Breakthroughs Blog: NIH

With the ever-present specter of federal budget reductions that could impact global health and development programs, the GHTC’s event on Friday to launch our new report couldn’t have come at a better time. The report, released by the GHTC and Policy Cures, provides new and exciting data on US investments over the past 10 years in global health research and development (R&D). It confirms that support from the US Government and its federal agencies has not only contributed to the largest pipeline of new global health products in history, but also that these investments make smart economic sense domestically. Dr. Javier Guzman, director of research at Policy Cures, shared the report’s main findings at Friday’s event, which was co-hosted by Research!America. Guzman outlined that the US Government has contributed significantly to global health R&D—indeed, the United States is the largest funder of R&D efforts worldwide, contributing $12.7 billion from 2000 to 2010. This support has translated into the largest pipeline of new global health drugs, vaccines, diagnostics, and other products, over half of which are supported by the United States. The US Government “needs to keep this momentum going” because “investments in global health R&D really work,” Guzman said. Guzman also stressed a key theme that emerged during the event—that of partnerships. According to Guzman, critical partnerships have formed between the US Government and the active global health R&D community that has emerged over the past ten years. This community includes philanthropic organizations, the pharmaceutical and biotechnology industries, academic institutions, and non-profit groups. The success of these partnerships can be seen in the development and delivery of new health tools like the Xpert ® MTB/RIF test for tuberculosis (TB), which Guzman said is projected to save 100,000 lives every year in India alone. Javier Guzman, Lee Hall, Eric Easom, and Heather Ignatius. Photo credit: PATH Dr. Lee Hall—chief of the Parasitology and International Programs Branch at the National Institute of Allergy and Infectious Diseases (NIAID)—also spoke about the importance of partnerships to his branch at the National Institutes of Health. According to Hall, partnerships between NIAID and academic institutions, industry, endemic country governments, and global organizations are “essential” to translating research into effective products. And Heather Ignatius, senior manager for policy at the Global Alliance for TB Drug Development, stressed that product development partnerships (PDPs) like her organization rely on collaboration with philanthropic organizations such as the Bill & Melinda Gates Foundation, which is currently the top funder of PDPs worldwide, as well as governments and industry groups. Additionally, the panelists emphasized how supporting R&D for new global health tools ultimately benefits the United States. Hall discussed how when President Roosevelt dedicated the NIH in 1940, he identified global health as a priority for the agency. “What perhaps started for humanitarian reasons also has benefits for the US,” Hall said, adding that better health products are needed in this era where infectious diseases know no borders. Guzman also shared how global health R&D creates jobs for Americans and boosts the economy. In 2007, for example, federal support for global health R&D paid $540 million in wages for employees in California. And Eric Easom, program leader for neglected diseases at the California biotechnology company Anacor Pharmaceuticals, reinforced this point when he said that conducting global health R&D allows his company to provide jobs for talented researchers and employees who “truly care about this work. It’s the reason we got into this business in the first place.” Finally, speakers addressed how continued support for research to develop new global health products has the potential to make an enormous impact across the globe. When discussing Anacor’s partnership with the Drugs for Neglected Diseases initiative to develop a new treatment for sleeping sickness, Easom said that the drug candidate is an “innovation that could totally change the game for patients” with the disease. Ignatius emphasized how the US Government must continue its support for global health R&D, and provide increased support for mechanisms like PDPs—much in line with the report recommendations. “We’re on the verge of developing and delivering drastically improved tools. They won’t get there if we can’t finish these final stages of testing,” she said, adding, “Donors must scale up their commitments, and we need increased involvement from the US Government. Now is the time.”

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. The National Institutes of Health has launched a new program that will pair researchers with certain pharmaceutical industry compounds to help scientists explore new treatment options for patients. The TuBerculosis Vaccine Initiative and GHTC member Aeras have announced a new memorandum of understanding to strengthen collaborative efforts to advance the world’s most promising tuberculosis vaccine candidates. The Senate Appropriations Committee has approved fiscal year (FY) 2013 budget legislation for the US Food and Drug Administration (FDA). The bill would provide the FDA with $2.524 billion for FY 2013, which is $12.5 million more than the spending plan submitted in February by President Obama and $27.47 million more than the current fiscal year. The House Appropriations Committee has proposed reducing the State and Foreign Operations account by more than $5 billion for FY 2013. The State and Foreign Operations account—which saw an overall increase in President Obama’s budget request—funds the US Agency for International Development and the State Department, as well as programs such as the President’s Emergency Plan for AIDS Relief.

Did you know that Americans spend more money on ice cream in three weeks than the US Government spends on global health research and development (R&D) in a year? And despite this relatively modest investment in the creation of new global health tools, the United States is the worldwide leader in R&D for neglected diseases. When we at the GHTC tell US policymakers the story of how global health R&D has revolutionized people’s lives around the world, we highlight the prominent role that the US Government plays in funding and supporting the development of new global health tools. We’ve always believed that US leads the world in investment in global health R&D, and that this comparatively small investment has paid off many times over in lives and dollars saved. We’ve seen the effectiveness of US-funded technologies like the polio vaccine, oral rehydration solution, and HIV and AIDS drugs. We’ve known that tools in the pipeline would have similar game-changing effects. And now we have the evidence to prove it. A new report, released today by the GHTC and Policy Cures, provides important confirmation that the US is the largest global health R&D funder in the world, investing $12.7 billion over the last decade. A new report, released today by the GHTC and Policy Cures, provides important confirmation that the US is the largest global health R&D funder in the world, investing $12.7 billion over the last decade. In fact, the US government in the last decade more than doubled its annual commitment to developing new global health products, from $685 million in 2000 to $1.4 billion in 2010. These investments represent just 0.01 percent of US gross domestic product. Our report confirms the strong leadership role the US has played in the global health R&D landscape, and also includes new information on spending and support by key US federal agencies—the Centers for Disease Control and Prevention, Department of Defense, US Food and Drug Administration, National Institutes of Health, and US Agency for International Development. New information also details the public health impact of products already on the market, and new health and economic projections detail the possible impact for health tools still in development. There is an exciting pipeline of 365 new vaccines, drugs, diagnostic, and other products for global health, and the US Government is supporting 200 of them. These are just a few examples of the immense impact of global health R&D, and the incredible benefits it will continue to have if robust support continues. The US is not alone in this effort—many governments around the globe, philanthropic foundations, and private industry partners provide large amounts of funding, technical expertise, and key intellectual property to develop new global health tools. The global health R&D landscape would not be the same without these important allies. While the success of US investment in global health R&D is exceptional, the report makes clear that sustained and increased US support is essential to ensuring that the potential of global health research is not lost. The report provides recommendations for how the US Government can maximize its investments in new global health products. The recommendations are: The US Government should maintain its funding for global health R&D, and increase this funding where possible. The US Government needs to have a greater focus on the later stages of research and product development to fully leverage its global health R&D investments. The US Government should increase funding to partnering mechanisms that are focused on translation of global health research. With all of the amazing results of global health R&D we have seen—and with this new evidence that global health R&D is a worthwhile, cost-effective investment—it would be a shame to retreat now. With the US leadership role comes the responsibility to continue pushing the boundaries of science and leading the world in combating global health diseases.

In this guest post, Dick Walker—director of the Enteric Vaccine Initiative at PATH’s Vaccine Development Program—writes about how the double-mutant heat-labile toxin is a potential booster for much-needed new vaccines against diarrhea. The leading bacterial causes of diarrhea are enterotoxigenic Escherichia coli (ETEC) and Shigella, and vaccines against these pathogens could save the lives of up to a half-million children each year. PATH’s enteric vaccine project is working to accelerate the development of several promising vaccine candidates. We are also looking at innovative ways to make these vaccines as effective, practical, and affordable as possible so they can be easily implemented in the developing world. Adjuvants, ingredients that may enhance the effectiveness of some vaccines, are one important area of focus. PATH is currently involved in research on a highly promising new adjuvant, the double-mutant heat-labile toxin (dmLT), which could open more doors to achieving immunity in the intestine. The dmLT is actually an ETEC antigen (a substance that causes the immune system to produce antibodies against it), so it may offer protection against both diarrhea and intestinal infection when used in conjunction with vaccines that are targeted to mucosal surfaces like the intestine. We aren’t exactly sure why, but children in resource-poor countries often have a lower response rate to mucosal vaccines. A wide range of factors may contribute to this issue, such as the influence of breastfeeding and maternal antibodies, poor nutrition, or increased exposure to intestinal pathogens. An adjuvant like the dmLT may help to bridge this gap in effectiveness. Dick Walker with children in Cambodia. Promising vaccine technologies can help expand and improve the arsenal of tools to use in the fight against diarrhea among children. Right now, we’re working with the National Institutes of Health’s (NIH) Division of Microbiology and Infectious Diseases on a Phase 1 clinical trial using oral doses of dmLT (not in conjunction with a vaccine) in healthy adults. We will have complete results later this year, but the preliminary data are quite promising. Meanwhile, we are about to launch our first Phase 1 trial of the dmLT combined with one of our oral ETEC vaccine candidates. In addition, we plan to start another Phase 1 trial to test the dmLT intradermally (through the skin), and we would also like to study its use sublingually (under the tongue), to see if these alternative delivery routes can achieve an immune response in the intestine. If preclinical studies accurately predict what will be seen in humans, delivering dmLT in these ways could open up a whole range of possibilities for developing more effective and practical vaccines. This type of innovative research is critical to moving forward the field of vaccines to address diarrheal disease, and support from the NIH and other partners is critical to this effort. As the dmLT and other promising vaccine technologies progress in their development, we know we are getting ever closer to expanding and improving the arsenal of tools to use in the fight against this major killer of children.