ATMP-Specific Needs
Although global regulators such as the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) have long presented guidelines regarding validation, production of ATMPs often cannot follow the same general rules as those developed for small and large molecules for traditional therapies.
The ISPE Guide: Advanced Therapy Medicinal Products (ATMPs) – Validation Methods and Controls Throughout the Cell and Gene Therapy Lifecycle offers practical guidance on applying validation methods and control. It also outlines risk-based strategies tailored to the unique nature of these products, addressing key areas such as analytical methods, equipment, processes, cleaning, and supply chain. It includes essential insights into regulatory expectations and phase-appropriate considerations that support timely development and commercialization.
“Regulatory agencies have advocated for risk-based approaches for a while,” said Guide Lead Biana Torres, Senior Director of Quality Assurance at Encoded Therapeutics. “Ultimately, the purpose of a risk-based approach is to understand what’s critical to your product quality, patient safety, and product variability. This understanding helps you focus on those elements to be able to ensure you have manufactured a safe product. If you don’t understand the risk, you won’t know where to focus and may neglect critical to quality elements. Ultimately, lack of risk-based approaches can lead to wasted resources, inefficient operations, or even prevent products from coming to market, thus limiting the intended benefit for patients.”
A Risk-Based Approach
A risk-based approach provides manufacturers with the flexibility necessary to adapt the best controls to the process while ensuring all requirements and critical aspects of the process are met. This is especially true for investigational medicinal product (IMP) phases. Applying risk-based approaches can support companies to be more efficient in overcoming regulatory hurdles.
The objective of this guide is to provide comprehensive risk-based validation strategies across the ATMP product life cycle.
“Companies want to adopt a risk-based approach, but there is not a standard way to implement this in the cell and gene therapy space,” said Torres. “This guide supports the industry and provides actionable insights to help navigate the complex ATMP landscape. It promotes flexible, science-driven development approaches that support informed decision-making and help accelerate patient access to these transformative therapies.”
Validation and Regulatory Compliance
Written by subject matter experts in regulatory, quality, engineering, and facility development that have developed risk-based validation approaches for ATMP companies, the Guide offers an in-depth analysis of validation activities specifically for ATMPs. This includes the validation of equipment, processes, analytical methods, cleaning procedures, and transportation logistics. Additionally, this guide outlines regulatory considerations to help streamline the path to commercialization and provides essential insights into the life cycle management of ATMPs.
Chapter three highlights some key regulations and guides pertinent to ATMP validation activities and risk management focused on the validation elements for analytical methods, process, equipment, cleaning, and supply chain. Because ATMPs are newer technologies, regulators are working with the goal of harmonizing regulations across the world and continue to release ATMP-specific regulations. The most recently released regulations (at the time of the guide’s publication) that are crucial for ATMP risk-based validation purposes include the following:
- PIC/S Annex 2A – Manufacture of Advanced Therapy Medicinal Products for Human Use
- PIC/S Annex 1 – Manufacture of Sterile Medicinal Products
- ICH Q9(R1) Quality Risk Management
The ultimate goal of the guide is to establish an effective control strategy that helps stakeholders navigate the complexities of ATMP validation, thereby facilitating regulatory compliance and fostering innovation in the field.
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