INSIGHTS ON CELL & GENE REGULATORY ISSUES

  • Real World Evidence (RWE) In Regulatory Decision Making: Establishing Study Credibility And Totality Of Evidence

    This blog shares some of the learnings from the recent Duke-Margolis webinar entitled, "Bolstering RWE Study Credibility and Its Role in a Totality of Evidence Approach".

  • Strategies To Address The Viral Vector Manufacturing Shortage

    Accelerating the production of your viral-vector based therapy requires you to understand the strategies available to address the viral vector manufacturing shortage as well as selecting the most cost-effective solution for advancing your therapeutic.

  • 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

    In this article, we'll examine key changes to FDA's six finalized draft guidance documents on its framework to advance gene therapy. We'll also review a new draft guidance on determining “sameness” of gene therapy products for orphan drug designation/exclusivity.

  • 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.