Cost-Utility Analysis of Early Detection with Ultrasonography of Differentiated Thyroid Cancer: A Retrospective Study on a Korean Population.

BACKGRUOUND: There is debate about ultrasonography screening for thyroid cancer and its cost-effectiveness. This study aimed to evaluate the cost-effectiveness of early screening (ES) versus symptomatic detection (SD) for differentiated thyroid cancer (DTC) in Korea. METHODS: A Markov decision analysis model was constructed to compare the cost-effectiveness of ES and SD. The model considered direct medical costs, health outcomes, and different diagnostic and treatment pathways. Input data were derived from literature and Korean population studies. Incremental cost-effectiveness ratio (ICER) was calculated. Willingness-to-pay (WTP) threshold was set at USD 100,000 or 20,000 per quality-adjusted life year (QALY) gained. Sensitivity analyses were conducted to address uncertainties of the model's variables. RESULTS: In a base case scenario with 50 years of follow-up, ES was found to be cost-effective compared to SD, with an ICER of $2,852 per QALY. With WTP set at $100,000, in the case with follow-up less than 10 years, the SD was cost-effective. Sensitivity analysis showed that variables such as lobectomy probability, age, mortality, and utility scores significantly influenced the ICER. Despite variations in costs and other factors, all ICER values remained below the WTP threshold. CONCLUSION: Findings of this study indicate that ES is a cost-effective strategy for DTC screening in the Korean medical system. Early detection and subsequent lobectomy contribute to the cost-effectiveness of ES, while SD at an advanced stage makes ES more cost-effective. Expected follow-up duration should be considered to determine an optimal strategy for DTC screening.

Cost-Effectiveness of Active Surveillance Compared to Early Surgery of Small Papillary Thyroid Cancer: A Retrospective Study on a Korean Population.

BACKGROUND: Recently, active surveillance (AS) has been introduced as an alternative to early surgery (ES) for the management of papillary thyroid microcarcinoma (PTMC), because of its indolent features and low mortality. However, its cost effects have not been determined and the findings of current studies differ, according to each country's medical system. METHODS: A Markov model was constructed to compare the cost-effectiveness of AS and ES, based on a reference case of a 40-year-old patient diagnosed with PTMC. Costs and transition probabilities were derived from previous clinical studies in Korean populations, and the incremental cost-effectiveness ratio (ICER) and net monetary benefit (NMB) were calculated. The willingness-to-pay (WTP) threshold was set at USD 100,000 per quality-adjusted life year (QALY) gained. Sensitivity analyses were conducted to address the uncertainties in the model's variables. RESULTS: From the base scenario, the cumulative costs and effectiveness were both higher in ES than AS. The ICER for ES, compared with AS, was USD 6,619.86/QALY, lower than the set WTP. The NMB difference between AS and ES increased across the stages (USD 5,980 at the first stage and USD 159,667 at the last stage). The ICER increased along with decreasing age and increasing cost of surgery. The higher the ES utility score and the lower that of AS, the more cost-effective ES, with WTP set at USD 30,000. CONCLUSION: In the current Korean medical system, ES is more cost-effective than AS. ES is more cost-effective as it is diagnosed at young age and followed-up for a long time.

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

INTRODUCTION: 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. METHODS: 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. RESULTS: 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%). CONCLUSIONS: 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.

The current state of patient access to new drugs in South Korea under the positive list system: evaluation of the changes since the new review pathways.

Objective: This study aims to provide an up-to-date analysis of the current state of patient access to new drugs in South Korea, focusing on the effect of new review pathways for reimbursement. Methods: We analyzed patients' access to new drugs, listing rate and lead time until listing from marketing authorization. New pathways were defined as 'price negotiation waiver,' 'risk-sharing agreements,' and 'pharmacoeconomic evaluation exemption.' Results: The listing rate for drugs increased after the introduction of the new pathways (93.7% vs. 77.9%, p < 0.001). Before the new pathways, the median lead time for listing was 21.0 months (95% CI: 16.9-25.0), while afterward it was shortened to 10.9 months (95% CI: 10.2-11.7) (p < 0.001). Conclusion: Although it has strengthened national health insurance coverage by positively impacting the rate and lead time, the lead time for the oncology and orphan drugs is substantially longer as compared to other drugs. Expanding the eligibility criteria to include non-life-threatening but rare or intractable diseases, and resolving the system's operational issues are still necessary.

Trends in the pricing and reimbursement of new anticancer drugs in South Korea: an analysis of listed anticancer drugs during the past three years.

Objective: This study aimed to examine patient accessibility to new anticancer drugs including reimbursement coverage, time to listing, and listing price during the recent 3 years after the introduction of alternative pricing and reimbursement pathways in South Korea.Methods: Anticancer drugs were selected for analysis from the new drugs reviewed from January 2017 to March 2020. Descriptive statistics were used to present the levels of the listing prices. Pearson's correlation analysis was used to analyze the relationship between the list price in comparison to the External Reference Price(ERP) and the time to listing.Results: Thirty-two anticancer drugs were included in analysis. The average time to listing for these drugs was 36.7 months. The ratio of the listing price in comparison with Average Adjusted Price from seven reference countries was from 12.6% to 90.2%. Pearson's correlation coefficient for the correlation between the ratio of the listing price to the ERP and the time to listing was -0.37 and was statistically significant (p = 0.035).Conclusions: Policies that relate to the scope of reimbursement, time to reimbursement, and list price should be able to equally reflect patient accessibility and national health insurance finances, as well as the impact on industry as a whole.

Post-metastasis survival in extremity soft tissue sarcoma: a recursive partitioning analysis of prognostic factors.

BACKGROUND: Recursive partitioning analysis (RPA) enables grouping of patients into homogeneous prognostic groups in a visually intuitive form and has the capacity to account for complex interactions among prognostic variables. In this study, we employed RPA to generate a prognostic model for extremity soft tissue sarcoma (STS) patients with metastatic disease. METHODS: A retrospective review was conducted on 135 patients with metastatic STS who had undergone surgical removal of their primary tumours. Patient and tumour variables along with the performance of metastasectomy were analysed for possible prognostic effect on post-metastatic survival. Significant prognostic factors on multivariate analysis were incorporated into RPA to build regression trees for the prediction of post-metastatic survival. RESULTS: RPA identified six terminal nodes based on histological grade, performance of metastasectomy and disease-free interval (DFI). Based on the median survival time of the terminal nodes, four prognostic groups with significantly different post-metastatic survival were generated: (1) group A: low grade/metastasectomy; (2) group B: low grade/no metastasectomy/DFI ⩾ 12 months or high grade/metastasectomy; (3) group C: low grade/no metastasectomy/DFI < 12 months or high grade/no metastasectomy/DFI ⩾ 12 months; and (4) group D: high grade/no metastasectomy/DFI < 12 months. The 3-year survival rates for each group were: group A, 76.1 ± 9.6%; group B, 42.3 ± 10.3%; group C, 18.8 ± 8.0%; and group D, 0.0 ± 0.0%. CONCLUSION: Our prognostic model using RPA successfully divides STS patients with metastasis into groups that can be easily implemented using standard clinical parameters.

Comparison of combined hepatocellular and cholangiocarcinoma with hepatocellular carcinoma and intrahepatic cholangiocarcinoma.

PURPOSE: Combined hepatocellular and cholangiocarcinoma (HCC-CC) is a rare primary hepatic neoplasm (PHN) with features of both hepatocellular and biliary differentiation. We compared the outcome of hepatic resection in patients with HCC-CC, those with hepatocellular carcinoma (HCC), and those with cholangiocarcinoma (ICC). METHODS: Between November 1994 and December 2003, 952 patients underwent hepatic resection for a PHN. RESULTS: The incidence of HCC-CC was 3.5%. Hepatitis B surface antigen was positive in 51.2% of these patients and the HCV antibody was positive in 12.2%. Positive hepatitis serology was more common in the HCC group (66.7%). The prevalence of underlying liver cirrhosis was significantly lower in the ICC group (7.8%) than in the HCC (49%) and HCC-CC (41.5%) groups (P < 0.0001). The median overall survival periods after hepatic resection of HCC-CC, HCC, and ICC were 47.3, 71.7, and 21.5 months, respectively (P < 0.0001). The median disease-free survival (DFS) periods after hepatic resection for HCC-CC, HCC, and ICC were 23.4, 68.2, and 15.5 months, respectively (P < 0.0001). CONCLUSION: Patients with transitional type HCC-CC had significantly poorer survival rates than those with HCC, after hepatic resection. Therefore, a more aggressive treatment modality should be explored to improve the survival rate of these patients.