What technologies are eligible for platform technology designation?

FDA developed the new platform technology designation program draft guidance to fulfill its requirement under Section 506K(b) of the Federal Food, Drug, and Cosmetic Act (FDCA), codified at 21 USC 356k, which was passed as part of the “PREVENT Pandemics Act” in December 2022. FDA clarifies in the guidance that the common understanding of what constitutes a “platform technology” — which has been viewed very broadly in the past — differs from the more narrow and precise definition of the term in the statute and guidance.

Under FDCA Section 506K(b), a platform technology is a “a well-understood and reproducible technology, which may include a nucleic acid sequence, molecular structure, mechanism of action, delivery method, vector, or a combination of any such technologies that [FDA] determines to be appropriate.” A platform technology “incorporated in or used by” an FDA-approved drug can be designated if it meets the following criteria:

1. The platform technology is included in, or utilized by, an approved drug or biologic;

2. preliminary evidence demonstrates that the platform technology has the potential to be incorporated in, or used by, more than one drug without an adverse effect on quality, manufacturing, or safety; and,

3. data or information submitted by the applicable person indicates that incorporation or usage of the platform technology has a reasonable likelihood to bring significant efficiencies to the drug development or manufacturing process and to the review process.

“Preliminary evidence,” as referred to in 21 USC 356k (b)(2), means information from completed tests or studies comparing the platform technology's use in approved drugs with its proposed use in drugs under investigation. To support a designation, this evidence must sufficiently demonstrate the potential for incorporating or using the platform technology in the investigational drug without adversely affecting quality, manufacturing, or safety. There should also be “minimal differences” between the approved or licensed drug or drugs using the platform technology and the investigational drug. Minimal differences may be supported by evidence such as similarities in structure, mechanism of action, formulation, or manufacturing processes between approved drugs and those under investigation.

What information should be included in a platform technology designation request?

A request for platform technology designation should include, among other things:

• A description of the platform technology and how it meets the statutory definition and eligibility requirements.

• Identification of the approved application where the technology was incorporated.

• • FDA has indicated that data from approved Abbreviated New Drug Applications (ANDAs) and 351(k) BLAs (biosimilars) can also be referenced in support of a designation request. However, the benefits of the platform technology designation program are not available to ANDAs or biosimilars.

• Identified shared structural elements between the approved and proposed product.

• Summary data of assessment of all the requestor’s products using or incorporating the platform technology, regardless of developmental or marketing status. Summary data from these assessments should be provided, along with justifications for their sufficiency.

• Justification of how the platform technology would bring significant efficiencies to the drug development or manufacturing process and to the review process. For example, how designation of the technology platform will reduce the need for testing or validation requirements.

We note that FDA emphasizes in the draft guidance that although NDA sponsors can simply cross-reference prior applications where a platform technology was leveraged, BLA holders must include full information because there is an expectation that a BLA holder is generally expected to have knowledge of and control over the manufacturing process for the biological product for which it has a license.

While designation requests can be submitted concurrently with an IND, FDA recommends sponsors consider waiting to submit a designation request until there is adequate product-specific data on both the prior product and subsequent product to permit a determination of suitability for platform technology designation.

Who can submit a designation request and leverage data from a designated platform technology?

The sponsor of an approved application containing the platform technology can submit a request for designation and can leverage the data in a subsequent application. In addition, a different sponsor may leverage the data and information in the approved application incorporating the platform technology if that subsequent sponsor receives full rights of reference to all data necessary to support the platform technology designation. Designation of a platform technology cannot be leveraged absent ownership of, or a right of reference to, the data or information.

Benefits of platform technology designation

A platform technology designation offers several potential benefits to sponsors that own the platform technology designation or have a right of reference to it, including:

• Early interactions and advice. Sponsors can engage with FDA early to discuss using the platform technology, including aspects related to safety, purity, potency, or quality.

• Continuous feedback and engagement during product development. Sponsors will receive timely advice from and additional engagement with FDA during the development program.

• Use of data from prior products. Sponsors can leverage data from prior products using the designated platform technology, such as batch and stability data, that either those sponsors own or to which they have rights of reference. This can supplement product development studies, support shelf-life extrapolation determination, and inform container closure system design.

• Use of nonclinical safety data from prior products. Nonclinical safety data from prior products using the platform technology can also be leveraged, potentially reducing the need for product-specific assessments for designated endpoints.

• Previous inspectional findings. Sponsors can consider previous FDA inspectional findings related to the manufacture of a drug using the platform technology.

However, it is important to note that platform technology designation does not automatically grant priority review for subsequent applications under the User Fee Authorization program or affect eligibility for expedited approval pathways if the relevant criteria are met.

Particular applicability to cell and gene therapy products

The platform technology designation may be especially beneficial for sponsors of cell and gene therapy products.[1] Dr. Nicole Verdun, Director of the Super Office of Therapeutic Products (OTP) within FDA’s Center for Biologics Evaluation and Research (CBER), has particularly emphasized the benefits of this program for cell and gene therapy sponsors.[2]

Next steps

FDA recommends requesting the designation of a platform technology during the IND phase of drug development for a planned subsequent NDA or 351(a) BLA. Requestors should submit their request when they have sufficient knowledge, including summary evidence from completed studies, to justify how the platform technology meets eligibility requirements for designation.

FDA will evaluate each request based on the provided data, the potential impact on product development and manufacturing, and the platform's ability to ensure product quality and safety. FDA will make a determination on the request within 90 days of receipt. If granted, the designation will be valid for a specified period, typically covering the duration of product development and initial marketing. Although specific circumstances are not described in the draft guidance, it should be noted that a platform technology designation status can be revoked if FDA determines that the technology no longer meets eligibility criteria.

Remaining questions and considerations

The guidance leaves open a number of issues. One issue is whether FDA will publish platform technology designations and related data publicly. Neither the statute nor the guidance explicitly contemplate publication of platform technology designations, or how follow-on sponsors could identify a designation. When determining whether to grant or obtain a right of reference to a platform technology designation, all parties should carefully consider their respective needs for the designation, and related information and data about the platform technology and the products into which it is incorporated, to be made public or to remain confidential. Consequently, they should scrutinize all rights of reference and related agreements carefully.

Another issue to consider is the potential implications of arguing about the similarity between the initially approved product incorporating platform technology and subsequent products incorporating that technology. It will be important to frame those arguments carefully so as to preserve arguments necessary to distinguish the prior and subsequent products where needed. For example, certain FDA-granted exclusivities require a product to be different from previously approved FDA products in particular ways, and a product is only eligible for a priority review voucher if its active ingredient has not been previously approved in another product. We believe that a product may obtain the benefits of a platform technology designation without losing these other regulatory benefits, but it will be important to be cognizant of the various criteria for such regulatory benefits when crafting arguments supporting the leveraging of platform technology designations for new product approvals.

FDA is seeking comments on the guidance through July 29, 2024. If you may be interested in submitting a comment or a request for platform technology designation, or if you have any questions on engaging with FDA during drug or biologic product development more generally, please contact any of the authors of this alert or the Hogan Lovells attorney with whom you regularly work.

Authored by Robert Church, Michael N. Druckman, Yetunde Fadahunsi, Bryan Walsh, and Ashley Grey