Health is the inevitable requirement to promote the all-round development of people,and accurate assessment of drug economy and scientific allocation of medical resources are the prerequisites for the sustainable development of medical and health care.Rare diseases are a type of disease with a low prevalence rate.Market of drugs is small because the number of patients is small.Drugs with only rare disease indications,also named orphan drugs,cannot meet the payment threshold of economic evaluation due to high prices and are difficult to be included in National Reimbursement Drug List.However,some orphan drugs can greatly improve the health of serious patients,which are of high economic and social value.Therefore,it is necessary to explore the deviation of traditional payment thresholds in evaluating orphan drugs with good therapeutic effects,and adjust the payment threshold reasonably to accurately assess the value of orphan drugs.Firstly,this study explores whether the payment threshold of economic evaluation can measure the value of drugs improving the health of seriously ill patients with almost zero initial QALYs,and proposes a theoretical method to adjust payment threshold.Secondly,this study carried out a questionnaire survey using person trade-off technique(PTO)to verify the S-curve relationship between incremental QALYs and health utility.Thirdly,applying the verified adjustment method of payment threshold to the evaluation of orphan drug,and calculating the relative payment threshold of orphan drugs relative to drugs that can only slightly improve the health of seriously ill patients.Finally,this study analyzes the admission of orphan drugs and its impact on medical insurance funds and health benefits,and provides a reference for improving the reimbursement of orphan drugs.Results of theory analysis showed that for seriously ill patients,as their health status improves,due to the cumulative effect of health improvement,the marginal health utility corresponding to each unit of QALY increases until the patient can normally participate in various social activities and then decrease,which indicates incremental QALYs and health effects show S-shaped curve.The survey results confirmed that incremental QALYs and health utility showed an S-shape.The marginal health utility reached the maximum when the incremental QALYs was about 9,the health utility was about 10.31,and the marginal health utility was 1.25.The adjusted payment threshold for 7 orphan drugs is 1.34 to 1.67 times that of cancer drugs.In the case of negotiating price cuts of 35.73% to 99.36%,the admission of 7 orphan drugs will increase the expenditure of the medical insurance fund by 1.08 to 2.27 billion,which can save 65673.00 QALYs and increase the utility of 72467.96.Comprehensive theoretical analysis and empirical research results shows that traditional payment threshold underestimate the value of orphan drugs that can greatly improve the health of patients with rare diseases,and the payment threshold should be adjusted according to the treatment effect.Therefore,it is recommended that government departments promote the development of basic research to provide data support for the economic evaluation of orphan drugs in China.Secondly,to adjust the payment threshold according to the treatment effect of orphan drugs,which can be achieved by setting the increment QALYs interval and weight,and set different payment thresholds for different orphan drugs.Thirdly,to clarify the contradictions in medical insurance price negotiations of orphan drugs,and improve the negotiation mechanism.Fourthly,to set reimbursement methods reasonably for orphan drug according to the admission and cost of orphan drugs,and do good connection with other medical insurance policies to improve the availability of orphan drugs. |