Breakthroughs Blog: FDA

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.

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.

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.