Trump Administration’s Impact on FDA Regulations: 5 Key Changes for Pharma & Biotech in 2025
Published Jan 28, 2025
FDA Guidance Radar: 4 Topics to Watch in 2025
Published 30th January 2025
This year, the Food and Drug Administration (FDA) has an ambitious and exciting agenda for draft and finalised guidance documents. On the Agency’s current trajectory, we can anticipate more detail on guidance documents. Focus areas include, accelerated approvals, expectations for laboratory developed tests, in vitro diagnostics at large, and requests for feedback on medical device submissions. This blog will summarise these key areas to watch for in CDER, CBER, and CDRH, in 2025.
An evolving area for CBER and CDER
During Trump’s first term, shortening times for FDA approval processes was a clear objective. Therefore, the accelerated approval pathway will be an important regulatory area to watch for within the Center for Biologics Evaluation and Research (CBER) and the Center for Drug Evaluation and Research (CDER) in 2025.
The accelerated approval pathway allows sponsors to gain approval for products intended to treat an unmet medical need. This pathway is an alternative and faster route compared to traditional approval. Sponsors must meet certain criteria and undertake confirmatory trials to verify surrogate endpoints. Examples of these endpoints include an effect on irreversible morbidity and mortality (IMM) as well as other clinical benefits. Once the clinical benefits of the product have been confirmed, the FDA considers the requirements to have been met. However, if the Sponsor fails to conduct a confirmatory trial with due diligence or the endpoints are not verified (in addition to other reasons), the FDA can withdraw this accelerated approval.
Updates to the Accelerated Approval Pathway
December 2024’s draft guidance updates the previous 2014 Expedited Programs guidance. The 2014 document encapsulated a broader scope of all expedited programs such as breakthrough therapy designation and fast track designation. The new draft guidance focusses solely on the accelerated approval pathway.
FDA Interactions
Firstly, compared to the 2014 guidance, the new draft guidance emphasises the importance of early consultation with the FDA to discuss and develop endpoints. Sponsors are able to request a Type C Meeting with the FDA, submit a Rare Disease Endpoint Advancement Pilot Program Proposal or have a consultation with FDA Review Teams. DLRC are experienced and well-equipped to assist Sponsors during these vital interactions with the FDA.
Confirmatory Trial Guidance
2014 guidance states that Sponsors must conduct confirmatory trials post-approval and with due diligence. The new draft guidance expands on the requirements for confirmatory trials. In January 2025, the publication of an additional draft guidance built upon determining whether a confirmatory trial is underway. Both the draft guidance documents note that confirmatory trials may be expected to be underway before accelerated approval is granted (or initiated within a specified time post-approval). Additionally, sponsors must report progress updates to the FDA approximately every 180 days.
Expedited Withdrawals
The 2024 draft guidance also introduces an expedited withdrawal of drugs approved under this pathway. This expedited withdrawal process aims to remove ineffective accelerated approval products from the market more efficiently.
Additional Initiatives for Accelerating Approvals
Throughout 2025 CDER and CBER are aiming to finalise draft guidance related to the accelerated approval of oncology therapeutics. These Centres also plan to issue further relevant guidance, including the “Accelerated Approval of Human Gene Therapy Products for Rare Diseases; Draft Guidance for Industry”. In line with this focus, CBER’s pilot programs, START and CoGenT, aim to increase the efficiency of the regulatory process and pace of development.
A Packed Agenda for the CDRH
FDA’s Center for Devices and Radiological Health (CDRH) is aiming to issue 18 guidance documents in FY 2025. These guidance dcuments stand to have a profound impact on the medical device industry. We’ll take you through some of the key topics to look out for:
Laboratory Developed Tests Guidance
Laboratory Developed Tests (LDTs) are in vitro diagnostic products (IVDs). LDTs are intended for clinical use and are designed, manufactured, and used within a single CLIA-certified laboratory. Historically, the Agency has distinguished LDTs from non-laboratory manufactured IVDs. In practice, LDTs were generally exempt from the measures imposed on medical devices. A May 2024 final rule, signalled a major change in the Agency’s approach. The rule made explicit that LDTs are considered devices. The Agency will phase out its longstanding practice of general enforcement discretion to ensure LDTs comply with applicable regulatory requirements.
The rule outlines a five-stage, four-year plan to increase FDA oversight of LDTs. This rule has a more targeted enforcement discretion to help maintain access to certain safe and effective LDTs that do not fully comply with the Agency’s requirements. From 6 May, regulators will expect most LDTs to comply with certain reporting requirements. This includes Medical Device Reporting (21 CFR 803) and Reports of Corrections and Removals (21 CFR 806), as well as requirements for processing complaints under 21 CFR 820.198.
On the Agency’s current trajectory, we should expect new guidance to further elucidate the changing approach to LDTs. This includes “Laboratory Developed Tests: Enforcement Discretion Policy Regarding Special Controls” and “Enforcement Discretion Policy for Certain Laboratory Developed Tests for Unmet Needs: Frequently Asked Question.” That said, the Agency’s LDT agenda faces strong headwinds from industry. Potential challenges include ongoing lawsuits from the ACLA and AMP as well as the incoming Administration. We still need to see if, when, and how the current trajectory for LDTs will change.
In vitro Diagnostics – at Large
The Agency’s final rule aims to regulate IVDs more consistently in the long term. This applies regardless of whether a laboratory manufactures them as LDTs. As part of this initiative, we should expect further insight into Agency expectations for IVDs at large. New guidance on “In vitro Diagnostics: Labeling” is designated a priority item for FY 2025. The Agency’s final rule puts an added time pressure on the issuance of this guidance: compliance with applicable labelling requirements is expected for all IVDs—including those manufactured as LDTs—from 6 May 2026, as part of stage 2.
Ultimately, by stage 5 of the plan (May 2028 onwards), the Agency would expect most IVDs—including those manufactured as LDTs—to comply with premarket review requirements congruent with the devices’ level of risk. Of note, the Agency’s approach to risk classification for IVDs is evolving. An initiative was launched at CDRH in January 2024 to reclassify most Class III (e.g. high risk) IVDs to Class II (e.g. moderate risk). There are special controls for safety and efficacy with an anticipated focus on infectious disease and companion diagnostic IVDs.
Classification of a device as moderate rather than high risk has profound implications on regulatory strategy. The route to market via a de novo or 510(k) submission is relatively quicker and less burdensome than premarket approval (PMA). The reclassification process, however, is ongoing with the Agency currently forecasting a November 2027 completion date.
Q-Submission Program Guidance
The Q-Submission program is the forum for formal advice and feedback from the Agency on submissions for medical devices, including IVDs. Developers should be aware that guidance on this program is currently being revised. New draft guidance was distributed for comment March 2024 and provides more detail and clarification on Agency expectations for meetings. Final guidance on this topic is a priority item for CDRH in FY 2025.
Summary
As the regulatory landscape for medicines and medical devices continues to shift and evolve in the US, it is clear that focused discussions with the Agency to de-risk and expedite product development are more essential than ever. With extensive experience in successfully navigating Agency interactions, DLRC is well equipped to be a strategic partner from concept to market.
