The Risk Based Approach to Nonclinical Development of Cellular Therapies
September 27, 2022
11:00 AM EDT | 4:00 PM BST | 5:00 PM CEST
A one-hour BSTP webinar, in collaboration with the Society of Toxicological Pathology (STP), will be held on Tuesday, September 27, 2022, at 11:00 AM Eastern Daylight Time (New York, GMT-4), 4:00 PM British Summer Time (London, GMT+1).
Title: The Risk Based Approach to Nonclinical Development of Cellular Therapies
Speakers: Michaela Sharpe, PhD, Senior Nonclinical Director at Moare Solutions Ltd; and Alys Bradley, BVSc, FRCVS, FIATP, Director of Pathology, European Safety Assessment at Charles River Laboratories
Abstract: Cell therapies have different challenges than other drug classes. Although cell therapies can share some of the same principal characteristics, they are not a homogeneous class of product. This is recognised by the regulatory agencies. The European Medicines Agency (EMA), for example, introduced the risk-based approach to determine the extent of quality, non-clinical and clinical data to be included in the Marketing Authorisation Application (MAA). This approach provides the possibility of omitting guideline-based nonclinical studies based on risk analyses. Therefore, although the basic aim for the nonclinical program, to determine the efficacy and safety of the product, there remains flexibility in how the safety and efficacy are demonstrated.
As an exemplar, the approaches that have been applied to cellular immunotherapies will be explored. Genetically-engineered T-cell therapies have revolutionized the treatment of cancer and these therapies have shown exceptional clinical efficacy especially in the field of B-cell malignancies. The power of these treatments is also linked with a distinct set of toxicities both predicted and unpredicted, including off-tumour activity, cytokine release syndromes, and neurotoxicity, occasionally with fatal consequences. As these therapies and next generation products begin to reach more patients, the nonclinical tools to determine the mechanisms driving these toxicities, to assess the safety risks of candidate products, and to develop strategies for safety management, have been rapidly evolving.
We look forward to your participation.