Taking a step toward fulfilling its requirements under Section 506K of the Federal Food, Drug, and Cosmetic Act (Section 506K), FDA has issued Draft Guidance describing how the agency intends to implement the Platform Technology Designation Program. Section 506K was established by Section 2503 of the Prepare for and Respond to Existing Viruses, Emerging New Threats, and Pandemics Act of 2022 and enacted as part of the 2023 Consolidated Appropriations Act.

Per Section 506K (as well as the draft guidance):

A platform technology is a well-understood and reproducible technology, such as a nucleic acid sequence or delivery method, that (1) is essential to a drug, (2) can be adapted for or otherwise used by multiple drugs that share common structural elements, and (3) facilitates the manufacture or development of multiple drugs through a standardized process.

This designation program marks a continuation and expansion of efforts by Congress and FDA relating to advanced manufacturing, including the related efforts regarding FDA’s advanced manufacturing technology program for manufacturing methods that incorporate a novel technology or use an established technology in a unique way to produce a drug of equivalent or superior quality.

Potential Benefits of Designation

If FDA grants a sponsor platform technology designation, the sponsor (or a third party with a full right of reference to necessary data) may have access to the following benefits:

• Engaging in early interactions with FDA to discuss the use of a platform technology.
• Leveraging data, when appropriate, from a prior product that used the designated platform technology.
• Leveraging certain nonclinical safety data, when appropriate, from prior products that used the designated platform technology.
• Considering previous inspectional findings by FDA for subsequent marketing applications related to the manufacture of a drug that inorporates or uses the designated platform technology.

Eligibility

Consistent with Section 506K, the Draft Guidance reiterates that the program is available to sponsors of drugs or biologics already approved under an Abbreviated New Drug Application, New Drug Application, or Biologics License Application that incorporates or uses a platform technology. Further, only technologies that FDA has determined qualify as a platform technology are eligible for platform technology designation.

In addition to being a well-understood and reproducible technology, a sponsor must also demonstrate that the platform technology is incorporated within or used by a drug or biological product to be eligible for designation. To establish this, FDA must find (1) that the platform technology is incorporated in, or used by, an approved drug; (2) that preliminary evidence, i.e., “information from completed tests or studies comparing the platform technology used in the approved or licensed drug(s) with the proposed use of the platform technology in the drug(s) under investigation described in the designation request” indicates that the platform technology has the potential to be incorporated in multiple drugs without an adverse effect on quality, manufacturing, or safety; and (3) the data demonstrates that incorporating the platform technology has a “reasonable likelihood to bring significant efficiencies to the drug development or manufacturing process and to the review process.” FDA recommends submitting the request for platform designation with the investigational new drug application to help expedite the review process.

Other Takeaways

Although the enactment of this program by Congress and its implementation by FDA do signal continued support for the development of advanced manufacturing and do serve as an acknowledgement of the role that manufacturing technologies will play in the successful development of emerging technologies generally, a number of questions remain.

For example, neither 506K nor the draft guidance elaborates on the meaning of a technology that is “reasonably likely to bring significant efficiencies”—a key consideration for program eligibility. Further, the benefits of designation remain, at this point, untested and unproven. We note that sponsors in the ordinary course can (and do) already rely on data and information that they own (or for which they have a right of reference) from previous applications when appropriate.

In addition, recent statements from leadership of the Center for Biologics Evaluation and Research (CBER) suggest that at least CBER is looking to apply the “concepts” of this guidance and authority more broadly than the four corners of eligible development programs. While this underscores CBER’s support for manufacturing technology development generally, it may also highlight the uncertain nature of benefits and value that designation program specifically may bring to the table. Interested parties may submit electronic or written comments on the proposed collection of information in the draft guidance by July 29, 2024.