【桑葛石原研翻译系列】风险投资在全球医疗领域中的作用分析

【桑葛石原研翻译系列】风险投资在全球医疗领域中的作用分析packagestoscan 作者：桑葛石 作者：Nina Rawal博士 全球医疗已经取得了很大的成就，但仍有很大提升空间 在过去几十年中，全球医疗领域取得了一些重大进步，霍乱等传染病的死亡率已经减半，脊髓灰质炎

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作者：桑葛石

作者：Nina Rawal博士

全球医疗已经取得了很大的成就，但仍有很大提升空间

在过去几十年中，全球医疗领域取得了一些重大进步，霍乱等传染病的死亡率已经减半，脊髓灰质炎在全世界几乎完全根除。自2000年以来，5岁以下儿童的疟疾死亡率下降了69%。这些不仅挽救了无数生命，减少了人类痛苦，而且大大加强了印度和几个非洲国家的经济。这是世界医疗组织（WHO）、全球防治疟疾、结核病和艾滋病基金、比尔和梅林达盖茨基金会等参与者的大规模努力的结果。他们都为长期健康改善目标投入了大量资源。

医疗领域仍存在着巨大的挑战，传染病仍然导致全球四分之一的人死亡，每年约有500万儿童在5岁之前死亡。除了结核病和麻疹等传染病，以及日益严重的抗菌素耐药性问题，中低收入国家越来越努力改善与生活方式相关的疾病，如糖尿病、心血管疾病和痴呆，即所谓的双重疾病负担。中低收入国家已经占全球所与生活方式相关死亡的70%，预计这一数字将持续增加。因此，在考虑未来的全球健康问题时，寻找对糖尿病和痴呆症具有成本效益的解决方案将是至关重要的。低收入国家和高收入国家面临不同疾病的观念差距越来越大，不过对中低收入国家具有成本效益的创新也可能对高收入国家产生大规模影响。Aravind眼科护理系统是一个非常好的例子，以最低的成本提供世界上最好的医疗结果；Leveraged Freedom轮椅，是一种比西方竞争对手更好的轮椅，价格却便宜一半，也是一个很好的例子。

“他们变得富有是因为他们很健康”——这是健康对一个国家提升经济繁荣阶梯的重要性

除了减少人类痛苦和提高个人生活质量外，健康还与GDP发展相关。事实上，健康的人造就了更健康的工人，他们可以为社会和经济增长做出更大贡献。日本等国家的经验表明，实行全民医疗保险导致了更强劲的财政增长，他们变得富有是因为他们健康。潜在的经济增长、将税收收入用于医疗保健的意愿以及对其引入的全系统视角也是重要因素。目前，195个国家中只有58个国家提供全民医疗保险，全球约有60亿人可以部分或全部自费支付医疗费用。

对于每天生活费不到2.50美元的27亿人口来说，这一等式几乎不可能平衡。当疾病袭击没有医疗保险的人时，贫困是一个最常见的结果。

展望未来，中低收入经济体的增长速度快于高收入国家，很快将占全球经济总量的50%。政府投资于健康不仅在财务上有意义，而且还增加了公司为这一细分市场开发产品的吸引力。

公平是医疗挑战的根源

公平获得医疗服务和产品可能是围绕全球医疗中最复杂的问题。一些人会认为，必须首先解决获得基本医疗的问题。这一挑战需要一套不同于这里概述的工具。然而，高质量和具有成本效益的产品，这份白皮书的重点，提供了无与伦比的可伸缩性，也可以允许关注最需要的医疗服务。

根据当前的国际知识产权(IP)立法，专利保护药物和其他治疗方案由拥有知识产权的公司控制，产品的分销由他们自行决定。低收入或无法负担药品成本的国家不能获得药品，除非公司决定降低价格，或其他利益相关方，如世卫组织或非政府组织介入，为药品购买提供资金。这是导致关注全球疾病的制药公司数量有限的主要原因之一。

印度和巴西等国对知识产权制度提出了质疑，认为它不成比例地针对了世界上最贫穷的人口。这些国家已经发展了大型仿制药产业，在这些产业中，药品的生产成本只是品牌药品的一小部分。印度仿制药公司现在是全球参与者，在药物逆向工程和低成本生产方面拥有强大的知识。

目前的系统在开始时通过为新疗法的开发提供激励而初衷良好，但在抗生素耐药性等领域的相关性也在逐渐降低。展望未来，我们有理由相信新的方法将会被发现。人们能够负担得起的低成本解决方案也会降低违规复制的动机。

医疗研发的长期资金不足，导致可用的治疗方案减少，医疗需求大量未得到满足

制药仍然是最可扩展的治疗方式，但这是一项困难的业务。一种药物从临床前阶段过渡到已批准产品的可能性仅为5%左右，总平均成本约为15亿美元。为了补偿风险和成本，制药公司享有其市场销售产品的溢价利润率。由于上述原因，中低收入国家支付高价药品的能力有限，导致制药公司将其研发工作集中在主要影响高收入国家的疾病上。从而导致医疗相对缺乏创新，高收入国家也无法获得药品。只有不到5%的私人研发支出属于全球医疗领域。宏观经济、政治风险和缺乏系统数据等问题也导致全球医疗被置于主流商业研发组织的聚光灯之外。

风险投资（VC）模式将科学转化为商业产品方面的力量

与此同时，在癌症和罕见疾病等领域正在取得巨大的进展。突破性的科学发现诸如基于基因治疗和免疫学的技术，针对以前无法治疗的疾病正在被转化为可治疗的和新疗法。罕见病与医疗领域的相似之处，都被认为是没有吸引力的创新市场。每种疾病很少被认为代表有限的市场机会。每一种疾病只有少数患者被认为代表着有限的市场机会。为了解决创新水平低的问题，引入了减少临床试验规模要求和监管审查时间的监管激励措施，结果是显著的，例如，2018年美国食品和药物管理局批准的59种新药中，有31种用于罕见疾病。此外，将患者分为更小、更同质的群体，再加上精准医学的发展，影响了其他医学领域应用相同的靶向药物开发方法——这是如何为特定人群进行创新也能对其他群体产生重大影响的一个例子。

VC和其他融资模式在这个时代发挥了重要作用，尤其是通过资助未经证实的高风险技术，并证明它们的临床效益。VC是资本和知识的结合，知道如何建立和加速科学类公司的市场。VC投资者将支持公司在临床和商业战略、商业、业务发展、融资、建立正确的网络，确定正确的董事会和顾问，所有的目的是加快开发过程和加速收入的产生。

典型的生命科学VC基金将投资周期设为5-8年。之后他们将出售给大型制药公司，或通过该公司在证券交易所上市进行交易。退出是VC过程不可分割的部分，因为它允许投资者反过来将资本返还给其投资者，或者在常青基金的情况下，投资于新公司。因此，大型制药公司和大型医疗器械公司在生态系统中的作用至关重要，它们是VC支持的初创公司的买家。

初创与大型药企合作模式显著提高了整个制药行业的创新率

在过去的几十年里，创新过程从大型制药公司和大型医疗设备公司广泛外包到小型的、通常是VC支持的初创公司。这一根本性转变在制药行业最为明显——自2013年以来，美国食品和药物管理局批准的新药中，有2/3最初由较小的生物制药公司开发，导致大型制药公司成为外部来源创新的大买家，随着时间的推移，各方在复杂的模型中分担开发和其他风险。VC初创公司-大型制药公司合作模式显著提高了整个制药行业的创新率。因此，创新产品的收购或许可对于大型制药公司保持创新并获得优质定价的追求同样重要。

除了通过对每家公司的广泛尽职调查和主动所有权模型掌握风险因素外，VC模型主要围绕能够获得溢价定价的产品构建。在罕见疾病的案例中，VC投资者发现了一个有吸引力的利基，鉴于患者相对缺乏，临床试验规模要求往往较小，同时有机会应用健康经济论证来获得溢价定价。

它还侧重于产品而不是服务，这主要是因为产品更容易跨地域、国家和文化进行扩张。

当然，VC模型并不完美，高风险高回报模式在过去几十年中产生了好坏参半的结果。此外，它有时与贪婪和非包容性文化有关。本文论述了VC方法论和工具箱的优点，它们绝不依赖于上述类型的组织文化。

在全球医疗领域进行VC的机会

鉴于VC在罕见疾病和肿瘤等具有挑战性领域的成功，但它回避了一个问题：VC模式可以做些什么来加速创新和解决全球卫生领域的市场失灵？

• 通过开发新产品和解决方案，挽救生命，减少人类的痛苦。
• 跨国家和区域扩大创新产品。在单独资助创新的能力下，中低收入国家可以获得大规模的健康收益
• 影响投资的案例已经提出，全球医疗是财政和社会回报可以一致的很好的例子。这个领域的创新产品可以挽救生命/减少人类的痛苦，也有助于中低收入国家的经济发展。
• VC投资者有机会在不那么拥挤的空间中，以合理的估值进行创新。为中低收入国家设计的低成本体外受精或糖尿病护理方案在高收入国家也可能具有有吸引力的市场潜力。
• 将更大比例的风险资本直接投入中低收入国家的方式。尽管许多创新最初可能来自欧美，但一些最具创新的解决方案应该来自当地国家本身，这些国家通常不在VC投资者或其他利益相关者的地图上。因此，资本流入有助于加强地方创新环境。

【原文】

Investing for good - the case for venture capital in global health

Nina Rawal, Ph.D

Global health - much has been won and much remains

The area of global health, here defined as diseases affecting low and middle income countries (LMIC), has seen some major wins over the past decades. Mortality rates for infectious diseases such as cholera have halved and polio is all but completely eradicated worldwide (1, 2). The malaria death rate in children under five has fallen by 69% since the year 2000 (3). These improvements have not only saved lives and reduced human sufferings they have also significantly contributed to strengthened economies in India and several African countries. They are the result of large scale efforts by players such as the WHO, The Global Fund to combat Malaria, TB, and HIV, and the Bill and Melinda Gates Foundation. They have all invested significant resources towards long term health improvement goals.

There are however large remaining challenges in the area of global health. Infectious diseases still cause one out of four deaths worldwide, and around five million children die every year before the age of five (4). In addition to communicable diseases such as tuberculosis and measles, and the increasing problem of antimicrobial resistance, LMIC increasingly struggle with lifestyle related diseases such as diabetes, cardiovascular disease and dementia 一 the so called double burden of disease. LMIC already account for 70% of all lifestyle related deaths worldwide and that number is expected to increase going forward (4, 5). Finding cost effective solutions for diabetes and dementia will thus be of utmost importance when thinking about global health issues ahead. The notion that low and high income countries face different disease challenges is increasingly outdated, cost effective innovations for LMIC could have large scale impact also in high income countries. Aravind Eye Care Systems which offer the best medical outcomes in the world at the lowest cost, and the Leveraged Freedom Chair, a wheelchair with superior features compared to Western competitors at half the price, are two examples of innovation with such potential (6).

'They became wealthy because they were healthy' - the importance of health for a country to move up the economic prosperity ladder

In addition to reducing human suffering and improving quality of life for the individual, good health also correlates with GDP development. Indeed, healthy people make for healthier workers, who can contribute more to society and economic growth. Experience from countries such as Japan illustrates that introduction of universal health coverage resulted in stronger financial growth 一 ‘they became wealthy because they were healthy' (7). Underlying economic growth, a willingness to spend tax income on health, and a system-wide perspective on its introduction are also important factors (8). Currently only 58 out of 195 countries offer universal health coverage, leaving around 6 bn people globally to finance their health care costs either partially or completely at their own expense (9).

For the 2.7 bn population living on less than 2.50 USD a day, that equation is virtually impossible to balance. When disease strikes people without health care insurance, poverty is a common consequence.

Looking ahead, LMIC economies are growing faster than high income countries and will soon account for 50% of the total global economy (2). Not only does it make financial sense for governments to invest in health, it also increases the attractiveness for corporations to develop product offerings tailored for this segment.

Equitable access - the mother of all global health challenges

Equitable access to health care services and products is perhaps the most complicated issue surrounding global health. Some will argue that access to basic health care must be addressed first. That challenge requires a different set of tools than outlined here. However, high quality and cost effective products, the focus of this whitepaper, offer unparalleled scalability and can also allow focusing of health care services where they are most needed.

Under current international Intellectual Property (IP) legislation, patent protected drugs and other treatment options are controlled by the company owning the IP and distribution of products is subject to their discretion. Low income persons or countries unable to afford the cost of drugs cannot access it unless the company decides to reduce the price or other stakeholders such as WHO or NGOs step in to finance the purchasing of product. This is one of the main reasons for the limited number of pharmaceutical companies focusing on global health diseases.

Countries such as India and Brazil have questioned the IP system, arguing that it disproportionately targets the world's poorest populations. These countries have developed large generics industries where the pharmaceutical is produced at a fraction of the cost of branded drugs. Indian generics companies are now global players with strong know how in reverse engineering of drugs and low cost production.

Well intended at the time of inception by providing incentive for development of new therapies, the current system is decreasingly relevant for areas such as antimicrobial resistance as well. Looking ahead, there is thus reason to believe that novel approaches will be identified. Low cost solutions that people can afford would also reduce the incentive to copy illegally.

Global health R&D is chronically underfunded, resulting in fewer available treatment options and large unmet medical needs

Pharmaceuticals remain the most scalable treatment modality but is a difficult business. The likelihood for a drug to pass from pre- clinical stage to approved product is only around 5%, and the total average cost amounts to around 1.5 bn USD (10, 11). In order to compensate for risk and cost, pharmaceutical companies enjoy premium margins on their marketed products. For the reasons outlined above, the limited ability for LMIC to pay for premium priced drugs have lead pharmaceutical companies to focus their R&D efforts on diseases mainly affecting high income countries. This results in a relative lack of innovation within the global health sector as well as lack of access to medicines available in high income countries. Less than 5% of total private R&D spending falls within the global health area (12). Issues such as macroeconomics, political risks, and lack of systematic data also contribute to global health being placed outside the spotlight of mainstream commercial R&D organizations.

The power of the venture capital model in translating science into commercial products

Meanwhile, great strides are being made in areas such as cancer and rare diseases. Breakthrough scientific discoveries such as gene therapy and immunology based technologies are being translated into cures and novel therapies for previously untreatable diseases. Rare diseases hold similarities with the global health area in that it was considered an unattractive market segment to innovate for. Few patients per disease were considered to represent a limited market opportunity. To address the low level of innovation, regulatory incentives reducing clinical trial size requirements and regulatory review time were introduced. The results have been remarkable；as an example 31 out of the 59 new drugs approved by the US Food and Drug Administration in 2018 were for rare diseases (13). Furthermore, the sub-categorization of patients into smaller, more homogenous groups, combined with the developments in precision medicine have influenced other areas of medicine to apply the same targeted drug development approach - an example of how innovating for a certain population can also have significant impact on other groups.

Venture capital (VC) and other active ownership financing models have played an essential role in this successful era by funding unproven high risk technologies and demonstrating their clinical benefits in patients. VC is the combination of capital and know how around building and accelerating science based companies to market. This is usually done through so called active ownership where the VC investor will support the company on clinical and commercial strategy, business development, financing, building the right networks, identifying the right board of directors and advisors - all with the purpose of speeding up the development process and accelerating revenue generation.

The typical life science VC fund will invest in a company with a 5-8 year investment horizon. After that, they will look to exit the company through an industrial trade sale to e.g. big pharma, or through listing of the company's shares on a stock exchange. The exit event is an integral part of the VC process as it allows the investor to in turn return capital to its investors, or in the case of evergreen funds, to invest in new companies. The role of big pharma and large medical device companies in the ecosystem is thus essential, acting as buyers of VC backed startup companies.

'The startup-big pharma partnership model has significantly increased the innovation rate in the pharmaceutical industry as a whole1

Over the past decades, there has been extensive outsourcing of the innovation process from big pharma and large medical device companies to small, often VC funded, startups. This fundamental shift, most clearly seen in the pharma industry - of the new drugs approved by the US Food and Drug Administration since 2013, 2/3 were initially developed by smaller biopharma companies (14)- has resulted in big pharma becoming large buyers of externally sourced innovation where development and other risks are shared between the parties over time in sophisticated models. The VC startup-big pharma partnership model has significantly increased the innovation rate in the pharmaceutical industry as a whole. The acquisition or in-licensing of innovative products is thus equally important to big pharma in their quest to stay innovative and command premium pricing.

In addition to mastering the risk component through extensive due diligence into each company, and the active ownership model, the VC model is mainly built around products that can command premium pricing. In the case of rare diseases, VC investors have identified an attractive niche where clinical trial size requirements tend to be smaller given the relative lack of patients, combined with the opportunity to apply health economic argumentation to command premium pricing.

It also focuses on products rather than services, mainly due to the easier scalability of products across geographies, countries, and cultures.

Of course the VC model is not perfect；the high risk high reward model has yielded mixed results over the past decades. Also, it is sometimes associated with greed and non-inclusive cultures. This paper addresses the merits of the VC methodology and toolbox, which in no way rely on the above mentioned types of organizational culture.

The opportunity for venture capital in global health

Given its success in challenging areas such as rare disease and oncology 一 areas until recently considered unsolvable, it begs the question what the VC model can do to accelerate innovation and address the market failure in global health?

• Save lives and reduce human suffering through the development of new products and solutions
• Scale innovative products across countries and populations. Large scale health gains could be achieved across LMIC with limited ability to individually fund innovation
• The case for impact investing has been made and global health is a great example of where financial and social returns can coincide. Innovative products in this space could save lives/reduce human suffering as well contribute to economic development in LMIC
• Opportunity for VC investors to access innovation in a less crowded space, at reasonable valuations. Low cost options for in vitro fertilization or diabetes care designed for LMIC could also have attractive market potential in high income countries
• A way to direct larger share of VC capital to LMIC. Although many innovations might initially come from USA/Europe, some of the most innovative solutions should be expected from the local countries themselves, which are typically not on the map of VC investors or other stakeholders in the innovation ecosystem. Inflow of capital could thus contribute to the strengthening of local innovation environments.

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