The new reality of R&D funding for neglected tropical diseases

One upside of the COVID-19 pandemic was the renewed attention on public health. However, just a few years later, “the party is over, very clearly”, according to Thi Hanh Cao, external affairs director and head of fundraising at the Drugs for Neglected Diseases initiative, or DNDi.

Despite affecting more than 1 billion people, neglected tropical diseases are, by name and nature, significantly overlooked.

Yet investing in this field clearly pays off. An analysis by Policy Cures Research estimated that every $1 invested in neglected disease research and development and developing life-saving products generates a societal and economic return of $405.

Those figures reflect the impact of products — including drugs, vaccines, and diagnostic tests — that have come to market in the past 20 years, plus the anticipated impact of entirely new innovations between now and 2040. If funding stops, those gains simply won’t materialize.

There’s good reason for concern: Overall investment in R&D for neglected diseases in 2023 was $3.7 billion, down from a high of $4.7 billion in 2018, according to Impact Global Health.

That’s a big issue for organizations such as DNDi — a nonprofit launched in 2003 to discover, develop, and deliver affordable treatments for neglected diseases. Together with its partners, it has delivered 13 new treatments for six diseases to date.

While one of its founding partners, Médecins Sans Frontières, and some governments, including the United Kingdom and Switzerland, continue to provide core funding, DNDi is increasingly looking to less conventional sources. One promising avenue is governments in the global south, whose own populations are disproportionately affected by NTDs.

Another is the use of priority review vouchers, or PRVs. Introduced by the U.S. Food and Drug Administration in 2007, PRVs incentivize the development of new drugs for certain diseases. Once a drug is registered with the FDA, the voucher grants the pharmaceutical company or applicant expedited review for any other product, and these vouchers can be sold for millions of dollars.

Cao spoke to Devex about the opportunities and challenges within this changing funding landscape.

This conversation took place before the USAID stop-work order and has been edited for length and clarity.

How would you describe the current state of funding for NTDs?

It's not an easy moment for funding for NTDs. … COVID was a time when there was a lot of focus on global health and research, not [specifically] for NTDs. A lot of existing donors increased their funding, and new donors came in.

The party is over, very clearly — and the world has changed. New political leaders have been elected. Wars have started. So there are very strong questions for a donor: Where do you put your money?

Government funding for NTD research has decreased. What does that mean for you?

We have to diversify funding sources. The trend I see is an increase in philanthropy funding, which is not the case for governments; but only a limited number of philanthropists are interested in funding NTD research.

There is also an opportunity with a new type of funding: impact investors. But we need support to better understand and tap into this new source because it's an entirely new world for us.

There are 21 NTDs on the WHO list, together affecting [over] 1 billion people. Everybody knows malaria, tuberculosis, and HIV, but very few know NTDs — diseases with sometimes unpronounceable names, such as leishmaniasis [3 types of diseases caused by a protozoa parasite] and mycetoma [a chronic, infectious disease of the subcutaneous tissue], affecting people in remote places.

Some NTDs affect a large number of people; others currently affect relatively few people. Our objective for those diseases is to eliminate them. So it’s not a question of numbers. We need to explain that to this new kind of investor who usually prefers a large number of patients, something they associate with a large impact.

For example, in the Democratic Republic of Congo, only [around] 400 cases of human African trypanosomiasis per year have been reported to the WHO, although the actual burden might be higher.

The majority of your funding is now earmarked to specific projects related to a disease or drug, whereas previously most were core funding. How is this affecting your work?

We have to change the way we manage our portfolio. … Projects can sometimes be delayed until we secure an earmarked grant. This can impact development timelines and makes it more difficult for us to develop several compounds in parallel. To find a molecule with potential to treat a disease, we screen millions of chemical compounds to identify a handful of promising ones that will then be optimized and enter our development pipeline.

Earmarked funding also means an increase in administrative complexity. For example, we had to submit 65 reports to donors last year. It draws on the time, energy, and brain capacity of the organization.

I'm not expecting that we will get money and not have to report to donors! The question is, how do we continue being efficient and focused on our R&D priorities, while complying with donor requirements?

What is the impact of not investing in NTDs?

A completely new treatment takes 10-15 years [to research and develop]. There are a large number of products in the pipeline and they need continued funding to be delivered in the next five to eight years.

Imagine if funding completely dried up today: we would lose all the investments made previously. It’s not only about money; lives can be changed.

How are PRVs, awarded by the U.S. FDA, helping to fund your work?

When we enter into negotiations with a pharmaceutical company, there is usually a discussion about the mechanisms to share a potential PRV — if our upcoming product will fit the FDA criteria to obtain a PRV, of course.

This PRV is awarded to the industrial pharmaceutical partner, not to us directly. To give you a concrete example, our partner Sanofi obtained such a voucher when it registered our new human African trypanosomiasis drug, fexinidazole, with the FDA, then gave us €39 million for it.

This is an amazing source of funding. Because this money has no strings attached, it allows us to be more nimble in pursuing new opportunities and partnerships to address the needs of neglected patients.  

For example, we are using some of this PRV money to invest into new parts of our portfolio, such as new medicines to prevent severe dengue.

Before a project is ready to be pitched and “sold” to donors, we need to do preliminary work and feasibility studies, we need a team, we need to have discussions with partners. … So this source of funding helps us kickstart new R&D projects.

What role does funding from the global south play?

Global south funding is part of our diversification strategy. DNDi was created by a group of research organizations from the global south, plus MSF, with the WHO and Institut Pasteur. So we are fortunate to have a really good footing in some countries in the global south and to have great partners.

We have been successful recently in securing some funds from Brazil. We've been working in Brazil for 20 years and recently received $1.5 million from the government — a very significant amount — to support research activities [for cutaneous leishmaniasis].

A specificity of financial support from the global south is that these countries usually focus on diseases endemic to their region. Brazil focuses on Brazil-endemic diseases, Malaysia on Malaysia, India on India, etc.

[At DNDi,] we usually conduct clinical trials in multiple countries. So we look forward to working with those countries so they can fund the trials in their own hospitals.

What other new sources of funding have emerged?

We've been successful in getting some funds from new actors from the private sector, such as the Novo Nordisk Foundation. They are funding the earliest part of our pipeline, which we call “drug discovery.” It is usually very difficult to convince donors to fund this work because it is very scientific with no guaranteed outcome.

This grant is a first [for DNDi], and even if it does not reach millions, I see it as a very interesting entry of a new player into R&D.

Update, Feb. 10, 2025: This article has been updated to clarify that Thi Hanh Cao’s interview was conducted before the USAID announcements.

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