Breakthroughs Blog: NIH

In this feature on Breakthroughs, we provide an update on what’s currently happening with global health in Congress and an outlook for future legislative activity. Foremost on Congress’ plate this week was, of course, the appropriations process. Last week, House Budget Committee Chair Paul Ryan (R-Wisconsin) released his fiscal year (FY) 2013 budget resolution, which the House Budget Committee has now approved by a 19-18 vote. The full House passed the resolution yesterday 228-191, making the appropriations process even more complicated, as the legislation conflicts with much of the Budget Control Act (BCA) signed into law last year. In the resolution, international affairs funding in the approved resolution totals $49.1 billion. This is a reduction of $7.1 billion, or 12.6 percent, compared with President Obama’s FY 2013 requested funding levels. A report accompanying the resolution outlines some potential cuts that could help achieve this $7.1 billion cut. The section that covers the State and Foreign Operations account does not mention any global health-specific policy changes, so it is unclear at this stage how global health, as well as research and development for new health tools, would fare under Ryan’s resolution. Regardless of the House’s approval, the resolution will be dead on arrival in the Senate. House Democrats introduced their own budget alternative, authored by Maryland Democrat Chris Van Hollen. Their approach follows what President Obama requested fairly closely, but both this alternative as well as a more conservative resolution—supported by the Republican Study Committee—did not garner the votes needed to pass. Also this week, the National Institutes of Health (NIH) Director Francis Collins testified before the Senate Appropriations Committee. When he spoke at a similar House hearing recently, the new National Center for Advancing Translational Sciences (NCATS)—which Collins stated has the potential to support the development of new tools for rare and neglected diseases—was a hot topic, and the same was true with the Senate. Collins was questioned on plans for future projects at NCATS, and whether they would affect the NIH basic research budget. The effect of across-the-board federal budget cuts on the NIH was discussed during the Senate hearing, with Collins stating that there would be hundreds of great research projects that would not be funded if these widespread cuts take place. You can read testimony from Collins—as well as National Institute of Allergy and Infectious Diseases Director Anthony Fauci and other NIH leaders—here. Now that most of the hearings have been completed on the President’s budget request, appropriations committees in both the House and Senate will soon move to approve legislation for all accounts in the federal budget. It’s likely that the House subcommittee will start to approve funding legislation in May, with the Senate following suit shortly after. The process is moving along normally—for now. With growing anxiety about whether Congress will alter the required across-the-board budget cuts, and the Presidential election drawing closer, we may still see a replay of previous years’ deadlock.

Rachel M. Cohen, MPP, is the regional executive director of the Drugs for Neglected Diseases initiative (DNDi) North America. One billion people. That’s roughly how many people live in North America and Europe combined. And that’s also roughly how many people have at least one neglected tropical disease (NTD). NTDs, as defined by the World Health Organization, are a set of 17 infectious diseases that severely debilitate or kill people, most of whom are poor, marginalized, or in other ways vulnerable. NTDs such as sleeping sickness and kala azar are 100 percent fatal if left untreated. Parasitic-worm diseases like river blindness and elephantiasis can blind and deform people, young and old. Chagas disease almost exclusively infects those in Latin America, but kills more people in this region than malaria. The United States Government has played a major role in ensuring that patients with certain NTDs receive urgently needed treatments through the US Agency for International Development (USAID) NTD Program, while simultaneously being the largest funder of basic research for NTDs through the National Institutes of Health. This commitment has spanned several presidential administrations, receiving widespread support from both Democrats and Republicans alike in Congress. However, today US Government funding for NTDs is under threat. The recently announced US fiscal year (FY) 2013 budget request from the Obama Administration has slashed the USAID NTD Program budget, which was already miniscule at $89 million, by nearly 25 percent to $67 million. That is one of the single largest percentage cuts in global health. This isn’t trimming the fat—it’s cutting into muscle. While the USAID NTD Program has made an enormous impact for millions of people, the current initiative only focuses on five of the 17 NTDs. It does not fund diagnosis or treatment of the NTDs with the highest death rates —sleeping sickness, Chagas disease, and visceral leishmaniasis—and it does not fund any research into new health tools, unlike every other disease-specific program at USAID. USAID must urgently invest in research and late-stage product development for NTDs in order to bring new, life-saving health technologies to patients. Photo: PATH/Evelyn Hockstein. If the requested budget cuts go through, existing programs will have to be cut, and there will be no room for urgently needed program expansions. This will have direct consequences in terms of lives lost. The NTD Program budget line needs to be increased to President Obama’s FY 2012 request of $100 million. USAID must also urgently invest in a broader scope of diseases, including the aforementioned deadly NTDs, and in late-stage product development for NTDs in order to bring new, life-saving health technologies to patients. But why are new drugs needed? Can we not make do with the drugs already available to treat NTDs? In short, no. Here is just one reason. Today, filarial parasitic-worm diseases (onchocerciasis, also known as river blindness, and lymphatic filariasis, also known as elephantiasis) are treated with the drug ivermectin. But in people co-infected with another filarial disease, African eye worm (Loa loa, or loaisis), this treatment, alone or in combination with another called albendazole, can lead to brain damage—or even death. More than 14 million people are at high risk. For these patients, a new treatment is needed. My organization, the not-for-profit research and development organization Drugs for Neglected Diseases initiative (DNDi), is developing a drug called flubendazole that, if successful, would be safe and effective in these patients. Without such a drug, control and elimination of river blindness and elephantiasis will not be possible. USAID has publicly committed to contributing to the global goals of elimination and control of these and other NTDs, both through the US Global Health Initiative targets and, most recently, by signing on to the “London Declaration on NTDs” on January 30 at the high-level event “Uniting to Combat NTDs.” But rather than sustaining or expanding its commitment to NTDs, USAID appears to be retreating. For nearly all other communicable diseases, USAID funds late-stage product development (phase IIb or later), to bring needed innovations through the pipeline and, ultimately, to patients. This is the case for malaria, tuberculosis, and HIV and AIDS, for example. But not for NTDs. USAID must without delay begin investing into late-stage product development for NTDs. This approach would align NTDs with other disease-specific USAID programs that currently allocate a percentage of their funding for late-stage product development, and would be entirely consistent with USAID’s vision of promoting science and technology to advance international development. In order to bridge the gap between innovation and equitable access to the benefits of scientific research, late-stage product development is crucial. USAID must act now to prioritize the needs of the world’s most neglected patients who urgently need new drugs, diagnostics, and vaccines. Without new tools, these patients will fall through the cracks and die in silence.

As people across the globe celebrate World TB Day this week, several groups are highlighting the fact that the current tools to prevent, test, and treat tuberculosis (TB) are greatly outdated. The world has been “beyond antiquated in our approach” to developing new TB tools. That is how Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases (NIAID) at the National Institutes of Health, described a not-so-bygone era in the history of TB research and development (R&D). Fauci was speaking at a briefing organized by the Critical Path to TB Drug Regimensin partnership with the Global Alliance for TB Drug Development (TB Alliance), US Agency for International Development (USAID), NIAID, and the US Food and Drug Administration (FDA). During the event, representatives from key US government agencies made the case for the urgent need for new technologies in the TB toolkit. “It can’t take 40 years for us to get our next TB drug,” Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research stated, highlighting one of the lesser known facts about TB—that the newest drug was approved over 40 years ago. “We need to capitalize on the momentum we have,” she added. Many current TB drugs are toxic and have serious interactions with other medicines, and most regimens take over six months to complete. The disease kills 1.4 million people and infects 8.8 million each year. As people across the globe celebrate World TB Day this week, several groups are highlighting the fact that the current tools to prevent, test, and treat TB are greatly outdated. Photo credit: PATH Joining Fauci and Woodcock were Robert Clay, deputy assistant administrator for Global Health at USAID; Mel Spigelman, CEO of the TB Alliance; and Christine Lubinski, vice-president for Global Health at the Infectious Diseases Society of America (IDSA). All speakers stressed the urgent need to conduct R&D for new and improved TB prevention and treatment options. They also lauded the use of partnerships between non-profit organizations, private pharmaceutical companies, and US Government agencies in the development of new drugs and other technologies. Fortunately, the TB Alliance, a GHTC member and product development partnership (PDP), announced the launch of a new combination drug trial that could pave the way for the next generation in TB treatment approaches. The group has launched stage II trials for NC-002—a novel drug regimen combining a well-known generic TB drug with two new drug candidates. The trials will take place in South Africa, Tanzania, and Brazil, and results will be known in roughly a year. This trial is also the very first of its kind. It will test both patients with ‘drug sensitive’ TB, who do respond to current treatments, along with patients with multi-drug resistant TB (MDR-TB), who do not respond well to currently available treatment, to determine differences in the groups’ response to the new drug regimen. As Spigelman outlined, the potential impact of this trial is immense—it could reduce the costs of treating MDR-TB by 90 percent, as well as decrease treatment time by over 80 percent. This exciting news in TB drug development follows closely on the heels of new funding for new prevention tools. Last week, GHTC member Aeras announced that it received a grantfrom the Bill & Melinda Gates Foundation of up to $220 million over five years to conduct R&D for new TB vaccines. Aeras stressed that it is developing new TB vaccines against the backdrop of a significant increase in drug-resistant strains of the disease worldwide. Additionally, today Aeras—along with the TuBerculosis Vaccine Initiative and the STOP TB Partnership—also announced a new blueprint for TB vaccine development. "To develop a new TB vaccine that will be fully effective, researchers, donors, and other partners will need to collaborate and coordinate their efforts as they address tough research questions," said Dr. Lucica Ditiu, executive secretary of the Stop TB Partnership. "We cannot allow ourselves to be overwhelmed by either the costs or the obstacles. It is time to be bold and dare to do more in TB, especially in supporting the development of a new vaccine." As we recognize World TB Day, the need for new and better prevention, diagnostic, and treatment options for the disease is clearer than ever before. And it’s encouraging that GHTC members like Aeras and the TB Alliance—along with many other partners—are at the forefront of R&D efforts to create these desperately needed health tools.

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. As the international community marks World TB Day, GHTC members Aeras and the Global Alliance for TB Drug Development announced research projects for new tuberculosis (TB) prevention and treatment tools. National Institutes of Health (NIH) Director Francis Collins testified at a Congressional hearing on the agency’s budget. Collins emphasized NIH’s commitment to fund basic biomedical research as the agency’s highest priority. Rajiv Shah, head of the US Agency for International Development, was also on Capitol Hill this week to discuss the agency’s budget with members of Congress. "While foreign assistance represents less than one percent of our budget, we are committed to improving our efficiency and maximizing the value of every dollar," Shah said at the hearing. Alberto Gutierrez—director of the Office of In Vitro Diagnostic Device Evaluation and Safety at the US Food and Drug Administration’s (FDA) Center for Devices and Radiological Health—writes about how the FDA is helping to improve access to TB diagnostic tools.