Applying Patient-centric Quality Standards Concepts to Biotechnology and Advanced Therapy Products

The current practice in establishing specifications as defined in ICH specification guidelines is essentially based solely on clinical experience. This can result in overly tight specifications that are not necessarily relevant to safety or efficacy and thus can lead to rejection of what is actually good product and limit the ability to continuously improve the manufacturing process. Patient-centricity in the development of specifications has additional focus on the impact to patient but using data beyond that simply what material was included in clinical studies. This data can be prior knowledge, in vitro models (e.g. immunogenicity), removing material from patient samples etc. Patient-centric quality standards (PCQS) are a set of patient relevant attributes and their associated acceptance ranges to which drug product should conform within the expected patient exposure range to deliver the therapeutic benefit indicated in the label. PCQS should be built within a patient centric control strategy (PCCS - which describe how and where the PCQS should be evaluated). The concept of PCQS has been around many years using other terms such as ‘clinically relevant specifications’ or ‘patient focused specifications.’ The term “patient centric” and “patient relevant” are currently preferred as these phrases emphasize the link to the patient and attempt to break the misconception that “clinical relevance” equates to clinical trial experience alone. The PCQS approach can benefit manufacturers who have an understanding of their process, product design and CQAs and their relation to patient impact. Applying this knowledge to the establishment of a PCCS can support opportunities for future continual improvement and reliable supply of medicines. This session introduces the PCQS concepts and provides case studies that describe how the new approach to setting specifications could be applied large molecules, cell therapy products and includes products approved via accelerated pathways. Speakers from industry will present case studies demonstrating the PCQS concepts and the panel discussion will include EU and US regulators.