Source: National Institute of Health (NIH)
With reporting by Jon Cohen and David Malakoff.
More than 100 treatments and vaccines are in development to stem the COVID-19 pandemic, and some onlookers have worried that this sprawling and potentially duplicative effort is wasting time and resources. Hoping to bring order to the chaos, the National Institutes of Health (NIH) and major drug companies today announced a plan to stage carefully designed clinical trials of the drugs and vaccines they have decided are the highest priorities for testing and development.
The public-private partnership involves NIH, other U.S. government agencies, 16 pharma companies and biotechs, and the nonprofit Foundation for the National Institutes of Health (FNIH). It aims to develop “an international strategy” for COVID-19 research, a press release states. However, NIH Director Francis Collins told reporters during a press call today that “it is primarily a U.S.-focused effort.”
The initiative, called Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV), aims to make efficient use of NIH funding and its clinical trial networks by working with companies to evaluate data on early candidates, selecting those that have the most promise and are not already part of rigorous human tests.
NIH is stepping in partly because of concerns that some potential treatments, such as hydroxychloroquine/chloroquine, are being touted by some researchers as effective based on poorly designed trials. “We need the best research standards,” says former NIH Director and Sanofi executive Elias Zerhouni, who sits on FNIH’s board. “To me, the whole effort is to create a synergy between all of the players.”
Collins said NIH began discussions with companies just 1 month ago, and plans gelled at a 3 April meeting. There was “unanimous agreement that the time has come to put aside any of the obstacles … [to] such a public-private partnership and bring all the full resources and ideas together in a variety of ways that neither sector could do alone,” Collins said.
ACTIV is partly modeled on a long-established FNIH-organized program called the Accelerating Medicines Partnership, in which companies join forces with NIH to generate shared data, such as biomarkers in Alzheimer’s disease. Its goals include giving companies access to animal models and drug screening programs in high-security biosafety labs, as well as developing a “master protocol” and setting clinical standards for assessing a drug’s efficacy that would be shared across trials. Companies will also share data on immune responses to candidate vaccines. Although focused on the United States, NIH says ACTIV is also working with the European Medicines Agency.
NIH is assembling working groups of scientists who will inventory drugs and vaccines at different stages of development, and then decide which are most promising for future testing and not already in well-designed studies. In making that list, the experts will also consider which untested compounds can most quickly move into human trials, and which could be manufactured in large quantities. The top-rated compounds will get priority for the $1.8 billion Congress gave NIH for COVID-19 research as part of pandemic recovery bills, and preferred access to NIH’s dozen or more clinical trial networks.
Collins noted that once ACTIV has started to coordinate clinical trials, it might become easier to rapidly start, end, or redesign trials to focus on the most promising candidates. As an example, he noted that NIH easily recruited 800 patients, 300 more than needed, to test the drug remdesivir. (Results are expected soon.) If an additional candidate compound was available, he noted, NIH could have offered those 300 patients the option of becoming involved in a different trial instead.
NIH and its partners say they have put off, for now, deciding how to answer potentially thorny questions about intellectual property (IP) ownership and the pricing of any new treatments developed under the partnership. That’s a reasonable strategy given the urgency, says science policy specialist Robert Cook-Deegan of Arizona State University, Tempe. “It’s pretty clear that they’re aware the IP could get complicated,” he says. And he doubts that pricing issues “will play out the way it usually does, with companies pricing to maximize and government passively going along,” if only because there is likely to be intense public scrutiny of the effort.
Collins pointed out that companies are unlikely to be motivated by profits after seeing their own clinical trial efforts shut down by COVID-19. Executives are “desperate” to get their businesses running again and “therefore willing to do whatever it takes both altruistically and practically,” he predicted.
Growing Efforts to Coordinate
The NIH project joins numerous other COVID-19 research coordination efforts around the world. In the United Kingdom, for example, a trial testing several drugs called RECOVERY is being led by the University of Oxford and tapping nearly 1000 patients at 132 hospitals run by the country’s National Health Service. Although the U.S. effort has been slower than some others to get started, “No doubt when the NIH gets going, it will be a … very efficient study,” says Mene Pangalos, executive vice president of biopharmaceuticals R&D at AstraZeneca. “Hopefully, they won’t do the same molecules they are doing in the U.K., and between us all, we’ll learn faster.” NIH says its working groups include representatives from efforts such as RECOVERY to avoid such duplication.
One global health leader is noticeably absent from the list of ACTIV’s partners: the World Health Organization (WHO). It, too, has been coordinating COVID-19 research, organizing a global trial of several drugs called SOLIDARITY.
WHO Chief Scientist Soumya Swaminathan said her agency welcomes the NIH effort. But for vaccines, she contends that WHO is well-positioned to coordinate the field with an expert group that’s now being formed. “The best and most efficient way would be to consider one large global study which would look at different vaccine candidates,” she says.
Collins says NIH is keeping tabs on WHO’s efforts as it makes its own plan. And infectious disease expert Daniel Bausch of the London School of Hygiene & Tropical Medicine says WHO does not have the capacity to launch the effort NIH has in mind. “It will be important, however, to have NIH and WHO coordination, especially to ensure equitable participation in the conduct of the trials and access to the products that they might produce,” Bausch says.
Richard Danzig, former secretary of the U.S. Navy who is heavily involved in international vaccine planning, says that although “the NIH effort is commendable,” it doesn’t directly take on another challenge once a vaccine has been developed: how to make enough to vaccinate the world’s population. “We need also,” Danzig says, “to prepare global manufacturing for vaccine production.”
With reporting by Jon Cohen and David Malakoff.