence of PIP was observed for the criteria of "first-generation antihistamines" (FGAH), followed by tricyclic antidepressants (TCA) in patients with prostatism and TCA in patients with dementia. The generalized estimating equation logistic regression analysis showed that the PIP of FGAH was significantly associated with polypharmacy (5-9 drugs: odds ratio (OR) 4.965, 95% confidence interval (CI) 4.936-4.994; ≥10 drugs: OR 5.704, 95% CI 5.604-5.807), less severe health conditions (Charlson Comorbidity Index (CCI)=2: OR 0.852, 95% CI 0.842-0.862; CCI=1: OR 0.975, 95% CI 0.964-0.986), prescriptions from clinics (OR>1.0), and outpatient care by general practitioners (OR>1.0). CONCLUSIONS: Appropriate interventions to reduce PIP should be made, especially for the criteria that indicate a high PIP prevalence. Targeted strategies are necessary to modify the risk factors of PIP identified from this study.