Peter Marks M.D., Ph.D.
Director, Center for Biologics Evaluation and Research (CBER), FDA
Thank you for this question.
FDA is steadfastly committed to working with stakeholders, including other government agencies, academia, industry, and product manufacturers, to foster beneficial innovation in this field and make the development and approval of innovative cell and gene therapies more efficient.
As we continue to develop an evidence-based framework for these products, FDA understands that we may need to re-evaluate and modernize our approach to the unique challenges of these products while also ensuring the resulting therapies are both safe and effective.
There are indeed steps that could be taken toward more efficient gene therapy product development. The sharing of best manufacturing practices among academic and industrial developers could result in advances associated with better product quality, including consistency and yield, along with reduced costs. Additionally, while countries around the world have their own regulatory authorities, there are not uniform global quality safety standard for the evaluation and regulation of cell and gene therapy products. FDA supports work toward regulatory convergence and, ultimately, global harmonization of regulations for these products. FDA is pursuing this goal with international partners, global regulators, and the World Health Organization (WHO).