Breakthroughs Blog: Guest post

Soma Ghoshal is a program officer with the Results for Development Institute. In this guest post, she writes about a recent event that examined a new World Health Organization (WHO) report on global health research and development (R&D) financing. The WHO Consultative Expert Working Group on Research and Development: Financing and Coordination (CEWG) recently released a new report, titled “Research and Development to Meet Health Needs in Developing Countries: Strengthening Global Financing and Coordination.” The report lays out the working group’s vision for principles, policies, and a framework that should be embodied in a global R&D system to meet the needs of those for whom markets fail. On May 7, the Results for Development Institute, Georgetown University’s O’Neill Institute for National and Global Health, and the Harvard Global Health Institute co-hosted an event to debate and discuss this critical report—the first of its kind for a multilateral institution like the WHO. Held at the Results for Development Institute offices in Washington, DC, the event was attended by 65 interested R&D practitioners, policymakers, and other global health stakeholders. John-Arne Rottingen, the chair of the CEWG, kicked off the event with a keynote presentation emphasizing the need for better and more sustainable financing and coordination mechanisms for diseases affecting low- to middle-income countries. He mentioned four underlying concepts that should be the foundation of a global framework for health R&D: Affordable products can best be achieved through free open market competition in production. R&D costs and prices of products should be delinked. Upfront public financing of R&D is needed. R&D is reestablished as a global public good where there is need for collective action and agreed financing contributions to avoid free riding. Rottingen emphasized that these elements should be embodied in a global binding instrument such as an international convention or treaty. Much like the WHO Framework Convention on Tobacco Control, agreeing to convention for R&D with legal force and an associated financial commitment would be intended to represent a systematic and sustainable solution, brought about by collective political leadership. “This is the challenge for the world as a whole, which has guided our discussions and deliberations. We have framed our recommendations to indicate that finding solutions is the responsibility of all of us in this interdependent world, in developed and developing countries alike,” Rottingen said at the event. After Rottingen’s presentation, the first panel took the stage, moderated by Robert Hecht, managing director at Results for Development Institute. Discussants included: Nils Daulaire, Office of Global Health Affairs, Department of Health and Human Services. Judit Rius, Médecines sans Frontières Access Campaign. Paul Wilson, Results for Development Institute and Columbia University. Rachel Cohen, Drugs for Neglected Diseases initiative. A lively discussion ensued regarding the proposals for new funding and coordinating mechanisms. The timeliness of the report and the US Government’s involvement in global health R&D were also topics of discussion. Rius spoke about the concept of delinkage, or separating the cost of R&D from the end product price. She explained that high product prices reflect high costs associated with R&D, and by de-linking these two aspects, products can be made more affordable while R&D can still persist. Daulaire articulated the US Government’s stance on global health R&D, explaining that the US Government clearly has a vested interest in global health R&D, which has been plainly shown through its investments. While it is promising that there seems to be an emerging consensus about how to address global health R&D needs throughout the world, there is still much work that needs to be done to create a viable framework that moves beyond (successful) voluntary bilateral and philanthropic efforts to more sustainable global commitments. After a brief question and answer period, the second panel began its discussion, moderated by Larry Gostin, director of the O’Neill Institute for National and Global Health Law at Georgetown University. The panelists included: Suerie Moon, Global Health Institute, Harvard University. Benn McGrady, O’Neill Institute, Georgetown University. Jamie Love, Knowledge Ecology International. This panel discussion focused on the binding instrument and the implications of using legal force to bring about collective action for R&D. Topics discussed included the international treaty on tobacco control and the merits of using a binding instrument for R&D. Moon highlighted that treaty backers will have to consider whether to seek an instrument that is universal, and potentially weaker, or go for a stronger treaty that includes fewer countries. McGrady noted how a convention on R&D may sort out problems that have kept R&D investments low, such as a lack of mobilization of relevant interest groups and a challenge in establishing common norms by which different countries should abide. In addition, Love commented on the merits of broadening the mandate of the WHO R&D Convention to encompass more than just developing countries, but rather to go for a “big” R&D treaty that includes R&D for non-communicable and other diseases affecting developed countries. Love felt that broadening the treaty scope has the potential to address more global health needs, which may be attractive to US or European country governments trying to control health care costs in times of austerity and who may be more willing to join if the treaty is seen as beneficial to their own populations. The event concluded with the clear message that although arriving at a consensus that more attention needs to be paid to R&D is progress in and of itself, this moment must be leveraged appropriately, and there is much work that needs to be done.

Michel Zaffran is the coordinator for the Expanded Programme on Immunization at the World Health Organization (WHO). He is also the director of Project Optimize at PATH. In this guest post, Zaffran writes about how the Bill & Melinda Gates Foundation is seeking new ideas for immunization systems and vaccine product characteristics. As one of the most cost-effective health interventions, new lifesaving vaccines bring great promise to communities around the world. However, these new vaccines often present challenges to the immunization supply systems of low- and middle-income countries. Some new vaccines can occupy more space, may be more complex to prepare for administration, and may have temperature requirements that are not suited to the challenging environment of low- and middle-income countries. The Bill & Melinda Gates Foundation’s Grand Challenges Explorations grants program has launched a new call seeking ideas to improve immunization supply systems, including vaccine product characteristics that could have implications for research and development. For example: Tools to enable countries, donors, and procurement agencies to assess the systems costs and benefits of vaccine product presentations and formulations, rather than product price alone, in order to inform their vaccine purchase decisions and optimize their relevance to immunization strategies. Low-cost vial-level temperature indicators that display both cumulative and peak exposures to heat. Non-formulation means to prevent vaccines from freezing and/or to guarantee the sterility of opened multi-dose vials during storage and use. Any other innovation relevant to vaccine characteristics that do not necessarily involve preclinical or clinical research or improving vaccine stability or formulation. Anyone with a good idea is eligible to submit a two-page proposal, including public health specialists, researchers, entrepreneurs, engineers, or students working in nongovernmental organizations, ministries of health, universities, or companies. The program offers $100,000 grants, and successful projects may be eligible for additional funding up to $1 million. The submission deadline is May 15, 2012. This call for proposals builds on work facilitated by Project Optimize—a collaboration between PATH and the WHO—to develop a common vision for the future of immunization supply systems and logistics. More information Grand Challenges Explorations call for proposals Grand Challenges Explorations program Project Optimize on the PATH and WHO websites.

In this guest post, Mandy Goldberg from Research!America writes about an event today in Washington, DC, that focuses on research and development (R&D) for new malaria tools. The event is sponsored by Research!America, the Global Health Technologies Coalition (GHTC), and several other partners. In the United States, the bite of a mosquito is uncomfortable and irritating. For 3.3 billion people abroad, a mosquito bite can be deadly. Almost half of the global population is at risk of contracting malaria from the bite of an infected mosquito. Although malaria is preventable and curable, an estimated 655,000 people, mostly children in Africa, died from the disease in 2010. On April 25, the global health community commemorates the fifth annual World Malaria Day by recognizing the progress that has been made in the fight against malaria and calling for increased investment to sustain this momentum and achieve malaria control worldwide. The current basis of malaria control efforts are mosquito control strategies and treatment. Studies have shown that bednets can reduce malaria transmission by 90 percent in areas with high coverage rates. In addition, indoor residual spraying with insecticides can also reduce malaria transmission in areas with at least 80 percent coverage. In the past decade, malaria deaths have decreased by one-third in Africa. In countries with improved access to malaria control interventions, child mortality rates have fallen by 20 percent. More research is needed, however, to develop new, longer-lasting tools to bring eradication within reach. Photo credit: PATH/Evelyn Hockstein The tide is turning against malaria, thanks in part to remarkable achievements in R&D. In the past decade, malaria deaths have decreased by one-third in Africa. In countries with improved access to malaria control interventions, child mortality rates have fallen by 20 percent. More research is needed, however, to develop new, longer-lasting tools to bring eradication within reach. Hope on the way: the malaria product pipeline Anti-malarial medicines are used for curative and preventive purposes, but the increasing threat of drug resistance is making R&D for new, more effective drugs even more essential. Widespread resistance to the drug chloroquine in the 1970s and 1980s necessitated the use of artemisinin-based combination therapies (ACT), currently the best available treatment. The plant from which artemisinin is derived is in short supply in nature, threatening future availability of ACT. OneWorld Health, a drug development affiliate of PATH, in collaboration with private-sector partners, is working to solve this problem through the development and production of semi-synthetic artemisinin at an affordable price that can sustain the supply of ACT for years to come. In addition, the product development partnership Medicines for Malaria Venture (MMV) has several promising drug candidates in its R&D pipeline. MMV is developing a one-dose cure to replace current three-day regimens, eliminating the need for treatment follow-up and reducing the likelihood of drug resistance. MMV is also working on the development of non-artemisinin-based treatments. Considering recent reports of increasing resistance to ACTs in Southeast Asia, increased investment is needed to support the development of new therapies that may be necessary to control malaria in the future. The greatest hope for malaria eradication is the development of a safe, effective vaccine to prevent infection. RTS,S, the most advanced malaria vaccine candidate, has been shown to reduce the risk of infection by 50 percent among children age 5-17 months in a recent late-stage clinical trial. Developed through collaboration between the Walter Reed Army Institute of Research, GlaxoSmithKline, and the PATH Malaria Vaccine Initiative, this vaccine could be recommended for widespread use by the World Health Organization as early as 2015. Research has played an essential role in the progress already made against malaria, and additional scientific breakthroughs may be around the corner due to the robust R&D pipeline. Malaria R&D has the potential to save hundreds of thousands of lives, including American troops stationed abroad, and also has important economic benefits here at home by creating jobs and stimulating economic growth. In order to fully realize these health and economic benefits, increased investment in malaria R&D is required.

Mark Alderson, PhD, is the director of the pneumococcal vaccine project at PATH , a nonprofit international organization based in Seattle, Washington, specializing in global health. In this guest post, he writes about PATH’s efforts to develop new vaccines to fight pneumonia among children. In honor of World Immunization Week, the global health community is coming together to engage in immunization campaigns, advocacy, and awareness activities to bring the power of vaccines into the spotlight. Vaccines are one of the most successful and cost-effective technologies that prevent childhood mortality. According to the World Health Organization, immunization prevents between two and three million deaths per year. Regardless, many vaccine-preventable diseases continue to take lives unnecessarily because of gaps in vaccine research and development (R&D) that neglect developing-world needs. Barriers to vaccine access also prevent low- and middle-income populations from receiving the protection they need. Pneumonia—the number one killer of children under five years of age worldwide—is one such disease for which vaccines and treatments could save millions of lives. However, achieving sustainable global health impact over the long-term requires further investment in new vaccines that cater specifically to the developing world, where pneumonia hits hardest. To this end, public-private sector partnerships are essential to maximizing resources and speeding development. In the fight against pneumonia, we have reason to celebrate. Overall, investments in combating pneumonia have helped contribute to a 35 percent reduction in child mortality since 1990—no small feat. We have vaccines for several top causes of the disease, including the pneumococcus bacterium, which is the most common cause of severe childhood pneumonia and takes the lives of an estimated 800,000 children annually. Pneumococcal vaccines, having been accessible in the industrialized world for over a decade, are now rolling out in low-resource countries with the help of the GAVI Alliance and other groups. This month, Ghana is making history by becoming the first GAVI-eligible country to introduce two vaccines simultaneously—pneumococcal and rotavirus vaccines. The power of vaccines is evident, but unless we continue to invest in new vaccine tools that can provide better, more affordable protection, our ability to protect our children will be incomplete. Photo credit: PATH Despite these great strides, we must not forget that there is still a long way to go. The pneumococcal vaccines in use today are effective against certain varieties of the bacterium, but no vaccine yet exists that can protect against all of the more than 90 pneumococcal strains, leaving children susceptible to strains not covered by these vaccines. Also, current pneumococcal vaccines are relatively expensive, making them difficult for low- and middle-income countries to afford without substantial assistance from international donors. To fight pneumonia, which takes the life of one child every 20 seconds, we must waste no time in striving for broadly effective and affordable vaccines that can be sustainably accessible in even the most impoverished regions of the world. Because market forces alone are not always strong enough to drive the development of vaccines most urgently needed in lower-income countries, PATH is stepping in to help fill the gap. Our pneumococcal vaccine project is advancing R&D for new, low-cost pneumococcal vaccines that are tailored to meet the needs of children in the developing world. We collaborate with an array of public- and private-sector partners on vaccine R&D projects, including universities, research institutions, vaccine manufacturers, and biotechnology companies. We also work with US and foreign government agencies on epidemiology, vaccine research, and regulatory issues. Several of the vaccine candidates we support could provide broad protection against pneumococcus by using proteins that could help the body’s immune system recognize and defend against virtually all pneumococcal varieties. If successful, these vaccines could change the face of pneumococcal disease by providing universal protection at low cost. For candidates ready to enter clinical trials, we coordinate closely with the US Food and Drug Administration (FDA) to ensure compliance with regulatory requirements. We are also partnering in India to develop a vaccine candidate using technologies similar to current pneumococcal vaccines, but at significantly lower inherent cost. This vaccine candidate targets only pneumococcal varieties common in the developing world. If successful, India could become a global manufacturer of pneumococcal vaccines, blazing the trail for endemic country vaccine producers in a market previously occupied solely by European and US manufacturers. Recognizing that successful vaccine development requires access to a full spectrum of scientific resources, we also support research that advances the vaccine field at large. Among our various research partnerships are several with the US Government, including FDA and the US Centers for Disease Control and Prevention. Collaborations of this nature are essential to helping lay the scientific groundwork for new pneumococcal vaccines to come to fruition. The power of vaccines is evident, but unless we continue to invest in new vaccine tools that can provide better, more affordable protection, our ability to protect our children will be incomplete. Tackling prolific killers like pneumonia in the developing world is a critical step toward truly making an impact on the global burden of disease. Working in partnership here in the United States and abroad can help get us there sooner rather than later.