In what circumstances may IBAI 's GaM trial not require a phase 3 given its EAP and short life expectancy6 Oct 2025 07:00
For drugs addressing a terminal illness with a short life expectancy, the FDA can use the Accelerated Approval pathway to grant approval based on convincing Phase 2 data, potentially making a Phase 3 trial unnecessary for initial market access.
Imaging Biometrics' GaM trial, which includes an Expanded Access Program (EAP) for glioblastoma, could be a candidate for this pathway due to the disease's poor prognosis and GaM's promising early results.
Accelerated Approval for serious conditions
The most direct circumstance is the use of the FDA's Accelerated Approval pathway, which is designed for drugs that treat serious or life-threatening conditions where there is an unmet medical need. This program was created to speed up the availability of new medicines for patients with a high degree of urgency. For GaM and glioblastoma, a disease with a notoriously short life expectancy, this pathway is a strong possibility.
Key factors that allow this pathway to potentially bypass a traditional Phase 3 include:
Surrogate endpoints: Instead of waiting years for a drug to prove a direct clinical benefit like prolonged survival, the FDA can grant accelerated approval based on a surrogate endpoint. This is a marker, such as tumor shrinkage, that is reasonably likely to predict a clinical benefit.
Post-marketing studies: The approval is conditional. The company, in this case, Imaging Biometrics, must still conduct a confirmatory Phase 4 trial to verify the drug's clinical benefit after approval. The recent FDORA legislation strengthens the FDA's ability to enforce these post-marketing study requirements.
Fast Track designation: Imaging Biometrics announced in early 2024 that GaM received Fast Track designation, which is intended to expedite the review of new drugs that treat serious conditions and fill an unmet medical need.
Role of the Expanded Access Program (EAP)
The use of an EAP can also contribute to a faster review process, though it does not eliminate the need for clinical trials.
Real-world data: As seen with Imaging Biometrics' EAP for GaM, these programs collect valuable "real-world" data from a broader patient population than is typically included in early-phase trials. This data can supplement clinical trial data and inform future development.
Safety and efficacy insights: The EAP provides information on the drug's performance outside the strict, controlled environment of a clinical trial, giving regulators a better understanding of its safety profile and effectiveness in a more representative patient group.
Demonstration of unmet need: The existence of an EAP underscores the clear unmet need and dire prognosis of the patient population, strengthening the case for accelerated review.
Continued
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