What the FDA Looks for in Global Development Programs Today
Multi-regional clinical trials (MRCTs) are now central to global oncology and rare disease development. Yet recent FDA decisions demonstrate that geographic reach alone does not determine regulatory success. What matters most is whether clinical evidence is applicable to U.S. patients and U.S. medical practice.
This white paper, authored by Dr. Harpreet Singh, Chief Medical Officer and former FDA Director Division of Oncology 2, examines how FDA evaluates MRCT data today — and why compelling global efficacy may still fall short without the right scientific and regulatory foundation.
Why this Whitepaper Matters
FDA’s approach to MRCTs has evolved through real-world regulatory decisions, draft guidance, and advisory committee deliberations. Across recent oncology programs, FDA has consistently emphasized:
- Applicability of trial results to U.S. patients
- Representativeness of enrolled populations
- Consistency of treatment effects across regions
- Alignment with U.S. standards of care
This white paper provides a clear, regulator-informed view of how these principles are applied in practice — and what sponsors should consider when designing global development programs.
What is Covered
- How FDA assesses applicability of MRCT data beyond geographic diversity
- Lessons from recent oncology cases, including ORIENT-11, JUPITER-02, STARGLO, and TRUST-I/II
- When single-country or heavily ex-U.S. data may be acceptable — and when it is not
- Strategic considerations for trial design, population selection, and regulatory engagement
Download to discover regulatory considerations shaping global development strategies.
Authored by a Former FDA Leader
Harpreet Singh, MD, Chief Medical Officer & Former FDA Director
Industry-leading oncologist and demonstrated medical leader in precision medicine, novel trial design and innovative regulatory initiatives designed to expedite drug approvals. Draws on nearly a decade leading oncology and rare disease programs at the FDA. Former fellow at the National Cancer Institute and frequent featured speaker at ASCO and AACR. CDER technical authority on oncologic drug and biological products for thoracic and head and neck malignancies, neurologic tumors, pediatric solid tumors and rare tumors.
