卫生研发活动经费流向调查指标与监测体系

在提高卫生研发活动资源配置效率日益成为全球卫生科技发展热点的情况下,掌握研发活动经费流向情况是改善资源配置效率的重要前提。以往关于卫生研发经费流向情况的信息十分缺乏,系统地追踪全球卫生研发经费流向情况是一个长期的目标。本就卫生研发活动经费调查在世界范围内所进行的情况,以及实施调查涉及的概念、分类指标进行概述。同时,就我国卫生研发经费调查的现状进行分析,并对建立我国卫生研发活动监测体系给予政策建议。     

Perspectives on stimulating industrial research and development for neglected infectious diseases.

This paper summarizes recent thinking on stimulating industrial research and development (R&D) for neglected infectious diseases and argues that it is critical to enlarge the value of the market for medicines and vaccines through, for example, global purchase funds. The most important economic barriers to R&D are that the commercial markets are small and that individual purchasing power is severely limited, even though the number of patients may be very large. Since R&D costs for all diseases are high, this means that returns will not cover investments. Various mechanisms have been proposed to address this economic imbalance (accepting that other barriers will also need to be considered). Economic devices which reduce the costs of R&D--push factors--are useful, but our review suggests that high costs do not explain the shortfall in R&D. Economic devices which address the lack of viable markets have been termed pull factors and are designed to create or secure a market, thereby improving the likelihood of a return on investments. One pull mechanism is the commitment in advance to purchase a product that meets specified criteria, if invented. The purchase-precommitment approach has a number of attractive features. For example, it only rewards successful outputs rather than supporting research that may not succeed. Pull programmes effectively mimic the market and lead companies to favour lines of attack that they believe will lead to marketable products. Overall, a combination of push and pull mechanisms is likely to represent an attractive approach. This could combine, for example, increased funding for public laboratories, public-private partnerships in R&D, purchases of underutilized existing products, and a precommitment to purchase new drugs and vaccines when developed.     

Stimulating pharmaceutical research and development for neglected diseases

New vaccines and drug treatments are needed for tackling the neglected diseases (NDs) of poor countries. These diseases are associated with high levels of mortality and/or morbidity, but lack appropriate vaccines and drug treatments because of bacterial resistance, toxicity, long-treatment protocols, problems with administration or because none are available. Current initiatives directed at research and development (R&D) of NDs are being led predominantly by the governmental, inter-governmental and private not-for-profit sectors implemented by way of public-private partnerships. Push and pull mechanisms targeted at the pharmaceutical industry have also been proposed as another way to stimulate R&D of NDs; however, these should be viewed with some caution. Stimulating R&D for NDs is important as part of a wider long-term public health strategy and must be addressed simultaneously with resolving more immediate problems of access to medicines and health system sustainability in less developed countries.     

An analysis of financial flows from the Brazilian Ministry of Health for research and development in 2003-2005 according to the National Agenda for Health Research Priorities

This study mapped the application of financing in research and development in health (R&D/H) by the Brazilian Ministry of Health in 2003-2005, according to the National Agenda for Health Research Priorities, created in 2004. The analysis was based on data from a study aimed primarily at measuring these investment flows during the same period. The calculations included only direct financing with actual outlays in research, including payroll expenditures. The studies were categorized according to the 24 sub-agendas of the national priority agenda by two independent researchers, and disagreements were resolved by consensus. Research and development expenditures in health totaled 409.7 million reais, concentrated mainly in the following sub-agendas: transmissible diseases, the health industry complex, clinical research, pharmaceutical care, and non-communicable diseases (79% of the total). All 24 sub-agendas received some financing during the period. The study established a baseline for subsequent evaluations of this financing instrument's inductive capacity and the relationship between R&D/H investments and the population's health needs.     

Routine monitoring of performance: what makes health research and development different?

Increasing attention is being directed to measuring and monitoring the use of health-related R&D funding, partly to justify this expenditure and partly to ensure that R&D effort is directed to achieving the paybacks desired by funders. These paybacks include contributing to knowledge, contributing to R&D capacity, political benefits, benefits to the health service and to patients, and more general economic benefits. This paper addresses the issues that must be considered when designing a routine performance management system for health R&D. Conventional methods of routine performance management are often rendered inappropriate in this context by the intangible and unpredictable outcomes of research, which are heterogeneous across projects and programmes and which can be hard to attribute to particular R&D support. Instead, to be effective in this context, a routine system must combine quantitative and qualitative indicators, utilising information from a number of different sources. The system must achieve acceptable levels (defined by the funder) on each of the following criteria: it must measure those dimensions of payback that are valued by the funder; it must be decision-relevant; it must be consistent with truthful compliance; it must minimise perverse incentives; and it must have acceptable net costs. It is vitally important that the system itself generates a positive payback. We illustrate these issues by outlining a system that might be used to monitor the payback from government-funded R&D.     

WHO's role in the global health system: what can be learned from global R&D debates?

Recent global debates on the research and development (R&D) of health technologies, such as drugs, diagnostics and vaccines, can be seen as a microcosm of discussions on the role of the World Health Organization (WHO) in the global health system more broadly. The global R&D system has come under heightened scrutiny with the publication of a 2012 report by the WHO Consultative Expert Working Group on Research and Development (CEWG), which made a number of recommendations to more equitably meet global health needs. The CEWG report followed a decade-long process of debate at the WHO on the weaknesses of the global R&D system, which include problems of affordability, limited research where market returns are small or uncertain (such as the ‘neglected diseases’ that predominantly affect the world's poorest), inefficient overlap of research efforts, and overuse of medicines such as antibiotics. The CEWG report called on WHO Member States to develop a global framework to improve monitoring, coordination and financing of R&D efforts through the establishment of a Global Health R&D Observatory and the negotiation of a binding treaty on R&D. While the treaty option has been put on the back-burner for several years, Member States nevertheless agreed at the 2013 World Health Assembly (WHA) on concrete steps towards a global framework. Progress at the 2013 WHA reaffirmed the central role of WHO as a convener, and the WHA's decision to create the Observatory within the WHO Secretariat underscored the organization's role as a source of strategic knowledge in the global health system. However, despite WHO's constitutional mandate as the ‘directing and coordinating authority on international health work’, in reality it faces major challenges in coordinating autonomous R&D actors such as states, firms and foundations in the global system. Strengthening its ability to do so requires, at a minimum, reforming its financing arrangements to provide it with a greater degree of independence from its largest donors. In addition, WHO may seem to be the natural arena for negotiating a binding R&D treaty, but negotiating new global agreements in other arenas such as the WTO, WIPO, or plurilateral fora offer the possibility of more enforceable and stronger public health norms. Nevertheless, no single arena in the existing system of global governance is perfectly suitable for the negotiation of progressive, inclusive, binding, enforceable, global health rules. While tradeoffs are inherent in the choice of any particular arena, leadership from either the multilateral institutions or influential governments can make a key difference in how beneficial any R&D treaty may be for health. In the coming years, global R&D debates will remain a critical issue to watch. The evolution of the global R&D system will be a harbinger not only of WHO's place in a rapidly-changing global health system, but also of our collective capacity to strengthen institutions of global governance for health.     

The antagonism of push and pull strategies, and the current funding campaigns to fight orphan diseases

We argue that an increase in investments in R&D for innovative treatments to eradicate neglected diseases in developing countries leads to a rational decrease in investments in available treatment technologies. In a formal model where the government of a developing country seeks to optimally allocate public resources, we show that the higher the odds of appearance of an innovative treatment, as occurring when investments in R&D increase, the lower the optimal provision of current treatments and other health expenditures. We also show that this phenomenon is aggravated when the opportunity cost of investments in current treatments increases. This implies that welfare in developing countries deteriorates as innovative treatments are more likely to become available. We also describe an insurance scheme that remedies these issues, and that leads to Pareto-optimal allocations regardless of the investment level in R&D for innovative treatments.     

Estimated research and development costs of rotavirus vaccines

Diseases like rotavirus afflict both upper- and lower-income countries, but most serious illnesses and deaths occur among the latter. It is a vital public health issue that vaccines for these types of global diseases can recover research and development (R&D) costs from high-priced markets quickly so that manufacturers can offer affordable prices to lower-income nations. Cost recovery depends on how high R&D costs are, and this study attempts to replace high, unverified estimates with lower, more verifiable estimates for two new vaccines, RotaTeq (Merck) and Rotarix (GlaxoSmithKline or GSK), based on detailed searches of public information and follow-up interviews with senior informants. We also offer a new perspective on “cost of capital” as a claim for recovery from public bodies. Our estimates suggest that companies can recover all fixed costs quickly from affluent markets and thus can offer these vaccines to lower-income countries at prices they can afford. Better vaccines are a shared project between companies and public health agencies; greater transparency and consistency in reporting of R&D costs is needed so that fair prices can be established.     

Evidence-based policy-making in the NHS: exploring the interface between research and the commissioning process.

BACKGROUND: The UK National Health Service (NHS) R&D strategy acknowledges the importance of developing an NHS where practice and policy is more evidence-based. This paper is based on a qualitative study which aimed to identify factors which facilitate or impede evidence-based policy-making at a local level in the NHS. METHODS: The study involved a literature review and case studies of social research projects which were initiated by NHS health authority managers or general practitioner (GP) fundholders in one region of the NHS. Data were collected through in-depth interviews with lead policy-makers, GPs and researchers working on each of the case studies and analysis of project documentation. RESULTS: An over-arching theme from the analysis was that of the complexity of R&D in purchasing. The two worlds of research and health services management often sit uncomfortably together. For this reason it was not possible to describe a 'blueprint' for successful R&D, although several important issues emerged. These include sharing an appropriate model for research utilization, the importance of relationships in shaping R&D, the importance of influence and commitment in facilitating evidence-based change, and the resourcing of R&D in purchasing. CONCLUSIONS: These issues have important implications for the strategic development of R&D as well as for individual project application. Moving beyond the rhetoric of evidence-based policy-making is more likely if both policy-makers and researchers openly acknowledge this complexity and give due concern to the issues outlined.     

Aid alignment for global health research: the role of HIROs

The lack of a mechanism that aligns financial flows for global health research towards public health priorities limits the impact of health research on health and health equity. Collaborative groups of health research funders appear to be particularly well situated to ameliorate this situation and to initiate discussion on aid alignment for global health research. One such group is the Heads of International Research Organizations (HIROs), which brings together a large number of major government and philanthropic funders of biomedical research. Surprisingly, there is hardly any information publicly available on HIROs' objectives, or on how it aims to achieve more harmonization in the field of research for health. Greater transparency on HIROs' objectives and on its current efforts towards addressing the gap between global health research needs and investments would be desirable, given the enormous potential benefits of more coordination by this group.     

Infectious disease research investments: Systematic analysis of immunology and vaccine research funding in the UK

Financing for global health is a critical element of research and development. Innovations in new vaccines are critically dependent on research funding given the large sums required, however estimates of global research investments are lacking. We evaluate infectious disease research investments, focusing on immunology and vaccine research by UK research funding organisations. In 1997–2010, £2.6 billion were spent by public and philanthropic organisations, with £590 million allocated to immunology and vaccine research. Preclinical studies received the largest funding amount £505 million accounting for 85.6% of total investment. In terms of specific infection, “the big three” infections dominated funding: HIV received £127 million (21.5% of total), malaria received £59 million (10.0% of total) and tuberculosis received £36 million (6.0% of total). We excluded industry funding from our analysis, as open-access data were unavailable. A global investment surveillance system is needed to map and monitor funding and guide allocation of scarce resources.     

Building a research conscious workforce

With the aim of building a research conscious workforce, the former Northern and Yorkshire Research and Development office explored the nature and purpose of research and development (R&D) support for NHS staff. Part of the study used a questionnaire to seek healthcare professionals' R&D education and training needs. Some findings were unexpected, such as the significant differences between professional groups' needs and preferences. Recommendations, therefore, include local, flexible R&D education and training programmes.     

Who needs what from a national health research system: lessons from reforms to the English Department of Health's R&D system

Health research systems consist of diverse groups who have some role in health research, but the boundaries around such a system are not clear-cut. To explore what various stakeholders need we reviewed the literature including that on the history of English health R&D reforms, and we also applied some relevant conceptual frameworks.     

SUPPORT Tools for evidence-informed health Policymaking (STP) 17: Dealing with insufficient research evidence

Background

Health systems research is being increasingly called upon to support scaling up of disease control interventions and to support rapid health sector change. Yet research capacity building and pay-back take years or even decades to be demonstrated, while leadership and institution building are critical for their success. The case of Mexico can be illustrative for middle income countries and emerging economies striving to build health research systems.

Methods

Historical reflection suggests the relationship between health sector reforms and economic crisis, on the one hand, and research capacity building and payback, on the other. Mexico's post-revolutionary background and its three health sector reforms are analyzed to identify the emphases given to health systems research.

Results

The first wave of health reform in the 1940s emphasized clinical and epidemiological research. Health systems research was not encouraged in a context of rapid economic development and an authoritarian regime. In contrast, health systems research was given a privileged place with the second wave of health reforms in the 1980s, which addressed health system coordination, decentralization and the universal right to health in a context of a deep economic crisis. The third wave of health reforms between 2003 and 2006 was based on the health system models proposed through research in the 90s. The credibility gained by research institutions was critical to ensure government uptake. Research influence can be traced through the role it played in defining a problem, in designing innovative insurance mechanisms and in establishing evaluation frameworks. It is argued that the Ministry of Health's budget increase of 56% between 2003 and 2006 and the reductions in inequity are pay-back to research investments since the 1980s.     

Incentives and disincentives for research and development of new drugs by the pharmaceutical industry

The authors present a model with factors that influence research and development decisions by the pharmaceutical industry: risk of disease transmission and possibility of control; case-fatality and the presence of cure or treatments; income; number of persons who demand the medicine; and opportunity costs for the company. Companies tend to invest in markets with inelastic demand (highly contagious diseases with no possibility of controlling transmission and/or very lethal diseases without treatment) and/or where there is a large population or high per capita income. Companies tend not to invest in markets where marginal costs exceed marginal income, particularly when costs increase permanently as a consequence of rising opportunity costs generated by foregoing profit in other markets. In such cases, policies to subsidize R&D are not effective, and policies must be orientated towards strengthening basic and applied research by public institutions.     

Shaping the future: a primary care research and development strategy for Scotland

Primary care is at the centre of the National Health Service (NHS) in Scotland; however, its R & D capacity is insufficiently developed. R&D is a potentially powerful way of improving the health and well-being of the population, and of securing high quality care for those who need it. In order to achieve this, any Scottish strategy for primary care R&D should aim to develop both a knowledge-based service and a research culture in primary care. In this way, decisions will be made based upon best available evidence, whatever the context. Building on existing practice and resources within primary care research, this strategy for achieving a thriving research culture in Scottish primary care has three key components: A Scottish School of Primary Care which will stimulate and co-ordinate a cohesive programme of research and training. A comprehensive system of funding for training and career development which will ensure access to a range of research training which will ensure that Scotland secures effective leadership for its primary care R&D. Designated research and development practices (DRDPs) which will build on the work of existing research practices, in the context of Local Health Care Co-operatives (LHCCs) and Primary Care Trusts (PCTs), to create a co-operative environment in which a range of primary care professionals can work together to improve their personal and teams' research skills, and to support research development in their areas. A modest investment will create substantial increases in both the quality and quantity of research being undertaken in primary care. This investment should be targeted at both existing primary care professionals working in service settings in primary care, LHCCs and PCTs, and at centres of excellence (including University departments). A dual approach will foster collaboration and will allow existing centres of excellence both to undertake more primary care research and to support the development of service based primary care professionals in their research. Resources should be distributed equitably, taking into account demography, geography and the health needs of patients in Scotland. The strategy and its components must be seen as a whole. The Scottish School of Primary Care will stimulate and co-ordinate both research and training programmes. DRDPs will become research active and will participate in School-led training and research, and will contribute to research programmes. Comprehensive funding for training and career development will ensure that staff have the skills to participate in both DRDPs and in the School's activities. Thus, inadequate commitment to any one component of the strategy will mean that other components will be less successful. Commitment to all three components will maximise the chances of success.     

Mixed signals: public policy and the future of health care R&D

The incentives facing health care research and development (R&D) are influenced by the ambiguous signals sent by private and public insurance decisions affecting the use of, and payments for, existing technologies. Increasingly, that uncertainty is exacerbated by confusion over technologies' impact on health care costs, how costs are to be measured, and the social difficulty of determining medical "need" for purposes of insurance coverage. R&D executives appear to believe that "major" advances are more likely to win such coverage and thus to be profitable. The products that result, therefore, may make the current policy dilemma of cost containment versus service restriction more acute rather than less so. If the aim of policy is to cut costs, innovative remedies are necessary.     

A research and development (R&D) roadmap for broadly protective coronavirus vaccines: A pandemic preparedness strategy

Broadly protective coronavirus vaccines are an important tool for protecting against future SARS-CoV-2 variants and could play a critical role in mitigating the impact of future outbreaks or pandemics caused by novel coronaviruses. The Coronavirus Vaccines Research and Development (R&D) Roadmap (CVR) is aimed at promoting the development of such vaccines. The CVR, funded by the Bill & Melinda Gates Foundation and The Rockefeller Foundation, was generated through a collaborative and iterative process, which was led by the Center for Infectious Disease Research and Policy (CIDRAP) at the University of Minnesota and involved 50 international subject matter experts and recognized leaders in the field. This report summarizes the major issues and areas of research outlined in the CVR and identifies high-priority milestones. The CVR covers a 6-year timeframe and is organized into five topic areas: virology, immunology, vaccinology, animal and human infection models, and policy and finance. Included in each topic area are key barriers, gaps, strategic goals, milestones, and additional R&D priorities. The roadmap includes 20 goals and 86 R&D milestones, 26 of which are ranked as high priority. By identifying key issues, and milestones for addressing them, the CVR provides a framework to guide funding and research campaigns that promote the development of broadly protective coronavirus vaccines.     

Measurement of the translation and impact from a childhood obesity trial programme: rationale and protocol for a research impact assessment

Background

There is growing recognition amongst health and medical research funders and researchers that translation of research into policy and practice needs to increase and that more transparency is needed on how impacts are realised. Several approaches are advocated for achieving this, including co-production of research or academic-practitioner research. The Population Health Unit (PHU) within the Hunter New England Local Health District in regional Australia, as an early adopter of this model, has been working to increase the likelihood that its research is translated into community health benefits. With the New South Wales Ministry of Health, the PHU responded to the burden of child overweight and obesity by combining service delivery with research expertise. The ‘Good for Kids, Good for Life’ (Good for Kids) dissemination trial was developed and implemented in seven community settings in the Hunter region of Australia between 2006 and 2010. This study aims to undertake a retrospective impact assessment to measure the research translation and impact of Good for Kids.

Methods

The method will be based upon the application of the Framework to Assess the Impact from Translational health research (FAIT), comprising three core elements, namely quantified metrics, economic assessment and a narrative of the process by which the research in question translates and generates impact.

Discussion

Increasingly, funders are interested both in the outcomes resulting from investments in health research and in the expected return on their investments. FAIT was developed specifically for this purpose and its use is anticipated to provide transparency to the pathway to translation and potentially drive increased investment in translational research programmes such as Good for Kids.

     

On being a good listener: setting priorities for applied health services research

In the last decade, explicit priority setting has become an integral part of health care systems. Indeed, there is even an International Society on Priorities in Health Care, created in 1997 ( Ham 1997 ). Whether it is Oregon's priority ordering of symptom treatment pairs to maximize the impact of a limited Medicaid budget (Fox and Leichter 1991), England's National Institute for Clinical Excellence's assessing priorities for new therapeutic innovations in the National Health Service ( Rawlins 1999 ), or New Zealand's setting priorities for patients' access to cardiovascular treatment ( Hadorn and Holmes 1997 ), techniques for judging the relative worth of different health service investments abound. As these techniques are refined, the most common addition is the incorporation of public values as part of the assessment. Priority setting is increasingly seen as combining an objective assessment of costs and effects with a more subjective assessment of patient or public preferences ( Lenaghan, New, and Mitchell 1996 ; Lomas 1997 ; National Institute for Clinical Excellence 2002 ; Stronks et al. 1997 ).