In recent years, a number of rare disease drugs have been approved for marketing and included in the national medical insurance catalog through negotiation, which will further encourage innovative investment by pharmaceutical companies while solving the problem of patient drug payment.

  On the occasion of the 15th International Rare Disease Day, Sullivan and the Pain Challenge Foundation released the "2022 China Rare Disease Industry Trend Observation Report" (hereinafter referred to as the "Report") on the 27th.

The "Report" studies and judges trends from the perspectives of rare disease drug research and development, supply guarantee, capital investment, and comprehensive service system, among which drug research and development is the focus.

  According to the "Report", the clinical development success rate of rare disease drugs is higher than that of other disease drugs, which will further encourage pharmaceutical companies to invest in innovation.

  In detail, the success rate of rare disease drugs in clinical phases I to II is 67%, 52% for all disease drugs, 44.6% for rare disease drugs in clinical phases II to III, and 28.9% for all disease drugs .

  From the perspective of the whole cycle from research and development to marketing, the success rate of rare disease drugs from clinical phase I to approval is 17%, while the success rate of research and development for all diseases is 7.9%, which is only half of rare disease drugs.

  To this end, Mao Hua, partner and managing director of Sullivan Greater China, said in an interview with China Business News that the increase in the success rate of clinical research and development of rare disease drugs, on the one hand, means that pharmaceutical companies are exploring the pathogenesis of rare diseases, More attention is paid to basic research. On the other hand, emerging technologies such as gene therapy are also providing strong support in cooperation with the research on the pathogenesis of rare diseases, and speeding up the research and development process.

  The "Report" also stated that top-level favorable policies provide direction for the strategic layout of pharmaceutical companies.

For example, overseas rare disease drugs can be exempted from clinical trials by being included in "urgent clinical need" or evaluated by CDE (the Center for Drug Evaluation of the State Food and Drug Administration) to meet the requirements of being safe, effective and non-racial, thus exempting from clinical trials; these greatly shorten the time for overseas rare diseases. The launch time of new drugs in China has increased the enthusiasm of pharmaceutical companies to introduce new drugs for rare diseases.

  The "Report" also shows that in addition to independent research and development, companies also tend to introduce overseas clinical Phase II to III projects to lay out product pipelines.

For example, Beihai Kangcheng, a biopharmaceutical company specializing in the field of rare diseases, has 13 innovative product pipelines for rare diseases with a large number of patients and rare tumor indications, of which 3 products have been launched and 4 products have entered clinical trials. Stage; Shufang Pharmaceutical also announced in January this year that it has reached a cooperation with Santhera Pharmaceuticals to obtain the rights to develop and commercialize the new rare disease drug Vamorolone in Greater China.

  Mao Hua told reporters that for drugs with clear indications, domestic pharmaceutical companies will still focus on original research and rarely make generic drugs; this is because many rare diseases still lack clear indications for the treatment of drugs, and most of them are in the In research and development, domestic pharmaceutical companies have the opportunity to stand out, and my country has also opened a green channel for innovative drugs to be listed.

For drugs that have been approved globally, they still have a long patent protection period. In the future, domestic pharmaceutical companies can also introduce foreign drugs to achieve patient accessibility.

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