More than 40 kinds of orphan drugs have been health insurance, but the payment problem still needs to be solved urgently

"Pharmaceutical network industry dynamics" rare diseases, not specially refers to a disease, but the prevalence of a very low prevalence of a large category of diseases, generally chronic, serious, most of the non-intervention will lead to death;
rare diseases in China, more than 80% are genetic defects caused by genetic diseases.
its treatment drugs are generally referred to as "orphan drugs".
because of the small number of patients, in normal market conditions, it is difficult to rely on sales to recover high investment in research and development, so the treatment of rare diseases is difficult to buy and high prices.
years, China has been exploring the establishment of a rare disease drug protection mechanism.
Since the establishment of the basic medical insurance system, the medical insurance department attaches great importance to the medical security of patients with rare diseases, and the national level has issued five editions of the medical insurance drug catalogue, increasing the number of medicines in the catalogue from 1535 to 2709.
the medical insurance catalogue, the scope of drug protection has been expanded and the structure of the catalogue has been optimized.
, the vast majority of rare diseases treatment drugs, as well as haemophilia, idiopathic pulmonary fibrosis, freezing disease and other rare diseases have been included in the medical insurance list.
2019, the national health insurance directory adjustment focused on the treatment of major diseases such as rare diseases, and the primary carnitine deficiency, youth Parkinson's disease, pulmonary hypertension and other rare diseases drugs are also included in the health insurance catalog.
According to incomplete statistics, among the 121 rare disease treatment drugs, there are more than 50 kinds of adaptive drugs listed in China, of which more than 40 kinds of drugs have been included in the national medical insurance drug list, and the price has dropped significantly.
, however, that while more and more rare disease drugs are being covered, they are still well-paid for the more than 7,000 rare diseases that have become clear.
that at present, China's rare disease patients have exceeded 20 million people, more than 200,000 new patients each year, the demand for drugs is huge.
addition, there is no uniform standard for how access to health insurance can be entered through access negotiations because of rare disease drugs, especially those that are those that are those that are those that are those that are those that are those that are those that are priced at sky-high prices.
, industry experts believe that the rare disease is characterized by low number of patients, high research and development costs, high prices, can not just reduce prices to solve the drug price problem.
if the price is reduced, and the price drop is too large, the enterprise will not have the incentive to research and development.
to solve the problem of access to rare disease drugs, on the one hand, to enable enterprises to reduce costs, the pursuit of reasonable profits, on the other hand, but also to build a reasonable reimbursement system.
can be based on individual efficacy, explore the rare disease high-value new drug payment method.
let the medical insurance bureau and the Drug Administration, health care committee and so on, according to clinical trial data to set up individual level efficacy indicators, so that all rare patients treated must register with the national rare disease diagnosis and treatment registration system to be allowed to reimbursement.
In addition, there are many analysts believe that to solve the problem of access to rare disease drugs, can not solve the problem of the payment of rare disease drugs, all to health insurance, health insurance can not afford such a high price, but to explore a variety of payment methods.
In general, in order to solve the protection of rare disease drugs, first, the whole country should establish a unified special protection plan for special rare disease drugs;
through a variety of safeguards, in order to better, longer-term reduction of the burden of rare disease patients medication.