Ma Aixia: It should be given a reasonable price of new high -value new drugs, and further encourage local medicine innovation

Since the reform of the pharmaceutical review system in 2015, the Chinese pharmaceutical industry has ushered in historic development opportunities. In the past seven years, domestic innovative drugs have accelerated their listing, and imported innovation medicines have come. Native innovative pharmaceutical companies, which are constantly exported to innovative vitality, have also promoted the rapid rise of my country's innovative pharmaceutical industry, and realized the major leap from "0" to "1", from "following" to "run". The transcript has attracted attention.

In the past few years, many of the local innovation fruits, many of them will have the ability to represent my country's innovation research and development strength to participate in the international market competition. Among them, the Orebatinib (product name "Nikk") was the first and only third-generation BCR-Abl inhibitor in China, which was approved for listing. It is the best Best in class ) Potential, which can fill the global clinical gap and solve the new type of Chinese original drugs that are urgently needed by clinical clinical. With excellent clinical data performance and indispensable importance, Orebatinib is expected to open a new chapter in the global blue ocean market in China.

Ma Aixia, director of the China Pharmaceutical University Pharmaceutical Evaluation Research Center and a professor at the International Medical Business School, told the Pharmaceutical Economic News: "Orebatinib is the only three-generation BCR-ABL inhibitor in China. T315i mutation patients with drug -resistant slow -capacity. Because this variety is an original high -value innovation drug that fills domestic clinical gaps, it can be accelerated by the National Drug Administration to obtain conditions. The treatment plan has a significant benefit to the patients with T315I mutations. "

With the booming development of the innovative drug industry, the price formation mechanism of innovative drugs dominated by state talks is gradually developing and improved. Compared with the international mature pharmaceutical markets such as Europe and the United States, China is still a very different emerging market. The value assessment of innovative drugs is a new challenge to the pharmaceutical company and the medical insurance department. How to give the reasonable pricing of innovative drugs from the perspective of pharmaceutical economics not only allows patients to benefit and protect the healthy development of the industry is the key to cracking the dilemma of innovative drug payment.

Professor Ma Aixia believes that different premium management of drugs of different values, especially for the evaluation threshold of the original high -value innovation drug that fills the original high -value innovation drugs that fills clinical gaps, can further encourage enterprises, especially local enterprises to innovate. The elimination of other policies in recent years can bring to the inhibitory effect of drug innovation. "In fact, innovative and precise drugs such as O'RERATININenibini are small because the target population is small, and even if a small amount of premium is performed, it will not have a great impact on the medical insurance fund."

Ma Aixia, director

01

Innovation and precision drug value assessment encounter challenge

"Medicine Economic News: In recent years, the importance of drug economics in medical insurance access has become more and more prominent. What does a drug economy evaluation of drugs needs to be considered?"

Ma Aixia: The economic evaluation of drugs is a relatively complicated class of research. Among them, evidence, parameters, methods and models of various sources need to be used. However, the ultimate goal is to evaluate the economy compared to the control intervention scheme. Usually we use it

To show the result, so ICER can discuss the factors that need to be considered.

First of all, the economic evaluation of drugs must be controlled. From the formula of ICER, it can be seen that the evaluation of the pharmaceutical economics itself is a comparative study, and the results of different evaluations must be different. Usually, the existing treatment drugs in the medical insurance directory under the same adaptation are required as a control. The "economy" in drug economics is relative, that is, compared to the control scheme is economical. Therefore Essence

Secondly, the economy of drugs is not an independent dimension, but it needs to be based on the safety and effectiveness of the drug. Therefore, the evidence of the safety and effectiveness of the drug for the treatment of specific diseases is required in the economy evaluation. If a drug has no reliable security and effective evidence, economics will not be able to talk about it.

Third, the economy of drugs is bound to be affected by cost. The cost is the result of the measurement of the medical resources consumed by the disease intervention plan. What needs to be emphasized here is the total cost of the drug intervention plan. It not only includes the price of the drug, but also the cost of considering the use of drugs, consolidated drugs, necessary examinations, nursing, and hospitalization. Symptoms, follow -up treatment and other costs.

Finally, the economic evaluation of the drug will encounter some more complicated problems. For example, indirect comparative comparative problems between drugs; the economic evaluation of multiple indications; the problem of "free is still not economical" in a certain drug in the complex intervention plan; Inadequate innovative drug value evaluation problems, etc.

"Medicine Economic Daily: For the original high -value innovation drugs that fill the clinical gap, how do you think it should evaluate its drug economy value? What are the challenges? What are the better ways and thinking?"

Ma Aixia: For the original high -value innovation drugs that fill the clinical gap, the basic ideas and paradigms of economic evaluation are the same. It is also necessary to evaluate the comparison of the cost of the treatment plan and the comparison of healthy output. However, in the context of precision treatment, drug research and development has gradually transformed from drug research and development from some large genetic targets to drug research and development of small genetic targets, that is, the treatment of drugs is becoming more and more accurate. Therefore, more and more innovative and precise therapy drugs have been listed in China in recent years have also been increasing. In the context of the continuous speed of review and approval, some precise treatment drugs with high levels of innovation, including the original high -value innovation drugs that fill the clinical gap, can often be approved by small samples and single -arm clinical trials. In this case, to carry out a high -quality pharmaceutical economics evaluation, it will encounter clinical data with a small amount of clinical data samples, no comparison with the control drug head, and only short -term clinical endpoints. This has brought great challenges to the value assessment in the process of innovative drug medical insurance.

Although it is very challenging to the value evaluation of innovation and precision drugs, you can still try to reduce the uncertainty of the uncertainty or uncertain analysis of the economic evaluation results through some methods. For example Simulation, or can carry out information value analysis (VOI), etc. Sometimes, although the evaluation results may have great uncertainty, they can still provide a certain basis for us to make decisions, which is better than there is no evidence, so as to ensure that we have been on the road of evidence -based decision -making, rather than decision -making.

Therefore, suggestions: Based on the establishment of a good value assessment framework, the focus is on the treatment field of heavy burden and clearly unsatisfactory treatment, and the value assessment of innovative and precise drugs is evaluated. Based on the results of value evaluation, the medicine is classified and managed:

1. Compared with the existing most effective treatment, there are no additional benefits or lower benefits, and directly enter the conventional negotiation process;

2. Compared with the existing most effective treatment, there are products that have significant additional benefits, enter the medical insurance reimbursement catalog, and conduct appropriate premium management (for example, based on more loose assumptions The cost of effective treatment is given a certain percentage of cost premiums), and it is required that the enterprise will continue to collect key clinical data during the two -year contract period, and then evaluate it after 2 years, and enter the conventional negotiation process.

02

Overcoming drug resistance to meet the urgent clinical needs

Orebitinib high -value innovation

"Medicine Economic Daily: The original intention of innovative drug development is the unsatisfactory clinical needs. Orebatinib was approved to treat patients with T315I mutations and drugs who had no medicine. From the perspective of the medicine, how do you think of the disease burden that the CML brings to patients with T315I mutations? "

Ma Aixia: According to the latest data released by the National Cancer Center, in 2016, the incidence of leukemia in China was 8.6/100,000, ranking 13th in the incidence of all cancer, but the mortality rate of leukemia can reach 56/100,000, ranking in 10th place in all cancers. Among them, chronic medulical leukemia (CML) accounts for about 15%-20%of all leukemia.

With the continuous progress of medical technology, especially the development of targeted therapy, the survival period of the vicious tumor of the hematopoietic system is particularly rapid. The 5-year relative survival rate of CML is only 22%in the mid-1970s. Increased to 71%, most patients who were treated with tyrosine kinase inhibitors (TKI) were expected to be close to normal people. Therefore, the treatment of CML is no longer limited to the short -term treatment goals of a complete cell genetic response (CCYR) and deep molecular academic reactions (DMR). Long -term goal.

However, the first and second-generation TKI resistance is still a clinical difficult problem. The BCR-ABL kinase region mutation is the most common TKI drug resistance mechanism, which accounts for about half of all patients with resistance. One of the mutation types.

Existing data show that the second -generation TKI treatment accompany T315i mutant CML patients with limited efficacy, only 36%hematological reactions, and no cytokine genetic response, which may not only make patients unable to achieve the long -term goal of CML, or even achieve short -term short -term short -term Treatment goals. Therefore, CML, which is accompanied by T315i mutation, still has huge unsatisfactory clinical treatment needs.

"Medicine Economic Daily: As China's first third-generation BCR-ABL inhibitor with the potential of BEST-in-Class potential in the world and has the potential of Best-IN-Class, from the perspective of pharmaceutical economics, how do you evaluate innovative drugs Auri The value of pharmaceutical economics of Batutini? "

Ma Aixia: According to clinical experts, the second -generation TKI that has been listed in China has no effect on patients with T315I mutations, and there are not many clinical use of transplantation. Orebatini is the only three -generation TKI in China, and its existing clinical data shows that its safety is higher than similar drugs. Therefore, for Orebatinib, there is no particularly suitable drug economics evaluation of drug economics.

However, from the key II clinical trials of Orebatinib's listing of Phase II, the effect is excellent. Patients with a chronic period (CP) have a 24 -month PFS of 92%and 95%of the 24 -month OS; 24 patients with acceleration (AP) The monthly PFS reaches 62%and the 24 months OS reaches 69%. Based on this, it is recommended to carry out the pharmaceutical economics evaluation of Orebatinib based on a relatively loose research, or to give the drug a higher pricing than other alternative treatments based on other basis.

03

Medical insurance negotiation reflects multi -dimensional value

Considering the global potential of innovative drugs

"Medical Economic Daily: Innovative drugs can achieve medical insurance access through national talks, which is of great significance for accelerating the process of commercialization and improving patients. In your opinion, what are the important considerations of national medical insurance negotiations on innovative drugs?"

Ma Aixia: This problem contains more than one more complicated problem. I want to say the following points:

First, the price of innovative drugs is only a small part of the national medical insurance negotiations. There are a large number of management processes, expert review and various evidence behind it. In general, the medical insurance access negotiations carried out by the National Medical Insurance Bureau in recent years have been carried out based on scientific evidence, and the process has become more and more complete and transparent.

Second, the price reduction cannot explain the problem, especially the newly added medicine. The leaders and relevant experts of the State Medical Insurance Bureau have also pointed out that there is no fixed or preset price reduction among medical insurance access negotiations. The key is how much the price is.

Third, the basic ideas in the national medical insurance negotiations are carried out in accordance with the value of value -based pricing. Therefore, for drugs with obvious innovation value, they can get higher prices. This is a policy that meets the goals of social welfare. For the development of the pharmaceutical industry, it can also inspire the rapid development of pharmaceutical companies with innovative capabilities. In this process, there is a special problem that needs to be noted that it may need to be further improved using generic drugs (especially generic drugs purchased with volume) to evaluate innovative drug values.

"Medicine Economic Daily: The industry generally believes that providing multi -level protection and reasonable returns is the hard -the -paths of the sustainable development of innovative pharmaceutical companies. What suggestions do you have?"

Ma Aixia: I personally think that multi -level guarantee is just a concept. The most critical problem is how multi -level guarantee is made and how to design. If there is no uniform and reasonable design, the multi -level guarantee system may not be able to bring goodness to innovative pharmaceutical companies. Development environment. For the reasonable return of innovative drugs, it is not given by the government or insurance company. More generally rely on the value of the innovative drug itself to win.

Therefore, to support the sustainable development of innovative pharmaceutical companies, it is very important to require relevant departments such as national medical insurance, health and health to design important management policies to formulate "game rules" reasonably, and fully consider encouraging real innovation. The impact of the item policy on the use of medical resources and the health of patients at the same time is that it is very important to consider the future development of the Chinese pharmaceutical industry.

"Pharmaceutical Economic News: The final journey of China Innovation Pharmaceuticals must be the global market. In your opinion, if the pricing in the Chinese market is too low, what impact will it affect Chinese innovative pharmaceutical companies in the global price system?"

Ma Aixia: At present, there are not many domestic innovative drugs to go abroad. Although there is still a certain gap between the pharmaceutical industry in our country compared with some developed countries, it is not a good innovative medicine. And risk, including more clinical trial data support, greater management and investment costs, more complex and changeable markets.

In addition, domestic innovative drugs also need to face the problem of building a global price system. The low price of innovative drugs in the domestic market may reduce the company's risk bearing capacity, and it may also make it difficult for enterprises to build a global price system.

However, we still need to encourage the innovative drugs produced by domestic companies to move towards the world, allowing our pharmaceutical companies to continue to improve and grow in the competition with large multinational pharmaceutical companies, thereby promoting the faster and better development of my country's pharmaceutical industry.

On the other hand, the risks and challenges brought by globalization will also bring further increase in drug costs. As the first country, China also needs to consider this when negotiating with innovative companies, instead of blindly pursuing low low pursuit of low low pursuit of low low pursuit of low low pursuit of low low pursuit of low low pursuit of low low pursuit. price.

Edit: Yu Chenglin

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