Ashley Bennett

Combating preventable child deaths worldwide is an “investment that will pay dividends for years to come,” Deputy National Security Advisor Dennis McDonough said last week at a major conference at Georgetown University. At the conference, representatives from over 80 countries met to pledge their support for an exciting new global campaign for child survival. Hosted by the United States, UNICEF, and the governments of Ethiopia and India, the summit convened high-ranking government officials, members of the faith community, advocates, and private sector representatives. Over the last 50 years, child mortality rates have dropped by 70 percent. However, 7.6 million children under age five still die each year—far too many. In 2010, 57 out of every 1,000 children died before age five. The goal of the new program is to reduce that global rate to 15 deaths per 1,000 children by 2035. Under the program, UNICEF will house a small secretariat that will coordinate global activities to increase access to existing technologies and improve treatment guidelines, while partner countries like the United States will implement five strategic shifts to accelerate progress. As US agencies work with their partners to implement a new child health program, they must invest in R&D for new global health technologies. It’s critical that the program include a dedicated and distinct focus on global investments in research and development (R&D) for new tools to combat global child deaths, we still lack the vaccines, drugs, and other technologies needed to save children’s lives around the world. Indeed, research and innovation for new tools have been a huge factor in the success made against child mortality in the past 20 years, and must be prioritized by the new program. Several speakers at the summit praised the impact that technologies like the polio vaccine, oral rehydration solution to treat diarrhea, the MenAfriVac™ meningitis vaccine, and others have made in saving the lives of millions of children. Imagine if we had never invented a polio vaccine, or malaria drugs for children, or diagnostic tools for tuberculosis—what would global child mortality rates be today? A historic commitment to child health R&D helped create the tools that have already made a huge dent in global childhood deaths. For this reason, we should not only increase access to these existing technologies—the US Government and global partners must also continue to support R&D for the next generation of health products for children. If they don’t, the world will never reach the goals set by the new program. Fortunately, several US officials stressed the role of R&D in this effort last week. Secretary of State Hillary Clinton emphasized the commitment of the US Government to the development of new tools, saying, “We will support innovative research into new life-saving cost-effective measures.” Health and Human Services Secretary Kathleen Sebelius echoed the US role in basic research to support global health programs. Additionally, health officials from countries such as Bangladesh, Pakistan, and Nigeria highlighted the immense impact of global health technologies on their national child health programs. These achievements can be furthered by R&D-focused programs supported by the US Government, such as the second round of the Saving Lives at Birth program at the US Agency for International Development (USAID) and a joint USAID/National Institutes of Health initiative (NIH) that will provide grants to researchers in developing countries. As USAID and other agencies work with their partners to implement this important new program, they must invest in R&D for new global health technologies and plan for their delivery. USAID, NIH, and several other US agencies have a history of investment in innovation and are experts in catalyzing new research for huge impact. If we truly want to make the amazing progress we are promising to the next generation, the US must continue to expand its investment in global health R&D.

Recently, a US Food and Drug Administration (FDA) panel recommended that the HIV and AIDS drug Truvada be approved for HIV prevention among certain groups at risk of infection. Known as pre-exposure prophylaxis (PrEP), this prevention method involves HIV-negative individuals taking antiretroviral drugs to prevent HIV infection. This recommendation from the FDA panel does not oblige the agency to follow suit; however, it often does. The Truvada hearing is illustrative of the important role that the FDA plays in the research, review, regulation, and licensure of global health technologies—both those that are brand new, as well as reviewing new uses for current tools. Indeed, the FDA has a vast amount of expertise that it can leverage to help address regulatory issues in the developing world, where countries often lack the expertise or resources to appropriately review new health tools. In addition to the regulation and review of health products, the FDA also plays a small but important role in research and development (R&D) for global health. Starting in 2010, the FDA engaged in R&D funding for new drugs to combat tuberculosis (TB) though its work with the Critical Path Institute. The Critical Path to TB Drug Regimens brings together leading pharmaceutical companies and other drug developers, global regulatory agencies, and nongovernmental organizations—like GHTC member the Global Alliance for TB Drug Development—to facilitate the development and availability of new TB treatments. In addition to the regulation and review of health products, the FDA also plays a small but important role in research and development (R&D) for global health. Photo credit: PATH/Evelyn Hockstein FDA clearly recognizes the importance of its position in the global regulatory world, as evidenced by its recent Pathway to Global Product Safety and Quality report. It’s equally important that Congress recognize the FDA’s increasing role in global health, and that it works with the agency to provide the funding needed to sustain and expand these activities. This week, the House Agriculture, FDA, and Related Agencies Appropriations Subcommittee will debate its version of fiscal year (FY) 2013 appropriations legislation. And recently both the House and the Senate approved very similar versions of the Food and Drug Administration Safety and Innovation Act (FDASI), including reauthorizations of the Prescription Drug User Fee and Medical Device User Fee programs – which provide a large chunk of FDA’s operating expenses, and must be reauthorized by September 30th. Included in FDASI is the Generating Antibiotic Incentives Now (GAIN) Act, a provision which would extend data exclusivity for new antibiotics to combat drug resistant bacteria—including multi-drug resistant TB—by five years. While incentives that engage the private sector in product development are urgently needed, data exclusivity alone may not solve the gap in antibiotics R&D, and should be combined with several other incentive mechanisms in order to truly leverage the knowledge and resources of the industry. The legislation does request a study be conducted outlining the use and effectiveness of such incentives, a positive step in the right direction. The next few months are a critical time for all of FDA’s programs—and also an excellent opportunity for Congress to make smart reforms that enhance the best of what FDA does, including its work to support global health research and development. Ashley Bennett is the GHTC’s senior policy associate.

Late last week, the House Appropriations Committee debated and approved Fiscal Year (FY) 2013 appropriations legislation for the State and Foreign Operations (SFOPS) account, which includes funding levels for the State Department and the US Agency for International Development (USAID). The bill that the SFOPs subcommittee previously approved—which we discussed here—went largely unchanged in the full appropriations committee vote. As we covered last week, global health funding under USAID was restored to FY 2012 levels for most accounts. However, USAID’s family planning and reproductive health efforts were capped at 25 percent below the FY 2012 level, and the United Nations Population Fund (UNFPA) was completely de-funded. Three separate efforts during the appropriations committee markup on Thursday to restore some funds to UNFPA failed. These cuts have been criticized by many in the global health community and on the Hill as harmful, short-sighted, and life-threatening to women worldwide. During the hearing, there were no amendments related to global health research and development (R&D) programs, although language in the report that accompanies the House bill highlights the need for research into health technologies appropriate for low-resource settings. Additional language in the accompanying report promotes R&D for microbicides, and HIV and malaria vaccines, among other health tools. The Senate will weigh in with its own (and likely very different) version of this legislation tomorrow, May 22nd. We hope that the Senate also recognizes the importance of USAID’s R&D efforts, and that it restores the agency’s family planning and reproductive health work. Congressional support for all areas of USAID’s global health programming—from R&D to women’s health—is critical to improve the health and well-being of people around the world.

On Thursday, the House Appropriations Committee will vote on a State and Foreign Operations (SFOPS) appropriations bill for fiscal year (FY) 2013, which will include funding levels for global health and other programs at the State Department and US Agency for International Development (USAID). This vote follows last week’s markup of the bill by the House SFOPS appropriations subcommittee. Overall, the subcommittee’s bill had mixed results for global health and other programs at USAID: while some programs were sustained at FY 2012 levels, others saw significant budget cuts that will affect the agency’s efforts worldwide. Under the subcommittee’s bill, the entire State and Foreign Operations account was cut by nine percent. Many bipartisan programs like the President’s Emergency Program for AIDS Relief (PEPFAR) were flat-funded at FY 2012 levels. USAID’s global health funding, which contributes to global health research and development (R&D) projects, was cut by six percent, a decrease of roughly $150 million. USAID’s operating budget was also slashed by 5%, which will affect all of its programs. No funding levels were outlined for the many disease-specific programs at USAID. These disease-specific funding levels could be added to later versions of the bill, or could be left up to the Obama Administration once the entire appropriations process is completed. The only exception was family planning, which was slashed by one-quarter and given a new overall funding cap of $461 million—$149 million below the FY 2012 levels. It’s important to remember that the final SFOPS budget for FY 2013 will probably look different from the House bill being considered this week. The Senate is working on its own version of the appropriations bill, and both versions will eventually have to be negotiated if Congress follows a typical appropriations process (which it hasn’t done in the past few years). Whatever the final SFOPS budget looks like, it’s critical that Congress fully funds USAID so it can continue its work in all areas of global health, including the agency’s support of R&D to develop new health tools. And if it’s left to the Administration to make decisions about disease-specific program funding at USAID, funding for R&D and innovation for all disease areas needs to remain a top priority at the agency. USAID plays a huge role not just in funding product development to combat global health challenges, but also ensuring that new technologies are appropriate for the communities where they will be used. Some of the tools that have benefited from USAID funding and expertise include the RTS,S malaria vaccine candidate, the groundbreaking CAPRISA microbicide trials, and the Xpert TB diagnostic. USAID also works in partnership with many critical players in the global health R&D space, such as other US agencies, the private sector, nongovernmental organizations, and universities. It’s clear that USAID has seen major successes in its support for global health R&D. As the House Appropriations Committee votes on the SFOPS bill on Thursday and the budget process continues, Congress will have to decide whether it should boldly support USAID’s goal of developing new health tools—tools that are projected to save millions of lives—or withdraw this support and risk halting scientific advancement in its tracks.

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.

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.