Analysis of regulatory and scientific activities of SFDA oncology and hematology section in sixteen months

Introduction Amid the global acceleration of the clinical development of drugs and biologics in oncology and hematology, the Saudi Food and Drug Authority (SFDA) established the Oncology and Hematology (OH) Section to evaluate the efficacy and safety of those drugs. We describe here the OH Section role in advancement of oncology-related regulatory science and policy in Saudi Arabia. Methodology We summarize scientific and clinical activities related to the OH efficacy and safety led by OH section's experts from July 2024-October 2025 using the OH excel database. Results The SFDA established OH section in July 2024 to evaluate the clinical efficacy and safety of applications of new clinical trials, new OH drugs and biologics and new clinical variations. The section is responsible for the expedited clinical review and designation of orphan drugs in rare OH diseases, breakthrough medicines in high unmet medical need in OH, fast-track and accelerated OH approvals based on validated surrogate endpoints. The OH section presented the oncology efficacy and safety considerations for OH approvals in Saudi Arabia including indications that received expedited review (based on validated surrogate endpoints) or regular review based on evident clinical benefits such as overall survival and patient reported outcomes. Since October 2024, experts in OH section participated with five abstracts in two AACR meetings. The section, experts also are participating in the development of guidelines and recommendations as follows: Early-phase clinical development of CAR T cells for patients with hematologic malignancies Biomarker and tissue agnostic oncology drug development Patient-Reported Outcomes (PROs) Regulatory considerations for use of minimal residual disease (MRD) for treatment hematologic malignancies Development of products in rare cancers and pediatrics Development of products in sickle cell disease Gene therapy in hematological diseases Also, the experts were qualified to join national and international relevant committees and working groups: ICH working group: E22 General Considerations for Patient Preference Studies The National Scientific Committee in the Saudi Cancer Institute Moreover, the OH section was responsible for The clinical development of the first in-house chimeric antigen receptor T-cells (CAR-T) in Saudi Arabia for acute lymphoblastic leukemia (ALL). Positive efficacy and safety opinions granted for at least 10 OH indications as first regulatory drug authority ever. First global regulatory approval for mitapivat in thalassemia Conclusion Since the formal establishment of the OH section in July 2024, OH experts contributed in hundreds of scientific and regulatory activities related to for OH products including developing clinical guidelines, evaluating the safety and efficacy, and facilitating the timely introduction of OH products and indications.