A Need for Enabling Technologies
Facilitating these standards will require the design and engineering of robust enabling technologies to address development and manufacturing challenges, and demonstrate product safety and/or efficacy.
These should be easy to use and provide reproducible results across multiple locations and usage environments, despite variation in user input. This approach also envisages the repositioning of existing platform technologies that are currently utilized outside the regenerative medicine field.
Take a specific example: the EC published Directive 2004/23/EC concerning standards of quality and safety for the donation, procurement, testing, processing, preservation, storage, and distribution of human tissues and cells. Article 23 of this directive requires establishments involved in distribution to comply with specific requirements to ensure the quality of tissues and cells during transit.
Here is an opportunity to develop enabling technologies that ensure that the environmental conditions in which tissues and cells are kept are within acceptable quality levels through the workflow. Furthermore, they should ensure that full recording of relevant conditions, such as temperature and physical shock, can be made.
Other common challenges pursuant to this sector, which may lend themselves to standardized approaches, include the following:
- bioprocessing, particularly manufacturing controls and metrology, including systems for batch release inspection and data logging; and
- physical delivery of the therapeutic into the subject—common or modular device platforms optimized to avoid cellular damage (through shearing forces) and capable of dealing with small delivery volumes.
In many cases the development of enabling technologies might most effectively be undertaken on a precompetitive footing in order to address generic processes faced by the industry as a whole.
To illustrate this point, consider an enabling technology widely used in respiratory pharmaceutical development. The Andersen Cascade Impactor (ACI) was developed for the size resolution of aerosolized particles, such as dry powder formulations intended for use in inhaled devices.
The ACI has been instrumental in standardizing a key aspect of regulatory development of inhaled medicines. The first generation of impactors required a labor-intensive protocol, which did not lend them to high-throughput analyses. Furthermore, the device was difficult to clean following each analysis.
To attempt to resolve these issues, a 15-member pharmaceutical industry consortium developed the Next-Generation Impactor (NGI). Substantial user involvement in the design process resulted in an easier to use, higher-performance, precision cascade impactor for testing metered-dose, dry-powder, and similar inhaler devices.
In this case, it was only when parties with a common interest got together on a collaborative footing to pool technical and commercial resources that the design for the NGI became more fit for purpose, rather than attempting to utilize nondedicated instrumentation for suboptimal development activities.
The example shows how collaborative development promises to support the industry as a whole where the players face specific challenges that are generic to the field and that the industry cannot afford to overlook in its pursuit of overcoming developmental or production bottlenecks.
As demonstrated in the examples in this article, the development of these enabling technologies is often best approached proactively through precompetitive collaboration. And, just as standards need to be harmonized globally, so too does the collaboration.
We believe that it is through the development and adoption of these harmonized and global standards, as well as the reduction of the cost of goods, that engineering and product development will be able to support the regenerative medicine industry finally going mainstream.