Recommendations for moving forward
9 October 2001
1. Because drug development is done almost exclusively within the context of the proprietary pharmaceutical industry, investment in R&D is guided by market considerations. Therefore R&D for diseases that mainly affect the poor is stifled. 2. Public policy has failed to correct this failure, with the result that some diseases are being neglected. 3. The dynamics of the neglect are different depending on the number of people affected and their purchasing power. Therefore it is impossible to develop a single strategy to stimulate R&D. It is crucial to acknowledge the different dynamics of neglected and most neglected diseases: each category will need distinct strategies. 4. A well-defined and needs-driven R&D agenda is required to assist policy makers, funding agencies and the research community in setting priorities for developing safe, effective and affordable medicines. The World Health Organization (WHO), as the only legally mandated international governmental agency responsible for global health, should work toward establishing an essential R&D agenda. WHO should lead this process. The DND Working Group, with input from WHO, has begun by drafting agendas that prioritize R&D needs for leishmaniasis, sleeping sickness, and malaria. These documents analyze the disease burden, current research strategies, and existing and potential treatments for each of these diseases. A critical next step is for governments and international organizations to examine carefully how they can contribute to dislodging the bottlenecks that currently restrict development of new treatments. 5. Governments in both developed and developing countries need to take comprehensive action to compensate for the market failure in drug development for neglected and most neglected diseases. Governments must lead in restarting R&D on diseases that are currently being ignored. They need to create and support new structures designed to develop essential medicines for diseases that are being sidelined by the private sector. The current model of profit-driven R&D should not be an exclusive model. Developing drugs as public goods should also be pursued. 6. Increased and reliable long-term funding for R&D into neglected diseases is urgently needed. The DND Working Group is exploring sustainable options to support R&D for neglected diseases through legal obligations. Governments can and do mandate industry spending in a wide range of areas. One example of a potential mandate would be an "essential research obligation" that would require companies to reinvest a percentage of pharmaceutical sales into R&D for neglected diseases, either directly or through public R&D programs. A global treaty on R&D for neglected diseases could provide a framework for such mandates. Such a treaty should correct the imbalance that exists between private sector rights and obligations under present international treaties and agreements (eg, the World Trade Organization's Agreement on Trade Related Aspects of Intellectual Property), and provide new legal options to make drugs for neglected diseases global public goods. 7. A complete cost analysis of the true costs of drug R&D should be carried out. Existing estimates on the costs of drug R&D vary widely and remain highly controversial. In order to address the R&D imbalance effectively and make informed funding decisions, policy-makers need objective, accurate figures on the true costs of developing drugs. Calculating drug development costs within a commercial context, which will include items such as opportunity costs, will be dramatically different from calculating the funding needed to develop a drug in a non-commercial setting. 8. Public funds for R&D into neglected diseases should be tied to guarantees of equitable access and affordability of the end product. Equitable access to medicines in developing countries should be a basic principle that guides policy initiatives from the start. If public funds are to be invested in correcting market failures in drug development, there must be guarantees that the new medicines developed are affordable to those who need them. 9. Focused capacity-building and technology transfer projects in developing countries should be encouraged as a direct way to increase R&D expertise and infrastructure. Long-term solutions to the current crisis in drug development for neglected diseases ultimately rest within developing countries. Therefore, the DND Working Group is cataloguing and examining means of increasing existing drug development capacity in developing countries, and is also working to promote technology transfer that will support sustainable drug development and production facilities. 10. An independent and thorough evaluation is needed of the current and future impact of the ongoing regulatory harmonization efforts (ICH process) on the ability of developing countries to increase their drug development efforts. 11. A new type of body is needed to contribute to drug development for the most neglected diseases. The DND Working Group is exploring the feasibility of a Not-for-Profit Initiative (DND NfPI) that would focus on drug development projects for neglected diseases. The DND Working Group's analysis has concluded that current approaches to address the lack of R&D for neglected diseases do not sufficiently address the most neglected diseases. To ensure a sustainable solution, a new approach is needed that would systemically harness funding, new science and technology, and foster public-private cooperation for these diseases. Encompassing the recommendations listed above, and based on the research of the DND Working Group, the vision of the proposed DND NfPI includes the following:
Ensuring equitable access to effective, field-relevant and easy-to-use drugs for neglected diseases.
Prioritizing the most neglected diseases, such as sleeping sickness, Chagas disease, and leishmaniasis.
Using sound science and management techniques to pursue a vision of developing new drugs for neglected diseases.
Collaborating closely with TDR, industry, and research institutes in developing and developed countries.
Securing support of public and private resources over the long term, with the majority of funding coming from the public sector.
Working with drug development experts in developing countries to build national capacity for future drug development.
It is hoped that the public sector will take a strong leadership role in the NfPI to establish its legitimacy and accountability to the public and provide it with the necessary funds.
Despite impressive advances in science and medicine, society has failed to allocate sufficient resources to battle the diseases that particularly affect people in poor countries. The vacuum in R&D for neglected and most neglected diseases means that doctors and nurses in developing countries still do not have effective medicines for many of the diseases they see every day. However, encouraging initiatives have emerged to counter the market and public policy failures that have led to this crisis. Many of these initiatives are new, and their effectiveness will need to be evaluated. For the most neglected diseases, implementation of new solutions, such as a not-for-profit initiative for developing drugs for neglected diseases, will be essential.