What is the clinical trial process for a new diabetes therapy?

The clinical trial process for a new diabetes therapy, or any new biologic or cell-based treatment, follows a structured regulatory pathway designed to evaluate safety and efficacy progressively before a therapy is made widely available to patients.

The process begins with preclinical development, in which the therapy is evaluated in laboratory studies and animal models to establish initial safety and efficacy data. This is the stage RMS is currently completing. A successful preclinical program supports the submission of an Investigational New Drug (IND) application to the FDA, which, if approved, permits the therapy to enter human trials.

Clinical trials are conducted in three phases. Phase I focuses primarily on safety, testing the therapy in a small number of participants to identify any adverse effects and establish safe dosing parameters. Phase II evaluates efficacy and further assesses safety in a larger group. Phase III involves large-scale trials comparing the therapy against existing treatments to generate the evidence required for regulatory approval.

For cell-based therapies like RMS’s, there are additional tools and designations that can streamline this process. Adaptive trial design allows Phase I and Phase II to be combined, evaluating safety and efficacy simultaneously rather than sequentially, potentially reducing the time and cost of reaching regulatory approval. The FDA also offers expedited programs specifically designed for therapies addressing serious unmet medical needs, including Regenerative Medicine Advanced Therapy (RMAT) designation, which provides intensive FDA guidance and priority review for cell and tissue-based therapies; Fast Track Designation, which accelerates development and review for therapies with the potential to address unmet medical needs; and Breakthrough Therapy Designation, which expedites development when early clinical evidence suggests substantial improvement over existing treatments. Cell-based therapies for diabetes may be eligible for one or more of these pathways, which can significantly shorten the journey from clinical trials to patients.

Learn about RMS’s diabetes research.