Study Of Management Strategies Of Improving Access To Orphan Drugs In China

Aim:In recent years, increasing reports concerning shortage of drugs for rare disease treatment have been seen in all kinds of media. The drug accessibility of rare disease patients is not satisfying. Their health can not be assured. The present study analyzed the current medication status of rare disease patient in China, the international orphan drug management system and the orphan drug policy construction environment, aiming to improve the medication accessibility of Chinese rare disease patients.Methods:Literature and evidence-based studies were conducted based on need. In literature-based study, description study and comparative analysis were used; in evidence-based study, existing statistics analysis, classic-case analysis, expert interview were used.Main results:1. Definition of rare disease and orphan drug According to WHO, rare disease refers to those the incidence is with 0.65‰～1‰. So far, there are 5000～6000 kinds of rare disease reported, accounting for 10% of all human diseases. Among which, 80% are genetic diseases, 90% are critical diseases. although the incidence of a certain rare disease is low, but the total population affected is huge. The total affected population is around 50～60 million. Currently, most of the rare disease patients didn't receive proper treatment. Orphan drugs are those intended to diagnose, prevent or treat rare diseases or pathologies that are serious or life threatening, and whose development costs are superior to the expected return on investment. Due to the low marketing and low profit and high cost, there is almost no drug company would like to invest on the R&D, production and supply. It is very necessary for the government to intervene to improve the accessibility of orphan drugs.2. Definition of accessibility of drugs for rare diseases we define the accessibility as the process of systemically transfer the drug from substance form to health value. This includes three steps: drug existence, drug availability, and the rpoper usage of the drugs. Five key factors were proposed to affect the accessibility of orphan drugs: availability, utility, deliverability, affordability and systemic reactivity.3. The current availability of orphan drug in China During 1983-2007.12, there are 254 orphan drugs for 315 rare diseases, among which, 130 were available in China, accounting for 51.18%. The orphan drugs for the top 8 systems involves 95 rare diseases and 178 orphan drugs in America, Japan and Europion Union. Among which, 85 were marketed in China, accounting for 47.75%.4. The research funding for rare disease in China During 1999-2007, there are 366 grants were funded by national natural science foundation of China for rare disease research, accounting for 89,358 thousand RMB. Among which, 63 grants were for drug research, accoungting for 14,870 thousand RMB and 16.7% in percentile. The rare diseases of digestive and metabolic system were funded relative lower, accounting for only 0.27%, 0.82% respectively. The rare diseases of hematopoietic system were funded relative higher, accounting for 66.12% in grant number and 69.41% in funding. There are seven"863"or"973"grants involving rare disease research. 15 grants were funded to pharmaceutical company to conduct orphan drug research. So far, the funding for orphan drug R&D is mainly form government and lack of systemic planning. The funding is too few and effect is not satisfying.5. Analysis of the factors affecting the accessibility of the medication of rare disease(1) utility disorder: not easy to diagnose; no proper drugs to use; too expensive; lack of proper information for rare disease;(2) production, supply and usage disorder; lack of profit motivation; lack of standard knowledge for rare disease;(3) affordability: long treating circle; lack of health insurance; high economic burden;(4) systemic reactivity disorder: no information platform for rare disease and orphan drugs; no standard training program for rare diseases;6. The analysis of foreign policies to improve orphan drug accessibility Industry motivation-Oriented public health policy. Combination of medical science, biotechnology, motivation of new drug R&D, risk investment policy, shortage-supply protocol, health insurance, rare disease information protocol, doctor training program.Conclusions: to improve the health status of rare disease patients, the following 10 aspects is noticed,(1) strengthen the government's responsibility on rare diseases;(2) clarify the definition of rare disease and orphan drug in china;(3) establish the information system for rare disease and orphan drug in China;(4) establish the stimulating mechanism for the R&D, production, supply and use of orphan drugs;(5) establish the hospital-oriented supply contract for orphan drugs;(6) improve the diagnosis and treatment level for rare disease;(7) strengthen the health education about rare disease and orphan drug; (8) improve the health insurance protocol concerning rare diseases;(9) increase orphan drug preservation;(10) establish the orphan drug review system and seek more international cooperation.Innovation of research(1) Study orphan drug management from patient drug access view,introducing health system reaction indicator firstly;(2) Setting up an orphan drugs supply international information data for 178 orphan drugs of eight disease types;(3) Some new ideas on solving orphan drug access.Such as drug access-Oriented policy system,reasons analysis of orphan drug policy from Interest Groups Theory and Mechanism Design Theory , pharmaceutical industry low interest loan policy, Public-Private Partnerships Financing Pattern,advising clinical service centres for rare diseases,promoting community health effective intervention level for rare disease, hospital-based orphan drugs supply protocol, reserving orphan drug approval number regulation for pharmaceutical company .