Advanced Therapy and Advanced Manufacturing for Unmet Medical Needs

To address the unmet needs and to enhance the living for our society, potential solutions encompass:

• Advanced Therapies, which offer groundbreaking new opportunities for the treatment of disease and injury.
• Advanced Technology to deliver medicines faster, cheaper, and with enhanced quality.
• The combination of the two above.

Numerous pharmaceutical companies are diversifying their portfolios, while some are taking a leap into the ATMP (Advanced Therapy Medicinal Product) era. The emergence of new therapies leads to the need for multi-modal manufacturing facilities. Given the need for multi-modality, the nature of ATMP products and their manufacturing processes, and the early-stage technological development, meticulous planning for facility segregation, flexibility / adaptability and capacity is imperative across different stages of the product lifecycle.

Beyond the technical complexities, the risk averse industry is facing other obstacles while shifting to new technologies. To name a few of them: regulatory uncertainties, the assessment on medicine’s Quality, Safety and Efficacy, and the impacts of the shifts on other areas (i.e., supply chain management, pharmaceutical quality system, and so on).

The 2023 ISPE Annual Meeting & Expo will feature the track Advanced Therapy and Advanced Manufacturing which will present attendees with the latest advancements and various real-life case studies by sharing the available solutions and best practices. Case studies, based on the invaluable project experiences of organisations like Wuxi Biologics, Bayer, United Therapeutics, Kite Pharma, Moderna and Spark Therapeutics, will be collaboratively presented by user organisations, their engineering partners, consultants, and equipment suppliers. The close collaborations between these organisations are pivotal for project success.

The commercialisation of an ATMP product has always been under the spotlight. While thousands of innovative therapies are in the pre-clinical and clinical phases, the approved products are only a handful (32 approved products3 under FDA’s jurisdiction by the time this article is published). Factors such as the number of ATMP products are developed in hospital or academia settings, the nature of personalised medicine, scarcity of automated solutions in the market, and the significant impact of the manufacturing process on the product, necessitate a mindset shift for the ATMP product commercialisation. This encompasses process development, facility design, equipment selection, quality system establishment, supply chain management, as well as personnel training. The speakers in the Advanced Therapy and Advanced Manufacturing track will offer their invaluable insights from the therapy development phase to the later phases of facility design and construction so to offer a comprehensive understanding of how to bring a successful therapy to the market.

Over the decades, the pharmaceutical industry, as well as the regulators, have embraced changes at its fastest pace. The need for building new facilities and revamping existing facilities (including manufacturing facilities, hospital facilities and academic facilities) to accommodate new therapies is evident. Equally vital is to deliver the new and modified facilities in an efficient and cost-effective manner. Accommodating multi-modality and new technology will profoundly influence the facility design. Equally crucial is the operation strategy, which should be established based on a thorough investigation not only on the product and process, but also on the business rationale and market dynamics (including the availability of skilful workforce). A multi-modal facility may either be designed for campaigning several modalities over the course of a year, or for concurrent processing of multiple modalities. The latter scenario will require a larger footprint, more stringent requirements on contamination control strategy (i.e., segregation, control of material and personnel flow) and a higher number of operation teams. While the campaigning facility may require higher flexibility for the facility and equipment. In “Transformation into ATMP” we'll take a closer look at how a conventional biologics manufacturing facility was transformed into a modern, multi-modal facility for ATMP products. Meanwhile, you will be presented with the comparison between different facility design approaches and the specific design considerations for each approach.

In comparison to the traditional site-built / stick-built methods, the pre-fabricated modular or even podular facilities offer advantages in terms of delivery timeline and standardisation. Furthermore, the possibility of incorporating robotics within the podular facilities will lead us to more opportunities and improvements. Our speakers in sessions “Therapy Development and Facility Design for Market Readiness” and “Technology Advancement” will illustrate diverse facility design solutions through tangible case studies for ATMP facilities as well as other aseptic filling operations.

While embracing the excitement and opportunities offered by the new therapies and technology, it’s also recognised that we are still in the early phases of development, and there will be new challenges and newer challenges to overcome. In this context, ISPE, as a leading industry society, serves to connect the pharmaceutical knowledge and facilitate idea exchange through platforms like the 2023 ISPE Annual Meeting & Expo. Join us on 15-18 October in Las Vegas, Nevada, USA to be a part of this journey of exploration and innovation.

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• 3Center for Biologics Evaluation and Research. “Approved Cellular and Gene Therapy Products.” U.S. Food and Drug Administration. Accessed September 7, 2023. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products.