U.S. effort to find treatment is described as chaos

In a desperate bid to find treatments for people sickened by the coronavirus, doctors and drug companies have launched more than 100 human experiments in the United States, investigating experimental drugs, a decades-old malaria medicine and cutting-edge therapies that have worked for other conditions such as HIV and rheumatoid arthritis.

Development of effective treatments for covid-19, the disease the virus causes, would be one of the most significant milestones in returning the United States to normalcy. But the massive effort is disorganized and scattershot, harming its prospects for success, according to multiple researchers and health experts. Researchers working around-the-clock describe a lack of a centralized national strategy, overlapping efforts, an array of small-scale trials that will not lead to definitive answers and no standards for how to prioritize efforts, what data to collect or how to share it to get to answers faster.

“It’s a cacophony — it’s not an orchestra. There’s no conductor,” said Derek Angus, chair of the department of critical care medicine at University of Pittsburgh School of Medicine, who is leading a covid-19 trial that will test multiple therapies. “My heart aches over the complete chaos in the response.”

The global biomedical research establishment could be one of most powerful assets in the campaign against the new virus, with experts all over the world — and especially the scientific and medical powerhouse of the United States — in rare alignment in their focus on a single enemy. Some large trials designed to be definitive have launched. But with more than 500 human clinical trials worldwide, the lack of coordination puts the world at risk of ending up with a raft of inconclusive and conflicting studies and little idea of what interventions work for the next wave of illness.

Francis Collins, director of the National Institutes of Health, the nation’s largest biomedical research agency, acknowledged researchers’ frustrations but said in an interview Wednesday he has been working behind the scenes to launch an unprecedented public-private partnership to address the problems. He said the framework involves top pharmaceutical companies such as Pfizer and Johnson & Johnson, domestic and international government agencies including the European Medicines Agency, and academic research centers.

Collins said the month-long discussions have been kept under wraps to ensure buy-in for an approach likely to require sacrifices of personal recognition, scientific credit and profit — a centralized decision, for example, not to proceed with tests of one company’s drug in order to move faster on a competitor’s.

“I think we have the necessary clout to steer this whole complicated ecosystem,” he said. “When you look at some of the things that are happening sporadically, we may be unlikely to learn what we need to from such disconnected, small trials. The whole point is to replace that with a coherent, evidence-based approach. … I want to know what works, and I want to have it answered by June or July.”

Agency officials said further details would be released in coming days.

Anthony S. Fauci, director of the National Institute of Allergy and Infectious Diseases (NIAID), said in an email Tuesday that the partnership led by Collins is the “functional equivalent of a National Strategy.”

While Collins was working on developing that strategy, hospitals, drug companies, government labs and individual doctors were flooding the system with proposals for drugs and other interventions to test against the virus — an outpouring that reveals how siloed and fragmented the research enterprise remains. For example, there are more than two dozen separate U.S. trials listed for the anti-malarial drug hydroxychloroquine, all with different designs. Some use the drug as a preventive, others as a treatment. Some use it alone, some with other drugs or vitamins, and some have no comparison group to tell if the drug was responsible for the outcome. That will make it more difficult to conclude whether, or in what circumstances, the drug may work.

Collins said a working group is addressing this problem by sifting through about 100 possible covid-19 treatments to decide which are the six to eight most promising drugs to move forward in large-scale trials. Those will be deployed in large clinical trial networks.

The new federal effort is motivated in part by what happened in China. Clifford Lane, deputy director for clinical research and special projects at NIAID, traveled to the origin of the outbreak in February as part of an international delegation to help the world learn from the Chinese experience. He was troubled by the lack of a strategic plan to prioritize and fast-track the most promising treatments, leading to a mosaic of inconclusive findings.

“We do have to have a bigger strategy than every university, every institute and — to be blunt — every country” working on their own research efforts, Lane said in an interview.

At the heart of the problem is the basic question of whether a drug really works. Typically, drugs and medical interventions are first tested in small clinical trials that establish safety before the most promising ones are funneled into bigger trials, in an iterative and years-long process. These trials, which typically randomly assign patients to receive either a drug or a placebo, prove that medicines, vaccines and medical procedures are effective and safe. But with the urgency of the coronavirus threat, timelines have been squeezed, doctors are doing uncontrolled experiments as they administer regular care, and the typical model for research is too slow.

David Boulware, an infectious-disease physician and scientist at the University of Minnesota, has gotten at least 50 emails from companies and researchers with treatments they want to test. The urgency to find something — anything — for patients who have nothing other than supportive care has led researchers to pull everything off the shelf: a mix of existing drugs that show promise, stem cell treatments and brand new compounds designed specifically against covid-19.

The energy is remarkable, but it needs to be channeled. Clinical trials, whether for an HIV drug or a brand new medicine, compete for many of the same patients. If there are too many trials at a hospital, none of them may enroll enough patients to get clear results. If there are too many similar small trials running in parallel, their results individually may be inconclusive and the data could have so many differences they may not be able to be pooled.

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