Breakthroughs Blog: Access

Interested in more global health innovation news? Every week GHTC scours media reports worldwide to deliver essential global health R&D news and content to your inbox. Sign up now to receive our weekly R&D News Roundup email. Researchers at Penn State have discovered a new storage technique that can keep protein-based drugs and vaccines stable without requiring a cold chain. This method could make these products easier and cheaper to store and distribute, enabling greater access and generating cost savings. Currently, most biologic drugs and vaccines require temperature-controlled conditions throughout the supply chain, from production to administration to patients. Products preserved with this new method, which involves an oil-based solution and a molecule to coat the surface of the protein, were found in mice to be just as effective as refrigerated versions, and there were no signs of adverse health effects. The researchers are planning to test their method with additional proteins and are also working to secure patent rights and partner with pharmaceutical companies.The first patients have been enrolled in a pivotal clinical trial, supported by the Africa Centres for Disease Control and Prevention (Africa-CDC), the Institut National de la Recherche Biomédicale, and PANTHER, testing promising mpox therapeutic options. Mpox continues to pose a significant global health threat with no approved therapeutics specifically for the virus. The first drug being tested is brincidofovir, an antiviral from Emergent BioSolutions. The trial, which aims to bolster our ability to respond to mpox, also demonstrates Africa’s proactive role in partnering to tackle this ongoing challenge that has caused numerous outbreaks across the continent.The Global Antibiotic Research and Development Partnership (GARDP) and Debiopharm have signed a memorandum of understanding to develop Debio 1453, a novel compound representing a new antibiotic class that targets the Neisseria gonorrhoeae bacteria, as multidrug-resistant strains of N. gonorrhoeae emerging around the world make infections harder to treat. The partnership aims to develop Debio 1453 for regulatory approval and scale-up. The compound, with ongoing support from the Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X), previously demonstrated efficacy in preclinical studies against N. gonorrhoeae strains resistant to ceftriaxone, the last available recommended treatment globally, and other multidrug- resistant strains.

In many ways, 2024 has been a year marked by change—the first new year since the official end of the COVID-19 pandemic and a record number of elections ushering in political transitions around the world. With the global spotlight shifting to antimicrobial resistance (AMR) and tuberculosis (TB) reclaiming the designation as the top infectious disease killer from COVID-19, it may seem, at first glance, that the world has moved on from the pandemic that dominated every aspect of life for the better part of four years. However, with a deeper look, it’s clear that the global community is still reacting to and learning from the pandemic—for better or worse.As the year comes to an end, GHTC is reflecting on the global health innovation news and policies that shaped 2024.1. New disease outbreaks arise around the world, spurring need for and progress in R&D.In 2024, disease outbreaks continued to dominate the headlines—from the first reports of avian flu in the United States in March to the rapid global spread of the clade 1b mpox strain over the summer to the outbreak of the deadly Marburg virus in Rwanda beginning in September. Each of these incidents exposed devastating gaps in our prevention and response toolkit and spurred important steps forward on research and development (R&D) and access.For example, in response to the worrisome spread of avian flu, both the World Health Organization (WHO) and the US government launched efforts to accelerate the development of mRNA vaccines that could be deployed in case of human-to-human transmission of the currently spreading strain of avian flu or redirected to target another form of pandemic influenza if needed. Additionally, following declarations from the Africa Centres for Disease Control and Prevention and WHO designating mpox a public health emergency this past August, WHO prequalified the first vaccine against mpox and approved the first mpox test for emergency use, with the goal of accelerating global access to these critically needed tools. The outbreak also provoked new research to develop improved vaccines against mpox and the broader range of orthopox viruses, with early studies of mRNA mpox vaccines in macaques and mice demonstrating promising results.This fall, as cases of Marburg were on the rise in Rwanda, and with no currently approved vaccines or treatments available, researchers fast-tracked efforts to evaluate tools to stem the current outbreak and curb future ones. Rwandan health authorities initiated an open label vaccine trial in health workers and others at heightened risk, and there has since been a randomized clinical trial arm launched, which will offer critical efficacy data. Additionally, scientists launched the first-ever clinical trial for a Marburg treatment this year.Beyond accelerating innovation against these outbreaks, 2024 also brought exciting research advancements targeting other pathogens scientists believe are most likely to cause the next pandemic, including trials that could lead to the first-ever approved vaccines for Sudan ebolavirus and Lassa fever, as well as further efforts to improve coronavirus vaccines and simplify and speed up vaccine development and manufacturing.2. Pandemic preparedness policy reforms march forward while post-COVID political backlash in United States grows.Multilateral efforts to strengthen the world’s pandemic preparedness and response architecture moved incrementally forward this year. At the World Health Assembly (WHA) in May, member states adopted several amendments to the International Health Regulations—an existing framework codifying international responsibilities during a pandemic—, including new commitments to solidarity and equity on financing and ensuring access to medical products. The World Bank-hosted Pandemic Fund, a financing mechanism launched in 2022 to help low- and middle-income countries strengthen their preparedness and response capacities, also raised $982 million in new contributions and an additional $1.8 billion in co-financing this year, as well as awarded $500 million in grants to countries to strengthen surveillance, laboratory, and workforce capacities and fast-tracked an additional $129 million for the mpox response. Under the Brazilian presidency, G20 nations established a voluntary coalition to strengthen local and regional production of health products, which, while focused foremost on neglected diseases, could also strengthen capacities critical for pandemic preparedness and response. Unfortunately, despite some progress on negotiations toward a new pandemic accord, countries failed to reach sufficient consensus before the end of this year, and negotiators are now pushing to wrap up by the next WHA in May of 2025.At the same time as some progress was achieved on the international front, in the United States, post-COVID political backlash has deepened and become more entrenched through congressional scrutiny and reform agendas targeting the National Institutes of Health (NIH) and the Centers for Disease Control and Prevention (CDC). Though the fiscal year 2025 budget is not expected to be finalized until next year, the health-related spending bill introduced by the House proposes spending cuts to CDC and a reorganization of NIH, which mirrors that put forward by House Republicans over the summer and could have serious implications for the agency’s core global health R&D capacities. Beyond appropriations, we also continued to see a plethora of congressional hearings targeting NIH and CDC on the origins of COVID-19, gain-of-function research, China’s influence, and the perception that these agencies are overstepping their mandates. The scrutiny and reform efforts we saw this year are expected to intensify in 2025, as Republicans gain control of both chambers of Congress and the White House.3. AMR captures the world’s attention as the global threat continues to grow.2024 was informally dubbed “the year of AMR,” with AMR elevated on the global agenda at several key moments. In May, a WHA resolution was adopted to accelerate national and global responses to AMR by advancing R&D, strengthening surveillance systems, and forging international partnerships. Later in September, a landmark report found that without further policy interventions, the death toll from AMR is expected to reach 39 million between 2025 and 2050—the equivalent of three deaths per minute.This shocking report set the scene for the United Nations High-Level Meeting on AMR in September, which led to the adoption of a political declaration that set the goal to reduce AMR deaths by 10 percent by 2030 and committed countries to promote the R&D of and equitable access to antimicrobials, vaccines, diagnostics, and other products. Global leaders also adopted an action plan in November, built off of the declaration, to provide actionable objectives and additional check-ins to monitor progress.This political momentum on AMR was also matched by exciting research breakthroughs that made the news this year. Researchers discovered a new compound that could potentially treat a resistant bacterial pathogen that can cause lethal hospital infections. Additionally, Phase 3 trials yielded promising results for both a therapeutic option for patients with serious bacterial infections and limited treatment options, as well as a new antibiotic that could cure uncomplicated gonorrhea infections, which could be approved sometime next year.4. TB reclaims title of deadliest infectious disease, but promising trials offer hope. This past year, TB surpassed COVID-19 to reclaim the spot as the world’s leading infectious disease killer, according to WHO’s Global Tuberculosis Report 2024. While this sobering news underscored major gaps in the tools needed to prevent, diagnose, and treat TB, especially drug-resistant TB, 2024 also brought exciting news on the research front that will hopefully help fill them.In March, a Phase 3 trial launched in South Africa, which is testing a TB vaccine candidate that could eventually become the first vaccine to prevent pulmonary TB in adolescents and adults. The only approved TB vaccine on the market, which was developed more than 100 years ago, is effective at preventing some types of TB in infants, but it offers inconsistent protection in adults against pulmonary TB. Additionally in November, the endTB consortium announced the completion of the first Phase 3 randomized controlled trial to exclusively enroll people with pre-extensively drug-resistant TB, a very hard-to-treat form of TB. Preliminary results demonstrated that a shorter, less toxic treatment strategy achieved high cure rates. Earlier in the year, WHO also recommended three new regimens for multidrug- or rifampicin-resistant TB, offering a much-needed variety of therapeutic alternatives for those with multidrug-resistant TB. 5. A major HIV breakthrough, but work remains on access. In what has been hailed as a breakthrough in HIV prevention, in 2024, exciting results were reported from a series of clinical trials for Gilead’s long-acting HIV prevention drug, lenacapavir, which found that the drug was 100 percent effective in preventing HIV in cisgender women and adolescent girls and 96 percent effective among cisgender men, transgender men and women, and gender nonbinary individuals who have sex with men. Both trials were so successful that they were unblinded early to offer the drug to all participants. Lenacapavir, which is administered via a twice-yearly injection, is currently approved as a treatment for multidrug-resistant HIV, but, if approved as a preventative regimen, the drug could be a game-changer for the HIV pandemic.Advocates applauded Gilead for its historic commitment to inclusion in these trials, while also urging that lenacapavir be made accessible and affordable as soon as possible to those that most need it. In October, Gilead announced a plan to allow generic versions of the drug to be sold in 120 countries, including all those with the highest HIV rates. However, the plan faced criticism for excluding most middle- and high-income countries that account for around 20 percent of new HIV infections. In these countries, the originator drug will only be available at higher prices, making it potentially inaccessible to at-risk populations.In other welcome HIV prevention news, in October, Population Council announced the results of a Phase 1 trial, which suggest that a three-month dapivirine vaginal ring for HIV prevention will be as effective as the currently approved one-month ring. The ring, which is self-inserted into the vagina and slowly releases an antiretroviral drug to prevent infection over three months, was found to deliver the drug at higher levels than the one-month ring. If the longer-acting ring continues to demonstrate success, it could offer a more cost-effective and convenient option for at-risk women. 6. Trump presidency and Republican trifecta portend changes to US leadership in global health.In the November election in the United States, former President Donald Trump won the presidency and Republicans gained control of both the US House of Representatives and the Senate. It was an across-the-board victory that will portend changes to US leadership on global health and medical research. If past is prologue, we’re likely to see President-elect Trump once again propose significant funding cuts to US global health programs and public health agencies like NIH and CDC, as well as withdraw the United States from WHO and other multilateral initiatives, the latter of which could derail efforts to finalize a pandemic accord. Given that the United States is the largest funder of global health programs and global health R&D efforts, these political changes could have a reverberating impact on health around the world.Since winning the presidency, President-elect Trump has also nominated several controversial picks to lead America’s public health agencies. These include Robert F. Kennedy Jr, a noted vaccine skeptic who has expressed a desire to cut the NIH workforce and refocus the agency on chronic diseases over infectious diseases, to lead the Department of Health and Human Services; Dr. Jay Bhattacharya, a health economist who criticized public health measures during the COVID-19 response and a vocal critic of NIH, to lead NIH; and Dr. Dave Weldon, a former Congressman who has questioned vaccine safety, to lead CDC.On the congressional front, with Republicans now in control of both chambers of the US Congress, as discussed, we’ll likely see continued scrutiny and reform efforts targeting NIH and CDC, as well as potential spending cuts to health and foreign assistance programs.7. New policies and innovative research approaches help reach underserved populations.Every person, everywhere, deserves to benefit from the fruits of science, and in this otherwise tumultuous year, there were several successful efforts to advance more inclusive clinical studies. In September, WHO released new guidance on clinical trial best practices outlining practical considerations for trials to include children and pregnant and lactating individuals, populations currently underrepresented in trials, leading to a lack of approved technologies for these groups. This guidance aligned with WHO’s living toolkit to facilitate the inclusion of pregnant and breastfeeding populations in clinical trials that was published earlier in the year. 2024 also brought the launch of two historic trials that aim to diversify research and thereby ensure that future technologies will serve the needs of underserved populations. This summer, a global consortium launched the first-ever Phase 3 clinical trial testing the efficacy and safety of antimalarials in women in their first trimester of pregnancy, which will hopefully not only lead to an urgently needed new treatment option for these women but also set a precedent for the inclusion of pregnant women in future clinical trials. Earlier this year, the US Administration for Strategic Preparedness and Response also announced funding to Walgreens to support a decentralized clinical trial testing COVID-19 vaccines. This unique approach will allow for a more diverse pool of study participants by bringing the study closer to where they live, ultimately ensuring that the resulting data reflects real-world use among a broader swath of the population. In the end, 2024 proved to be a complicated year, marked by both scientific and political steps forward and back. As new innovations and policies emerge in 2025 and beyond, GHTC will continue to work to ensure support for global health R&D remains a political priority and that the gains we have already made on neglected and emerging health challenges are not only sustained but also strengthened to combat an evolving health landscape.For other fascinating global health news delivered to your inbox weekly and to stay abreast of developments in 2025 and beyond, sign up now to receive GHTC’s R&D News Roundup newsletter.

Interested in more global health innovation news? Every week GHTC scours media reports worldwide to deliver essential global health R&D news and content to your inbox. Sign up now to receive our weekly R&D News Roundup email. A research team was able to induce broadly neutralizing antibodies targeting hepatitis C virus (HCV) in laboratory models for the first time, providing a promising approach for a vaccine targeting the virus, which an estimated 58 million people globally are chronically infected with and which causes 290,000 deaths annually. HCV is identified as a globally prioritized pathogen for vaccine research and development by the World Health Organization, but developing a vaccine has been a challenge due to the high genetic diversity of the virus. This new approach, if proven successful in further studies, could not only bring us closer to an effective HCV vaccine that could help limit the spread of the virus but could also lay the foundation for a new generation of vaccines against other priority viruses.Last week, the World Health Organization (WHO) announced that it had granted prequalification to Cepheid’s Xpert MTB/RIF Ultra rapid diagnostic test for tuberculosis (TB), the first TB diagnostic test to be prequalified by WHO. The designation will hopefully help ensure broader access to the product, allowing for accurate and early detection of TB, especially drug-resistant TB. Within hours, the test detects the genetic material of Mycobacterium tuberculosis in sputum samples and identifies mutations associated with rifampicin resistance, a key indicator of multidrug-resistant TB. A recent report found that 8.2 million people—the highest number of cases recorded by WHO since it began global TB monitoring—were diagnosed with TB in 2023, underscoring the need for greater access to new and improved tools.Last week, the World Health Organization (WHO) announced that it had granted prequalification to tafenoquine, the first single-dose medicine for the prevention of Plasmodium vivax malaria, and also included tafenoquine, co-administered with chloroquine, in the new WHO malaria guidelines for South America. P. vivax is the dominant malaria parasite in most countries outside of sub-Saharan Africa, with children under five and migrant populations at particular risk. The prequalification and inclusion of tafenoquine in the updated guidelines are aimed at improving access to the treatment, which is shorter and simpler than current treatment regimens.

Interested in more global health innovation news? Every week GHTC scours media reports worldwide to deliver essential global health R&D news and content to your inbox. Sign up now to receive our weekly R&D News Roundup email. A study published last week tested a promising new malaria vaccination strategy that aims to boost immunity through bites from mosquitoes carrying a genetically engineered version of the malaria parasite, which could pave the way for improved malaria vaccines. There are two approved vaccines for malaria, but they are only about 75 percent effective and require booster shots. In a trial, researchers exposed participants to bites from mosquitoes that had a version of the malaria parasite that was genetically modified to stop developing after delivery into a human, finding that nearly 90 percent of participants exposed to the modified parasites avoided contracting the disease after being bitten. The next step will be a larger trial to confirm this strategy as a viable candidate to advance malaria control efforts.A new study found that vaccines for diarrhea could reduce bacteria-associated diarrhea and antibiotic use among young children in low- and middle-income countries, which could also help reduce the emergence of antimicrobial resistance due to antibiotic overuse in low-resource settings where cases of diarrheal disease are most common. The research team used data from a previously published study exploring enteric infections and diarrheal illness in children in low- and middle-income countries and generated simulations to estimate the hypothetical impact of nine vaccine scenarios, including six single-pathogen vaccines and three combination vaccines. They found that, compared to the other single-pathogen vaccines, a vaccine against Shigella could cause the greatest reduction in antibiotic courses, and an adenovirus-norovirus-rotavirus vaccine could cause the greatest reductions in antibiotic use compared to the other combination vaccines.Last week, the Coalition for Epidemic Preparedness Innovations (CEPI) announced $1.46 million in funding for PATH to develop antibody reagents for developing vaccines against Severe Acute Respiratory Syndrome, or SARS. Antibody reagents are critical for measuring and analyzing a vaccine’s potency and ability to induce an immune response. PATH will lead efforts to develop and test reagents, as well as collaborate with the Texas Children’s Center for Vaccine Development to select and scale up a SARS-CoV-1-specific antibody to ensure these reagents are available globally, particularly in low and middle-income countries. The project aims to strengthen global health security by allowing researchers and manufacturers the tools to develop and scale up vaccines quickly in the face of an outbreak or new variant.