Journal: Clinical cancer research : an official journal of the American Association for Cancer Research
This publication is a concise report from an FDA Oncology Center of Excellence mini‑symposium held in May 2023, focused on clinical trial design for relapsed and refractory osteosarcoma in children and adolescents.
Key points:
- Clinical context:
- Osteosarcoma is the most common primary malignant bone tumor in children and adolescents, peaking around puberty.
- Around 10%–20% present with metastatic disease, most often in the lungs.
- With current multimodality therapy, 5‑year overall survival is ~70% for localized disease but only ~30% for metastatic or recurrent disease in the pediatric population.
- There is no standard of care or approved therapy for relapsed/refractory disease.
- Barriers to progress:
- Complex biology and the rarity of osteosarcoma limit drug development.
- Conventional clinical trial endpoints, especially objective response rate, are unreliable indicators of meaningful drug activity in this setting.
- Trial design and implementation in this rare population are logistically and methodologically challenging.
- Symposium focus:
- Brought together patient advocates, FDA regulators, and pediatric sarcoma experts to review current obstacles and discuss how to improve trial design.
- Emphasized the need for endpoints and study designs that better capture clinical benefit and are feasible in small, heterogeneous populations.
- Main conclusion:
- Moving the field forward will require deliberate, sustained collaboration among all stakeholders—patients, regulators, and investigators—to create trials that are patient-acceptable and capable of generating evidence robust enough to support future marketing applications.
- The article summarizes the core themes and outlines future directions discussed at the meeting, aiming to guide more effective trial strategies in relapsed/refractory osteosarcoma.