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. Newly published research suggests that a patient-centered approach, combining novel diagnostic tools with targeted therapies, could be a game changer in tuberculosis (TB) treatment. Current TB treatment requires a complicated, months-long regimen that can be hard for patients to adhere to, leading to treatment failure and the emergence of drug-resistant TB strains. The researchers behind the new study suggest that new diagnostic tools that can better distinguish between patients with less and more severe forms of TB combined with, respectively, shorter or longer, more resource-intensive treatment courses could improve patient outcomes, reduce the burden on health systems, and limit the emergence of drug resistance.Last week, Bavarian Nordic announced the start of the first of two clinical trials testing the use of its Jynneos mpox/smallpox vaccine in vulnerable populations, including infants and children two years old and younger and pregnant or breastfeeding women. If the vaccine demonstrates safety and efficacy, the trial data could help support expanded access to the vaccine for the most vulnerable populations. The trial is being led by the University of Antwerp and the University of Kinshasa and supported financially by EDCTP3 and the Coalition for Epidemic Preparedness Innovations.A recently published study found that a single-dose, long-lasting flu drug could provide protection for an entire flu season, offering longer and more effective protection than our current seasonal flu vaccines. The clinical trial followed 5,000 healthy adults for the entire duration of the most recent flu season, finding that the highest dose of the drug provided more than 76 percent protection compared with a placebo shot. The drug has also demonstrated efficacy against a wide variety of flu strains in a previous study in mice, compared to the season influenza vaccines, which are designed (prior to flu season based on expert assumptions) to protect against at least three of the seasonal strains that circulate each season and whose efficacy depends on how well they match the flu strains that end up circulating.

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. Last Wednesday, Gilead announced that its twice-yearly injectable drug for HIV prevention has received approval from the US Food and Drug Administration, the first and only such shot for HIV prevention. The drug, lenacapavir, which is already used to treat HIV in certain populations, was found to dramatically reduce the risk of infection, providing near total protection among cisgender men and women, transgender men and women, and nonbinary people in clinical trials. The twice-a-year injection offers a significant improvement over current HIV prevention options that require a daily pill or every two-month injections, helping improve uptake and adherence by providing a more discreet, private, and less cumbersome alternative. Pricing and access plans for the United States and the rest of the world will determine the scale of lenacapavir’s impact on the broader HIV epidemic.Researchers from Cornell University have found that the antibiotic rifampin, which is already widely used, is 99.9 percent effective against Salmonella Typhi, the bacterium that causes typhoid fever, which a growing threat due to antibiotic resistance. The researchers found that S. Typhi is still highly susceptible to the antibiotic and that rifampin-resistant typhoid is extremely unlikely to emerge, despite concern due to the previous emergence of rifampin-resistant tuberculosis cases. The researchers found that rifampin may also be effective against other life-threatening diseases like bacterial pneumonia and meningitis.Newly published research found that a simple, affordable device made with strips of paper was more effective than other commonly used testing methods at detecting malaria in asymptomatic people in Ghana. Malaria testing, particularly at the point of care, is essential to malaria control in endemic areas, especially as increased uptake of the new preventive malaria vaccine lowers natural immunity, raising the need for widespread surveillance. The new test is highly sensitive, offers results in about 30 minutes, and can detect even a low density of parasites in the blood, catching malaria before a patient is sick.

The recent staffing cuts at the U.S. Food and Drug Administration (FDA), which affected roughly 20 percent of its workforce, have prompted concerns about the agency’s ability to fulfill its mission. The cuts would severely impact US companies and US global leadership in the biomedical space.  The global implications would be even more grievous—particularly for low- and middle-income countries (LMICs) that depend on the FDA’s regulatory leadership. Many LMICs and other countries rely on the FDA’s assessments, approvals, and post-market surveillance data to inform their own regulatory decisions. These programs, often referred to as “reliance programs,” streamline national approval processes by referencing trusted decisions from agencies like the FDA. As the agency grapples with significant staffing reductions, there could be ripple effects on these international structures.What are reliance programs?Reliance programs allow regulatory authorities in one country to draw on the evaluations, inspections, and approvals of trusted agencies—known as Stringent Regulatory Authorities (SRAs)—such as the FDA. Many LMICs use FDA decisions to streamline access to essential medicines and vaccines, avoiding duplicative reviews and accelerating timelines. This model is especially valuable where regulatory resources are limited, allowing local agencies to focus on post-market surveillance and health care delivery. The FDA’s assessments serve as a de facto quality mark, reinforcing confidence in products worldwide.One example is the World Health Organization’s (WHO’s) Collaborative Registration Procedure (CRP), which enables countries to fast-track the registration of WHO-prequalified medicines by leveraging FDA and other SRA reviews. Between 2015 and 2021, the CRP facilitated 59 approvals for 16 medicines across 23 countries, demonstrating its impact on timely access to quality-assured medicines for patients.Implications for the United StatesIf the FDA’s reduced capacity compromises its ability to effectively conduct timely reviews, update guidance documents, or perform inspections, the most immediate impacts will be felt in the United States. The United States could lose influence in setting international safety, efficacy, and quality standards if other regulatory authorities begin supplanting the FDA's role in global reliance networks. Over time, this erosion of regulatory leadership could diminish the FDA’s status as the gold standard globally—a position that has long bolstered American soft power in global health and trade. Manufacturers based in the United States that are developing products for diseases endemic in LMICs may also find their innovations at a disadvantage if the FDA lags behind other regulators in review speed or capacity or if the WHO prequalification program, which allows US manufacturers to leverage mechanisms that streamline and accelerate the deployment of their innovations in LMICs,  no longer recognizes the validity of FDA review. In 2024, for example, WHO prequalified a Hepatitis C self-test manufactured by Bethlehem, Pennsylvania-based OraSure Technologies. An innovative mpox point-of-care molecular test developed by Sunnyvale, California-based Cepheid became the first such diagnostic ever approved by WHO for emergency use. These FDA-WHO collaborations are vital for the purchase and procurement of American products. If the FDA retreats from this space, it could reduce the competitiveness of American firms in emerging markets and make the United States a less attractive regulatory partner for international collaboration. Preserving the FDA’s global standing is, therefore, not only a matter of public health but also of economic and geopolitical significance.Potential impacts on LMICsHowever, the secondary consequences could be more acute for countries relying on the FDA’s efficiency to inform their regulatory decisions. Delays in product approvals may extend the time before essential medicines reach patients in LMICs—where the burden of disease is often highest and timely access is critical. The loss of experienced personnel and institutional knowledge may also weaken confidence in the consistency and rigor of FDA assessments, prompting some regulators to reevaluate the extent of their reliance. The FDA should build its internal capacity to address neglected diseases rather than reduce staff, providing training opportunities and ensuring their workforce has the prerequisite expertise.Looking aheadTo help safeguard both domestic and global health outcomes, it will be important for the FDA to maintain its capacity to carry out timely, science-based evaluations. One upcoming opportunity to support this effort is the Prescription Drug User Fee Act (PDUFA) reauthorization, which allows the FDA to collect fees from pharmaceutical companies to help fund its drug review programs. PDUFA has long been a bipartisan priority and is key in supporting the agency’s scientific workforce and review capabilities. Ensuring the continuity of this program will help the FDA meet its responsibilities at home and abroad. As global reliance on the FDA remains high—particularly among countries with limited regulatory resources—continued investment in the agency’s capacity will be necessary both for US innovation and global health systems that depend on its leadership.Now more than ever, we must protect the FDA’s ability to lead. A weakened FDA puts American innovation and jobs at risk, slows access to breakthrough products, and opens the door for other countries to set the standards we once defined. The world looks to the FDA, not just for safety and science, but for leadership. With PDUFA reauthorization on the horizon, Congress has a critical opportunity to invest in the FDA’s workforce and secure the United States’ role as the global leader in health and biomedical innovation.

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 new study found that LC16m8, an attenuated vaccinia virus strain originally developed in Japan for smallpox and later approved for mpox in 2022, induced strong responses and was safe in mice, monkeys, and healthy adult humans, potentially paving the way for the development of broadly effective mpox vaccines. The researchers vaccinated mice with LC16m8 to see how it protected them from mpox, later vaccinating monkeys and healthy adult volunteers as well to study their blood samples for immune responses and monitor them for safety. The results underscore LC16m8’s potential as a safe, effective, and scalable choice for mpox vaccination and also provide a blueprint for developing next-generation vaccines against other poxviruses and emerging pathogens.Researchers from Abertay University and Bangor University have developed a prototype blood test that can detect up to five infections at the point of care, providing results quickly via a smartphone app. The tool, which was piloted using tuberculosis as a test case, showed high sensitivity and an ability to accurately identify cases. The speed and convenience, as well as the much lower cost compared to lab testing, demonstrate the test’s potential to be a game changer in ensuring access to timely testing and diagnosis, particularly in remote locations and other settings lacking laboratory infrastructure.  The results of a Phase 3 clinical trial published last week found that Moderna’s combination COVID and flu shot outperformed existing individual vaccines for both viruses. The combination shot uses the same mRNA technology as Moderna’s approved vaccine for COVID-19, but there are no currently approved mRNA flu vaccines. In a trial that included more than 8,000 adults, the combination vaccines generated higher antibody levels for COVID and all but one influenza strain in trial participants who got the combination vaccine compared to those who got individual vaccines. A combination vaccine could help boost vaccination levels for both viruses, and an mRNA vaccine, in particular, could also speed up production because they are faster to make than current flu shots.