Breakthroughs Blog: FDA

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 led by a multi-country research team has found that the Xpert MTB/Ultra molecular diagnostic test for stool samples, which is currently only recommended for tuberculosis (TB) diagnosis in children, could serve as an alternative TB diagnosis option for adults living with HIV—a potential gamechanger for improving diagnosis among this population. Thirteen percent of the 1.25 million TB deaths in 2012 were among people living with HIV, but access to the sputum- and urine-based tests currently recommended for TB diagnosis in this population is limited in many settings, and they are not effective for all people living with HIV. The researchers found that, compared to current tests, the stool test was effective, and, in some cases, even identified additional cases that were not detected in sputum or bacterial culture.The Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) last week announced a $3.7 million award to ArrePath to support its first-in-class antibiotic for treating complicated urinary tract infections caused by multidrug-resistant Enterobacterales bacteria. If it proves successful, the novel antibiotic class could target the vast majority of multidrug-resistant Enterobacterales infections. The class of antibiotics also has attributes that suggest it could be administered orally or intravenously, allowing more flexibility for patients. ArrePath identified the new class using an artificial intelligence and machine-learning-enabled approach.Last week, the US Food and Drug Administration approved a freeze-dried formulation of Bavarian Nordic’s vaccine for mpox and smallpox, Jynneos, enabling additional flexibility in stockpiling doses of the vaccine to prepare for future outbreaks of smallpox or mpox. The freeze-dried vaccines are easier to store and transport and have a longer shelf life than the frozen liquid version, which was approved in 2019. Bavarian Nordic will deliver the first freeze-dried vaccines later this year.

The World Health Organization’s (WHO’s) prequalification (PQ) program plays a critical role in certifying the quality, safety, and efficacy of medical products to expand access to health technologies in low- and middle-income countries (LMICs). In our PQ Pulse interview series, GHTC speaks with stakeholders involved in the PQ process about persistent challenges within this evolving regulatory ecosystem and potential reforms to promote equitable access to innovative medical products.  Our first guest is Pierre Hugo, Vice President of Market Dynamics and Global Supply Security at the Medicines for Malaria Venture (MMV), a nonprofit product development partnership working to advance accessible medicines to treat, prevent, and eliminate malaria. MMV works end-to-end along the research and development pipeline, developing novel products, engaging with regional regulatory bodies, and supporting local manufacturers.In our interview, which has been lightly edited and condensed below, Pierre discusses MMV’s experience navigating WHO PQ and his hopes for the future of the R&D ecosystem.Can you tell us about MMV and the unique role and perspective it brings to product development?We fill the gap and, by and large, are a risk mitigation tool as one of the product development partnerships, or PDPs, within the malaria space to discover, develop, and deliver malaria products. Regarding access, our key ethos is that no drug is a medicine until it's in the hands of the patient. It's all very good and well developing drugs, but if they aren't developed correctly or affordably for the target population and ultimately made available, it's a dead-end game. So, we try to ensure that in the last mile, or the death-mile, drugs are getting to the patients. WHO PQ and organizations like MMV play a very important role.Broadly speaking, how would you describe MMV's experience with engaging in the WHO PQ process?  We’ve got a number of products that are now WHO prequalified, working with our pharmaceutical partners. We see the whole PQ process as a critical mechanism to ensure that quality standards are adhered to and, ultimately, to make quality-assured products available in LMICs, where maybe those regulatory pathways are a little bit less mature. I think what's great to see is that the landscape is changing, with a lot more countries achieving WHO maturity-level 3 status [Editor note: a WHO designation for countries with stable, well-functioning regulatory systems]. Yes, there have been challenges. WHO has been very collaborative, the team has been engaging, and we are grateful for the support that WHO PQ provides.Looking forward, it's going to be critical to see how WHO PQ needs to evolve and change to further strengthen and work more closely with countries’ regulatory mechanisms or pathways.How do you see the regulatory landscape evolving in the coming years, including the potential role of the African Medicines Agency (AMA) and other regional bodies?  The AMA I think is the future; it's critical that it is a success. There's a fair amount of, I wouldn't say pessimism but [questions about] how long it will take for them to be up and running, simply because they just have so many different countries and regional regulatory systems.By and large, the East African collaborative registration or collaborative processes [The Eastern African collaborative registration process is a regional initiative that aims to facilitate the registration of medicines and medical products through a collaborative process, including joint assessments and mutual recognition of regulatory decisions] have been working to some success. There are other regional harmonization networks, but it takes time, and there is still a lot of work that is needed.How do you view recent reforms made to the PQ program? Overall, we are encouraged to see efforts that enable the fast-tracking of innovative product introduction. The new WHO framework for novel products [a reform implemented in 2024 to initiate parallel, rather than sequential, processes for recommending the use of novel products and evaluating them through PQ] is just entering its pilot phase. The new web portal [an online PQ application platform that will provide real-time information for applicants, regulators, and other stakeholders on the status of products in review] will give us greater transparency. The Coordinated Scientific Advice [a new procedure that allows product developers to engage with WHO early on to better understand the evidence that they will need to generate to support PQ review and generation of policy recommendations] should ensure that later review of the finished product is as streamlined as possible.There also needs to be some thought on how AMA will work with the European Medicines Agency, the US Food and Drug Administration, and WHO PQ to avoid complexity for local manufacturers.  If you had a hypothetical magic wand to wave to influence the program, what changes would you make?Something that is critical is to have clarity on timelines and the interaction between PQ and national regulatory bodies and lessons learned. Ultimately, what is the incentive for a manufacturer to go that extra mile to ensure that a product is quality approved [through PQ]? That's where the funders play a critical role in endorsing and supporting the process. Without that support, if you speak to a lot of the manufacturers, they say, “Well, there's no way I'll be going through the WHO PQ process. It's a major investment, and it's an additional long-term commitment that you need to make as a manufacturer.” So, ensuring that there is coordination between normative bodies, funders, procurement partners, and regulators is critical to ensure fast uptake of innovative products.The dream is to have a lot more quality-approved manufacturers in Africa, where over 95 percent of the patient population is. If you look at our new products coming down the line, like single-dose cures or long-acting injectables, greater upfront alignment is key, between WHO PQ and these stringent regulatory authorities, to offer clearer guidance in terms of what those regulatory expectations are for new products. We need to ensure that global procurement agencies, like the Global Fund to Fight AIDS, Tuberculosis and Malaria, the President’s Malaria Initiative, or UNICEF, are working as closely as possible with AMA and with WHO PQ to ensure the rapid uptake of new qualified drugs.I would love to see harmonization between PQ and national regulatory authorities to fast-track important innovation and ensure that new quality-assured drugs get to market faster.Putting on your advocacy hat, where do we, as an advocacy community, need to put our energy in the next couple of years to make some of those improvements become reality?  To unleash the potential [of PQ], we need to simplify the processes for manufacturers, ensure procurement commitments for a clear return on investment, and provide continuous and sustained funding to support manufacturers to embark on the lengthy quality assurance process. If I look at the manufacturers, we did an extensive exercise with the Africa Centres for Disease Control and Prevention to identify manufacturers throughout the whole continent that would want to take malaria products forward to be WHO PQ’d. We only had about 50 manufacturers that were interested in taking part in this initiative [out of 400], so that's a very low number. I mean, we're talking small molecules, not vaccines, and you've got to ask yourself why? They obviously don't see a need to have products WHO PQ’d, which is, on the one hand, a problem. The Africa Centres for Disease Control and Prevention and MMV and other partners believe that the African manufacturing sector can take its place long term alongside manufacturers from India and China, who today supply the vast majority of public health medicines for malaria on the continent.Big Pharma, if anything, is disinvesting in Africa. So, having those in-Africa partners is critical within the malaria space and, obviously, not just for malaria, but for HIV and tuberculosis and so forth.So, I'm just thinking that if manufacturers are going down the WHO PQ route, it might be a really nice carrot to say, “Look, if you are applying for the WHO PQ process, this is a financial incentive in place.” That could be a really nice carrot to keep the Big Pharma guys in the game.