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In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week. The recently released annual G-FINDER survey found that total global funding for neglected disease research in 2012 amounted to $3.2 billion, which included an increase of $92.1 million—or 3.2 percent—over 2011 levels. Unfortunately, non-US government funding fell by 12.4 percent in 2012, with 11 governments either cutting or freezing funding. This drop was offset by an increase in US government funding by 6.4 percent from 2011. After returning from the 8th International Summit of Heads of Medicines Regulatory Agencies in Amsterdam, US Food and Drug Administration (FDA) Commissioner Margaret Hamburg wrote in FDA Voice about the increasing need for international regulatory agencies to work together. The FDA has granted GHTC member Medicines for Malaria Venture and pharmaceutical company GlaxoSmithKline Breakthrough Therapy designation for tafenoquine, an investigational medicine for the treatment and relapse prevention of Plasmodium vivax malaria. The Bill & Melinda Gates Foundation has named Susan Desmond-Hellmann, chancellor of the University of California, San Francisco, as its new CEO. A new article on SciDev.net examines how science is now more influential within the US Agency for International Development than it has been for the last two decades. Civil society groups are reacting to a set of demonstration projects recently selected by a World Health Organization expert panel to find new ways to fund health research for neglected diseases.

The US Agency for International Development (USAID) is the nation’s lead foreign assistance agency. Throughout its 50-year history, USAID has worked with other government agencies, the private sector, and nongovernmental organizations to support the development and introduction of affordable health products appropriate for addressing the diseases and health issues in developing countries. The GHTC has launched a new blog series to highlight the impact of USAID’s commitment to global health research and development (R&D). The series will showcase stories from individuals who have benefited from the agency’s support and investment in R&D—as well as people whose lives would vastly improve if USAID were to expand its support for health R&D into new areas. Our second post is by Natalie Nelson, a tuberculosis (TB) survivor from Tennessee who believes that if USAID expanded its support for health R&D to include TB vaccine research, the agency could help change the lives of TB patients like her worldwide. Q: Many people think of TB as a disease of the past—or a disease that only impacts people in developing countries. But your experience with TB demonstrates that the disease impacts Americans, too. How did you contract TB, and how has the disease impacted your life in the US? A: I was working as a volunteer physical therapist in a small healthcare facility in South Africa when I contracted TB. I assisted patients infected with TB and HIV. After I came home to the US, I started getting sick. It took an entire year to be correctly diagnosed—and after finding out I had multi-drug resistant TB (MDR-TB), my life drastically changed. TB forced me to quit my job as a physical therapist. I received a 2-year treatment program, a mask, an isolation sentence, and with that, a new stigma. My life and my desire to return to South Africa were both placed on hold as I accepted a new lifestyle: swallowing drugs as I was directly observed by a health care provider each morning and afternoon and a drug infusion for 2 hours, 5 days a week for the first 6 months, then 3 days a week for the next year and a half. Natalie Nelson is a TB survivor from Tennessee who believes that if USAID expanded its support for health R&D to include TB vaccine research, the agency could help change the lives of TB patients like her worldwide. Credit: Aeras. Although the treatment was completely exhausting both physically and mentally, having TB brought an awareness that I never would have experienced otherwise. I feel blessed to have the care that I received, and now I can fight for and speak up for those who do not have the same options. Q: Your story also demonstrates the growing issue of resistance to currently available TB treatment options, which makes the disease much more difficult and expensive to treat. What was that experience like for you, in terms of undergoing TB treatment, its side effects, and its costs? A: Having TB was extremely scary. So many things were uncertain, and the questions were endless. Was the medicine working? Would I eventually need surgery? Would I even survive? No one could tell me definite answers. I just had to keep pressing forward and swallowing the pills, as there was no alternative. There were numerous side effects from the medication—it seemed like a new one would surface each week. First, I was terribly weak and frail. I lost over 20 pounds and my iron levels bottomed out, so I was tired all the time. Then, I developed excruciating kidney stones in both kidneys. Every afternoon around 3:00 my face would flush, my temperature spiked, and I had to lie down. At times, my fingers went numb, and I dropped things constantly. Though all of this was terrible, the worst side effect was short term memory loss from Cycloserine—the drug I was taking to treat my TB. I forgot what I was saying mid-sentence, and constantly repeated myself. I got lost driving and forgot to show up to appointments. I became socially awkward and utterly undependable during this time. This was really difficult and embarrassing. The costs were astronomical. TB cost me my job, my energy, years of my life, special moments with friends and family, my health—and at times—my sanity. I am unsure of the exact dollar amount because there are some losses that can't be calculated, but the cost was easily over half a million dollars. Q: Currently, there is only one vaccine to prevent TB—the Bacille Calmette-Guérin (BCG) vaccine. While BCG is the most widely used vaccine in the world, it has not successfully eliminated the disease due to its limited efficacy. Based on your personal experience with TB, how would new and improved vaccines—as well as treatment options—help in the fight against TB worldwide? A: We can never truly eradicate TB without a vaccine, better diagnostics, and treatment options. I passionately believe that prevention is key and that new vaccines and treatments could mean billions of dollars saved every year. It would mean that TB would not be a plane ride away. It would mean the college student on spring break traveling to Africa for mission work wouldn't have to worry about his safety. Currently, it would mean that the person who dies every 30 seconds of every day of TB would not. Q: As you know, USAID currently does not support research to develop new TB vaccines. How would support from USAID for TB vaccine research help patients like you in the US and around the world? A: Support from USAID to develop new vaccines will mean moments and years and lives saved around the world. A vaccination before my trip to South Africa might have changed everything. The truth is I will never know what could have been. But what IF I had received an improved TB vaccine before traveling? Maybe I would have returned home and never lost my job as a physical therapist. Maybe I would have stood next to my friend Laural as she said her vows, rather than lying in bed on isolation. Maybe I would have been more present in my friends’ and family's lives during those three years. Maybe I would have gone back to South Africa. For countless others around the world, a better TB vaccine would be life-changing. Maybe a mother will not worry about passing the disease to her children. Maybe a doctor will save millions of lives. TB does not discriminate. It does not matter who you are—if you breathe, you are at risk. This is why support for research of new vaccines is absolutely imperative. USAID should absolutely choose to be a part of this global fight. After all, who would willingly choose to NOT be a part of claiming lives were SAVED, lives were LIVED, and dreams were PURSUED? I hope we can all confidently say we were a part of this solution.

In this guest post, Kari Stoever—vice president of External Affairs at Aeras—writes about European support for new vaccines against tuberculosis (TB). TB is a disease most often associated with countries in the developing world, particularly in Asia and Africa. While India and China had the largest number of cases in 2012, Europe is not immune. Europe accounts for almost 18 percent of global cases of multidrug-resistant and extensively drug-resistant TB, with the highest burden in Eastern Europe. TB is estimated to cost the European Union around €6 billion (US$8 billion) each year. New vaccines to prevent TB will be the most cost-effective and long-term approach to saving lives and money, so it makes sense that Europe leads the world in the research and development (R&D) of TB vaccines. Europe is a major contributor to the development of vaccines globally, with as much as 75 percent of the global TB vaccine portfolio either originating in Europe or being developed in partnership with European public or private organizations. Of the five major donors contributing to TB vaccine R&D, three of them are European. Since 2000, the European Commission (EC) has invested more than €34 million (over US$46 million) in research for new TB vaccines. Along with European Union Member States, the EC has also invested more than €42 million towards clinical trial site preparedness, capacity building, and support in Africa through the European and Developing Countries Clinical Trials Partnership (EDCTP). Recently the EC announced a €25 million (approximately US$34 million) call for proposals to accelerate the development of new TB vaccines. Europe is a major contributor to the development of vaccines globally, with as much as 75 percent of the global TB vaccine portfolio either originating in Europe or being developed in partnership with European public or private organizations. Credit: PATH. The announcement comes as an important new collaboration between European funders and a global network of researchers is forming. The new initiative, the Global TB Vaccine Partnership (GTBVP), aims to channel finances to TB vaccine R&D to establish a robust and diversified global portfolio of clinical and preclinical candidates. One of the top priorities for the GTBVP will be to strengthen and diversify the preclinical and clinical portfolio to facilitate a more rational, cost-effective, and accelerated vaccine development process through the sharing of technical, financial, and market risks. The GTBVP’s role will be to foster greater alignment among researchers and funders’ objectives, and implement an effective mechanism for collaboration, information-sharing, and decision-making. Aeras, the Netherlands-based TuBerculosis Vaccine Initiative (TBVI), the EC, the European Investment Bank (EIB), the Bill & Melinda Gates Foundation, and the EDCTP are spearheading the development of the GTBVP. The evolution of the collaboration The push for a new financing mechanism and governance structure began in March 2012 when the EC, with support of the EIB, expanded its engagement with TBVI and Aeras to develop a Business Case for Investment. The document provides evidence of a viable market incentive for new TB vaccines, opening the door for more expensive later stage trials to be financed through cost-sharing arrangements with industry and the use of other risk-sharing financial instruments. Public sector grant funding for early stage research would act as a catalyst for this private sector investment, providing significant leverage to donors. By working with researchers around the world, it is envisioned that the GTBVP will play a crucial role in ensuring that research into life-saving TB vaccines continues to progress in the most efficient way possible. Europe’s leadership has been critical to the formation of the partnership, and the EC is to be commended for its excellent commitment to much-needed health interventions, especially a vaccine that would drastically reduce the number of TB cases. TB is a global threat requiring global action, and through the new partnership there is a historic opportunity for donors, disease-endemic countries, researchers, and other stakeholders to work together and achieve the goal of having new, more effective TB vaccines available globally.

In this guest post, Claire Wingfield—product development policy officer at PATH—writes about a new paper she developed for the GHTC that outlines key issues surrounding access to global health products. In order to achieve impact, a health technology must be more than just safe and effective. It must also be affordable, available, and acceptable to ensure that it is accessible to those who need it most. The pipeline of products addressing public health needs in low- and middle-income countries (LMICs) has grown substantially over the past two decades. Much of that success can be traced back to the 1990s when a number of nonprofit product development organizations (NPPDs) were established to accelerate the development and accessibility of new health technologies targeting poverty-related diseases and conditions. Since that time, NPPDs and their partners have contributed to the development, evaluation, and/or introduction of 42 new health products. The GHTC has launched a new paper that outlines key issues surrounding access to global health products. Photo credit: PATH Because NPPDs were established to develop—not deliver—health technologies, they negotiate collaborations with partners from the public and private sectors that are designed to ensure access to the resulting products and knowledge. Today, the GHTC is launching a paper that explores how NPPDs approach access and how they manage partnerships and negotiate agreements to improve access – which they define as the affordability, availability, and acceptability of technologies targeting the health needs of LMICs. Although there is no “one size fits all” model for how NPPDs operate, they do employ some common strategies to ensure that the resulting products benefit those most in need: Spreading investment in research and development across a portfolio of technologies, thereby allowing NPPDs to select only the most promising products to advance through the pipeline. Prioritizing access to these technologies for poor populations. Planning for access from the beginning of the product development process. Working closely with manufacturing partners to guarantee that consistent supplies of high-quality, cost-effective products are available to target populations. Engaging end users and beneficiaries in the design and the development of the product. Negotiating collaborations with partners from the public and private sectors that create a framework for access in LMICs. NPPDs also shared lessons that they and their partners have learned in trying to improve the affordability, availability, and acceptability of new health technologies for those most in need: Defining the value of a technology must be driven by the local communities and countries that will use the product. Product developers must understand and address the needs and wants of those who will ultimately be implementing and benefiting from the product. Achieving global access does not guarantee local access. NPPDs and partners may achieve global access targets (such as receiving World Health Organization prequalification) but this does not guarantee that the technology will be accessible at the national or subnational level. Relying on national average income status can undermine access for the poorest populations. In many middle-income countries, the burden of disease is among poorer populations who have not benefitted from strengthening economies. Therefore, the poorest populations, often the most at risk, are unable to access new technologies. Securing donor recognition that access activities need to be initiated early is critical. In order to ensure there is not a lag between licensing a product and making it available in the health system, NPPDs and partners must start planning for access from the beginning of the development process. Support from donors for these activities is critical. Demonstrating a niche in the market for manufacturers is essential to incentivize their investment. Manufacturing partners must understand the value that they bring to a market to enable them to invest time, effort, and expense to developing products for poverty-related and neglected diseases and conditions. Any delays in planning and implementing an access strategy can result in significant lag time from licensure to actual delivery in country. A product that needs to be retrofitted for affordability, availability, and acceptability will result in a costly and time-consuming development process. Therefore, as the GHTC’s new paper reveals, it is critical that NPPDs and partners address these principles from the beginning of the product development process.