Breakthroughs Blog: Access

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.

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 week, the Coalition for Epidemic Preparedness Innovations (CEPI) announced a $43.5 million deal with AstraZeneca to further the development of novel antibodies designed to target four pandemic influenza virus strains, which could reduce costs and improve access to monoclonal antibody treatments. The antibodies, called VHH, have unique potential to be a key medical countermeasure in the event of an influenza pandemic, and this investment marks the first time they are being looked at for pandemic flu protection, although they are currently being studied as a potential tool to address chronic diseases, including cancers. If proven effective down the line, VHH antibodies could serve as a stopgap in the case of an influenza pandemic before vaccines can be widely rolled out.A team of Chinese researchers has discovered a bacterial compound that can kill infectious fungi, including antifungal-resistant strains, as antimicrobial resistance, including resistance to antifungals, continues to rise around the world. The compound, mandimycin, was effective against World Health Organization fungal priority pathogens Candida auris, Cryptococcus neoformans, and Aspergillus fumigatus in lab tests and was able to treat an antifungal-resistant Candida auris strain in mice. The compound also seemed less likely than other compounds to lead to the development of resistance in the fungi exposed to it, underscoring its potential as a key tool to address this growing global public health crisis.Last week, the Medicines for Malaria Venture (MMV) and deepmirror announced a new disease-agnostic platform, Drug Design for Global Health, designed to accelerate drug discovery through machine learning and artificial intelligence in low- and middle-income countries. The partners aim to bolster global health security by speeding up the development of new drugs for a variety of significant diseases, including malaria, tuberculosis, and neglected tropical diseases. Specifically, the platform aims to eliminate financial barriers for scientists in low- and middle-income countries and give them access to cutting-edge technology, enabling them to drive innovation in drug discovery research efforts.

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: 

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.