Regulatory Requirements for Cell-Based Therapy for Degenerative Retinal Disease

Abstract

Cell therapies present many unique challenges to regulators. The science is continuing to advance. Cell replacement therapy is part of the Audacious Goals Initiative of the U.S. National Eye Institute, and viewed as having great promise in restoring vision through regeneration (Levin et al., Ophthalmology 124:926–934, 2017). However, the risks to patients, including unethical promotion by unscrupulous organizations, are real. The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) each have published guidance on many aspects of the investigational use of cell therapy—part of the larger group of human cells, tissues, or cellular or tissue-based products (HCT/Ps). HCT/Ps are regulated primarily as biologics.

Government regulatory guidances are continually being updated with changing science and input from patients, physicians, pharmaceutical/biotechnology firms, and the National Institutes of Health. Development of cell therapy follows the same pattern as with small molecules—i.e., discovery, chemistry, manufacturing and controls (CMC), nonclinical pharmacology, pharmacokinetics and toxicology, and, finally, clinical trials with increasing doses, duration, and number of patients. However, as cells are a living, biologic therapy, additional efforts are required in each of these disciplines to provide a known, high-quality therapeutic product to patients. For the most part, the same regulatory oversight is required for cell therapy as for small molecules—e.g., Investigational New Drug (IND) exemption and marketing applications (the latter called a Biologic Licensing Application (BLA) in the USA).

Given the unique, variable, and changing nature of cell therapy, and the ongoing update of regulatory guidances, it is not possible to give a “one-size-fits-all” summary of the regulatory pathway for cell-based therapy for degenerative retinal disease.

This work has not been previously presented.