A key rapid-response tool for the next pandemic? Drug repurposing

Approved drugs can be tested quickly, but a coordinated repurposing system is needed

What if a life-saving treatment for the next pandemic is already on pharmacy shelves? But what if we never learn of its benefits because we again find ourselves unorganized when the pandemic hits, lacking a system for rapidly and effectively testing approved products?

One of the most effective treatments for COVID-19 that saved over 1 million lives globally was approved by FDA in 1958 for the treatment of rheumatoid arthritis. Dexamethasone was among one of the first drugs tested by the U.K.’s RECOVERY trial for treatment of COVID-19 and was shown to reduce mortality in hospitalized patients receiving respiratory support. Because it had been on the market for over 60 years, it was ideal for wide-spread use and access with its readily available generics that were cheap to produce and purchase.

This experience with dexamethasone is referred to as repurposing — research to identify new indications for already-approved drugs. Many approved drugs have more than one use or benefit but are rarely researched beyond the initial indication for which they are approved. Drug repurposing is often viewed as a faster, cheaper and less risky approach to drug development. The development of a new drug is reported to take an average of 8-10 years and cost as much as $2.8 billion. Since they have been approved by a regulatory body, repurposed compounds have already demonstrated safety in humans. Therefore, researchers can bypass earlier stages of drug discovery and clinical trials, and development efforts can focus on demonstrating efficacy for the new indication.  

On Day 1 of an emerging pandemic or health threat, drug repurposing represents a critical path to quickly identify potential treatments. This approach should be the first line of defense against the emerging disease, offering the best option to rapidly identify and develop therapies to treat or reduce the severity of the disease.

It is critical to develop quality evidence that can address questions about the safety and efficacy of these products.

The COVID-19 pandemic demonstrated that physicians and the public will try a wide range of unproven but FDA-approved products (e.g., ivermectin). However, in contrast to RECOVERY’s success with dexamethasone, most of the early work on repurposing of approved medicines during the COVID pandemic was uncoordinated and did not yield actionable data.

We must not let that happen again. Coordinating our efforts to establish an efficient and effective drug repurposing ecosystem is an essential component of preparing for the next pandemic.

Despite the advantages and need for drug repurposing studies, several challenges persist — inadequate resources, poor trial data access, lack of expertise, uncertainty about value, liability risks, and intellectual property challenges are among the obstacles that need to be addressed.

By reflecting on the lessons learned from the COVID-19 pandemic and consulting with experts in a recent roundtable meeting hosted by the Duke-Margolis Health Policy Center, we have identified three components of successful drug repurposing: clear governance to set priorities and guide investments; ready infrastructure and capacity to facilitate rapid research on repurposed compounds; and administrative structures and support systems to drive efficiency and close coordination across sectors. 

U.S. drug repurposing efforts were slowed because they lacked these necessary components. Most notably, there was a lack of central governance or recognition of the unique role that the federal government has in virtually every aspect of driving a repurposing effort — from setting priorities to allocating resources and establishing incentives for coordination among all stakeholders. 

Finally, despite the vast resources U.S. companies focused on developing vaccines and treatments, greater private sector coordination between the government and the private sector would have sped repurposing efforts.

Now that the public health emergency declaration for COVID-19 has officially come to an end, this is the time to reflect on the lessons learned and take steps to improve preparedness for future threats.

The reauthorization of Pandemics and All-Hazards Preparedness Act (PAHPA), which is currently under discussion among lawmakers, provides the opportunity to take the necessary actions to strengthen the country’s pandemic response capacity. Congress should consider effective and fiscally responsible approaches that leverage existing resources and funding and create clear governance. Establishing the necessary groundwork for a drug repurposing ecosystem that can rapidly identify and study promising approved and available drugs should be chief among those efforts. 

We estimate around $50 million — a mere 1.5% of the annual budget of the Administration for Strategic Preparedness and Response — would be needed to establish a drug repurposing program for pandemics, and complementary efforts to build warm clinical trial networks and manufacturing capacity could reduce the cost even further. Creating an efficient drug repurposing ecosystem can save precious time when looking for treatments to an emerging health threat. Taking action now to establish a centrally coordinated repurposing effort can better position the U.S., and the world, to respond to emerging health threats.

Tanisha Carino is a visiting fellow and Beth Boyers a policy research associate at the Duke-Margolis Center for Health Policy.

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