Let "orphan medicine" no longer be lonely

Source: on February 19, 2016, xinhuanet.com, according to the definition of the World Health Organization, rare disease is a single disease or pathological change with the number of patients accounting for 0.65 ‰ - 1 ‰ of the total population, while "orphan drug" refers to the drug used to prevent, treat and diagnose rare diseases In terms of availability and affordability, the current situation of orphan drugs in China is not optimistic Drugs are basically imported, with a small number of products on the market, far from meeting the demand for treatment of rare diseases Moreover, they are expensive, few in the list of basic drugs and medical insurance, and the proportion of reimbursement is not high For example, there are about 1000 patients with Gaucher's disease in China, but there are no domestic drugs for the treatment of this disease, which are completely dependent on imports The drug cost of an adult patient is more than 2 million yuan per year, and it needs to be used for a lifetime Few patients with Gaucher disease can buy their own drugs Only by donating drugs to some Chinese patients through charities, can only meet the treatment needs of a few patients The experience of some countries shows that legislation has an immediate effect on the R & D and production of orphan drugs These countries promote the research and development of "orphan drugs" through tax credit, registration fee exemption and priority review, and at the same time give "orphan drugs" market monopoly and medical insurance policy support The United States provides that orphan drugs enjoy a 50% tax deduction preference in the clinical research and development period, which can be extended for 3 years forward and 15 years backward The total tax deduction can reach 70% of the total cost of clinical research In addition, it also provides that the registration fee of orphan drugs can be reduced, which allows them to price separately and give them 7-year market monopoly Compared with the increasingly sound laws and regulations for the research and development of "orphan drugs" in developed countries, China's relevant policies and legislation are not perfect, and there are almost no incentive policies in tax credit and other aspects, which leads to the low enthusiasm of pharmaceutical enterprises in the research and development and production of "orphan drugs" To make orphan drugs no longer lonely, we should learn from foreign experience, establish a recognition system, and regard this recognition as the pre-processing of the registration and approval of orphan drugs At the same time, simplify the review and approval procedures of "orphan drugs" and formulate the implementation rules of "orphan drugs" priority review, including clinical trial relief and special approval In addition, the state may set up a special fund to support the research and development of original "orphan drugs", encourage the imitation of foreign patent expired "orphan drugs", and formulate preferential policies for market monopoly, independent pricing and tax deduction of "orphan drugs" The medical security system for rare diseases also needs to be strengthened at the same time Some rare diseases should be included in the serious disease insurance, and "orphan drugs" should be included in the national medical insurance drug catalog as special drugs.