Critical Path Institute® (C-Path) today announced the launch of One to Millions, a multi-stakeholder public-private global initiative to enable the scalable development of advanced therapies for highly personalized conditions. Rapid advances in technologies such as antisense oligonucleotides, genome editing, gene therapies, and RNA-based therapies make it possible to design precisely targeted interventions for very small patient populations, even individual patients. However, existing regulatory and reimbursement frameworks, built for population-based medicines and linear growth models, are ill-equipped to keep up, creating a growing gap that delays patient access.
To help fill this gap, One to Millions is advancing the US Food and Drug Administration’s (FDA) Mechanism Framework and Rare Disease Evidence Principles by leveraging C-Path’s centralized regulatory-grade data platform to support evidence generation, regulatory decision-making, and scalable development pathways.
Words cannot fully express how pivotal this moment is in transforming lives and realizing a long-awaited innovative vision. Designed to make personalized treatments scalable for even more people, One to Millions is a partnership that only C-Path could convene. It has a central data platform, ready for regulatory settings. a unique ecosystem-wide pre-competitive environment; integrated preclinical, translational, clinical and patient-level outcomes. active evidence frameworks to optimize efficacy and safety assessment; and the ability to create the regulatory-grade tools needed to create a continuous process of learning and confirmation. There is simply no other initiative like it.”
Klaus Romero, MD, MS, FCP, Chief Executive Officer, Critical Path Institute
A modernized platform approach brings essential consistency and reliability to advanced therapeutic technologies. Standardization of manufacturing and release testing protocols directly addresses the disproportionate costs that typically plague low-volume drug development. By building new treatments on top of an established architecture, developers can leverage prior knowledge, allowing regulatory reviews to focus strictly on new elements rather than evaluating the entire base from scratch.
Julia Vitarello, founder of Mila’s Miracle Foundation and co-founder of the N=1 Collaborative, emphasized the urgent need for a coordinated approach to address the shortcomings of today’s landscape. “This is a very exciting time in genetics. Today, we have the science to help a huge number of children with serious, rare, life-changing diseases, but our access system was not designed for thousands of genetic diseases, each affecting small populations,” said Vitarello. “We are excited to work with regulators to move from approving one drug for one disease at a time to creating processes that can work across multiple diseases. This change could be a game-changer for millions of patients, but it will only succeed if we ensure continuous, iterative learning from these treatments, systematically collecting and sharing data to develop safer, more effective drugs.”
Integrating post-approval evidence generation into the development model is a defining feature of the initiative. Integrating longitudinal registries directly into the framework ensures that evidence generated for regulatory decision making can simultaneously inform payer assessments of durability, safety, and efficacy. Integrating information from across the ecosystem will prevent duplication of effort and accelerate learning, a point emphasized by Janet Woodcock, MD, longtime Director of CDER and former Deputy Commissioner, FDA. “New technologies enable potential correction of the root causes of devastating single-gene diseases. But progress can stall and regulatory requirements remain overly conservative when information is not available for collective analysis and learning,” Woodcock said. “We must not repeat the mistakes of the past; we must strive for rapid knowledge turnarounds and agile development in this new field, sharing what we have learned. Our patients deserve no less.”
Collection of existing preclinical, translational, and clinical data sources will help optimize toxicology and dose selection. Collecting robust data maximizes the utility of alternative methodologies, which help reduce unnecessary reliance on animal testing while building a continuous paradigm of learning and confirmation.
“This represents a critical new setting for interventional genetics, providing a long-missing piece for approval and reimbursement and completing the arc that began with the FDA’s 2021 guidance for personalized antisense therapies,” said Timothy Yu, MD, Ph.D., Division of Genetics and Genomics of the Co-=founds. Cooperative. “Effectively doubling modularity, this framework allows developers to leverage data into therapies targeting different genetic variants without restarting the regulatory process for each mutation. It points to a plug-and-play future of genetics, but such a system cannot be built in isolation. The discovery informs the next.”
“At n-Lorem, we have built a robust and scalable process to deliver personalized ASO drugs to nano-rare patients. We have discovered and developed more than 25 ASOs that have enabled the treatment of more than 45 nano-rare patients to date. Many of these new ASO drugs can be used to treat more patients, and we are committed to reaching these patients.” n-Lorem Foundation. “With the encouraging clinical benefit we are seeing in our patients, we believe there is significant momentum to solve some of the challenges facing the nano-rare community, to expand the accessibility of these drugs, and to find commercial solutions that will make these drugs more widely available. We are encouraged to join the One to Millions effort and look forward to working with the learnings from the community.”
