By Michele Dougherty, Ph.D., Head of Regulatory Affairs Practice and Steven Bowen, Ph.D., Principal Consultant, CMC Biologics

FDA’s release of the draft Guidance for Industry:  Platform technology Designation Program for Drug Development was highly anticipated and provides insight into the Agency’s current thinking on platform technology in drug development, manufacturing and streamlining the review process.

The guidance disusses how platform technology is codified in the FD&C act to ensure that drug developers have a shared understanding of the statutory meaning of platform technology.  The guidance further explains the eligibility factors that must be met to achieve the statutory designation of a platform technology.  The information, data and rationale are compiled into a platform technology designation request that is submitted to the FDA for review.  A successful designation request should be responsive to the statutory requirements as interpreted in the guidance.

To be defined as a platform technology, the technology must be well-understood and reproducible and   

  1. Incorporated and used in a drug or biological product and is essential to the structure of function of said project
  2. Can be adapted for or incorporated into or used by more than one drug or biological product sharing common structural features
  3. Facilitates the manufacture of more than one drug or biological product through a standardized production or manufacturing process

Once the technology has been shown to meet the definition of a platform technology, the FDA considers whether the technology is eligible for a platform designation. 

A designation can be achieved only if the platform technology is used and described in a marketing or licensing application that has been approved by FDA, and if the Sponsor seeking the platform technology designation is the same Sponsor or has full right of reference to the leveraged data in the approved application.  Once that milestone is achieved, a sponsor is positioned to pursue the additional eligilbility requirements.  The approval pre-requisite ensures a comprehensive set of data and information that has been successfully assessed by FDA as sufficient to support safety and efficacy of the product for the proposed platform technology.  From that point, a sponsor can begin to build out the additional data, rationale and justification for the designation and use of the platform technology with subsequent products.  

The additional data contribute to meeting the next eligibility requirement,  preliminary evidence that the platform technology has the potential to be used in subsequent drug development programs without adversely affecting quality, safety and manufacturing.  Preliminary evidence should support that there are minimal differences between the approved drug product and the drugs under development.  The evidence should focus on aspects of structure, mechanism of action, biological effect, manufacturing processes and formulation.  Preliminary evidence should be generated and summarized in a designation request for any product under development that proposes to use the platform technology. 

Finally, data and information should demonstrate the designation will result in significant efficiencies to drug development, manufacturing and to the review process.  The second and third eligibility requirements ensure that sponsors can achieve a streamlined development and review process, potentially reducing development timelines and reducing uncertainty in the review process. 

Potential benefits of the platform technology designation include earlier and more frequent interactions with FDA regarding the use of the platform technology.  The designation should allow sponsors to  leverage prior knowledge and data from products that incorporate use of the platform technology in subsequent development programs, with appropriate rationale and justification.  The guidance indicates that data from stability studies, nonclinical studies and inspection findings could potentially be leveraged across applications. The designation request can be made concurrent with submission of the original IND application, or at any point thereafter.

The platform technology designation holds the promise of streamlining and potentially reducing the time and cost of drug development.  How the designation is implemented in practice is likely to present a challenge for both industry and regulators.  A critical piece of information, namely the preliminary evidence to support that a platform technology can be implemented across products without adversely affecting quality, safety and manufacturing will likely represent an area where agreement will be challenging to achieve.  FDA’s expectations on the type and amount of preliminary evidence needed to support a designation will depend on the type of product and the technology being implemented.  Additional clarity around data expectations for preliminary evidence and how the data can support a designation will be key to successful implementation of the program. Sponsors considering pursuing platform technology designation should utilize pre-submission meetings for their specific program to gain alignment on the data and information needed for a successful request package.