Breakthroughs Blog: Medicines

In 2001, I traveled to Ghana as a graduate student working on a US Agency for International Development-funded decentralization program. I arrived armed with materials on local governance best practices. But the mayors I met were focused on urgent public health issues: clean water, HIV/AIDS, tuberculosis, and malaria. That trip, meant to be an early lesson in governance, became my first and most personal education in global health. It also nearly killed me. I was taking mefloquine weekly to prevent malaria. Toward the end of our stay, my partner and I set off to explore the country. Somewhere along the way, I developed what I believed to be giardia. That infection likely compromised the absorption of the mefloquine and left me dangerously vulnerable. One night in the village of Kokrobite, I woke with a high fever and chills so intense I could barely stand. A local man saw me and immediately recognized it as malaria. He arranged for a taxi to take me back to Accra. Before I left, he handed me a small, crumpled packet labeled in Chinese characters. Inside was a blister pack of pills. The only word written in English on the package was artesunate. He told me it would help, and, in that moment, sick, scared, and weak, I took it. At the hospital, a blood smear confirmed that I had cerebral malaria caused by Plasmodium falciparum, the deadliest strain. Cerebral malaria can cause brain swelling, coma, and death. I drifted in and out of consciousness for days. At one point, hospital staff told my partner that there was nothing more they could do. I should “go home” and be “as comfortable as possible.”My partner worked relentlessly to get me on one of the next flights back to the United States. I was so sick that the airline nearly refused to let me board. Once we arrived home, I was seen right away by an infectious disease doctor at Johns Hopkins Hospital. After reviewing my labs and the herbal remedy package, he said plainly: this saved your life. The artesunate had bought me enough time to survive the journey and begin treatment. At the time, the World Health Organization had not yet formally endorsed artemisinin-based combination therapies (ACTs) as the global standard for P. falciparum malaria. That came in 2006. But the effectiveness of artesunate alone was already well known in many parts of the world. That medicine saved my life.I missed several weeks of school but made a full recovery. Without that drug and the help of my partner (now my husband) and friends in Accra, I wouldn’t be here today. More than two decades have passed, and we've made tremendous progress. Insecticide-treated bed nets, rapid diagnostic tests, and ACTs have saved millions of lives. Two malaria vaccines, RTS,S and R21/Matrix-M, have been approved. Long-acting monoclonal antibodies are showing promise as seasonal protection for children. Newer insecticide-treated nets help counter resistance. And researchers are exploring RNA-based therapeutics and single-dose cures.Many of these breakthroughs are being developed by members of the Global Health Technologies Coalition, where I now serve as Executive Director. These tools represent our best chance not just to control malaria but to end it.But progress is not guaranteed. Cuts to foreign assistance and global health research and development are already slowing the progress of innovations in the pipeline. Some promising tools may be delayed or abandoned. That means more children at risk, more untreated infections, and more drug-resistant strains emerging. The parasite has evolved before. It will do so again if we let it. And malaria is no longer a disease that happens mostly outside of the United States. In the past two years, locally acquired cases have been reported in Florida, Texas, and Maryland. Climate change, global travel, and changing mosquito habitats are making malaria a threat we can no longer treat as distant. I know how thin the line is between surviving this disease and not. For me, it’s not abstract. It’s the memory of a fevered body, the generosity of strangers, and a medicine in the right place at the right time. This World Malaria Day, I urge leaders and policymakers to stay the course. The science is moving forward. But science alone isn’t enough. If we don’t finish what we’ve started, we risk letting malaria surge back and take more lives with it. Science has given us the tools. The question now is whether we will act in time or let the disease outpace us once again. 

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.

In 2023, tuberculosis (TB) retook the spot as the world’s leading infectious disease killer from COVID-19, claiming the lives of around 1.25 million people that year. Insufficient and inadequate tools and treatments and growing drug resistance have challenged global efforts to bring the TB epidemic to an end. In 2023, approximately 8.2 million people were newly diagnosed with TB, the highest number recorded since the World Health Organization (WHO) began global TB monitoring in 1995. At the same time, global funding for TB diagnostic, treatment, and prevention services fell in 2023 and globally, TB research remains greatly underfunded, with only 24 percent of the $5 billion annual target reached in 2023. Scientists hope that the tide can be turned as new technologies and innovations advance to market—but to achieve that, more investment is urgently needed to boost resources and reverse setbacks. In recognition of World TB Day, test your knowledge of innovations to end TB with GHTC’s quiz: 

There is a well-known Chinese proverb that says, “Women hold up half the sky,” but when it comes to global health, sadly, humanity has a lot of ground to make up to close the stark gap between the health outcomes of women and men.While women tend to live longer than men, this longevity often comes with a greater burden of disease and disability, underscoring the urgency of addressing sex-based disparities in health care. Women, on average, spend 25 percent more of their lives in poor health compared to men, they are 52 percent more likely than men to have an adverse drug reaction and 29 percent more likely to die from one, and they’ve been found in one multi-decade study to be diagnosed later than men for more than 700 diseases. Research and development (R&D) for female-specific conditions also remains vastly underfunded: only 1 percent of R&D funding focuses on non-cancer-related women’s health.Yet despite these sobering statistics and new threats to the future of women’s health care and research funding, GHTC remains optimistic. With sustained support and investment, the scientific landscape holds immense potential to drive breakthroughs that can close these gaps and improve health outcomes for women worldwide.Thus, in recognition of International Women’s Day, GHTC is spotlighting five promising technologies in the research pipeline that could improve women’s health globally.1. A new treatment for gonorrheaGonorrhea is a sexually transmitted infection, more prevalent among women than men, that can cause serious health issues—from pelvic inflammatory disease to infertility and ectopic pregnancies—if left untreated. Combating gonorrhea has become more complicated in recent years, as growing drug resistance has reduced the arsenal of available treatment options.But, excitingly, a new, much-needed breakthrough is on the brink. A first-in-class antibiotic, zoliflodacin, developed by GHTC member the Global Antibiotic Research and Development Partnership, was found to be effective in a large-scale global Phase 3 clinical trial in 2023. If approved, it would become the first new antibiotic for treating gonorrhea in decades. The drug is also taken orally, which would simplify treatment administration for clinicians around the world, and its nonprofit developer, which is committed to delivering affordable access to the product, has the right to commercialize it in more than three-quarters of the world’s countries, including all low-income countries and most middle-income countries. Another second promising antibiotic for gonorrhea from GSK, gepotidacin, is also in late-stage development.2. A therapeutic vaccine for cervical cancerCervical cancer is the fourth most common cancer in women globally, claiming the lives of 350,000 women each year. Nine out of every ten cases of cervical cancer are caused by human papillomavirus (HPV) infection.Since the introduction of the first preventative HPV vaccine nearly two decades ago, global efforts have focused on expanding immunization coverage worldwide, as well as advancing research that has reduced vaccine schedules from multiple doses to just a single dose, easing administrative costs. Yet, despite these efforts, there remain significant gaps in immunization coverage—only 27 percent of girls worldwide received an HPV vaccine dose as of 2023—underscoring the need for additional complementary treatment and prevention tools.That’s where therapeutic cervical cancer vaccines could offer promise. What if we could administer a shot to patients who have already developed precancerous lesions due to HPV that could eliminate HPV-infected calls before those cells become cancerous? This theoretical possibility could become a reality in the future. A Phase II trial of the investigational therapeutic HPV vaccine Vvax001 reported results in January, finding that the vaccine successfully induced regression of precancerous lesions in half of all trial participants. Among patients with advanced precancerous lesions, the traditional intervention is surgery to remove the affected areas, which carries the risk of side effects and complications. An effective therapeutic vaccine could reduce the number of patients who ultimately require surgery and offer advantages as an intervention that is far easier and less costly to administer in resource-limited settings worldwide.3. Inhalable oxytocin to reduce PPHPostpartum hemorrhage (PPH), or excessive bleeding after childbirth, is the leading cause of maternal deaths worldwide. While the hormone oxytocin is often administered to women following childbirth to prevent PPH by causing the uterus to contract, oxytocin products are traditionally heat-sensitive and administered via injection or intravenously, which can make this intervention challenging to administer in certain settings around the world.To solve this issue, researchers at Monash University have developed an inhalable form of oxytocin that is formulated as a dry powder requiring no refrigeration. Prior clinical research has shown that the product is comparable in effect to injected oxytocin, and now, scientists are advancing research on the best means to administer it. In late 2023, a Phase I trial assessed administration of the product using a low-cost inhaler system called the ICOone. If this product ultimately reaches the market, it could offer a much-needed alternative that would make this lifesaving drug easier to store and administer to women in need, no matter where they live.4. Biodegradable contraceptive implantsMany women worldwide choose to use contraceptive implants as their contraceptive method. These products offer unique advantages for certain women: they are long-acting and easy to use; once inserted, there is no need for users to take or do anything on a daily, weekly, or monthly basis. But eventually, they must be removed when they are no longer effective or when users change their reproductive plans. This removal procedure requires trained health care providers, and it can create an access barrier for women worldwide who live far away from health care facilities.That’s why researchers are working to advance biodegradable implants, which would dissolve into the body over time, eliminating the need for removal. In February 2024, GHTC member FHI 360 launched a Phase 1 trial of a biodegradable implant, Casea S, which aims to prevent pregnancy for 18 to 24 months. At the time, it marked the first biodegradable contraceptive implant to undergo human trials in over 20 years. While the trial will still take a few years to complete and further research will be required, if the implant is successful, it could not only help solve health care access challenges but could also address a middle-ground need between existing implants that last years and long-acting injectables, which usually last three months. FHI 360 also has a second biodegradable contraceptive implant in its portfolio, which is in early-stage development and uses a different contraceptive hormone.5. A new drug to prevent preeclampsiaPreeclampsia is a dangerous pregnancy-related condition that affects between 2 and 10 percent of pregnancies worldwide and claims the lives of more than 70,000 mothers and 50,000 babies each year. While low-dose aspirin and calcium supplements are recommended to women who are at high risk of the condition, there is currently no medicine approved specifically to prevent preeclampsia. That could change in the coming years thanks to emerging research.A British biotech company has developed an investigational, once-daily oral drug, MZe786, to prevent preeclampsia, which is in early-stage development. In animal studies, the drug, which is a form of aspirin that also releases hydrogen sulfide, elicited an improvement in maternal and fetal outcomes compared to aspirin alone. While further confirmation of these effects in human clinical trials is needed and it could be many years before such a product potentially reaches market, in 2022, the drug was awarded a fast-track development status by the United Kingdom’s regulatory agency, becoming the first pregnancy-related drug to receive this status, signifying its potential to address an urgent and unmet need.All five of these promising products are inspiring examples of the stepwise progress achieved through past investments in women’s health research, but we also hope that they will serve as a rallying cry to what more could be possible if we were to devote adequate resources to addressing these challenges.This International Women’s Day, we hope that the global community will further commit to investing in innovations for women’s health and to expanding the representation of women in clinical research. After all, closing the women’s health gap is not only essential to the well-being and future of millions of women and girls worldwide, but it will also enrich the well-being and economic stability of their families, communities, and nations. It’s a responsibility and opportunity we can’t ignore.