INSIGHTS ON CELL & GENE REGULATORY ISSUES

• CCDS Or Local Labeling, A Chicken-And-Egg Situation?

  In this blog, UBC’s Dr. Luca Cariolato, Sr. Safety Scientist, speaks to the importance of developing a Company Core Data Sheet (CCDS) before local safety information.

• An Analysis Of FDA’s Recently Issued Gene Therapy Guidances

  The FDA has finalized six draft guidance documents intended to serve as part of a modern, comprehensive framework for how the agency will help advance the field of gene therapy. At the same time, it issued a new draft guidance on determining “sameness” of gene therapy products for the purposes of orphan drug designation/exclusivity. This article highlights key changes made in each of the final guidances and also reviews the new draft guidance.

• FDA FY2019 Human Tissue And Cell Therapy Observations And Trends

  Very soon after publication of the FY2018 data on human tissue and cell therapy products, the FDA released the data from FY2019, which we address here. We examine data from FY2019 and a total of five years’ worth of trends in GMP inspection enforcement in this area. The regulations that govern this area, 21 CFR 1271, are the same regulations that govern segments of human cell therapy and gene therapy products. Human cell and gene therapy products are part of FDA’s Regenerative Medicine framework.

• Considerations For Operationalizing Pediatric Oncology Studies

  Remarkable progress has been made in our understanding of the genomics of pediatric cancers, and these advancements have led to the recognition that products being studied for use in adult cancer indications may have health benefits for pediatric patients. By closing the orphan drug exemption loophole and enabling earlier discussions with the FDA, the Research to Accelerate Cures and Equity (RACE) for Children Act has the potential to accelerate the development of novel treatment options for children with cancer. Read more how RACE and the FDA guidance will affect the future of pediatric oncology.

• 3 Keys To Realizing FDA’s Vision For CAR-T And Other ATMPs

  While cell therapy remains a very promising approach to developing much-needed new immunotherapies, significant challenges will have to be overcome in order to realize the FDA’s twenty-first century vision of making complex ATMPs widely and cost effectively available to patients.

• Sampling And Testing Of ATMPs: Gaining Insights From PIC/S Annex 2A

  With minimal specific guidance on commercial manufacturing from the FDA and a lack of robust platform commercial processes, some of the finer details of cell and therapy manufacturing can make operating companies feel like they are walking on a precarious limb.

• Planning Is Everything - Quality Control Testing In Cell And Gene Therapy

  Compliance with quality control (QC) standards is a basic requirement for products manufactured under good manufacturing practices (GMP) conditions. In this post we will explore the main compendial QC tests that ensure cell and gene therapies are safe for use in patients, and important considerations for integrating QC testing into the manufacturing process.

• Understanding Recent Regulatory Changes For Pediatric Oncology Trials

  While children typically have a better cure rate for cancer, biopharma companies have traditionally shied away from pediatric oncology trials, resulting in fewer treatment options. However, the regulatory landscape is evolving, and the demand for innovative therapies for pediatric cancers is becoming increasingly vocal.

• Automating Cell Therapy Manufacturing For GMP Compliance And Consistency

  Exploring the use of automation in cell therapy and advanced therapeutic medicinal product manufacturing to improve safety, quality, and compliance is a key factor in advancing patient care.

• FDA FY2018 Human Tissue And Cell Therapy Observations And Trends

  This article presents the most recent publication of GMP Human Tissue for Transplantation Form 483 observation data from the FDA. We examine data from FY2018 and evaluate a total of four years’ worth of trends in GMP inspection enforcement in this area.