Research Roundup: Research initiative to boost African vaccine access, Pfizer/BioNTech sign vaccine manufacturing agreement, and FDA approves all-oral sleeping sickness treatment

The Biden administration has proposed a new, cutting-edge research entity that has the potential to benefit global health the way the Defense Advanced Research Projects Agency (DARPA)—the research agency behind the internet, GPS, and self-driving cars—benefited the rest of society. This proposal, the Advanced Research Projects Agency for Health (ARPA-H), could lead to transformative health innovations for neglected diseases and address gaps caused by market failures, ultimately bolstering global health research and development (R&D). What is ARPA-H? Initial proposals from the Biden administration and allies in Congress have described ARPA-H as a driver of scientific breakthroughs for innovations to prevent, detect, and treat diseases that will be housed within the National Institutes of Health (NIH). The idea for ARPA-H was originally conceived by the Suzanne Wright Foundation, a pancreatic cancer advocacy organization, which outlined a Health Advanced Research Projects Agency that would focus on overcoming market failures by de-risking investments from the private sector. Now that the proposal has been taken into the mainstream by the Biden administration and biomedical R&D champions like Representatives Diana DeGette (D-CO) and Fred Upton (R-MI), advocates hope this mandate of addressing market failures might also encompass global health issues including HIV/AIDS, malaria, tuberculosis, and neglected tropical diseases. The scope of research to be conducted under ARPA-H has not yet been set in stone, even though the president’s fiscal year 2022 (FY22) budget request lists as initial focus areas cancer, diabetes, and Alzheimer’s in its $6.5 billion request for the agency. NIH has organized listening sessions, including one for allergies, infectious diseases, and global health, to further discuss the potential research areas for ARPA-H.  To promote high-risk, high-reward research, ARPA-H will likely be modeled after DARPA, which has a flat and nimble organizational structure and a large budget with limited oversight. In contrast to NIH’s sometimes slow-moving peer review process and multiyear grants, DARPA utilizes program managers who make unilateral funding decisions based on research milestones achieved. If the agency is stood up, GHTC supports an ARPA-H funding framework that is similar to DARPA, as it could enable easy, flexible collaborations with product development partnerships—nonprofit partnerships that address public health goals.The Biden administration’s proposal to house ARPA-H within NIH has raised concerns among some advocates and skeptics that this could limit its flexibility and independence, two critical aspects of the ambitious high-risk, high-reward ARPA-H agenda. An additional concern is that placement within NIH could eventually limit growth of top-line funding available for other NIH institutes and centers, and the lack of clarity regarding the scope of focus areas for research has created hesitancy among congressional appropriators to fund the full $6.5 billion proposed. However, if ARPA-H is created with the flexibility and independence of DARPA, and its funding is additive so as not to diminish work on any other NIH program, the agency holds great promise for global health innovations. Global health potential With the right influence from advocates, ARPA-H has the potential to advance research that could benefit the lives of people living in impoverished and underprivileged communities worldwide. Poverty-related and neglected diseases (PRNDs) affecting these communities remain inadequately addressed by the market, as they offer little commercial incentive for the private sector to invest in developing medical products to diagnose, treat, or prevent them. The lessons of the COVID-19 pandemic highlight the need to address gaps in existing PRNDs as their impacts are exacerbated during health emergencies. With a focus on PRNDs, ARPA-H could make a substantial impact toward developing innovations to address the world’s most overlooked diseases.GHTC is not the only stakeholder that recognizes the global health potential for ARPA-H. During a Senate budget hearing for FY22 on NIH, Dr. Francis Collins, NIH’s director, said that one focus for ARPA-H could be the development of a universal vaccine that protects against the ten most common infectious diseases in a single shot. The opportunity for further influencing the scope and mission of ARPA-H remains open. Just recently, Representatives DeGette and Upton, lead sponsors of the landmark 21st Century Cures Act of 2016 who are now developing a follow-up bill, issued a request for information on the possible authorization of ARPA-H in their bill, with questions including, “To ensure it has the biggest impact, on what activities or areas should ARPA-H focus?” In response, GHTC submitted feedback advocating for a focus on PRNDs, including HIV/AIDS, tuberculosis, malaria, and neglected tropical diseases, as well as antimicrobial resistance, arguing that a focus on these areas of historic market failures holds the greatest potential for ARPA-H’s societal impact. ARPA-H could produce transformative health innovations in chronically underfunded areas. If the agency is authorized, GHTC and other advocates should encourage lawmakers to ensure R&D for PRNDs is prioritized. The innovations that result could yield additional advancements across the entire field of biomedical R&D, improving health for all.

Interested in more global health innovation news? Every week GHTC scours media reports worldwide to deliver essential global health R&D news and content to your inbox. Sign up now to receive our weekly R&D News Roundup email. An adapted version of an existing treatment regimen to combat highly drug-resistant tuberculosis (TB) maintains efficacy against the disease and reduces side effects, according to results from the ZeNix trial announced last week. The ZeNix study, led by TB Alliance, is a follow-up to the Nix-TB trial, which published results last year showing 89 percent of participants were cured of TB when treated for six months with a combination of bedaquiline, pretomanid, and linezolid—known as the BPaL regimen. ZeNix evaluated the same regimen among 181 people with cases of highly drug-resistant TB in South Africa, Russia, Georgia, and Moldova, but varied the doses and durations of the use of linezolid. Results showed significantly reducing doses and duration of linezolid can have the same levels of efficacy in addition to reducing the side effects significantly.A new partnership will aim to help endemic countries eliminate Plasmodium vivax malaria, a complex and persistent type of malaria that poses a risk to more than one-third of the world’s population. The Partnership For Vivax Elimination (PAVE), led by the Medicines for Malaria Venture and PATH, will conduct feasibility studies to evaluate the best treatment options for P. vivax relapse in diverse populations and settings. The partnership will scale up the single-dose treatment tafenoquine as well as a higher dose of a second drug, primaquine, that cuts treatment time to seven days compared to the 14-day regimen currently in use. PAVE will also generate and consolidate evidence to support malaria-endemic countries in developing and implementing new strategies for P. vivax malaria.The COVAX vaccine-sharing facility is launching a marketplace to address supply chain bottlenecks and accelerate the global production of COVID-19 vaccine doses. The platform will match vaccine manufacturers with suppliers of key inputs to boost production of vaccines for equitable distribution through COVAX. The marketplace will initially focus on six critical areas where there have been shortages, including bioreactor bags, lipids, and vials, but could eventually facilitate supplies for other lifesaving medicines and vaccines hit by global shortages, according to the Coalition for Epidemic Preparedness Innovations, which will facilitate the initiative. COVAX aims to make 2 billion vaccine doses available by the end of the year, but so far has only delivered 118 million vaccines to 135 countries.