This study aimed to identify orphan drug accessibility and impact on pharmaceutical budgets in South Korea by analyzing the status of orphan drug designation, approval, reimbursement, and pharmaceutical expenditure. We analyzed the dataset on orphan drugs designated, approved, and reimbursed from 2007 to 2019 based on long-term real-world data. The designated and approved orphan drugs were 165 and 156, respectively, and 88 out of 156 approved products were reimbursed. Total expenditure on orphan drugs increased annually to account for about 1.44% of total pharmaceutical expenditure in 2018. Orphan drug expenditure per patient increased on average by 8.7% per year. The average annual cost of orphan drugs was USD 27,000–USD 47,000, with the maximum value of USD 260,000–USD 560,000. As there are a number of orphan drugs that have not yet been reimbursable after approval, a reimbursement policy should be established that considers the characteristics of orphan drugs. Since the rapid increase in orphan drug expenditure can be a potential threat to the insurance budget, budget management should also be considered. In conclusion, it is necessary to take preemptive measures to manage the health insurance budget efficiently while improving patient accessibility to orphan drugs.

Background: Mobile health technologies show promise in addressing metabolic syndrome, but their comparative effectiveness in large-scale public health interventions remains unclear. Objective: This study aims to compare the effectiveness of wearable devices (wearable activity trackers) and mobile app–based activity trackers (built-in step counters) in promoting walking practice, improving health behaviors, and reducing metabolic syndrome risk within a national mobile health care program operated by the Korea Health Promotion Institute. Methods: This retrospective cohort study analyzed data from 46,579 participants in South Korea’s national mobile health care program (2020‐2022). Participants used wearable devices for 12 weeks, after which some switched to built-in step counters. The study collected data on demographics, health behaviors, and metabolic syndrome risk factors at baseline, 12 weeks, and 24 weeks. Outcomes included changes in walking practice, health behaviors, and metabolic syndrome risk factors. Metabolic syndrome risk was assessed based on 5 factors: blood pressure, fasting glucose, waist circumference, triglycerides, and high-density lipoprotein cholesterol. Health behaviors included low-sodium diet preference, nutrition label reading, regular breakfast consumption, aerobic physical activity, and regular walking. To address potential selection bias, propensity score matching was performed, balancing the 2 groups on baseline characteristics including age, gender, education level, occupation, insurance type, smoking status, and alcohol consumption. Results: Both wearable activity tracker and built-in step counter groups exhibited significant improvements across all evaluated outcomes. The improvement rates for regular walking practice, health behavior changes, and metabolic syndrome risk reduction were high in both groups, with percentages ranging from 45.2% to 60.8%. After propensity score matching, both device types showed substantial improvements across all indicators. The built-in step counter group demonstrated greater reductions in metabolic syndrome risk compared to the wearable device group (odds ratio [OR] 1.20, 95% CI 1.05‐1.36). No significant differences were found in overall health behavior improvements (OR 0.95, 95% CI 0.83‐1.09) or walking practice (OR 0.84, 95% CI 0.70‐1.01) between the 2 groups. Age-specific subgroup analyses revealed that the association between built-in step counters and metabolic syndrome risk reduction was more pronounced in young adults aged 19‐39 years (OR 1.35, 95% CI 1.09‐1.68). Among Android use subgroups, built-in step counters were associated with a higher reduction in health risk factors (OR 1.20, 95% CI 1.03‐1.39). Conclusions: Both wearable devices and built-in step counters effectively reduced metabolic syndrome risk in a large-scale public health intervention, with built-in step counters showing a slight advantage. The findings suggest that personalized device recommendations based on individual characteristics, such as age and specific health risk factors, may enhance the effectiveness of mobile health interventions. Future research should explore the mechanisms behind these differences and their long-term impacts on health outcomes.

Aceclofenac controlled-release (CR) is a once-a-day tablet with 200 mg of aceclofenac, and is bioequivalent to conventional aceclofenac. However, its safety in humans has not been well studied in Korea. Therefore, we aimed to evaluate the overall incidence and patterns of adverse events (AEs), the effectiveness of aceclofenac CR, and the differences in incidence rates of the AEs based on each patient’s baseline charateristics. This study was conducted on patients receiving aceclofenac CR in clinical practice at each investigational institution to treat musculoskeletal pain and inflammation. The subjects were administered one tablet of aceclofenac CR (200 mg once-a-day) and were observed for 4 weeks post-administration. Factors affecting the occurrence of AEs were evaluated, and the Visual Analogue Scale (VAS) was used to measure the pain intensity. Among 14,543 subjects, the incidence rate of AEs was 0.86%, and that of adverse drug reactions was 0.74%. No serious AEs and unexpected adverse drug reactions were monitored. The incidence rates of AEs were significantly higher in females, inpatient treatment, individuals with concurrent disorders, and those receiving concomitant medications, respectively (all P < 0.05). Four weeks post-using aceclofenac CR, the mean changes in VAS was significantly decreased compared to prior administration. The overall clinical efficacy rate was 91.63%. This study confirmed that no severe adverse reactions were observed for aceclofenac CR exceeding those previously reported for safety results of conventional formulation of this drug in routine clinical practice settings. The use of aceclofenac CR might not violate the previously reported information on the safety and effectiveness of aceclofenac.

This study aimed to restore the skin barrier function from atopic dermatitis (AD) via treatment with leucine-rich glioma inactivated 3 (LGI3) peptide. Male NC/Nga mice (7 weeks, 20 g) were randomly allocated into three groups (control, AD, and LGI3 group). After induction of AD skin lesions with Dermatophagoides farinae ointment, mice were treated with LGI3. The clinical score of AD was the highest and the dorsal skin thickness was the thickest in the AD group. In contrast, LGI3 treatment improved the clinical score and the dorsal skin thickness compared to the AD model. LGI3 treatment suppressed histopathological thickness of the epithelial cell layer of the dorsal skin. LGI3 treatment could indirectly reduce mast cell infiltration through restoring the barrier function of the skin. Additionally, the filaggrin expression was increased in immunohistochemical evaluation. In conclusion, the ameliorating effect and maintaining skin barrier homeostasis in the AD murine model treated with LGI3 could be attributed to complete re-epithelialization of keratinocytes. Hence, LGI3 might be considered as a new potential therapeutic target for restoring skin barrier function in AD.

Background: Orphan drug pricing in South Korea, including its workings and determinants, is a complicated aspect of the country’s healthcare system. Therefore, this study identified the factors associated with the pricing of orphan drugs in South Korea.Methods: The study screened 66 orphan drugs out of the whole set of newly reimbursed drugs from 2012 to 2022. The analysis presented the characteristics of the orphan drugs and accounted for correlations between treatment cost and other seemingly related variables. Formal multivariate regression analysis indicated associated factors in multivariate settings.Results: Annual treatment cost per patient displayed a weakly negative correlation with the number of patients. Meanwhile, treatment cost exhibited a strongly positive correlation with the average of adjusted prices in seven advanced countries, reaching an adjusted R-squared value of 0.99. Treatment cost also showed a positive correlation with budget impact.Conclusion: The pricing of orphan drugs in South Korea is predominantly influenced by external reference pricing. Drugs targeting smaller patient populations tend to be priced higher. This finding can guide policymakers, pharmaceutical companies, and healthcare providers in their efforts to balance patient accessibility with the sustainable development of orphan drugs and the financial sustainability of public health insurance.