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Maximilian Geigenmueller, advocacy officer at the Berlin office of DSW, answers questions about the German PDP funding call. Earlier this month, Germany’s Federal Ministry of Education and Research (BMBF) the renewal of the German funding program for product development partnerships (PDPs) conducting research and development (R&D) for health technologies to combat poverty-related and neglected diseases (PRNDs). The call for proposals for €50 million over five years (2016–2021) is expected to be published shortly. GHTC Breakthroughs blog is joined by Maximilian Geigenmueller, advocacy officer at the Berlin office of Deutsche Stiftung Weltbevoelkerung (DSW)—an international development organization that conducts global healthR&D advocacy—to learn more about this funding call and its importance in advancing R&D for lifesaving global health technologies. Q: Why is this funding call significant among efforts to advance global health R&D? A: This funding call is very significant in the sense that it ensures the continuation of German PDP funding. The first funding round was considered to be a pilot, and it was by no means a given that there would be a second one. Germany was late in joining the group of governments funding PDPs in 2011, when the United States and United Kingdom had already been funding the model for years. Germany also started out at a small scale—€20 million over four years. Over the duration of this time, it was gradually increased to over €26 million, which I think was an indication that the German government began to understand and increasingly appreciate the PDP model. This was further reinforced by an independent evaluation of the first funding round which was very positive. I think with this second call Germany has established itself as a PDP supporter in the medium and long term—albeit at a still rather modest funding level. Q: How does this second funding call differ from the first? A: First of all, there will be more money: €50 million over five years from 2016 to 2021, which is still not quite what we had hoped for but almost double the first funding round. Secondly, this call will be open for proposals not only on the 17 neglected tropical diseases (NTDs) and malaria, which were included in the first funding round, but also HIV and AIDS and tuberculosis (TB). Q: The announcement of this funding call occurred at the G7 Research Ministers conference which Germany chaired. How does this funding call reflect Germany’s broader global health priorities? And how does it align or differ with the priorities agreed upon at the G7 conference? Photo: PATH/Jennifer Fox A: I think the German government finally decided it was time to take on a leadership role in global health that appropriately reflects the country’s economic and financial standing in the world. I am sure the Ebola crisis—an outbreak of an infectious disease spreading at a breathtaking rate with no immediate treatment available and relatively close to home—had a lot to do with it. The prioritization of PRNDs/NTDs, antimicrobial resistance, and Ebola on the G7 agenda has given a lot of visibility to health issues in the international debate this year. For a while it looked like the G7 were leaving out the big three diseases of poverty—HIV and AIDS, malaria, and TB—and would only discuss NTDs, particularly as the G7 leaders’ summit communiqué only referred to NTDs. Fortunately, at the G7 Research Minister Conference in Berlin on October 8–9, the ministers of research included not only NTDs but also explicitly mentioned malaria, tuberculosis, HIV and AIDS, and several other infectious diseases. There are some interesting commitments in the communiqué, such as better coordination of research efforts. Germany’s Minister of Research Johanna Wanka has offered to host a brainstorming workshop on this in 2016. Also, the communiqué states the intent to develop a joint G7 pilot research activity for R&D projects to build research capacities in high PRND/NTD burden countries. Combined with the Japanese 2016 G7 presidency which is very likely to have health as one of its priorities as well, I think we have a good chance that health is going to stay at the center of the international agenda for a while. Q: As a global health R&D advocate, what excites and disappoints you about this funding call? A: I am excited about it being out and that there is significantly more budget available this time. It is of course not enough, not by a long shot, but maybe there will be some top-ups to the second funding round, as was the case in the first funding round when Germany started out with €20 million and ended up with €26 million. There is also the prospect of institutionalizing German PDP funding, moving away from providing limited funding for a number of years to making it a permanent feature in the German budget, but this is most likely only going to happen at the end of the second funding round. What was also really exciting was the process of ensuring this second funding round happened. I think all advocates—PDPs, nonprofits, philanthropic organizations, and others—did a great job in keeping global health R&D in the political debate. For example, during the summer the All Parliamentary Group on Population and Development of the German Bundestag wrote a letter to Chancellor Angela Merkel voicing their support for global health R&D and the PDP model, which was signed by 19 members of parliament (MP). I think nineteen MPs coming together from three out of the four parties in the Bundestag to sign a letter in support of a measure which represents only a minuscule share of the research ministry’s annual budget is fairly remarkable.

Photo: National Institute of Allergy and Infectious Diseases (NIAID) Researchers at Tulane University successfully tested an experimental aerosol tuberculosis (TB) vaccine in monkeys, demonstrating that the candidate was more effective in protecting against infection than BCG—the only TB vaccine on the market. To create the vaccine, the researchers genetically modified the TB bacterium, removing a gene that plays a key role in infection. The vaccine was then administered to seven monkeys, while another seven each received either an aerosol version of BCG or no vaccine. After eight weeks, the monkeys were exposed to TB at a lethal level. The experimental vaccine not only was more protective than BCG, but it also induced a stronger immune response in the lungs, which is thought to be key to preventing infection. A new formulation and coating for existing HIV and AIDS medicines could significantly decrease the frequency with which the drugs must be taken. Researchers at the University of Nebraska Medical Center used a process called nanoformulation, which involves modifying drugs at the most basic level (i.e., atomic or molecular level) in order to convert them into nanoparticle crystals. In this experiment, the antiretroviral medicine crystals were coated with fat and protein, allowing them to last much longer in the body. In the lab, the drug proved to be effective in combatting HIV and AIDS. The team hopes its findings could eventually lead to the development of an HIV and AIDS treatment that could be administered once or twice per year, rather than daily. Resistance to the leading antimalarial—artemisinin—was first reported in Cambodia in 2008, and a new study in Nature Communications suggests that the drug-resistant parasite has the ability to infect African mosquito species. A team of researchers at the US National Institute of Allergy and Infectious Disease infected two Asian and one African mosquito species with the drug-resistant malaria parasite, and all three species proved to be susceptible. Furthermore, the African species were no less vulnerable to infection, which came as quite a surprise as the species have evolved independently for more than 30 million years. Health officials from the United States and Saudi Arabia are considering a collaboration for the development of a vaccine against Middle East Respiratory Syndrome (MERS), which has infected more than 1,500 people across 27 countries. There are currently a number of MERS vaccines in development and several have yielded promising results in animal studies. The Saudi Ministry of Health has US$70 million available for the project; however, the proposed role of the US government remains unknown.

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.