With the continuous adjustment and improvement of the national medical insurance directory, the research and development of drugs for rare diseases are gradually gaining attention from all sectors of society. Professor Liu Guoen, Dean of the Institute for Global Health and Development at Peking University, stated in an interview that the medical conditions faced by patients with rare diseases are more severe compared to common diseases. Due to the substantial investment required for the research and development of drugs for rare diseases, and the lower market return rate, the current production of research and development remains insufficient.

Professor Liu pointed out that the research and production of drugs for rare diseases require a significant amount of time and capital. The development of a new drug often takes over 10 years and requires an investment of at least 1 billion. Due to the small population of patients with rare diseases and the limited market capacity, social investment institutions often maintain a cautious attitude towards the return rate of drugs for rare diseases.

In response to this issue, the national medical insurance directory has increased the payment intensity for innovative drugs during the formulation process. Professor Liu stated that the payment standards for drugs for rare diseases in the medical insurance directory are higher than those for general drugs. However, the specific payment standards depend on the scale of the medical insurance fund each year and the health benefits brought by the drugs themselves to patients with rare diseases. Health economics research has become the core content determining drug payment standards, ensuring that medical insurance funds are used more accurately.

Although there is still a gap in payment standards for innovative drugs and drugs for rare diseases compared to developed countries, it is expected that this gap will gradually narrow with the improvement of medical insurance policies and payment capabilities. Professor Liu believes that the relatively large population of patients with rare diseases in China provides a market advantage for drug development enterprises. Additionally, continuous guidance and scientific and systematic management of medical insurance policies will create favorable conditions for the development of such drugs.

Professor Liu emphasized that scientific evaluation and economic analysis of drugs are crucial for better supporting the research and use of drugs for rare diseases. These methods can help decision-makers more accurately understand the benefits to patients and cost increases compared to old drugs, thereby more reasonably allocating medical insurance funds. Meanwhile, with the continuous improvement of payment methods, the efficiency of medical insurance fund utilization will also be improved, further promoting the research and development of drugs for rare diseases.

(Interpreted by Waverly Shi)