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GHTC submitted the below response to a request for information (RFI) on proposed changes to reporting outcomes from US National Institutes of Health (NIH) Peer Review, urging the agency to maintain a rigorous, transparent, and effective peer review process through any reforms to the process.The Global Health Technologies Coalition (GHTC), a coalition of more than 50 nonprofit organizations, academic institutions, and aligned businesses working to accelerate the development and delivery of new drugs, vaccines, diagnostics, and other health technologies, is committed to ensuring the NIH sustains a strong, predictable environment for scientific discovery to advance global health research and development (R&D). GHTC supports NIH’s goal of ensuring funding decisions reflect the full substance of scientific peer review. If NIH moves toward broader categories for reporting review outcomes, it should ensure that qualitative feedback is sufficiently clear and actionable to help researchers understand the scientific strengths and weaknesses identified through peer review and, where appropriate, improve future applications.  Peer review enables global health R&D NIH’s investments in early-stage biomedical research are critical for the global health R&D ecosystem. NIH support has helped advance more than 45 new global health technologies since 1999, including Lenacapavir for long-acting HIV prevention, monoclonal antibodies to treat Ebola, and new drugs to combat drug-resistant tuberculosis. The science leading to each of these transformative innovations moved through the NIH peer review process. For global health R&D, where promising discoveries often move through long and interconnected development pathways, GHTC’s primary interest is ensuring that NIH maintains a rigorous, predictable, and scientifically independent research environment that enables strong ideas to advance and researchers to continue improving the science. Transparency for researchers As NIH simultaneously implements a more flexible approach to funding decisions under the Unified Funding Strategy, transparency regarding how scientific merit is considered alongside programmatic priorities, portfolio needs, workforce considerations, and available resources will be important. Clear and consistently applied processes can help preserve confidence in the independence of scientific review while giving NIH appropriate flexibility to steward its research portfolio.  Researchers should be able to understand the substantive scientific feedback on their applications and use that feedback to inform future research and applications. Historically, scores, percentiles, and paylines have provided researchers with useful reference points in the review and funding process. If NIH moves away from this traditional system, the qualitative feedback provided through peer review becomes even more important. Written critiques and panel discussion summaries should provide researchers with sufficient information to understand the scientific strengths and weaknesses identified during review and assess whether and how to revise and resubmit an application.  Maintain scientific independence  Researchers and institutions have experienced significant changes to NIH funding policies, priorities, and grantmaking practices over the past year. Additional reforms should be implemented in a measured and thoughtful manner and clearly communicated to stakeholders to minimize unnecessary disruption to the research ecosystem. As the peer review and funding processes evolve, NIH should continue to provide clear safeguards and transparency regarding the respective roles of scientific peer review and programmatic considerations in final funding decisions. Preserving confidence in the independence and integrity of peer review is essential to maintaining researcher participation and ensuring that NIH investments continue to support high-quality science. A rigorous, transparent, and effective peer review process will help ensure that NIH resources continue to support scientific discovery and that today’s research can become tomorrow’s health technologies. 

Recently published results from a large multi-country study found that pooled tuberculosis (TB) testing could greatly reduce costs and boost access to diagnosis. Access to the gold standard of rapid molecular testing is limited—only just over half of people diagnosed with TB in 2024 were first tested using the technology. Instead, they were diagnosed using older technology, which can take weeks to produce results, during which the infection could progress or be transmitted to others. Millions of additional individuals with TB go completely undiagnosed. A major contributing challenge is that rapid molecular testing requires an expensive diagnostic cartridge for each sample. Pooled testing involves testing samples from multiple individuals with one cartridge. Patients are only retested individually if the result is positive. Unlike new diagnostic technologies, like AI-enabled portable X-ray machines, that are promising but would require additional funding to implement, pooled testing could be scaled up rapidly without new funding because it utilizes technologies that many labs already have.Medicines for Malaria Venture (MMV) is advancing the novel, first-in-class antimalarial compound, cabamiquine, into the next stage of clinical development, with the hope that the compound could be part of a single-dose, long-acting treatment for the prevention and treatment of malaria, which could greatly simplify care compared to current options. Cabamiquine was discovered by researchers at the University of Dundee, with financial and scientific support from MMV, and then advanced by Merck to proof of concept. Recently published results from two Phase 2a trials showed that a regimen of cabamiquine and pyronaridine was able to clear uncomplicated Plasmodium falciparum malaria in more than 90 percent of participants and demonstrated a strong safety profile. MMV will now lead Phase 2b clinical development of the compound. The Biomedical Advanced Research and Development Partnership (BARDA) recently announced that it has awarded Scynexis a $214 million contract for the development of an antifungal to treat and prevent invasive fungal infections, the less common but more serious form of fungal infection that penetrates deep into the tissues of the body. Fungal infections are a growing global challenge, but there is a significant gap in the pipeline of new tools to address them. Last year, the company reported positive findings from a Phase 1 trial of an oral dose of the candidate and is currently conducting a Phase 1 trial of an intravenous formulation. The award includes initial funding to advance the company’s antifungal candidate into Phase 2 trial, with an option for additional, longer-term funding. 

Recently published results from a Phase 2b trial testing a new regimen for people with chronic Trypanosoma cruzi infection could offer a safer, more practical treatment option for the infection, which affects more than seven million people globally, mainly in Latin America, and can develop into Chagas disease. Compared to the current standard regimen that involves twice-a-day treatment for 60 days and is frequently accompanied by uncomfortable side effects, leading to treatment failure, the new regimen involves a 30-day course taken once daily. Trial participants treated with the shorter regimen had significantly reduced side effects, found it easier to follow, and saw lower treatment interruption. If it continues to demonstrate success in large Phase 3 trials, the regimen could transform treatment for patients globally, as T. cruzi infection becomes a growing challenge in the United States, Europe, and Japan.The first participants have been vaccinated in a trial in the Democratic Republic of the Congo (DRC), where the country’s ongoing outbreak of the Bundibugyo strain of Ebola has now become the largest Ebola outbreak in the country’s history and the second largest in history. Health workers in Bunia, a DRC city at the center of the outbreak, were the first to be vaccinated in the real-world efficacy trial evaluating whether the Ervebo vaccine, which is designed to target the more common Zaire strain, offers some cross-protection against Bundibugyo. There are no approved vaccines for Bundibugyo Ebola, and there were no candidates in late-stage development before the start of the outbreak of the rarer strain of the virus, which has only caused three outbreaks since it was first discovered around 20 years ago. Since the outbreak began, global partners have fast-tracked the development of earlier-stage candidates, but researchers hope the Ervebo vaccine could provide some protection before those candidates are ready for evaluation in DRC. As artificial intelligence (AI) continues to rapidly transform many aspects of technology and science, including health research, a new report from the World Health Organization (WHO) speaks to how stakeholders can help ensure AI-enabled health research is conducted responsibly and benefits everyone. According to the report, while AI has the potential to accelerate health innovation and improve health outcomes, it also presents risks related to equity, privacy, safety, transparency, and more. WHO suggests that researchers, ethics committees, regulators, funders, and policymakers can act to strengthen ethical oversight of AI throughout the health research continuum. WHO also notes that most AI development remains concentrated in high-income settings and contends that strengthening leadership, capacity, and participation in AI in low- and middle-income countries will be important to ensuring the technology does not exacerbate existing inequities in health and health research. 

Last week, Gilead Sciences announced that it has expanded the scope of its voluntary licensing agreements with six manufacturing partners, who are already manufacturing the recently approved twice-yearly lenacapavir for HIV prevention, to include investigational once-yearly lenacapavir. Following the unprecedented Phase 3 trials for twice-yearly lenacapavir, an ongoing Phase 3 trial is evaluating a once-yearly option that could provide a full year of prevention with just one annual dose, further expanding prevention options and addressing persistent barriers to uptake and adherence. Establishing these agreements at this early stage helps manufacturers, regulators, health systems, and communities prepare for the broad, rapid, and equitable scale-up of the once-yearly option, if it is eventually approved. The World Health Organization (WHO) has updated its malaria guidelines to include Coartem® Baby, the first antimalarial developed specifically for newborns and young infants weighing between two and five kilograms. This patient group previously had no medicine specifically designed for their unique needs, leaving them to be treated with modified doses of formulations intended for older children, raising the risk of treatment failure or toxicity. The drug was developed by Novartis and partner Medicines for Malaria Venture (MMV), with financial support from the European & Developing Countries Clinical Trials Partnership and the Swedish International Development Cooperation Agency. WHO’s inclusion of Coartem® Baby in the guidelines informs and enables malaria-endemic countries to introduce the treatment in national malaria programs, supporting broader access. After receiving prequalification by WHO and approval from Swiss and Ghanian regulatory authorities, Coartem® Baby has so far been rolled out in 11 countries. The Coalition for Epidemic Preparedness Innovations (CEPI) recently announced that it is awarding $3.9 million to support research projects aimed at improving understanding of the Bundibugyo ebolavirus behind the ongoing outbreak in the Democratic Republic of the Congo. Specifically, the funding will support modelling and analytic teams led by the Institut National de Recherche Biomédicale, Imperial College London, and Makerere University School of Public Health. These teams will collect, integrate, and analyze outbreak data to guide the outbreak response; inform the design of fast, efficient vaccine clinical trials; and support longer-term preparedness against similar outbreaks in the region. To ensure long-term applicability, the partners have placed a strong emphasis on capacity strengthening and open sharing of data and methods, so that the tools and knowledge these projects generate can be utilized in future outbreaks in the region.