Patients in low- and middle-income countries largely left out of clinical trails, limiting access to new treatments

• Only 43% of all clinical trials (297/685) analysed in the 2024 Index are conducted in any low- and- middle-income countries (LMICs), despite being home to nearly 80% of the global population.

• Pharmaceutical companies typically prioritise market access in countries where clinical trials are conducted, leaving much of the world behind.

• Clinical trials should be more diverse, with overall access planning during research and development encompassing all geographies to ensure equitable access.

Clinical trials evaluate the effect that new treatments, such as vaccines and medicines, have in human populations to ensure that only reliable, safe treatments reach patients. Typically, pharmaceutical companies conduct these trials in countries where they plan to market their products and provide posttrial availability through access plans, which are a crucial step to ensuring broad access to these products upon launch. Industry wide, the quality of access plans during product development varies widely and these plans tend to prioritise a small number of low-and middle-income countries (LMICs)^*, usually those in which clinical trials have been conducted. As a result, the location of these trials and patient populations included often determines where new healthcare products will become accessible to people once approved.

Companies prioritise high-income countries for clinical trials

The 2024 Index finds that most clinical trials are concentrated in high-income countries, leaving populations in lower-income regions underserved. In analysing companies’ clinical trials for 81 diseases that disproportionately impact people living in LMICs, the 2024 Index found a significant gap, with less than half (43%) of these trials carried out in any LMICs (see the figure below).

The disparity in the clinical trial landscape has significant implications for patients living in LMICs:

1. Companies prioritise access planning in countries where clinical trials are conducted, leading to delays or reduced access in countries without trials.

2. Clinical trials offer patients access to potentially lifesaving investigational medicines, but many people in LMICs miss this opportunity.

3. The resulting lack of research data from populations in LMICs limits the understanding of how diverse patient populations respond to new therapies.

Access planning often limited to the few countries where clinical trials take place

Should a clinical trial prove successful, the first crucial step is to ensure the product's availability in the country where the trial took place. To this end, access planning during clinical trials is critical to ensure that new medical treatments are available swiftly and widely once they are launched on the market. This link between clinical trials and access means that, when LMICs are excluded from clinical trials, they are also excluded from the access plans tied to those trials. As a result, people living in these countries face significant delays before new treatments become available and, in many cases, may never have the chance to access new therapies.

Most companies (15/20) have public commitments to plan for access by registering the product for approval in countries where they conduct trials. However, since clinical trials are only conducted in a small cohort of LMICs, access plans tend to be limited to those countries. On average, an access plan includes only six of the 113 LMICs covered in the Index, typically focusing on a select number of upper-middle-income countries or emerging markets with higher commercial potential. Lower-middle-income-countries are included in access plans to a lesser extent, highlighting significant room for improvement to expand plans to include a broader range of countries.

Expanding the scope of clinical trials to more LMICs can play a significant role in broadening the geographic reach of companies’ access plans, ultimately helping to narrow gaps in access both pre- and post-approval of new, innovative products.

Expanding the scope of clinical trials in LMICs

Indeed, the capacity to conduct clinical trials is constrained in some LMICs for a few reasons; essential infrastructure may be limited, with a lack of adequate clinical facilities and trained healthcare personnel, for example. Regulatory frameworks may also be complex or fragmented. Furthermore, the absence of contract research organisations – which provide outsourced support to pharmaceutical companies to conduct trials – in some LMICs makes it more challenging to operationalise the trials on the ground. However, it should be noted that the capacity to conduct trials varies significantly in different countries and regions analysed by the Index, with the landscape evolving and established clinical trial networks in place in many LMICs. As a first step to widening the scope of their clinical trials, companies can expand trial sites to routinely include LMICs where the necessary infrastructure is already in place, which would inherently strengthen local research capacity. In addition to this, broadening clinical trials to include more diverse populations would ensure more equitable representation of the world's population in clinical research. Consequently, the resulting treatments would have broader, more reliable applications globally, reducing health disparities. For trials in lower-resource settings, companies can work with partners to build local research capabilities and conduct clinical research. This is particularly important to discover suitable treatments and cures for diseases that are endemic in these countries. In the future, as healthcare needs of patient populations in these countries evolve, companies can explore opportunities to collaborate with partners to expand clinical trials to other disease areas.

Alongside partners, some companies are conducting clinical trials in low-income countries

The 2024 Index identified a few examples of companies making efforts to increase their focus in poorly resourced settings. For instance, although only 3.5% of clinical trials take place in low-income countries, at least eight companies – Bayer, Eisai, Gilead, GSK, Johnson & Johnson, Merck KGaA (Merck), Novartis, and Sanofi – are engaged in these trials either through their R&D efforts or by partnering with organisations that lead the trials.

As it stands, clinical trials in low-income countries are focused predominantly on communicable diseases, such as HIV/AIDS (6) and malaria (7) or neglected tropical diseases (6) reflecting the high burden of these diseases in these regions (see the figure above).

Specific examples of companies’ clinical trials in low-income countries:

• Gilead conducted clinical trials testing lenacapavir (a long-acting injectable) for HIV prevention in Uganda, a low-income country where approximately 1.5 million people are living with HIV. As part of its access plans, the company announced a non-exclusive voluntary licensing agreement, allowing manufacturers to make and supply generic lenacapavir in 120 LMICs, including Uganda, a low-income country.

• Novartis and Merck KGaA are both conducting clinical trials in low-income countries for new antimalarials as part of the PAMAfrica consortium, led by the Medicines for Malaria Venture. Trials for Novartis' new IV formulation of cipargamin for severe malaria are ongoing in Burkina Faso, the Democratic Republic of Congo, Rwanda and Uganda. Novartis has developed a comprehensive access plan if the product is successful in clinical trials that includes equitable pricing and broad registration plans in countries with high disease burdens. Merck's cabamiquine is currently being tested in clinical trials in Burkina Faso, Mozambique and Uganda for both the treatment and prevention of Plasmodium falciparum malaria. Merck has an access plan in place, including a regulatory strategy for countries with high disease burdens, and plans to ensure sustainable supply.

Some companies are also collaborating with international and local partners to build clinical trial capacity in LMICs. The Clinical Trials Community Africa Network (CTCAN), of which Johnson & Johnson is a partner, aims to build clinical trial capacity by combining data from research centres across Africa. Similar efforts are underway in Asia, with partnerships like the one between AstraZeneca and the Cancer Research and Clinical Trials Centre (CRCTC) in Vietnam focusing on oncology treatments. These partnerships can be further expanded to build sustainable clinical trial infrastructure in more regions, and for multiple products and therapeutic areas.

What’s next?

To improve access and reduce delays in access to the newest medicines for those who need them most, companies can expand clinical trials to include more diverse populations, especially in LMICs. At first, companies can expand the scope of their clinical trials, routinely including LMICs where the necessary infrastructure and regulatory systems are already in existence. In tandem, it is also essential for companies to develop comprehensive access plans that cover a wider range of countries and diseases. Such plans should consider, among other factors, plans for registering in more countries, affordability, collaboration with local partners, and securing consistent supply chains.

Additionally, pharmaceutical companies play a vital role in fostering local R&D capacity. As innovators and patent holders, they are uniquely positioned to collaborate with partners in LMICs, helping to bridge gaps and build clinical trial capabilities in these countries. This includes developing centralised clinical trial infrastructure that would facilitate a coordinated and streamlined process for monitoring data, which can be scaled across all products in development, expediting the expansion of clinical trials to more regions.

_{*The term LMICs is used to denote all low- and middle-income countries in scope of the Index. This encompasses countries all countries classified as low-income, lower-middle income as per the World Bank income groups classification and some upper-middle income countries with a high inequality-adjusted HDI ratio as defined by the UN Inequality-Adjusted Human Development Index.}