Organ Chips Offer $3 Billion Annual Boost to Drug Development by Improving Preclinical Success Rates

Boston, MA – The pharmaceutical industry is increasingly looking to advanced preclinical models, such as organ chips, to mitigate the high failure rates and significant costs associated with drug development. Aaron VanDevender, Chief Scientist and Principal at Founders Fund, recently highlighted the critical need for better preclinical tools, stating, "Early failures in organ transplantation, gene therapy, and CAR-T caused a backlash of skepticism and set those therapies back a decade or two. It's important to develop better preclinical models like organ chips to avoid these pitfalls in the future." This perspective underscores a growing industry consensus on the transformative potential of these innovative technologies.

Organ-on-a-chip technology, pioneered by companies like Emulate Inc., aims to replicate human physiology with greater precision than traditional animal models or cell cultures. Emulate's Human Emulation System, which includes Organ-Chips mimicking organs such as the liver, intestine, and brain, offers a more human-relevant testing environment. This system is designed to predict human responses to drugs, chemicals, and diseases more accurately, thereby reducing the reliance on less predictive animal testing.

A recent study published in Communications Medicine, co-authored by scientists from Emulate Inc. and with contributions acknowledged from VanDevender, demonstrated the significant predictive power of a human Liver-Chip. The study, which evaluated 27 drugs, showed the Liver-Chip achieved an 87% sensitivity and 100% specificity in predicting drug-induced liver injury when protein binding was corrected. This performance notably surpasses that of 3D hepatic spheroids, which showed only 47% sensitivity for the same drug set.

The economic implications of such improved predictability are substantial. The Communications Medicine study estimated that widespread adoption of the Liver-Chip for predicting drug-induced liver injury could generate approximately $3 billion annually for the pharmaceutical industry due to increased research and development productivity. If similar organ-chip models were developed for other major causes of drug attrition—cardiovascular, neurological, immunological, and gastrointestinal toxicities—the annual industry-wide gain could exceed $24 billion.

The call for improved preclinical models stems from historical setbacks in advanced therapies. Early gene therapy trials, for instance, faced severe challenges, including the tragic 1999 death of Jesse Gelsinger due to a fatal immune response to a viral vector, and instances of leukemia in children treated with early retroviral vectors. These events led to a significant slowdown in gene therapy research and heightened regulatory scrutiny. Similarly, CAR-T cell therapies, while now highly successful for certain cancers, also navigated a complex development path with initial safety concerns and manufacturing hurdles.

As VanDevender emphasized, "The dependence of drug development on animal models for safety and efficacy testing has hit a wall." He noted that limitations of legacy animal models increasingly prevent accurate predictions of drug responses in humans, leading to patient risks and hindering approval of novel life-saving therapies. The continued development and adoption of organ-on-a-chip technology represent a strategic shift towards more effective, ethical, and economically beneficial drug development pathways.