News

Moderator Lisa Cohen and panelist Tony Pipa. With the Sustainable Development Goals (SDGs) freshly adopted, the Global Health Technologies Coalition (GHTC) gathered representatives from the private, public, and nonprofit sectors on Capitol Hill this past Wednesday to discuss what it will take to achieve the global goals. One message rang loud and clear during the briefing: health innovation will be critical to achieving the SDGs, and it will take political commitment, funding, partnerships, and accountability mechanisms to deliver the next generation of health innovations needed to get us there. The panelists agreed that the SDGs—which set ambitious targets to end poverty, infectious disease epidemics, and preventable maternal and child deaths—are an important device for catalyzing political commitment and driving collective action to improve global health. Anthony Pipa, the US special coordinator for the Post-2015 Development Agenda, called the SDGs “a very optimistic global moment” and noted the power of the 193 United Nations member states coming together to agree on a set of goals, a point reinforced by moderator Lisa Cohen, executive director of the Washington Global Health Alliance. Pipa explained that the success of the Millennium Development Goals (MDGs) demonstrates the power of goal setting in advancing global health, and that the SDGs must build on that success. But he also clarified that “the SDGs are not just the MDGs 2.0,” as the SDGs are broader and more ambitious than the MDGs when it comes to health—they set goals to eliminate disease and preventable deaths, not just reduce them. He also described the inclusivity of the development process and the universality of the agenda, which promoted a sense of ownership among member states that are now starting to integrate the SDGs into their own national agendas. In the case of the US government, Pipa explained that the SDGs closely align with US priorities in global health and reflect the government’s longstanding commitment to health research and development (R&D). Panelists Dr. Dean Jamison and Kaitlin Christenson The need for strong and sustained investment in global health R&D to reach the SDGs was highlighted throughout the briefing. Dr. Dean Jamison, co-chair of the Lancet Commission on Investing in Health, explained that “we benefit today from investments in R&D of products made decades ago” and that health innovations have driven significant progress in global health over the past few decades. Jamison noted the Lancet Commission report’s conclusion that it is possible to achieve the ambitious SDG vision, but not without greater investment in R&D for new health technologies. He emphasized that, although health R&D can be a long and expensive process, it delivers huge returns on investment. The panelists all underscored that, because the SDGs are so ambitious, no one sector alone can ensure their success. Instead, it will take partnerships between the public, private, academic, philanthropic, and nonprofit spheres to reach the targets, particularly for health. Kaitlin Christenson, acting director of Advocacy and Public Policy at PATH, discussed the importance of product development partnerships (PDPs) in advancing the development of health technologies. Christenson remarked that PDPs leverage resources, expertise, and financing from across sectors and help “multiply” the impact of public sector investment. Gary Cohen Gary Cohen, senior vice president at BD, echoed this sentiment, noting that “so much of what we’ve achieved in health is due to collaboration, we couldn’t see doing it any other way.” Moving a promising idea through product development, regulatory approval, scale up of manufacturing, and market strengthening, in order to ultimately get the health innovation into the hands of patients who need it, is a daunting process. It’s not possible to do this, Cohen explained, without the involvement of the private sector. Cohen noted that throughout the process of developing the SDGs, it was widely acknowledged that the private sector would be critical to their successful implementation. Christenson pointed out that, while it is clear new health tools are needed to reach the global goals, it is not just about designing any technology, but the right technology. She explained that developers must stay focused on the needs of the end user and ensure that the product is affordable and contextually appropriate in order to achieve the desired health impact. She also noted that the community must be diligent in identifying health technologies with the greatest potential to drive dramatic improvements in health and rally the necessary investment and political support to bring them to scale. As an example, she highlighted PATH’s Innovation Countdown 2030 initiative, which identifies 30 innovations with the greatest promise to accelerate progress toward the SDGs. Attendees check out health technologies funded by US government investment. Pipa also emphasized that reaching the SDGs will require “doing business differently” and pointed to innovation within the US government to promote new thinking in global health, like USAID’s Saving Lives at Birth Grand Challenge. In the same vein, Christenson pointed out that “innovation comes from everywhere, not just white-coated scientists” and called for systems and policies that support a broad view of innovation. Cohen said that “innovation in business models will be just as important as innovation for technologies” and called for greater collaboration among key players to ensure that R&D investments are translated from the development of new tools and technologies to the ultimate delivery and use of lifesaving products. An audience member from PATH pointed out that, despite the importance of new health technologies to achieving the SDGs, the agenda does not currently include any indicator to measure progress in global health R&D, citing concern that “unless something is measured, it doesn’t necessarily happen.” Panelists agreed that data and measurement is a critical part of ensuring we are on the right track to reach the health targets. Pipa highlighted the expanded availability of data and the need to leverage data from different sectors, while Jamison cited the importance of data and monitoring in holding actors accountable to their SDG commitments. Though it will be a challenging road ahead to reach the SDG targets by 2030, the panelists were optimistic it could be done. Cohen emphasized that “these seemingly intractable problems are solvable.” Pipa agreed, saying, “the political consensus around ending extreme poverty is really powerful, and we shouldn’t underestimate that power.”

In this guest post, Dr. Jayasree K. Iyer—Chief Scientific Officer at the Access to Medicine Foundation—writes about the findings from their newest study, which examines the current state of the vaccine pipeline and how large pharmaceutical companies are considering access to these products. Vaccine manufacturers play a critical role in innovating new vaccines and making existing vaccines more suitable for use in developing countries. Apart from developing effective, appropriate vaccines, manufacturers work with relevant stakeholders to ensure that access to these vaccines is ramped up upon market introduction, addressing issues like supply and affordability. Vaccines are the cornerstone of disease prevention: mass immunization has led to the eradication of smallpox, and between 2000 and 2010, it helped cut the global childhood mortality rate in half. Yet many people cannot access full immunization, especially with newer vaccines. Recent attempts to boost immunization coverage (e.g., of the relatively new pneumococcal and rotavirus vaccines) shows that immunization coverage and access can be increased when good vaccines, committed funding, and strong global partnerships come together. The vaccine R&D challenge The challenge with vaccine development and access is twofold: the need for new vaccines for diseases where there are no vaccines on the market (e.g., for HIV and AIDS, malaria, and for almost all neglected tropical diseases (NTDs)) and the need for vaccines that are more suitable for use in the developing world (e.g., thermostable vaccines, oral, more effective vaccines and combination vaccines). The research and development (R&D) pipeline for vaccines is primarily driven by the vaccine industry, product development partnerships (PDPs)—such as the PATH Malaria Vaccine Initiative (MVI) and Sabin Vaccine Institute—and other research institutes. With the recent outbreak of Ebola, it is clear that vaccine innovators play a crucial role in the development of safe, quality, effective vaccines, and that R&D must be stimulated by donors and regulators, as well as by market forces. Tracking vaccine development The Access to Medicine Foundation has been tracking the development and deployment of medicines and vaccines for use in developing countries since 2008. Our almost ten years of research into the world’s 20 largest research-based pharmaceutical companies has given rise to special insight into the opportunities for addressing specific challenges. These insights can be used to inform the work of other pharmaceutical companies, as well as governments, investors, donors, and market shapers. Our research prompted us to embark on the first Access to Vaccines Index (due early in 2017). While talking with vaccine stakeholders and defining appropriate, robust metrics and analysis scopes for this new access-analysis tool, we also published a preliminary report on vaccine R&D, which offers a baseline on industry efforts in this area. Data from the 2014 Access to Medicine Index was used to report on (a) the R&D pipeline for high burden vaccines and (b) the use of mechanisms that ensure rapid access and uptake of vaccines upon market approval (termed “access provisions”). Promise in the pipeline The study highlights and confirms that the vaccine R&D pipeline covers the highest burden diseases. There appears to be relatively promising candidate vaccines emerging for diseases where no vaccine yet exists on the market (e.g., for malaria and dengue). However the pipeline for these types of vaccines remains relatively empty. Considering the large development costs associated with vaccine development and the high attrition rate of vaccine R&D projects, there is little promise that other new high-need vaccines products will enter the market in the coming years. Persistent R&D gaps remain Considering the large development costs attributed with vaccine development and the high attrition rate of projects in the pipeline, there may be few vaccines entering the market in the coming years from industry. There may be other projects under development by research institutes, PDPs, and biotech companies and by other smaller vaccine manufacturers. However, without significant investment (which many of the stakeholders may not have access to), chances of successful products reaching the market remain limited. And with so many global priorities competing for investments, product development is almost certain to take a strong hit. Interestingly, the majority of vaccines in the pipeline are aimed at adapting or improving existing vaccines (e.g., to improve their efficacy or give longer-lasting immunization or to develop combination vaccines), or creating the next generations of existing vaccines. Many of these projects target leading causes of child death including diarrheal disease and pneumonia. This is important, as coverage, adherence, and acceptance is also improved with these types of products. Accelerating access to suitable new vaccines Having a vaccine on the market that meets a health need and is suitable for LMICs does not automatically resolve access issues. Photo: PATH/Aaron Joel Santos To drive strategic R&D decisions that meet public health needs, particularly for low- and middle-income countries, the World Health Organization develops Preferred Product Characteristics (PPC)– recommendations for priority indications, target groups, possible immunization strategies, and desired clinical data, as well as ensuring that vaccine presentation and packaging is suitable for low-resource settings (such as remote populations, or where infrastructure is weak). Together these PPCs and presentation and packaging recommendations form an essential part of vaccine developers’ Target Product Profiles (TPPs). Companies can use these TPPs to assist in defining target product characteristics, such as dosage, target price, target population, and route of administration. Just having a vaccine in the pipeline or even on the market that meets a priority need and is suitable for use in a low- and middle-income country will not automatically solve the world’s access problems. Companies and other stakeholders have a joint responsibility to ensure that there are mechanisms in place to ensure the vaccine’s quick roll-out, thus ramping up coverage of vaccines upon market introduction. Companies are expected to continuously innovate through R&D towards the goals defined in the TPP, and ensure that access provisions are aligned with the needs and intended reach of vital products. Big pharma actions to drive access In our vaccine R&D study, we found that companies have used at least one access provision in 17 percent of the vaccine projects in the pipeline. Out of 70 projects analyzed, 42 are nearing the end of the pipeline, yet only 8 projects have provisions in place for driving access once they hit the market. It is not clear whether access provisions are truly lacking, or whether low transparency accounts for this gap. A close look at the usage of the provisions revealed examples such as tiered pricing strategies aimed at ensuring affordability, the use of local manufacturing capacity based in a developing country to reduce cost and ensure local supply, the use of advanced market commitments, commitments for discounted prices, and commitments to manufacture large volumes at the point of market entry. The systematic application of access provisions will help ensure that successful vaccine candidates are suitable for use in the countries that need them most and align with the needs outlined in the PPCs and TPPs. This could help ensure uptake, acceptance, and coverage is ramped up upon market entry and ultimately more lives are saved. While we indeed need clear incentives for companies to invest in vaccine pipelines, they are encouraged to consider and put in place access provisions as early as phase II clinical stages to develop vaccine candidates with poor, rural, and isolated populations in mind.

Dr. Robert Gallo co-discovered the HIV virus and subsequently led the development of the first HIV and AIDS blood test. Now, his team at the University of Maryland School of Medicine is initiating human clinical trials of an experimental HIV and AIDS vaccine that aims to prevent the virus from attaching to immune cells. The HIV virus uses a protein on its surface—gp120—to bind onto one part of human immune cells—the CD4 receptor—and then subsequently bind onto a second part of the cell—CCR5. This vaccine candidate interrupts this process by preventing the HIV virus from attaching to CCR5, therefore disrupting the process of infection. While many vaccines target specific viral strains, this candidate is intended to neutralize all strains of the HIV-1 virus. The first phase of the trial will enroll 60 healthy, adult volunteers to determine the vaccine’s safety and to ascertain if it successfully triggers an immune response. A 23-year-old engineer has developed an inflatable incubator that is designed to be easy to transport, use, and maintain, which could dramatically reduce deaths of premature babies in refugee camps. Developer Briton James Roberts has created electronic and functional prototypes which indicate that the incubator, known as MOM, will provide the required warm, stable, and sterile environment. Roberts designed the product with Syrian refugee camps in mind, as an estimated three-fourths of premature infant deaths in camps could be prevented with proper incubation. The incubator can be collapsed (see Figure 1) to fit in care packages commonly distributed in refugee camps and while it does require electricity, it can run off of a car battery or solar power. Roberts recently won two cash prizes to support product development and manufacturing but is continuing to fundraise to support the costly process of regulatory approval for medical devices. Roberts believes the product could be ready for market by 2018. Photo: CDC/ Dr. Scott Smith The US Department of Defense (DoD) and National Institutes of Health (NIH) have both supported the research and development (R&D) of drugs and vaccines for Ebola since the early 2000s. In both agencies, budget constraints halted progress despite promising clinical trials. In 2004, a researcher at army installation Fort Detrick was exposed to Ebola, prompting the DoD to partner with pharmaceutical company Sarepta Therapeutics to rapidly develop an experimental treatment for Ebola. The treatment was given emergency approval by the US Food and Drug Administration (FDA), but the researcher did not become infected and the treatment remained unused. In 2010, the DoD awarded Sarepta a US$291 million contract to continue its work. Sarepta’s drug demonstrated 60 to 80 percent efficacy in clinical trials with primates and proved safe for use in phase 1 human clinical trials. In September 2012, the FDA granted Sarepta’s drug fast-track status—to expedite its regulatory review—but just one month later, the DoD discontinued the project, in light of looming budget cuts that seemed inevitable due to congressional gridlock. In mid-2014, after the onset of the Ebola epidemic in West Africa, Sarepta CEO Chris Garabedian approached the DoD about the possibility of continuing their work, however, the agency chose to fund products that were not as far along as Sarepta’s. While the DoD has since reconsidered—inviting Sarepta to apply for funding for phase II trials—Sarepta declined, and under new leadership, has terminated their Ebola R&D. The US Department of Agriculture (USDA) has contracted two veterinary pharmaceutical companies—Harrisvaccines and Ceva Santé Animale—to manufacture, store, and ship vaccines to prevent the strain of avian influenza which caused an outbreak and consequent shortage of eggs and turkey in the United States earlier this year. The contract requires the companies to store the vaccines for up to five years, regularly testing them for quality assurance, and will ship them within ten days to anywhere in the United States if requested by the USDA National Veterinary Stockpile. To improve clarity and expediency of communication—for which the agency was criticized by veterinary, poultry, and egg groups during this year’s outbreak—the USDA has also worked with Iowa State University to translate avian influenza–related biosecurity materials for Spanish-speaking farm workers.

Photo: Heng Chivoan When most people think of hunger, they think of a lack of food—a nation suffering from crop failure and famine, or a family that can’t afford to put enough food on the table. But hunger is a far more complex issue with many dimensions. Tackling hunger is not only about ensuring reliable and affordable access to food, but access to the right types of food that are rich with the nutrients human bodies need to function and thrive. Two billion people—more than a quarter of the world’s population—suffer from micronutrient deficiencies, an affliction GHTC member HarvestPlus calls “hidden hunger.” Micronutrient deficiencies, or insufficient amounts of vitamins and minerals, including vitamin A, zinc, and iron, are a major public health problem, particularly in developing countries where people’s diets rely on inexpensive staple crops that often lack nutrients. While those affected may not show the visible symptoms of hunger and malnutrition, micronutrient deficiencies can still have a devastating impact on health, resulting in stunting, blindness, and lowered IQ in children, as well as increased vulnerability to infectious diseases and complications in pregnancy and childbirth. This World Food Day, we’re joined by Peg Willingham, head of Advocacy and Policy at HarvestPlus, to discuss biofortification—an innovative approach to tackling hunger and malnutrition. What role can biofortification play in eradicating hunger, and in particular, micronutrient deficiency? PW: Ideally, everyone should have access to fruits, vegetables, and protein, or to important interventions like vitamin supplements. Unfortunately, these are often out of reach for smallholder farmers who subsist on the food they grow on an acre or two of land—which constitutes the majority of people living in developing countries. A cost-effective new tool to target these families is biofortification—using conventional breeding techniques to increase the levels of vitamins and minerals in the most widely consumed staple foods in Africa, Asia, and Latin America. Can you talk a bit about the process of biofortification and how these crops are introduced and scaled up in developing countries? PW: Until recently, crop developers have focused on higher yield per acre and resistance to diseases, pests, drought, and heat. With support from HarvestPlus, researchers are now including nutrition as an important trait. They search existing seeds banks—which contain 300,000 plant varieties—to identify ones that are naturally high in these important micronutrients, but which cost the same, produce the same yield, and don’t require any extra inputs like more water or fertilizer. National agricultural research authorities around the world then test these crops locally to make sure they grow properly in different soils and climates. We then work with a broad array of partners to get these healthier crops to farmers. This varies from country to country and from crop to crop. For example, most farmers in Zambia buy their corn seed from local private companies, so we work with those firms to get them to make the switch to seeds that are high in vitamin A. In Uganda, farmers who receive donated vitamin A sweet potato vines share them with other farmers the following season, in a constantly expanding cycle. Ten million people are currently growing and eating these healthier foods in more than 30 countries, and testing is underway in almost 20 more countries. What’s in HarvestPlus’ pipeline of biofortified crops? What crops have already been released or are currently in development? PW: Biofortification is centered on the staple foods that people already eat regularly. More than a dozen crops are currently available to farmers: bananas, beans, black-eyed peas, cassava, corn, lentils, pearl millet, plantains, potatoes, pumpkin, rice, sorghum, sweet potato, and wheat. We are excited by recent data demonstrating the nutritional impact of biofortified crops. A study in Mozambique showed vitamin A sweet potatoes significantly reduced the incidence and duration of diarrhea, one of the leading causes of mortality in African children. Pearl millet extra rich in iron was able to reverse iron deficiency in school-aged Indian children in six months. More studies are underway. What role can policymakers—at the national level, as well at the multilateral and global level—play in advancing biofortification and other innovations to improve nutrition? PW: We have been gratified to see how quickly policymakers have embraced this innovation, paving the way for better health and livelihoods. The World Bank has begun funding countries to scale-up biofortification, and the African Union’s commissioner for Rural Development and agriculture and health ministers around the globe have publicly endorsed it. Several countries have included biofortification in their national health and agriculture strategies and are investing their own money in scaling-up access. Additional resources will accelerate this process.