Sarcoma's Rarity Limits Trials and Biomarker-Driven Treatment Options: Alexander Spira, MD, PhD, FACP, FASCO

The rarity of sarcoma complicates clinical trial design and biomarker-driven treatment, explains Alexander Spira, MD, PhD, FACP, FASCO, medical oncologist at Virginia Cancer Specialists and chief scientific officer of NEXT Oncology, in this interview with The American Journal of Managed Care^® (AJMC®), where he discusses sarcoma treatment and highlights ongoing gaps in physician willingness to treat the disease.

Spira notes that sarcoma is extremely rare and only affects roughly 10,000 patients annually in the US, a population further fragmented across numerous distinct subtypes. That scarcity, combined with the disease's molecular diversity, makes it difficult to design clinical trials and has slowed the broader treatment advances seen in more common cancers.

Biomarker-driven therapy remains an area of unmet need, according to Spira. While oncologists should be testing for actionable alterations such as microsatellite instability-high (MSI-H) status and other rare mutations, he notes that no biomarker-driven therapies are yet approved specifically for soft tissue sarcoma. Even so, he recommends that all patients undergo full next-generation sequencing (NGS) panels, since testing can still uncover rare, targetable mutations or identify eligibility for a relevant clinical trial, even in the absence of an approved companion therapy.

Spira also points to a persistent gap in physician comfort treating sarcoma. Because the disease is uncommon, many oncologists rarely encounter it and may be uncertain about how to proceed when they do. He emphasizes that clinicians need to recognize when to seek outside expertise, ensuring patients have access to physicians who can either manage their care directly or guide it collaboratively.

Spira's remarks raise considerations around coverage for NGS testing in a rare cancer with few approved biomarker-driven therapies, as well as the importance of referral networks and physician education in ensuring patients with sarcoma can access appropriately experienced care regardless of where they are first seen.