药物经济学评价 企业该不该知难而上

┃ 来源：中国医疗保险

什么是药物经济学？

企业为什么重视？

有什么关键障碍？

如何克服？

What Is Pharmaceutical Economics?

Why Do Enterprises Attach Importance to It?

What Are the Key Obstacles about Its Application?

What Are the Solutions?

5月20日，在《中国医疗保险》杂志社主办的第6期“青年药政论坛”上，前美国通用医疗集团中国区市场准入总监陶立波，就药物经济学在医药行业中的应用及其关键问题做了深入的分析。

Tao Libo, former Director of Market Access of GE Healthcare China, presented an insightful analysis on the application of pharmaceutical economics in the pharmaceutical industry and key challenges therein at the “6th Youth Forum on Pharmaceutical Administration” held by the Journal of China Health Insurance on May 20.

药物经济学是经济学原理与方法在药品领域内的具体运用。

Pharmaceutical economics is the application of economic principles and methodologies in pharmaceutical area.

广义的药物经济学（Pharmaceutical Economics）主要研究药品供需方的经济行为，供需双方相互作用下的药品市场定价，以及药品领域的各种干预政策措施等。

In a broad sense, Pharmaceutical economics studies the economic behavior of demand and supply sides in pharmaceutical market, the pricing of drugs, and different kinds of policy and governance on the market.

狭义的药物经济学（Pharmacoeconomics）是一门将经济学基本原理、方法和技术运用于临床药物诊疗过程，并以药物流行病学的人群观为指导，从社会、医保、医院、患者等多个角度展开研究，以求最大限度的合理利用现有医药卫生资源的综合性应用科学。

In a narrow sense, Pharmaceutical economics, (also named as PharmacoEconomics), is an interdiscipline which applies economic principles and technologies in pharmaceutical area, and carries out studies from multiple perspectives including society, medical insurance, hospitals and patients, to help decision maker to make best use of limited resources in the healthcare system.

企业希望全面地阐述其产品的价值，形成完整的产品证据链，并基于此和政府与医院开展沟通。一个好的药品，既要安全有效，又要价格合理。安全性和有效性主要是通过临床研究和观察结果来得到体现，而价格、成本是否合理,需要通过药物经济学研究来进行评价。所以一个完整产品的证据链应该包含着经济学研究证据，以此回应社会和政府的诉求。

Enterprises hope to demonstrate the integrated value of their products and develop a comprehensive evidence chain to support communication with government and hospitals. A good drug should be safe & effective, and also reasonable in price as well. Safety and effectiveness evidence are mainly generated via clinical studies, while the reasonableness of price should be evaluated through pharmaceutical economic research. Therefore, a complete evidence chain of a drug product should encompass evidence from economic research on responding the concern of society and government.

在药品的生命周期的各个阶段，都可以选择适合的时机开展药物经济学研究。

Pharmaceutical economic research could be conducted in different stages when properly during the life cycle of the drug.

首先，在经过化合物筛选和动物实验之后，医药企业会面临一个非常重要的抉择：是否要进入临床试验阶段。一些领先的国际医药企业在这个环节上就开始进行药物经济学评估工作，开展经济学探讨：这个药品将来上市，临床上会有什么样的效果？能定什么样的价格？能获得什么样的商业回报？企业以此来帮助决策该产品是否要进入临床试验，因为开展临床试验的投资是相当巨大的。

Firstly, in stage after chemical screening and animal experiment, pharmaceutical enterprise will face an important question, i.e., whether to proceed for clinical trial or not. At this point of time, some leading MNCs will carry out pharmaceutical economic study on topics: What will be the clinical effect of the product like when it is introduced to the market? How will it be priced? And what business return will it achieve? These studies could help the enterprises to make proper decision on whether to start the clinical trial as the investment of clinical trial could be huge.

然后，在药品的临床试验阶段，尤其是在三期临床研究的后期，即上市前半年到一年的时间，是开展药物经济学研究非常好的时机。因为此时企业获得了三期临床的结果，就可以基于临床数据来开展药物经济学评价。这样，当产品上市时，就不仅有临床数据还有经济学证据，从而构成完整的证据链对新产品的市场准入和营销进行支持。

Then, in the clinical trial, particularly at the end stage of phase III clinical study, i.e., half to one year before the launching, there is a wonderful time point to conduct pharmaceutical economic research. At that time, enterprises have acquired the results of phase III trial and can perform pharmaceutical economic evaluation based on the trial data. In this way, not only clinical data but also economic evidences will be available when the product is brought to market, providing support for market access and marketing of the new product with a complete evidence chain.

最后，当产品上市之后，基于市场策略的需要，可以灵活的开展各种药物经济学研究，以帮助医药企业更好地推广其产品。

Finally, when the product has been put on the market, various pharmaceutical economic researches might be conducted flexibly, to meet the needs of marketing and sales.

最后需要指出的是，企业开展药物经济学是和企业的定价策略密切相关的，在各个环节做的经济学研究都是为了支持它的定价和市场策略。

It should be noted that pharmaceutical economic researches are closely related to pricing strategies and should be used aligning with the pricing and market strategies.

2017年版我国医保目录调整提出了动态调整、谈判目录等机制，并且开始要求企业提交药物经济学资料，这大大促进了该学科在我国医药行业中的应用，很多企业开始积极地做这件事情了。

In 2017, for the adjustment of National Reimbursement Drug List (NRDL), the government applies negotiation mechanism and requires enterprises to submit pharmaceutical economic materials, which greatly promotes the application of the discipline in the pharmaceutical industry of China. Many enterprises start to carry out pharmaceutical economic research actively nowadays.

药物经济学研究包括性价比研究和预算分析研究。性价比就是这个药品的投入产出比如何，预算分析研究是研究医保为这个药品要花多少钱，能否买得起。这次国家医保谈判准入中，对药品的预算影响预测是必需的，即要求企业估测产品进入医保后的销量、价格打算降多少，以此算出这个药品进入医保后对医保预算的影响。对于性价比研究则是企业自选的，医保没有强制和明确的规定，目前很多企业其实是缺乏性价比研究资料的。

Pharmaceutical economic research includes the cost-effectiveness analysis (CEA) and budget impact analysis (BIA) generally. CEA studies the input and output of the treatment with drugs, while BIA studies how much medical insurance will pay and whether it is affordable. In this round of NRDL adjustment, the estimation of budget impact is a necessity, i.e., enterprises should propose a price and estimate the sales volume so as to find out the budget impact. CEA is not asked mandatory this time. In fact, such data are rarely available in enterprises.

地方医保也有不少这方面的政策探索。比方说天津、广州等地医保就有委托研究机构探索基于药物经济学评价的准入决策支持方法，包括企业需提交的资料有哪些、怎么评估数据质量、如何评分等。这些地方探索经验相信将来也会影响国家医保的政策制定。

Pilot policy in this area could be found in local medical insurance. E.g., in Tianjin and Guangzhou, local government asked research institutes to develop the decision making mechanism based on pharmaceutical economic evaluation, including what materials should be submitted, how to assess the quality of data and how to fulfill rating, and so on. Such local pilot policy will surely effect the formulation of national medical insurance policies.

医保对药物经济学资料的要求和政策尝试，说明了我们国家对药物经济学的正式应用开始重视了。虽然我国的药物经济学应用水平还处在一个初步发展阶段，有缺陷、有障碍，但也要看到有希望、有进步。这是一个过程，更是未来的趋势，企业应当未雨绸缪了！

The new policy of national insurance for asking pharmaceutical economic data indicates that China has begun to attach importance to the formal application of pharmaceutical economics. Though the application of the discipline is still at its beginning and has lots of challenges, we shouldn’t neglect the progress here. It is a tendency for the future. Enterprises should get fully prepared.

由于缺乏明确的规范，对企业来讲，药物经济学研究这个事情可做可不做，就算想做也不太清楚该做什么、怎么做，才能够满足以后政策的需求。在这种情况下企业会怎么样呢？第一，企业可能会觉得花这个钱没有意义，还不如干点别的更有帮助的事情。第二，在国家没有明确规范的情况下，企业有太多活动的余地，如果花了钱，一定会想法得出有利于自己的结果。这虽然给了企业一定的空间，但也伤害了药物经济学研究的质量，从而阻碍了决策者对研究结果的采信。

Due to lack of clear requirement and guideline of pharmaceutical economic research, it is just optional for enterprises, and they have no idea what to do and how to do it properly when they want to do something of pharmaceutical economic research. For the enterprises, they might think spending money on this research is meaningless. And because of the lack of guideline, if they spend the money on it, they’d certainly try to achieve what they want. This will harm the quality of pharmaceutical economic research and hence stop the decision makers from adopting the results of the research.

药物经济学是非常科学、循证的方法，但同时也是很复杂、很“柔”性的方法，对于数据的要求非常高，计算过程非常复杂，以至于决策者很难把握其质量。药物经济学评价中会涉及到很多数据计算和医学事件的互动，不同的专家即使非常严格地照原则指南去做，非常公正地做，做出来的结果也有可能差别甚大。这并不是说专家受到了利益上的诱导，而是这个研究过程本身很复杂。

Pharmaceutical economics is a scientific and evidence-based approach but very complex and “flexible” as well, requiring tremendous data and complicated calculation. The decision-makers could hardly tell its quality. And, because the research involves many calculations as well as interactions of medical events, consequently the study results got by different experts might vary greatly though they follow the principles and guidelines in an fair manner. The difference of results does not mean that the experts are interest-oriented; it is resulted from the complex research itself.

上述的阻碍药物经济学研究开展的主要障碍，都指向一个重要因素：研究的质量和可靠性。如果质量无法把控，研究结果就难以为决策者所用，最终也会使企业失去开展研究的动力。因此，我们应该考虑建立新的药物经济学应用和发展模式以及更加公开透明的决策机制。

All these obstacles for pharmaceutical economic research described above point to an important factor, i.e., the quality and reliability of the research. If research results can scarcely be used by decision-makers due to quality concern, the enterprises will finally lose their momentum to conduct research. We should, therefore, consider creating new pattern for the application and development of pharmaceutical economics with a more open and transparent decision-making mechanism.

这其中的解决方案之一，是建立标准化的疾病评价模型。虽然药品多种多样，但最终是去治疗某个疾病，而疾病发生发展的过程架构是比较稳定的。学术界应该去探讨建立标准化的疾病发生发展的评价模型，不断研究模型参数，不断完善模型。待模型获得共识和认可后，提交给决策者进行应用。

One of the solutions is to develop standard disease evaluation models. The drugs are varied but individual disease progression pathways are relatively stable. The academia should explore to develop standard models for individual diseases, and make constant efforts to study proper parameters of the models. The standard models could be submitted to decision-makers for application when consensus on and recognition of the models has been achieved.

药物经济学评价怎么做呢？就利用这么个透明的标准化模型，将药品价格数据、相关成本数据和临床研究的结果填入这个模型，模型输出评价结果。这样的结果是容易获得认可的，因为企业的价格数据、成本数据和临床数据都比较容易被验证。中间的分析模型虽然复杂，但通过学术研究透明化、标准化了，实现过程比较清楚，大家用标准化模型工具，得出的结果自然具有可信度。这样药物经济学分析的结果就容易被各方认可并应用。

When the standard models are available, researchers could just fill the price data and the results of clinical study into the model and get evaluation outcome. Such outcome is liable to be trusted and accepted, for the price and clinical data can be verified easily. Though the analytical model is complicated, its transparency and standardization is achieved through academic research. Since the process is clear, the results of pharmaceutical economic analysis obtained with standard model are certainly reliable and more easily to be recognized by decision-maker.

所以，我们现在需要静下心来把每一个疾病的标准化模型建起来，但这显然也不是件容易的事，需要各方的共同努力。

Therefore, what we need to do right now is to build up standard models for diseases, which is evidently not an easy job and requires joint efforts of all sides.