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COVID-19 could change clinical trials for the better

In the race to find effective COVID-19 treatments, scientists are setting up clinical trials that test multiple drugs simultaneously, in the hopes of quickly determining which ones work best.

These “platform” trials have already led to important breakthroughs.

One, sponsored by the University of Oxford, tested multiple treatments, including the antimalarial hydroxychloroquine, HIV protease inhibitors lopinavir and ritonavir, and steroid dexamethasone. The conclusion? Hydroxychloroquine and the protease inhibitors don’t work—but dexamethasone significantly reduces mortality among hospitalized patients. Many people credit this steroid for President Trump’s speedy recovery.

Another platform trial at the National Institutes of Health this spring found antiviral remdesivir could shorten COVID-19 patients’ hospital stays by four days, on average—a game changer in the battle to keep our health care system from being overwhelmed with coronavirus patients.

These platform trials are far superior to traditional trials—and it’s ridiculous that it took a global pandemic to spur their widespread adoption. Making platform trials the industry standard would help patients battling conditions from Alzheimer’s to cancer to rare diseases.

Traditional trials evaluate whether a single experimental treatment improves patient outcomes compared to a placebo or existing standard of care. This approach is scientifically sound, but quite inefficient.

That’s because if multiple experimental drugs are ready for human testing at roughly the same time, those treatments must be evaluated individually. Each traditional trial requires its own funding sources, collaborating physicians, and patients. And since data collection techniques might vary across those trials, it’s difficult to make head-to-head comparisons about which drug works best.

By contrast, in platform trials, researchers test multiple interventions simultaneously. The most promising candidates move on to the next stage of the trial, while treatments that don’t perform well get dropped. And researchers can introduce new potential treatments at each phase.

This approach has a number of advantages. It’s quicker, uses fewer participants, requires fewer patients to receive either a placebo or the standard of care as part of the control group, and allows for head-to-head comparisons.

Here in the U.S., North Carolina–based Wake Forest Baptist Health recently became the country’s first hospital to enroll patients in a platform trial. And the University of Texas Health Science Center at Houston is in the early stages of its own COVID-19 platform trial.

Platform trials would prove especially helpful in rare disease research. Since these illnesses affect small to very small numbers of patients, traditional trials requiring large groups of participants aren’t feasible. Rare diseases also tend to attract less funding. Platform trials can provide high-quality evidence without a large participant pool—and at a lower cost than standard trials.

Recognizing these benefits, the Children’s Tumor Foundation, where I serve as president, recently launched INTUITT-NF2. This innovative platform trial is able to evaluate treatments for multiple tumor manifestations in neurofibromatosis type 2 (NF2) at the same time. NF2 is a rare genetic disorder that results in the growth of tumors on the nerves and in the brain.

Our organization is also playing a key role in the EU Patient-Centric Clinical Trial Platforms (EU-PEARL) project, a public-private partnership aimed at promoting the use of platform trials.

While this promising approach is hopefully becoming mainstream, the drug industry will need to be incentivized to collaborate. Specifically, the legal departments at competing pharmaceutical firms will need to find acceptable ways to work together in co-designing and co-executing drug trials without compromising any company’s intellectual property.

Such challenges are well worth overcoming. Patients’ lives are at stake.

Platform trials are already playing a vital role in beating back the COVID-19 pandemic. Making them the industry standard would bring lifesaving treatments to patients quicker, more efficiently, and more often.

Annette Bakker, Ph.D., is president of the Children’s Tumor Foundation.

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