Neglected diseases: On the possible role of public research.

Currently, respiratory infections, malaria and tuberculosis are the leading causes of death and morbidity in Africa, Asia and South America, where four-fifth of the world's population live. Effective, affordable and easy-to-use medicines to fight these infectious diseases are nearly absent. With market prospects rather than global health needs guiding drug development, the pipeline of new drugs for these neglected diseases is almost empty. Less than 10 per cent of the worldwide expenditure on health research and development (R&D) is devoted to the major health problems of 90 per cent of the population. Exacerbated by the HIV/AIDS pandemic, awareness is growing that firmly increased and focused R&D efforts are needed to address this global public health crisis. To correct the current market failure, a variety of measures are proposed to mobilize existing R&D capacity in both the public and the private sector.

While private sector incentives focus on improving profitability prospects, the public research community is attracted by increased funding opportunities, implicitly assuming this suffices to direct researchers towards neglected diseases R&D. However, while ensuring the development of new health tools for neglected diseases is ultimately a public responsibility, public research policies are not always at ease with a leading role in it and encounter several constraints.

Financing health research

With an estimated US$ 60 to 70 billion in 2001, worldwide spending on health R&D is higher than ever, but the commercial sector has become by far the biggest investor in health research and dominates priority setting, based on market prospects. It pays off, as according to Fortune500 , pharmaceutical companies top the industry performance list for return on revenue. Meanwhile, in the current transition towards a global knowledge economy, public sector policies increasingly view public research as an investment that needs to generate financial return. Scientists are not only requested to publish their research and advance science, but also to promote and actively pursue the possible commercialisation of research findings. This so-called valorization of research has become an important objective of public research, especially in the biotechnology and health sector where financial returns prove very attractive. As valorization prospects are now included in the evaluation criteria for public research, research choices inevitably start reflecting market opportunities. Thus, the same market failure that deters the pharmaceutical industry from investing in neglected diseases also discourages public research.

Drug R&D is not innovative science

Developing a new drug is a highly complex process, requiring the co-ordinated mobilization of different kinds of expertise and activities over a protracted period of time. To turn a viable idea from basic research into a pharmaceutical product can easily take ten years. Although scientific research underlies the process, the most innovative part is the initial target discovery and the identification of lead components. The subsequent work of product optimization and validation, represents very little scientific interest. However, the performance criteria for public research, both for an individual career in terms of funding opportunities, are based on scientific excellence and on creative and innovative research publishable in high-ranking journals. True drug development activities do not qualify for this and so are unattractive for academic scientists and public science funding agencies. Not surprisingly, the most important gap in the drug R&D process for neglected diseases is the transfer from basic to pre-clinical research. For a drug candidate with promising market prospects this would normally coincide with the transfer from the public to the private sector.

Incompatible organization forms

In general, the public research sector has little expertise for true product development (which has little to do with science), and typically operates with short-term research grants of two to three years. It uses a bottom-up approach in which a large number of small research groups with specific expertise contribute in a fragmented way to the general scientific progress. This open-ended and non-goal directed nature of scientific research lies at the heart of scientific creativity and is essential. It however contrasts sharply with the focused, long-term project management and leadership that is required to take a drug candidate through the development pipeline.

New approaches to ensuring drug development for neglected diseases rightly expect a significant contribution of public sector research. But the mere existence of scientific expertise does not suffice. Where the market fails, a global non-profit strategy for drug development should be developed and implemented within the public sector. Existing counter-currents in public science policy need to be overcome. First, in the expected 'valorization' of public research, non-commercial goals such as pursuing public health interests and social returns should be emphasised over the economic goals. Second, the current public research focus on innovative science needs to be extended to include more technical drug development activities, and appropriate funding opportunities and career incentives should be developed accordingly. Finally , public leadership should be created for managing the R&D process from basic science to clinical product development. Only in this way can the public sector play its requisite role in ensuring drug development for neglected diseases.