Before any cell therapy company can begin manufacturing a therapy, or designing and engineering the production equipment, it is necessary to engage in detailed brainstorming and planning. You must first define your product—who do you want to serve, and what do you want your product to achieve?
The FDA has provided draft guidance for creating a Target Product Profile (TPP) to document product attributes in a format that can evolve into the label claims for the product. While not required, a TPP or similar approach should be created and maintained by cell therapy developers. The FDA has also provided guidance via ICH Q8 for pharmaceutical development (where Quality by Design (QbD) principles are presented) for establishment of a Quality Target Product Profile (QTPP). Using a target product profile template as a key source of input, the QTPP is derived and maintained to detail the targeted post-manufacturing product attributes needed to support safety and efficacy of the product.
While there is certainly latitude in how a QTPP is constructed, a recommended format provides the following categories of information:
- Characteristics profile (e.g. description, formulation, dosage, potency, volume, shelf life)
- Safety profile (e.g. microbial assurance, cellular impurities, manufacturing residuals)
- Use profile (e.g. indications for use, treatment timing, preparation and use)
- Business profile (e.g. geographies, market projections, clinical/commercial milestones, cost of goods targets, IP)
Often when cell therapy clients begin clinical development of a product, many of the elements of the QTPP are not fully known. Generally, the tendency is for the QTPP to be largely overlooked until a majority of the information can be specified, but that often leads to poor manufacturing development decisions that are regretted later on. The QTPP should be developed—at least as an initial draft—very early on, albeit with some areas that will need to be more thoroughly fleshed out at later stages. For example, elements of the business profile are often difficult to specify at an early stage of product development.
When developed early, the QTPP provides the input to establish a rational development plan that starts with the end in mind. The plan can then be confidently used to maintain strategic alignment among the ever-growing and ever-changing stakeholders as the therapeutic program develops, and to manage the risk of manufacturing development drifting away from the intended target. When a cell therapy developer is ready to engage external resources such as a contract development and manufacturing organization, this strategic alignment provides the right foundation for success.
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To learn more about the Quality Target Product Profile, click the button below to request a PDF copy of the complimentary white paper, “Manufacturing Cell Therapies: Development Strategies for Commercial Vision.”
