Fact sheet series: R&D across health areas

HIV/AIDS

R&D for HIV/AIDS

How new tools can transform the fight

Since emerging in the 1980s, the HIV/AIDS pandemic has claimed the lives of more than 42 million people around the world—reversing gains in life expectancy and economic development in the world’s most under-resourced places. The development of antiretroviral drugs (ARVs) turned the trajectory of the global pandemic, extending the life expectancy of people living with HIV and preventing transmission between mothers and children and people of different HIV statuses.

Yet, progress toward ending the HIV pandemic has slowed in recent years, and major gaps remain in our arsenal of treatment and prevention tools. We will not bring about an end to the HIV pandemic without new and improved technologies.

3.79 million

people living with HIV/AIDS
1.3 million

people became newly infected with HIV in 2023
250 children

die every day from AIDS-related causes

Research successes

Technologies have transformed the fight against HIV/AIDS:

ARVs—developed with NIH support—are today used to treat 30.7 million people globally and have cumulatively averted 16.5 million AIDS-related deaths since 2001.
Pre-exposure prophylaxis (PrEP)—the use of ARVs to prevent infection—was shown to be effective through NIH, CDC, and USAID research and is now widely used globally.
Approaches to prevent mother-to-child HIV transmission—developed with NIH support—have contributed to a 62% decline in new infections in children since 2010.
New long-acting PrEP options, including a monthly vaginal ring and an every-two-month injectable, developed with NIH and USAID support, now offer an alternative for those who struggle to take daily pills.
Child-friendly therapies—including a sweet-tasting, heat-stable combination therapy—are improving care for HIV-positive children.
New diagnostic innovations, such as rapid tests, urine and oral tests, and at-home tests, have increased the percentage of people aware of their HIV status.
Recency tests, which distinguish if an infection occurred in the last year or before, are enabling health programs to identify HIV transmission clusters and better target services.

Continued progress is possible, not inevitable

Key missing tools

To end HIV/AIDS, we need new prevention and treatment tools, including:

Vaccines to prevent and reduce new infections.
A cure to control infection or eliminate it from the body.
New treatment and PrEP regimens to help expand choice and improve adherence, including additional long-acting options, fixed-dose combinations, and simplified treatments with fewer side effects.
Additional microbicide options to prevent infections in women and men who have sex with men—populations disproportionately impacted by the pandemic.
Expanded therapies for young children that are safe, palatable, and easy to administer.
Additional treatment options to combat resistance to existing regimens.
Additional multipurpose prevention technologies that simultaneously prevent HIV, other sexually transmitted infections, and/or unintended pregnancy.

Breakthroughs on the brink

Long-acting biomedical products that could revolutionize both HIV treatment and prevention in hard-to-reach populations by reducing frequency of use and providing a more discreet, convenient alternative to daily pills. Products now in development include a once-weekly ARV pill, a three-month vaginal ring, injectable preventative drugs taken every four or six months, and implants that slowly release anti-HIV drugs over time for continuous protection.
Multipurpose prevention products—including pills, injections, and vaginal rings that combine HIV protection with contraception—could provide more convenient options for women. The two products furthest in development are a dual prevention pill and a multipurpose vaginal ring, both of which were developed with USAID support.
Many clinical trials are underway to test broadly neutralizing antibodies (bNAbs)—proteins shown to neutralize many different genetic variants of HIV—as treatment and prevention products delivered via infusion or injection. These include trials of several bNAbs developed or isolated at NIH labs.
Several mRNA-based HIV vaccine candidates have advanced into first-in-human trials, including candidates supported by NIH, USAID, and PEPFAR, bringing new hope to the quest for an HIV vaccine. Additionally, new vaccine research strategies are reinvigorating the field, including approaches to induce bNAbs such as germline targeting, which uses a series of stepwise shots to coax the immune system to create bNAbs, and T-cell approaches, which induce T cells that act as either killer cells to directly attack the virus or helper cells that support other B cells that generate antibodies against HIV.
Seven patients appear to have been effectively cured of HIV or are in long-term remission, demonstrating that a cure for HIV infection, while difficult, is scientifically possible. Several cure strategies are also advancing in the research pipeline including a “kick and kill” approach to prompt latent HIV to reactivate so it can be targeted and neutralized and gene modification approaches to change human DNA in immune cells to produce cells that are resistant to HIV.

US government investment in HIV/AIDS R&D for low-resource settings (in 2022) US$ millions

US Government R&D efforts

The US government is leading efforts to advance research and development (R&D) to end the HIV/AIDS epidemic through a whole-of-government approach:

National Institutes of Health (NIH) conducts basic science and clinical research to advance products to prevent, diagnose, and treat HIV/AIDS, as well as social behavioral research to improve the use of existing interventions.
US Agency for International Development (USAID) advances R&D for HIV/AIDS technologies designed for low-resource settings, including research for a vaccine, microbicides, and multipurpose prevention products, and helps accelerate the introduction of and access to new tools.
Department of State oversees the President’s Emergency Plan for AIDS Relief (PEPFAR), which finances certain HIV/AIDS research programs, alongside delivering health products and services.
Centers for Disease Control and Prevention (CDC) develops improved diagnostics and conducts research to inform the use of existing tools and the risk factors influencing the spread of HIV/AIDS to better target interventions to those in need.
Department of Defense undertakes research to protect US service members, including vaccine research.
Food and Drug Administration operates a tentative regulatory approval program to allow PEPFAR to distribute generic ARVs for use outside the United States.

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Introduction

39.9 million living, 1.3 million newly infected, 42 million deaths: UNAIDS. Global HIV & AIDS statistics — Fact sheet. UNAIDS; 2024. https://www.unaids.org/en/resources/fact-sheet

250 children: UNICEF. Global and regional trends. Accessed August 9, 2024. https://data.unicef.org/topic/hivaids/global-regional-trends/

Research successes

ARVs, developed with NIH support: National Institute of Allergy and Infectious Diseases. Antiretroviral drug discovery and development. Accessed August 8, 2024. https://www.niaid.nih.gov/diseases-conditions/antiretroviral-drug-development

ARVs, used to treat 30.7 million people: UNAIDS. Global HIV & AIDS statistics — Fact sheet. UNAIDS; 2024. https://www.unaids.org/en/resources/fact-sheet

ARVs, averted 16.5 million AIDS-related deaths: UNAIDS. Global roll-out of HIV treatment has saved millions of lives. September 6, 2021. Accessed July 17, 2024. https://www.unaids.org/en/resources/presscentre/featurestories/2021/september/20210906_global-roll-out-hiv-treatment

Pre-exposure prophylaxis: The Global PrEP Tracker. AVAC; 2022. Accessed July 17, 2024. https://data.prepwatch.org/

Pre-exposure prophylaxis, shown to be effective through NIH research: National Institute of Allergy and Infectious Diseases. Pre-exposure prophylaxis (PrEP) to prevent HIV. Updated March 1, 2024. Accessed July 17, 2024. https://www.niaid.nih.gov/diseases-conditions/pre-exposure-prophylaxis-prep

Pre-exposure prophylaxis, shown to be effective through CDC research:

Nicol M, Adams J, Kashuba A. HIV PrEP trials: The road to success. Clinical Investigation. 2013; 3(3): 295-308. doi:10.4155/cli.12.155

US Centers for Disease Control and Prevention. TDF2 study of pre-exposure prophylaxis (PrEP) among heterosexual men and women in Botswana: Key facts. August 26, 2011. Accessed July 17, 2024. https://stacks.cdc.gov/view/cdc/23283

Pre-exposure prophylaxis, shown to be effective through USAID research: US Agency for International Development. PrEP. Accessed July 17, 2024: https://www.usaid.gov/global-health/health-areas/hiv-and-aids/technical-areas/prep

Approaches to prevent mother-to-child HIV transmission, developed with NIH support: NIH-sponsored study identifies superior drug regimen for preventing mother-to-child HIV transmission. Press release. National Institutes of Health; November 17, 2014. https://www.nih.gov/news-events/news-releases/nih-sponsored-study-identifies-superior-drug-regimen-preventing-mother-child-hiv-transmission

Approaches to prevent mother-to-child HIV transmission, 62% decline in new infections in children: UNAIDS. Global HIV & AIDS statistics — Fact sheet. UNAIDS; 2024. https://www.unaids.org/en/resources/fact-sheet

Long-acting PrEP options, monthly vaginal ring: Population Council. The Dapivirine Vaginal Ring for HIV prevention. Accessed July 17, 2024. https://popcouncil.org/project/the-dapivirine-vaginal-ring-for-hiv-prevention/

Long-acting PrEP options, every-two-month injectable: PrEPWatch. Injectable cabotegravir for PreP. Updated July 30, 2024. Accessed August 9, 2024. https://www.prepwatch.org/products/injectable-cab-for-prep/

Child-friendly therapies, combination therapy: Cipla and DNDi launch child-friendly 4-in-1 antiretroviral treatment for young children living with HIV in South Africa. Press release. Drugs for Neglected Diseases Initiative; June 14, 2022. https://dndi.org/press-releases/2022/cipla-dndi-launch-child-friendly-4-in-1-antiretroviral-treatment-young-children-hiv-south-africa/

Diagnostic innovations:

Unitaid, World Health Organization. Market and Technology Landscape 2020: HIV Rapid Diagnostic Tests for Self-Testing. Unitaid, World Health Organization; 2020. https://unitaid.org/assets/Unitaid-WHO-HIVST-landscape-report-2020.pdf

Unitaid, World Health Organization. HIV/AIDS Diagnostics Technology Landscape: 5th Edition. Unitaid, World Health Organization; 2015. http://www.unitaid.org/assets/UNITAID_HIV_Nov_2015_Dx_Landscape-1.pdf

Recency tests: Facente S, Grebe E, Maher D, et al. Use of HIV recency assays for HIV incidence estimation and other surveillance use cases: Systematic review. JMIR Public Health and Surveillance. 2022;8(3):e34410. https://doi.org/10.2196%2F34410

Continued progress is possible, not inevitable

Bill & Melinda Gates Foundation. Goalkeepers 2024 Report. The Race to Nourish a Warming World. Bill & Melinda Gates Foundation; 2024. https://www.gatesfoundation.org/goalkeepers/report/2024-report/

Key missing tools

AVAC. Prevention options. Accessed July 17, 2024. https://avac.org/prevention-options/

Breakthroughs on the brink

Long-acting biomedical products, once-weekly ARV pill, injectable drugs every four months, one weekly ARV pill: Mandavilli A. Long-acting drugs may revolutionize HIV prevention and treatment. The New York Times. April 17, 2024. Accessed July 18, 2024. https://www.nytimes.com/2024/04/17/health/hiv-long-acting-shots-pills.html

Long-acting biomedical products, three-month vaginal ring: HealthDay News. Vaginal ring may shield women from HIV for 3 months at a time. UPI. March 9, 2021. Accessed July 17, 2024. https://www.upi.com/Health_News/2021/03/09/vaginal-ring-protects-hiv-three-months-study/7961615322193/

Long-acting biomedical products, injectable drugs taken every six months: Gilead’s twice-yearly lenacapavir demonstrated 100% efficacy and superiority to daily Truvada® for HIV prevention. Press Release. Gilead; June 20, 2024. https://www.gilead.com/news-and-press/press-room/press-releases/2024/6/gileads-twiceyearly-lenacapavir-demonstrated-100-efficacy-and-superiority-to-daily-truvada-for-hiv-prevention

Long-acting biomedical products, implants: HIV.gov. Long-acting HIV prevention tools. Updated June 28, 2022. Accessed July 18, 2024. https://www.hiv.gov/hiv-basics/hiv-prevention/potential-future-options/long-acting-prep

Multipurpose prevention products, overall: AVAC. Advocates’ Guide to Multipurpose Prevention Technologies. AVAC; 2021. https://avac.org/resource/advocates-guide-to-multipurpose-prevention-technologies/

Multipurpose prevention products, dual prevention pill: Population Council. Dual prevention pill for the prevention of HIV and unintended pregnancy. Accessed July 18, 2024. https://popcouncil.org/project/dual-prevention-pill-for-the-prevention-of-hiv-and-unintended-pregnancy/

Multipurpose prevention products, multipurpose vaginal ring: Population Council. The Dapivirine-Levonorgestrel vaginal ring for HIV prevention and contraception. Accessed July 18, 2024. https://popcouncil.org/project/the-dapivirine-levonorgestrel-vaginal-ring-for-hiv-prevention-and-contraception/

Multipurpose prevention products, USAID support: US Agency for International Development. Microbicides: New HIV prevention products for women. Accessed July 18, 2024. https://www.usaid.gov/basic-page/microbicides-new-hiv-prevention-products-women-0

Broadly neutralizing antibodies, overall:

AVAC. Antibody related research. Accessed July 18, 2024. https://avac.org/prevention-option/antibody-related-research/

HIV Prevention Clinical Trials Database. AVAC; 2024. Accessed July 18, 2024. https://avac.org/hiv-prevention-clinical-trials-database/?_prevention_options=antibody-related-research&_pxrd_trial_status=ongoing

Broadly neutralizing antibodies, trials with NIH support: AVAC. Broadly Neutralizing Antibody Combinations. AVAC; 2021. https://avac.org/resource/infographic/broadly-neutralizing-antibody-combinations-2/

mRNA-based HIV vaccine candidates:

AVAC. Phase 1 mRNA HIV Vaccine Trials. AVAC; 2023. https://avac.org/resource/report/phase-1-mrna-hiv-vaccine-trials/

Jefferys R. Pipeline Report 2023: HIV Vaccines and Passive Immunization. Treatment Action Group; 2023. https://www.treatmentactiongroup.org/wp-content/uploads/2023/07/pipeline_HIV_VAX_2023_final.pdf

New vaccine strategies:

AVAC. Vaccines for prevention. Accessed July 18, 2024. https://avac.org/prevention-option/vaccines/

AVAC. HIV Vaccines: An Introductory Factsheet. AVAC; 2023. https://avac.org/resource/hiv-vaccines-an-introductory-factsheet/

Russel S. A promising HIV vaccine candidate gets a little help. Fred Hutch New Service. May 24, 2023. Accessed July 18, 2024. https://www.fredhutch.org/en/news/center-news/2023/05/hiv-vaccine-broadly-neutralizing.html

Seven patients cured/in remission: AmfAR. Curing HIV—How Far Have We Come? July 31, 2024. Accessed July 18, 2024. https://www.amfar.org/news/how-many-have-been-cured/

Cures strategies:

AVAC. Cure. Accessed July 18, 2024. https://avac.org/prevention-option/cure/

Treatment Action Group. Research Toward a Cure Trials. July 16, 2024. Accessed July 18, 2024. https://www.treatmentactiongroup.org/cure/trials/

Jefferys R. Pipeline Report 2023: Research Toward a Cure and Immune-Based Therapies. Treatment Action Group; 2023. https://www.treatmentactiongroup.org/wp-content/uploads/2023/07/pipeline_HIV_cures_ibt_2023_final.pdf

US government investment in HIV R&D for low-resource settings

G-FINDER data portal. Policy Cures Research; 2023. Accessed May 14, 2024. https://gfinderdata.policycuresresearch.org/

US Government R&D efforts

US federal funding for HIV/AIDS: Trends over time. Kaiser Family Foundation. March 26, 2024. Accessed July 18, 2024. https://www.kff.org/hivaids/fact-sheet/u-s-federal-funding-for-hivaids-trends-over-time/

Michaud J, Moss K, Kates J. The U.S. Department of Defense and Global Health. Kaiser Family Foundation; 2012. https://www.kff.org/global-health-policy/report/the-u-s-department-of-defense-global/

US Food & Drug Administration. President's Emergency Plan for AIDS Relief (PEPFAR). Updated May 15, 2022. Accessed July 18, 2024. https://www.fda.gov/international-programs/presidents-emergency-plan-aids-relief-pepfar

Malaria

R&D for Malaria

How new tools can transform the fight

About half of the world’s population is at risk of malaria—a life-threatening mosquito-borne disease that devastates communities and countries. In 2022, there were an estimated 249 million cases of malaria in 85 countries, claiming the lives of approximately 608,000 people—80% of whom were young children. Thanks to malaria control interventions, more than 1.5 billion malaria cases and 7.6 million deaths were successfully averted between 2000 and 2019. Yet, despite this progress, challenges like growing drug and insecticide resistance hinder control efforts. Unfortunately, 2022 saw a continued increase in new cases—indicating stalled progress and underscoring the urgent need for new solutions to complement existing interventions.

Nearly half

the world’s population is at risk
249 million

cases of malaria in 2022
608,000

deaths from malaria worldwide in 2022

Research successes

Technologies have transformed the fight against malaria:

Vector control tools, like insecticide-treated nets (ITNs) and indoor residual spraying, have driven dramatic declines in malaria. Recently, a new generation of dual-ingredient ITNs was introduced to respond to rising insecticide resistance.
Artemisinin-based combination therapies (ACTs), first developed in the 1990s, have become the gold standard treatment. Child-friendly ACTs followed, like Coartem® Dispersible, which has saved more than 1 million child lives since 2009.
Seasonal malaria chemoprevention (SMC), the use of antimalarials for prevention, was first introduced in 2012. It has saved 700,000 child lives and prevented 160 million infections.
Rectal artesunate and injectable artesunate, key combined medicines addressing severe malaria, have saved more than 98,000 and 1.25 million additional child lives, respectively.
Two malaria vaccines have been approved since 2021. Eight countries now offer one in their childhood immunization programs.
Tafenoquine, a single-dose medicine for Plasmodium vivax malaria, was approved in 2018, with a pediatric version approved in 2022. A point-of-care diagnostic test to guide its use was approved in 2019
Since 2019, more than 1 billion genetically modified mosquitoes have been released globally to reduce malaria’s spread.

Continued progress is possible, not inevitable

Key missing tools

To end malaria, we need new prevention and treatment tools including:

New vector control tools, including new and longer-lasting insecticides to address resistance and biological control methods to reduce mosquito populations.
New treatments and approaches to stem drug resistance, including artemisinin alternatives, novel regimens, easier-to-administer solutions like single-dose cures, and innovative delivery approaches to reduce pressure on existing treatments.
Novel drugs to block transmission and prevent reinfection, endectocides to kill the mosquito vector, and more antimalarials approved for children and pregnant individuals.
Novel, single-dose preventative therapies like monoclonal antibodies and long-acting injectables, which can complement other prevention tools like bednets, vaccines, and SMC.
Improved rapid diagnostic tests for low-resource settings that can detect all stages and all species of malaria equally well for early, accurate diagnosis and effective surveillance.
Next-generation malaria vaccines with longer duration and/or increased efficacy, including vaccines that block human-to-mosquito transmission of the parasite.

Breakthroughs on the brink

Three promising monoclonal antibodies for malaria, all supported by NIH, are in development, as well as several long-acting injectable malaria prevention drugs, including one supported by NIH, which could not only simplify and improve malaria prevention by eliminating the need for daily pills but could also reduce the emergence of drug resistance.
More than a dozen malaria vaccine candidates are in late-stage clinical development, including candidates supported by NIH, DoD, and USAID.
A novel vector control solution called Attractive Targeted Sugar Bait, which uses plant sugars to attract mosquitoes combined with an ingestible toxin that kills them but is safe for humans, holds promise to address the growing threat of outdoor biting by mosquitoes. The tool, which is commercially available in the United States, is undergoing validation testing for malaria control in Africa.
A first-of-its-kind test that diagnoses malaria using a patient’s saliva, rather than blood, is in commercial development. Developed with NIH support, the test is less invasive than other methods and delivers results quickly at the point of care.
A first-of-its-kind malaria treatment designed specifically for babies less than 10 pounds has completed Phase 2/3 trials. There is no approved treatment for small babies, who are currently treated with a partial dose of medicines made for larger children, which can heighten the risk of overdose among this very vulnerable population.
A new malaria treatment combination, gananplacide/lumefantrine-SDF, which consists of a novel drug and a new, optimized formulation of an existing drug, is currently in Phase 3 trials. It has the potential to clear malaria infection, including drug-resistant strains, and block transmission of the malaria parasite.
Progress is being made toward additional simpler dosing treatment regimens that could require as little as one dose.

US government investment in malaria R&D (in 2018) US$ millions

US Government R&D efforts

The US government is leading efforts to advance research and development (R&D) to combat malaria through a whole-of-government approach:

National Institutes of Health (NIH) conducts basic and clinical research for new treatments, vaccines, diagnostics, and vector control products.
Department of Defense (DoD) undertakes research to protect US service members against malaria—the leading infectious disease threat to US service members abroad—including drug, vaccine, and vector control research.
US Agency for International Development (USAID) leads the interagency President’s Malaria Initiative (PMI) and supports the development of new vaccines, antimalarials, insecticides, and novel vector control tools for low-resource settings.
Centers for Disease Control and Prevention jointly implements PMI, conducts surveillance and monitoring research, and develops and evaluates malaria control interventions such as bednets and other tools to improve public health efforts.
Food and Drug Administration administers the Tropical Disease Priority Review Voucher Program to incentivize investment in products for neglected diseases, including malaria.

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Introduction

249 million cases in 2022, 608,000 deaths in 2022: World Health Organization. Malaria. Updated December 4, 2023. Accessed July 17, 2024. https://www.who.int/news-room/fact-sheets/detail/malaria

Nearly half of world’s population at risk: US Centers for Disease Control and Prevention. Malaria’s impact worldwide. Updated April 1, 2024. Accessed July 17, 2024. https://www.cdc.gov/malaria/php/impact/index.html

1.5 billion cases, 7.6 million deaths averted: World Health Organization. World Malaria Report 2020: 20 Years of Global Heath Progress & Challenges. World Health Organization; 2020. https://www.who.int/publications/i/item/9789240015791

Continued increase in cases: World Health Organization. World Malaria Report 2023. World Health Organization; 2023. https://www.who.int/teams/global-malaria-programme/reports/world-malaria-report-2023

Research successes

Vector-control tools, driven dramatic declines in malaria: Bhatt S, Weiss DJ, Cameron E, et al. The effect of malaria control on Plasmodium falciparum in Africa between 2000 and 2015. Nature. 2015;526:207–211. https://doi.org/10.1038/nature15535

Vector-control tools, recent introduction of new generation of dual-ingredient ITNs: World Health Organization. World Malaria Report 2023. World Health Organization; 2023. https://www.who.int/teams/global-malaria-programme/reports/world-malaria-report-2023

Artemisinin-based combination therapies:

Medicines for Malaria Venture. History of antimalarial drugs. Accessed July 17, 2024. https://www.mmv.org/malaria-medicines/history-antimalarials-drugs

Tse EG, Korsik M, Todd MH. The past, present and future of anti-malarial medicines. Malaria Journal. 2019;18(93). doi:10.1186/s12936-019-2724-z

Artemisinin-based combination therapies, Coartem® dispersible: Medicines for Malaria Venture. Antimalarial drugs for children. Accessed July 22, 2024. https://www.mmv.org/our-work/antimalarial-drugs-children

Artemisinin-based combination therapies, Coartem® dispersible, 1 million lives saved: Data provided from Medicines for Malaria Venture.

Seasonal malaria chemoprevention, 700,000 children saved, 160 million infections prevented: SMC Alliance. From Concept to Scale: Celebrating 10 Years of Seasonal Malaria Chemoprevention. SMC Alliance; 2023. https://www.smc-alliance.org/resources/from-concept-to-scale-celebrating-10-years-of-seasonal-malaria-chemoprevention

Rectal artesunate:

Medicines for Malaria Venture. Artesunate rectal capsules. Accessed August 12, 2024. https://www.mmv.org/mmv-pipeline-antimalarial-drugs/artesunate-rectal-capsules-0

Medicines for Malaria Venture. Artesunate rectal capsules, Artecap™, Larinate® 60 mg for injection. Accessed August 12, 2024. https://www.mmv.org/mmv-pipeline-antimalarial-drugs/artesunate-rectal-capsules

Lives saved is compared to prior standard of care.

Injectable artesunate:

Medicines for Malaria Venture. Artesunate for injection, Larinate® 60 mg for injection. Accessed August 12, 2024. https://www.mmv.org/mmv-pipeline-antimalarial-drugs/artesunate-injection

Medicines for Malaria Venture. Artesunate for injection, Artesun®. Accessed August 12, 2024. https://www.mmv.org/mmv-pipeline-antimalarial-drugs/artesunate-injection-0

Rectal artesunate, injectable artesunate, lives saved: Data provided from Medicines for Malaria Venture. Lives saved is compared to prior standard of care.

Two approved malaria vaccines: Malaria vaccines (RTS,S and R21). World Health Organization. July 19, 2024. Accessed July 24, 2024. https://www.who.int/news-room/questions-and-answers/item/q-a-on-rts-s-malaria-vaccine

Tafenoquine, approval: US FDA approves Krintafel (tafenoquine) for the radical cure of P. vivax malaria. Press release. GSK; July 20, 2018. https://www.gsk.com/en-gb/media/press-releases/us-fda-approves-krintafel-tafenoquine-for-the-radical-cure-of-p-vivax-malaria/

Tafenoquine, pediatric version: Single-dose Kozenis (tafenoquine) approved for children with Plasmodium vivax malaria by Australian Therapeutic Goods Administration. Press release. Medicines for Malaria Venture; March 14, 2022. https://www.mmv.org/newsroom/news-resources-search/single-dose-kozenis-tafenoquine-approved-children-plasmodium-vivax

Tafenoquine, companion diagnostic test approval: Expert review panel for diagnostics approval expands access to a G6PD deficiency test that supports P. vivax malaria treatment and elimination initiatives. PATH. October 3, 2019. Accessed July 22, 2024. https://www.path.org/our-impact/articles/expert-review-panel-diagnostics-approval-expands-access-g6pd-deficiency-test-supports-p-vivax-malaria-treatment-and-elimination-initiatives/

Genetically modified mosquitoes:

Wangira D. GMO mosquitoes released in Djibouti to fight malaria. BBC. May 23, 2024. Accessed July 22, 2024. https://www.bbc.com/news/articles/cw551yelwz5o

Nolen S. The gamble: Can genetically modified mosquitoes end disease? The New York Times. September 29, 2023. Accessed July 22, 2024. https://www.nytimes.com/2023/09/29/health/mosquitoes-genetic-engineering.html

Continued progress is possible, not inevitable

Bill & Melinda Gates Foundation. Goalkeepers 2024 Report. The Race to Nourish a Warming World. Bill & Melinda Gates Foundation; 2024. https://www.gatesfoundation.org/goalkeepers/report/2024-report/

Key missing tools

World Health Organization. World Malaria Report 2023. World Health Organization; 2023. https://www.who.int/teams/global-malaria-programme/reports/world-malaria-report-2023

Based on consultation with topic area experts from GHTC member organizations.

Breakthroughs on the brink

Monoclonal antibodies:

Mundel T. The quest for transformative tools to eradicate malaria. The New England Journal of Medicine. 2024;390(17):1620-1621. doi:10.1056/NEJMe2402430

Global Health Technologies Coalition, Policy Cures Research. Doing Well by Doing Good: Why Investing in Global Health R&D Benefits the United States and the World. Global Health Technologies Coalition; 2024. https://www.ghtcoalition.org/resources-item/doing-well-by-doing-good-why-investing-in-global-health-r-d-benefits-the-united-states-and-the-world.

Johns Hopkins study supports potential for injectable ‘chemical vaccine’ for malaria using atovaquone. Press release. Johns Hopkins Bloomberg School of Public Health; October 20, 2023. https://publichealth.jhu.edu/2023/johns-hopkins-study-supports-potential-for-injectable-chemical-vaccine-for-malaria-using-atovaquone

Malaria vaccine candidates: Infectious disease R&D tracker. 2023. Accessed July 22, Policy Cures Research; 2024. https://www.policycuresresearch.org/pipeline-database/

Attractive Targeted Sugar Bait:

Innovative Vector Control Consortium. Attractive Targeted Sugar Baits (ATSB®). Accessed July 22, 2024. https://www.ivcc.com/research-development/atsb/

Westham Co. Accessed July 22, 2024. https://westhamco.com/

Saliva-based malaria test: Diaz J, Gusto C, McCoy K, et al. A mixed methods study assessing the adoption potential of a saliva-based malaria rapid test in the Democratic Republic of Congo. Malaria Journal. 2023;22(180). https://doi.org/10.1186/s12936-023-04599-y

Malaria treatment for babies under 10 pounds: Medicines for Malaria Venture and Novartis announce positive efficacy and safety data for a novel treatment for babies <5 kg with malaria. Press release. Medicines for Malaria Venture; April 24, 2024. https://www.mmv.org/newsroom/news-resources-search/medicines-malaria-venture-and-novartis-announce-positive-efficacy

Simpler dosing treatment regimens:

Medicines for Malaria Venture. MMV533. Accessed July 22, 2024. https://www.mmv.org/mmv-pipeline-antimalarial-drugs/mmv533

Medicines for Malaria Venture. MMV183. Accessed July 22, 2024. https://www.mmv.org/mmv-pipeline-antimalarial-drugs/mmv183

US government investment in malaria R&D

G-FINDER data portal: Tracking funding for global health R&D. Policy Cures Research; 2023. Accessed July 22, 2024. https://gfinderdata.policycuresresearch.org/

US government R&D efforts

USAID: The President’s Malaria Initiative and other US government global malaria efforts. KFF. December 20, 2023. Accessed July 22, 2024. https://www.kff.org/global-health-policy/fact-sheet/the-presidents-malaria-initiative-and-other-u-s-government-global-malaria-efforts/

DoD: The US Department of Defense & global health. KFF. September 29, 2012. Accessed July 22, 2024. https://www.kff.org/global-health-policy/report/the-u-s-department-of-defense-global/

FDA: US Food & Drug Administration. Tropical disease priority review voucher program. Updated June 13, 2024. Accessed July 22, 2024. https://www.fda.gov/about-fda/center-drug-evaluation-and-research-cder/tropical-disease-priority-review-voucher-program

Tuberculosis

R&D for Tuberculosis

How new tools can transform the fight

Tuberculosis (TB) has burdened humanity for thousands of years. Today, it is the world’s leading infectious disease killer: 10.8 million people fell ill with TB and 1.25 million died in 2023 alone. Additionally, growing resistance to available drugs is making the disease more deadly and difficult to treat.

To end the epidemic, new technologies to prevent, treat, and diagnose TB are urgently needed, including a new vaccine with better protective efficacy, shorter and safer treatment regimens, and improved diagnostics designed specifically for underserved populations and low-resource settings.

1.25 million

people die annually from TB
400,000 people

develop MDR-TB each year
$16.7 trillion

cost of MDR-TB to global economy by 2050

Research successes

Technologies have transformed the fight against TB:

Bedaquiline, a drug for multidrug-resistant TB (MDR-TB) developed with NIH and USAID support, was approved by the FDA in 2012. A pediatric version was later approved in 2020.
The first child-friendly TB medicines, developed with USAID support, were introduced in 2016. They are appropriately dosed, dissolvable, and fruit-flavored.
Pretomanid, a drug for highly drug-resistant TB (DR-TB), developed with USAID and NIH support, was approved by the FDA in 2019. Pretomanid-based regimens have dramatically improved DR-TB treatment outcomes and reduced treatment time from up to two years to six months.
A shorter, four-month treatment regimen for drug-susceptible TB, developed with CDC and NIH support, was endorsed by the World Health Organization in 2022, marking the first major advancement in drug-susceptible treatment in nearly 50 years.
3HP and 1HP, short-course preventative regimens to stop latent TB from becoming active, developed with CDC and NIH support, were introduced in 2011 and 2018, respectively, making treatment more tolerable and shorter (one to three months versus six).
New diagnostic innovations, including the Xpert and Truenat rapid, automated molecular tests, which are suitable for lower-level health facilities.

Continued progress is possible, not inevitable

Key missing tools

To end TB, we need new tools to detect, prevent, and treat infection, including:

Shorter, safer, and simpler treatment regimens for active TB that include both novel drugs and next-generation versions of existing drugs to improve tolerability, acceptability, adherence, and treatment outcomes and address rising drug resistance.
Specifically dosed and formulated treatment regimens for pregnant people and children.
New vaccines for prevention and treatment that are cost-effective, safe for pregnant people, and protect across ages. The current vaccine, developed in 1921, is effective at preventing some types of TB in infants, but it offers inconsistent protection in adults against pulmonary TB.
Rapid, non-sputum-based, point-of-care diagnostics that are accurate, affordable, and suitable for use in low-resource settings and primary health care facilities, as well as rapid DR-TB tests that enable treatment to be tailored to individuals and help safeguard against drug resistance.

Breakthroughs on the brink

Twenty-two new or repurposed drugs for TB are in clinical development, including 11 from a new class or with a new mechanism of action. Ongoing research will determine how to optimally combine this next generation of new drugs to further shorten the duration and improve the efficacy and safety of TB treatment regimens and preserve the longevity of new agents and drug classes against the development of drug resistance.
There are 17 potential preventative and therapeutic TB vaccines in development, including a vaccine candidate, M72/AS01E, currently in a Phase 3 trial, that shows promise in preventing pulmonary TB in adolescents and adults and, if proven successful, could become the first new TB vaccine in more than 100 years.
New approaches and strategies for TB vaccine research are invigorating the field, including research on new routes of administration, such as using inhaled, aerosolized TB vaccines and mRNA technology, and new models for vaccine testing, including preclinical animal and in vitro models that can mirror humans.
New, innovative methods to administer treatment, such as long-acting injectable agents, microarray patches, and implants, may lower the cost and burden of treatment by reducing the frequency and number of treatments that patients need. Nanomedicine is another area that could have the potential to reduce drug doses and side effects, improving treatment compliance.
More portable and affordable chest X-ray screening devices, point-of-care ultrasound devices, digital stethoscopes, and cough apps that use artificial intelligence are in the research pipeline, along with less-expensive, next-generation urine- and swab-based molecular tests, all of which could contribute to bringing care closer to patients and improving diagnostics access for underserved populations.

US government investment in tuberculosis R&D (in 2018) US$ millions

US Government R&D efforts

The US government is leading efforts to advance research and development (R&D) to end the TB epidemic through a whole-of-government approach:

National Institutes of Health (NIH) conducts basic, translational, and clinical research to accelerate the development of new tools to diagnose, prevent, and treat TB.
US Agency for International Development (USAID) supports R&D for new TB diagnostics and drugs. The agency also works with CDC and NIH to support basic and applied R&D for MDR-TB as part of the National Action Plan for Combating Multidrug-Resistant Tuberculosis.
Centers for Disease Control and Prevention (CDC) supports clinical and epidemiological research for TB through national and international partnerships, such as the TB Trials Consortium, which has supported the development and implementation of new TB technologies and significantly improved global TB treatment and prevention guidelines.
Department of Defense has funded research on TB vaccines, drugs, and diagnostics through the Congressionally Directed Medical Research Programs (CDMRP) and other contracts, but, to enable the former, TB must be added each year to the CDMRP list of eligible diseases.
Food and Drug Administration (FDA) administers the Tropical Disease Priority Review Voucher Program to incentivize investment in products for neglected diseases, including TB, and implements an expedited approval pathway for antibiotics that can apply to DR-TB products.

Download PDF

Introduction

10.8 million people fell ill, 1.25 million died, TB leading infectious killer: World Health Organization. Tuberculosis. Accessed October 31, 2024. https://www.who.int/news-room/fact-sheets/detail/tuberculosis

400,000 people develop MDR-TB: World Health Organization. Global Tuberculosis Report 2024. World Health Organization; 2024. https://www.who.int/teams/global-tuberculosis-programme/tb-reports/global-tuberculosis-report-2024

$16.7 trillion cost of MDR-TB: Zweynert A. Drug-resistant TB threatens to kill 75 million people by 2050, cost $16.7 trillion. Reuters. March 23, 2015. Accessed July 17, 2024. https://www.reuters.com/article/us-health-tuberculosis-economy/drug-resistant-tb-threatens-to-kill-75-million-people-by-2050-cost-16-7-trillion-idUSKBN0MK00520150324/

Research successes

Bedaquiline: Thomas K. F.D.A. Approves Drug for Resistant Tuberculosis. New York Times. December 31, 2012. Accessed July 17, 2024. https://www.nytimes.com/2013/01/01/business/fda-approves-new-tuberculosis-drug.html

Bedaquiline, pediatric version: U.S. FDA Approves New Pediatric Formulation of SIRTURO® (bedaquiline) as Part of Combination Therapy to Treat Children with Pulmonary Multidrug-Resistant Tuberculosis. Press release. Johnson & Johnson; May 27, 2020. https://www.jnj.com/media-center/press-releases/u-s-fda-approves-new-pediatric-formulation-of-sirturoo-bedaquiline-as-part-of-combination-therapy-to-treat-children-with-pulmonary-multidrug-resistant-tuberculosis

Child-friendly TB medicines: TB Alliance. Child-friendly medicines. Accessed July 17, 2023. https://www.tballiance.org/child-friendly-medicines

Pretomanid, new drug for highly drug-resistant TB: FDA Approves New Treatment for Highly Drug-Resistant Forms of Tuberculosis. Press release. TB Alliance; August 14, 2019. https://www.tballiance.org/news/fda-approves-new-treatment-highly-drug-resistant-forms-tuberculosis

Shorter, four-month treatment regimen: Mckenna L. Pipeline Report 2023: Tuberculosis Treatment. Treatment Action Group; 2023. https://www.treatmentactiongroup.org/wp-content/uploads/2024/03/pipeline_TB_Treatment_2023_final.pdf

3HP and 1 HP, short-course preventative regimens: Mckenna L. Pipeline Report 2023: Tuberculosis Treatment. Treatment Action Group; 2023. https://www.treatmentactiongroup.org/wp-content/uploads/2024/03/pipeline_TB_Treatment_2023_final.pdf

Diagnostic innovations, general: Branigan D. Pipeline Report 2023: Tuberculosis Diagnostics. Treatment Action Group; 2023. https://www.treatmentactiongroup.org/wp-content/uploads/2023/11/2023_pipeline_TB_diagnostics_final.pdf

Diagnostic innovations, Xpert: Stop TB Partnership Global Drug Facility. Xpert MTB/RIF and Ultra Technical Information Note. Stop TB Partnership Global Drug Facility; 2019. https://stoptb.org/assets/documents/gdf/drugsupply/Xpert_info_note.pdf

Diagnostic innovations, Truenat: Foundation for Innovative New Diagnostics. WHO endorses Truenat molecular diagnostic tests for TB. News Medical Life Sciences. July 3, 2020. Accessed July 17, 2024. https://www.news-medical.net/news/20200703/WHO-endorses-Truenat-molecular-diagnostic-tests-for-TB.aspx

Continued progress is possible, not inevitable

Bill & Melinda Gates Foundation. Goalkeepers 2024 Report. The Race to Nourish a Warming World. Bill & Melinda Gates Foundation; 2024. https://www.gatesfoundation.org/goalkeepers/report/2024-report/

Key missing tools

Madlen N, Branigan D. Getting Better Faster: Delivering on the Promise of New TB Treatments. 1/4/6X24 Campaign; 2024. https://www.treatmentactiongroup.org/wp-content/uploads/2024/03/146_report_final.pdf

World Health Organization. Global Tuberculosis Report 2023: Research & innovation. World Health Organization; 2023. https://www.who.int/teams/global-tuberculosis-programme/tb-reports/global-tuberculosis-report-2023/tb-research-and-innovation

Breakthroughs on the Brink

Twenty-two new or repurposed drugs: Mckenna L. Pipeline Report 2023: Tuberculosis Treatment. Treatment Action Group; 2023. https://www.treatmentactiongroup.org/wp-content/uploads/2024/03/pipeline_TB_Treatment_2023_final.pdf

17 potential preventative and therapeutic TB vaccines: Frick M. Pipeline Report 2023: Tuberculosis Vaccines. Treatment Action Group; 2023. https://www.treatmentactiongroup.org/wp-content/uploads/2023/10/2023_pipeline_TB_vaccines_final.pdf

17 potential preventative and therapeutic TB vaccines, M72/AS01E: Adepoju P. New TB vaccine on trial. Nature Africa. March 24, 2024. Accessed July 17, 2024. https://www.nature.com/articles/d44148-024-00101-1

New approaches and strategies for TB vaccine research, aerosolized TB vaccine: University of Oxford. Breakthrough aerosol human infection model gives hope for future tuberculosis vaccine development. University of Oxford. April 15, 2024. Accessed July 27, 2024. https://www.ox.ac.uk/news/2024-04-15-breakthrough-aerosol-human-infection-model-gives-hope-future-tuberculosis-vaccine

New approaches and strategies for TB vaccine research, mRNA technology: Aizenman N. Frozen cells reveal a clue for a vaccine to block the deadly TB bug. NPR Goats and Soda. March 6, 2023. Accessed July 17, 2024. https://www.npr.org/sections/goatsandsoda/2023/03/06/1161248152/it-appears-scientists-are-closer-to-making-a-vaccine-that-blocks-tuberculosis

New approaches and strategies for TB vaccine research, preclinical animal and in vitro models: Chugh S, Kar Bahal R, Dhiman R, Singh R. Antigen identification strategies and preclinical evaluation models for advancing tuberculosis vaccine development. NPJ Vaccines. 9, 57 (2024). https://doi.org/10.1038/s41541-024-00834-y

New innovative methods to administer treatment: Branigan D. Injectables Redux: Developing Acceptable Long-Acting Formulations for TB Prevention Amidst a Push for All-Oral Treatment. Treatment Action Group TAGline newsletter. October 2021. Accessed July 17, 2024. https://www.treatmentactiongroup.org/resources/tagline/tagline-october-2021/injectables-redux-developing-acceptable-long-acting-formulations-for-tb-prevention-amidst-a-push-for-all-oral-treatment/

New innovative methods to administer treatment, nanomedicine: Slater K B, Kim D, Chand P, et al. A Current Perspective on the Potential of Nanomedicine for Anti-Tuberculosis Therapy. Tropical Medicine and Infectious Disease. 2023; 8(2): 100. https://doi.org/10.3390%2Ftropicalmed8020100

Devices/diagnostics: Branigan D. Pipeline Report 2023: Tuberculosis Diagnostics. Treatment Action Group; 2023. https://www.treatmentactiongroup.org/wp-content/uploads/2023/11/2023_pipeline_TB_diagnostics_final.pdf

US government investment in tuberculosis R&D

G-FINDER data portal. Policy Cures Research; 2023. Accessed May 14, 2024. https://gfinderdata.policycuresresearch.org/

US government investment in tuberculosis R&D

All: Kaiser Family Foundation. The U.S. Government and Global Tuberculosis Efforts. Accessed July 17, 2024. https://www.kff.org/global-health-policy/fact-sheet/the-u-s-government-and-global-tuberculosis-efforts/

Department of Defense: Search Awards. Department of Defense Congressionally Directed Medical Research Programs; 2023. Accessed July 27, 2024. https://cdmrp.health.mil/search.aspx

Food and Drug Administration, Tropical Disease Priority Review Voucher: US Food and Drug Administration. Tropical Disease Priority Review Voucher Program. Accessed July 17, 2024. https://www.fda.gov/about-fda/center-drug-evaluation-and-research-cder/tropical-disease-priority-review-voucher-program

Food and Drug Administration, expedited approval pathway: US Food and Drug Administration. Limited Population Pathway for Antibacterial and Antifungal Drugs – the LPAD Pathway. Accessed July 17, 2024. https://www.fda.gov/drugs/development-resources/limited-population-pathway-antibacterial-and-antifungal-drugs-lpad-pathway

Neglected Tropical Diseases

R&D for Neglected Tropical Diseases

How new tools can transform the fight

Neglected tropical diseases (NTDs) are a group of diseases that exert a crushing burden in poor and underserved communities around the world. Named for the limited attention they have historically received, NTDs are inextricably linked to poverty and inequality. These diseases exact a heavy toll, causing chronic illness, severe disfigurement, blindness, or even death. They can also exacerbate malnutrition and delay cognitive development, as well as reduce economic productivity, trapping families and communities in poverty. Increased attention in recent years has brought new resources to the fight against NTDs and fueled research breakthroughs. Yet, very significant gaps remain in the arsenal of tools needed to control and eliminate these diseases, underscoring the need for the research and development (R&D) of new tools.

1 billion+people

worldwide are affected by NTDs
179 countries

reported an NTD in 2021
21 conditions

are classified as NTDs

Research successes

Technologies have transformed the fight against NTDs:

The world’s first vaccine for chikungunya, a disease causing debilitating joint pain, which was developed with DoD support, was approved in 2023.
A second dengue vaccine that is safe for use in individuals with and without prior infection was approved in 2022.
The first all-oral cure for sleeping sickness, a fatal neurological disease, was approved in 2018. Fexinidazole cures all stages of the disease in ten days, replacing prior treatments that involved two weeks of intravenous injections at a hospital.
The first new drug for river blindness in 20 years, moxidectrin, was approved in 2018 to treat this disfiguring and blinding disease.
Child-friendly drugs for schistosomiasis and Chagas disease were approved in 2024 and 2011, respectively, enabling children to receive appropriate care for these painful parasitic diseases.
New shorter, more effective combination therapies for visceral leishmaniasis—a disease causing spleen and liver damage—, introduced in the 2000s, are improving care.
New diagnostics, including rapid tests for river blindness and elephantiasis, developed with NIH research, and a test that detects all four types of dengue, developed by CDC, have been introduced.

Continued progress is possible, not inevitable

Key missing tools

To eliminate NTDs, we need new and improved tools, including:

New and improved diagnostics to rapidly detect infection at the point-of-care in low-resource settings and, in some cases, to distinguish between different strains or stages of a disease.
Vaccines to prevent and treat infection. Beyond dengue and chikungunya, no vaccines exist for the remaining 20 NTDs prioritized by the World Health Organization.
Antivenoms to treat snakebites.
New vector and biological control tools, like space spray insecticides and Wolbachia bacteria that disrupt reproduction, to reduce mosquito and other vector populations.
New and improved treatments and cures, including therapies designed for children and shorter, simplified regimens with fewer side effects. While there are low-cost, effective interventions to combat some NTDs, in many cases, these available treatments are still lengthy and burdensome with significant side effects.

Breakthroughs on the brink

An all-oral, single-dose treatment for sleeping sickness, acoziborole, which is in late-stage development, could simplify treatment administration, laying the groundwork for the eventual elimination of this deadly disease. A pediatric version of this medicine is also in development.
An oral treatment for dengue, developed with NIH support, showed promising results in Phase 2 clinical trials, offering hope that in the coming years, we will see the first-ever treatment for this disease.
Researchers have created a synthetically produced antibody that can neutralize a neurotoxin found in the venom of four different deadly snake species, laying the foundation for the eventual development of a universal antivenom for snakebites.
Several vaccines for schistosomiasis—a disease that causes liver damage or kidney failure if left untreated—are in development, including a candidate now in Phase 2 trials developed with NIH and DoD support.
Fosravuconazole, a potential new weekly treatment for eumycetoma, a fungal disease that can cause severe deformity of the limbs, demonstrated promising results in Phase 2 trials. Existing treatments for this disease are toxic, costly, only cure a fraction of patients, and require daily dosing.
Two NIH-funded vaccine candidates against hookworm infection are in clinical development, including one in Phase 2 development. This parasitic infection causes gastrointestinal issues and protein deficiency and is linked to impaired learning in children.
Several diagnostic tests for Buruli ulcer—a disease that causes painful ulcers and swelling—are in development, including an instrument-free, point-of-care test and other tools for remote health care settings. Today, diagnosis is typically done by appearance, which leads to high rates of misdiagnosis and delayed treatment.
Mosquitoes infected with Wolbachia, a bacteria that impact their reproduction and reduce population size, have been released to fight dengue and other mosquito-borne diseases in locations ranging from Indonesia to the Florida Keys, with early results showing promise. NIH and USAID have funded research into this approach.

US government investment in NTD R&D (in 2022) US$ millions

US Government R&D efforts

The US government is advancing R&D to control and eliminate NTDs through a whole-of-government approach:

National Institutes of Health (NIH) conducts R&D for new treatments, vaccines, diagnostics, vector control products, and other tools to combat NTDs.
Centers for Disease Control and Prevention (CDC) advances research to develop new and improved diagnostics and interventions to strengthen NTD control and elimination efforts.
Department of Defense (DoD) undertakes R&D to create vaccines, drugs, diagnostics, and vector control products for NTDs that threaten US service members stationed abroad.
US Agency for International Development (USAID) has in the past supported the development of new drugs and diagnostics for a select group of NTDs as part of its flagship NTD Program but largely focuses on deploying existing interventions.
Food and Drug Administration administers the Tropical Disease Priority Review Voucher Program to incentivize investment in products for NTDs.

Download PDF

Introduction

1 billion people affected; 21 conditions*: World Health Organization. Neglected tropical diseases page. Accessed July 17, 2024. https://www.who.int/health-topics/neglected-tropical-diseases

*WHO designated NTDs include: Buruli ulcer; Chagas disease; dengue and chikungunya; dracunculiasis; echinococcosis; foodborne trematodiases; human African trypanosomiasis; leishmaniasis; leprosy; lymphatic filariasis; mycetoma, chromoblastomycosis and other deep mycoses; noma; onchocerciasis; rabies; scabies and other ectoparasitoses; schistosomiasis; soil-transmitted helminthiases; snakebite envenoming; taeniasis/cysticercosis; trachoma; and yaws.

179 countries reported: More countries eliminate neglected tropical diseases but investments key to sustain progress. Press release. World Health Organization; January 30, 2023. https://www.who.int/news/item/30-01-2023-more-countries-eliminate-neglected-tropical-diseases-but-investments-key-to-sustain-progress

Research successes

World’s first vaccine for chikungunya: FDA Approves First Vaccine to Prevent Disease Caused by Chikungunya Virus. Press release. US Food and Drug Administration; November 9, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-vaccine-prevent-disease-caused-chikungunya-virus

Second dengue vaccine: Takeda’s QDENGA®▼ (Dengue Tetravalent Vaccine [Live, Attenuated]) Approved in Indonesia for Use Regardless of Prior Dengue Exposure. Press release. Takeda; August 22, 2022. https://www.takeda.com/newsroom/newsreleases/2022/takedas-qdenga-dengue-tetravalent-vaccine-live-attenuated-approved-in-indonesia-for-use-regardless-of-prior-dengue-exposure/

First all-oral cure for sleeping sickness: Drugs for Neglected Diseases initiative. Sleeping Sickness: Fexinidazole for T.b. gambiense. Accessed July 17, 2024. https://dndi.org/research-development/portfolio/fexinidazole/

New drug for river blindness: First new treatment for river blindness approved by U.S. FDA in 20 years. Press release. World Health Organization Special Programme for Research and Training in Tropical Diseases; June 14, 2018. https://tdr.who.int/newsroom/news/item/14-06-2018-first-new-treatment-for-river-blindness-approved-by-u-s-fda-in-20-years

Child-friendly drugs for schistosomiasis: The European Medicines Agency adopts positive opinion of our treatment option for schistosomiasis in preschool-aged children. Press release. Pediatric Praziquantel Consortium; December 15, 2023. https://www.pediatricpraziquantelconsortium.org/newsroom/european-medicines-agency-adopts-positive-opinion-our-treatment-option-schistosomiasis

Child-friendly drugs for Chagas disease: Drugs for Neglected Diseases initiative. Chagas disease: Paediatric Benznidazole. Accessed July 17, 2024. https://dndi.org/research-development/portfolio/paediatric-benznidazole/

Combination therapies for visceral leishmaniasis: Drugs for Neglected Diseases initiative. Visceral leishmaniasis. Accessed July 17, 2024. https://dndi.org/research-development/portfolio/ssg-pm/

Rapid tests for river blindness and elephantiasis: Innovative partnership brings to market new tools for neglected tropical diseases. Press release. PATH; April 11, 2016. https://www.path.org/our-impact/media-center/innovative-partnership-brings-to-market-new-tools-for-neglected-tropical-diseases/

Test that detects all four types of dengue: CDC Begins Distribution of New Dengue Fever Tests. *Global Biodefense. June 18, 2012. Accessed July 17, 2024. https://globalbiodefense.com/2012/06/28/cdc-begins-distribution-of-new-dengue-fever-tests/

Continued progress is possible, not inevitable

Bill & Melinda Gates Foundation. *Goalkeepers 2024 Report. The Race to Nourish a Warming World. Bill & Melinda Gates Foundation; 2024. https://www.gatesfoundation.org/goalkeepers/report/2024-report/

Key missing tools

Bill & Melinda Gates Foundation. *Goalkeepers 2024 Report. The Race to Nourish a Warming World. Bill & Melinda Gates Foundation; 2024. https://www.gatesfoundation.org/goalkeepers/report/2024-report/

Breakthroughs on the brink

Single-dose treatment for sleeping sickness: Drugs for Neglected Diseases initiative. Sleeping sickness: Acoziborole. Accessed July 17, 2024. https://dndi.org/research-development/portfolio/acoziborole/

Oral treatment for dengue: Janssen Announces Promising Antiviral Activity Against Dengue in a Phase 2a Human Challenge Model. Press release. Johnson & Johnson; October 20, 2023. https://www.jnj.com/media-center/press-releases/janssen-announces-promising-antiviral-activity-against-dengue-in-a-phase-2a-human-challenge-model

Antivenom: Wilcox, C. Powerful new antivenom raises hopes for a universal solution to lethal snakebites. *Science. February 21, 2024. Accessed July 17, 2024. https://www.science.org/content/article/powerful-new-antivenom-raises-hopes-universal-solution-lethal-snakebites

Several vaccines for schistosomiasis: Infectious Disease R&D Tracker. Policy Cures Research; 2023. Accessed July 17, 2024. https://www.policycuresresearch.org/pipeline-database/

Potential new weekly treatment for eumycetoma: Drugs for Neglected Diseases initiative. Mycetoma: Fosravuconazole. Accessed July 17, 2024. https://dndi.org/research-development/portfolio/fosravuconazole/

Vaccine candidates against hookworm: Infectious Disease R&D Tracker. Policy Cures Research; 2023. Accessed July 17, 2024. https://www.policycuresresearch.org/pipeline-database/

Vaccine candidates against hookworm, candidate in phase II development/NIH funding: Efficacy of Na-GST-1/Alhydrogel Hookworm Vaccine Assessed by Controlled Challenge Infection. ClinicalTrials.gov identifier. NCT03172975. Accessed July 17, 2024. https://clinicaltrials.gov/study/NCT03172975

Diagnostic tests for Buruli ulcer: Infectious Disease R&D Tracker. Policy Cures Research; 2023. Accessed July 17, 2024. https://www.policycuresresearch.org/pipeline-database/

Diagnostic tests for Buruli ulcer, instrument-free, point-of-care test: FIND. Rapid tests for Buruli ulcer control. Accessed July 17, 2024. https://www.finddx.org/what-we-do/projects/rapid-tests-for-buruli-ulcer-control/

Mosquitoes infected with *Wolbachia: World Mosquito Program. Accessed July 17, 2024. https://www.worldmosquitoprogram.org/

Mosquitoes infected with *Wolbachia, NIH support: National Institute of Health National Institute of Allergy and Infectious Diseases. Neglected Tropical Diseases Scientific Findings. Accessed July 17, 2024. https://www.niaid.nih.gov/research/neglected-tropical-diseases-scientific-findings

Mosquitoes infected with *Wolbachia, USAID support: USAID. *Combating Zika and Future Threats: A Grant Challenge for Development: Using Wolbachia to prevent disease transmission. USAID; 2021. https://www.usaid.gov/sites/default/files/2022-05/2a_-_Lessons_Learned_on_Supporting_Crisis-Related_Innovations_-_MAY_2021_-_WMP_Success_Story_FINAL_1.pdf

US government investment in NTD R&D

G-FINDER data portal. Policy Cures Research; 2023. Accessed May 14, 2024. https://gfinderdata.policycuresresearch.org/

US government R&D efforts

Kaiser Family Foundation. The U.S. Government and Global Neglected Tropical Disease Efforts. Accessed July 17, 2014. https://www.kff.org/global-health-policy/fact-sheet/the-u-s-government-and-global-neglected-tropical-diseases/

US Food and Drug Administration. Tropical Disease Priority Review Voucher Program. Accessed July 17, 2014. https://www.fda.gov/about-fda/center-drug-evaluation-and-research-cder/tropical-disease-priority-review-voucher-program

Emerging Infectious Diseases

R&D for Emerging Infectious Diseases

How new tools can transform the fight

In a world experiencing increasing human mobility and ecological change, emerging infectious diseases (EIDs) pose a growing threat to health security. As we saw with COVID-19, an outbreak in one corner of the world can quickly become a global pandemic with devastating human and economic costs. New vaccines, treatments, diagnostics, and other tools are urgently needed to outsmart epidemics. However, research and development (R&D) for EIDs is particularly challenging.

The market potential for tools against EIDs is too uncertain to drive commercial investment, and testing these products is especially difficult. Strong government support and smart collaboration between nations are vital to overcome these barriers and develop the tools needed to prevent, detect, and respond to these threats.

$12.5 trillion

global economic cost of COVID-19
11,000+ lives

lost from 2014-16 West Africa Ebola outbreak
3x projected increase

in annual probability of extreme epidemics in coming decades

Key missing tools

We are without essential tools to combat many EIDs considered most likely to cause the next epidemic:

	Vaccines	Treatments	Diagnostics
COVID-19	Several vaccines are approved but will require routine updates for variants.	Oral and intravenous treatments are approved, but continued innovation is needed to optimize treatment and address variants.	Several rapid, point-of-care (POC) tests and laboratory-based tests are available, but continued validation is needed for new variants.
Crimean-Congo Haemorrhagic fever (CCHF)	No FDA-approved vaccine is available. A Bulgarian-licensed vaccine exists, but its efficacy is unclear	No specific licensed treatment is available. An existing antiviral has been used during outbreaks, but its efficacy remains unclear.	Commercial tests are available but require sophisticated laboratory infrastructure. Rapid POC tests are needed.
Ebola Sudan	No licensed vaccine.	No specific licensed treatment.	Confirmatory tests are available but require sophisticated laboratory infrastructure. Rapid POC tests are needed.
Ebola Zaire	Two vaccines are approved by stringent regulators.	Two licensed treatments are FDA-approved and in use globally.	Several rapid POC tests and laboratory-based tests are in use globally, including FDA-approved and -authorized options.
Lassa fever	No licensed vaccine.	No specific licensed treatment. The IV-administered antiviral ribavirin is effective if used early, but more robust research is needed, as are oral antivirals.	Confirmatory tests are available but require sophisticated laboratory infrastructure, and validation is needed for different lineages. Rapid POC tests are needed.
Marburg	No licensed vaccine.	No specific licensed treatment.	Commercial tests are available but require sophisticated laboratory infrastructure. Rapid POC tests are needed.
Middle East respiratory syndrome (MERS)	No licensed vaccine.	No specific licensed treatment.	Commercial tests are available, including FDA-authorized options, but they require sophisticated laboratory infrastructure. Rapid POC tests are needed.
Mpox	Three vaccines are approved by stringent regulators, two of which are FDA-approved.	No specific licensed treatment. An FDA-approved smallpox drug that has been used on a compassionate use basis was found ineffective.	Commercial tests are available, including FDA-authorized options, but they require sophisticated laboratory infrastructure or specialized testing systems. Further validation is needed for different clades, as are additional rapid POC tests.
Nipah	No licensed vaccine.	No specific licensed treatment.	Commercial tests are available but require sophisticated laboratory infrastructure. Rapid POC tests are needed.
Rift Valley fever (RVF)	No licensed human vaccine.	No specific licensed treatment.	Commercial tests are available but require sophisticated laboratory infrastructure. Rapid POC tests are needed.
Severe Acute Respiratory Syndrome (SARS)	No licensed vaccine.	No specific licensed treatment.	Commercial tests are available but require sophisticated laboratory infrastructure. Rapid POC tests are needed.
Zika	No licensed vaccine.	No specific licensed treatment.	Rapid POC tests and laboratory-based tests are available, including FDA-approved or -authorized options.
Pandemic influenza	No licensed universal vaccine. Existing flu vaccines may offer some cross protection.	Existing antivirals may be effective. Additional broad-spectrum antivirals are needed.	Confirmatory tests are available but not all commercially. Rapid POC tests will be needed.

Research progress

Several COVID-19 vaccines, therapeutics, and rapid POC diagnostic tests were developed with cross-US government support, significantly changing the course of the pandemic. Next-generation nasal COVID-19 vaccines, combination influenza and COVID-19 vaccines, and pan-coronavirus vaccines are now in development with US support.
Two licensed vaccines, two approved therapeutics, and a rapid POC test for Ebola Zaire were developed via various partnerships with NIH, DoD, and BARDA. Since their introduction, these tools have helped bring subsequent outbreaks under control more quickly, dramatically reducing their scale and toll.
A promising vaccine for Marburg—a deadly cousin of the Ebola virus—which was developed by NIH, advanced with BARDA support, and trialed at a DoD-supported overseas lab, was deployed under clinical trial protocols to respond to the 2024 outbreak in Rwanda. A vaccine for Ebola Sudan that is built on the same vaccine platform is also undergoing a Phase 2 trial with US support.
Thanks to investments from BARDA, the United States was prepared with an approved mpox vaccine when the global epidemic hit. Researchers are now advancing an adapted version of this vaccine for children.
Vaccines for several other EIDs have recently entered or advanced in clinical trials, including the first human trial of a Nipah vaccine, the first Phase 2 study of a Lassa vaccine, and a Phase 2 trial of an mRNA-based Zika vaccine.
Clinical trials are underway for several potential universal flu vaccine candidates, including an mRNA-based candidate developed at NIH’s Vaccine Research Center.
Numerous diagnostic tools for EIDs beyond Ebola and COVID-19 have been advanced to market, including four tests for Zika that have full FDA approval, as well as 14 tests for Zika, 8 for mpox, and 2 for MERS that received Emergency Use Authorization.

US government investment in R&D for EIDs (in 2022) US$ millions

US Government R&D efforts

The US government is developing tools to improve health security through a whole-of-government approach:

Biomedical Advanced Research and Development Authority (BARDA) supports the advanced development of vaccines, drugs, diagnostics, and other medical countermeasures against EIDs considered a threat to national security.
National Institutes of Health (NIH) conducts basic and clinical research to advance new treatments, vaccines, diagnostics, and vector control products to combat EIDs.
Department of Defense (DoD) supports R&D to create vaccines, drugs, and other tools for EIDs considered a threat to US service members or to national security.
Centers for Disease Control and Prevention works to develop new and improved diagnostics and surveillance tools to improve global capacity to prevent, detect, and respond to outbreaks.
US Agency for International Development has supported the development of select tools to combat EIDs designed for low-resource settings and contributes to the Coalition for Epidemic Preparedness Innovations (CEPI), a global vaccine development partnership.
Food and Drug Administration (FDA) administers the Tropical Disease and Material Threat Medical Countermeasures Priority Review Programs to incentivize industry investment in products for select EIDs and grants Emergency Use Authorization to permit the use of not-yet-approved products during an emergency.

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Introduction

$12.5 trillion cost: Reuters. IMF sees cost of COVID pandemic rising beyond $12.5 trillion estimate. Reuters. January 20, 2022. Accessed July 22, 2024. https://www.reuters.com/business/imf-sees-cost-covid-pandemic-rising-beyond-125-trillion-estimate-2022-01-20/

11,000+ lives lost Ebola: World Health Organization. Ebola: West Africa, March 2014-2016. Accessed July 22, 2024. https://www.who.int/emergencies/situations/ebola-outbreak-2014-2016-West-Africa

3x project increase in probability of epidemics: Joi P. New study suggests risk of extreme pandemics like COVID-19 could increase threefold in coming decades. Vaccines Work blog. September 5, 2022. Accessed July 22, 2024. https://www.gavi.org/vaccineswork/new-study-suggests-risk-extreme-pandemics-covid-19-could-increase-threefold-coming

Key missing tools

COVID-19: MedicalCountermeasures.gov. COVID-19 Portfolio. Accessed July 22, 2024. https://medicalcountermeasures.gov/app/barda/coronavirus/COVID19.aspx

Crimean-Congo Haemorrhagic fever:

World Health Organization. Crimean-Congo haemorrhagic fever. Accessed July 22, 2024. https://www.who.int/news-room/fact-sheets/detail/crimean-congo-haemorrhagic-fever

World Health Organization. Crimean-Congo haemorrhagic fever Research and Development (R&D) Roadmap. World Health Organization; 2019. https://www.who.int/publications/m/item/crimean-congo-haemorrhagic-fever-(cchf)-research-and-development-(r-d)-roadmap

Ebola Sudan: Ibrahim S, Ndwandwe D, Thomas K, et al. Sudan virus disease outbreak in Uganda: urgent research gaps. BMJ Global Health. 2022;7:e010982. https://doi.org/10.1136/bmjgh-2022-010982

Ebola Zaire:

MedicalCountermeasures.gov. FDA Approvals, Licensures & Clearances, BARDA Supported Products: Accessed July 22, 2024. https://medicalcountermeasures.gov/barda/fdaapprovals

MedicalCountermeasures.gov. Global collaboration with BARDA scientists successfully develop Ebola countermeasures. Medicalcountermeasures.gov. June 21, 2021. Accessed July 25, 2024. https://medicalcountermeasures.gov/stories/ebola-story/

US Food and Drug Administration. Emergency Use Authorization. Accessed July 22, 2024. https://www.fda.gov/emergency-preparedness-and-response/mcm-legal-regulatory-and-policy-framework/emergency-use-authorization#ebola

Lassa fever: World Health Organization. Lassa fever. Accessed July 25, 2024. https://www.who.int/news-room/fact-sheets/detail/lassa-fever

Marburg: World Health Organization. Marburg virus disease. Accessed July 25, 2024. https://www.who.int/news-room/fact-sheets/detail/marburg-virus-disease

Middle East respiratory syndrome:

World Health Organization. Middle East respiratory syndrome coronavirus (MERS-CoV). Accessed July 25, 2024. https://www.who.int/health-topics/middle-east-respiratory-syndrome-coronavirus-mers

US Food and Drug Administration. Emergency Use Authorization. Accessed July 22, 2024. https://www.fda.gov/emergency-preparedness-and-response/mcm-legal-regulatory-and-policy-framework/emergency-use-authorization

Mpox:

Administration for Strategic Preparedness & Response. SNS Products: Vaccines and Treatment Available for Use in the Mpox Response. Accessed July 22, 2024. https://aspr.hhs.gov/SNS/Pages/Mpox.aspx

National Institutes of Health National Institute of Allergy and Infectious Diseases. Mpox (Formerly Monkeypox) Vaccines. Accessed July 25, 2024. https://www.niaid.nih.gov/diseases-conditions/mpox-vaccines

World Health Organization. Mpox Questions and Answers. Accessed September 13, 2024. https://www.who.int/news-room/questions-and-answers/item/mpox

Mast J. Antiviral used for mpox no better than placebo, NIH says. STAT. August 15, 2024. Accessed September 13, 2024. https://www.statnews.com/2024/08/15/mpox-antiviral-tecovirimat-no-better-than-placebo-nih/

US Food and Drug Administration. Monkeypox (mpox) Emergency Use Authorizations for Medical Devices. Accessed July 22, 2024. https://www.fda.gov/medical-devices/emergency-use-authorizations-medical-devices/monkeypox-mpox-emergency-use-authorizations-medical-devices

Nipah:

World Health Organization. Nipah virus. Accessed July 25, 2024. https://www.who.int/news-room/fact-sheets/detail/nipah-virus

World Health Organization. Nipah Research and Development (R&D) Roadmap. World Health Organization; 2019. https://cdn.who.int/media/docs/default-source/blue-print/nipah_rdblueprint_roadmap_advanceddraftoct2019.pdf?sfvrsn=4f0dc9ad_3&download=true

Rift Valley fever:

World Health Organization. Rift Valley Fever. Accessed July 25, 2024. https://www.who.int/news-room/fact-sheets/detail/rift-valley-fever

Petrova V, Kristiansen P, Northeim G, Yimer A. Rift valley fever: diagnostic challenges and investment needs for vaccine development. BMJ Global Health. 2020:5:e002694 https://doi.org/10.1136/bmjgh-2020-002694

Severe Acute Respiratory Syndrome: World Health Organization. Severe Acute Respiratory Syndrome (SARS). Accessed July 25, 2024. https://www.who.int/health-topics/severe-acute-respiratory-syndrome

Zika:

World Health Organization. Zika virus. Accessed July 25, 2024. https://www.who.int/news-room/fact-sheets/detail/zika-virus

US Food and Drug Administration. Emergency Use Authorization. Accessed July 22, 2024. https://www.fda.gov/emergency-preparedness-and-response/mcm-legal-regulatory-and-policy-framework/emergency-use-authorization#zika

Pandemic influenza:

Fuller D. How do you make a universal flu vaccine? A microbiologist explains the challenges, and how mRNA could offer a promising solution. The Conversation. February 7, 2023. Accessed July 25, 2024. https://theconversation.com/how-do-you-make-a-universal-flu-vaccine-a-microbiologist-explains-the-challenges-and-how-mrna-could-offer-a-promising-solution-195807

Jones J, Yen H, Adams P, et al. Influenza Antivirals and Their Role in Pandemic Preparedness. Antiviral Research. 2023 Feb;210:105499. https://doi.org/10.1016/j.antiviral.2022.105499

Diagnostics, all: Consultation with topic area experts from FIND.

Research progress

COVID-19 vaccines, therapeutics, rapid POC tests: MedicalCountermeasures.gov. COVID-19 Portfolio. Accessed July 22, 2024. https://medicalcountermeasures.gov/app/barda/coronavirus/COVID19.aspx

Next-generation COVID-19 vaccines:

MedicalCountermeasures.gov. Project NextGen: Next Generation Medical Countermeasures. Accessed July 22, 2024. https://medicalcountermeasures.gov/nextgen

Moderna Announces First Participant Dosed in Phase 3 Study of mRNA-1083, a Combination Vaccine Against Influenza and COVID-19. Press release. Moderna; October 24, 2023. https://investors.modernatx.com/news/news-details/2023/Moderna-Announces-First-Participant-Dosed-in-Phase-3-Study-of-mRNA-1083-a-Combination-Vaccine-Against-Influenza-and-COVID-19/default.aspx

Ebola Sudan vaccine:

Sabin Vaccine Institute Begins Phase 2 Clinical Trial for Sudan Ebolavirus Vaccine. Press release. Sabin Vaccine Institute; July 15, 2024: https://www.sabin.org/resources/sabin-vaccine-institute-begins-phase-2-clinical-trial-for-sudan-ebolavirus-vaccine/

Sabin Vaccine Institute Delivers Marburg Vaccines to Combat Outbreak in Rwanda. Press release. Sabin Vaccine Institute; October 5, 2024. https://www.sabin.org/resources/sabin-vaccine-institute-delivers-marburg-vaccines-to-combat-outbreak-in-rwanda/

Marburg vaccine: Sabin Vaccine Institute Begins Phase 2 Clinical Trial for Marburg Vaccine in Uganda. Press release. Sabin Vaccine Institute; October 19, 2024. https://www.sabin.org/resources/sabin-vaccine-institute-begins-phase-2-clinical-trial-for-marburg-vaccine-in-uganda/

Mpox vaccine: Administration for Strategic Preparedness & Response. SNS Products: Vaccines and Treatment Available for Use in the Mpox Response. Accessed July 22, 2024. https://aspr.hhs.gov/SNS/Pages/Mpox.aspx

Mpox vaccine adapted for children: Schnirring L. Bavarian Nordic, CEPI announce plan to advance mpox vaccine for African children. CIDRAP. May 30, 2024. Accessed July 22, 2024. https://www.cidrap.umn.edu/mpox/bavarian-nordic-cepi-announce-plan-advance-mpox-vaccine-african-children

Nipah vaccine: Pickover E. Scientists test world-first Nipah virus vaccine. The Independent. January 11, 2024. Accessed July 22, 2024. https://www.independent.co.uk/news/health/sarah-gilbert-nipah-scientists-nipah-virus-covid-b2476686.html

Lassa vaccine: Participants in Nigeria vaccinated in first-ever Phase 2 Lassa fever vaccine clinical trial, sponsored by IAVI. Press release. IAVI; April 24, 2024. https://www.iavi.org/press-release/iavi-c105-lassa-fever-vaccine-clinical-trial/

mRNA-based Zika vaccine: Bayer M. Moderna won't advance US-backed Zika vaccine without more outside funding. Fierce Biotech. May 25, 2024. Accessed July 22, 2024. https://www.fiercebiotech.com/biotech/moderna-says-it-wont-advance-us-backed-zika-vaccine-without-more-outside-funding

Universal flu vaccine: Universal Influenza Vaccine Technology Landscape. University of Minnesota; 2024. Accessed July 22, 2024. https://ivr.cidrap.umn.edu/universal-influenza-vaccine-technology-landscape

Universal flu vaccine, mRNA-based candidate: Clinical trial of mRNA universal influenza vaccine candidate begins. Press Release. National Institutes of Health; May 15, 2023. https://www.nih.gov/news-events/news-releases/clinical-trial-mrna-universal-influenza-vaccine-candidate-begins

Diagnostic tools for EIDs, Zika: US Food and Drug Administration. Emergency Use Authorization. Accessed July 22, 2024. https://www.fda.gov/emergency-preparedness-and-response/mcm-legal-regulatory-and-policy-framework/emergency-use-authorization#zika

Diagnostic tools for EIDs, mpox: US Food and Drug Administration. Monkeypox (mpox) Emergency Use Authorizations for Medical Devices. Accessed July 22, 2024. https://www.fda.gov/medical-devices/emergency-use-authorizations-medical-devices/monkeypox-mpox-emergency-use-authorizations-medical-devices

Diagnostic tools for EIDs, MERS: US Food and Drug Administration. Emergency Use Authorization. Accessed July 22, 2024. https://www.fda.gov/emergency-preparedness-and-response/mcm-legal-regulatory-and-policy-framework/emergency-use-authorization#MERS

US government investment in HIV R&D for emerging infectious diseases

G-FINDER data portal. Policy Cures Research; 2023. Accessed May 14, 2024. https://gfinderdata.policycuresresearch.org/

US Government R&D efforts

The White House. U.S. Government Global Health Security Strategy 2024. The White House; 2024. https://www.whitehouse.gov/wp-content/uploads/2024/04/Global-Health-Security-Strategy-2024-1.pdf

US Agency for International Development. Global Health Security Areas of Investment. Accessed July 22, 2024. https://www.usaid.gov/global-health/health-areas/global-health-security/global-health-security-areas-investment

Maternal, Newborn & Child Health

R&D for Maternal, Newborn & Child Health

How new tools can transform the fight

The chances of a mother and her child surviving and thriving are vastly different depending on where in the world they live. For example, around 83% of all global maternal and child deaths occur in sub-Saharan Africa and southern Asia, yet these regions only account for 57% of live births. Having the right intervention or tool at the right time is one of the most crucial factors for survival. Thanks to global efforts to develop and scale up health solutions, child deaths have declined worldwide by 62% and maternal deaths by nearly 46% since 1990. But vast challenges remain. Intensified research and development (R&D) efforts are needed to deliver pediatric versions of existing and novel medicines and to ensure products are suitable for use in pregnant and lactating women and individuals, as well as to create low-cost, easy-to-use technologies to address common causes of maternal and child deaths in the world’s poorest places.

4.9 million

children under five die each year
287,000 women

die each year in pregnancy and childbirth
218 million

women have an unmet need for contraception

Research successes

New technologies have driven significant improvements in maternal, newborn, and child health (MNCH):

Vaccines against childhood diseases, including polio, measles, diphtheria, tetanus, pneumonia, and other illnesses, have saved more than 150 million children over the past 50 years.
Contraceptive innovations, from decades-old tools like oral pills and implants to newer technologies like self-insertable, three-month and one-year vaginal rings, as well as discreet, self-injectable, and long-acting contraception options, have helped millions of women meet their unique family planning (FP) needs.
Tools to prevent and treat malaria in mothers and children—including insecticide-treated bednets, chemoprevention for children and pregnant women, child-friendly medicines, and vaccines—have significantly reduced maternal and child deaths.
Interventions to treat and prevent diarrhea—including oral rehydration solutions, zinc supplements, and low-cost vaccines against rotavirus, cholera, and typhoid—have helped reduce child mortality from diarrheal disease by more than half since 2000.
Nutrition innovations, like biofortified crops and vitamin supplements, are helping more children thrive.
Approaches to prevent maternal-to-child transmission of HIV have contributed to a 62% decline in new infections in children since 2010.

Key missing tools

New tools that are designed specifically for low-resource settings are needed to advance MNCH, including:

Better tools to manage respiratory disorders and pneumonia in facilities without high-tech equipment, including low-cost, durable, and easy-to-use neonatal resuscitators and ventilators to help babies breathe and affordable, easy-to-operate tools to monitor respiratory rates and oxygen levels in children.
Vaccine innovations to improve immunization coverage for vulnerable, hard-to-reach populations, such as heat-stable versions of vaccines that can be stored without refrigeration or easy-to-use, pain-free oral formulations.
Child-friendly formulations of existing and new medicines that are appropriately dosed, dissolvable, and more palatable.
Better tools to prevent postpartum hemorrhage, the leading cause of maternal death, including new, easily administered, and heat-stable formulations of the drug oxytocin for use in settings without reliable electricity and low-cost, simple devices to control and monitor the severity of bleeding after childbirth.
Simple, low-cost tools to detect preeclampsia, a dangerous pregnancy complication, including handheld blood pressure monitoring devices and other improved point-of-care diagnostics.
New contraceptive options with minimal side effects that are suitable for people living in remote areas with limited access to health care services, including multipurpose products for women that simultaneously prevent HIV and sexually transmitted infections while providing contraception and novel contraceptive options for men.
Better treatments and prevention options for pregnant and lactating women and individuals across a range of health areas.
New lost-cost and easy-to-administer tools to address other leading MNCH challenges, including obstructed labor, preterm birth, diarrheal disease, malaria, HIV/AIDS, and malnutrition.

Continued progress is possible, not inevitable

Breakthroughs on the brink

An oxytocin inhaler designed to manage postpartum hemorrhage after childbirth, even in settings with no electricity and refrigeration and limited access to trained health workers, is under development. The product, which received support from USAID, could save the lives of almost 20,000 new mothers each year.
A first-of-its-kind malaria treatment designed specifically for babies less than ten pounds has completed Phase 2/3 trials. There is no approved treatment for babies in this weight group, who are currently treated with a partial dose of medicines made for larger children, which can heighten risk of overdose among this very vulnerable population.
The OdonAssist™, a promising new tool previously supported by USAID, that uses air pressure and suction to gently assist with delivery during obstructed labor, is advancing through clinical trials.
Vaccines against shigella and enterotoxigenic Escherichia coli, two leading causes of diarrheal disease, are in clinical development with DoD support. To date, no effective vaccines are available against these pathogens.
New contraceptive innovations, including a microneedle contraceptive patch lasting six months supported by USAID, a male contraceptive gel now in clinical trials supported by NIH, and a vaginal ring and dual-use prevention pill that combine contraception with antiretroviral treatments to prevent HIV infection supported by USAID and NIH.
Zoliflodacin, a new antibiotic that completed Phase 3 trials, which, if approved, would become the first new antibiotic for treating gonorrhea in decades. In addition to causing health issues in women, gonorrhea can be passed from a pregnant person to their baby, leading to serious problems, including blindness and a life-threatening blood infection.
Affordable, rapid, and point-of-care urine tests to diagnose preeclampsia, including a simple paper urine test supported by USAID, are now undergoing validation testing. These tools, designed for low-resource settings, will improve the detection of this deadly condition that impacts 1 in 20 women during pregnancy.

US Government R&D efforts

The US government is advancing MNCH research through a whole-of-government approach:

US Agency for International Development (USAID) develops and distributes affordable vaccines, treatments, and other tools to improve MNCH globally and address FP and reproductive health (RH) needs, leading implementation of the US government’s MNCH and FP/RH activities.
National Institutes of Health (NIH) conducts basic science and implementation research to develop and improve tools to advance MNCH and FP/RH.
Centers for Disease Control and Prevention operates immunization programs, conducts surveillance and epidemiological research to inform the use of existing MNCH tools, and provides technical assistance to country partners to strengthen public health capacity.
Department of Defense (DoD) supports research against diseases like malaria and diarrheal diseases that pose a risk to service members stationed abroad and that are also leading killers of children.

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Introduction

Percentage of maternal deaths in sub-Saharan Africa and southern Asia: World Health Organization. Maternal mortality. Updated April 26, 2024. Accessed July 23, 2024. https://www.who.int/news-room/fact-sheets/detail/maternal-mortality

Percentage of under five deaths in sub-Saharan Africa and southern Asia, 62% reduction in child deaths, 57% of live births: United Nations Inter-agency Group for Child Mortality Estimation. Levels & Trends in Child Mortality: Report 2023, Estimates developed by the United Nations Inter-agency Group for Child Mortality Estimation. UNICEF; 2024. https://data.unicef.org/resources/levels-and-trends-in-child-mortality-2024/

46% reduction in maternal deaths:

World Health Organization, UNICEF, United Nations Population Fund, World Bank Group, United Nations Population Division. Trends in Maternal Mortality: 1990 to 2015. Work Health Organization; 2015. https://iris.who.int/bitstream/handle/10665/193994/WHO_RHR_15.23_eng.pdf

UNICEF. Maternal mortality. Accessed July 26, 2024. https://data.unicef.org/topic/maternal-health/maternal-mortality/

4.9 million children die: United Nations Inter-agency Group for Child Mortality Estimation. Levels & Trends in Child Mortality: Report 2023, Estimates developed by the United Nations Inter-agency Group for Child Mortality Estimation. UNICEF; 2024. https://data.unicef.org/resources/levels-and-trends-in-child-mortality-2024/

287,000 women die: World Health Organization. Maternal Health. Accessed July 23, 2024. https://www.who.int/health-topics/maternal-health#tab=tab_1

218 million women with unmet need for contraception: Guttmacher Institute releases family planning investment impact calculator, a new online tool for estimating health benefits of investing in family planning in low- and middle-income countries. News release. Guttmacher Institute; September 26, 2022. Accessed July 23, 2024.https://www.guttmacher.org/news-release/2022/guttmacher-institute-releases-family-planning-investment-impact-calculator-new

Research successes

Vaccines against childhood diseases: Ritchie H. Vaccines have saved 150 million children over the last 50 years. Our World in Data. May 6, 2024. Accessed July 23, 2024. https://ourworldindata.org/vaccines-children-saved

Contraceptive innovations, overall: World Health Organization. Family planning/contraception methods. Updated September 5, 2023. Accessed July 23, 2024. https://www.who.int/news-room/fact-sheets/detail/family-planning-contraception

Three-month vaginal ring: Undie CC, RamaRao S, Mbow FB. Choosing and using the progesterone vaginal ring: Women's lived experiences in three African cities. Patient Preference and Adherence. https://pubmed.ncbi.nlm.nih.gov/33061318/

One-year vaginal ring: Population Council. The Nestorone®/Ethinyl estradiol one-year vaginal contraceptive system. Accessed July 23, 2024. https://popcouncil.org/project/the-nestorone-ethinyl-estradiol-one-year-vaginal-contraceptive-system

Discreet, self-injectable, long-acting contraception: PATH. The power to prevent pregnancy in women’s hands: DMPA-SC injectable contraception. PATH. September 12, 2018. Accessed July 23, 2024. https://www.path.org/our-impact/articles/dmpa-sc/

Tools to prevent and treat malaria, insecticide-treated bednets: US Centers for Disease Control and Prevention. Insecticide-treated nets. Updated April 2, 2024. Accessed July 23, 2024. https://www.cdc.gov/malaria/php/public-health-strategy/insecticide-treated-nets.html

Tools to prevent and treat malaria, chemoprevention for children and pregnant women: Updated WHO recommendations for malaria chemoprevention among children and pregnant women. World Health Organization. June 3, 2022. Accessed July 23, 2024. https://www.who.int/news/item/03-06-2022-Updated-WHO-recommendations-for-malaria-chemoprevention-among-children-and-pregnant-women

Tools to prevent and treat malaria, child-friendly malaria medicines: Medicines for Malaria Venture. Artemether-lumefantrine dispersible. Accessed July 23, 2024. https://www.mmv.org/mmv-pipeline-antimalarial-drugs/artemether-lumefantrine-dispersible

Tools to prevent and treat malaria, vaccines:

Wadman M. First malaria vaccine slashes early childhood mortality. Science. October 24, 2023. Accessed July 23, 2024. https://www.science.org/content/article/first-malaria-vaccine-slashes-early-childhood-deaths

WHO prequalifies a second malaria vaccine, a significant milestone in prevention of the disease. News release. World Health Organization; December 21, 2023. https://www.who.int/news/item/21-12-2023-who-prequalifies-a-second-malaria-vaccine-a-significant-milestone-in-prevention-of-the-disease

Reduced child mortality from diarrheal disease: Child mortality, stillbirth, and causes of death estimates. United Nations Inter-agency Group for Child Mortality Estimation; 2023. Accessed July 23, 2024. https://childmortality.org/causes-of-death/data?causes=DIARRHOEAL&d_refArea=WORLD&type=DEATHS

62% decline in new maternal-to-child HIV infections: Global HIV & AIDS statistics — Fact sheet. UNAIDS; 2024. Accessed August 13, 2024. https://www.unaids.org/en/resources/fact-sheet

Continued progress is possible, not inevitable

Bill & Melinda Gates Foundation. Goalkeepers 2024 Report. The Race to Nourish a Warming World. Bill & Melinda Gates Foundation; 2024. https://www.gatesfoundation.org/goalkeepers/report/2024-report/

Key missing tools

Based on consultation with experts from GHTC member organizations.

Breakthroughs on the brink

Oxytocin inhaler: Monash University. The project. Accessed July 23, 2024. https://www.monash.edu/iop/home/the-project

Malaria treatment for babies under 10 pounds: Medicines for Malaria Venture and Novartis announce positive efficacy and safety data for a novel treatment for babies <5 kg with malaria. Press release. Medicines for Malaria Venture; April 24, 2024. Accessed July 23, 2024. https://www.mmv.org/newsroom/news-resources-search/medicines-malaria-venture-and-novartis-announce-positive-efficacy

OdonAssist: Maternal Newborn Health Innovations. OdonAssist™. Accessed July 23, 2024. https://www.mnhi.com/odonassist-tm

Vaccines for diarrheal diseases: When it comes to vaccines, bigger isn’t always better. PATH. November 9, 2021. Accessed July 23, 2024. https://www.path.org/our-impact/articles/when-it-comes-to-vaccines-bigger-isnt-always-better/

New contraceptive innovations, microneedle patch: Georgia Institute of Technology. Long-acting contraceptive designed to be self-administered via microneedle patch. Medical Xpress. January 14, 2019. Accessed July 23, 2024. https://medicalxpress.com/news/2019-01-long-acting-contraceptive-self-administered-microneedle-patch.html

New contraceptive innovations, male contraceptive gel: Population Council. Nestorone®/Testosterone transdermal gel for male contraception. Accessed July 23, 2024. https://popcouncil.org/project/nestorone-testosterone-transdermal-gel-for-male-contraception/

New contraceptive innovations, vaginal ring: Population Council. The dapivirine-levonorgestrel vaginal ring for HIV prevention and contraception. Accessed July 23, 2024. https://popcouncil.org/project/the-dapivirine-levonorgestrel-vaginal-ring-for-hiv-prevention-and-contraception/

New contraceptive innovations, dual use prevention pills

Population Council. Dual prevention pill for the prevention of HIV and unintended pregnancy. Accessed July 23, 2024. https://popcouncil.org/project/dual-prevention-pill-for-the-prevention-of-hiv-and-unintended-pregnancy/

PrEPWatch. Dual prevention pill. Updated July 19, 2024. Accessed July 23, 2024. https://www.prepwatch.org/products/dual-prevention-pill/

Affordable, rapid point-of-care urine tests to diagnose preeclampsia: Research finds simple urine test allows for rapid diagnosis of preeclampsia. Press release. The Ohio State University Wexner Medical Center; March 13, 2019. Accessed July 23, 2024. https://wexnermedical.osu.edu/mediaroom/pressreleaselisting/preeclampsia-test.

US government R&D efforts

The US government and international family planning & reproductive health efforts. KFF. January 2, 2024. Accessed July 23, 2024. https://www.kff.org/global-health-policy/fact-sheet/the-u-s-government-and-international-family-planning-reproductive-health-efforts/.

The US government and global maternal & child health efforts. KFF. September 29, 2022. Accessed July 23, 2024. https://www.kff.org/global-health-policy/fact-sheet/the-u-s-government-and-global-maternal-and-child-health/.

The US Department of Defense & global health. KFF. September 29, 2012. Accessed July 23, 2024. https://www.kff.org/global-health-policy/report/the-u-s-department-of-defense-global/.

Antimicrobial Resistance

R&D for Antimicrobial Resistance

How new tools can transform the fight

Antimicrobial resistance (AMR) occurs when microbes evolve to no longer respond to treatment. The World Health Organization considers it one of the top global health threats, given it puts the gains of modern medicine at risk and threatens progress against some of the world’s deadliest diseases. In recent years, governments have increased action to reinvigorate the antimicrobial research pipeline and tackle major drivers of AMR, such as antimicrobial misuse and overuse, but much work remains to be done. The current clinical pipeline for products to address priority pathogens is insufficient, and growing resistance to antibiotics, antivirals, antifungals, and antiparasitic drugs is steadily reducing our arsenal of treatments for common threats. To confront this crisis, we need new antimicrobials and antimicrobial alternatives, as well as new diagnostics and vaccines to improve surveillance and prevention.

5 million

deaths associated with AMR every year
$100 trillion

projected cost of AMR to global economy from 2014 to 2050
1 in 5

cancer patients in treatment are hospitalized due to an infection

Research successes

New technologies are aiding the fight against AMR:

Cifiderocol and sulbactam-durlobactam were approved by the FDA in 2020 and 2023, respectively, as new antibiotics for hospital-acquired and ventilator-associated bacterial pneumonia, providing additional treatment options for bacterial strains prone to resistance.
Several new antibiotics, including pivmecillinan, vaborbactam, plazomicin, and cefiderocol, have been approved by the FDA in recent years to treat urinary tract infections, providing new options for those with infections unresponsive to existing drugs.
Pretomanid, a new drug for highly drug-resistant tuberculosis (TB) developed with USAID and NIH support, was approved by the FDA in 2019 as part of a combination regimen that has dramatically improved treatment outcomes and reduced treatment times.
Pneumococcal vaccines for children, first introduced in the 2000s, have saved millions of lives while also reducing the use of antibiotics. Universal coverage of these vaccines could prevent 11.4 million days of antibiotic use per year in children under five.
New tests that identify whether infections are resistant to certain treatments have been introduced, including a BARDA-supported panel test that detects 13 resistance genes and rapid, automated molecular tests for drug-resistant TB.

Deaths from drug-resistant infections set to skyrocket

Key missing tools

To control AMR, we need innovative tools to prevent, diagnose, and treat drug-resistant infections, including:

Novel antimicrobials to treat priority pathogens.
Expanded antimicrobial options designed for children and infants.
Oral formulations of existing and investigational antimicrobials, which are easier to administer in outpatient settings and can improve treatment adherence to prevent further resistance.
Rapid, point-of-care diagnostic tests that identify infections and determine if they are resistant to ensure appropriate treatment and prevent the misuse or overuse of existing antimicrobials.
Vaccines and other prevention options to avert infections in the first place.
Alternatives to antimicrobials, such as bacteria-targeting viruses called bacteriophages, anti-virulence therapies, and innovative approaches to manipulate the gut microbiome.

Breakthroughs on the brink

Zoliflodacin, a new first-in-class antibiotic that completed Phase 3 trials, which, if approved, would become the first new antibiotic for treating gonorrhea in decades.
VE303, a new oral treatment for Clostridioides difficile, a potentially life-threatening bacterial infection that causes diarrhea and colon inflammation, which is undergoing Phase 3 trials with support from BARDA.
Cefepime-taniborbactam, a new antibiotic developed for treating complicated urinary tract infections in adults, which completed Phase 3 trials. A pediatric formulation is also being advanced for children and newborns.
A protective vaccine against typhoid fever and invasive nontyphoidal Salmonellosis, a disease caused by salmonella subspecies found almost exclusively in sub-Saharan Africa—now in first-in-human studies—could help address the estimated 70,000 deaths from the disease and 20,000 deaths from Salmonella enetrica serovar Typhi annually in the region.
A maternal vaccine to prevent sepsis in newborns targeting Escherichia coli, the bacteria responsible for a large portion of sepsis infections in newborns. It is being advanced with BARDA and NIH support and would be administered to expectant mothers who pass antibodies on to their babies in utero and through breast milk.
Dosing validation of two existing antibiotics (fosfomycin and flomoxef) in newborns to expand treatment options for neonatal sepsis. Forty percent of infections causing neonatal sepsis in hospitals are resistant to standard treatments.
A portable, rapid diagnostic test to diagnose gonorrhea infection and detect resistance to ciprofloxacin, a former frontline oral antibiotic that can no longer treat drug-resistant infections. The test, being advanced with BARDA and NIH support, will enable doctors to treat patients with ciprofloxacin when appropriate while reserving ceftriaxone, the only other antibiotic effective against drug-resistant gonorrhea.
A first-of-its kind infectious disease diagnostic platform, advanced with BARDA and NIH support, that rapidly identifies the pathogen causing an infection and profiles its antibiotic susceptibility in hours, rather than the days typically required for culture-based tests, the current standard.
Phage therapy, a promising alternative to antibiotics, which uses specialized viruses called bacteriophages to target, infect, and kill bacteria. With NIH support, researchers are advancing potential phage therapies to treat TB, cholera, staph infections, and more.

US Government R&D efforts

The US government is leading efforts to advance research and development (R&D) to combat AMR through a whole-of-government approach:

National Institutes of Health (NIH) conducts basic science and clinical research to advance new antimicrobials and other technologies to combat AMR and provides in-kind assistance to the global Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) nonprofit partnership.
Biomedical Advanced Research and Development Authority (BARDA) supports early-stage and advanced development of antimicrobials and other products through direct R&D investments and financial support of CARB-X.
Centers for Disease Control and Prevention (CDC) develops diagnostic tools, operates an AMR isolate bank to provide samples to product developers, and supports domestic and global surveillance and stewardship activities, including operating a global laboratory and response network.
Department of Defense supports global surveillance activities through its overseas laboratories and supports the development of select antimicrobials to combat sepsis and the threat of drug resistance in combat wounds.
US Department of Agriculture conducts veterinary surveillance and stewardship activities and advances R&D for antimicrobials and vaccines for animal populations.
US Agency for International Development (USAID) advances product development and implementation research for select global threats impacted by rising drug resistance, including malaria, TB, and HIV/AIDS.
Food and Drug Administration (FDA), alongside approving products for use in the United States, also cooperates the AMR isolate bank with CDC and provides guidance to product developers.

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Introduction

Antimicrobial resistance: World Health Organization. Antimicrobial resistance. Updated November 21, 2023. Accessed July 23, 2024. https://www.who.int/news-room/fact-sheets/detail/antimicrobial-resistance

5 million deaths: Institute for Health Metrics and Evaluation. Antimicrobial resistance (AMR). Accessed July 23, 2024. https://www.healthdata.org/research-analysis/health-risks-issues/antimicrobial-resistance-amr

$100 trillion lost: King A, Chemistry World. Antibiotic resistance will kill 300 million people by 2050. Scientific American. December 16, 2014. Accessed July 23, 2024. https://www.scientificamerican.com/article/antibiotic-resistance-will-kill-300-million-people-by-2050/

1 in 5 cancer patients: Union for International Cancer Control. AMR and cancer series. Accessed July 23, 2024. https://www.uicc.org/what-we-do/sharing-knowledge/virtual-dialogues/amr-and-cancer-series

Research successes

Cefiderocol: Shionogi announces FDA approval of FETROJA® (Cefiderocol) for the treatment of hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia. Press release. Shionogi; September 28, 2024. https://www.shionogi.com/us/en/news/2020/9/shionogi-announces-fda-approval-of-fetroja-cefiderocol-for-the-treatment-of-hospital-acquired-bacterial-pneumonia-and-ventilator-associated-bacterial-pneumonia.html

Sulbactam-durlobactam: FDA approves new treatment for pneumonia caused by certain difficult-to-treat bacteria. Press release. US Food and Drug Administration; May 23, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-pneumonia-caused-certain-difficult-treat-bacteria

Several new antibiotics for urinary track infections:

Jacobs A. FDA approves antibiotic for increasingly hard-to-treat urinary tract infections. The New York Times. April 24, 2024. Accessed July 23, 2024. https://www.nytimes.com/2024/04/24/health/fda-urinary-tract-infection-antibiotic.html

World Health Organization. 2023 Antibacterial agents in clinical and preclinical development: An overview and analysis. World Health Organization; 2024. https://www.who.int/publications/i/item/9789240094000

Pretomanid: FDA approves new treatment for highly drug-resistant forms of tuberculosis. Press release. TB Alliance; August 14, 2019. https://www.tballiance.org/news/fda-approves-new-treatment-highly-drug-resistant-forms-tuberculosis

Pneumococcal vaccines: O’Neill J. Vaccines vs superbugs: A new weapon to fight antibiotic resistance? World Economic Forum. February 12, 2016. Accessed July 23, 2024. https://www.weforum.org/agenda/2016/02/vaccines-vs-superbugs-a-new-weapon-to-fight-antibiotic-resistance/

New tests:

T2 Biosystems’ T2Resistance™ Panel is first diagnostic to graduate from CARB-X portfolio. Press release. Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator; September 24, 2019. https://carb-x.org/carb-x-news/t2-biosystems-t2resistance-panel-is-first-diagnostic-to-graduate-from-carb-x-portfolio/

Nandlal L, Perumal R, Naidoo K. Rapid molecular assays for the diagnosis of drug-resistant tuberculosis. Infection and Drug Resistance. https://pubmed.ncbi.nlm.nih.gov/36060232/

Deaths from drug-resistant infections set to skyrocket

Fleck A. Deaths from drug-resistant infections set to skyrocket. Statista. May 26, 2023. Accessed July 23, 2024. https://www.statista.com/chart/3095/drug-resistant-infections/

Key missing tools

World Health Organization. Global research agenda for antimicrobial resistance in human health. World Health Organization; 2023. https://www.who.int/publications/m/item/global-research-agenda-for-antimicrobial-resistance-in-human-health

World Health Organization. Antimicrobial resistance. Updated November 21, 2023. Accessed July 23, 2024. https://www.who.int/news-room/fact-sheets/detail/antimicrobial-resistance

Breakthroughs on the brink

Zoliflodacin: Positive results announced in largest pivotal Phase 3 trial of a first-in-class oral antibiotic to treat uncomplicated gonorrhoea. Press release. Global Antibiotic Research & Development Partnership; November 1, 2023. https://gardp.org/positive-results-announced-in-largest-pivotal-phase-3-trial-of-a-first-in-class-oral-antibiotic-to-treat-uncomplicated-gonorrhoea/

VE303: BARDA and Vedanta Biosciences partner for Phase 3 clinical trial of VE303 as a potential treatment for C. difficile bacterial infections. Web announcement. Medical Countermeasures.gov; October 5, 2021. https://medicalcountermeasures.gov/newsroom/2021/vedanta/

Cefepime-taniborbactam: Global Antibiotic Research & Development Partnership. Serious bacterial infections & sepsis. Accessed July 23, 2024. https://gardp.org/serious-bacterial-infections/

Vaccine to protect against typhoid fever and invasive nontyphoidal salmonellosis: Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator. Portfolio product developers. Accessed July 23, 2024. https://carb-x.org/portfolio/portfolio-companies/

Maternal vaccine for sepsis: CARB-X funds GlyProVac to develop a novel vaccine to prevent sepsis in newborns. Press release. Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator; February 29, 2024. https://carb-x.org/carb-x-news/carb-x-funds-glyprovac/

Validation of dosing of two existing antibiotics in newborns: Global Antibiotic Research & Development Partnership. Neonatal sepsis. Accessed July 23, 2024. https://gardp.org/neonatal-sepsis/

Portable, rapid diagnostic test for gonorrhea: CARB-X funds Visby Medical to develop a portable rapid diagnostic for gonorrhea including antibiotic susceptibility. Press release. Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator; February 8, 2024. https://carb-x.org/carb-x-news/carb-x-funds-visby-medical/

First-of-its kind infectious disease diagnostic platform: Pattern Bioscience secures $28.7 million in Series C financing. Press release. Pattern Bioscience; April 27, 2023. https://pattern.bio/pattern-bioscience-secures-28-7-million-in-series-c-financing/

Phage therapy: NIH awards grants to support bacteriophage therapy research. News release. US National Institutes of Health; March 11, 2021. https://www.nih.gov/news-events/news-releases/nih-awards-grants-support-bacteriophage-therapy-research

US government R&D efforts

Federal Task Force on Combating Antibiotic-Resistant Bacteria. Natifonal Action Plan for Combating Antibiotic-Resistant Bacteria: 2020-2025. U.S. Department of Health & Human Services; 2020. https://aspe.hhs.gov/reports/national-action-plan-combating-antibiotic-resistant-bacteria-2020-2025

Fact sheet series: R&D across health areas

R&D for HIV/AIDS

Research successes

Continued progress is possible, not inevitable

Key missing tools

Breakthroughs on the brink

US Government R&D efforts

R&D for Malaria

Research successes

Continued progress is possible, not inevitable

Key missing tools

Breakthroughs on the brink

US Government R&D efforts

R&D for Tuberculosis

Research successes

Continued progress is possible, not inevitable

Key missing tools

Breakthroughs on the brink

US Government R&D efforts

R&D for Neglected Tropical Diseases

Research successes

Continued progress is possible, not inevitable

Key missing tools

Breakthroughs on the brink

US Government R&D efforts

R&D for Emerging Infectious Diseases

Key missing tools

Research progress

US Government R&D efforts

R&D for Maternal, Newborn & Child Health

Research successes

Key missing tools

Continued progress is possible, not inevitable

Breakthroughs on the brink

US Government R&D efforts

R&D for Antimicrobial Resistance

Research successes

Deaths from drug-resistant infections set to skyrocket

Key missing tools

Breakthroughs on the brink

US Government R&D efforts

Other resource items you may like:

WHO prequalification process scorecard

US agency series: Global health R&D across the US government

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