Roman Grosiberg, MD, Medical Oncologist and Director of the Sarcoma Program at Rutgers Cancer Institute of New Jersey/RWJBarnabas Health, discusses strategies for designing clinical trials with rare disease patient samples such as ones for sarcoma patients.
One strategy that Dr. Grosiberg suggests is to isolate an individual rare disease subtype of which the biological causes and symptoms are well known such as epithelioid sarcoma. Then he advises that researchers partner with institutions and get the drug they think will help the subset of patients. Afterwards, clinicians may connect with other clinicians who see sarcoma patients or patient advocacy groups in order to recruit enough participants.
A second strategy Dr. Grosiberg offers is to put patients on basket studies which identify patients with the same kind of mutations and treat them with the same drug, regardless of their specific cancer type. For instance, IDH mutations are common in chondrosarcoma patients but are not exclusive to this subset of sarcoma. In a basket study, all patients with an IDH mutation would be accepted even if not all patients have chondrosarcoma.
To learn more about sarcomas and other rare cancers, visit checkrare.com/cancers.