BioPharm International - February 2020

BioPharm-Outsourcing eBook

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14 BioPharm International eBook February 2020 www.biopharminternational.com contract development and manu- facturing organizations (CDMOs) that support them is: "How do we robustly produce, ensure quality, and deliver these transformative and life-saving products to patients around the world safely?" STANDARDIZED PLATFORMS NEEDED As patient numbers addressable by cell and gene therapies grow, new technologies will be needed to meet manufacturing demands. Standardized and robust manufac- turing platforms will need to be established, and a global network of facilities will be required to serve the main cell and gene therapy develop- ment hubs across the globe. CDMOs are responsible not only for manufacturing dr ugs safely and robustly to meet customer demands, but also for supporting innovation from these small bio- techs. This requires facilitating the delivery of therapies to the market under the right conditions, while accelerating an increase in overall capacity, and the infrastructure required to meet increased demand for these therapies at scale. For example, Lonza has been working with a small biotech that is innovating within the oncology and immune tolerance space. As their CDMO, we are committed to lever- age our experience and expertise to de-risk their product's pathway from lab to market and maximize product safety by industrializing its manufacturing process. For any manufacturer, successful industrial- ization requires focus on the "cubic effect," a concept to be discussed later in this article, which includes technical and operational develop- ment, as well as cost. MASS CUSTOMIZATION First, consider that cell and gene therapy is an ever evolving and com- plex science dealing with autologous and allogeneic cell therapies as well as viral vector gene therapies. There are variations in approaches to man- ufacturing between different ther- apies, and in the critical steps that surround streamlining and ensuring good process development. However, when it comes to the reg u lator y pat hway to sca l i ng up processes and ensuring con- sistency, success depends on the manufacturing process and asso- ciated critical quality attributes. A fully optimized and robust process is needed, one that can be "mass customized." For autologous thera- pies, for example, the process must be scaleable, reproducible, com- mercially viable, and customized for every single patient at a large scale. This is where CDMO expe- rience and capabilities are most critical for customers who have been focusing on the safety and the efficacy of their therapy and now face an accelerated path to commercialization. Reflecting on the difficulties of optimizing the process to ensure its scalability is the trouble that some innovators are currently experienc- ing in commercializing therapies. The importance of good process development is illustrated by the dif- ficulty some therapies are encounter- ing when moving to the commercial phase. Once approved, cell and gene therapies must comply with more stringent characteristics defined by regulatory bodies. Getting the process and the manufacturing approach right from the start can facilitate scale-up and reduce the risk of quality and consistency issues, which often result in delays due to the need to tweak processes at the critical commercialization point. Regulatory agencies are offering increased support to help advance the growth of innovative therapies that aim to meet unmet medical needs. Given the levels of efficacy that some cell and gene therapies have shown, FDA and other global regulators have created accelerated approval pathways and allowed unprecedented small-scale clinical trials for these therapies. Regulators are also supporting technologies that can advance manufacturing and shorten the timeline between clin- ical trials and commercial scale-up. PREVENTING OVERCAPACITY At this crucial point in the devel- opment of cell and gene therapies, developers need to constantly reas- sess the demand forecast and scale their production accordingly to ensure the right investments are made into CAPEX (capital expendi- ture) and OPEX (operating expense). Overestimating the figure will result in overcapacity in five or 10 years. This is particularly true since some of these therapies are curative and only administered to patients once, unlike treatments for chronic condi- tions, which require multiple doses or treatments over time. Once the current prevalent patient population has been treated, therapies would only be needed for newly diagnosed patients. The need for industrialization is not new for the life-sciences indus- try. In fact, with cell and gene thera- pies, we are seeing history repeating similar patterns that were seen in the past, when the first monoclo- Partnerships for Outsourcing Manufacturing Getting the process and manufacturing approach right from the start can facilitate scale-up and reduce the risk of quality and consistency issues.

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