Old Medicines, New Hope: The Revolution in Repurposed Drugs (2025)

Most new drugs released in the U.S. are the product of 15-year development projects that cost billions and frequently result in failure. An easier, faster way is hiding in plain sight. Thousands of generic medications sitting in our medical cabinets are a potential goldmine of new applications that could be developed in years, not decades. The Trump administration can revolutionize healthcare by establishing a systematic framework to study and develop repurposed generic drugs.

Diverse cancer hallmarks targeted by repurposed non-oncology drugs. This figure was created with Biorender.com. Source: Nature 2024

For millennia medicine has been practiced by observation. Physicians tried different approaches to treatment, gauged a patient’s response, and shared their findings. Over the past century, that practice gave way to a mechanical approximation of this process, whereby patented medicines are tested through large-scale randomized controlled trials. This approach has yielded incredible breakthroughs, allowing millions to live longer, healthier lives. But these trials are incredibly expensive and carry a great risk of failure. Today the biopharmaceutical industry spends roughly $200 billion per year on R&D for cancer drugs, roughly 70% of which—some $60 billion—results in failed projects.

More importantly, our regulatory system relies too heavily on these trials to affirm new drugs, leaving little room for the time-tested practice of observation in medicine. This came to a head during the COVID pandemic, when physicians who treated patients with generic repurposed medicines to observe potential benefit lost their jobs for bucking institutional protocols. These men and women spent their lives in service to others, meticulously practicing medicine for decades, trying new treatment approaches to gauge patient response, and healing the sick. Many lives were destroyed merely for want of practicing medicine as it had always been done, in search of a way to alleviate suffering.

It’s time to change course. We need to rethink how we as a nation fund the development of new medicines. Yes, we need research-based pharma companies investing in the clinical trial process to make new discoveries. But clinical observation is essential and must return to prominence.

Consider how hydroxychloroquine came to be the standard treatment for lupus. Originally developed as an anti-malarial, people with lupus taking the medicine noticed that it actually dissipated their symptoms. They reported it to their doctors, and as a result the treatment was incorporated into practice. To this day hydroxychloroquine is the first-line drug for every lupus patient, and the only one to improve their life expectancy. Yet we still do not understand how exactly it works—only that it works.

Lyme disease is another instructive case. It was first recognized by mothers from Lyme, Connecticut and whose sons had been diagnosed with juvenile rheumatoid arthritis. These mothers, on their own in the early 1970s, determined that the rate of disease in Lyme was far above the national rate. They drove down to Yale with a hypothesis and eventually met Dr. Allan Steere, and within a few months he had defined a working definition of Lyme disease.

Our federal government can be a partner in catalyzing insights like these by funding and systematizing the collection and reporting of observational data across the country. As our understanding of medicine evolves, and AI-powered technology is creating new opportunities for sophisticated drug testing and discovery. We have tools to accelerate drug repurposing as never before, provided we can ensure the integrity of data submitted through hospitals and outpatient offices. Machine learning methods can rapidly screen databases for drug-disease connections that would traditionally take years to uncover.

The government can provide incentives under a new regulatory framework to ensure pharmaceutical companies are remunerated for developing new uses for generic drugs to treat major diseases. Helping agencies design, run and analyze trials could be rewarded with tax breaks, priority FDA review for a company’s other novel drug candidates, and guaranteed bulk purchase agreements. Prizes or mega-funds could provide payouts for successful trials that discover a new use, with the amount reflecting the anticipated public health impact of the innovation.

There are several means by which FDA could give a generic drug new market exclusivity, generating additional revenue for companies. The MODERN Labeling Act of 2020 provides authority to update labeling for certain generic drugs if doing so would benefit the public health there is a “relevant accepted use in clinical practice” for the new use. FDA also allows for a new 3-year exclusivity if a trial sponsor reports new clinical investigations related to the drug’s approval. When a label is updated under these circumstances, it changes how the drug can be marketed, prescribed, administered, and ultimately enforced by FDA. Prescribing physicians, insurance companies and pharmacies would all be bound to the new label and indication.

Systematic study of repurposed drugs is a promising path to finding novel therapeutics. We must swing the pendulum back toward observational medicine, powered by modern tools, to find what actually works in patients.

About the Author: Stephen M. Smith, M.D. is president of The Smith Center for Infectious Diseases and Urban Health. 

Source: https://www.realclearhealth.com/articles/2025/09/18/repurposed_drugs_is_a_promising_path_1135701.html

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