The pharmaceutical industry, while generally supportive of the US Food and Drug Administration’s (FDA) draft guidance on its platform technology designation program, called for greater clarity on the designation process, review timelines, and eligibility criteria.
This draft guidance, which was issued in May 2024, provided details on the program, which can be used by drug sponsors to manufacture more than one drug or biological product using a standardized production or manufacturing process.
The program formalizes the existing pathway for getting products made on novel platforms approved. Comments were due on 28 August.
The Biotechnology Industry Organization (BIO) said it “welcomes” the guidance, which is a “first step toward implementation of this promising program.” The group added, “We strongly believe the program could provide opportunities for early and continuous engagement with the Agency that will ultimately streamline drug development.”
Yet BIO took issue with several elements of the guidance and called for greater flexibility for leveraging platform knowledge and submitting requests for designation.
The guidance states that FDA “recommends requesting the designation of a platform technology during the [investigational new drug] IND phase of drug development for a planned subsequent [new drug application] NDA or 351(a) [biologics license application] BLA, because by this stage of development, the sponsors should have sufficient knowledge to outline for FDA how the proposed platform technology would meet the eligibility factors as outlined in section 506K(b) of the [Federal Food, Drug, and Cosmetic Act] FD&C Act.”
The draft guideline, said BIO, “Sets the expectation that the designation request is submitted during the IND phase, but that the information is leveraged only at the NDA or BLA stage, which is unnecessarily restrictive.”
BIO adds that “platform knowledge can be applied at any point in a product’s lifecycle. Rather than specifying a specific stage for submitting this designation request, FDA could focus on availability of data and ensuring adequate product-specific data.”
BIO requests shorter reviews
BIO also suggested that FDA take up to 60 days, rather than the 90 days proposed in the guidance, to review designation requests.
“A 90-day review timeline could substantially delay key development decisions, for example, whether redundant tests could be eliminated due to receiving a PTD. We suggest that a timeline of 60 days would be more appropriate to mitigate such delays and would be in line with other existing determination timeframes.”
BIO, ISPE want delivery devices included, clarity on program benefits
Both BIO and the International Society for Pharmaceutical Engineering (ISPE) suggested that device makers should also be eligible to participate in the platform designation program.
BIO states that “the definition of a platform technology is only focused on drugs” but suggested that delivery devices should also be eligible for inclusion.
ISPE said that it “considers that the guidance would benefit from allowing platform technology designation to have broader eligibility criteria, for example, to cover devices and a greater range of small and “more conventional” technologies, such as monoclonal antibody biologic product technologies.”
BIO and ISPE also suggested that the guidance expand on the benefits of requesting a platform technology designation. BIO said in its comments that “more information is needed to fully understand the upside of utilizing this designation in development, registration, and post-approval.”
BIO recommended adding potential benefits including supporting comparability process/scale changes, supporting process performance qualification (PPQ) stability studies, process characterization, and designating active pharmaceutical ingredient (API) starting materials.
ISPE said that “it would also be helpful if the guidance could be clearer on what the benefits of designation would produce.”
Regeneron wants clarity on platform approaches for biologics
Biologics maker Regeneron wanted FDA to clarify how platform data can be leveraged for BLA products.
The guidance states that “sponsors of NDAs can leverage platform technology information from other applications submitted by the same sponsor using the cross-reference mechanism. However, BLA sponsors seeking to leverage data and information from a platform technology in a prior application should include the full information in their subsequent application.”
“Although Regeneron understands that certain statutory requirements preclude the cross-referencing of data in the BLA, Regeneron recommends that FDA provide guidance as to how the leveraged PT data may be presented in a BLA to clearly identify the content as derived from a formal PTD program,” the drugmaker wrote.
ISPE further noted not treating BLAs and NDAs differently and that both types of products should be able use the same cross-reference mechanism.
ISPE states that “sponsors with a platform technology designation should be able to leverage prior information through the same cross-reference mechanism for both NDAs and BLAs owned by the same sponsor.”
NORD requests additional clarity on eligibility criteria
In its comments, the National Organization for Rare Disorders (NORD) requested FDA provide greater clarity on its eligibility criteria for platform designation.
The guidance states that “Under section 506K(h)(1) of the FD&C Act, a platform technology is 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.”
NORD also points out that “what exactly the agency considers ‘well-understood and reproducible technology,’ significant efficiencies,’ or ‘developed and reviewed in a more streamlined manner’ may be variable or subject to interpretation, in particular for nascent areas of rare disease drug development.”
NORD also suggested that FDA include more information on patient engagement and include more patient perspectives.
“NORD understands and appreciates the guidance’s intentional focus on increasing efficiencies for FDA review and medical product development. However, patients and caregivers play a number of vital, unique, and outsized roles in rare disease drug development. It is deeply concerning that the word ‘patient’ is not mentioned once in the 18-page draft guidance.”
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