In the race to find effective treatments for COVID-19, scientists are setting up clinical trials that test multiple drugs simultaneously, hoping to quickly determine which ones work best.
These “platform” tests have already led to significant progress.
One, sponsored by the University of Oxford, tested several treatments, including the antimalarial hydroxychloroquine, HIV protease inhibitors, lopinavir and ritonavir, and the steroid dexamethasone. The conclusion? Hydroxychloroquine and protease inhibitors do not work, but dexamethasone significantly reduces mortality in hospitalized patients. Many people credit this steroid for President Trump fast recovery.
Another platform trial at the National Institutes of Health this spring found antiviral remdesivir could shorten hospital stays for patients with COVID-19 four days on average, which is a game-changer in the battle to prevent our healthcare system from being overwhelmed with coronavirus patients.
These platform trials are far superior to traditional trials – and it’s ludicrous that it took a global pandemic to spur their widespread adoption. Making platform trials the industry standard would help patients with conditions ranging from Alzheimer’s disease to cancer to rare diseases.
Traditional trials assess whether a single experimental treatment improves patient outcomes compared to a placebo or an existing standard of care. This approach is scientifically sound, but completely ineffective.
Indeed, if several investigational drugs are ready for human testing at around the same time, these treatments must be evaluated individually. Each traditional test requires its own sources of funding, its collaborating physicians and its patients. And since data collection techniques may vary depending on these trials, it is difficult to make direct comparisons which drug works best.
In 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 work well are dropped. And researchers can introduce potential new treatments at each phase.
This approach has a number of advantages. It’s faster, uses fewer participants, requires fewer patients to receive a placebo or standard of care as part of the control group, and allows for one-to-one comparisons.
Here in the United States, 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 at Houston Health Sciences Center is in its infancy of its own COVID-19 testing platform.
Platform trials would prove particularly useful in rare disease research. Since these diseases affect a small or very small number of patients, traditional trials requiring large groups of participants are not feasible. Rare diseases also tend to attract less funding. Platform testing can provide high-quality evidence without a large pool of participants—And at a lower cost than standard testing.
Recognizing these benefits, the Children’s Tumor Foundation, of which I am president, recently launched INTUITT-NF2. This innovative platform trial is capable of simultaneously evaluating treatments for multiple tumor manifestations in neurofibromatosis type 2 (NF2). NF2 is a rare genetic disease this is explained by tumor growth on the nerves and in the brain.
Our organization also plays 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 testing.
While this promising approach will hopefully become mainstream, the pharmaceutical industry will need to be encouraged to collaborate. Specifically, the legal departments of competing pharmaceutical companies will need to find acceptable ways to work together on the co-design and co-execution of drug trials without compromising a company’s intellectual property.
These challenges are worth overcoming. The lives of patients are at stake.
Platform trials are already playing a vital role in the fight against the COVID-19 pandemic. Making it the industry standard would bring life-saving treatments to patients faster, more efficiently, and more often.
Annette Bakker, Ph.D., is President of the Children’s Tumor Foundation.
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