Breakthroughs Blog: FDA

Interested in more global health innovation news? Every week GHTC scours media reports worldwide to deliver essential global health R&D news and content to your inbox. Sign-up now to receive our weekly R&D News Roundup email. The Coalition for Epidemic Preparedness Innovations (CEPI) is investing US$34 million into the German biotech company CureVac to develop vaccine “printing” technology that could rapidly manufacture vaccines to treat multiple diseases. Specifically, this funding will help CureVac work on a prototype of its RNA printer product, a transportable automated printing facility for molecules known as messenger RNA (mRNA)—used to transport genetic information from DNA into a cell, instructing it to produce a specific antigen to induce an immune response. As part of this three-year deal with CEPI, CureVac will use its mRNA platform to produce vaccines for Lassa fever, Rabies, and Yellow fever. If preclinical tests are successful, two of the vaccine candidates will be developed through early-stage safety trials in people. As epidemics of infectious diseases can be unpredictable and fast-moving, CureVac’s vaccine platform could help improve our ability to defend against such pathogens. Treating bednets with an antimalarial drug could be a promising new approach to controlling the disease. Although insecticide-treated bednets have been credited with averting 68 percent of all malaria cases since 2000, scientists believe mosquitoes are developing a resistance to pyrethroid, the insecticide predominantly used to treat bednets. Researchers at Harvard University demonstrated that an antimalarial drug can be transmitted to mosquitoes through brief direct contact and effectively halt the transmission of the malaria-causing parasite. In the study, researchers coated a petri dish with atovaquone, a commonly used antimalarial drug. Mosquitoes landed on the treated surface for approximately six minutes and were then fed a blood meal rife with the malaria parasite. Results showed that none of the mosquitoes treated with antimalarial drugs harbored the parasite and demonstrated proof of concept that antimalarial-treated bednets could be a powerful new tool in the fight against malaria. The US Food and Drug Administration has granted Fast Track designation to the Themis Bioscience chikungunya vaccine candidate, which allows the product to receive an expedited agency review given its potential to meet an unmet medical need. Themis hopes this designation will support its phase 3 development efforts for the vaccine, which showed encouraging results in phase 2. Two other chikungunya vaccine candidates have also received Fast Track designation, though the Themis vaccine is the most advanced in development. There are no current treatments or vaccines for chikungunya, a disease for which symptoms include debilitating joint pain.

On February 15, President Trump signed into law a bipartisan spending bill—averting another partial government shutdown and ensuring full-year funding for the government for the remainder of fiscal year (FY) 2019. The move followed several days of speculation about whether discord between Congress and the President on funding for a border wall would imperil the spending measure passed by Congress to fund the remaining one-quarter of the federal government.The months-long saga ended with some great news for global health and research and development (R&D)—a roughly US$150 million funding increase for global health programs in the State and Foreign Operations (SFOPs) bill, as well as a significant boost to the Food and Drug Administration (FDA) under the Agriculture bill included in the package.The bill included some welcome increases to global health accounts which have been flat-funded for five years running, including a $50 million (1.16 percent) increase to the President’s Emergency Plan for AIDS Relief (PEPFAR) and a $2.5 million (2.5 percent) increase to the US Agency for International Development’s (USAID) Neglected Tropical Disease Program compared to FY 2018. Nutrition saw a $20 million (16 percent) increase and Maternal and Child Health got a $5.5 million (0.66 percent) boost. But perhaps the most notable win in the bill was a long-sought increase to USAID’s tuberculosis (TB) program. After TB was in the spotlight at the United Nations High-Level Meeting on TB in September, the United States backed up its pledge of additional support for the global fight against TB by including a $41 million (15.71 percent) increase to the program in the final spending package for FY 2019—the largest increase to the program in more than five years. All other global health accounts were level-funded compared to FY 2018, resulting in an overall increase of 3.23 percent for USAID Global Health—a stark difference from the 36 percent cut proposed in the President’s budget.While most accounts of relevance to global health R&D within the Department of Health and Human Services were funded for the entire fiscal year in a spending package passed in September, the FDA was one agency whose funding was still in limbo. The FDA’s unique funding structure—divided between budget authority, or discretionary funding (appropriated by Congress), and fees (paid by FDA users) —shielded the agency from some of the worst impacts of the partial government shutdown in December and January as it drew down on user fees to continue most operations. But the final FY 2019 funding package included a welcome $460 million (9 percent) increase for the agency, including a $269 million (9 percent) increase in budget authority.As the FY 2019 appropriations process lurched across the finish line, advocates were already gearing up for another budget battle for FY 2020. Check back for regular updates as appropriations hearings begin and GHTC continues monitoring the prospects for global health R&D accounts in the coming year.  For reference, the text of the Conference Report to accompany H.J. Res. 31, the Consolidated Appropriations Act, 2019, is available here, the text of the Joint Explanatory Statement is here (with the Global Health Program breakdown on page 454 of the PDF), and a division-by-division summary is available here.

As the partial government shutdown stretches into a fifth week – the longest in United States history – news of its impact on agencies essential to global health broadly, and research and development in particular, is taking clearer shape. While less visible than long lines at Transportation Security Administration (TSA) airport check-points, the consequences of shuttering the US Agency for International Development and furloughing broad swaths of the State Department and Food and Drug Administration workforce for more than a month will only become more obvious as the dispute between Congress and the administration over funding for a border wall continues.Here’s what we know now, based on news reports, anecdotal evidence, and intel from agency contacts:Food and Drug AdministrationThe Food and Drug Administration (FDA) has managed to continue many activities in the midst of the shutdown by drawing down on prescription drug user fees, with reviews of submissions filed and paid for ahead of the partial shutdown on December 22, but drug reviews will stop completely as that funding dwindles in the coming weeks. A sign-on letter sent this week on behalf of “millions of American patients, caregivers, healthcare providers, and researchers” raised alarm that the shutdown “has begun to put future scientific discovery and innovation in jeopardy” by forcing the FDA to “make difficult choices regarding to which essential functions its greatly reduced resources are directed,” and urging that “the health and safety of Americans today should never be weighed against the prospect of new life-saving therapies for patients.”State Department/PEPFAROn January 17, the State Department announced it would be “taking steps to make additional funds available to pay employee salaries” to resume most personnel operations and fund most salaries beginning this week and continuing through at least February 14. The announcement from Bill Todd, Deputy Under Secretary for Management, specified that “although most personnel operations can resume, bureaus and posts are expected to adhere to strict budget constraints with regard to new spending for contracts, travel, and other needs.” For the remainder of this interim fix, it appears most of the President’s Emergency Plan for AIDS Relief (PEPFAR), funded through the State Department, should be operating as usual.  US Agency for International DevelopmentIn advance of the shutdown, the US Agency for International Development (USAID) released guidance noting that about half of its 3,100 person staff would be furloughed, with the remainder exempt and expected to continue working, including “those who are funded through mechanisms other than annual appropriations; employees whose jobs are considered necessary for the protection of life and property or necessary to carry out the president’s constitutional duties; or those who are authorized because of their connection to operations that will continue.” While this allows many contract employees across the agency to continue working, only a handful of full-time agency staff within the Global Health Bureau are reported to be in the office.The development and global health community is slowly beginning to feel and communicate the impact of USAID’s staffing and funding shortages. Last week, Devex detailed one example of a USAID funding commitment delayed by the shutdown, though most development leaders they contacted for comment suggested that the shutdown has had limited effect thus far as USAID-funded programs continue operating on prior year funds, but could soon slow funding approvals and dry up grant funds if protracted. This week, we began to hear anecdotes from implementing nongovernmental organization partners to that end, detailing agency guidance to slow spending and even cease activities on some USAID-funded projects.The Washington Post reported this week that White House Acting Chief of Staff Mick Mulvaney has asked agency leaders to provide him with a list of high-impact programs at risk if the shutdown continues into March and April, which increased concern that a compromise is not imminent. On Thursday, two bills with the potential to end the shutdown failed in the Senate, and no immediate path to resolve this impasse and reopen the government has emerged. Of note, a spending package released by House Democrats last week – based on compromise bills drafted last year by House and Senate negotiators – which is expected to shape final funding outcomes, increased global health programs by US$147 million over fiscal year 2018 enacted levels, including boosts to HIV and AIDS, nutrition, tuberculosis, maternal, newborn and child health, and neglected tropical diseases, as well as positive language about USAID’s role in product development.GHTC continues to track reports of how the shutdown may be affecting global health research and development (R&D) activities. Please email Emily Conron (econron@ghtcoalition.org) to share details of any impacts your organization or partners are experiencing as a result of agency closures.

Interested in more global health innovation news? Every week GHTC scours media reports worldwide to deliver essential global health R&D news and content to your inbox. Sign-up now to receive our weekly R&D News Roundup email.Scientists from the Fraunhofer Society have developed a first-of-its kind technology that uses electron beams to produce inactivated vaccines quickly and reproducibly without the use of chemicals. Inactivated vaccines contain pathogens that have been killed so they are no longer harmful to patients. Often these pathogens are killed using chemicals, such as diluted formaldehyde. This process is time consuming and can have a negative impact on both the structure of the pathogen and the reproducibility of the vaccine. The new technique uses electrons to fragment the DNA of the pathogens while maintaining their external structure, which is key to triggering an effective immune response. It will likely be another two to four years before vaccines produced using electron beams can be tested in clinical trials. The US Food and Drug Administration (FDA) has granted Fast Track designation to Valneva’s chikungunya vaccine candidate, which will allow the product to receive expedited review by the agency given it holds potential to fill an unmet medical need. The company is expected to release phase 1 data for the vaccine candidate in the coming weeks. Currently, there are no approved treatments or vaccines for chikungunya‒a disease that causes debilitating joint pain‒though there are several candidate vaccines in clinical testing.The Marburg virus, a deadly cousin of Ebola, has been isolated in fruit bats in Sierra Leone, marking the first time the virus has been found in West Africa. According to two teams of scientists involved in this discovery, five bats caught in three health districts tested positive for the virus. Genetic testing revealed multiple strains of the virus in each bat, which suggests that the virus has circulated in West Africa for many years. This testing was done as part of a project funded by the US Agency for International Development, known as PREDICT, which aims to detect dangerous pathogens in animals and take steps to prevent them from crossing over into humans and triggering disease outbreaks. PREDICT takes a “one health” approach by uniting the work of medical doctors, veterinarians, and occasionally plant biologists to detect threats in other species.