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.