Breakthroughs Blog: Congress

Pyramax® Granules (Photo: MMV) After four successful Phase 3 clinical trials enrolling more than 3,500 patients in 18 countries, the European Medicines Agency (EMA) has granted Pyramax® Granules, a pediatric malaria medicine, a positive opinion under Article 58—a mechanism through which the EMA reviews and issues a scientific opinion on products that are not intended for use in Europe. Pyramax® Granules is a combination of two antimalarials and was developed by Medicines for Malaria Venture (MMV), a member of the GHTC, in partnership with Shin Poong Pharmaceutical Co. Ltd. The treatment is designed to be easy for children to consume: its bitter taste is masked and it is taken in liquid form, as children often struggle to swallow pills. Next, MMV and Shin Poong will work to introduce Pyramax® Granules as well as Pyramax®, the tablet version which is suitable for adults, in malaria-endemic countries. A recent article in NPR: Goats & Soda examines Zika virus, a disease transmitted by the same mosquito vector of dengue fever and chikungunya, that has been rapidly spreading throughout South America and the Pacific Islands in recent years. One in five individuals infected with the virus experience Zika fever, which in addition to fever involves rashes, joint pain, conjunctivitis, headaches, vomiting, and muscle pain. Cases of Zika fever are likely underdiagnosed, as awareness of the disease is limited and its symptoms are similar to infections associated with other members of the Flaviviridae virus family, including dengue fever, yellow fever, West Nile, and Japanese encephalitis. There are no reported deaths to date, but there is growing evidence that the virus can be transmitted sexually or from mother to infant during pregnancy. As there are no vaccines or drugs for Zika fever, treatment involves addressing the symptoms, using fever reducers and painkillers. A group of global health advocacy organizations—including GHTC members the Drugs for Neglected Diseases initiative, TB Alliance, Infectious Diseases Society of America Center for Global Health Policy, Sabin Vaccine Institute, and Treatment Action Group—sent an open letter to the US Senate Health, Education, Labor and Pensions (HELP) committee, calling for amendments to the priority review voucher (PRV) program at the US Food and Drug Administration (FDA) to ensure it adequately incentivizes research and development (R&D) for neglected diseases, as intended. Under the program, companies that receive FDA approval for a drug for a neglected disease (see list of eligible diseases) are awarded a voucher for expedited FDA review (six months rather than ten) of a future product. The vouchers are transferable and have been sold at increasing rates, the most recent going for US$350 million. The letter calls for PRVs to only be awarded for products that are "truly new" as two of the six vouchers that have been awarded were for products that had long been in use outside of the United States.[1] The organizations are also calling for recipients of the voucher to be required to develop a strategy to ensure the product is made affordable and accessible to patients in the countries in which the eligible disease is endemic. The United Kingdom (UK) announced last week the creation of the Ross Fund which will provide £1 billion ($1.5 billion) over five years to fight malaria and other infectious diseases. The funding, which will come from the UK overseas aid budget, includes £90 million for malaria eradication; £100 million for infectious disease R&D; £115 million for the development of drugs, diagnostic, and insecticides for malaria, tuberculosis, and other infectious disease resistance; and £188 million for biodefense and rapid response systems for epidemic preparedness. Global Good has developed a cooler that can store and transport vaccines at freezing temperatures for over a month using just ice and no electricity. The Arktek cooler uses vacuum thermal insulation, an approach used to preserve spacecraft entering extreme temperatures. The cooler has been tested in Senegal (with support from GHTC member PATH) and Ethiopia, and last year, the Arktek was modified for use in Ebola vaccine clinical trials, during which the cooler kept the vaccines between -76 and -112 degrees Fahrenheit for three days. [1] There are two separate PRV programs at the FDA, one for neglected tropical diseases and one for rare pediatric diseases. Of the six PRVs that have been awarded, three each have been for neglected tropical diseases and rare pediatric diseases. Since the beginning of the PRV program, six PRVs have been awarded—three for neglected tropical diseases and three for rare pediatric diseases.  

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. Now that members of Congress are back in their Capitol Hill offices after five weeks of recess, lawmakers must decide what to prioritize and what to push off in the few short weeks left in session before members hit the campaign trail for the upcoming November mid-term elections. In the latest post on Breakthroughs, GHTC Senior Policy and Advocacy Associate, Jenny Howell, provides a breakdown of what lawmakers should do to ensure global health research and development gets the support it needs. Federal funding in the United States for biomedical research has declined by more than 20 percent in the last decade. At the same time, more scientists are competing for a smaller pot of money. NPR highlights two stories of researchers forced out of science because they either can’t get research funding or “because the rat race has simply become too unpleasant.” Scientists at the National Institutes of Health (NIH) have discovered an HIV antibody that could be effective in fighting the deadly disease. According to the scientists, the antibody—called 35O22—can prevent 62 percent of known HIV strains from infecting cells. The NIH-led scientists also found that the antibody closely resembles antibodies that naturally occur in HIV, which could help in the development of an eventual vaccine. Results from a recent clinical trial could change the approach researchers take on the delivery and development of tuberculosis (TB) drugs in the future. While the REMoxTB trial did not successfully prove TB treatment times could be shortened from six to four months by substituting antibiotic moxiflacin for a currently used drug, it did prove that moxiflacin was safe to use to treat TB on its own. Also, because the trial was one of the largest ever for a new TB regimen, the trial left a stronger research infrastructure to conduct other trials in the future. The Bill & Melinda Gates Foundation has committed $50 million to fight Ebola in West Africa. According to Gates Foundation CEO Susan Desmond-Hellmann, the new money will be available for emergency operations and to help develop drugs, diagnostics, and vaccines to fight the disease. The Gates Foundation’s commitment is the largest sum to ever for one outbreak.

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. The Senate Appropriations Labor and Health and Human Services (LHHS) Subcommittee released its fiscal year 2015 appropriations legislation, after a lengthy internal debate on domestic health concerns. The legislation is—for the most part—good news for funding levels and policy issues for several key global health research programs at the National Institutes of Health, the Centers for Disease Control and Prevention (CDC), and the Office of Global Affairs at the Department of Health and Human Services. Since its discovery nearly 40 years ago, we have learned a lot about Ebola. Yet there are still no effective treatments or vaccines to protect people against this deadly disease. According to an article in Vox, this is not because of “a lack of human ingenuity or scientific capacity,” but rather due to a global innovation system that does not prioritize the health research needs of world’s poorest populations. Drs. S. Patrick Kachur, MD, MPH, FACPM—chief of the Malaria Branch in the Division of Parasitic Diseases and Malaria at CDC’s Center for Global Health—and Larry Slutsker, MD, MPH—director of the Division of Parasitic Diseases and Malaria at CDC’s Center for Global Health— write in Breakthroughs about the emerging global security threat of anti-malaria drug resistance and CDC’s efforts to prevent, detect, and respond to this growing threat. They write: “…to sustain momentum and move toward elimination of malaria, new tools and technologies will be required.” Despite progress made to prevent maternal and child deaths by the United Nations’ Millennium Development Goals (MDGs), still too many mothers and children are compromised or killed by preventable diseases. PATH President and CEO Steve Davis writes for Devex about the need to take advantage of the progress made thus far by the MDGs and accelerate innovation to meet the health needs of millions of people in low- and middle-income countries. It’s World Breastfeeding Week (August 1-7) and as breastfeeding has served as the primary method for a mother to feed her infant since the dawn of humanity, we wonder if this could be a way for a mother to administer life-saving medicines to her baby? According to a group of researchers in the United States and United Kingdom, the answer is yes. Two recent promising developments in the fight against malaria were announced last week. Malaria is one of the leading killers of women and children in the world, and in sub-Saharan Africa particularly—where 90 percent of the more than 620,000 annual malaria deaths occur—new tools to fight the disease are desperately needed.

Last week, we reported on exciting policy language included in the Senate Department of Defense appropriations legislation, and earlier this summer, the House and Senate State and Foreign Operations bills also included recognition of the role that new technologies could play in advancing global health. Today we have even more good news to report: The Senate Appropriations Labor and Health and Human Services (LHHS) Subcommittee released its own fiscal year (FY) 2015 appropriations legislation, after a lengthy internal debate on domestic health concerns. Senate supports global health R&D in LHHS appropriations bill. Photo: NIAID Senate calls for increase in NIH funding; still lags behind need The legislation covers funding and policy issues for several key global health research programs at the National Institutes of Health (NIH), the Centers for Disease Control and Prevention (CDC), and the Office of Global Affairs at the Department of Health and Human Services (HHS). At GHTC we’ve voiced concern that funding for NIH has lagged behind both inflation and the research program needs of the agency. Last week, we learned that the Senate LHHS appropriations subcommittee has appropriated $30.6 billion for NIH—which represents an increase of $606 million over FY 2014. This is a welcome increase; however, as the subcommittee noted in its report introduction, NIH’s funding is still unacceptably low, even with this improvement. Report language supports global health programming at NIH Within NIH, several key research centers were funded at reasonable levels, including the National Center for Advancing Translational Sciences (NCATS) and the National Institute for Allergy and Infectious Disease. The committee expressed its support for continued funding for neglected tropical diseases (NTDs), microbicides, and antibiotic research, as well as government-wide research collaboration, among other priorities. In an exciting move, the committee also included NCATS report language requesting that NCATS include a report in its FY 2016 budget justification “on initiatives targeted at neglected diseases and global health conditions, with specific focus on global health technologies and devices.” Global health is an essential part of NCATS’s mandate, and this language will ensure that it is prioritized within the program. CDC global health programming receives increased funding in bill The legislation also provides strong support for research at the CDC’s global health programs. Under the bill, the National Center for Emerging Zoonotic and Infectious Diseases (NCEZID) would receive an increase over FY 2014 funding levels. In addition, the subcommittee’s report mentions the Chikungunya outbreak in the Caribbean and the United States and encourages coordination between NCEZID and the CDC’s Center for Global Health on global health security efforts. Speaking of the Center for Global Health, this program received a rare increase in the Senate bill over even the President’s FY 2015 budget request. This is likely a result of support for the new Global Health Security Agenda. Most notable was the inclusion of a new line item in the Center’s budget for “Global Health Protection.” The bill also included positive language on CDC’s work on malaria and NTDs and provided continued funding for the National Public Health Institutes—an epidemiology and research capacity-building program in select countries. House LHHS bill has not been released; continuing resolution likely The House Appropriations LHHS Subcommittee has not released a bill for FY 2015 and may not do so at all. As it looks more likely that a continuing resolution will be necessary to continue uninterrupted government funding for NIH, HHS, and CDC global health R&D programs, Congress should work to come to an agreement that robustly supports funding and smart policy improvements for these programs. Ashley Bennett is GHTC’s policy officer.