Enhancing Predictive Outcomes: How New Approach Methodologies Are Advancing Precision Medicine

New Approach Methodologies (NAMs) give researchers better tools to study disease, understand treatment response, and define practical paths to precision medicine.

Despite years of advances in biomedical research, developing new therapies is still a costly and uncertain process. Nearly 90% of drugs that show promise in animal studies ultimately fail in human clinical trials, a gap that drives up costs, slows progress, and delays potential treatments for patients. 

New Approach Methodologies (NAMs) are gaining momentum as a way to address this challenge. By relying on human‑relevant, data‑driven models, NAMs give researchers better tools to study disease, understand treatment response, and make more informed decisions earlier in development. These advantages support a more practical path to precision medicine. 

To explore how NAMs are being applied today, the Institute for Precision Medicine at UPMC and the University of Pittsburgh convened leaders from academia, government, and industry for an April 2026 webinar focused on how NAMs are reshaping discovery and care. 

Why NAMs Matter Now

NAMs, including organoids, micro physiological systems (organs‑on‑chips), patient‑derived stem cell models, and AI‑driven computational approaches, provide a more accurate window into human biology than traditional models alone. Rather than relying on averages, these approaches make it possible to test therapies across diverse patient‑derived systems, revealing variability in response earlier in development.

As moderator, Kambez Benam, DPhil, associate professor of medicine and bioengineering at the University of Pittsburgh, emphasized: “NAMs shift the focus from generalized biology to human‑centric biology. The opportunity is not just to build more sophisticated models, but to design tools that meaningfully improve decision‑making across research and clinical translation.”

This focus on human relevance aligns closely with UPMC Enterprises’ mission to bridge innovation and real‑world impact by accelerating technologies that improve outcomes and reduce friction across the health care system. UPMC Enterprises collaborates with the Institute for Precision Medicine on advancing life sciences innovation.

Building Confidence Through Collaboration

From a policy and research infrastructure perspective, Christine Happel, PhD, program officer at the National Institutes of Health’s National Center for Advancing Translational Sciences (NCATS), highlighted the importance of validation, standardization, and coordinated investment.

“A key priority right now is building confidence — confidence in the data, confidence in the methods, and confidence across the ecosystem that these models can support meaningful clinical and regulatory decisions,” she said.

NIH has increasingly taken on a convening role, aligning academic researchers, industry leaders, and regulators to create shared frameworks for evaluating NAMs. These efforts aim to reduce fragmentation and accelerate adoption.

Precision Medicine Requires Human‑Centric Models

For Joseph Wu, MD, PhD, director of the Stanford Cardiovascular Institute, NAMs unlock a more realistic understanding of how treatments work across patients, not just in theory, but in practice.

“When you test therapies across patient‑derived models, the variability becomes immediately clear. That variability is not noise, it’s the signal that precision medicine is built on,” he said.

By combining genomics, patient‑specific stem cell models, organoids, and AI, researchers can identify which therapies are more likely to benefit subpopulations earlier in development. This approach holds promise for lowering attrition, improving trial design, and supporting more targeted care strategies.

From Innovation to Use: Purpose Matters

From an industry and translational lens, Lorna Ewart, PhD, Chief Scientific Officer at Emulate, emphasized that successful adoption of NAMs depends on clarity of purpose and real‑world applicability.

“Novelty alone isn’t enough. These tools must be designed for a specific question, validated for a defined use, and reproducible across settings if they’re going to make a difference,” she said.

Her perspective accentuated a recurring theme: the future of NAMs will be shaped not just by technological sophistication, but by execution, scalability, and trust.

The Role of AI and Advanced Analytics

Across the discussion, panelists agreed that AI and advanced analytics are no longer optional complements to NAMs, they are essential enablers. From interpreting complex biological data to automating workflows and improving predictive modeling, these technologies are accelerating insights while reducing burden.

For the Institute for Precision Medicine, this convergence reinforces the importance of integrated innovation strategies that bring together biology, data science, and clinical operations to drive sustainable transformation.

Looking Ahead

The evolution of NAMs reflects a practical shift in health care, away from broad assumptions and toward more precise, predictive, and patient‑specific decision‑making. While real challenges remain, including funding, standardization, and adoption at scale, momentum across the field continues to advance. 

What comes next for precision medicine won’t be defined by discovery alone, but by how effectively insights move from the lab into everyday care. As NAMs, AI, and collaborative innovation efforts mature, they offer a clearer path to developing therapies that work better for patients. 

Next Steps

• Watch the full webinar recording.  

• Download the research report, “Operationalizing Precision Medicine: How U.S. Health Systems are Implementing Genetic Testing.” 

• Explore the Institute for Precision Medicine at UPMC and University of Pittsburgh.