International rare disease day: the situation of difficult and expensive drug purchase for patients will be improved

[China Pharmaceutical network industry trends] February 28 is the "international rare disease day" The rare disease is also called orphan disease, which has the characteristics of less clinical cases and less experience, leading to high misdiagnosis, high missed diagnosis and difficult medication WHO (WHO) defines rare diseases as diseases with 0.65-1 incidence rate The global global estimates of 300 million rare diseases are about 7000-8000 rare diseases identified But for the definition of rare diseases, the standards of different countries and regions are different It is estimated in the industry that there are nearly 20 million cases of rare diseases in China, but the drugs and treatment standards for rare diseases are still lacking In terms of drugs for rare diseases, due to the high cost of research and development of patients and drugs for rare diseases, it is difficult for pharmaceutical companies to actively participate in the production of drugs for rare diseases, which leads to the situation that it is difficult and expensive for some patients with rare diseases to buy drugs At present, however, the situation is improving On the one hand, the speed of review and approval of imported drugs was accelerated to further meet the drug demand of patients For example, at the end of 2018, the State Food and Drug Administration approved the application for registration of the new imported drug for the treatment of hemophilia type A The news that the drug passed the approval of "green channel" brought good news to hemophilia patients On January 11 this year, China held a meeting to guarantee the medication for 20 million patients with rare diseases At that time, the 20 million patients with these rare diseases will no longer have no medicine available On the other hand, the drug burden of patients will be further reduced On February 22, the drug administration and other four ministries and commissions announced the notice on the value-added tax policy for drugs with rare diseases It is mentioned that from March 1, 2019, the general VAT payers can choose to pay the VAT at a simple rate of 3% for the production, sale, wholesale and retail of rare disease drugs For the imported drugs with rare diseases, the import value-added tax shall be reduced by 3% According to the industry, the 3% tax on drugs with rare diseases will further encourage the development of the pharmaceutical industry with rare diseases and reduce the burden on patients In addition to focusing on rare disease treatment drugs, disease treatment has also attracted the attention of relevant departments It is understood that rare diseases are often misdiagnosed because they are rare For example, multiple sclerosis is also a rare disease, its early symptoms are complex, and young people are more sick, treatment interruption is easy to relapse, also easy to be misdiagnosed as other diseases Industry data shows that about 38% of multiple sclerosis patients in China are misdiagnosed as cerebrovascular disease, myelopathy and other diseases Therefore, the standard treatment of this kind of disease needs to be promoted urgently In order to attach importance to rare diseases, in 2016, Beijing Union Medical College Hospital took the lead to establish a national rare disease registration platform with unified standards in 19 hospitals including West China Hospital At present, more than 120 rare diseases have been registered on the platform On May 22, 2018, the national health and Health Commission, the Ministry of science and technology, the Ministry of industry and information technology, the drug administration and other five departments jointly issued the catalogue of rare diseases, involving 121 kinds of diseases, including albinism, amyotrophic lateral sclerosis, angel syndrome, Zener syndrome, autoimmune encephalitis, biotinase deficiency, heart free passage disease, congenital myasthenia syndrome, Fabre disease, and Gaucher disease At the end of 2018, with the support of the national health and Health Commission, Beijing Union Medical College Hospital, China Association of hospitals, China Association for the promotion of pharmaceutical innovation and other institutions launched the China rare diseases alliance, which will explore Chinese programs for patients with rare diseases With the establishment of China rare diseases alliance and the recognition and popularization of rare diseases, it is believed that the clinical misdiagnosis will be reduced in the future.