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Summary

This workshop of the Institute of Medicine's Forum on Emerging Infections set out to learn from experience what has been done and what is needed for the public and private sectors to collaborate effectively and productively for the health of the public. The emphasis was on cooperation in those product areas where returns from the market might be perceived as too small or too complicated by other factors to compete in industrial portfolios with other demands for investment. Quintessential examples of such products are vaccines, and in some instances therapies, for the diseases of children, for malaria, and for HIV/AIDS. Each of these offers lessons for attempts to deal systematically with emerging infectious diseases. While there are differences between the public health requirements of developing countries and industrialized countries, the growth of the middle class in the former and the vulnerability of the latter to diseases once thought to reside permanently ''offshore" are doing much to narrow those differences.

The primary study case for the workshop was the Children's Vaccine Initiative (CVI), formally established in 1991 as the first comprehensive effort to yoke public- and private-sector scientific advances to a global public health priority through purposive intersectoral collaboration. The lessons learned from the CVI were integrated at the workshop with other experience from disease-focused efforts, notably malaria and HIV/AIDS. The purpose of this report was to integrate that learning and the tasks it suggests as points of reference for further action.

Lessons

The lessons learned fall into four sets of messages around: what makes intersectoral collaboration "real," the notion of the product "life cycle," the implications of divergent sectoral mandates and notions of risk, and the roles of advocacy and public education (see Table 1).



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--> Summary This workshop of the Institute of Medicine's Forum on Emerging Infections set out to learn from experience what has been done and what is needed for the public and private sectors to collaborate effectively and productively for the health of the public. The emphasis was on cooperation in those product areas where returns from the market might be perceived as too small or too complicated by other factors to compete in industrial portfolios with other demands for investment. Quintessential examples of such products are vaccines, and in some instances therapies, for the diseases of children, for malaria, and for HIV/AIDS. Each of these offers lessons for attempts to deal systematically with emerging infectious diseases. While there are differences between the public health requirements of developing countries and industrialized countries, the growth of the middle class in the former and the vulnerability of the latter to diseases once thought to reside permanently ''offshore" are doing much to narrow those differences. The primary study case for the workshop was the Children's Vaccine Initiative (CVI), formally established in 1991 as the first comprehensive effort to yoke public- and private-sector scientific advances to a global public health priority through purposive intersectoral collaboration. The lessons learned from the CVI were integrated at the workshop with other experience from disease-focused efforts, notably malaria and HIV/AIDS. The purpose of this report was to integrate that learning and the tasks it suggests as points of reference for further action. Lessons The lessons learned fall into four sets of messages around: what makes intersectoral collaboration "real," the notion of the product "life cycle," the implications of divergent sectoral mandates and notions of risk, and the roles of advocacy and public education (see Table 1).

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--> TABLE 1 Lessons Learned   Authentic Intersectoral Collaboration • Implementing collaboration between the public and private sectors that goes beyond rhetoric is difficult. • Beginning partnerships early and sustaining them with regular interactions at several institutional levels is at the core of making cross-sectoral collaboration real and useful. • A critical—and informative—test of real collaboration is the sharing of information not customarily shared, for example, product leads in the pipeline, pricing rationales, early data from trials, and ongoing priority-setting processes. The Product "Life Cycle" • Pharmaceutical research and development are most usefully addressed as a total process that expresses the push-pull dynamic between supply and demand, and offers opportunities for incentives all along the pathway from bench to market. • The market for publicly needed products is not self-evident, so that clear, consistent pictures of public-sector needs, priorities, and policies, as well as potential market sizes and characteristics are essential. Divergent Sectoral Mandates and Notions of Risk • In the private sector the bottom line is defined by timely financial return. The public sector also needs to take account of competing priorities for resource allocation in terms of public health results. • The cost of pharmaceutical R&D is sizable but its dimensions are poorly understood. • For both sectors, R&D investment decisions derive from interactions among costs, time, and predictability; how those compare across different investment options; and the relative risks they produce. • Public- and private-sector views of risk differ but since both sectors confront it, pooling at least some risks is likely to motivate taking them. Roles of Advocacy and Public Education • The high value of advocacy and public education in promoting individual disease priorities and catalyzing public awareness is increasingly well appreciated. • However, the public sector and important elements of the nonprofit sector underuse their powers for advocacy for generic public health needs such as vaccines, as well as for needs seen as most important outside national borders. These lessons, taken together, signal needs for: (1) more information, (2) more predictability; and (3) more sharing of costs and risks, if the requirements for products for emerging infectious diseases are to be satisfied. Looked at systematically across the product cycle, these sort into more specific categories where incentives might be developed to bolster the competitiveness of such public health products in industrial portfolios. Table 2 on the next two pages lays out these categories, highlighting actions expected to be especially critical for advancing the infectious disease enterprise as a whole. Because a fresh look at the market for these products is believed to be primary for awakening commercial interest, the demand side is presented as leading the

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--> cycle. The emphasis here is on interventions that can make markets more attractive by expanding knowledge, limiting demand uncertainties, and generating appealing economies of scale, with most dramatic effect likely when these are conceptualized early in the R&D cycle. Emphasis on the supply side is focused on solidifying the financial resource base, sharing information, balancing out investment risks, and reducing the time and costs of clinical research. The sense of the workshop was that this was a reasonable framework for action, with the next logical steps being decisions about who might stimulate action most quickly and effectively in each area, perhaps beginning with those requiring building consensus and possible legislative modifications. Since microbes are agile and fast, it might be said that there is little time to lose.

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--> TABLE 2 Incentives for Increasing Pharmaceutical Research and Development for Priority Infectious Diseases What Is Needed Demand Side Supply Side: Basic Research Supply Side: Clinical Phases More information Market identification; • epidemiologic/burden of disease data; • ongoing, accessible, integrated, comprehensive surveillance data on disease trends and resistance patterns Priority setting; • well articulated, consensus-based public health agendas; • clear portrayals of specific disease priorities; • product characterization Disease-specific bioinformation system; • research data from universities, research councils, biotechnology companies on product leads for possible development by industry Development of surrogate endpoints; • generic categories of endpoints for use with a range of infectious diseases; • alternatives to correlates of protection for vaccines for which clinical trials difficult or impossible More predictability Market assessment; • early forecasting of demand based on epidemiologic criteria from surveys, demographic analysis; • segmentation by size, ability to pay, disease profile; • cost-effectiveness analysis International regulatory harmonization/reinforcement of intellectual property rights Restricted distribution/product labeling; • systematic exploration of tension between need to conserve usable life of antimicrobials while conserving market appeal for R&D investment

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--> More cost- and risk-sharing Market creation; • procurement guarantees via: high-volume bulk orders, extended contracts, product "bundling" subsidies for poorest countries; • revolving funds for national and/or regional purchasing; • ODA for health infrastructure and education, drug logistics; Multi-tiered pricing; • careful articulation of rationales to re-start legislative dialogue Patent extension; • exploration of extension of patents for expired compounds with potential utility in new antimicrobials; • explicit inclusion of antibiotics under Patent Term Restoration Act; Stabilization of funding; • examination of R&D implications of ODA decreases, annual volatility; Financial support to universities and biotechnology companies for taking promising leads to proof-of-principle; Venture fund for early-stage development of product leads Orphan drug legislation; • exploration of applications of OD law in developing products for emerging infections, especially niche products, with primary market outside U.S.A.; Accelerated regulatory approval; • accelerated enrollment in trials; • aggregation of efficacy data from multiple sources; • continuing the progress in predictability and efficiency in regulatory oversight processes; Building CRO capability in developing countries to reduce costs and enhance infrastructure for clinical trials; Financial subsidy for phase II/III trials, with payback on success NOTE: ODA = Official Development Assistance; R&D = Research and Development; OD = Orphan Drug; and CRO = Contract Research Organization. We want to acknowledge the importance to this table of the list, presented at the workshop by the representative from the World Bank, of suggestions from a survey of pharmaceutical company executives on how to promote health product development for low-income countries. The tabular material presented here is a broader synthesis that draws on the whole range of workshop presentations, including discussions of public-sector needs and priorities; industry perspectives on needs; lessons learned from the Children's Vaccine Initiative and other models; and legal and regulatory issues involved in development of drugs and vaccines for emerging infections. That there is so much concordance about which areas merit attention, as well as the kind of attention they require, is impressive.