Critical Path Institute® (C-Path) today announced the launch of One to Millions, a global, multi-stakeholder public-private initiative to enable the scalable development of advanced treatments for highly individualized conditions. Rapid advances in technologies such as antisense oligonucleotides, genome editing, gene therapy, and RNA-based therapies have made it possible to design precisely targeted interventions for very small patient populations and even individual patients. However, existing regulatory and reimbursement frameworks built for population-based medicines and linear development models do not have the capacity to accommodate, creating growing gaps that slow patient access.
To bridge this gap, One to Millions leverages C-Path’s centralized regulatory-level data platform to advance the U.S. Food and Drug Administration’s (FDA) Adequacy Mechanism Framework and Rare Disease Evidence Principles to support evidence generation, regulatory decision-making, and scalable development pathways.
Words cannot adequately express how important this moment is for transforming lives and realizing the long-awaited innovative vision. One to Millions is a partnership unique to C-Path, built to make personalized care scalable to even more people. It features a central, regulatory-ready data platform. A unique pre-competitive environment across the ecosystem. Integrated preclinical, translational, clinical, and patient-level outcomes. A practical evidence framework for optimizing efficacy and safety assessments. Ability to generate regulatory grade tools needed to establish continuous learning and validation processes. There is no other initiative like this. ”
Klaus Romero, MD, MSc, FCP, CEO, Critical Path Institute
A modernized platform approach provides the consistency and reliability essential to advanced treatment techniques. Standardization of manufacturing and release testing protocols directly addresses the unreasonable expense that typically burdens low-volume drug development. Building new treatments on established architectures allows developers to leverage prior knowledge and allows regulatory reviews to focus strictly on new components rather than evaluating the entire foundation from scratch.
Julia Vitarello, founder of Mira’s Miracle Foundation and co-founder of the N=1 Collaboration, stressed the urgent need for a coordinated approach to address the shortcomings of the current situation. “These are very exciting times in genetics. Today we have the science to help a huge number of children with serious, life-altering rare diseases, but our access system was not designed for the thousands of genetic diseases that each affect a small population,” Vitarello said. “We are excited to work with regulators to move from approving one drug for one disease at a time to creating a process that can be applied to many diseases. This shift has the potential to be game-changing for millions of patients, but we will only succeed if we ensure we continually and iteratively learn from these treatments by systematically collecting and sharing data that informs the development of safer and more effective medicines.”
Integrating post-approval evidence generation into the development paradigm is a hallmark of this effort. Incorporating longitudinal registries directly into the framework allows evidence generated for regulatory decision-making to simultaneously inform payer durability, safety, and effectiveness assessments. Integrating information from across the ecosystem prevents duplication of effort and accelerates learning. This point was emphasized by Janet Woodcock, MD, long-time CDER director and former acting FDA commissioner. “New technologies have the potential to correct the root causes of devastating monogenic diseases. However, if the information is not available for collective analysis and learning, progress may stall and regulatory requirements remain overly conservative,” Woodcock said. “We should not repeat the mistakes of the past. We should strive for rapid knowledge transformation and agile development in this new field by sharing what we have learned. Our patients do not deserve that either.”
Compiling existing preclinical, translational, and clinical data sources can help optimize toxicology and dose selection. Collecting robust data can maximize the utility of alternative methods and reduce unnecessary reliance on animal experiments while creating a paradigm of continuous learning and confirmation.
“This represents an important new tent pole in interventional genetics, providing a long-missing piece for approval and reimbursement and completing the arc that began with FDA’s 2021 guidance on personalized antisense therapies,” said Timothy Yu, MD, PhD, of the Division of Genetics and Genomics at Boston Children’s Hospital. “By effectively enforcing modularity, this framework enables developers to leverage data across therapies targeting different genetic mutations without having to re-invent the regulatory process for each mutation. “We cannot build such a system in isolation; to advance treatments that are truly greater than the sum of their parts, we must share learning through robust data sharing, with each breakthrough leading to the next.”
“At n-Lorem, we have built a robust and scalable process to deliver personalized ASO medicines to nanorare patients. We have discovered and developed over 25 ASOs and have enabled the treatment of over 45 nanorare patients to date. Many of these new ASO medicines can be used to treat more patients, and we are committed to reaching them.” “With the encouraging clinical effects we are observing in patients, we believe there is significant momentum to solve some of the challenges facing the nanorare community, expand the availability of these medicines, and find commercial solutions that will make these medicines more widely available. We are encouraged to join the One to Millions initiative and look forward to providing the insights we have learned from the research we are conducting in the community.”
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Critical Path Research Institute (C-Path)
