A sponsor-held platform IND model for personalized immunotherapy.

Abstract

e23044 Background: Despite strong biological rationale, personalized cancer immunotherapies have struggled to scale beyond clinical trials. A challenge is the mismatch between population-level drug development and therapies intended for individual patients. Additional limitations include drug-centric trial designs, fragmented regulatory oversight, and limited infrastructure for real-world evidence (RWE) generation. These factors have constrained institutional adoption. Methods: We developed a centralized clinical model in which a sponsor provides a platform IND, predefined design and safety rules, centralized pharmacovigilance, and digital infrastructure, while participating oncology centers retain responsibility for patient care. Patient-specific immunotherapies are designed based on tumor and immune profiling and manufactured by sponsor-qualified vendors. Treatment planning is guided by prospective assessment of biological relevance and safety with predefined monitoring rules. Participating institutions enroll patients under the IND and use program-provided tools for coordination, safety reporting, and data capture. Each patient-specific immunotherapy is regulated under 21 CFR 312 with sponsor-controlled release and safety oversight. Results: This model enables institutions to deliver personalized immunotherapy as a structured clinical program. Key features include: (1) site-level participation under sponsor-held IND, (2) centralized safety oversight and regulatory accountability, (3) physician-directed individualized treatment within predefined program rules, and (4) systematic generation of structured RWE suitable for regulatory review and payer engagement. The framework reduces operational burden for clinical sites and enables learning across patients and institutions without altering local standards of care. Conclusions: This platform IND model provides a scalable pathway for personalized immunotherapy within existing regulatory structures. By shifting focus from population-level development to patient-level delivery with centralized oversight, this approach aligns clinical practice, safety monitoring, and evidence generation, potentially facilitating broader adoption and supporting future regulatory and reimbursement pathways. Traditional IND(Population-Level Drug Approval) Platform IND(Personalized Oncology Care) Personalized Oncology Outcomes Population-based drug design Patient-specific therapy design Personalized care beyond standard of care Efficacy and safety assessed at the population level Biological relevance and safety assessment Personalized immunotherapy for all patients Restricted trial eligibility Individual-level eligibility RWE for coverage decisions Static treatment paradigm after approval Structured learning from care delivery Centralized safety oversight and evidence generation