A survey of international legal instruments to examine their effectiveness in improving global health and in realizing health rights.

Many global health issues, almost by definition, do not recognize state borders and therefore require bi-lateral, or more often multi-lateral international solutions. These latter solutions are articulated in international instruments (declarations, conventions, treaties, constitutions of international bodies, etc). However, the gap between formal adoption of such instruments by signatory states and substantive implementation of the articulated solutions can be very wide. This paper surveys a selection of international legal instruments, including those where the sought after positive outcomes have been achieved, and those that have been ineffective, with little or no real progress being made. The paper looks for commonalities, both in the nature of the problems and the forms of the international legal instruments, to seek answers as to why some instruments ultimately succeeded where others failed. It also provides some guidance to law/ treaty makers to help ensure that they frame future instruments in such a way as to maximize the probability that those instruments will have a substantive positive impact on global health and health rights.

Emergence of biopharmaceutical innovators in China, India, Brazil, and South Africa as global competitors and collaborators.

Biopharmaceutical innovation has had a profound health and economic impact globally. Developed countries have traditionally been the source of most innovations as well as the destination for the resulting economic and health benefits. As a result, most prior research on this sector has focused on developed countries. This paper seeks to fill the gap in research on emerging markets by analyzing factors that influence innovative activity in the indigenous biopharmaceutical sectors of China, India, Brazil, and South Africa. Using qualitative research methodologies, this paper a) shows how biopharmaceutical innovation is taking place within the entrepreneurial sectors of these emerging markets, b) identifies common challenges that indigenous entrepreneurs face, c) highlights the key role played by the state, and d) reveals that the transition to innovation by companies in the emerging markets is characterized by increased global integration. It suggests that biopharmaceutical innovators in emerging markets are capitalizing on opportunities to participate in the drug development value chain and thus developing capabilities and relationships for competing globally both with and against established companies headquartered in developed countries.

Stakeholder involvement in expensive drug recommendation decisions: an international perspective.

OBJECTIVES: To describe stakeholder involvement in the priority setting and appeals processes across five drug reimbursement recommendation committees. METHODS: We conducted qualitative case studies of how five independent drug advisory committees from Canada, Israel, England and Wales, Australia, and the USA made funding decisions for six expensive drugs. Interviews with 48 informants were conducted with committee members, patient groups, and industry representatives. RESULTS: Different stakeholders were allowed, in varying degrees, to participate in the formal mechanisms for revisions and appeals of decisions. Participants identified a number of stakeholder groups who were already involved in the process, as well as stakeholders whom they believed should be included in the decision-making process. CONCLUSIONS: A central component of a legitimate and fair priority setting process is to make priority setting explicit and to involve both pertinent values and stakeholders in decision-making. Study participants believed that the involvement of multiple stakeholder groups within the deliberative and appeals/revisions processes would contribute to a fair and legitimate drug reimbursement process.

Health biotechnology innovation on a global stage.

With increasing globalization, infectious diseases are spreading faster than ever before, creating an urgent need for international collaboration. The rise of emerging economies has changed the traditional collaborative landscape and provided opportunities for more diverse models of collaboration involving developing countries, including North-South, South-South and North-South-South partnerships. Here, we discuss how developing countries can partner with other nations to address their shared health problems and to promote innovation. We look specifically at what drives collaborations and at the challenges that exist for them, and we propose actions that can strengthen these partnerships.

Priority setting for orphan drugs: an international comparison.

OBJECTIVES: To describe the process of priority setting for two orphan drugs - Cerezyme and Fabrazyme - in Canada, Australia and Israel, in order to understand and improve the process based on stakeholder perspectives. METHODS: We conducted qualitative case studies of how three independent drug advisory committees made decisions relating to the funding of Cerezyme and Fabrazyme. Interviews were conducted with 22 informants, including committee members, patient groups and industry representatives. RESULTS: (1) DESCRIPTION: Orphan drugs reimbursement recommendations by expert panels were based on clinical evidence, cost and cost-effectiveness analysis. (2) EVALUATION: Committee members expressed an overall preference for the current drug review process used by their own committee, but were concerned with the fairness of the process particularly for orphan drugs. Other informants suggested the inclusion of other relevant values (e.g. lack of alternative treatments) in order to improve the priority setting process. Some patient groups suggested the use of an alternative funding mechanism for orphan drugs. CONCLUSIONS: Priority setting for drugs is not solely a technical process (involving cost-effective analysis, evidence-based medicine, etc.). Understanding the process by which reimbursement decisions are made for orphan drugs may help improve the system for future orphan drugs.

Venture funding for science-based African health innovation.

BACKGROUND: While venture funding has been applied to biotechnology and health in high-income countries, it is still nascent in these fields in developing countries, and particularly in Africa. Yet the need for implementing innovative solutions to health challenges is greatest in Africa, with its enormous burden of communicable disease. Issues such as risk, investment opportunities, return on investment requirements, and quantifying health impact are critical in assessing venture capital's potential for supporting health innovation. This paper uses lessons learned from five venture capital firms from Kenya, South Africa, China, India, and the US to suggest design principles for African health venture funds. DISCUSSION: The case study method was used to explore relevant funds, and lessons for the African context. The health venture funds in this study included publicly-owned organizations, corporations, social enterprises, and subsidiaries of foreign venture firms. The size and type of investments varied widely. The primary investor in four funds was the International Finance Corporation. Three of the funds aimed primarily for financial returns, one aimed primarily for social and health returns, and one had mixed aims. Lessons learned include the importance of measuring and supporting both social and financial returns; the need to engage both upstream capital such as government risk-funding and downstream capital from the private sector; and the existence of many challenges including difficulty of raising capital, low human resource capacity, regulatory barriers, and risky business environments. Based on these lessons, design principles for appropriate venture funding are suggested. SUMMARY: Based on the cases studied and relevant experiences elsewhere, there is a case for venture funding as one support mechanism for science-based African health innovation, with opportunities for risk-tolerant investors to make financial as well as social returns. Such funds should be structured to overcome the challenges identified, be sustainable in the long run, attract for-profit private sector funds, and have measurable and significant health impact. If this is done, the proposed venture approach may have complementary benefits to existing initiatives and encourage local scientific and economic development while tapping new sources of funding.

Science-based health innovation in Tanzania: bednets and a base for invention.

BACKGROUND: Tanzania is East Africa's largest country. Although it is socially diverse, it has experienced general political stability since independence in 1964. Despite gradual economic development and Tanzania's status as one of the biggest recipients of aid in Africa, health status remains poor. This paper explores Tanzania's science-based health innovation system, and highlights areas which can be strengthened. METHODS: Qualitative case study research methodology was used. Data were collected through reviews of academic literature and policy documents, and through open-ended, face-to-face interviews with 52 people from across the science-based health innovation system over two visits to Tanzania from July to October 2007. RESULTS AND DISCUSSION: Tanzania has a rich but complex S&T governance landscape, with the public sector driving the innovation agenda through a series of different bodies which are not well-coordinated. It has some of the leading health research on the continent at the University of Dar es Salaam, Muhimbili University of Health and Applied Sciences, the National Institute for Medical Research and the Ifakara Medical Institute, with strong donor support. Tanzania has found developing an entrepreneurial culture difficult; nevertheless projects such as the clusters initiative at the University of Dar es Salaam are encouraging low-tech innovation and overcoming knowledge-sharing barriers. In the private sector, one generics company has developed a South-South collaboration to enable technology transfer and hence the local production of anti-retrovirals. Local textile company A to Z Textiles is now manufacturing 30 million insecticide impregnated bednets a year. CONCLUSIONS: To have a coherent vision for innovation, Tanzania may wish to address some key issues: coordination across stakeholders involved with health research, increasing graduates in health-related disciplines, and building capabilities in biological testing, preclinical testing, formulation and standardization, and related areas important to moving from basic research to applications. The private sector can be encouraged to innovate through improved access to financing, and incentives for R&D. The diaspora community represents an untapped source for partnerships and access to other developing world markets and technology. The government may wish to set up mechanisms to encourage south-south collaborations, and to bring the public and private sector together around specific projects to help realize the country's innovation potential.

Regenerative medicine in Brazil: small but innovative.

AIMS: Although Brazil has received attention for conducting one of the world's largest stem cell clinical trials for heart disease, little has been published regarding Brazil's regenerative medicine (RM) sector. Here we present a comprehensive case study of RM in Brazil, including analysis of the current activity, the main motivations for engaging in RM and the remaining challenges to development in this field. METHODS: Our case study is primarily based on semi-structured interviews with experts on RM in Brazil, including researchers, policymakers, clinicians, representatives of firms and regulators. RESULTS: Driven by domestic health needs and strategic government support, Brazil is producing innovative RM research, particularly for clinical research in cardiology, orthopedics, diabetes and neurology. We describe the main RM research currently taking place in Brazil, as well as some of the economic, regulatory and policy events that have created a favorable environment for RM development. Brazilian RM researchers need to overcome several formidable challenges to research: research funding is inconsistent, importation of materials is costly and slow, and weak linkages between universities, hospitals and industry impede translational research. CONCLUSIONS: Although Brazil's contribution to the RM sector is small, its niche emphasis on clinical applications may become of global importance, particularly if Brazil manages to address the challenges currently impinging on RM innovation.

Shared principles of ethics for infant and young child nutrition in the developing world.

BACKGROUND: The defining event in the area of infant feeding is the aggressive marketing of infant formula in the developing world by transnational companies in the 1970s. This practice shattered the trust of the global health community in the private sector, culminated in a global boycott of Nestle products and has extended to distrust of all commercial efforts to improve infant and young child nutrition. The lack of trust is a key barrier along the critical path to optimal infant and young child nutrition in the developing world. DISCUSSION: To begin to bridge this gap in trust, we developed a set of shared principles based on the following ideals: Integrity; Solidarity; Justice; Equality; Partnership, cooperation, coordination, and communication; Responsible Activity; Sustainability; Transparency; Private enterprise and scale-up; and Fair trading and consumer choice. We hope these principles can serve as a platform on which various parties in the in the infant and young child nutrition arena, can begin a process of authentic trust-building that will ultimately result in coordinated efforts amongst parties. SUMMARY: A set of shared principles of ethics for infant and young child nutrition in the developing world could catalyze the scale-up of low cost, high quality, complementary foods for infants and young children, and eventually contribute to the eradication of infant and child malnutrition in the developing world.

Globetrotting firms: Canada's health biotechnology collaborations with developing countries.

A survey of Canadian biotech firms reveals that their biotech collaborations with developing countries are not only significant but also increasingly reciprocal in terms of the exchange of financial resources and technological know-how.

Small but tenacious: South Africa's health biotech sector.

Despite a challenging business environment, entrepreneurial health biotech companies in South Africa are finding ways to succeed.