Breakthroughs Blog: NIH

Late last week, the National Center for Advancing Translational Science (NCATS) at the National Institutes of Health (NIH) unveiled a pilot program that aims to speed the creation of new drugs. Under the program, three pharmaceutical companies have agreed to make dozens of their failed compounds available to researchers, who will investigate if the compounds can be re-purposed into successful treatments for other diseases. The HIV and AIDS drug AZT, which was born out of an unsuccessful cancer treatment, has been cited as a prime example of the potential for this new program. At the launch event, NIH Director Francis Collins highlighted that the NIH needs to “speed the pace at which we are turning discoveries into better health outcomes.” He also emphasized the urgent need for new cures for patients suffering without any available treatment options. With NCATS, an exciting potential exists for new tools to combat neglected global health diseases. Photo credit: PATH The new program embodies the public-private partnership model. In 2013, $20 million in US Government grants from the NIH will fund researchers to work with compounds provided by private-sector pharmaceutical companies in pursuit of new treatments. As of now, over 20 compounds have been committed by the three companies. Kathy Hudson, acting deputy director of NCATS, hopes that many more will be available by the time the program releases its first Request for Applications (RFA) next month. No information is currently available from NCATS on what compounds will be available, or what diseases the program hopes to tackle. We won’t have that information until the RFA is public. In setting his goals for NIH at the beginning of his tenure, Collins stated that global health would be a top priority for NIH as an organization. Now that NCATS—which focuses on spurring drug development for neglected diseases of the developing world, as well as rare diseases—is established and this new program has been launched, an exciting potential exists for new tools to combat neglected global health diseases. Since its inception, NCATS has unfortunately been slow to take up initiatives that target global health diseases, as many of the NCATS programs announced thus far focus primarily on rare diseases that affect US populations. Rare diseases absolutely deserve funding, support, and targeted research—as do global health diseases. As shown in our recently released report, the United States leads the world in global health research and development. Under this leadership, who knows what groundbreaking cures for global health diseases could be created with the compounds included in the new program? At the press conference, patient advocate Michael Manganiello spoke enthusiastically about his experience as a patient saved by AZT. “Without partnerships between the NIH and industry, I would not be here to give this statement today,” he said. HIV and AIDS is just one example of the range of global health diseases that remain in urgent need of new tools. Think of how far we have advanced from a time when there were no treatments for HIV and AIDS, and think of what progress could be made in other neglected diseases under this new program. With this new therapeutics program, NIH and NCATS have created an exciting, innovative model for collaboration between the public and private sectors. They should be applauded for it. Now NCATS must ensure that research for neglected diseases is encouraged and supported through this initiative. NCATS has the opportunity to jump start innovation that will one day save lives and create better health outcomes—an opportunity that should be shared with patients around the world.

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.