Abstract A017: Retrospective descriptive analysis of FDA CDER and CBER drug development tool qualification programs in oncology and hematology: Implications for orphan drug submissions at the Saudi Food & Drug Authority

Abstract Purpose: This study aims to retrospectively analyze the qualification process of the US FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) Drug Development Tool (DDT) Qualification Programs, with a focus on investigational new drug applications in oncology and hematology. The objective is to identify trends and key factors in biomarker and clinical outcome assessment qualifications that could inform and support orphan drug regulatory submissions at the Saudi Food & Drug Authority (SFDA). Methods: A retrospective descriptive analysis was conducted using publicly available data from the FDA’s DDT Qualification Programs. Projects included biomarker (BMQ) and clinical outcome assessment (COA) submissions related to oncology and hematology. Submissions were categorized by cancer type, therapeutic area, biomarker or COA type, qualification status, and context of use. Key patterns and outcomes were reviewed to assess factors associated with successful qualification and to inform regulatory strategies for orphan drugs. Results: The analysis included 13 biomarker projects and 6 clinical outcome assessment projects submitted between 2011 and 2024. Most biomarker submissions aimed to support pharmacodynamic or diagnostic purposes, using molecular or imaging modalities. COA submissions primarily involved patient-reported outcomes related to symptom severity and treatment impact. A limited number of tools achieved full qualification, while others were withdrawn, delayed, or issued Letters of Support without formal qualification. The data highlighted the importance of a clearly defined context of use, robust validation plans, and early engagement with regulatory authorities. These insights offer a basis for enhancing SFDA’s approach to evaluating similar tools in rare disease settings. Conclusions: While the FDA’s DDT Qualification Program provides a structured framework to support regulatory decision-making in drug development, qualification remains a resource-intensive process with variable timelines and outcomes. Lessons learned from this retrospective analysis can guide the SFDA in strengthening its regulatory support for investigational and orphan drug development. Integrating these insights can streamline IND submissions, promote the use of innovative tools in clinical trials, and ultimately improve access to novel therapies for patients in Saudi Arabia. Citation Format: Meshal A. Al-Kalabi, Tariq H. Alqurayshah. Retrospective descriptive analysis of FDA CDER and CBER drug development tool qualification programs in oncology and hematology: Implications for orphan drug submissions at the Saudi Food & Drug Authority [abstract]. In: Proceedings of the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics; 2025 Oct 22-26; Boston, MA. Philadelphia (PA): AACR; Mol Cancer Ther 2025;24(10 Suppl):Abstract nr A017.