ABSTRACT
OBJECTIVE：To evaluate the price and affordability of rare disease drugs in China and provide the suggestions for the improvement of rare disease drug affordability in order to provide reference for the relevant decision-making of government departments. METHODS ：According to the List of the First Batch of Rare Diseasesand Diagnosis and Treatment Guideline for Rare Diseases（2019 edition），rare disease drugs were selected. The median price ratio （MPR）was used to evaluate the drug price level ， and the ratio of the annual drug costs to the annual disposable income of urban or rural residents was used to evaluate the affordability，and the impact of price management policieson drug prices and affordability. RESULTS and CONCLUSIONS ：A total of 71 kinds of rare disease drugs were included ，and the median MPR of them was 0.83；among them ，the median MPR of the original drugs was 1.13，and the median MPR of the generic drugs was 0.37. The annual cost of 71 rare disease drugs was 0.001-178.43 times the average annual income of urban residents ，and 0.003-456.57 times the average annual income of rural residents. There were 21 and 28 drugs whose annual cost exceeded the annual income of urban and rural residents.After the implementation of national medical insurance negotiation or volume based procurement of 14 drugs，the median MPR of these drugs decreased from 1.71 to 0.46. For urban residents ，the number of unaffordable drugs had been reduced from 8 to 0；for rural residents，it had been reduced from 10 to 5. In summary ，the price level of orphan drugs in China was slightly lower than international reference price ，but the price level of original drugs was higher than the international reference price ，and the cost of some drugs far exceeded the residents ’ability to pay. It is suggested that the government should strengthen the price management of original drugs ，promote the substitution of high-quality generic drugs for original drugs ，and improve the payment system for high-value rare disease drugs.
ABSTRACT
OBJECTIVE:To provide reference for formulating drug price regulation policy and promoting reasonable drug price. METHODS:According to WHO and HAI drug price standard investigation,questionnaire survey(supplemented by field investigation)about sale price of 63 commonly used drugs with large consumption sum was conducted among 3 districts of different economic development levels in Hubei province. Median price ratio(MPR)was calculated statistically. Primary interview survey(supplemented by literature investigation)about drug distribution cost was conducted among managerial staff of pharmaceutical wholesale and retail enterprises,hospital pharmacy administrators. Suggestions were put forward to control distribution cost and standardizing drug price based on analysis and discussion. RESULTS:Totally 120 questionnaires were sent out,and 118 were effectively received,with effective recovery of 98.33%. Thirty managerial staff from drug wholesale and retail enterprises,hospital pharmacy department were interviewed for investigation of drug distribution cost. MPR of 63 drugs ranged 0.05-44.55 in different types of sample institutions. Among 67 specifications of 53 kinds of drugs,median retail price of 38 specifications(56.72%)was higher than international reference price in first-level sample medical institutions. Among 79 specifications of 63 kinds of drugs,median retail price of 57 specifications(72.15%)was higher than international reference price in second-level sample medical institutions. Among 80 specifications of 63 kinds of drugs,median retail price of 63 specifications(78.75%)was higher than international reference price in third-level sample medical institutions. Among 50 specifications of 37 kinds of drugs,median retail price of 42 specifications(84.00%)was higher than international reference price in sample pharmaceutical retail enterprises. In all sample institutions,maximum MPR of 13 specification were lower than 1;those of 12 specification ranged from 1 to <2;those of 23 specifications ranged from 2 to <5;15 specification ranged from 5 to <10;those of 17 specification were higher than 10. MPR of third-level medical institution samples were higher than those of second-level and first-level ones,and pharmaceutical retail enterprises(P<0.01). MPR of second-level medical institution samples and pharmaceutical retail enterprises were higher than those of first-level medical institutions(P<0.01). There was no statistical significance in the levels of MPR between second-level medical institutions and pharmaceutical retail enterprises(P>0.05). MPR of sample institutions in well-developed regions were higher than in medium-developed or less-developed regions(P<0.01). MPR of sample institutions in medium-developed regions were higher than in less-developed regions(P<0.01). MPR of original drugs were all higher than those of generic drugs in different types of sample institutions(P<0.01). There was no statistical significance in MPR of original drugs among different types of sample institutions(P>0.05). At the same time,MPR of generic drugs in third-level medical institution samples were higher than in second-level and first-level ones and pharmaceutical retail enterprises (P<0.05 or P<0.01). MPR of second-level medical institution samples and pharmaceutical retail enterprises were higher than in first-level ones(P<0.05). There was no statistical significance between second-level medical institution samples and pharmaceutical retail enterprises(P>0.05). Drug production and circulation cost,centralized bidding and purchasing price in Hubei province were higher than the national level. CONCLUSIONS:The drug price of Hubei province is in high level,especially that of three-level medical institutions is higher than other institutions;drug price of well-developed region is higher than those of medium-level and less-developed region;the price of original drugs are higher than those of generic drugs. The cost of pharmaceutical industry,drug circulation cost and the addition of drug price during application link eventually lead to the higher retailing price of drugs. Comprehensive policy measures need to be taken to control the cost of the whole process of drug distribution and to regulate the price of drugs.