(Characters of the Two Sessions) Ding Lieming, an innovative drug researcher: Why is "orphan drug" not "orphan"?
China News Agency, Hangzhou, March 4th: Ding Lieming, an innovative drug researcher: Why is "orphan drug" not "orphan"?
Author Zhang Yuhuan
"There are nearly 7,000 known rare diseases in the world, and only 10% of rare diseases have corresponding treatments, and the remaining 90% of rare diseases are still incurable or even undiagnosed." Representatives of the National People's Congress, Betta Pharmaceuticals In an interview with a reporter from China News Agency, Ding Lieming, chairman and CEO of the industry, mentioned the current situation of rare disease drug research and development.
He introduced that drugs used for the treatment of rare diseases internationally are called "orphan drugs".
At present, the shortage of "orphan drugs" has become a major global public health problem.
Ding Lieming (right), deputy to the National People's Congress and chairman and CEO of Betta Pharmaceuticals, was interviewed by a reporter from China News Agency.
Photo by Qian Peng
Over the years, Ding Lieming, who has been engaged in the research and development of new drugs, has built a leading tumor molecular targeted drug research and development and industrialization platform in China, filling many drug research and development gaps.
In the eyes of this former doctor scientist, the "helplessness" of rare disease patients is also the crux of the hearts of countless drug developers.
"Many people have seen the movie "I'm Not the God of Medicine", which tells the reality that some cancer patients can't afford medicine." Ding Lieming said that through independent innovation in recent years, China has made continuous progress in medical innovation, and with more and more The more sky-high "life-saving drugs" are included in medical insurance, the economic burden of the people is greatly reduced.
However, in the development of rare disease drugs, it seems that there is still a rather difficult road ahead.
"A major obstacle to the research and development of 'orphan drugs' is the low enthusiasm of enterprises." Ding Lieming explained that the research and development costs of "orphan drugs" are high, and the number of patients with a single rare disease is small, and the post-marketing income is not enough to recover the research and development costs.
In addition, due to the small number of patients, the recruitment of clinical subjects is also very difficult.
Ding Lieming emphasized that although the incidence and prevalence of rare diseases are low, due to the huge population base in China, the absolute number of rare disease patients is not large.
The research and development of "orphan drugs" can hardly be solved by simply relying on enterprise-driven and market mechanisms.
He mentioned that there are relatively few incentive policies for the research and development of "orphan drugs" in the country. At present, the policies are mainly concentrated in the review stage, and the incentive effect on basic research on rare diseases and original innovation of "orphan drugs" is not obvious.
Ding Lieming proposed that a systematic incentive mechanism should be established throughout the stages of R&D, registration, and post-marketing to encourage more "Chinese innovation" in the field of "orphan drugs".
"I propose to strengthen the coordination of the diagnosis and treatment of rare diseases at the national level, and at the same time establish a special working committee for rare diseases at the national level to coordinate the formulation of systematic policies and mechanisms around the diagnosis and treatment of rare diseases, 'orphan drug' research and development and medical insurance reimbursement, etc., and encourage scientific research institutes and pharmaceutical companies to increase the research and development of 'orphan drugs'." Ding Lieming said.
R&D expenses are also a big mountain for pharmaceutical companies.
Ding Lieming hopes to increase the scientific research funding support for "orphan drug" research and development projects in the application of national scientific research projects, such as giving companies tax relief for 50% of the "orphan drug" research and development expenses.
At the same time, pharmaceutical companies that have obtained the marketing authorization of "orphan drugs" for children can be rewarded with priority review vouchers that are not restricted in use. The priority review vouchers are anonymous and tradable, which can greatly stimulate the enthusiasm of pharmaceutical companies.
In addition, he also proposed to grant market exclusivity protection to pharmaceutical companies.
It introduced that the "Opinions on Deepening the Reform of the Review and Approval System and Encouraging the Innovation of Drugs and Medical Devices" proposed that a certain data protection period should be given to the self-obtained and undisclosed test data and other data submitted by applicants for drug registration for the treatment of rare diseases. .
During the protection period, other applicants will not be approved for marketing applications of the same variety.
"But in practice, due to the small number of subjects, the data duplication of 'orphan drug' research and development is low, and the incentive effect of data protection is limited." He suggested that a certain period of market exclusivity should be given to the approved "orphan drug". During this period, the listing of the same product with the same indication will not be approved, and the market exclusivity period will be used as an incentive to replace the data protection policy.
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