No abstract

Regenerative medicine is a rapidly evolving field that faces novel scientific and regulatory challenges. In September 2013, the International Workshop on Regulatory Pathways for Cell Therapies was convened to discuss the nature of these challenges and potential solutions and to highlight opportunities for potential convergence between different regulatory bodies that might assist the field's development. The workshop discussions generated potentially actionable steps in five main areas that could mitigate cell therapy development pathway risk and accelerate moving promising therapies to patients. These included the need for convergence of regulatory guidelines on donor eligibility and suitability of lines for use in clinical trials and subsequent commercialization for cell therapies to move forward on a global basis; the need to challenge and encourage investigators in the regenerative medicine field to share information and provide examples of comparability studies related to master cell banks; the need for convergence of guidelines across regulatory jurisdictions on requirements for tumorigenicity studies, based on particular cell types and on biodistribution studies; the need to increase transparency in sharing clinical trial information more broadly and disseminating results more rapidly; and the need to establish a forum for sharing the experiences of various approaches being developed to expedite regulatory approvals and access for patients to innovative cell and regenerative therapies in the different regulatory jurisdictions and to assess their potential strengths and weaknesses.

Effective interaction between key stakeholders and the U.S. Food and Drug Administration (FDA) is central to successfully navigating the regulatory process and advancing new therapies into clinical trials. This is especially true when developing cell-based therapies, which pose unique challenges to demonstrating safety and effectiveness. There are numerous opportunities for developers of a new cell therapy to interact with the regulatory agency, through both formal and informal processes. It is important to understand how to maximize the productivity of dialogue with the FDA and develop an effective regulatory strategy. This article provides an overview of the types of interactions with the FDA that are available throughout the regulatory process. This article also notes some common pitfalls to avoid and directs readers to additional references and resources to help inform cell therapy researchers and product developers and enable successful regulatory interactions. STEM CELLS TRANSLATIONAL MEDICINE 2012;1:825-832

Establishing and maintaining consistency and reliability of raw materials and the product throughout development stages and in the fi nal commercial process is critical and presents a challenge for complex biological systems due to multiple factors that aff ect their viability and function.The leveraging of clinical data throughout development can occur only if the drug product has similar or comparable qualities throughout development. Thus, comparability for CGT therapy products is a critical aspect of process development and is required to link clinical data throughout progressive phases of product development.Assays measuring product potency are of central importance to all product development and quality control activities. Potency assays serve many functions. They help to identify and control sources of variation in product activity and to test for product comparability across lots, process changes, sites, scale changes, and stability testing.