The usage of enzyme replacement treatments, economic burden, and quality of life of patients with four lysosomal storage diseases in Shanghai, China

Background Lysosomal storage diseases (LSDs) are a group of rare diseases that caused progressive physical dysfunction and organ failure, which significantly affected patients’ quality of life. Enzyme replacement treatments (ERTs) are now acknowledged as the advanced therapies for LSDs while cost millions per patient per year. Previous studies seldom reported the usage of ERTs and disease burden of patients with LSDs in China. The objective of this study was to explore the characteristics and usage of ERTs of patients with the four different LSDs (Gaucher, Fabry, Pompe disease and Mucopolysaccharidosis) in Shanghai and then evaluate the economic burden and quality of life of these patients. Methods The study used data extracted from a large survey of living conditions of patients with rare diseases in Shanghai, which was conducted from April to August 2020. A total of 31patients, involving 5, 14, 4 and 8 patients with Gaucher, Fabry, Pompe disease and Mucopolysaccharidosis, respectively, was included in analysis. Descriptive statistics was used to describe the socio-demographic information (age, gender, education and etc.), economic burden caused by LSDs (direct medical and non-medical costs, and indirect cost in 2019), the treatment (usage of drugs) and the patients’ quality of life. Results Five Gaucher disease patients in Shanghai used Imiglucerase in 2019, while the other 26 patients with the other three LSDs didn’t receive ERTs. The total health expenditure of Gaucher disease patients was 2,273,000CNY on average mainly resulted by the high cost of Imiglucerase. The total health expenditure of the other 26 patients was 37,765CNY on average. The average total disease burdens of Gaucher disease patients and the patients with the other three LSDs were 164,301CNY and 58,352CNY, respectively. The mean EQ-VAS score of GD patients was 76.4 ± 15.5, which was higher than that of the other three LSDs. All the patients with LSDs in this study reported poor quality of life, which was significantly worse than the Chinese general population. Conclusion Few patients with LSDs in Shanghai could have access to available ERTs without a high reimbursement level. Though the cost-sharing mechanism of basic medical insurance, charity fund and patients had been explored for Gaucher disease in Shanghai, the Out-of-pocket part still laid a heavy economic burden on the patients and their families. The scope of drug reimbursement list and the reimbursement level should be further expanded and raised to help improve the quality of life of patients with LSDs.

Participants’ satisfaction on China's New Rural Cooperative Medical System:a cross-sectional Survey in Liaoning province of China.

Background:China launched New Rural Cooperative Medical System (NRCMS) in 2003. However, satisfaction and voluntary participation in NRCMS of Liaoning province was unknown after ten years launching of NRCMS. Methods: In 2014, a total of 1500 individuals from 12 general hospitals in Liaoyang City, Dalian City, Panjin City and Tieling City of Liaoning Province were investigated using self-administered questionnaire by trained staff. Univariate and multivariate logistic regression analyses were applied to find out the satisfaction with NRCMS and its influencing factors among population from Liaoning province. Results: Of all respondents, 180 individuals (13.8%) were highly or relatively satisfied with the program. Moreover, 1092 individuals (84.0%) were fairly satisfied with the program, whereas 29 individuals (2.2%) were not. Multivariate analysis showed the price and range of drug, reimbursement ratio, simplicity of reimbursement procedures and medical technology level of hospitals were the main factors influencing the satisfaction.Conclusion: The overall satisfaction degree of Liaoning province's participating in NRCMS participant is at the middle level. In the process of seeking medical treatment, efforts are made to improve the reimbursement level, simplify the reimbursement procedures, increase the types of drugs in the reimbursement catalogue, and simultaneously take effective measures to control drug prices.

Public Health Insurance with Monopolistically Competitive Providers and Optional Spot Sales

We study the implications of extending public-insurance coverage over differentiated medical products of the same therapeutic group to market outcomes. The public insurer can set the reimbursement level for medical providers and the copayment for the insured for medical care provided under the policy coverage, but cannot directly control providers’ spot sales (outside of insurance) price. In this setup, the price offered by the public insurer to medical providers must maintain their reservation profit from selling on the spot market directly to consumers. We show that the public insurer can manipulate this reservation profit by setting the copayment rate, and thereby promote market welfare while increasing consumers’ surplus due to lower medical prices and lower market entry. The results survive generalizations including moral hazard and incomplete insurance coverage.

The Impact of Medicaid Primary Care Case Management on Office-Based Physician Supply in Alabama and Georgia

The success of the “primary care case management (PCCM)” form of managed care implemented in many state Medicaid programs over the past several years depends in part on the expanded availability of primary care physician sites to substitute for hospital-based outpatient care and to provide a medical home for enrollees. However, the PCCM requirement for physicians to accept assignment of a caseload of patients and to provide all of their primary care likely conflicts with the approach of limited Medicaid participation favored by many Medicaid physician participants. This study examines the early impact of PCCM implementation, in the absence of physician reimbursement level increases, on the patterns of Medicaid participation by physicians in communities in Georgia and Alabama. We find that the implementation of PCCM under these conditions often was associated with reductions in the proportion of physicians participating in Medicaid, reductions in the number of very small Medicaid practices, and declines in Medicaid visit volumes across all participating physicians. We also find evidence of an overall reduction in the number of primary care visits per Medicaid enrollee, but an increase in the proportion of these visits that were for preventive care services associated with initial PCCM implementation.