Barriers to rare cancer treatment and research

Medical experts and advocacy groups are identifying systemic barriers to treatment and research for rare cancers and vulnerable patient populations.

In the United States, insurance coverage gaps have been highlighted by cases where insurers refuse to cover precision medicines for rare tumors due to a lack of established clinical guidelines or FDA-approved labels, even when specialists recommend specific drugs. This creates a barrier where scientific evidence for targeted therapies exists, but reimbursement is denied because of a lack of formal regulatory precedents.

On an international level, specialists have noted that the low incidence of rare soft tissue sarcomas, such as leiomyosarcoma, complicates clinical trial enrollment and reduces commercial investment in new drugs. Additionally, a gap in care has been identified for adolescents aged 15 to 19, who often fall into a “middle ground” between pediatric and adult oncology, resulting in lower access to specialized centers and innovative protocols compared to younger children.