Breakthroughs Blog: FDA

  As 2014 gets into full swing, the GHTC is looking ahead to many exciting developments on both the global health research and development (R&D) and policy landscapes: A step in the right direction: Policymakers in Congress recently passed both a budget deal for fiscal year (FY) 2014 and FY 2015, as well as a final appropriations package for FY 2014. The package included mixed results for global health research, however the compromise may hallmark a step towards a more “normal” appropriations process for FY 2015. We’ll be watching to see if policymakers boost support for the accounts—like those at the National Institutes of Health—that were hammered by the shutdown and budget sequester. Progress towards new malaria tools: In late 2013, GHTC member Medicines for Malaria Ventures received expedited regulatory approval from the US Food and Drug Administration to move treatment candidate tafenoquine into Phase III trials. And, the most recent clinical trial results of the RTS,S malaria vaccine candidate, released last year by partners PATH Malaria Vaccine Initiative and GlaxoSmithKline (GSK), have spurred excitement that the vaccine may be WHO-approved by 2015. To start the process, GSK announced plans to submit a regulatory application through the European Medicines Agency later this year. 21st Century Global Health Technology Act: Now that Congress has finalized its FY 2014 budget and moved on to other priorities, at the top of our list is the 21st Century Global Health Technology Act. The legislation was introduced in April of 2013 in the House of Representatives by Reps. Albio Sires (D-NJ) and Mario Diaz-Balart (R-FL) and boosts global health R&D programming at the US Agency for International Development. Rumors are a companion Senate version is coming very soon, so stay tuned! Exciting new research is coming up in 2014. Photo: PATH/Evelyn Hockstein R&D takes the stage at WHO: The World Health Organization (WHO) has made some progress in implementing the health R&D resolution it passed in 2013—including reviewing proposals for demonstration projects aimed at promoting R&D for medical products. The World Health Assembly will have eight finalists to choose from at its meeting this May. Horizon 2020 begins: In late 2013, the European Union approved €80 billion in funding for Horizon 2020—the European Parliament’s framework for research and innovation. This represents an increase in funding compared with previous years, and includes an emphasis on public-private partnerships for research. It will be interesting to see how this program affects the global health R&D landscape. State of the Union: This time last year, we were anticipating the start of President Obama’s 2nd term, and what shifts in agency and congressional leadership would mean for US global health research programs. The 2012 and 2013 State of the Union addresses emphasized harnessing innovation to make progress in health, education, and other areas. We’ll be watching this Tuesday night to see how the President plans to build on progress made in the remainder of his term. A new era for the President’s Emergency Plan for AIDS Relief: In other Administration news, Dr. Deborah Birx was recently nominated to be the new US Global AIDS Coordinator. Dr. Birx currently heads the Global HIV and AIDS program at the Centers for Disease Control and Prevention (CDC), and previously ran the Military HIV Research Program (MHRP) at the Department of Defense. We’re excited to see a leader in global health research take the reins of one of the US government’s most important global health programs. Pending her confirmation, it will be intriguing to see where Dr. Birx takes the agency, and how research for new global health tools will play a role. All health is global: Speaking of the CDC, two of the five most pressing health priorities that CDC’s Director Dr. Tom Frieden identified in a recent interview involve big impacts in global health research: eradicating polio and combating disease that originates outside of the United States. Between new research advancements and policy progress, there is clearly a lot we’re excited about for 2014. What new developments in global health R&D are you looking forward to? Ashley Bennett is GHTC's policy officer.

UPDATE: since this post, the Senate also approved the spending package and President Obama has signed the final 2014 omnibus bill into law. In an increasingly rare show of bipartisanship, Congress has moved forward an appropriations agreement that should finally settle the issue of fiscal year (FY) 2014 funding levels. Yesterday, the House of Representatives approved the $1.1 trillion Omnibus spending bill, and the Senate is likely to pass the legislation later this week. In line with budget numbers previously agreed to, the bill includes an increase in discretionary spending of $45 billion, split equally between defense and non-defense spending. For global health research, the news is mixed. Funding for many accounts that fund research for new global health tools—such as those at the US Agency for International Development (USAID)—was higher than FY 2013 sequestered levels, and a few accounts even saw further increases. However, the National Institutes of Health (NIH) remains woefully underfunded, even as Congress increased its budget by $1 billion over last year’s post-sequestration levels. Below are a few key highlights from the omnibus agreement: State and Foreign Operations (SFOPS)—Although overall International Affairs funding saw a cut of $6.5 billion (39.7 percent) from last year’s levels, funding for global health actually increased by 4.7 percent over FY 2013 levels to $8.44 billion. Certain subaccounts that contain global health research programs within the SFOPs budget fared particularly well, including funding for the Maternal and Child Health account ($705 million) which saw a 12.4 percent increase from 2013 levels. Additionally, appropriators went above and beyond the President’s request for tuberculosis (TB) funding, allocating $236 million for bilateral TB programs, $45 million over the President’s request and an increase of 5.1 percent from 2013 enacted levels. In another positive move, the USAID Neglected Tropical Diseases program is funded at $100 million—potentially opening the door for the inclusion of research and development (R&D) programming. The bill also provides for $4.067 in funding for the President’s Emergency Plan for AIDS Relief program—which includes key product development programs for malaria, HIV and AIDS, and TB—an overall increase of 3.9 percent. Funding for both family planning and malaria remained similar to last year’s levels. Policy language that accompanied the omnibus also contained support for the continuation of important global health research programming. NIH—The omnibus sets overall 2014 funding for the NIH at $29.934 billion—a $1 billion increase from 2013 post-sequester levels. While this is certainly an improvement from the current situation, this number falls short of White House and Senate requests and represents a $950 million cut from 2012 levels. In more reassuring news, the NIH National Center for Advancing Translational Science (NCATS) was funded at a fairly robust $633 million, however report language included in the omnibus text requested that all funding for the Clinical and Translational Science Awards come out of NCATS funding in FY 2014, whereas in the past funding has been drawn from several centers. Other important centers such as the National Institute of Allergy and Infectious Diseases and the National Institute of Child Health were funded at $4.359 billion and $1.283 billion, respectively—both decreases from FY 2012 levels and the FY 2014 budget request. Centers for Disease Control and Prevention (CDC)—Overall funding for the CDCis set at $6.9 billion, a $567 million increase from 2013 post-sequestration levels. Within the CDC, the National Center for Emerging and Zoonotic Infectious Diseases was funded at $287.3 million, $93 million below the President’s request. CDC’s Global Health account, which includes funding for HIV and AIDS was funding at $383 million, slightly below the President’s request, but a $24 million increase from 2013 post-sequestration levels. In an intriguing move, language included in the CDC section calls for increase coordination of research programming between CDC, the US Food and Drug Administration (FDA), and NIH. You can read the full report here. FDA—Out of the major US agencies involved in global health research, FDA fared the best in the omnibus. FDA is funded at a total of $2.552 billion in discretionary spending, an increase of $91 million from 2013 post-sequestration levels. Notably the omnibus allows FDA to get back $85 million in user fee money that was withheld as a result of the sequester. Department of Defense (DoD)—While we’re still poring through the DoD’s appropriation package, early analysis reports that medical R&D funding saw increases, even as DoD’s overall budget was decreased. This may be a positive sign; as AAAS reports, research programs here may help lessen the impact of dire cuts at NIH. While Congress should be applauded for protecting many of the programs that fund global health research in the omnibus package, the recent downward trend in NIH funding is troubling. NIH is the world’s leading research institution, and has funded the groundbreaking work behind the majority of global health tools available today. The role of NIH in the global health product development cannot be replaced or replicated elsewhere; if support continues to decline, urgently needed innovations will not reach those who need them. Congress and the Administration must continue to push global health progress forward by supporting robust funding for all US agencies that fund global health R&D during the FY 2015 budget and appropriations process. Additional analyses: Kaiser Family Foundation AAAS InterAction budget table

In the past, the United States has contributed to important breakthroughs in global health—from the eradication of smallpox to a 74 percent drop in measles deaths worldwide. Because of longstanding US leadership and support for global health research and development (R&D), Americans and millions around the world no longer live in fear of diseases like polio and measles, and many more are on treatment for diseases like HIV and AIDS and malaria. In fact, the US government is the largest supporter of global health R&D in the world, supporting the development and delivery of vaccines, drugs, diagnostics, microbicides, and devices. However, as we sit at a very important moment in global health product development, the political and economic environment in the United States is causing some to worry. Budget constraints have put funding at risk at many of the federal agencies involvement in global health R&D. Because of longstanding US leadership and support for global health research and development (R&D), Americans and millions around the world no longer live in fear of diseases like polio and measles, and many more are on treatment for diseases like HIV and AIDS and malaria. Photo: PATH/Evelyn Hockstein Today, the GHTC launches a new fact sheet outlining the important contributions the US government has made to global health R&D and why it’s critical the United States continues to robustly support the global health research pipeline. There are currently more global health products in the pipeline than ever before—with more than 365 new tools under development as of April 2012. Contributing to the success of many of these products is the US government’s partnerships with nonprofit product developers. These organizations partner with the public and private sectors to maximize resources and create tools for patients in low- and middle-income countries. Increased investments in global health research by the United States have the potential to increase the amount of tools combating global diseases. Just some of the products currently in the research pipeline include: The RTS,S malaria vaccine candidate, which is currently being tested and a policy recommendation for its use could be issued as early as 2015. Such a vaccine could reduce the burden of sickness and death from malaria. A new oral drug for sleeping sickness, fexinidazole, which recently entered late-stage clinical trials in patients in the Democratic Republic of the Congo. As a simple, short-course oral pill, fexinidazole could transform care for African sleeping sickness, which is fatal without treatment, and could potentially reduce its incidence among the most afflicted populations and accelerate elimination of the disease. Cost-effective diagnostics that are under development for malaria, tuberculosis, and neglected tropical diseases. These diagnostics could be administered in a variety of health care settings and could rapidly and accurately identify sick patients. An antiretroviral‐based microbicide which offers hope for women’s HIV prevention. Though more testing is needed following promising results from a 2010 advanced clinical trial, a microbicide could give women the power to protect themselves with or without their partners’ cooperation. In sub-Saharan Africa, where the epidemic has hit hardest, women 15 to 24 years old are at least twice as likely to be infected with HIV as young men.   In the research pipeline for global health products, 200 of the 365 products under development have some sort of US government support. With so much investment from the United States, there is even more at risk if funding is cut. Over the past several years, the US Congress has not been able to agree on a long-term budget for the federal government. Policymakers have allowed sequestration, or widespread automatic budget cuts, to affect all federal programs. These constraints have the potential to push back or halt promising research supported by several federal agencies involved in global health R&D—including the Centers for Disease Control and Prevention, the Department of Defense (DoD), the US Food and Drug Administration, the National Institutes of Health (NIH), and the US Agency for International Development (USAID). Due to sequestration: DoD is expected to receive budget cuts between 8 and 10 percent in fiscal year (FY) 2013. The DoD has been instrumental in the development of several global health technologies, many of which are used to protect Americans soldiers serving abroad. These cuts could affect the Walter Reed Army Institute of Research, the Naval Medical Research Center, and the Military HIV Research Program. NIH, the largest funder of global health R&D in the world, was forced to cut $1.7 billion from its budget by the end of FY 2013. NIH conducts nearly half the early-stage global health research, and supports thousands of domestic jobs all around the United States. USAID—with a presence in more than 100 countries—is best suited to support the clinical trials in low- and middle-income countries that ensure basic research is translated into appropriate health products. USAID is the largest supporter of HIV vaccine and microbicide development—both of which will be essential to achieve an AIDS-free generation. For many of the products currently in the global health research pipeline, continuous US support is essential. Without it, much of the breakthrough research that pushed these products to later-stage development will be lost—sometimes forever. US leaders should seize upon the recent successes in global health R&D and pass a long-term budget that ensures America’s legacy as a leader in innovation and science is not forgotten.

The GHTC has long stressed that the US Food and Drug Administration (FDA) plays a critical part in efforts to improve global public health. While the FDA is best known for its work to protect the health of US consumers, the agency has a strong history of sharing its expertise to benefit people worldwide and is playing an increasingly vital role in global health issues. The GHTC has released two new resources that provide fresh evidence on the FDA’s growing global health presence, while also offering recommendations for how the agency can continue to strengthen its efforts to improve health and save lives worldwide. Our new fact sheet and policy brief focus on five key areas at the FDA. First, the FDA leads numerous international programs from its headquarters in the United States and operates offices and posts in ten countries across the globe. As the agency expands and deepens its contributions to global health, it is essential to ensure FDA’s activities contribute to an agency-wide strategy. Therefore, the FDA should create an office of neglected diseases so that it can better drive coordination among its centers and offices to maximize its global—and domestic—health impacts. This new program should be housed within the FDA’s Office of the Commissioner. By formalizing the agency’s global health activities, better oversight can help ensure that related programs and initiatives contribute to a unified goal. The GHTC has released two new resources that provide fresh evidence on the FDA’s growing global health presence, while also offering recommendations for how the agency can continue to strengthen its efforts to improve health and save lives worldwide. Credit: PATH Second, the FDA should continue to build partnerships with global regulatory stakeholders, such as the World Health Organization and national regulatory authorities. Regulatory review of products designed for use in the developing world can be particularly challenging, especially when the national regulatory authorities do not have the capacity or resources to manage the reviews. To address this issue, the FDA offers trainings and contributes to global efforts to improve regulatory harmonization. The agency also supports a variety of global regulatory initiatives. But this is just the beginning. To realize the positive impacts of these investments, FDA should continue to contribute to and expand these efforts. Third, while FDA has taken impressive steps to increase its engagement globally, without formalized reporting and oversight mechanisms the outcomes of this work can wane in the face of other priorities and funding constraints. To increase transparency and accountability for its global health commitments, the FDA should take steps to establish formalized processes to report to Congress on its neglected disease activities. Fourth, while its first priority is the health of Americans, it is essential that the FDA arms itself with global health experts who can help carry out a comprehensive plan. To strengthen the work already being conducted in the agency’s international offices and posts, FDA should dedicate additional staff members and resources to global health engagement. Finally, FDA—like so many agencies across the US government—is facing severe financial constrains due to sequestration and broader budget cuts. We’re facing the possibility of a government shutdown at the end of this month, as congressional policymakers still have not reached an agreement on the federal budget. Without question, this financial uncertainty hinders FDA’s ability to strengthen its role in global health and safeguard the health of American citizens. It’s critical that congressional policymakers pass a smart budget agreement that avoids another round of sequestration cuts while protecting key programs at FDA and other federal agencies that work in global health. As House and Senate leaders continue their budget negotiations, they have the opportunity to demonstrate their commitment to lifesaving and efficient programs at leading agencies like the FDA.