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 global HIV/AIDS epidemic has claimed the lives of more than 32 million people around the world—reversing gains in life expectancy and economic development in the world’s poorest places. The development of antiretroviral drugs (ARVs) has turned the trajectory of the epidemic, extending life expectancy of those living with HIV/AIDS and enabling the prevention of transmission between mothers and children and HIV-positive and negative partners.

Yet progress against the epidemic has slowed in recent years, and major gaps remain in our arsenal of treatment and prevention tools. We will not reach targets to end the HIV/AIDS epidemic without new and improved technologies.

37.9 million

people are living with HIV/AIDS globally
1.7 million

people became newly infected with HIV in 2018
300+ 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 21.7 million people globally and have cumulatively averted more than 9.5 million AIDS-related deaths since 1995, with global economic benefits of US$1.05 trillion.
Pre-exposure prophylaxis, or PrEP—the use of ARVs to prevent infection—was shown to be effective through NIH, CDC, and USAID research and is today being introduced worldwide.
Approaches to prevent mother-to-child HIV transmission—developed with NIH support—that involve treating women during pregnancy and their newborns with ARVs have contributed to a 41% decline in new infections in children since 2010.
New diagnostic innovations, from rapid tests, to urine and oral tests, to 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:

HIV/AIDS vaccines to prevent and reduce new infections.
A cure to control or eliminate infection from the body.
Microbicides to prevent infections in women and men who have sex with men—populations disproportionately impacted by the epidemic.
Improved therapies for young children that are safe, palatable, shorter course, and easier to administer.
New treatment and PrEP regimens to help expand choice and improve adherence, such as long-acting injectable ARVs, fixed-dose combinations, and simplified treatments with fewer side effects.
Additional treatment options for those whose infection has become resistant to existing treatments.

Breakthroughs on the brink

A first-of-its-kind, monthly ARV-based microbicide vaginal ring, supported by NIH and USAID, is now under regulatory review and could expand prevention options for at-risk women.
A promising “mosaic” vaccine designed to address several HIV strains at once is one of two HIV vaccine candidates now in late-stage clinical trials in sub-Saharan Africa. This and other scientific breakthroughs bring new hope to the quest for an HIV/AIDS vaccine, with around 40 vaccine candidates now in late-stage development.
A 4-in-1 child-friendly ARV regimen for infants and young children that is palatable, easy-to-administer, and requires no refrigeration is now under regulatory review.
Many clinical trials are underway to test broadly neutralizing antibodies (bNabs)—proteins shown to neutralize many different genetic variants of HIV—as treatment and preventions products, including late-stage clinical trials of a bNAb developed at NIH labs.
Long-acting injectable formulations of PrEP, which could be administered monthly or every two months, are now in late-stage clinical trials and could soon offer a new option for patients.
Multipurpose prevention products, including pills, injections, and a vaginal ring that combines ARVs with contraception, could expand HIV prevention options for women.
A second patient appears to be effectively cured of HIV as of 2019, demonstrating that a cure for HIV infection, while difficult, is scientifically possible.

US government investment in HIV/AIDS R&D for low-resource settings (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 HIV/AIDS epidemic through a whole-of-government approach:

National Institutes of Health conducts basic science and clinical research for new therapies, vaccines, microbicides, and a cure for HIV/AIDS, as well as research to improve use of existing interventions.
US Agency for International Development advances R&D for HIV/AIDS technologies designed for low-resource settings, including research for a vaccine and microbicides, and helps accelerate introduction 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.
Centers for Disease Control and Prevention develops improved diagnostics and conducts research to inform 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 from HIV/AIDS, 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

HIV/AIDS epidemic claimed the lives of more than 32 mllion people; 37.9 million living with HIV/AIDS, 1.7 people became newly infected: UNAIDS website. Global HIV & AIDS statistics — 2019 fact sheet page. https://www.unaids.org/en/resources/fact-sheet. Accessed August 26, 2019.

300+ children die every day form AIDS-related causes: UNICEF. UNICEF Data. New York City, NY: UNICEF; 2019. https://data.unicef.org/topic/hivaids/global-regional-trends/. Accessed August 26, 2019.

Research successes

ARVs, developed with NIH support: National Institute of Allergy and Infectious Diseases website. Antiretroviral drug discovery and development page. https://www.niaid.nih.gov/diseases-conditions/antiretroviral-drugdevelopment. Accessed August 26, 2019.

ARVs used to treat 21.7 million people: UNAIDS website. Global HIV & AIDS statistics — 2019 fact sheet page. https://www.unaids.org/en/resources/fact-sheet. Accessed August 26, 2019.

ARVs, averted more than 9.5 million AIDS deaths since 1995; global economic benefits of US$1.05 trillion: Forsythe S, McGreevey W, et al. Twenty years of antiretroviral therapy for people living with HIV: global costs, health achievements, economic benefits. Health Affairs. 2019;38(7). https://doi.org/10.1377/hlthaff.2018.05391

Pre-exposure prophylaxis, shown to be effective through NIH research: National Institute of Allergy and Infectious Diseases website. Pre-exposure prophylaxis (PrEP) to reduce HIV risk page. https://www.niaid.nih.gov/diseases-conditions/pre-exposure-prophylaxis-prep. Accessed August 26, 2019.

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 Mar; 3(3): 10.4155/cli.12.155. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3882078/#!po=3.12500.

Centers for Disease Control and Prevention. TDF2 Study of Pre-exposure Prophylaxis (PrEP) Among Heterosexual Men and Women in Botswana: Key Facts. Atlanta: Centers for Disease Control and Prevention; 2011: https://www.cdc.gov/nchhstp/newsroom/docs/PrEP-Heterosexuals-Factsheet.pdf.

Garcia-Lerma JG, Paxton L, Kilmarx PH, et al. Oral pre-exposure prophylaxis for HIV prevention. Trends in Pharmacological Science. 2010; 31(2):74-81. https://doi.org/10.1016/j.tips.2009.10.009.

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

Celum C. Open Label Extension studies: Findings from Partners PrEP Study. Presented at 2014 Microbicides Trial Network Regional Meeting. October 25, 2014. https://mtnstopshiv.org/sites/default/files/attachments/CELUM2014%20MTN%20Regional%20mtg_Partners%20PrEP%20OLE.pdf.

Clinicaltrials.gov. NCT00625404: FEM-PrEP (Truvada®): Study to Assess the Role of Truvada® in Preventing HIV Acquisition in Women. https://www.clinicaltrials.gov/ct2/show/NCT00625404. Accessed January 30, 2020.

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]. Bethesda, Maryland: National Institutes of Health; November 17, 2014. https://www.nih.gov/news-events/newsreleases/nih-sponsored-study-identifies-superior-drug-regimen-preventing-mother-child-hiv-transmission.

Approaches to prevent mother-to-child HIV transmission, 41% decline in new infections in children since 2010: UNAIDS website. Global HIV & AIDS statistics — 2019 fact sheet page. https://www.unaids.org/en/resources/factsheet. Accessed August 26, 2019.

Diagnostic innovations:

Unitaid, World Health Organization. Market and Technology Landscape: HIV Rapid Diagnostic Tests For SelfTesting, 4th Edition. Geneva, Switzerland: Unitaid; 2018. https://unitaid.org/assets/HIVST-landscape-report.pdf.

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

Recency testing: PEPFAR Solution Platform (Beta) website. Surveillance of recent HIV infections: using point-ofcare recency tests to rapidly detect and respond to recent infections page. https://www.pepfarsolutions.org/emerging-technologies-innovations/2018/7/11/surveillance-of-recent-hivinfections-using-point-of-care-recency-tests-to-rapidly-detect-and-respond-to-recent-infections. Accessed October 17, 2019.

Continued progress is possible, not inevitable

Bill & Melinda Gates Foundation and Institute for Health Metrics and Evaluation. The Goalkeepers Report 2019: How Geography and Gender Stack the Deck For (or Against) You. Seattle: Bill & Melinda Gates Foundation; 2019. https://www.gatesfoundation.org/goalkeepers/report/2019-report/#ExaminingInequality.

Breakthroughs on the brink

Microbicide vaginal ring: IPM’s application for dapivirine vaginal ring for reducing HIV risk in women now under review by European Medicines Agency [press release]. Silver Spring, Maryland: International Partnership for Microbicides; July 13, 2017. https://www.ipmglobal.org/content/ipm%E2%80%99s-application-dapivirine-vaginalring-reducing-hiv-risk-women-now-under-review-european.

Mosaic vaccine: Leary, K. A vaccine for HIV is about to be tested in thousands of people. Futurism. November 30, 2017. https://futurism.com/neoscope/vaccine-hiv-tested-thousands-people.

Two HIV vaccine candidates in late-stage clinical trials: Engel M. New HIV vaccine study launched for World AIDS Day. Fred Hutch News Service. December 2, 2017. https://www.fredhutch.org/en/news/centernews/2017/12/new-hiv-vaccine-test-launched-world-aids-day.html.

40 vaccine candidates in late-stage development: Policy Cures Research. Neglcted Disease R&D Product Pipeline. Sydney, Australia: Policy Cures Research; 2015. https://www.pipeline.policycuresresearch.org/august2017. Accessed August 26, 2019.

Child-friendly ARV regimen: Drugs for Neglected Diseases initiative. Ending the Neglect of Paediatric HIV: Improving HIV Treatment for Children: An Update. Geneva, Switzerland: Drugs for Neglected Diseases initiative; 2018. https://www.dndi.org/wp-content/uploads/2018/08/DNDi_Paediatric-HIV_2018.pdf.

Several clinical trials are underway to test broadly neutralizing antibodies: AVAC. Broadly Neutralizing Antibody Combinations. Seattle; AVAC; 2019: https://www.avac.org/infographic/bnab-combinations

BNAb developed at NIH lab: NIH Launches Large Clinical Trials of Antibody-Based HIV Prevention [press release]. Bethesda, Maryland: National Institute of Allergy and Infectious Diseases; April 7, 2016. https://www.niaid.nih.gov/news-events/nih-launches-large-clinical-trials-antibody-based-hiv-prevention

Long-acting injectable formulations of PrEP: NIH trial evaluates long-acting HIV medication unable to adhere to strict daily regimens [press release]. Bethesda, Maryland: National Institutes of Health; May 9, 2019. https://www.nih.gov/news-events/news-releases/nih-trial-evaluates-long-acting-hiv-medication-unable-adherestrict-daily-regimens.

Multipurpose prevention products: AVAC. Multipurpose Prevention Technologies (MPTs): An Introductory Factsheet. New York City, NY: AVAC; 2016. https://www.avac.org/resource/multipurpose-prevention-technologiesmpts-introductory-factsheet.

Multipurpose prevention products, vaginal ring: International Partnership for Microbicides website. Our products page. https://www.ipmglobal.org/our-work/product-pipeline. Accessed August 26, 2019.

Second patient appears to be effectively cured of HIV: Mandavilli A. H.I.V. is reported cured in a second patient, a milestone in the global AIDS epidemic. The New York Times. March 4, 2019. https://www.nytimes.com/2019/03/04/health/aids-cure-london-patient.html.

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

Policy Cures Research. G-FINDER Public Search Tool. Sydney, Australia: Policy Cures Research; 2019. https://gfinder.policycuresresearch.org/PublicSearchTool/. [Early access to 2018 data was provided by Policy Cures Research in December 2019. Data is now available online as of January 30, 2019.]

US government R&D efforts

Kaiser Family Foundation. U.S. Federal Funding for HIV/AIDS: Trends Over Time. Washington, DC: Kaiser Family Foundation; 2019. https://www.kff.org/hivaids/fact-sheet/u-s-federal-funding-for-hivaids-trends-over-time/.

Kaiser Family Foundation. The U.S. Department of Defense & Global Health. Washington, DC: Kaiser Family Foundation; 2012. https://www.kff.org/global-health-policy/report/the-u-s-department-of-defense-global/.

U.S. Food & Drug Administration website. President's Emergency Plan for AIDS Relief (PEPFAR) page. https://www.fda.gov/international-programs/presidents-emergency-plan-aids-relief-pepfar. Accessed August 26, 2019.

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 2017, there were an estimated 219 million cases of malaria in 87 countries that claimed the lives of approximately 435,000 people—61% of whom were young children. Despite gains that have been made over the past two decades in expanding access to malaria prevention tools and treatments, challenges such as drug and insecticide resistance continue to hamper control efforts. Unfortunately, 2017 marked the second consecutive year that malaria cases have increased—indicating progress against the disease has stalled and underscoring the urgent need for new solutions. To eradicate malaria, we need new treatments, vaccines, diagnostics, and vector control technologies.

half

the world’s population is at risk
219 million

cases of malaria in 2017
435,000

deaths from malaria worldwide in 2017

Research successes

Technologies have transformed the fight against malaria:

Vector control tools, including insecticide-treated bednets and indoor residual spraying, have driven dramatic declines in malaria. These two tools are responsible for an estimated 78% of malaria cases averted between 2000 and 2015.
Artemisinin-based combination therapies (ACTs), first developed in the 1990s, have become the gold standard treatment and have helped contribute to a 62% decline in malaria mortality between 2000 and 2015.
The world’s first malaria vaccine, RTS,S, which provides partial protection in young children, began pilot implementation in parts of three sub-Saharan African countries in 2019. Several US agencies supported its development.
Tafenoquine, a single-dose medicine to prevent the relapse of Plasmodium vivax malaria, which was developed with support from USAID and DoD, was approved in 2018—becoming the first new treatment for this indication in more than 60 years. A companion point-of-care diagnostic test to guide its use was approved in 2019.
A child-friendly malaria medicine, Coartem® Dispersible, developed with support from USAID, has saved an estimated 825,000 child lives since its launch in 2009.

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 insecticides for use in interventions like bednets and indoor residual spraying to address growing insecticide resistance, and biological control methods, like gene drives, to reduce mosquito populations and block parasite transmission.
New treatments for infections that have become resistant to currently available drugs including ACTs, as well as treatments to block transmission and to protect vulnerable populations like children and pregnant women.
A single-dose cure for Plasmodium falciparum infection, the most severe form of malaria, that will be easier and faster to administer and prevent the emergence of drug resistance.
Improved, more sensitive rapid diagnostic tests that are suitable for use in low-resource settings and that can detect 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 prevent infection or block human to mosquito transmission of the malaria parasite.

Breakthroughs on the brink

Several long-acting injectable malaria prevention drugs are under development, including one supported by NIH, which uses nanotechnology to intravenously deliver existing antimalarials that confer protection against the disease for weeks or months. This technology could simplify and improve malaria prevention by eliminating the need for daily pills.
An experimental single-dose cure for Plasmodium falciparum malaria, DSM265, developed with support from USAID, has shown promise in an early-stage field trial, curing patients with just one dose.
More than a dozen malaria vaccine candidates are in late-stage clinical development, including candidates supported by NIH, DoD, and USAID.
A promising vector control tool called Attractive Targeted Sugar Bait is undergoing trials in three countries. The brand-new product class, which uses plant sugars to attract mosquitoes combined with an ingestible toxin that kills them but is safe to humans, is being developed to address the growing threat of outdoor biting by mosquitoes.
A first-of-its-kind test that diagnoses malaria using a patient’s saliva, rather than blood, is now undergoing field trials. Developed with support from NIH, the test is less invasive than other methods, delivers results in 5 to 20 minutes at point of care, and can detect the disease before patients even show symptoms.

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 conducts basic and clinical research for new treatments, vaccines, diagnostics, and vector control products.
Department of Defense 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 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, which jointly implements PMI, conducts surveillance and monitoring research, as well as 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

About half of the world’s population is at risk of malaria: Kaiser Family Foundation. The President’s Malaria Initiative and Other U.S. Government Global Malaria Efforts. Washington, DC: Kaiser Family Foundation; 2019. https://www.kff.org/global-health-policy/fact-sheet/the-u-s-government-and-global-malaria/.

219 million cases of malaria in 87 countries that claimed the lives of approximately 435,000 people—61% of whom were young children; 2017 marked second consecutive year malaria cases have increased: World Health Organization. World Malaria Report 2018. Geneva: World Health Organization; 2018. https://www.who.int/malaria/publications/world-malaria-report-2018/en/

Research successes

Vector-control tools, two tools are responsible for an estimated 78% of malaria cases averted between 2000 and 2015: S Bhatt, Weiss J, et al. The effect of malaria control on Plasmodium falciparum in Africa between 2000 and 2015. Nature International Journal of Science. 2015; 15(526); 207–211. https://www.nature.com/articles/nature15535.

Artemisinin-based combination therapies (ACTs): From plant to medicine: how an ancient plant-based remedy became a modern malaria medicine [blog post]. Sandoz. August 25, 2017. https://www.sandoz.com/stories/access-healthcare/plant-medicine-how-ancient-plant-based-remedy-became-modern-malaria.

ACTs, 62% decline in malaria mortality between 2000 and 2015: World Health Organization. World Malaria Report 2016. Geneva: World Health Organization; 2016. https://www.who.int/malaria/publications/world-malaria-report-2016/en/.

The world’s first malaria vaccine, RTS,S: PATH. The RTS,S Malaria Vaccine. Seattle: PATH; 2019. https://www.malariavaccine.org/sites/www.malariavaccine.org/files/content/page/files/PATH_MVI_RTSS_Fact%20sheet_042019.pdf.

Tafenoquine, a single-dose medicine to prevent the relapse of Plasmodium vivax malaria:

Medicines for Malaria Venture website. First single-dose medicine to prevent the relapse of P. vivax approved by US FDA page. https://www.mmv.org/our-impact/achievements/first-single-dose-medicine-prevent-relapse-p-vivax-approved-us-fda. Accessed September 25, 2019.

US Agency for International Development website. Medicines for Malaria Venture (MMV) page. https://www.usaid.gov/global-health/health-areas/malaria/research-innovation/medicines-malaria-venture-mmv. Accessed September 25, 2019.

US FDA approves Krintafel (tafenoquine) for the radical cure of P. vivax malaria [press release]. London, UK: 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, a companion point-of-care diagnostic: Expert Review Panel for Diagnostics approval expands access to a G6PD deficiency test that supports P. vivax malaria treatment and elimination initiatives [press release]. Seattle, Washington: PATH; October 3, 2019. Available at https://www.path.org/articles/expert-review-panel-diagnostics-approval-expands-access-g6pd-deficiency-test-supports-p-vivax-malaria-treatment-and-elimination-initiatives/.

A child-friendly malaria medicine, Coartem® Dispersible:

Medicines for Malaria Venture website. Key achievements 2018. https://www.mmv.org/sites/default/files/uploads/images/achievements/MMV_KA2018.jpg. Accessed September 25, 2019.

US Agency for International Development website. Medicines for Malaria Venture (MMV) page. https://www.usaid.gov/global-health/health-areas/malaria/research-innovation/medicines-malaria-venture-mmv. Accessed September 25, 2019.

Continued progress is possible, not inevitable

Bill & Melinda Gates Foundation and Institute for Health Metrics and Evaluation. The Goalkeepers Report 2019: How Geography and Gender Stack the Deck For (or Against) You. Seattle: Bill & Melinda Gates Foundation; 2019. https://www.gatesfoundation.org/goalkeepers/report/2019-report/#ExaminingInequality.

Breakthroughs on the brink

Several long-acting injectable malaria prevention drugs, including one supported by NIH which uses nanotechnology: Martin A. Researchers develop long-acting malaria prevention drug. Homeland Preparedness News. January 24, 2018. https://homelandprepnews.com/stories/26379-researchers-develop-long-acting-malaria-prevention-drug/.

An experimental single-dose cure for Plasmodium falciparum malaria, DSM265: Anti-Malarial Shows Promise in Human Clinical Study [press release]. Washington, DC: American Society for Microbiology; March 11, 2019. https://www.asm.org/Press-Releases/Anti-Malarial-Shows-Promise-in-Human-Clinical-Stud.

More than a dozen malaria vaccine candidates are in late-stage clinical development: Policy Cures Research. Neglected Disease R&D Product Pipeline. Sydney, Australia: Policy Cures Research; 2019. https://www.pipeline.policycuresresearch.org/august2017.

A promising vector control intervention called Attractive Targeted Sugar Bait: Fiorenzano J, Koehler P, Zue R. Attractive toxic sugar bait (ATSB) for control of Mosquitoes and its impact on non-target organisms: a review. International Journal of Environmental Research and Public Health. 2017; 14(4): 398. https://doi.org/10.3390/ijerph14040398.

A first-of-its kind test that diagnoses malaria using a patient’s saliva: Farmer B. Saliva tests to diagnose malaria one step closer as researchers get £1m for trials. The Telegraph. October 1, 2019. https://www.telegraph.co.uk/global-health/science-and-disease/saliva-tests-diagnose-malaria-one-step-closer-researchers-get/.

US government investment in malaria R&D

Policy Cures Research. G-FINDER Public Search Tool. Sydney, Australia: Policy Cures Research; 2019. https://gfinder.policycuresresearch.org/PublicSearchTool/. [Early access to 2018 data was provided by Policy Cures Research in December 2019. Data is now available online as of January 30, 2019.]

US government R&D efforts

Kaiser Family Foundation. The President’s Malaria Initiative and Other U.S. Government Global Malaria Efforts. Washington, DC: Kaiser Family Foundation; 2019. https://www.kff.org/global-health-policy/fact-sheet/the-u-s-government-and-global-malaria/.

Kaiser Family Foundation. The U.S. Department of Defense & Global Health. Washington, DC: Kaiser Family Foundation; 2012. https://www.kff.org/global-health-policy/report/the-u-s-department-of-defense-global/.

US Food & Drug Administration website. Tropical disease priority review voucher program page. https://www.fda.gov/about-fda/center-drug-evaluation-and-research-cder/tropical-disease-priority-review-voucher-program. Accessed October 23, 2019.

Tuberculosis

R&D for Tuberculosis

How new tools can transform the fight

Tuberculosis (TB) has burdened humanity with symptoms including cough, fever, and emaciation for thousands of years. Today it is the world’s leading infectious disease killer: 10 million people fell ill from TB and 1.5 million died in 2018 alone. Yet only one low-efficacy TB vaccine exists, treatment takes months to years, and improved diagnostics designed specifically for low-resource settings are needed.

Meanwhile, 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.

1.5 million

people die annually from TB
$393,000

treatment cost and productivity loss of MDR-TB patient in US
45%

treatment failure rate for MDR-TB

Research successes

Technologies have transformed the fight against TB:

The first child-friendly TB medicines, developed with support from USAID, were introduced in 2015. Appropriately-dosed, dissolvable, and fruit-flavored for palatability, they have transformed treatment for children, with over 1 million courses ordered in 93 countries.
Bedaquiline, a drug to combat multidrug-resistant TB (MDR-TB), was approved by the FDA in 2012. Developed with early support from NIH, at the time it was the first new drug approved to treat TB in over 40 years.
Pretomanid, a new drug for highly drug-resistant TB, developed with USAID and NIH support, was approved by the FDA in 2019 as part of a combination regimen with bedaquiline and linezolid. The regimen reduces treatment time from up to two years to six months, while significantly improving treatment outcomes.
Xpert MTB/RIF, a fully automated diagnostic test, developed with NIH and DOD support, was introduced in the early 2010s. It is simple to use and produces results in two hours, compared to prior methods which took up to six weeks.
3HP, a shorter preventative regimen that can be taken weekly, rather than daily, to prevent latent TB from becoming active, was first introduced in 2011. Developed with CDC and NIH support, it is improving treatment completion.

Continued progress is possible, not inevitable

Key missing tools

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

Shorter, simplified treatment regimens for active TB to improve adherence and treatment outcomes and stem the rise of drug-resistant TB (DR-TB). Existing TB treatments can require thousands of pills and painful injections over the course of 6 to 20 months or longer, and certain drugs can cause severe side effects like liver damage and deafness.
Improved treatments for drug-resistant strains to lower the mortality rate of MDR-TB and extensively drug-resistant TB, the deadliest and most difficult to treat forms of TB.
New vaccines for prevention and treatment that are cost effective and address antimicrobial resistance (AMR). The TB vaccine currently is use was developed in 1921. Though effective at preventing some types of TB in infants, it offers inconsistent protection in adults against pulmonary TB, which affects the lungs.
Rapid, non-sputum-based diagnostics, suitable for low-resource settings and primary healthcare facilities, as well as rapid DR-TB tests that enable treatment to be tailored to individuals and help safeguard against AMR.

Breakthroughs on the brink

A new all-oral treatment regimen, BPaMZ, developed with NIH and USAID support, is in late-stage clinical trials, with the goal of reducing treatment time for drug-sensitive TB from 6 months to 4 months and for MDR-TB from 9 to 24 months to 6 months. Beyond BPaMZ, 13 new TB drug compounds are undergoing clinical trials.
More than ten potential preventative and immunotherapeutic TB vaccines are in development, including a vaccine candidate, M72/AS01E, that prevented active pulmonary TB from developing in just over half the adults who received it in a phase 2 clinical trial.
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 new models for vaccine testing, such as the controlled human infection model, which exposes trial participants to a pathogen in a highly-controlled, safe environment.
New innovative methods to administer treatment may lower the cost and burden of treatment by reducing the frequency and number of treatments that patients need. One example is a coiled wire device that slowly administers antibiotics into the stomach over several weeks, eliminating the need for daily oral pills.
A new point-of-care urine test to detect TB in HIV-positive individuals is currently being evaluated, while subsequent iterations of the test for use in broader populations are in development. DNA-based tests, including next-generation sequencing, and digital tools, including computer-assisted X-rays, are also in development, which could more quickly detect and differentiate strains of TB and MDR-TB, leading to faster and more appropriate treatments for patients.

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 conducts basic, translational, and clinical research to accelerate the development of new tools to diagnose, prevent, and treat TB.
US Agency for International Development 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 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.
Food and Drug Administration 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.

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Introduction

It is today the world’s leading infectious disease killer: 10 million people fell ill from TB and 1.5 million died: World Health Organization. Global Tuberculosis Report 2019. Geneva: World Health Organization; 2019. https://www.who.int/tb/publications/global_report/en/.

Only one low-efficacy vaccine exists, treatment takes months to years, and improved diagnostics designed specifically for low-resource settings are needed:

World Health Organization. Global investments in tuberculosis research and development: past, present, and future. A policy paper prepared for the first WHO global ministerial conference on ending tuberculosis in the sustainable development era: a multisectoral response. Geneva: World Health Organization; 2017. https://apps.who.int/iris/bitstream/handle/10665/259412/9789241513326-eng.pdf;jsessionid=BC5E45D3DD06E1CD7934C3B2172C6557?sequence=1.

Suleiman K, Lessem E. An Activist’s Guide to Tuberculosis Diagnostic Tools. New York: Treatment Action Group; 2017. http://www.treatmentactiongroup.org/sites/default/files/TB%20Diagnostics%20Guide.pdf.

$393 thousand treatment cost and productivity loss of MDR-TB patient in US: Centers for Disease Control and Prevention. The Costly Burden of Drug-Resistant TB in the U.S. Atlanta: Centers for Disease Control and Prevention; 2018. https://www.cdc.gov/nchhstp/newsroom/docs/factsheets/costly-burden-dr-tb-508.pdf.

45% treatment failure rate for MDR-TB: World Health Organization. Global Tuberculosis Report 2019. Geneva: World Health Organization; 2019. https://www.who.int/tb/publications/global_report/en/.

Research successes

First child-friendly TB medicines: TB Alliance website. Child-friendly medicines page. https://www.tballiance.org/child-friendly-medicines. Accessed August 27, 2019.

Bedaquiline, a drug to combat multi-drug resistant TB:

Mirsaeidi M. After 40 years, new medicine for combating TB. International Journal of Mycobacteriology. 2013;2(1):1–2. http://www.ijmyco.org/article.asp?issn=2212-5531;year=2013;volume=2;issue=1;spage=1;epage=2;aulast=Mirsaeidi.

NIH statement on World Tuberculosis Day [press release]. Bethesda, Maryland: National Institutes of Health; March 24, 2017. https://www.niaid.nih.gov/news-events/nih-statement-world-tuberculosis-day-0.

Pretomanid, a 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.

Xpert MTB/RIF, a fully automated diagnostic test: Lawn SD, Nicol MP. Xpert® MTB/RIF assay: development, evaluation and implementation of a new rapid molecular diagnostic for tuberculosis and rifampicin resistance. Future Microbiology. 2011 Sep;6(9): 1067–1082. https://www.futuremedicine.com/doi/10.2217/fmb.11.84?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%3dwww.ncbi.nlm.nih.gov.

3HP, a shorter preventative regimen: Centers for Disease Control and Prevention website. 3HP FAQs for providers page. https://www.cdc.gov/tb/education/FAQforProviders.htm. Accessed October 15, 2019.

Continued progress is possible, not inevitable

Bill & Melinda Gates Foundation and Institute for Health Metrics and Evaluation. The Goalkeepers Report 2019: How Geography and Gender Stack the Deck For (or Against) You. Seattle: Bill & Melinda Gates Foundation; 2019. https://www.gatesfoundation.org/goalkeepers/report/2019-report/#ExaminingInequality.

Key missing tools

Existing TB treatments take 6 to 20 months or longer, and certain drugs can cause severe side effects like liver damage and deafness: World Health Organization. WHO consolidated guidelines on drug-resistant tuberculosis treatment. Geneva: World Health Organization; 2019. https://apps.who.int/iris/bitstream/handle/10665/311389/9789241550529-eng.pdf?ua=1.

New vaccines: Schrager L, Harris R, Vekemans J. Research and development of new tuberculosis vaccines: a review. F1000 Research. 2019 Feb;7(1732). https://doi.org/10.12688/f1000research.16521.2.

Breakthroughs on the brink

A new all-oral treatment regimen, BPaMZ: TB Alliance. SimpliciTB and the BPaMZ Regimen. TB Alliance; 2019. https://www.dropbox.com/s/c1u2yj8zpz1pmha/tba_SimpliciTB_Factsheet.pdf?dl=0.

13 new compounds are undergoing clinical trials: World Health Organization. Global tuberculosis report 2019. Geneva: World Health Organization; 2019. https://www.who.int/tb/publications/global_report/en/.

More than ten potential preventative and immunotherapeutic TB vaccines are in development:

Tuberculosis Vaccine Initiative. Pipeline of Vaccines. Netherlands; 2015. https://www.tbvi.eu/what-we-do/pipeline-of-vaccines/. Accessed August 29, 2019.

Schrager L, Harris R, Vekemans J. Research and development of new tuberculosis vaccines: a review. F1000 Research. 2019 Feb;7(1732). https://doi.org/10.12688/f1000research.16521.2.

M72/AS01E vaccine candidate: GSK candidate vaccine helps prevent active pulmonary tuberculosis in HIV negative adults in phase II study [press release]. London: Aeras; September 25, 2018. https://www.aeras.org/pressreleases/gsk-candidate-vaccine-helps-prevent-active-pulmonary-tuberculosis-in-hiv-ne#.XaYRKkZKiUk.

New approaches and strategies for TB vaccine research: Schrager L, Harris R, Vekemans J. Research and development of new tuberculosis vaccines: a review. F1000 Research. 2019 Feb;7(1732). https://doi.org/10.12688/f1000research.16521.2.

New innovative methods to administer treatment:

Verma M, Vishwanath K, Eweje F, et al. A gastric resident drug delivery system for prolonged gram-level dosing of tuberculosis treatment. Science Translational Medicine. 2019;11(483):eaau6267. https://doi.org/10.1126/scitranslmed.aau6267

Omwoyo WN, Ogutu B, Oloo F, et al. Preparation, characterization, and optimization of primaquine-loaded solid lipid nanoparticles. International Journal of Nanomedicine. 2014;9(1):3865–3874. https://doi.org/10.2147/IJN.S62630.

A new point-of-care urine test: Mahon C. New rapid test significantly more effective for TB-diagnosis in people with HIV. Avert. June 11, 2019. https://www.avert.org/news/new-rapid-test-significantly-more-effective-tb-diagnosis-people-hiv.

DNA-based tests and digital tools: World Health Organization. Global tuberculosis report 2018. Geneva: World Health Organization; 2018. https://www.who.int/tb/publications/global_report/en/.

US government investment in tuberculosis R&D

Policy Cures Research. G-FINDER Public Search Tool. Sydney, Australia: Policy Cures Research; 2019. https://gfinder.policycuresresearch.org/PublicSearchTool/. [Early access to 2018 data was provided by Policy Cures Research in December 2019. Data is now available online as of January 30, 2019.]

US government R&D efforts

Department of Defense Congressionally Directed Medical Research Programs. Search Awards. Fort Detrick, Maryland: Congressionally Directed Medical Research Programs. https://cdmrp.army.mil/search.aspx. Accessed August 26, 2019.

Kaiser Family Foundation. The U.S. Government and Global Tuberculosis Efforts. Washington, DC: Kaiser Family Foundation; 2019. https://www.kff.org/global-health-policy/fact-sheet/the-u-s-government-and-global-tuberculosis-efforts/.

US Food & Drug Administration website. Tropical Disease Priority Review Voucher Program page. https://www.fda.gov/about-fda/center-drug-evaluation-and-research-cder/tropical-disease-priority-review-voucher-program. Accessed October 23, 2019.

US Food & Drug Administration website. Limited Population Pathway for Antibacterial and Antifungal Drugs – the LPAD Pathway. https://www.fda.gov/drugs/development-resources/limited-population-pathway-antibacterial-and-antifungal-drugs-lpad-pathway. Accessed October 23, 2019.

Neglected Tropical Diseases

R&D for Neglected Tropical Diseases

How new tools can transform the fight

Neglected tropical diseases (NTDs) are a group of infectious diseases that exert a crushing burden in poor and vulnerable communities around the world. Named for the limited attention they’ve 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 research and development (R&D) of new tools.

1 in 6 people

worldwide are infected with one or more NTD
149 countries

in which NTDs are endemic
<1% of new drugs

approved between 1975–2011 were for NTDs

Research successes

New tools are advancing control and elimination of NTDs:

The first all-oral cure for sleeping sickness, a fatal neurological disease, was approved in 2018. This new drug, fexinidazole, cures all stages of the disease in ten days, replacing previous treatments that involved two weeks of intravenous injections at a hospital.
The first new treatment for river blindness in 20 years, moxidectrin, was approved in 2018, adding ammunition to the fight against this disfiguring and blinding disease.
A dissolvable, easy-to-use child-friendly Chagas drug, pediatric benznidazole, was approved in 2011 to combat this parasitic disease that can cause heart failure.
New shorter, more effective combination therapies for visceral leishmaniasis—a disease that causes spleen and liver damage—that were introduced in the 2000s, are improving treatment and slowing the rise of drug resistance.
Several diagnostics, including rapid tests for river blindness and elephantiasis, developed using NIH research, and a test that detects all four types of dengue, developed by the CDC, were introduced in the last decade.

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 point of care in low-resource settings, and in some cases, to distinguish between different strains or stages of a disease.
New and improved treatments and cures including therapies designed for children and shorter, simplified regimens with fewer side effects. Only seven NTDs are considered “tool-ready”—meaning we have low-cost, effective interventions to combat them—but in many cases these available treatments are still lengthy and burdensome to take with significant side effects.
Vaccines to prevent and treat infection. No vaccines exist for 20 of the 21 NTDs prioritized by the World Health Organization, and the existing vaccine for dengue is recommended for use only in select populations.
New vector and biological control tools, like space spray insecticides and Wolbachia bacteria that disrupts reproduction, in order to reduce mosquito and other vector populations.
Antivenoms to treat snakebites.

Breakthroughs on the brink

Several chikungunya vaccine candidates are in late-stage development, including one NIH-supported candidate ready to enter phase 3 clinical trials and a second NIH-supported candidate in phase 2 trials, bringing new hope that in the coming years we will have a vaccine against this disease that causes debilitating joint pain and fever.
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.
New tools to control schistosomiasis—a disease that causes liver damage or kidney failure if left untreated—are being developed, including an oral dispersible formulation of the medicine praziquantel designed for children soon entering phase 3 trials and a vaccine candidate Sm14 that has completed a phase 2 trial.
Phase 2/3 clinical trials are underway for fosravuconazole, a potential new treatment for eumycetoma, a fungal disease that can cause severe deformity of the limbs. Existing treatments for this disease are toxic, costly, only cure a fraction of patients, and require 12 months of treatment.
Two NIH-funded vaccine candidates against hookworm infection are in clinical development, including one in phase 2 trials. This parasitic infection causes gastrointestinal issues and protein deficiency and is linked to impaired learning in children.
Mosquitoes infected with Wolbachia, a bacterium that impacts their reproduction and reduces 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 2018) 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:

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

Download PDF

Introduction

1 in 6 people worldwide are infected with one or more NTD: Kaiser Family Foundation. The U.S. Government and Global Neglected Tropical Disease Efforts. Washington, DC: Kaiser Family Foundation; 2019. http://files.kff.org/attachment/fact-sheet-the-u-s-government-and-global-neglected-tropical-diseases.

149 countries in which NTDs are endemic: World Health Organization (WHO) website. Neglected tropical diseases page. https://www.who.int/neglected_diseases/diseases/en/. Accessed June 26, 2019.

<1% of new drugs approved between 1975–2011 were for NTDs: Weng H, Chen H, Wang M. Innovation in neglected tropical disease drug discovery and development. Infectious Diseases of Poverty. 2018;7(67). https://doi.org/10.1186/s40249-018-0444-1

Research successes

First all-oral cure for sleeping sickness: Drugs for Neglected Diseases initiative website. Fexinidazole to treat sleeping sickness page. https://www.dndi.org/achievements/fexinidazole/. Accessed June 26, 2019.

New treatment for river blindness: First new treatment for river blindess approved by U.S. FDA in 20 years [press release]. Geneva, Switzerland: WHO Special Programme for Research and Training in Tropical Diseases; June 14, 2018. https://www.who.int/tdr/news/2018/moxidectin-approved-as-treatment-for-river-blindness/en/.

Child-friendly Chagas drug: Drugs for Neglected Diseases initiative website. Paediatric benznidazole page. https://www.dndi.org/achievements/paediatric-benznidazole/. Accessed June 26, 2019.

Combination therapies for visceral leishmaniasis: Drugs for Neglected Diseases initiative website. Sodium stibogluconate & paramomycin (SSG&PM) page. https://www.dndi.org/achievements/ssg-pm/. Accessed June 26, 2019.

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

Test that detects all four types of dengue: Global Biodefense. CDC begins distribution of new dengue fever tests. Global Biodefense. June 28, 2012. https://globalbiodefense.com/2012/06/28/cdc-begins-distribution-of-new-dengue-fever-tests/

Continued progress is possible, not inevitable

Bill & Melinda Gates Foundation and Institute for Health Metrics and Evaluation. The Goalkeepers Report 2019: How Geography and Gender Stack the Deck For (or Against) You. Seattle: Bill & Melinda Gates Foundation; 2019. https://www.gatesfoundation.org/goalkeepers/report/2019-report/#ExaminingInequality.

Key missing tools

Only seven NTDs are considered “tool-ready”: Kaiser Family Foundation. The U.S. Government and Global Neglected Tropical Disease Efforts. Washington, DC: Kaiser Family Foundation; 2019. http://files.kff.org/attachment/fact-sheet-the-u-s-government-and-global-neglected-tropical-diseases.

No vaccine exists for 20 of the 21 NTDs: WHO website. Neglected tropical diseases page. https://www.who.int/neglected_diseases/diseases/en/. Accessed June 26, 2019.

Breakthroughs on the brink

Several chikungunya vaccine candidates, candidate ready to enter phase 3 trials:

Liu A. Themis’ chikungunya vaccine, furthest in development, nabs fast FDA review. FiercePharma. February 26, 2019. https://www.fiercepharma.com/vaccines/themis-chikungunya-vaccine-furthest-development-nabs-fast-fda-review.

NIAID-sponsored trial of experimental chikungunya vaccine begins [press release]. Bethesda, MD: National Institutes of Health; June 5, 2017. https://www.nih.gov/news-events/news-releases/niaid-sponsored-trial-experimental-chikungunya-vaccine-begins.

Several chikungunya vaccine candidates, candidate in phase 2 trials:

Liu A. Themis’ chikungunya vaccine, furthest in development, nabs fast FDA review. FiercePharma. February 26, 2019. https://www.fiercepharma.com/vaccines/themis-chikungunya-vaccine-furthest-development-nabs-fast-fda-review.

Shanley M. FDA grants fast track designation to chikungunya vaccine. MD Magazine. May 4, 2018. https://www.mdmag.com/medical-news/fast-track-designation-chikungunya-vaccine.

Diagnostic tests for Buruli ulcer: FIND. DX Pipeline Tracker. Geneva, Switzerland: FIND; 2019. https://www.finddx.org/dx-pipeline/. Accessed June 26, 2019.

New tools to control schistosomiasis, praziquantel designed for children: Pediatric Praziquantel Consortium website. The pediatric formulation page. https://www.pediatricpraziquantelconsortium.org/pediatric-formulation. Accessed June 26, 2019.

New tools to control schistosomiasis, vaccine candidate SM14: ClinicalTrials.gov. NCT03799510: Anti-Schistosomiasis Vaccine: SM14 Phase 2b-Sn in School Children. https://clinicaltrials.gov/ct2/show/NCT03799510. Accessed June 26, 2019.

Fosravuconazole, a potential new treatment for eumycetoma: Drugs for Neglected Diseases initiative website. Fosravuconazole page. https://www.dndi.org/diseases-projects/portfolio/fosravuconazole/. Accessed June 26, 2019.

Vaccine candidates against hookworm infection: Bottazzi MA. Human hookworm vaccine initiative: a public health value proposition leading to societal impact and positive financial returns. Presented at: the Third Global Vaccine and Immunization Research Forum, March 22, 2018; Bangkok, Thailand [poster presentation]. https://www.who.int/immunization/research/forums_and_initiatives/gvirf/forum_2018/en/index2.html.

Mosquitoes infected with Wolbachia: World Mosquito Program website. Projects page. https://www.worldmosquitoprogram.org/. Accessed June 26, 2019.

National Institute of Allergy and Infectious Diseases website. Neglected tropical diseases scientific findings page. https://www.niaid.nih.gov/research/neglected-tropical-diseases-scientific-findings. Accessed June 26, 2019.

Combating Zika and Future Threats: A Grand Challenge for Development website. Wolbachia-infected mosquitoes page. https://www.zikagrandchallenge.net/project/wolbachia-infected-mosquitoes.html. Accessed June 26, 2019.

US government investment in NTD R&D

Policy Cures Research. G-FINDER Public Search Tool. Sydney, Australia: Policy Cures Research; 2019. https://gfinder.policycuresresearch.org/PublicSearchTool/. [Early access to 2018 data was provided by Policy Cures Research in December 2019. Data is now available online as of January 30, 2019. Disease categories included in calculation are kinetoplastid diseases, dengue, helminth infections, leprosy, buruli ulcer, leptospirosis, trachoma, mycetoma, and snakebite envenoming.]

US government R&D efforts

Kaiser Family Foundation. The U.S. Government and Global Neglected Tropical Disease Efforts. Washington, DC: Kaiser Family Foundation; 2019. http://files.kff.org/attachment/fact-sheet-the-u-s-government-and-global-neglected-tropical-diseases.

Salaam-Blyther T. Neglected Tropical Diseases: Background, Responses, and Issues for Congress. Washington, DC: Congressional Research Service; 2011. https://fas.org/sgp/crs/misc/R41607.pdf.

US Food & Drug Administration website. Tropical disease priority review voucher program page. https://www.fda.gov/about-fda/center-drug-evaluation-and-research-cder/tropical-disease-priority-review-voucher-program. Accessed October 23, 2019.

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.. An outbreak in a remote corner of the world can quickly become a global crisis with devastating economic and human 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 is vital to overcome these barriers and develop the tools needed to prevent, detect, and respond to these threats when we need them most.

$570 billion

projected average annual cost of pandemics over coming decades
11,310 lives

human loss from 2014 West Africa Ebola outbreak
3x increase

in annual desease outbreaks since 1980

Key missing tools

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

	Vaccines	Treatments	Diagnostics
Crimean-Congo Haemorrhagic fever (CCHF)	No FDA-approved vaccine is available. A Bulgarian-licensed vaccine exists, but its efficacy is unclear. Most advanced other candidate is in early human trials.	No specific licensed treatment is available. An existing antiviral has been used during outbreaks, but its efficacy remains unclear, and an experimental Ebola drug has shown initial promise.	Commercial tests are available but require sophisticated lab infrastructure. Rapid, point-of-care (POC) tests are needed.
Ebola Zaire	One vaccine, VSV-ZEBOV, is approved by the FDA and European regulators, while a second is being used on an emergency basis. Additional vaccines are in early development stages.	Two experimental treatments have been found highly effective and are being used on an emergency basis, but neither has full regulatory approval.	One rapid test is FDA-approved, while additional diagnostics, including rapid and portable tests, have been approved for emergency use with further validation studies needed.
Marburg & Ebola Sudan	No licensed vaccine is available. Most advanced candidate is in early human trials.	No specific licensed treatment is available. Most advanced candidate is in early human trials.	Confirmatory tests are available but require sophisticated lab infrastructure. Rapid POC tests are needed.
Lassa fever	No licensed vaccine is available. Most advanced candidate is in early human trials.	No specific licensed treatment is available. The antiviral ribavirin is effective if used in early disease stages, but more robust assessment is needed.	Confirmatory tests are available but require sophisticated lab infrastructure. Rapid POC tests and tests to distinguish symptomatic stages are needed.
Middle East respiratory syndrome coronavirus	No licensed vaccine is available. Most advanced candidate is in early human trials.	No specific licensed treatment is available. Most advanced candidate is in early human trials.	Confirmatory tests are available at a laboratory level and rapid tests are FDA-authorized for emergency use. POC tests for humans and animals are needed.
Nipah	No licensed vaccine is available. Most advanced candidate is in preclinical research.	No specific licensed treatment is available. Most advanced candidate is in early human trials.	Confirmatory tests are available but require sophisticated lab infrastructure and could benefit from further validation. Rapid POC tests are needed.
Rift Valley fever (RVF)	No licensed human vaccine is available, though animal vaccines are used. Several candidates are in early stages of development.	No specific licensed treatment is available.	Confirmatory tests are available but require sophisticated lab infrastructure. Rapid POC tests for humans and animals are needed.
Zika	No licensed vaccine is available. Several candidates are in development, including ones in late-stage human trials.	No specific licensed treatment is available. Several therapies are in clinical development	Commercial tests are available with additional tests authorized under emergency use protocols.
Pandemic influenza	No licensed universal flu vaccine is available. Most advanced candidate is in late-stage human trials.	Existing antivirals may be effective. Additional broad-spectrum antivirals are needed.	Rapid, POC tests will be needed.

Research progress

The VSV-ZEBOV Ebola vaccine, which is now approved by the FDA and European regulators, demonstrated a 97% protection rate during the 2018/2019 outbreak. The development of this vaccine, as well as a second deployed vaccine and other candidates in early stages of development, are supported by partnerships with NIH, DoD, and BARDA.
Two experimental Ebola treatments were found to dramatically improve survival rates during a NIH-funded trial during the 2018/2019 outbreak. One was developed by scientists at DoD and NIH, while the other was advanced through a partnership with BARDA.
A candidate vaccine for Marburg—a deadly cousin of the Ebola virus—which was developed by NIH, entered first-in-human trials led by the DoD Walter Reed Army Institute of Research (WRAIR) in 2018. Development of this vaccine is now being continued with BARDA support.
A candidate vaccine for Middle East respiratory syndrome coronavirus (MERS-CoV), a severe respiratory disease, completed first-in-human studies in July 2019 and will progress to phase 2 studies in the Middle East. DoD funded the study which was conducted at WRAIR.
The most advanced universal flu vaccine candidate, which was developed with support from NIH, began phase 3 clinical trials in 2018. Additional candidates supported by NIH are in clinical development with the goal of achieving a vaccine that is highly effective against multiple strains of influenza.
Over a dozen Zika vaccine candidates are in clinical development, including candidates developed with support from NIH, DoD, and BARDA.
Numerous diagnostic tools for EIDs have been advanced, including 1 test for Ebola and 2 tests for Zika that have FDA approval, as well as additional tests authorized by the FDA for emergency use including 10 tests for Ebola, 2 for MERS-COV, and 16 for Zika.

Global funding for R&D for World Health Organization R&D Blueprint EIDs, 2017

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 supports advanced development of vaccines, drugs, diagnostics, and other medical countermeasures against EIDS considered a threat to national security and has authority to add them to the US Strategic National Stockpile.
National Institutes of Health conducts basic and clinical research to advance new treatments, vaccines, diagnostics, and vector control products to combat EIDs.
Department of Defense 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 development of select tools to combat EIDs designed for low-resource settings, primarily through the Grand Challenges for Ebola and Zika programs.
Food and Drug Administration administers the Tropical Disease Priority Review Program to incentivize industry investment in products for select EIDs and grants emergency use authorization to permit use of unapproved products during an emergency.

Download PDF

Introduction

$570 billion projected average annual cost of pandemics over coming decades/11,310 lives lost from 2014 West Africa Ebola outbreak: Center for Strategic and International Studies. Harnessing Multilateral Financing for Health Security Preparedness. Washington, DC: Center for Strategic and International Studies; 2019. https://www.csis.org/analysis/harnessing-multilateral-financing-health-security-preparedness

3x increase in annual disease outbreaks: Smith K, Golberg, M, Rosenthal S, et al. Global rise in human infectious disease outbreaks. Journal of the Royal Society. 2014; 11(101) 1742-5662. https://doi.org/10.1098/rsif.2014.0950.

Key missing tools

Crimean-Congo Haemorrhagic fever:

World Health Organization website. Crimean-Congo haemorrhagic fever page. https://www.who.int/news-room/fact-sheets/detail/crimean-congo-haemorrhagic-fever. Accessed August 16, 2019.

World Health Organization. Roadmap for Research and Product Development against Crimean-Congo Heamorrhagic Fever (CCHF)-Draft. Geneva: World Health Organization; 2018. https://www.who.int/blueprint/priority-diseases/key-action/cchf-draft-r-and-d-roadmap.pdf.

Clinicaltrails.gov. NCT03020771: Phase I Study to Evaluate Basic Pharmacodynamic, Pharmacological and Toxicological Effects of the Newly Developed Crimean-Congo Hemorrhagic Fever Vaccine for Humans. https://clinicaltrials.gov/ct2/show/NCT03020771. Accessed August 16, 2019.

Mazzola L, Cassandra KC. Diagnostic tests for Crimean-Congo haemorrhagic fever: a widespread tickborne disease. BMJ Global Health. 2019; 4e001114. http://dx.doi.org/10.1136/bmjgh-2018-001114.

Ebola Zaire:

Branswell H. FDA approves an Ebola vaccine, long in development, for the first time. STAT. December 19, 2019. https://www.statnews.com/2019/12/19/fda-approves-an-ebola-vaccine-long-in-development-for-the-first-time/

WHO website. Ebola virus disease page. https://www.who.int/news-room/fact-sheets/detail/ebola-virus-disease. Accessed October 22, 2019.

WHO. Ebola/Marburg Research and Development (R&D) Roadmap. Geneva: WHO; 2018. https://www.who.int/blueprint/priority-diseases/key-action/Ebola-Marburg_Draft_Roadmap_publiccomment_MAY2018.pdf?ua=1.

Branswell, Helen. Ebola vaccine approved in Europe in landmark moment in fight against a deadly disease. STAT. November 11, 2019. https://www.statnews.com/2019/11/11/ebola-vaccine-approved-in-europe-in-landmark-moment-in-fight-against-a-deadly-disease/.

Second Ebola vaccine to complement “ring vaccination” given green light in DRC [press release]. Geneva, Switzerland: World Health Organization; September 23, 2019. https://www.who.int/news-room/detail/23-09-2019-second-ebola-vaccine-to-complement-ring-vaccination-given-green-light-in-drc.

McNeil D. A cure for Ebola? Two new treatments prove highly effective in Congo. The New York Times. August 12, 2019. https://www.nytimes.com/2019/08/12/health/ebola-outbreak-cure.html.

FDA allows marketing of first rapid diagnostic test for detecting Ebola virus antigens [press release]. Silver Spring, Maryland: US Food and Drug Administration; October 10, 2019. https://www.fda.gov/news-events/press-announcements/fda-allows-marketing-first-rapid-diagnostic-test-detecting-ebola-virus-antigens.

WHO website. Essential medicines and health products: Ebola diagnostics page. https://www.who.int/medicines/ebola-treatment/emp_ebola_diagnostics/en/. Accessed August 16, 2019.

US Food and Drug Administration website. Emergency Use Authorization page. https://www.fda.gov/emergency-preparedness-and-response/mcm-legal-regulatory-and-policy-framework/emergency-use-authorization#ebola. Accessed October 24, 2019.

Marbug & Ebola Sudan:

WHO website. Marburg virus disease page. https://www.who.int/en/news-room/fact-sheets/detail/marburg-virus-disease. Accessed August 16, 2019.

WHO. Ebola/Marburg Research and Development (R&D) Roadmap. Geneva: WHO; 2018. https://www.who.int/blueprint/priority-diseases/key-action/Ebola-Marburg_Draft_Roadmap_publiccomment_MAY2018.pdf?ua=1.

WRAIR begins Phase 1 clinical trial of Marburg vaccine [press release]. Silver Spring, Maryland: Military Health System; October 19, 2018. https://health.mil/News/Articles/2018/10/19/WRAIR-begins-Phase-1-clinical-trial-of-Marburg-vaccine?page=3#pagingAnchor.

Sabin Vaccine Institute receives $20.5 million from BARDA with potential of up to $128 million to develop Ebola Sudan and Marburg Vaccines [press release]. Washington, DC: Sabin Vaccine Institute; October 2, 2019. https://www.globenewswire.com/news-release/2019/10/01/1923382/0/en/Sabin-Vaccine-Institute-Receives-20-5-Million-From-BARDA-With-Potential-of-up-to-128-Million-to-Develop-Ebola-Sudan-and-Marburg-Vaccines.html.

BioCryst initiates phase 1 clinical trial of Galidesivir [press release]. Research Triangle Park, North Carolina: BioCryst; January 2, 2019. http://ir.biocryst.com/news-releases/news-release-details/biocryst-initiates-phase-1-clinical-trial-galidesivir.

Lassa fever:

World Health Organization website. Lassa fever page. https://www.who.int/en/news-room/fact-sheets/detail/lassa-fever. Accessed August 16, 2019.

World Health Organization. Lassa Fever Research and Development (R&D) Roadmap. Geneva: World Health Organization; 2018. https://www.who.int/blueprint/priority-diseases/key-action/LassaFever_Draft_Roadmap_publiccomment_MAY2018.pdf?ua=1.

Inovio first to advance Lassa fever candidate vaccine into a clinical trial [press release]. Plymouth Meeting, Pennsylvania and Oslo, Norway: Inovio; May 21, 2019. http://ir.inovio.com/news-and-media/news/press-release-details/2019/Inovio-First-to-Advance-Lassa-Fever-Candidate-Vaccine-Into-a-Clinical-Trial/default.aspx.

GeoVax announces upcoming presentation of Lassa fever vaccine data [press release]. Atlanta, Georgia: GeoVax; July 16, 2019. https://www.globenewswire.com/news-release/2019/07/16/1883361/0/en/GeoVax-Announces-Upcoming-Presentation-of-Lassa-Fever-Vaccine-Data.html.

Middle East respiratory syndrome coronavirus:

World Health Organization website. Middle East respiratory syndrome coronavirus (MERS-CoV) page. https://www.who.int/news-room/fact-sheets/detail/middle-east-respiratory-syndrome-coronavirus-(mers-cov). Accessed August 16, 2019.

Modjarrad K, Moorthy V, Embarek P, et al. A roadmap for MERS-CoV research and product development: report from a World Health Organization consultation. Nature Medicine. 2016; 22(7):701-5. https://doi.org/10.1038/nm.4131.

Modjarrad K. Research and Development Activities for Middle East Respiratory Syndrome: The Current Landscape. Geneva: World Health Organization; 2015: https://www.who.int/blueprint/priority-diseases/key-action/mers-landscape.pdf?ua=1.

Galford C. Phase 1 first-in-human clinical trial shows promise for MERS CoV vaccine candidate. Homeland Preparedness News. July 26, 2019. https://homelandprepnews.com/stories/35014-phase-1-first-in-human-clinical-trial-shows-promise-for-mers-cov-vaccine-candidate/.

Cassandra K, Mazzola L, Chua A, et al. An updated roadmap for MERS-CoV research and product development: focus on diagnostics. BMJ Global Health. 2019; 4(2): e001105. https://dx.doi.org/10.1136%2Fbmjgh-2018-001105.

Nipah:

WHO website. Nipah virus page. https://www.who.int/news-room/fact-sheets/detail/nipah-virus. Accessed August 16, 2019.

Denis M. NIPAH Baseline Situation Analysis – Draft. Geneva: WHO; 2018. https://www.who.int/blueprint/priority-diseases/key-action/WHO_NIPAH_baseline_situation_analysis_27Jan2018.pdf?ua=1.

WHO. Nipah Research and Development (R&D) Roadmap. Geneva: WHO; 2018. https://www.who.int/blueprint/priority-diseases/key-action/Nipah_Draft_Roadmap_publiccomment_MAY2018.pdf?ua=1.

Pacha A. Nipah virus: experimental antiviral drug shows promise. The Hindu. June 29, 2019. https://www.thehindu.com/sci-tech/science/nipah-virus-experimental-antiviral-drug-shows-promise/article28228467.ece.

Rift Valley fever:

WHO website. Rift Valley fever page. https://www.who.int/news-room/fact-sheets/detail/rift-valley-fever. Accessed August 16, 2019.

Wyckoff J. Colorado State University receives $9.5M to develop Rift Valley fever vaccine. Healio Infectious Disease News. July 22, 2019. https://www.healio.com/infectious-disease/vaccine-preventable-diseases/news/online/%7Bb2508152-67f9-4dec-b3bf-ceef43314a45%7D/colorado-state-university-receives-95m-to-develop-rift-valley-fever-vaccine.

Zika:

WHO. Current Zika Product Pipeline. Geneva: WHO; 2016. https://www.who.int/blueprint/priority-diseases/key-action/zika-rd-pipeline.pdf?ua=1.

WHO website. WHO vaccine pipeline tracker page. https://www.who.int/immunization/research/vaccine_pipeline_tracker_spreadsheet/en/. Accessed August 16, 2019.

US Food and Drug Administration website. Emergency Use Authorization page. https://www.fda.gov/emergency-preparedness-and-response/mcm-legal-regulatory-and-policy-framework/emergency-use-authorization#ebola. Accessed October 24, 2019.

Pandemic influenza:

Tayor A. first universal flu vaccine to enter phase 3 trial. The Scientist. November 12, 2018. https://www.the-scientist.com/news-opinion/first-universal-flu-vaccine-to-enter-phase-3-trial-65073.

U.S. Department of Health and Human Services. Pandemic Influenza Plan: 2017 Update. Washington, DC: U.S. Department of Health and Human Services; 2017. https://www.cdc.gov/flu/pandemic-resources/pdf/pan-flu-report-2017v2.pdf.

Research Progress

Ebola vaccine:

Branswell H. FDA approves an Ebola vaccine, long in development, for the first time. STAT. December 19, 2019. https://www.statnews.com/2019/12/19/fda-approves-an-ebola-vaccine-long-in-development-for-the-first-time/

Branswell, Helen. Ebola vaccine approved in Europe in landmark moment in fight against a deadly disease. STAT. November 11, 2019. https://www.statnews.com/2019/11/11/ebola-vaccine-approved-in-europe-in-landmark-moment-in-fight-against-a-deadly-disease/?.

Ebola vaccine more than 97% effective in DRC outbreak. Healio Infectious Disease News. April 15, 2019. https://www.healio.com/infectious-disease/emerging-diseases/news/online/%7B595b1729-f589-4a9a-8aef-ef4b4b05866b%7D/ebola-vaccine-more-than-97-effective-in-drc-outbreak.

Second Ebola vaccine to complement “ring vaccination” given green light in DRC [press release]. Geneva, Switzerland: WHO; September 23, 2019. https://www.who.int/news-room/detail/23-09-2019-second-ebola-vaccine-to-complement-ring-vaccination-given-green-light-in-drc.

National Institute of Allergy and Infectious Diseases website. Ebola vaccines page. https://www.niaid.nih.gov/diseases-conditions/ebola-vaccines. Accessed August 16, 2019.

HHS accelerates development of first Ebola vaccines and drugs [press release]. Washington, DC: Department of Health and Human Services; September 29, 2017. https://www.hhs.gov/about/news/2017/09/29/hhs-accelerates-development-first-ebola-vaccines-and-drugs.html.

U.S. Department of Defense website. DoD Helps Fight Ebola in Liberia and West Africa page. https://archive.defense.gov/home/features/2014/1014_ebola/. Accessed August 16, 2019.

Experimental Ebola treatments: A cure for Ebola? Two new treatments prove highly effective in Congo. The New York Times. August 12, 2019. https://www.nytimes.com/2019/08/12/health/ebola-outbreak-cure.html.

Vaccine for Marburg:

WRAIR begins phase 1 clinical trial of Marburg vaccine [press release]. Silver Spring, Maryland: Military Health System; October 19, 2018. https://health.mil/News/Articles/2018/10/19/WRAIR-begins-Phase-1-clinical-trial-of-Marburg-vaccine?page=3#pagingAnchor.

Sabin Vaccine institute receives $20.5 million from BARDA with potential of up to $128 million to develop Ebola Sudan and Marburg vaccines [press release]. Washington, DC: Sabin Vaccine Institute; October 2, 2019. https://www.globenewswire.com/news-release/2019/10/01/1923382/0/en/Sabin-Vaccine-Institute-Receives-20-5-Million-From-BARDA-With-Potential-of-up-to-128-Million-to-Develop-Ebola-Sudan-and-Marburg-Vaccines.html.

Vaccine for Middle East respiratory syndrome coronavirus: Galford C. Phase 1 first-in-human clinical trial shows promise for MERS CoV vaccine candidate. Homeland Preparedness News. July 26, 2019. https://homelandprepnews.com/stories/35014-phase-1-first-in-human-clinical-trial-shows-promise-for-mers-cov-vaccine-candidate/

Universal flu vaccine: First universal flu vaccine to enter phase 3 trial. The Scientist. November 12, 2018. https://www.the-scientist.com/news-opinion/first-universal-flu-vaccine-to-enter-phase-3-trial-65073.

Dozen Zika vaccine candidates: WHO website. WHO vaccine pipeline tracker page. https://www.who.int/immunization/research/vaccine_pipeline_tracker_spreadsheet/en/. Accessed August 16, 2019.

Diagnostic tools for EIDS:

FDA allows marketing of first rapid diagnostic test for detecting Ebola virus antigens [press release]. Silver Spring, Maryland: US Food and Drug Administration. October 10, 2019. https://www.fda.gov/news-events/press-announcements/fda-allows-marketing-first-rapid-diagnostic-test-detecting-ebola-virus-antigens.

US Food and Drug Administration website. Emergency Use Authorization page. https://www.fda.gov/emergency-preparedness-and-response/mcm-legal-regulatory-and-policy-framework/emergency-use-authorization#ebola. Accessed August 16, 2019.

Global Funding for R&D for World Health Organization Blueprint Priority EIDs, 2017

Data from forthcoming report from Policy Cures Research assessing global investment in R&D for EIDs identified in the World Health Organization’s R&D Blueprint.

US government R&D efforts

Office of the Assistant Secretary for Preparedness and Response website. Biomedical Advanced Research and Development Authority page. https://www.phe.gov/about/barda/Pages/default.aspx. Accessed August 16, 2019.

National Institute of Allergy and Infectious Diseases website. Biodefense and emerging infectious diseases page. https://www.niaid.nih.gov/research/biodefense-emerging-infectious-diseases. Accessed August 16, 2019.

Cullison T, Morrison JS. The U.S. Department of Defense’s Role in Health Security: Current Capabilities and Recommendations for the Future. Washington, DC: Center for Strategic and International Studies; 2019. https://csis-prod.s3.amazonaws.com/s3fs-public/publication/190701_CullisonMorrison_DoDHealthSecurity_WEB_v2.pdf.

7 ways CDC is keeping us safe from deadly diseases [blog post]. Washington, DC: Global Health Technologies Coalition; August 27, 2017. https://www.ghtcoalition.org/blog/7-ways-cdc-is-keeping-us-safe-from-deadly-diseases.

Fighting Ebola: A Grand Challenge for Development website. http://www.ebolagrandchallenge.net/. Accessed August 16, 2019.

Combating Zika and Future Threats: A Grand Challenge for Development website. https://www.usaid.gov/grandchallenges/zika. Accessed August 16, 2019.

US Food & Drug Administration website. Tropical disease priority review voucher program page. https://www.fda.gov/about-fda/center-drug-evaluation-and-research-cder/tropical-disease-priority-review-voucher-program. Accessed October 23, 2019.

US Food and Drug Administration website. Emergency Use Authorization page. https://www.fda.gov/emergency-preparedness-and-response/mcm-legal-regulatory-and-policy-framework/emergency-use-authorization#ebola. Accessed August 16, 2019.

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 80% of all global maternal and child deaths occur in sub-Saharan African and Southern Asia. Having the right intervention or tool—drug, vaccine, device, or diagnostic—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 nearly 60% and maternal deaths by nearly 45% since 1990. But vast challenges remain. Intensified research and development (R&D) efforts are needed to create low-cost, easy-to-use technologies to address common causes of maternal and child deaths in the world’s poorest places.

5.3 million

children under five die each year
290,000

women die each year in pregnancy and childbirth
214 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, pneumonia, and other illnesses, save two to three million lives each year.
Contraceptive innovations, from decades-old tools like oral pills and implants, to newer options like a self-insertable, one-year vaginal ring and a discreet, self-injectable, long-acting contraception, have helped millions of women meet their unique family planning needs. Research shows that expanding access to contraception could reduce maternal deaths by nearly one-third and child deaths by one-quarter.
Tools to combat childhood malaria—including insecticide-treated bednets and child-friendly malaria medicines—have significantly reduced child deaths.
Interventions to treat and prevent diarrhea—including oral rehydration solutions, zinc supplements, and low-cost vaccines against diarrheal diseases like rotavirus, cholera, and typhoid—have helped reduce child mortality from diarrheal disease by more than one-third since 2005.
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 41% 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 that lack access to high-tech equipment, including low-cost, durable, easy-to-use neonatal resuscitators 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 to improve adherence in children.
Tools to prevent postpartum hemorrhage, the leading cause of maternal death, including new easier-to-administer, heat-stable formulations of the drug oxytocin for use in settings without reliable electricity and low-cost, simple uterine balloon devices to control bleeding after childbirth.
Simple, low-cost tools to detect preeclampsia, a dangerous pregnancy complication, including improved point-of-care diagnostics and handheld blood pressure monitoring devices.
New contraceptive options with low side effects and suitable for women who live far from healthcare settings, as well multipurpose products that combine contraception with prevention of HIV and other sexually transmitted infections.
New low-cost tools to address other leading MNCH challenges including obstructed labor, 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, refrigeration, and limited access to trained health workers, is under development with support from USAID. It could save the lives of almost 20,000 new mothers each year.
The Odon Device, a promising new tool that uses air pressure and suction to gently assist with delivery during obstructed labor, which was inspired by a trick to remove a cork from inside a wine bottle, is advancing through clinical trials. Developed with USAID support, this device could save an estimated 249,000 maternal and newborn lives by 2030.
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.
The Bilistick, a simple, low-cost, portable device to diagnose dangerous levels of jaundice in newborns, designed for use by health workers in remote areas without laboratory facilities, is now undergoing clinical trials with USAID funding. At less than $1 per test, it could help protect the lives of the estimated 60% of newborns worldwide affected by jaundice.
New contraceptive innovations are in development including a microneedle contraceptive patch lasting six months, a male contraceptive gel now in clinical trials supported by NIH, and a vaginal ring which combines contraception with antiretroviral treatments to prevent HIV infection, supported by USAID and NIH.
Affordable, rapid point-of-care urine tests to diagnose preeclampsia are now undergoing validation testing with USAID support. These tools, designed for low-resource settings, will improve 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 develops affordable vaccines, treatments, and other tools to improve MNCH globally and address family planning (FP) and reproductive health (RH) needs, leading implementation of the US government’s MNCH and FP/RH activities.
National Institutes of Health 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 research to inform use of existing MNCH tools, and provides technical assistance to country partners to strengthen public health capacity.
Department of Defense support 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

80% of all global maternal and child deaths occur in sub-Saharan Africa and Southern Asia: More women and children survive today than ever before – UN report [press release]. New York: World Health Organization (WHO); September 19, 2019. https://www.who.int/news-room/detail/19-09-2019-more-women-and-children-survive-today-than-ever-before-un-report.

Child deaths declined by nearly 60%: UN Inter-agency Group for Child Mortality Estimation. Levels & Trends in Child Mortality: Report 2019. New York: UNICEF; 2019. https://data.unicef.org/resources/levels-and-trends-in-child-mortality/.

Maternal deaths declined by nearly 45%:

WHO, UNICEF, UNFPA and The World Bank. Trends in Maternal Mortality: 2000 to 2017. Geneva: WHO; 2019. https://www.who.int/reproductivehealth/publications/monitoring/maternal-mortality-2015/en/.

WHO, UNICEF, UNFPA and The World Bank. Trends in Maternal Mortality: 1990 to 2015. Geneva: WHO; 2015. https://www.who.int/reproductivehealth/publications/monitoring/maternal-mortality-2013/en/.

5.3 million children under five die each year/290 thousand women die each year: Surviving birth: Every 11 seconds, a pregnant woman or newborn dies somewhere around the world [press release]. New York City, New York: UNICEF; September 19, 2019. https://www.unicef.org/press-releases/surviving-birth-every-11-seconds-pregnant-woman-or-newborn-dies-somewhere-around#targetText=In%202017%2C%20according%20to%20the,and%20childbirth%2C%20or%20over%20290%2C000.

214 million women have an unmet need for contraception: Guttmacher Institute. Adding It Up: Investing in Contraception and Maternal and Newborn Health, 2017. Washington, DC: Guttmacher Institute; 2017.https://www.guttmacher.org/fact-sheet/adding-it-up-contraception-mnh-2017.

Research successes

Vaccines against childhood diseases save two to three million lives each year: WHO website. Immunization coverage page. https://www.who.int/news-room/fact-sheets/detail/immunization-coverage. Accessed August 30, 2019.

Contraceptive innovations: WHO website. Family planning/contraception page. https://www.who.int/news-room/fact-sheets/detail/family-planning-contraception. Accessed October 22, 2019.

Contraceptive innovations, self-insertable, one-year vaginal ring: Population Council website. The Nestorone®/ethinyl estradiol one-year vaginal contraceptive system page. https://www.popcouncil.org/research/one-year-contraceptive-vaginal-ring. Accessed August 30, 2019.

Contraceptive innovations, discreet, self-injectable, long-acting contraception: The power to prevent pregnancy in women’s hands: DMPA-SC injectable contraception [blog post]. PATH. November 12, 2018. https://www.path.org/articles/dmpa-sc/.

Contraceptive innovations, expanding access to contraception could reduce maternal deaths by nearly one-third and child deaths by one-quarter: US Agency for International Development (USAID) website. Family planning and reproductive health page. https://www.usaid.gov/global-health/health-areas/family-planning#dataSources. Accessed August 30, 2019.

Tools to combat childhood malaria, insecticide-treated bednets: Centers for Disease Control and Prevention (CDC) website. Insecticide-treated bed nets page. https://www.cdc.gov/malaria/malaria_worldwide/reduction/itn.html. Accessed August 30, 2019.

Tools to combat childhood malaria, child-friendly malaria medicines: Medicines for Malaria Venture website. 385 million treatments of Coartem® Dispersible delivered to over 50 countries page. https://www.mmv.org/our-impact/achievements/385-million-treatments-coartem-dispersible-delivered-over-50-countries. Accessed August 30, 2019.

Interventions to treat and prevent diarrhea have reduced child mortality from diarrheal disease by more than one-third since 2005: GBD Diarrhoeal Diseases Collaborators. Estimates of global, regional, and national morbidity, mortality, and aetiologies of diarrhoeal diseases: a systematic analysis for the Global Burden of Disease Study 2015. Lancet Infectious Diseases. 2017; 17: 909–948. https://doi.org/10.1016/S1473-3099(17)30276-1.

Nutrition innovations: Global Health Technologies Coalition website. Why research & development page. https://www.ghtcoalition.org/why-research-and-development. Accessed August 30, 2019.

Nutrition innovations, biofortified crops: HarvestPlus website. https://www.harvestplus.org/. Accessed August 30, 2019.

Approaches to prevent maternal-to-child transmission of HIV have contributed to a 41% decline in new infections in children since 2010: UNAIDS website. Global HIV & AIDS statistics — 2019 fact sheet page. https://www.unaids.org/en/resources/fact-sheet. Accessed August 26, 2019.

Key missing tools

PATH. Reimagining global health: 30 high-impact innovations to save lives. Seattle: PATH; 2015. http://ic2030.org/wp-content/uploads/2015/07/ic2030-report-2015.pdf.

Continued progress is possible, not inevitable

Bill & Melinda Gates Foundation and Institute for Health Metrics and Evaluation. The Goalkeepers Report 2019: How Geography and Gender Stack the Deck For (or Against) You. Seattle: Bill & Melinda Gates Foundation; 2019. https://www.gatesfoundation.org/goalkeepers/report/2019-report/#ExaminingInequality

Breakthroughs on the brink

Oxytocin inhaler: El-Noush H, Silver K, Pamba A, Singer P. Innovating for women’s, children’s, and adolescents’ health. BMJ. 2015; 351:h4151. https://doi.org/10.1136/bmj.h4151.

Odon device:

PATH. Reimagining global health: 30 high-impact innovations to save lives. Seattle: PATH; 2015. http://ic2030.org/wp-content/uploads/2015/07/ic2030-report-2015.pdf.

El-Noush H, Silver K, Pamba A, Singer P. Innovating for women’s, children’s, and adolescents’ health. BMJ. 2015; 351:h4151. https://doi.org/10.1136/bmj.h4151.

Vaccines against shigella and Enterotoxigenic Escherichia coli: When it comes to vaccines, bigger isn’t always better [blog post]. Seattle: PATH; June 29, 2019. https://www.path.org/articles/when-it-comes-to-vaccines-bigger-isnt-always-better/.

Bilistick: Bilimetrix website. Bilistick® page. https://www.bilimetrix.net/bilistick-system/. Accessed August 30, 2019.

USAID. Saving Lives at Birth: A Grand Challenge for Development: Innovation Catalogue. Washington, DC: USAID; 2017. https://issuu.com/savinglivesatbirth/docs/sl_b_v11_pdf_version.

Bilistick, 60% of newborns worldwide affected by jaundice: CDC website. What are jaundice & kernicterus? page. https://www.cdc.gov/ncbddd/jaundice/facts.html. Accessed October 22, 2019.

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

New contraceptive innovations, male contraceptive gel: First trial launches to test effectiveness of male contraceptive gel [press release]. New York: Population Council; November 28, 2018. https://www.popcouncil.org/news/first-trial-launches-to-test-effectiveness-of-male-contraceptive-gel.

New contraceptive innovations, vaginal ring which combines contraception with antiretroviral treatment: IPM advances three-month HIV prevention and contraception ring to clinical trial [press release]. Silver Spring, Maryland: International Partnership for Microbicides (IPM); May 3, 2017. https://www.ipmglobal.org/content/ipm-advances-three-month-hiv-prevention-and-contraception-ring-clinical-trial.

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

US government R&D efforts

Kaiser Family Foundation (KFF). The U.S. Government and International Family Planning & Reproductive Health Efforts. Washington, DC: KFF; 2019. https://www.kff.org/global-health-policy/fact-sheet/the-u-s-government-and-international-family-planning-reproductive-health-efforts/.

KFF. The U.S. Government and Global Maternal & Child Health Efforts. Washington, DC: KFF; 2019. https://www.kff.org/global-health-policy/fact-sheet/the-u-s-government-and-global-maternal-and-child-health/.

KFF. The U.S. Department of Defense & Global Health. Washington, DC: KFF; 2012. https://www.kff.org/hivaids/fact-sheet/u-s-federal-funding-for-hivaids-trends-over-time/. https://www.kff.org/global-health-policy/report/the-u-s-department-of-defense-global/.

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

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