Breakthroughs Blog: WHO

Few diseases have shaped global health like the HIV/AIDS pandemic. Since it began, more than 40 million lives have been lost, leaving an enduring impact on families, communities, and health systems worldwide. But HIV has also become one of the clearest examples of what sustained research and development (R&D) can achieve. From antiretroviral therapies (ART) to long-acting prevention tools, decades of scientific innovation have fundamentally changed what is possible in the fight against HIV.This year, HIV innovation stands at a pivotal moment. Shifting funding landscapes and evolving global health priorities have raised important questions about how R&D for HIV will be sustained in the years ahead. As global leaders have reaffirmed the importance of continued R&D investment—such as through the recently adopted United Nations High Level Meeting political declaration on HIV/AIDS—we must also focus on the systems and programs that translate scientific breakthroughs into impact. Few advances better illustrate this balance than lenacapavir, or LEN, an injectable form of HIV prevention requiring only two injections per year, spaced 6 months apart. Unlike daily oral pre-exposure prophylaxis (PrEP), which depends on consistent adherence, LEN offers protection by eliminating daily pill burden for consumers and communities vulnerable to HIV.The journey of LEN reflects the long arc of HIV innovation. Decades of research supported by the US National Institutes of Health helped establish the HIV capsid as a promising drug target, creating the foundation for a new class of HIV medicines. Then, in 2006, researchers at Gilead Sciences began developing capsid inhibitors designed to suppress HIV replication while offering the potential for long-acting treatment. At the time, ART had transformed HIV from a fatal disease into a manageable chronic condition for millions of people, but treatment relied on daily medication, creating ongoing challenges for adherence and access.Following years of research and clinical development, the US Food and Drug Administration (FDA) approved LEN in 2022 as a treatment for adults living with multidrug-resistant HIV whose existing treatment regimens were no longer effective. While this approval marked an important milestone, researchers at Gilead continued exploring the drug's potential as a long-acting option for HIV prevention and found remarkable resultsThe first pivotal late-stage prevention trial of the drug, called PURPOSE 1, conducted among women in South Africa and Uganda—including pregnant and lactating women—found that LEN provided near-complete protection against HIV infection and outperformed daily oral PrEP. PURPOSE 2, a subsequent study, conducted across seven countries and including men and gender-diverse people, confirmed similarly high levels of protection in late 2024. Beyond their clinical success, these studies also represented an important step toward more inclusive HIV research by intentionally enrolling populations that have been historically underrepresented in clinical trials.  Just six months after Gilead submitted LEN for review as an HIV prevention tool, the FDA approved the drug in June 2025 under a priority review designation pathway. Because regulators were able to rely on the extensive safety data generated during the drug's earlier approval for HIV treatment, they could focus their review on its use as a novel preventive intervention. The result was an efficient regulatory process that maintained rigorous standards while accelerating future access to an important new prevention tool. Swift regulatory action extended beyond the United States. In October 2025, the World Health Organization (WHO) prequalified LEN in just 36 days using its abridged prequalification pathway. This expedited process enabled WHO to leverage assessments already completed by the European Medicines Agency’s (EMA) European Union-Medicines for all program, which provides scientific opinions on medicines for use in low- and middle-income countries, while conducting its own review focused on issues relevant to the global procurement and implementation of LEN.  This rapid review mattered because WHO prequalification serves as a critical gateway for procurement by many international organizations and countries, particularly in low- and middle-income countries. Accelerating this process helps ensure that countries are not waiting months or years for access after a product has already demonstrated its safety and effectiveness through other rigorous stringent regulatory approval processes.Accelerated regulatory approval and collaboration continued across multiple regions. Following FDA, EMA, and WHO approval, LEN rapidly advanced through regulatory processes in South Africa, Australia, Brazil, and Canada, and, as of today, it is approved by 17 regulatory bodies, including 12 countries in Africa. In several cases, regulators leveraged reliance mechanisms, such as WHO’s collaborative registration procedure, international collaboration, and priority review pathways to streamline review and accelerate access to this important breakthrough without compromising regulatory rigor. Swift regulatory action was soon matched by commitments from the United States; the Global Fund to Fight AIDS, Tuberculosis and Malaria; and Gilead to rapidly scale up access to LEN and accelerate generic production in high-burden countries.This coordinated approach, supported by political commitment and strategic public-private partnership, created an unusually efficient pathway from scientific discovery to implementation. Compared with earlier HIV prevention technologies, such as oral PrEP and the dapivirine vaginal ring, LEN has progressed through key milestones at a rapid pace. This allowed for accelerated product introduction of lifesaving technologies at the country level, while simultaneously shaping the market and demand for LEN and future prevention options. While every health technology faces unique scientific, regulatory, and access considerations, LEN demonstrates how thoughtful planning, regulatory preparedness, and collaboration between researchers, regulators, countries, multilateral organizations, and communities can significantly shorten the time between discovery and access.As conversations about the future of HIV innovation continue, LEN offers an important blueprint for global health innovation more broadly. It reminds us that sustained investment in R&D produces transformative breakthroughs, inclusive clinical trials generate stronger science, and innovative regulatory pathways can accelerate, rather than compromise, rigorous review. Moreover, the accelerated path from research to rollout illustrates the coordination and collaboration required to advance technologies through regulatory approval while simultaneously shaping markets to enable introduction at scale. The lessons from LEN are already informing the next generation of HIV prevention innovations, with developers pursuing earlier access and licensing strategies for products such as Merck's MK-8527. While the US government, the Global Fund, and Gilead have recently expanded commitments to scale up LEN, the ultimate measure of success will be whether LEN—and the innovations that follow it—reach the people who need them most.  The future of innovations for HIV, as well as tuberculosis, malaria, emerging threats with pandemic potential, and beyond, will require the same integrated approach. Scientific discovery, product development, efficient regulatory pathways, and equitable access cannot be treated as separate objectives; they are mutually reinforcing components. Innovation succeeds not when a product is approved, but when it reaches the communities whose lives it has the power to change. LEN has shown us what’s possible when the entire R&D ecosystem works together. The challenge now is ensuring that this success becomes the standard rather than the exception.

Interim study findings that will be presented at this week’s AIDS 2026 conference show that a new, once-weekly antiretroviral combination pill for HIV seems to be effective and well tolerated, with comparable results to currently available daily pills. The findings are from two ongoing large, multi-country Phase 3 trials comparing the new combination pill to current treatment options. The new combination treatment has the potential to greatly simplify HIV treatment for patients and health care providers by reducing treatment frequency—and potentially cost—which could help improve access and adherence. A new study in humans suggests that Merck’s Ervebo vaccine, which is licensed for the Zaire strain of ebolavirus, could offer some protection against the Bundibugyo strain of ebolavirus, fueling a growing number of calls to evaluate Ervebo in a clinical trial in the Democratic Republic of the Congo (DRC), the site of the rapidly expanding outbreak of Bundibugyo, for which there are no licensed vaccines. The World Health Organization has so far prioritized evaluation of Bundibugyo-specific vaccines, but the few in development are still months away from clinical trials in the DRC. A trial of Ervebo could provide some protection to people at risk in the outbreak sooner, since there is an international stockpile of Ervebo doses. The Africa Centres for Disease Control and Prevention is planning a trial that would test a two-dose vaccine regimen of Ervebo and an experimental vaccine targeting Sudan Ebola in health workers. Last week, IAVI and Biofabri announced that their Phase 2b clinical trial of tuberculosis (TB) vaccine candidate MTBVAC has reached full enrollment. The trial is evaluating the safety and efficacy of MTBVAC in preventing active TB disease in adolescents and adults with latent TB infection, as well as an additional cohort of participants who have never been exposed to TB infection, across 16 clinical research centers in South Africa, Kenya, and Tanzania. The current TB vaccine is more than 100 years old and provides incomplete protection in young children and does not prevent TB disease in adolescents and adults. MTBVAC, which is single-dose and well-suited for use in remote locations, could be a game-changer for TB control. 

On Thursday, the World Health Organization (WHO) announced the first patient enrollment in a critical clinical trial evaluating investigational antiviral therapies to treat Ebola caused by the Bundibugyo virus, as the worrying outbreak in the Democratic Republic of the Congo continues to grow. There are currently no approved treatments specifically for the Bundibugyo strain. The trial is supported by a range of multilateral, regional, national, and scientific partners and is being coordinated by the Institut National de Recherche Biomédicale, the Institute of Tropical Medicine and the University of Oxford. Two therapies, the monoclonal antibody MBP134 and remdesivir, which were selected by WHO technical advisors, are being evaluated for their ability to improve survival among patients, with the goal of saving lives and helping curb the spread of the current outbreak, as well as generating evidence to improve care for future outbreaks. A comprehensive review of COVID-19 mRNA vaccines published last week in The Lancet found that they are safe and effective, as skepticism around the safety and trustworthiness of the technology has persisted in recent years, while new research efforts have demonstrated the technology’s promise to address a range of priority disease and health challenges, including influenza and cancer. The researchers found that serious events are rare and the odds of them occurring were outweighed by significant protection against severe disease, hospitalization, and death. The researchers do acknowledge challenges with mRNA technology, including that they mRNA vaccines are not as effective at providing long-last immunity as they are at generating short-term responses and they have significant, expensive storage resource requirements, challenging access in low-resource areas. Sustained innovation and a commitment to improving access could help address these shortcomings and ensure the promise of mRNA technology is fully realized around the world.Early results from a new study published in Nature last week found that an investigational HIV vaccine demonstrated unprecedented success in primates in a preclinical trial. The vaccine, which was developed through a prolonged collaboration between scientists at La Jolla Institute for Immunology and Scripps Research, works by prompting the immune system to produce high numbers of broadly neutralizing antibodies, which are capable of neutralizing a wide range of strains of the virus and are recognized as a promising approach in the HIV vaccine development field. One of the challenges of developing a vaccine for HIV has been the genetic diversity of the virus, with various distinct strains circulating. People living with HIV naturally develop broadly neutralizing antibodies that can neutralize various strains but designing a vaccine that can induce them has proven to be a challenge for researchers. 

Every May for the last 79 years, member states of the World Health Organization (WHO) have gathered in Geneva, Switzerland for the annual World Health Assembly (WHA). The Assembly remains one of the most important moments on the global health calendar, offering insight into how countries are responding to emerging health challenges and where political momentum is building around the future of global health governance, financing, and innovation. This year, however, Geneva felt markedly different. Conversations throughout the week reflected an ecosystem in transition. The global health community is grappling with constrained resources and evolving geopolitical priorities. Meanwhile, WHO reform efforts, persistent budget challenges, and upcoming leadership transitions across multilateral institutions contributed to a broader sense that global health is entering a new phase. Adding to this uncertainty, the United States did not formally participate in assembly proceedings for the first time in WHO's history following its withdrawal from the organization earlier this year.  The assembly also convened amid an evolving Ebola emergency and recent hantavirus outbreak, grounding at times abstract debates about preparedness, coordination, and health security in the realities of active disease outbreaks affecting communities around the world. Against this backdrop, WHA served not only as a decision-making forum but also as a barometer of where global health is headed. For GHTC, three high-level themes emerged that will help shape our advocacy in the months ahead:  1. Innovation must be matched by scale, delivery, and sustainable financing.  Throughout the week, one message surfaced consistently across discussions with WHO leaders, product developers, donors, and civil society organizations: scientific breakthroughs are essential to improving health outcomes, but their impact depends on whether new tools can be produced, financed, delivered, and accessed by the communities that need them most. Today, the challenge is twofold. Continued investment in R&D remains critical to delivering the next generation of vaccines, diagnostics, and therapeutics, particularly for diseases and conditions where significant gaps persist. At the same time, many effective technologies already exist but are not reaching those who need them. Addressing both realities will require increased financing across the R&D to delivery continuum, accelerated regulatory pathways, increased local manufacturing capacity, and more responsive procurement and delivery systems to ensure that innovations are both advanced and translated into impact.  This dual imperative carries particular urgency amid ongoing uncertainty in global health financing. As donors reassess priorities and international institutions face budget constraints, important questions remain about who will finance the next generation of health innovations and ensure their deployment at scale.  Discussions throughout WHA highlighted growing concerns that funding across the entire innovation continuum, from R&D to product introduction, market shaping, and implementation, may not keep pace with scientific advances and need, widening the gap between what is possible and what is accessible. 2. Country ownership is operational, not aspirational.  Perhaps the most significant shift visible throughout Geneva was the growing expectation that countries and regions will increasingly drive and implement their own health priorities.This was particularly evident in discussions marking the first anniversary of the Global Coalition for Local and Regional Production, Innovation, and Equitable Access. Representatives from France, the United Kingdom, the European Union, Indonesia, South Africa, and the African Union repeatedly emphasized the coalition's goal of enabling countries—particularly in the Global South—to become producers, developers, and innovators of health technologies. This conversation reflected a fundamental evolution in how many stakeholders view health security and access: local and regional production is no longer seen primarily as a pandemic preparedness objective but rather as a cornerstone of resilient health systems, economic development, and long-term sustainability. At the same time, discussions throughout the week underscored that manufacturing alone is insufficient; strong regulatory systems are equally critical to ensuring that health products reach communities efficiently and safely. This was reinforced by WHO's newly announced partnership with the African Medicines Agency (AMA) aimed at strengthening regulatory harmonization and reliance, improving safety surveillance, building regulatory capacity, and supporting innovation and local manufacturing across the continent. Similar themes also emerged from GHTC's dialogue with WHO's Prequalification (PQ) team. WHO officials described PQ as an indispensable but ultimately transitional program. The organization's long-term vision is for mature national and regional regulatory authorities to reduce reliance on PQ over time. For that vision to become reality, institutions like AMA and other regional regulatory bodies must develop the technical capacity, regulatory maturity, and trust needed to independently oversee health products.3. AI and emerging technologies are already being deployed.  During WHA week, it became clear that artificial intelligence (AI) and other emerging technologies are no longer hypothetical future solutions, they are already being integrated into health systems and product development. The focus has shifted from how they could be used to how they can be deployed and governed more effectively.  For instance, within WHO's PQ program, the PQ team highlighted the growing use of digital tools and AI-enabled systems to improve efficiency and transparency. Earlier this year, WHO launched its electronic prequalification submission portal, now the mandatory application submission pathway for manufacturers. Looking ahead, the PQ team is exploring how AI tools can streamline product reviews and reduce administrative burdens. Beyond regulatory systems, member states and stakeholders debated WHO's broader role in shaping governance frameworks for AI and digital health as part of the official WHA agenda. While many emphasized AI's potential to boost productivity, discussions consistently returned to concerns of how the organization will navigate challenges in transparency, accountability, data privacy, and public trust.Another major recurring concern was the risk that technological advances could deepen existing disparities if lower-resource countries lack the infrastructure, expertise, or regulatory frameworks necessary to benefit from them. As AI becomes increasingly integrated into global health R&D and delivery, ensuring that these technologies narrow, rather than widen, health gaps will be an increasingly important challenge.Carrying these lessons forward WHA79 underscored that global health is entering a new chapter, one defined by financial constraints, shifting political dynamics, evolving models of partnership, and rapid technological change. Yet amid this uncertainty, one message remained clear: the pursuit of equitable access to health innovation is both a global priority and a shared responsibility. As a coalition, GHTC will carry three priorities forward from Geneva: 1. We will continue advocating for innovation across the full product lifecycle. Investments in research, development, and access pathways must occur in tandem, so new technologies can reach the communities they are intended to serve at scale. For GHTC, this reinforces the importance of maintaining a diverse ecosystem of product developers, manufacturers, implementers, procurement agencies, regulators, civil society organizations, and community leaders to transform scientific advances into public health gains.  2. We recognize the growing emphasis on country ownership and the value of local leadership. As governments increasingly prioritize local production and health sovereignty, GHTC will work to ensure that R&D is a core pillar of these efforts through our Global Partners Network, dialogues with WHO, and engagement in global forums.  3. We will advocate for a future in which all countries have the capacity to contribute to and see their people benefit from the development of lifesaving technologies. Emerging technologies hold tremendous promise for advancing R&D against global health threats. Realizing that promise, however, will require intentional efforts to accelerate R&D, strengthen local capabilities, expand access, build trust, and promote equitable governance. GHTC will support policies that bolster innovation ecosystems worldwide, not only in countries with the greatest resources.The conversations in Geneva made clear that the future of global health will be shaped by parallel progress in innovation and access—driving new breakthroughs while strengthening the systems and local leadership needed to deliver them equitably at scale to the people who need them most.