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Transforming the Future of Personalized Medicine: One to Millions

On March 27, 2026, the Critical Path Institute (C-Path) officially launched the One to Millions initiative. This cutting-edge global public-private partnership aims to revolutionize personalized medicine by making advanced therapeutic treatments accessible to patients worldwide. As rapid technological advancements provide unprecedented opportunities for individualized care, this initiative seeks to bridge the gap between innovation and the existing regulatory and reimbursement frameworks that often hinder patient access.

Addressing the Challenges of Individualized Treatment

One to Millions focuses on leveraging the latest technologies like antisense oligonucleotides, gene editing, gene therapies, and RNA-based treatments. These advances enable precise interventions tailored to extremely small patient populations, sometimes even individual cases. However, today's regulatory systems, originally designed for mass-market medications, struggle to keep pace with these rapid developments. As a result, many patients are left without timely access to potentially life-saving treatments.

To combat these limitations, One to Millions aims to enhance the U.S. Food and Drug Administration (FDA)'s regulatory framework and evidence standards concerning rare diseases. C-Path's centralized data platform will bolster evidence generation, regulatory decision-making, and adaptable development pathways, thereby accelerating the introduction of therapeutic innovations.

A Commitment to Transform Lives

Klaus Romero, M.D., the CEO of C-Path, emphasized the urgency of this initiative: "This moment is pivotal for transforming lives and realizing an innovative vision. Designed to make individualized therapies accessible to broader populations, One to Millions can be realized only through the unique partnership that C-Path has established. Our centralized data platform is ready for regulatory compliance and built upon collective preclinical, translational, clinical, and patient-level outcomes, setting the foundation for a continuous learning process in medicine. There's simply no other initiative quite like this."

Creating a Unified Approach

The initiative advocates for a modern platform approach that ensures consistency and reliability in the advancement of therapeutic technologies. By standardizing manufacturing and testing protocols, One to Millions directly addresses inefficiencies and high costs typically associated with limited drug development. This innovative design allows developers to leverage existing knowledge while enabling regulatory bodies to concentrate on new components rather than re-examining established processes from scratch.

Julia Vitarello, the founder of Mila's Miracle Foundation, expressed the urgent need for coordinated approaches to tackle existing gaps in the healthcare landscape. "We are living in an exciting time for genetics. Today, we have the scientific knowledge needed to help countless children suffering from rare, severe, and disabling diseases. Yet, the access system has not been designed for thousands of genetic disorders affecting small populations. We are thrilled to collaborate with regulatory bodies to shift from approving drugs for individual diseases to establishing processes that can accommodate multiple conditions. This evolution is crucial for millions of patients, but it must align with a continuous learning framework that systematically collects and shares data to facilitate safer and more effective drug development."

Utilizing Post-Market Evidence

A notable feature of the One to Millions initiative is its integration of post-market evidence into the therapeutic development paradigm. By directly incorporating longitudinal data, the initiative ensures that evidence generated for regulatory decision-making remains valuable for payers assessing sustainability, safety, and efficacy. This comprehensive ecosystem collaboration is designed to prevent unnecessary duplication of efforts, expediting collective learning—an essential point underscored by Janet Woodcock, M.D., who has a long history with the FDA.

"New technologies present the opportunity to address the root causes of devastating monogenic diseases. However, progress can stall when regulatory requirements become overly cautious due to a lack of shared information for collective analysis and learning," Dr. Woodcock stated. "We must not repeat past mistakes; we need to advance knowledge rapidly and support agile development in this new field through shared insights. Our patients deserve nothing less."

Future Directions

One to Millions will compile existing preclinical, translational, and clinical data sources to optimize toxicity assessments and dosage choices. A robust data collection strategy enhances the utility of alternative methodologies, reducing unnecessary reliance on animal experimentation while fostering an ongoing learning and confirmation paradigm.

As Timothy Yu, M.D., Ph.D. from Boston Children’s Hospital detailed, “This initiative provides a long-overdue component for approval and reimbursement in interventional genetics, starting with the FDA's 2021 guidelines on individualized antisense therapies. By doubling modularity efforts, developers can utilize data from therapies targeting different genetic variants without needing to restart regulatory procedures for every mutation. A future of 'ready-to-use' genetics is within reach, but it won't materialize in isolation. Each breakthrough must contribute to the next for the treatments to exceed the sum of their parts.”

Starting a Collaborative Endeavor

The collaboration involves several organizations, including n-Lorem Foundation and Mila's Miracle Foundation, with founding members convening to advance this effort. For more information, please visit c-path.org/programs/one-to-millions or contact [email protected]

Join the upcoming C-Path webinar titled "Transforming Drug Development for Precision Medicines" to learn more and register for the next session on April 8, 2026, focusing on scalable approaches to patient-centered benefit-risk in precision medicine.

About Critical Path Institute

Founded in 2005 as a non-profit public-private partnership in response to the FDA’s Critical Path initiative, the Critical Path Institute (C-Path) is dedicated to improving treatments for populations globally. Renowned for its pioneering efforts in accelerating drug development, C-Path has launched numerous consortia, programs, and international initiatives involving over 1,600 scientists and representatives from regulatory agencies, academic institutions, patient advocacy groups, disease foundations, and pharmaceutical and biotechnology companies. C-Path’s global headquarters is in Tucson, Arizona, with a European branch located in Amsterdam, Netherlands. For more information, visit c-path.org.

C-Path is supported by the FDA and funded 56% by the FDA/HHS, totaling $23,740,424, and 44% by non-governmental sources, totaling $18,881,611. The views expressed in this content do not necessarily reflect the official positions of the FDA/HHS or the United States Government.