Claire Wingfield

  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 challenges and solutions facing research and development (R&D) for diseases of poverty. In April 2012, a World Health Organization (WHO) working group released a report outlining recommendations to improve the coordination and financing of R&D targeting the health needs of low- and middle-income countries (LMICs). The recommendations were hotly debated at last year’s World Health Assembly, but did not have enough support among WHO Member States to pass. A new resolution will be considered at this year’s World Health Assembly, being held this week in Geneva. In an effort to inform this and other global debates, the GHTC has released a paper providing new insights from nonprofit product development organizations (NPPDs) working to advance R&D for poverty-related and neglected diseases and conditions. NPPDs are nongovernmental organizations that partner with the public, philanthropic, and private sectors to develop technologies—including diagnostics, drugs, devices, vaccines, and microbicides. NPPDs were established to accelerate R&D and improve access to products that would not be available otherwise for commercial reasons. This paper provides examples of how NPPDs approach product development for diseases and conditions prevalent in LMICs, as well as outlines the key challenges they face. We surveyed a cross-sampling of NPPDs to capture their perspectives on the gaps in the current global pipeline, key challenges as technologies advance through the pipeline, and potential solutions to solving those challenges. The GHTC's new paper finds that NPPDs play a vital role in bringing together the fragmented resources and expertise of the various sectors involved in neglected disease R&D. Credit: PATH/Evelyn Hockstein We found that NPPDs play a vital role in bringing together the fragmented resources and expertise of the various sectors involved in neglected disease R&D—not only by developing and delivering health products that would not be available otherwise, but also by improving scientific understanding for many of these conditions. For example, the surveyed NPPDs reported contributing to the development of more than 450 technologies across neglected and infectious diseases, as well as maternal, newborn, and child health, and sexual and reproductive health conditions. Despite this progress, NPPDs cited several significant challenges that hinder their work. Not surprisingly, funding was the most commonly cited challenge. NPPDs noted that there are too few funders investing too few resources in R&D for neglected diseases and poverty-related conditions. As a result of a relatively small number of funders, shifts by donors can have significant effects. For example, a cut of just 10 percent by the National Institute of Health—the largest funder of R&D for LMICs—would have a disproportionately large impact on the overall funding picture. Another main challenge is a lack of consistent regulatory requirements and limited capacities of national regulatory authorities, which NPPDs cited as major contributors to delays and increased costs. NPPDs noted that each delay increases the complexity, duration, and cost of conducting research targeting the health needs of LMICs. For instance, PATH Malaria Vaccine Initiative had to clear 40 independent review boards—many reviewing the same information against different standards—in order to initiate clinical trials of one of its malaria vaccine candidates. NPPDs noted that limited local research and manufacturing capacity constrain their and their partners’ ability to keep the pipeline filled and bring new and improved technologies to patients. Many local research sites have not conducted studies that meet international standards. Manufacturing partners in many of these countries lack the experience producing products that meet standards necessary to secure approval from WHO and international procurement agencies, such as the GAVI Alliance and United Nations Children’s Fund . This means NPPDs and their partners must invest in strengthening local research and manufacturing. As just one example, the International Partnership for Microbicides established its South Africa office to enhance local research in communities with high HIV prevalence, and as a result, 17 research centers in seven African countries are equipped to conduct clinical trials in line with international guidelines. Fortunately, NPPDs also offered the following solutions, which they cited as necessary to enabling the successful development and delivery of new health tools for LMICs: Sustainable and consistent funding. More predictable, stable, and long-term funding across all phases of product development is needed, as well as diversified funding sources and improved coordination across funders. Better coordination could be achieved through a centralized mechanism tasked with identifying R&D needs, gaps, and priorities. Capacity building for local partners. NPPDs and their partners need sufficient resources to invest in strengthening their local partners’ research and manufacturing capacities. Improving regulatory pathways and capacities. NPPDs noted that policy changes and adequate, sustainable funding are needed to support capacity building and alignment of regulatory requirements. This paper is the first in a series from the GHTC that will examine financing, accessibility, regulatory, and capacity issues facing NPPDs. This first paper provides a high-level overview of the greatest gaps and challenges faced by NPPDs, and further papers will explore these issues in greater detail. The paper is available here on the GHTC’s website.

In this guest post, Claire Wingfield—product development policy officer at PATH—writes about a new paper that examines partnerships between the public and private sectors to advance global health innovation, research, and development. For more than 30 years, PATH has been working to advance innovative global health solutions. Partnering is a core part of PATH’s strategy and is fundamental to how we develop and deliver high-impact, cost-effective technologies. Our partners come from international health agencies, governments, universities, other nongovernmental organizations (NGOs), and private-sector companies. Each partnership is unique, but no matter who the partner is, the collaboration must be mutually beneficial and fulfill our mission to improve the health of people around the world. Public-private partnerships provide a forum for maximizing complementary areas of expertise. They improve knowledge of the most pressing global health needs and lead to solutions that improve health equity. By strengthening the capacity of different sectors, these partnerships accelerate the development and introduction of the most promising technologies for those most in need. Finally, forging new cross-sector partnerships with public institutions and commercial entities enable organizations like PATH to maximize the limited global health resources of both sectors—financial, technical, and structural—to reduce costs and avoid redundancy in our efforts to ensure the affordability of much-needed global health technologies. For instance, in many low-income countries, where budget resources are stretched thin, health systems may not be able to ensure the safety of health tools and products. This leads to situations where the very injections meant to prevent or treat disease can end up causing it instead because under-resourced, overburdened health care programs reuse or fail to properly sterilize contaminated injection equipment. Recognizing the tremendous negative impact that unsafe injection practices can cause, PATH researched how to improve injection practices to reduce the risk of transmitting bloodborne diseases such as HIV and hepatitis B in low-resource settings. The US government, through the US Agency for International Development, provided funding to PATH, enabling us to develop and patent a design for an auto disable (AD) syringe that automatically locks after a single injection. A partnership between PATH, the US government, and Becton, Dickinson and Company led to the development of SoloShot™, the world’s first commercially available auto-disable syringe. Credit: PATH/Eric Becker While PATH had the scientific and technical know-how to design the technology, a commercial partner was needed to validate the design, support large-scale production, and enable widespread distribution. PATH licensed the technology to Becton, Dickinson and Company (BD), the world’s largest syringe manufacturer. BD brought product development expertise to advance the technology beyond proof of concept, manufacturing capabilities to produce a global supply, and distribution channels to ensure the availability of the technology in the public and private sectors. The US government’s investment in the product’s development—harnessed many times over by BD’s own investment in the device—enabled PATH and BD to pursue an aggressive distribution program that led to SoloShot™, the world’s first commercially available AD syringe. A major boost to the introduction and use of AD syringes occurred when the United Nations Children’s Fund and the GAVI Alliance began purchasing and distributing AD syringes for their programs. The US government also accelerated the adoption of the technology by aggregating demandthrough the US President’s Emergency Plan for AIDS Relief (PEPFAR) program. Because injection safety was one of PEPFAR’s top priorities, the US government procured more than 250 million AD syringes for 11 countries through the Making Medical Injections Safer project over its five-year life. Since 1992, more than six billion vaccinations have been delivered using SoloShot™ in more than 40 countries. Studies have shown that transmission of bloodborne diseases caused by dirty needles has been reduced by 90 percent in programs using some form of AD syringe. Creating partnerships that harness the assets of the private sector and the public sector together is a powerful means to ensure impact, scale, and sustainability for the world’s most vulnerable populations. The partnerships help align the strengths and requirements of each sector to ensure that appropriate technologies and innovations reach communities and individuals with the greatest need.

Claire Wingfield is a Product Development Policy Officer with PATH. This week, she’s blogging from the World Health Summit in Berlin, Germany. The third annual World Health Summit is currently underway in Berlin. The Summit brings together researchers, policymakers, funders, advocates, and health care providers to discuss global public health policy and research. This year’s theme is “Today’s Science – Tomorrow’s Agenda,” but the hot topic in almost every session that I have attended so far has been the need for innovative and sustainable funding mechanisms for global health in this time of financial uncertainty. Incentive prizes were highlighted as a mechanism for encouraging investment in research and development (R&D) of neglected and orphan diseases. These results-based rewards (as opposed to recognition awards like the Nobel Prize) “pull” innovation by encouraging applicants to submit ideas. The models and types of prizes vary. Some may offer millions of dollars while others provide the winner with access to laboratory services for future research. The first incentive prize, sponsored by the British Parliament in 1714, resulted in breakthroughs in navigation with the winner getting a whopping £100,000. The prize model often attracts new ideas and people to a field and is results focused which is attractive in an environment where funders want to see “quick wins.” Product development partnerships (PDPs) have also been highlighted at the summit as a successful model for accelerating R&D. Regina Rabinovitch of the Bill & Melinda Gates Foundation attributed the PDP model with feeding the largest research pipeline for neglected disease tools, increasing collaboration across the public and private sectors, and promoting a better understanding of the complexity of the product development process. While the successes are promising, she warned that as the number of products moving through the pipeline grows, so do the costs. Each phase of clinical studies requires more volunteers, more data points, more demands on the research site, longer trials, and increased regulatory complexity. Given these challenges in a constrained financial environment, PDPs and other research networks must increase collaboration and share costs by doing things like site sharing and harmonized training. They also need to focus their research efforts by prioritizing the products in the pipeline and weighing their potential public health impact. Bernard Pécoul, Executive Director of the Drugs for Neglected Diseases initiative (DNDi), stressed that it is critical for public sector partners to set objectives and priorities to ensure that product development is driven by health needs. Representatives from pharmaceutical companies—Novartis and Sanofi in the sessions that I attended—acknowledged that there are inherent difficulties with trying to reconcile limited profit potential with great public health need. There are many differing views on how to address this challenge with no clear consensus. One suggestion that seemed to be well received was that pharmaceutical companies can offer untapped areas of expertise, specifically in regulatory science, manufacturing, distribution, and pharmacovigilance. These are critical components of the R&D process, and many academic and public sector research institutions would benefit from training and mentoring in these areas. Recognizing that there is no magic bullet and that no one organizational or funding model can address the R&D needs for all neglected diseases, it is clear that limited resources must be leveraged, communication and coordination among research institutions across sectors needs to be increased, funding sources must be diversified, and current levels of support need to be sustained in order to maintain the recent momentum in global health research.