Health technology assessment in traditional and complementary medicine: a scoping review of international activity and examples of acupuncture.

BACKGROUND: Traditional therapies are crucial in maintaining and improving human well-being. China's healthcare policymakers are attempting to use health technology assessment (HTA) as a decision-making supportive tool. The value assessment framework for Chinese patent medicine (CPM) has been developed and is being adopted and validated widely by research institutions. Subsequently, the healthcare decision-makers particularly hanker for the value framework of traditional non-pharmacological therapies. METHODS: To construct a practical value framework for traditional non-pharmacological therapies, a scoping review methodology was adopted to identify the evaluation domains and obstacles. A search, screening, and analysis process was conducted according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews (PRISMA-ScR). Evidence was retrieved from scientific databases and HTA agencies' websites. RESULTS: The search strategy identified 5 guidelines records and 17 acupuncture HTA reports. By synthesizing the valuable reports of CPM and acupuncture evaluation in representative countries, this study found that Mainland China was promoting the comprehensive value assessment of CPM, whereas the United Kingdom, Singapore, Canada, the United States, and Malaysia had carried out the HTA evaluation of acupuncture for various conditions among which chronic pain was the most common. UK and Singapore applied the HTA results to support acupuncture reimbursement decisions. Three domains, including safety, effectiveness, and economy, were commonly adopted. The identified biggest challenge of evaluating traditional non-pharmacological therapies is the scarce high-quality clinical evidence. CONCLUSIONS: This study identified value domains and issues of traditional therapies, and pointed out future research implications, to promote the development value framework of traditional therapies.

Budget Impact Analysis of Nusinersen for Spinal Muscular Atrophy in China.

OBJECTIVES: This study aimed to evaluate the potential budget impact of nusinersen in the treatment of spinal muscular atrophy (SMA) from the perspective of the basic medical insurance payer in China. METHODS: A budget impact analysis model was developed according to ISPOR budget impact analysis guidelines; we integrated health resource consumption, epidemiology, and market data for the treatment of patients with SMA in China, to estimate the impact of nusinersen on basic medical insurance. The microcosting method was conducted to evaluate the treatment cost. One-way sensitivity analysis was performed to explore the stability of the results. RESULTS: If nusinersen could be reimbursed, the impact on the medical insurance funds in the next 1 to 3 years will be 253 million, 333 million, and 426 million, respectively. In addition, it can reduce 0.73 million, 2.17 million, and 2.22 million in complication costs, respectively. CONCLUSIONS: SMA is a rare disease with a very low prevalence; reimbursement of nusinersen will lead to a limited impact on the basic medical insurance and improve the accessibility of treatment.

Assessment of health-related quality of life in patients with spina muscular atrophy in China.

Spinal muscular atrophy (SMA) is a rare disease that has attracted considerable interest in China due to its severity and hefty treatment costs. Few studies have been conducted on Chinese patients. The objective of this study was to assess the quality of life of SMA patients in China and to investigate the real impact of new treatments. We used the Pediatric Quality of Life Inventory (PedsQL) to analyze the Health-related quality of life (HRQoL) of patients with SMA in China. Information on demographics, disease-specific characteristics, and treatment were collected using a child-reported or proxy-reported questionnaire. The mean scores of HRQoL for the Nusinersen treatment group and conventional treatment groups are 55.6 and 48.4, respectively. Patients with SMA type I have the lowest scores, while those with type III have the highest scores. A higher proportion of the medication group showed improvement in the condition in the past six months (56.9% vs. 17.1%). Our results show that the clinical type, motor function and treatment strategy have a significant influence on HRQoL. The findings imply that Nusinersen benefits patients by slowing the progression of the disease and increasing their quality of life in the real world.