ObjectivesTo provide a reference for the evaluation procedures of genetic testing technology applicable to China by combining the existing evaluation frameworks and procedures for genetic testing techniques globally, and also put forward design suggestions for the construction of evaluation procedures in China.MethodsThe literature research method was primarily used to summarize different evaluation progress, as well as put forward design suggestions.ResultsAt present, numerous developed countries have organized genetic testing technology evaluation projects. The various evaluation frameworks developed were based on the ACCE or HTA framework. The evaluation and decision-making procedures were similar in general, including topic selection, evaluation implementation, results reporting and making recommendations. However, there still remained difficulties such as limited evidence and uncertainty in decision-making.ConclusionsTo establish the procedures of genetic testing technology applicable in China, the following specific procedures are recommended: selecting target genetic testing technology topics; analyzing necessity and feasibility of target testing technology evaluation; evaluating and reviewing the evidence; applying results and decision-making transformation; developing regular review and revision mechanisms.
Survival prognosis in patients with terminal cancer plays an important role in clinical decision-making, policy formulation, and end-stage patient with relatives. To date, foreign researchers have developed multiple survival prediction models based on patient clinical performance, biomarkers and other indicators, along with a large number of studies which have been externally verified, including Palliative Performance Scale (PPS), Palliative Prognostic Score (PaP), Delirium-Palliative Prognostic Score (D-PaP), and Palliative Prognostic Index (PPI), etc. China's research on this topic remains in the primary stage. Therefore, this article reviews the prognostic factors of terminal cancer and survival prediction models as well as applications, in order to provide references for the subsequent construction of survival prediction models for patients with terminal cancer in line with Chinese characteristics.
ObjectivesTo initially construct a scientific, reasonable and precision medicine technology value judgment framework suitable for China’s national conditions based on expert consultation method, so as to provide scientific value judgment system support for China's medical insurance decision-making.MethodsThe preliminary evaluation indicator system for precision medicine technology value was established by using literature analysis and expert consultation method, and the direct weighting method was used to determine the indicators weight.ResultsAfter two rounds of expert consultation, an indicator system suitable for the value judgment of precision medicine technology in China was constructed, including 5 primary indicators (health needs, health effects, economics, innovation and suitability) and 14 secondary indicators. Each indicator was weighted according to importance.ConclusionsA set of precision medicine value judgment indicator system suitable for China has been initially established, which lays a certain foundation for further measurement research of the indicator system and provides a scientific basis for medical insurance decision-making.
ObjectiveTo systematically review the economics evaluation studies on the early screening or diagnosis of primary immunodeficiency diseases (PID). MethodsWeb of Science, CRD, PubMed, The Cochrane Library, CNKI and WanFang Data databases were electronically searched to collect the economics evaluation studies on the early screening or diagnosis of PID from inception to July 1st, 2022. Two reviewers independently screened literature, extracted data and assessed the risk of bias of included studies, then, a descriptive systematic review was performed. ResultsA total of 10 studies focusing on SCID were included. The results showed that under a relatively high threshold, the early screening and diagnosis of SCID were cost-effective, which can reduce severe infections in patients and treatment costs while improving patient’s survival. ConclusionCurrent evidence shows that early diagnosis of PID can reduce costs and improve benefits. Due to limited quality and quantity of the included studies, more high quality studies are needed to verify above conclusion.
ObjectiveTo systematically review the pharmacoeconomics of hemodialysis and peritoneal dialysis in patients with end-stage renal disease. MethodsCRD, NICE, CADTH, HITAP, NECA, IWIQG, ISPOR, PubMed, EMbase, CNKI, and WanFang Data databases were electronically searched to collect pharmacoeconomic studies on the hemodialysis and peritoneal dialysis in the treatment of end-stage renal disease from inception to December 2020. Two reviewers independently screened literature, extracted data, and assessed the risk of bias of included studies. Then, the conclusions of research models, pharmacoeconomic evaluation results, and sensitivity analysis were summarized. ResultsA total of 15 pharmacoeconomic studies were included, among which 9 studies used the Markov state transition model, and 6 were observational studies. From the perspective of health outcomes, peritoneal dialysis had cost-effectiveness advantages over hemodialysis in the treatment of end-stage renal disease under the condition of a clear threshold. ConclusionsCurrent evidence shows that compared with hemodialysis, peritoneal dialysis has certain cost-effectiveness advantages in the treatment of end-stage renal disease. Due to limited quality and quantity of the included studies, more high-quality studies are required to verify the above conclusions.