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This post originally appeared on the Center for Global Health R&D Policy Assessment blog. Depending on who you ask, you’ll come up with a number of different definitions for what exactly characterizes a neglected disease, as there’s no generally accepted definition. I personally like the one offered by the NIH: “Global health organizations define neglected diseases as those that are prevalent among impoverished and marginalized populations in the developing world. Tropical environments, poor living conditions, and limited access to health care contribute to the risk and spread of these diseases.” But even this definition leaves something lacking. There are a number of conditions that cause severe morbidity or mortality in low and middle-income countries and still go unrecognized when we talk about neglected diseases. For instance, we at the Global Health Technologies Coalition (GHTC) tend to think of “neglected” in terms of the urgent need for tools to prevent, diagnose, treat, and generally improve the health of people in low- and middle-income countries around the world. If increased investments from governments and other stakeholders in research and development (R&D) are needed to create new and improved products for a certain disease or health need in the developing world, then we would define that health issue as neglected. As an example, reproductive health issues and sexually transmitted infections can have devastating health effects for women on par with infectious diseases like tuberculosis, malaria, and neglected tropical diseases. Although pregnancy is not often thought of as a neglected condition, it could be, as there is an incredible demand for contraceptive options that don’t yet exist. Additionally, issues like non-communicable diseases are not traditionally neglected when considering the amount of funding and other investments pharmaceutical companies devote to developing products for conditions like diabetes. However, the distinct technological and infrastructure needs of health systems in the developing world, as well as the alarming climb in the prevalence of non-communicable diseases in these settings, have made them a much more urgent concern. But why should a definition matter anyway? For those of us thinking about global health policy, terms and nomenclature are important. Consider, for instance, one of the many programs implemented by the US government to improve public health around the world. The US Food and Drug Administration’s (FDA) priority review voucher program lists specific diseases that are eligible for the agency’s incentive program. This list leaves out a whole host of neglected diseases. Among them is Chagas disease, which Congress has been attempting to include via new legislation for the past two years. This disease remains ineligible for the incentive according to the legislation and guidelines governing the program. So we need to adapt the way we think about neglected health needs worldwide to reflect conditions—like pregnancy and reproductive health needs—that impact people worldwide, in addition to infectious diseases. Perhaps instead of framing our thinking around neglected diseases, we should instead consider a different term: neglected conditions. By changing our thinking on neglected conditions, we open the door to increased attention, support, and programming to address all of these pressing health needs as a package—from infectious disease to childhood health needs like nutrition, reproductive health issues, and pregnancy-related complications. For instance, new and improved contraceptive and microbicide products, including multipurpose prevention technologies under development, could be an easy, cost-effective way to help women plan their pregnancies, address reproductive health needs, and prevent HIV and other STIs. We also may be able to leverage funding and government policies to give greater attention to the important conditions and other contributing factors to illness that don’t fit precisely into current definitions of neglected disease. Programs like PEPFAR have evolved from a narrow focus on urgent needs related to HIV and AIDS to one that considers the impact of other diseases and conditions, like malaria, TB, nutrition, family planning, and others. This evolution has created important successes for the program, and has pushed programs and policymakers to think outside typical disease boundaries. The US government is the largest supporter of research and other programs for neglected conditions worldwide and, as demonstrated by the FDA’s priority review program above, implements a number of programs that can have important impact on the development of critically-needed new tools. Given that the US government has long been a leader in supporting R&D for a range of global health diseases and conditions, leadership cannot abandon this legacy now. There are significant gaps in the development of new tools to address neglected conditions, and there is a critical unmet need for tools that address reproductive health, non-communicable disease in low-resource settings, and other health issues. By changing our definition of neglected diseases to include neglected conditions, the US government and other governments, donors, and advocates can make an even greater impact on health worldwide.

Ahead of World AIDS Day, Secretary of State Hillary Rodham Clinton launched a new State Department blueprint to reach an “AIDS-free generation” within five years. “An AIDS-free generation will be within our sight,” Clinton said as she described the blueprint, which outlines key strategies the US will undertake to fight the HIV and AIDS pandemic. She also warned: “Now we have to deliver. … The history of global health is littered with grand plans that never panned out.” According to Clinton, an AIDS-free generation means that the world “can reach a point where virtually no children are born with the virus, and as these children become teenagers and adults, they are at a far lower risk of becoming infected than they are today. And if they do acquire HIV, they have access to treatment that helps prevent them from not only from developing AIDS, but from and passing the virus on to others.” The blueprint outlines several key actions to reach this goal, including: Scaling up the most effective prevention and treatment interventions. Targeting the populations at the greatest risk of contracting HIV, including people who inject drugs, sex workers, those trafficked into prostitution, and men who have sex with men. This also includes ensuring that HIV and AIDS programs recognize the particular needs of women and girls. Promoting sustainability, efficiency, and effectiveness. Promoting a global effort to achieve an AIDS-free generation. There have been several notable breakthroughs in HIV and AIDS science and research in recent years, and it’s critical that the US government prioritizes the development of new tools in any HIV and AIDS strategies and goals it creates. Photo credit: PATH. According to Clinton, science and research are key principles that must underlie each aspect of the AIDS-free generation blueprint. This includes US support for research to develop the next generation of “innovative technologies for prevention and treatment, such as microbicides and approaches that stave off opportunistic infections” like tuberculosis. It’s commendable that the US government is continuing its legacy as a leader in the fight against HIV and AIDS, as this blueprint indicates. It’s also encouraging that the blueprint focuses on developing new scientific breakthroughs in the fight against the disease. As Mitchell Warren from AVAC said in response to Clinton’s speech, “Powerful new HIV prevention options could together lead to dramatic reductions in HIV infections, but we don’t have all the information we need to scale them up in the right combinations for various communities. Urgent questions about the real-world use of new prevention tools in combination have been clear for months or even years, yet the work to answer them is barely under way. That’s as unconscionable as it is unnecessary.” There have been several notable breakthroughs in HIV and AIDS science and research in recent years, and it’s critical that the US government prioritizes the development of new tools in any HIV and AIDS strategies and goals it creates. But as Clinton stressed, it’s vital that the United States and its partners deliver on the promises outlined in the blueprint. According to the International AIDS Vaccine Initiative, the “realization of all this promise depends on sustained funding for HIV prevention research. Political will, too, must be sustained to secure the progress already made in the campaign against HIV. We must remember that any call to bring about the ‘end of AIDS’ will only be fulfilled if HIV prevention remains high on the list of global health priorities.” As the Obama Administration enters its second term in the new year, leaders across the Administration must ensure that the priorities outlined in this blueprint are fulfilled, particularly the US commitment to research and new HIV and AIDS tools. We cannot reach an AIDS-free generation otherwise.

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. The latest G-FINDER report finds that annual investment in neglected disease research and development (R&D) from donors worldwide has been in the decline in recent years. And while the report finds that the United States has maintained its position as the top funder of neglected disease R&D, US funding still decreased in 2011 by $30.6 million. A treatment for sleeping sickness has entered clinical trials in the Democratic Republic of the Congo, and trials will start soon in Central African Republic. The study was initiated by the Drugs for Neglected Diseases initiative (DNDi) and its partners. A new opinion piece argues that sequestration “would significantly delay new, life-saving medical treatments. But what many people don’t realize is that the massive cuts also would put a chokehold on the creation of high-tech jobs and threaten US global competitiveness.” The US Food and Drug Administration approved 35 new medicines in its fiscal year 2012, which ended in September, according to a new report. That number is one of the highest levels in years. Senators Patrick Leahy (D-VT) and Christopher Coons (D-DE) have introduced legislation that aims to bolster the US Patent and Trademark Office’s Patents for Humanity initiative by making certificates awarded through the program transferable to third parties.

In this guest post, Eleonora Jiménez-Levi—senior researcher at the Treatment Action Group (TAG)—writes about the latest report on the state of funding for tuberculosis (TB) research and product development. Mycobacterium tuberculosis (MTB) is one of the oldest known human pathogens that continues to pose a global public health threat in the 21st century. The World Health Organization reports that MTB—the bacterium that causes TB disease—was responsible for 1.4 million deaths in 2011, including 430,000 deaths among people with HIV. Current strategies and tools cannot effectively prevent and control TB. BCG, the only vaccine approved for TB, is over 100 years old. BCG cannot offer protection against pulmonary TB, and cannot be used among HIV-positive infants. The most widely used diagnostic tool—sputum smear microscopy—detects roughly 50 percent of pulmonary TB cases, and fares far worse among people with immature or compromised immune systems. Treatment against drug-sensitive TB involves 6–9 months of first-line drugs, and drug-resistant TB requires 20 months or more. The lengthy treatment regimen results in suboptimal and, at times, deadly outcomes because of several challenges including difficulty of adhering to treatment, drug stock-outs, toxic side effects, treatment costs, and incompatibility between TB drugs and antiretroviral medicines for people with HIV. A doctor examines the chest x-ray of a TB patient. Photo credit: PATH. For the seventh year, TAG and the Stop TB Partnership have published the latest investment data and analysis on the state of global TB research and development (R&D) funding. The 2012 Report on Tuberculosis Research Funding Trends: 2005–2011 builds on seven years of investment data to report on annual funding trends and gaps among the leading TB R&D donors. The report analyzes current spending levels across six research areas to assess how they compare to the R&D funding targets in the Global Plan to Stop TB 2011-2015. The new report found that in 2011, 81 donors invested $649.6 million in TB R&D. This is an 82 percent increase from the baseline year of 2005, but only a 3 percent increase over 2010 funding. The top ten donors disbursed $506.7 million of the $649.6 million total, or 78 percent of the global share. Despite the small funding increase observed between 2010 and 2011, the world continues to fall $1.35 billion short of the annual $2 billion funding target recommended by the Global Plan. To accelerate global TB R&D and capitalize on the scientific gains achieved in the last decade for drug, vaccine, and diagnostic development, more financial resources are needed, particularly in: Biomarker discovery to modernize TB drug and vaccine development by demonstrating the progress and effects of treatment or immunity early on, which in turn radically reduces the time and costs of the clinical trials. Biomarker discovery for the development of a point-of-care diagnostic test that can identify people with latent TB at risk of developing active TB, as well as biomarkers associated with infection, treatment response, cure, and drug susceptibility or resistance. TB sample banks that support biomarker discovery with well-characterized specimens. Late-stage drug and vaccine clinical trials, many of which will take place in high-burden countries with limited laboratory capacity or infrastructure that meet good clinical practice standards. Basic science research to enhance our scientific understanding of TB disease and the MTB pathogen. The global TB community should also give further consideration to: Establishing access plans to optimize the rational use of new TB drugs and regimens when they become available, as well as prevent the emergence of resistance to new TB treatments. Developing pediatric investigational plans to ensure child-friendly formulations are available as quickly as possible. Conducting drug-drug interaction studies for novel TB drug regimens. Providing compassionate use and expanded access, which are particularly important for people with drug-resistant TB who have limited treatment options. Modernizing TB regulatory guidelines and streamlining regulatory requirements across and within regions. All of these recommendations and more can be accomplished if policymakers, researchers, activists, clinicians, donors, implementers, and people affected by TB pledge their commitment to achieve zero TB deaths, zero new TB infections, and zero suffering from TB. The Zero Declaration, created by a worldwide group of TB stakeholders in June 2012, is a new strategy that calls on the international community to embrace a “new global attitude in the fight against TB … and mobilize sufficient political will and resources to prevent needless transmission, sickness, and death.” The goal of the campaign is to organize and inspire people to work toward eliminating TB as a public health threat with greater urgency, more political will, and increased investments. Promising new tools are on the horizon, but without political will and adequate funding to support the development of new vaccines, diagnostics, and drugs, the world will not be able to contain the growing problem that drug-resistant TB poses, much less eliminate TB as a public health threat by 2050.