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Pharmaceuticals save lives and improve the quality of life of people. The pharmaceutical industry is also a key sector for the European economy in terms of employment and production. This study investigates the functioning of the EU market of pharmaceuticals as part of the market monitoring of the pharmaceutical market in the European Union and as a follow-up to the Single Market Review. The study builds upon the outcomes of the Pharmaceutical Sector Inquiry carried out by the Competition Directorate General of the Commission (2009).

This study also complements the evaluation of the European Medicines Agency (EMEA) (forthcoming) with its focus on the process of marketing authorisation in the EU. This Volume II of the study presents the results of a research into the competitive position of the EU pharmaceutical sector. It analyses the economic outlook for the sector, focusing on demand, followed by the implications for the business model of the sector (supply) and distinguishes between the main trends that have been observed in recent years and expectations for the medium term.

In a separate volume I the study investigates the implications for economic welfare – for the efficient production and effective use of pharmaceuticals in our societies- of the regulation of pharmaceuticals in the EU. During the study we have spoken to various people from different organisations. We would like to express our gratitude to all people who have shared their valuable insights with us. The study is carried out by an independent team of consultants from ECORYS, IDEA Consult and in cooperation with several other experts.

Therefore, the views in this report represent the views of the consultants, which do not necessarily coincide with those of the European Commission. Rotterdam, December 2009 Arthur ten Have (Team Leader) Prof. Dr. Marcel Canoy Emmy Nelissen Lars Meindert Nora Plaisier Douwe Wielenga Vincent Duchene (IDEA Consult) Eveline Durinck (IDEA Consult) Paul Baker Prof. Dr. F. F. H. Rutten (Erasmus University Rotterdam, EUR) Prof. Dr. A. J. McGuire (London School of Economics) Executive Summary Background Pharmaceuticals save lives and improve the quality of life of people.

The pharmaceutical industry is also a key sector for the European economy. The sector is a major employer and it combines a large production value with high levels of innovation. It follows that the functioning the pharmaceutical market is crucial for Europe. The examination of market functioning was the focus of the Pharmaceutical Sector Inquiry carried out by the Competition Directorate General of the Commission published in July 2009. The Inquiry concluded that improvements were possible which would add up to €3 billion in gains for European consumers.

The current study investigates the competitiveness and functioning of the EU market of pharmaceuticals as part of the market monitoring of the pharmaceutical market in the European Union and as a follow-up to the Single Market Review. The study is divided into two volumes. This Volume II of the study focuses on the competitiveness of the sector. It describes the main characteristics of the sector, the trends and developments in the EU pharmaceutical markets, the competitive position of the industry and the main prospects for its future development, with a special focus on R&D and innovation.

Next to the competitiveness of the industry and the functioning of the market, the regulatory system has important implications on the sector and hence on economic welfare. As this issue has received somewhat less attention in other studies, in Volume I of the study ECORYS puts particular emphasis is on the implications of the regulation of pharmaceuticals in the EU for economic welfare. In this way the study builds upon the outcomes of the Pharmaceutical Sector Inquiry. This study also complements the (forthcoming) evaluation of the European Medicines Agency (EMEA) with its focus on the process of marketing authorisation in the EU.

Main characteristics of the pharmaceutical sector The pharmaceutical market is a complex market, given the great variety of stakeholders, the significant involvement of the government and a high degree of regulation aimed at achieving different objectives. These objectives range from supporting innovation to ensuring a high degree of public health and keeping public expenditure under control. 11 The demand side of the pharmaceutical sector is characterised by a complex interrelationship between patients, doctors, hospitals, insurance providers and reimbursement systems.

For prescription medicines, the ultimate consumer (i. e. the patient) systematically differs from the decision maker (generally the prescribing doctor) and very often also from the bearer of the costs (generally the insurance companies or the health system). On the supply side, a distinction can be made between two types of producers: originator companies and generic companies: • Originator companies undertake research into new pharmaceuticals, develop them from the laboratory to marketing authorisation and sell them on the market.

These companies range from very large multinationals to SMEs concentrating on certain niche products. • Generic companies use a business model aimed at the development of a medicine which is identical or equivalent to originator products. Generic companies market their products as soon as the originator product encounters loss of exclusivity, and their products are sold at a much lower price than the original product. Generic companies active on the European market tend to be significantly smaller than originator companies.

The difference in business models between originator and generic companies is also reflected in their cost structure: whereas for generic companies manufacturing costs account for the largest share of the costs, for originator companies research and development, marketing and sales together account for a much larger share of total costs than the manufacturing process. There is a clear focus on research and innovation in the sector. R&D is very important in the lifecycle of drugs as it takes some 12 to 14 years on average to develop a new medicine.

On a global scale, the pharmaceutical sector is the highest R&D spending sector. Given the importance of R&D in the sector and the high costs and risks associated with it, access to capital is important for the sector. Especially SMEs are dependent on external sources of finance, notably when they first enter the market. Venture capital and grants are among the most important sources of funding in the discovery and development of new drugs. The sector is heavily regulated. Important examples of policies and regulation are patent protection, and pricing and reimbursement policies.

A more detailed description of the various policies and regulation that affect the sector as well as their impact is presented in Volume I of this report. The pharmaceutical sector in the EU Value added The pharmaceutical sector occupies an important position in the EU economy with valueadded production of €70. 5 billion (2006), accounting for some 4% of total manufacturing value added. This share is much higher in some Member States, such as Belgium, 12 Denmark, Sweden and Slovenia, reaching between 8. 5 and 10% of manufacturing value added.

The value added of the sector has grown significantly, with a nominal compound annual growth rate of 7% in the period from 2002-2006. The pharmaceutical sector also accounts for over 600,000 jobs (2006), some 2% of total manufacturing employment in the EU. R&D The sector is even more important in terms of R&D and innovation and thus, in terms of its potential contribution to meet the ambitions of the Lisbon Agenda. The sector accounts for some 18% of EU R&D and 33% of total high-tech R&D expenditure1, and is also among the fastest growing sectors in terms of real business R&D expenditure.

The sector records the second highest R&D intensity (as measured by the share of R&D in value added). Data for the period 1995-2008 show that R&D intensity has increased considerably during this period. In the period 1995-2003, the sector recorded the highest growth in R&D intensity of all high-tech sectors in the EU, and available data for 20042008 suggest a further incr ease in the R&D intensity. Trade Whilst the US is still the most important market in terms of the total value of pharmaceutical sales, the value of EU pharmaceutical production surpasses that of the US.

This is reflected in the EU’s strong position in international trade of pharmaceuticals, with a significant and growing trade surplus of some €32 billion. The EU’s external exports accounting for over 70% of total world trade in pharmaceuticals. Its exports are not concentrated towards a limited number of markets: while the US is clearly the most important export destination, the EU exports significant amounts to other countries as well, and as a result the EU accounts for a major share of imports in many countries, including many of the emerging countries that experience high growth in pharmaceutical sales.

Within the EU, notably Germany, France, Ireland, the UK, and Belgium show a sizeable trade surplus in the sector. Market structure In terms of market structure, the sector is moderately concentrated. It is characterised by a relatively small group of big pharmaceutical companies that represent a significant share of the annual European turnover: in 2006, companies with more than 250 employees represented some 10% of the total number of enterprises, but accounted for more than 80% on the EU pharmaceutical turnover.

Strikingly, the numb er and share of small enterprises (less than 20 employees) has increased over the last decades, but their share in total EU turnover has decreased. Performance Although the overall performance of the EU pharmaceutical sector appears to be good notably in terms of value added growth, export performance, and R&D intensity, there are some indicators showing potential weaknesses. For example, the US has a better performance with respect to pharmaceutical innovation.

The business R&D intensity in the US is superior because high-tech industries represent a larger share of the economy and high-tech industries are more research-intensive than in the EU. Moreover, the US 1 OECD, Anberd, April 2008 13 pharmaceutical industry seems to have a higher productivity rate. In terms of profitability, EU companies are broadly comparable with US companies. Demand for pharmaceutical products Expenditures on pharmaceuticals are part of total healthcare expenditure accounting on average for about 16% of total healthcare expenditure in the EU15, although differences are substantial.

For EU12 the numbers are higher. Pharmaceutical expenditure as a share of GDP in the EU amounted to 1. 7% in 2008. When examined over a longer period of time (1980-2005), pharmaceutical expenditure has increased more than total health expenditure. In more recent years most OECD countries have experienced a consolidation or even a decrease in pharmaceutical expenditure as a share of total healthcare expenditure. It should be noted that these average figures hide the variation between individual Member States, both in the trends and (relative) size of pharmaceutical expenditure.

Within the EU, France and Germany have the largest pharmaceutical markets in absolute terms, followed at some distance by Italy, the UK and Spain. The US market is still larger than the EU market, but this difference has decreased over the last years. Prescription drugs account for some 80% of total pharmaceutical expenditures. Looking at the different types of medicines by therapeutic class (ATC), ATC-N (Nervous system) and ATC-C (Cardiovascular system) are by far the largest categories in terms of sales, both at world level and in the EU.

The use of generic medicines has increased worldwide, at least partially as a result of promotion through government policies. Although the EU Member States are not among the top countries with a high penetration rate of generic medicines, they have been catching up in terms of growth rates. However, the share is still small compared to original products: all forms of generic products represented about 17% of the total value of sales of pharmaceutical products. Figures for 2007 demonstrate big differences between Member States, ranging from just over 10% in Ireland to over 40% in Poland.

Expected growth of the pharmaceutical market The pharmaceutical sector is less sensitive to the business cycle than most other sectors of manufacturing. Alternative projections for growth in world pharmaceutical markets show continued positive growth, but at a declining trend. Growth of the world pharmaceuticals market has almost halved from 10% in 2003 to 5. 1% in 2008. The average annual growth rate for the world market over the next five years is expected by IMS Health to be 4. 4%2 and by EvaluatePharma by 2. 9% per year3.

The more detailed IMS forecast shows an annual growth rate in the EU pharmaceutical market of 3. 9% for the 20008-2013 period, representing an increase in overall sales by US$ 40 billion (€27 billion). Whilst this is below the world average, it is markedly higher than in Japan (2. 4%) and North America (0. 2%).

Moreover, because of the larger size of the EU market, its growth in absolute 2 3 14 For the period 2008-2013, IMS Health, 2009, IMS Market Prognosis 2009-2013. For prescription markets only over the period 2009-2014, EvaluatePharma, 2009, World preview 2014. terms is comparable with that of the Chinese market (US$ 42 billion), despite China’s considerably higher average annual growth rate of 21. 8%.

Growth on the pharmaceutical world market is thus especially driven by high growth rates in the EU’s New Member States and emerging markets, where the increased demand for pharmaceuticals can be explained by increased access to health care. More generally it should be noted that, while growth rates may decline, a predicted world market growth rate of 3-4% is much better than in many of the other manufacturing sectors, as the pharmaceutical sector is less sensitive to the business cycle.

In terms of type of drugs, the pharmaceutical industry is increasingly shifting towards biotechnology. According to EvaluatePharma the Top 100 drugs in 2014 will for the first time be evenly split between biotechnology and small molecule technology. 4 IMS sees cancer therapies spearheading the growth in the pharmaceutical market (ATC L). Cardiovascular drugs (ATC C) seem to be an important target for cost containment measures, and the market for this class is therefore expected to show the lowest growth in the EU. Business strategies.

There are various trends and factors that will impact the business strategies. Here we distinguish between four broad elements of the business strategy: R&D, mergers and acquisitions, sale strategies and the supply chain. R&D An important issue for the sector is that it is confronted with rising R&D costs while at the same time the success rate of innovation seems to have declined.

The rising R&D costs seems to be mainly the result of two factors: 1) many of the “easy” inventions have already been made making current clinical development more complex; and 2) regulation (e. g.requirements on clinical development) has become stricter, and differs by country, which makes testing more expensive.

5 Regarding the decreasing success rate of innovation, PWC (2009) reports that the pharmaceutical industry is currently investing twice as much as it was a decade ago to produce 40% of the new medicines it then produced. In other research it is observed that, although the total number of projects in the R&D pipeline has continued to increase in recent years, there is only limited growth in the number of candidate products for Phase III and the success rates of moving from Phase II to III are in decline.

6 At the same time, partly as a result of the comp etition of generics, it has become important to regain investments in R&D quicker, and companies seem to apply more a cost-benefit approach. As a result, we observe that the rise in R&D costs makes that the R&D processes are increasingly geared towards “failing early”, which means that a much stricter and shorter monitoring process is elaborated and applied at each stage of the R&D trajectory, aiming to detect as early as possible potential failures. The fact that R&D 4 5 6 Ibid.

W ilsdon T, Attridge J, Chambers G, 2008, Current state of innovation in the pharmaceutical industry, CRA International. Ibid. 15 processes are geared towards failing early in turn leads also to the broader product portfolio. Another consequence of the amplified R&D costs is that companies also prefer to develop a broader portfolio of products (with less sales per product) over focusing only on block busters, as this reduces the risk per product. Related to the above, pharmaceutical companies are focussing their R&D efforts towards “small indications” (i. e. focus on specific niches within particular diseases).

First of all, because the R&D costs in these areas are lower. Secondly because it is easier to prove the value added of new drugs in these areas – due to e. g. the fact that there are no other drugs available yet in this area or that clinical trials can be conducted on a smaller scale, thus leading to faster market access of the new drug and a longer period to regain the R&D investments. These trends combined suggest that there will be a larger number of more complex and specialised drugs, which may pose a challenge to the authorisation authorities, especially at national level, in terms of their capacity.

In terms of the business model, there is increased attention on co-operation with third parties on R&D, either to gain access to specialised knowledge, and/or to reduce costs. Companies perform only those processes and products in which they excel themselves, while outsourcing the remaining processes and products to other parties that can handle these better and/or cheaper. The outsourcing occurs mostly vertically, towards smaller companies; horizontal cooperation is much less prevalent. The outsourcing trend is also reflected in the increased internationalisation of R&D.

T he US and EU are no longer seen as the only places with a suitable R&D environment, and the required infrastructure in many of the emerging countries (notably India and China) is comparable to that of the EU and US, while costs are generally lower. Therefore R&D is likely to increasingly shift out of the EU, although some of the new Member States also seem to benefit from the relocation of pharmaceutical R&D. Focus areas for R&D seem to be oncology, vaccines and biologics, as in these areas there is still a relatively high unmet demand, and the possibility to create genuine improvements appears to be higher.

Developments in the vaccine market are specifically increasing as a result of an increasing focus on the prevention of diseases and due to the absence of generic competition within the vaccines segment (also relevant for biologics). Within the vaccine market, most R&D for vaccines is in the field of oncology (cervical cancer), pneumococcal and influenza vaccines.

7 New medicines are often very expensive, which sometimes causes market access problems as they are not put on the reimbursement lists. IMS reports that companies come up with creative approaches to overcome this problem, e.g. by concluding “performance-based” agreements. 7 16 EvaluatePharma, 2009, World preview 2014.

Mergers and acquisitions (M&A) and licensing With deteriorating growth rates, pharmaceutical companies are increasingly focusing on mergers and acquisitions (M&A), both horizontal (e. g. the 2009 mergers of MerckSchering, Pfizer-Wyeth, and the 2008 merger of Roche-Genentech) and vertical (e. g. with pharmacy benefit companies) as a cost cutting measure, and also to diversify their portfolio in health-related business in another attempt to maintain shareholder value.

8 Literature shows that leading pharmaceutical companies are increasingly making (biotech) acquisitions in order to refill their product pipelines. For a lot of smaller companies, acquisition is the (only) way to bring their product to the market, because they lack funds and expertise. Selling the company (or product) is also a way to convert the previous investments and efforts into cash. 9 Another way for SMEs focused on R&D to market their products is through other strategic alliances like licensing deals.

The fact that the number of SMEs is increasing, while their share in the industry’s turnover is decreasing, seems to support this observation. Sale strategies The sector also experienced an increase in marketing and sales expenses over the past decades, making this the largest cost category of the industry leaders in the sector. Some see this as a sign of a lack of new innovative medicines, and many have criticized the industry for it, which has led to increased regulation of the promotion of medicines in several countries. In the coming years, a consolidation or even a decrease in the marketing and sales process is expected.

The industry will mainly focus on policy makers and on those that pay for the products (e. g. insurance companies). There is also an increased focus on patients in the industry, as they pay for an increasing share of their health care costs. As the pool of marketing and sales targets will then become smaller, it will become more important for pharmaceutical companies to differentiate by offering additional services like home delivery. The trend towards diversification is reported widely. Next to providing additional services an important element in the diversification strategy seems to be the focus on personalised healthcare.

Personalised healthcare is a comprehensive approach as it incorporates gene-biological information to understand each patient’s unique requirements for health maintenance, disease prevention and therapy tailored to genetic or molecular profiles. In addition, it includes consideration of each individual patient’s environment, health-related behaviour, culture and values. Personalised healthcare is currently regaining momentum through the surge of research in the genomics field and related areas of biology. These new insights compel the optimisation of healthcare and therapy for individual patients.

While the potential benefits are large, substantial obstacles need to be overcome for attaining clinical utility in general medial practice. These range 8 9 Moody’s investment services, 2009, Global Pharmaceutical outlook, as reported by http://www. moodys. com/cust/content/content. ashx?

source=StaticContent/Free%20Pages/Products%20and%20Services/ Downloadable%20Files/Global%20pharma%20outlook. pdf; EvaluatePharma, 2009, World preview 20014. Malik NN, 2009, Biotech acquisitions by Big Pharma: why and what is next, Drug Discovery Today, Volume 14 (17/18), September 2009, p. 819. 17 from scientific hurdles emerging from biological and genetic complexity, to cultural, economic, legal, regulatory and ethical issues.

10 Although Marketing and Sales budgets are generally expected to decline, the share of the budgets targeted at growing pharmaceutical markets in emerging economies still seem to be increasing. To be successful in these countries, it is important for EU pharmaceutical companies not to adopt a “one-size-fits-all” approach, as these countries differ greatly in terms of for example age structure, type of diseases, protection of intellectual property, cultural factors, etc.

This calls for a tailor-made approach for operating on these markets. 11 The supply chain With the trend towards increased pharmaceutical sales in the developing world (see also before); increased outsourcing of both R&D and other parts of the production process, and an increased chance for pandemics,12 it is expected that higher drug volumes must be transported further, necessitating the use of logistics coordination on a global scale. 13 Globalisation of the supply chain will make supply chain management much more complex.

Other factors that will affect the supply chain are the growing product diversification strategies of pharmaceutical companies and increasingly complex technologies. At the same time, there is increasing pressure on manufacturing costs. Many blockbusters that allow for large scale production become off-patent, which r educes the possibilities for large scale production. There is already a lot of overcapacity in manufacturing according to PWC (2007), with utilization rates of less than 50% in some plants, although it should be noted that manufacturing is already mainly located outside the EU.

All these developments may lead to different organization of production into more flexible systems that can be adapted in the light of changes in market conditions, e. g. outsourcing to contract manufacturers. Also in the distribution, more fragmentation is expected, with a smaller role for wholesalers, and more integrated with support services to patients of pharmacies. Opportunities and threats for the EU Pharmaceutical industry We conclude with the opportunities and threats that the sector faces.

Opportunities There are a number of trends that the EU pharmaceuticals sector may benefit from. First of all, the high expected growth rates in GDP and pharmaceutical sales in the new Member States and emerging economies imply a larger market for pharmaceutical 10 11 12 13 18 Xu L H, Zheng H, Sedmark D D, Sadee W, 2008, The Re-emerging Concept of Personalized Healthcare, Personalized Medicine. 2008;5(5):457-469. PWC, 2007, Pharma 2020:

The vision, Which path will you take? ’, retrieved from: http://www. pwc.com/extweb/pwcpublications. nsf/docid/91BF330647FFA402852572F2005ECC22. ibid. LeadDiscovery, 2008, Pharmaceutical Supply chain strategies: Reassessing product distribution to cut costs and improve Supply. companies, even more so because these countries are catching up on spending across the full range of remedies. In particular, the expected growth in absolute terms in the EU market is greater than the growth in China and India combined. EU pharmaceuticals firms need to make the most of this projected growth in markets.

The EU pharmaceuticals industry must ensure that it benefits from the growth in its internal market and its relatively strong trade position in these countries . Secondly, demand for pharmaceuticals may rise because of the increase in the chronic disease burden owing to the aging population, the higher risk of pandemics due to globalization and urbanization, and the emergence of new diseases. Concerning the aging population, Europe is facing lower growth in t he age category of 65 years and older in the next five years than other regions. 14 Unhealthy lifestyles will also likely accentuate the chronic disease burden in the EU.

In a longer-term perspective, however, population growth both at EU and worldwide level is slowing down, which could result in a flattening off in the demand for pharmaceutical products. Thirdly, the EU pharmaceuticals industry has some notable strengths in R&D and innovation. The sector accounts for a much higher share of high-tech R&D in the EU than in the US. Moreover, within the EU high-tech sectors, the pharmaceutical sector has experienced by far the highest increase of R&D expenditure and R&D intensity over the past decade. R&D has grown fast at an approximate rate of 15% over the period 20002007.

EU firms have strong positions in the field of cancer remedies and other complex drugs for which the demand are expected to grow strongly in coming years. EU firms have also begun to make innovations in drug delivery and services and can rely on a very solid scientific base and a large pool of highly-skilled staff. In this context the knowledge and equipment available in many hospitals in the EU, for instance, allows for complex testing and advanced clinical trials. Even though emerging economies are rapidly catching up in this regard too, this remains an important relative strengths for pharmaceutical R&D in the EU.

Finally, the EU industry would benefit if the proposals from Volume I are to be implemented since inter alia this creates business opportunities for European pharmaceutical companies. Threats There are also a number of trends that are likely to negatively affect the EU sector in terms of sales. First of all, the current economic crisis and increased demands from a rising chronic disease burden and due to an ageing population will also increase pressures on health budgets, pushing governments to find ways to cut costs.

Pharmaceutical expenditures have become a favoured cost-containment target, as witnessed by the consolidation of pharmaceutical expenditure as a share of total health spending and GDP in recent years. Pricing policies, more incentives for using generic medicines, and increasing the own contribution of patients in pharmaceuticals are among the policies to respond to the rising costs of pharmaceuticals. 14 Source: IMS (2009) IMS Market prognosis, global 2009. 19.

Secondly, the expiration of a number of important patents in the coming years is expected to affect the value of pharmaceutical sales and negatively affect the finance available for R&D in the short and medium run. In particular, the segment of generics is expected to grow further. Generic entry has increased over the 2000-2007 time period and has been quicker over the past five years and in some cases significantly quicker in markets that were lagging behind in generic penetration of off-patent markets – e. g. France and Italy in comparison to some northern European markets (such as UK, Germany, and the Netherlands).

This is the result of a great deal of policy emphasis that has been placed in the last years on the potential benefits from generic competition and generic substitution. As noted above, all forms of generic products represented about 17% of the value of sales in the EU market in 2007 and the value of sales has grown in the same year by 7%. To put this in context, original products experienced a growth in value of sales of approximately 5%: those still with exclusivity protection grew with approximately 13%, but those that had lost exclusivity declined substantially with almost 9%.

15 The increase in the share of generics in pharmaceutical sales will increase competition in the market. The effects on R&D and innovation in the sector are uncertain: whilst generic companies have a much lower R&D intensity than originators, nevertheless increased competition from generics will provide incentives for originator companies to increase the efficiency of R&D spending and focus on more innovative business models and improving costefficiency etc. Finally, despite the increase in R&D intensity in the EU, the success rate of innovation seems to have declined.

The rising R&D costs, partially explaining the increased R&D intensity, result from the fact that many of the “easy” inventions have already been made making current clinical development more complex; and also that regulatory requirements (e. g. on clinical trials) have become stricter and differ by country, which makes testing more expensive. 16 Regarding the decreasing success rate of innovation, the pharmaceutical industry is currently investing twice as much as it was a decade ago but achieving only some 40% of the previous number of new medicines launches.

R&D outputs have lowered in recent years inter alia due to launch delays and non-approvals. These developments make it especially important to ensure that successfully launched products have timely access to the EU-wide pharmaceuticals market. 15 16 20 W ilsdon T, Attridge J, Berdellima A, 2008, Factors affect ting generic entry in Europe, CRA International. W ilsdon T, Attridge J, Chambers G, 2008, Current state of innovation in the pharmaceutical industry, CRA International. 1 Introduction 1. 1 Background of the study.

The first aim of this study is the monitoring of the pharmaceutical market in the European Union. As a follow-up to the Single Market Review, the Commission’s services began a more systematic and integrated monitoring of the functioning of key goods and services markets. As announced in the Commission Communication “A Single Market for 21st Century Europe” and the accompanying Commission S taff Working Document this product market monitoring approach is one of the main components of the new strategy for the Single Market, which aims at delivering more evidence based and impact driven policies.

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