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Critical Path Institute Unveils 'One to Millions'

On March 27, 2026, the Critical Path Institute (C-Path) unveiled a groundbreaking initiative known as 'One to Millions'. This global public-private partnership is designed to reshape the future of personalized medicine, scaling up the development of advanced therapies for highly individualized conditions while accelerating patient access worldwide.

Recent advancements in technologies such as antisense oligonucleotides, gene editing, gene therapies, and RNA-based treatments make it possible to devise targeted interventions for very small patient populations, even for individual patients. However, existing regulatory and reimbursement frameworks, which were established for population medicine, are not adequately equipped to keep pace with these innovations. This limitation has resulted in a growing gap that delays patients' access to new therapies.

To bridge this gap, One to Millions aims to promote the FDA’s framework for Rare Disease Evidence Standards while utilizing C-Path's centralized, regulatory-grade data platform. This resource will support evidence generation, regulatory decision-making, and scalable development pathways.

Klaus Romero, M.D., MS, FCP, the CEO of C-Path, expressed the significance of this initiative: "Words cannot express the magnitude of this moment for transforming lives and fulfilling an innovative vision. Designed to make personalized therapies scalable for a larger number of people, One to Millions is an alliance that only C-Path could convene. With a centralized and regulatory-ready data platform, a unique pre-competitive environment, and practical evidence frameworks, we are poised to optimize efficacy and safety evaluations. Simply put, there is no other initiative like this."

A modernized platform approach brings essential consistency and reliability to advanced therapeutic technologies. By standardizing manufacturing protocols and release testing, the initiative directly addresses inefficiencies and high costs often seen in low-volume drug development. As new therapies are built upon an established architecture, developers can leverage prior knowledge, allowing regulatory reviews to focus solely on new components rather than reassessing the entire framework.

Julia Vitarello, founder of Mila's Miracle Foundation and co-founder of the N=1 Collaborative, emphasized the pressing need for a coordinated approach to tackle current deficiencies: "We're living in an exciting time for genetics. We now possess the science to assist a large number of children with severe, life-changing rare diseases, but our access system wasn't designed to handle thousands of genetic conditions affecting small populations each. We are excited to collaborate with regulators to transition from approving one drug for one disease at a time to developing processes that work for multiple diseases. This shift could be revolutionary for millions of patients, but it will only be successful if we ensure ongoing, iterative learning based on these treatments through the systematic collection and exchange of data."

A fundamental feature of One to Millions is the integration of post-authorization evidence generation into the development paradigm. By incorporating longitudinal registries into the framework, the evidence generated for regulatory decision-making can inform payers' evaluations regarding durability, safety, and effectiveness, thereby preventing duplicated efforts and speeding up learning processes. Dr. Janet Woodcock, former director of the CDER and interim commissioner of the FDA, reinforced this point: "New technologies allow us to address the root causes of devastating monogenic diseases. However, progress can stagnate, and regulatory requirements remain excessively cautious when information for collective analysis and learning is lacking. We must not repeat past mistakes; we need to swiftly transfer knowledge and adopt agile development in this emerging field. Our patients deserve nothing less."

The collection of existing preclinical, translational, and clinical data will help optimize toxicology and dose selection. Gathering robust data enhances the utility of alternative methodologies, thus reducing the unnecessary reliance on animal testing while fostering a continuous learning and confirmation paradigm.

Timothy Yu, M.D., Ph.D., from Boston Children's Hospital, highlighted the initiative's potential: "This represents a new cornerstone for interventional genetics, filling a crucial gap for approval and reimbursement. By enhancing modularity, this framework allows developers to leverage data from various therapies targeting different genetic variants without having to restart the regulatory process for each mutation, paving the way for a plug-and-play genetics future. However, such a system cannot be built in isolation. To achieve cures that are greater than the sum of their parts, there needs to be shared learning through robust data exchange, where each advancement builds upon the last."

Additionally, Sarah Glass, Ph.D., Director of Operations at the n-Lorem Foundation, noted the foundation’s commitment to implementing scalable processes for personalized ASO drugs for patients with ultra-rare diseases, stating, "We have developed more than 25 ASOs that enabled treatment for over 45 patients. Many of these new ASOs can help more patients, and we are dedicated to reaching them. We are excited to join the One to Millions initiative and look forward to sharing our insights from our work within the community."

Collaborators involved in this initiative include the n-Lorem Foundation, Mila's Miracle Foundation, and the N=1 Collaborative, with founding members convening for further discussions.

For more information, visit C-Path’s website or contact them directly. Additionally, interested parties can attend C-Path's upcoming webinar titled, "Transforming Drug Development for Precision Medicines: A Practical Path from Evidence Generation," scheduled for April 8 at noon ET, with another session on patient-centered approaches to drug development.

About Critical Path Institute

The Critical Path Institute (C-Path) is a non-profit organization founded in 2005 as a public-private alliance in response to the FDA's Critical Path Initiative. C-Path's mission is to lead collaborations that accelerate better treatments for people worldwide. Recognized globally for its pioneering work in drug development acceleration, C-Path has established numerous consortia, programs, and international initiatives involving over 1600 scientists and representatives from regulatory agencies, academic institutions, patient organizations, disease foundations, and pharmaceutical companies. With a dedicated team worldwide, C-Path’s global hub is in Tucson, Arizona, and its European headquarters is in Amsterdam, Netherlands. For more information, visit c-path.org.

The Critical Path Institute receives funding from the U.S. Department of Health and Human Services' Food and Drug Administration (FDA) at a rate of 56%, totaling $23,740,424, with 44% from non-governmental sources, amounting to $18,881,611. The content of this communication is solely the authors' responsibility and does not necessarily represent the official views or endorsement by the FDA/HHS or the U.S. Government.