ABSTRACT
OBJECTIVE： To investigate the feasibility of multi-payment for drugs for rare diseases and also provide reference for rare disease treatment and the formulation of related policy. METHODS： Taking Gaucher’s disease in Qingdao as an example， the relevant medical insurance policies and drug supply were analyzed； according to the sources of financing， the economic burden of treatment drugs for patients with Gaucher’s disease in Qingdao was estimated. Based on the average total cost of patients with Gaucher’s disease in Qingdao， the drug cost burden of patients with Gaucher’s disease in the whole country was simulated according to the relevant epidemiological survey data， and the problems of medical insurance model for rare diseases in Qingdao were analyzed. RESULTS & CONCLUSIONS： A series of medical insurance policies were formulated in Qingdao. The financing mode of medical insurance includes social funds （from Qingdao Charity Federation， drugs are provided by pharmaceutical manufacturers）， medical insurance funds （used for personal self-payment assistance within the scope of medical insurance co-ordination） and civil assistance （used for assistance outside the scope of medical insurance co-ordination）. Imidase is currently the only approved specific drug for Gaucher’s disease in China. The designated physician， treatment and drug-taken system is adopted in Qingdao. By the end of 2017， the annual drug consumption of 8 patients in Qingdao was 38-170 bottles， totaling 686 bottles. The annual cost of treatment ranged from 786 600 to 3 519 000 yuan， totaling 1 420 200 yuan. The self-paid expenses ranged from 9 800 to 197 400 yuan （the self-paid ratio ranged from 0.46％ to 8.87％）， totaling 661 400 yuan. The reimbursement cost by supplementary medical insurance was 509 800 to 1 789 800 yuan （accounting for 54.59％ to 65.94％）， totaling 8 577 800 yuan. Three patients received civil assistance， the amount of which ranged from 23 100 to 13 000 yuan （accounting for 1.89％ to 4.18％）， totaling 241 400 yuan. According to the relevant epidemiological survey data （the prevalence rate was 0.15 per 100 000）， it is estimated that there are about 2 093 patients with Gaucher’s disease in China. Referring to Qingdao multi-payment model， it is estimated that the total cost of drugs for Gaucher's disease in China is about 3.715 billion yuan， charitable assistance can bear 1.238 billion yuan， while medical insurance expenditure is about 2.255 billion yuan， and individual self-payment is about 222 million yuan. From the point of view of drug cost burden， the multi-payment model in Qingdao has lightened the personal burden of patients’ has achieved significant results. There are problems in Qingdao’s multi-payment model， such as “medical insurance immigration”， low drug accessibility， drug price monopoly， three-level disease prevention needs to be strenghten， etc. The state or provinces and cities can refer to Qingdao model when formulating policies related to rare diseases. Great importance should be paid attention to the existing problems.
ABSTRACT
The ultimate goal of synthetic biology is to build customized cells or organisms to meet specific industrial or medical needs. The most important part of the customized cell is a synthetic genome. Advanced genomic writing technologies are required to build such an artificial genome. Recently, the partially-completed synthetic yeast genome project represents a milestone in this field. In this mini review, we briefly introduce the techniques for de novo genome synthesis and genome editing. Furthermore, we summarize recent research progresses and highlight several applications in the synthetic genome field. Finally, we discuss current challenges and future prospects.
Subject(s)
Animals , Humans , CRISPR-Cas Systems , Gene Editing , Methods , Genetic Engineering , Methods , Genome, Human , High-Throughput Nucleotide SequencingABSTRACT
Objective To investigate the clinical efficacy of percutaneous nephrolithotomy with holmium laser(YAG laser lithotripsy)for the treatment of renal calculi.Methods Percutaneous puncture was performed under continuous epidural anesthesia,and then a pyeloscope(Wolf F8.5/11.5)was inserted and lithotripsy was carried out by using 200-?m holmium laser fiber(0.6-1.2 J/6-10 Hz).After the procedure,the pieces of the calculi were removed.After the operation,renal fistula was maintained for 3-5 d,urethral catheter was retained for 5-7 d,and double-J catheter was kept for 4-8 weeks.Results Stone removal was achieved with one-stage operation in 130 cases [mean operation time,60 minutes(40-150 minutes);and mean hospital stay,8.5 d(7-10 d)];with two-stage operation in 15 cases [mean hospital stay,14 d(12-16 d)];and with three-stage operation in 5 [mean hospital stay,17 d(15-20 d)].In all the 150 cases,by using single tract,stone-free rate reached 86.0%(129/150)after the first-stage operation,and final stone-free rate was 92.0%(138/150).None of the patients had severe complications after the operation.Conclusion Percutaneous nephrolithotomy by using holmium laser is minimally invasive,effective and safe for patients with renal calculi.