Rare diseases pose a challenge to thepharmaceutical industry

　　"The slow progress of rare diseasedrug development is not just a matter of lack of money." Deng Zhidong,general manager of Hainan Boao Medical Technology Co., Ltd. told the SurfingNews that rare diseases, as the name implies, are rare, difficult to treat, fewand of high value, which means that even if a truly effective drug is developed,there are not that many patients to pay for it. Companies hope to have outputand return after investment, the time period can not be too long, and the drugresearch and development ultimately can not be successful need to put aquestion mark, 98% to end in failure.

　　Deng Zhidong also mentioned that patientdata is the basis for targeted research on diseases, and most rare diseases aredifficult to clarify the pathogenesis, precisely due to the lack ofunderstanding of the causative genes, rare diseases are extremely expensive totreat, patients often give up treatment, and there is no diagnosis and datawithout patients. Many busy doctors are also reluctant to attend trainingrelated to rare diseases because they may only encounter one or two patients ayear, and would rather spend this time doing patients and funding for morediseases.

　　Wang Lei also mentioned the difficulty ofrare disease drugs in the above interview: "Rare disease drugs are verycostly and difficult to find patients because of the low incidence and smallproduction, so it is often a case of waiting for patients with the drugs. Thisis also an area where we want to be able to find rare disease patients as muchas possible and give them the treatment they need, so that rare diseasepatients can be treated early and find a doctor."

　　For local innovative drug companies, howto achieve better commercialization is a challenge for rare disease drugs.Beihai Kangcheng mentioned in its semi-annual report that the current focusremains on expanding the number of patients by creating a stronger diagnosticnetwork. The company plans to expand its internal dedicated commercial team andplans to form a full-fledged rare disease commercialization team in China, ateam capable of commercializing multiple rare disease products.

　　Rare disease drugs are not easy todevelop, and the drugs are often expensive when they are available, so how tomake them affordable for the rare disease community has become an inescapabletopic for drug companies. In the year 2021, for example, the phrase "everysmall group should not be abandoned" was widely resonated, and finally therare disease drug Nocinasan sodium was reduced from 700,000 to 30,000 per shot.2022 national medical insurance negotiations also proposed an appropriate tiltto rare disease patients, and several rare disease drugs passed the preliminaryreview list.

　　For the accessibility of rare diseasedrugs, Wang Lei told the surging news reporter that it is difficult to meet thediagnosis, medication and payment needs of Chinese rare disease patients by oneparty alone, and the country continues to put forward new protection ideas interms of medical insurance and urban customized commercial medical insurance.As a pharmaceutical company, we will also actively support the construction ofa multi-level protection system for rare diseases. Based on strengthening ourown R&D capabilities, we will carry out multi-lateral cooperation tosupport the construction of a multi-level payment guarantee system, so thatinnovative rare disease drugs and treatment solutions can meet the unmet needsof patients.