RESUMEN
At present, we are facing challenges in the development of rare disease drugs. The paper introduces the ethical theories of rare disease treatment, the characteristics of pharmacoeconomic evaluation on orphan drugs, the risk of reimbursement in medical insurance funds, the international experiences on the balance between the accessibility and cost containment of high-cost orphan drugs, the orphan drug list promulgated by Hong Kong SAR of China in 2019 and the status quo of orphan drugs in China. The author also analyzes the international practice from different perspec-tives, such as, the Orphan Drug Act(ODA)in the United States, the new concept of high specific technology introduced by National Institute Health and Clinical Excellence(NICE)in the UK, the retrospective review of pharmacoeconomics of orphan drugs by National Centre for Pharmacoeconomics(NCPE)in Ireland, the principles of orphan drug evaluation by TLV in Sweden, a special research program on the cost of orphan drugs in Turkey, and the four measures to control the cost of high-priced drugs in the Republic of Korea. The aim of this paper is to introduce international experiences for reference on the formulation of drug policies in rare diseases and development of related pharmacoeconomics in China.
RESUMEN
Shanghai is the earliest city in China for starting family doctor system at national level. It has actively explored the contract service mechanism between residents and family doctors. It has developed from "soft contract signing of guiding residents to understand, contact and gradually accept family doctor services" to the " main and close contracting using the comprehensive service from medical institutions". This paper systematically analyzed the implementation status in " one community health center, one district hospital and one municipal hospital (1 + 1 + 1)" contractservice system of family doctor in Shanghai from the aspects of adjustment background, design ideas, progress and obstacles of the policy. Based on the analysis of barriers to the policyimplementation, some suggestions were put forward including the improvement of the contract service system in order to increase the number and build the capacity of general practitioners, and the optimization of the existing payment distribution systems.
RESUMEN
Objective To analyze the factors influencing the long-term allograft survival in cadaver- ic renal transplantation.Methods Parameter data were obtained from 249 patients who received immuno- suppressive agents after renal transplantation.Health resources were evaluated for 1-5 years;and 14 relevant factors,including age,sex,therapeutic regimen choice and complications,etc were analyzed.Life table method and COX regression model were used to analyze the risk factors influencing the outcomes and to calculate the survival rates.Results Following renal transplantation,the survival rates of recipients who lived for 1,2 and 3 years were 72.6%,56.0% and 40.8%,respectively;and the rates of those who lived for 4 and 5 years both were 22.5%.The median survival time was 34.9 month.With the therapy prolonged,the survival benefit in MMF group was superior to that in AZA group,with the median survival time being 38.9 months 30.6 months,respectively.COX regression model showed that the main predictive factors were treatment regi- men(P=0.000),follow-up period(P=0.000),patient's compliance(P=0.000),acute rejection episode (P=0.020),sex(P=0.001)and hospitalization period(P=0.040).Conclusions Life table and COX regression model are useful methods for evaluating long-term outcome and influencing factors in renal trans- plant patients.