Study On The Policy Of Drug Security For Rare Diseases

Objective:To analyze the situation of registration,medical insurance payment,procurement and use of orphan drugs.To summarize the characteristics of health care insurance modes for rare diseases in various regions of China,and to analyze the economic burden of patients with rare diseases under different policy modes.To predict the budget impact of orphan drugs which have been included in the National Basic Medical Insurance Medicine Catalogue and simulate a accessible path to medical care for rare diseases,in order to provide reference for the improvement of medical insurance policies for rare diseases.Methods:The basic information of orphan drugs involved in“the First Batch of Rare Diseases Catalogue”was obtained by searching public database.By searching policy documents of Zhejiang,Chengdu,Shanxi,Qingdao,Foshan and other areas,the characteristics of health care for rare diseases modes in China were summarized according to types of medical insurance,financing situation,reimbursement list and reimbursement proportion.The national drug procurement data were obtained from the National Health Commission,among which procurement and use of some orphan drugs from 2017 to 2019 were described in the ratio and constituent ratio.Based on the operation data of basic medical insurance for urban and rural residents in Xianning City,Hubei Province from 2015 to 2019,this study used the budget impact analysis to analyze the impact of orphan drugs’being involved in the National Basic Medical Insurance Medicine Catalogue,and analyze the accessible path of rare diseases based on the practice of Zhejiang Province.Results:1.The situation of market access and medical insurance payment of drugs which have been involved in“the First Batch of Rare Diseases Catalogue”:By March 31^th,2021,75 rare diseases in“the First Batch of Rare Diseases Catalogue”which have specific therapeutic drugs have been marketed worldwide,among which 19 drugs of 16 rare diseases have been marketed in the United States,the European Union,Japan and other countries and regions,but no drugs have been approved in China.In China,99 drugs of 59 rare diseases have been approved,and 75drugs of 39 rare diseases have registered for rare disease indications,and 50 drugs of25 rare diseases have been included in the National Basic Medical Insurance Medicine Catalogue,among which,13 drugs of 9 rare diseases and 37 drugs of 23rare diseases are fully and partly reimbursed respectively.In addition,24 drugs of 30 rare diseases had the problem of off-label drug use,among which all drugs in the catalogue of 20 rare diseases（a total of 18 drugs）had this problem.2.Summary of modes of medical insurance for rare diseases in China（1）Special fund model:Take Zhejiang Province and Chengdu as representatives.Zhejiang province raises funds by individual contribution（2 yuan per person annually）and from critical illness insurance and pays cost by annual accumulative and piecemeal reimbursement,with the patients’personal burden capping at 100,000 yuan.In Chengdu,2%of the total amount of critical illness insurance was raised last year,with the maximum limit of 400,000 yuan for medical insurance and 60,000 yuan for patients respectively.（2）Special drugs for critical illness model:Take Shanxi Province and Hunan Province as representatives.Orphan drugs are included in the special drug list of basic medical insurance or critical illness insurance,and are paid according to a certain proportion of drug classification.（3）Financial contribution model:Take Qingdao as the representative.Funding is mainly based on financial contribution.For special drugs included in the insurance scope,there is no minimum or maximum limit for payment,and the reimbursement rate is 80%.（4）Commercial supplementary insurance model:Take Guangzhou as the representative.Orphan drugs included in the insurance scope will be reimbursed in a certain proportion on the basis of the deductible amount through individual voluntary payment,with the annual guarantee amount ranging from 100,000 yuan to 1 million yuan.（5）Medical assistance model:Take Foshan as the representative.Funds are raised from medical assistance fund.The expenses can be reimbursed at the proportion of 80%after the reimbursement of basic medical insurance,critical illness insurance and supplementary insurance,with a maximum limit of 300,000 yuan annually.3.Procurement and use of orphan drugs in China from 2017 to 201945 orphan drugs were purchased in 2019.From 2017 to 2019,DDDs of orphan drugs were 18.400 billion,123.569 billion and 140.765 billion respectively,and expenditures were 10.516 billion,15.271 billion and 19.153 billion yuan respectively.The actual number of patients using drugs covered by the purchase amount of rare disease drugs accounted for the proportion of the estimated number of patients with the same rare disease in China.The lowest proportion was less than 0.01%（Iloprost）and the highest was 5.15%（Bosentan）.4.Budget impact analysisAfter involving xaltrexate,imidase and alabidase in the National Basic Medical Insurance Medicine Catalogue,the expenditure of the basic medical insurance fund for urban and rural residents in Xianning will increase by 101.456～198.578 million yuan,accounting for 0.36%～0.70%in total expenditure of medical insurance fund and 2.01%～3.94%in accumulated balance amount respectively,which will make the fund failing to meet its expenses,and may cause deficit.Conclusion:（1）The approval and registration of orphan drugs in China need to be improved,and the problem of off-label drug use need to be tackled by“Three Medical Linkage”.（2）The rare disease insurance policy in China has limited coverage and regional differences,so it is urgent to establish the principle and consensus of drug security for rare diseases in China.（3）The basic medical insurance cannot meet the needs for orphan drugs.Drug insurance for rare diseases requires the joint action of various types of supplementary medical insurance,so that they can connect and complement each other well.