Will NIH Funding Cuts Fuel Drug Repurposing Strategies?

The pharmaceutical industry is facing a critical juncture. For years, the staggering 90% failure rate of drugs in clinical trials has cost pharmaceutical companies an estimated $45 billion annually. A significant portion of this waste is attributed to inefficiencies in the drug development process, an issue highlighted by the Trump Administration’s Department of Government Efficiency (DOGE). In an effort to reduce inefficiencies and government spending, DOGE has cut funding to the National Institutes of Health (NIH). Now, with fewer dollars flowing into research, the pharmaceutical industry must reevaluate and adapt its approach to drug development. 

The journey from discovery to market for any new drug is long, costly, and fraught with uncertainty. With such high risks that any particular clinical trial will fail, it’s no surprise that the financial burden on pharma companies is immense. This failure rate is driven by several factors, including ineffective patient recruitment, suboptimal patient selection, and statistical under-powering. A fundamental question now facing the industry is:  How can we reduce these risks without compromising patient safety or wasting critical resources, particularly if reduced NIH funding results in greater expenditures or risks on the part of trial sponsors?

One answer may lie in a largely underexplored area - drug repurposing. 

Drug repurposing involves finding new therapeutic uses for drugs that have already been developed for a different disease, may have already received FDA approval, and may have even reached loss of exclusivity (LOE; the point at which biosimilar or generic versions of the drug can also be marketed). It allows companies to bypass many early-stage hurdles in the drug development pipeline and get life-saving treatments to patients faster. This model holds promise for both accelerating the drug development process and reducing the financial burden of development.

A compelling case for the effectiveness of drug repurposing was the response to the COVID-19 pandemic. This rapid response not only saved time but also allowed for faster deployment of therapies to the public in a time of need. When faced with adversity, the medical community has demonstrated it is well-equipped to adapt. As the industry continues to face global health crises, whether another pandemic or reduced funding, drug repurposing will play a pivotal role in addressing urgent medical needs with greater speed and precision. More recently, a number of academic researchers have dedicated their efforts toward using AI in efforts to repurpose drugs, either alone or in combination with others.

The cost of developing a new drug typically ranges from $1-2 billion. This, combined with the recent cuts to NIH funding, represents a paradigm shift in how the healthcare industry must approach drug development. Historically, the NIH has been seen as one of the pillars of medical research funding,  with 80% of NIH funding previously going to drug development. Now, that pillar is facing its own financial headwinds.

As the future of NIH funding remains uncertain, AI-driven drug development platforms, such as our cross-functional clinical development platform, are important as ever. These advanced technologies allow researchers to simulate clinical trials and predict drug effectiveness, including that of repurposed drugs, before they are tested on patients. By leveraging vast datasets encompassing patient responses, genetic information, and historical drug performance, AI can forecast a drug's performance in a new patient cohort or for a new indication. This capability significantly reduces the costly trial-and-error phase of drug development, making drug repurposing a more viable and efficient strategy for pharma companies seeking innovation without excessive financial risk.

As we look toward the future, drug repurposing, supported by AI innovation, will likely become a cornerstone of drug development strategies. Cuts to NIH funding may ultimately be a catalyst that forces the industry to rethink its approach and embrace smarter, data-driven solutions. We believe that now is the time for pharma companies to invest in AI, and to embrace the promise and potential of drug repurposing, to ensure that we are not only meeting the challenges of today but also paving the way for a healthier, more sustainable future.

The views expressed in this op-ed are those of the author and do not necessarily reflect the opinions or positions of BioBuzz.