Clinical Research: Phase 1 - Phase 4

Premier Insight 267: From KOLs to Patient Advocates, Using Every Tool to Fight a Rare Disease

The indication: a basal cell carcinoma known as Gorlin syndrome, a disease that manifests itself in large numbers of benign tumors that grow on various parts of the body. The sponsor: a very small, virtual company consisting of a handful of contractors pursuing success in the form of a topical gel, which had only ever been tried as an oral drug in the past. This had demonstrated several undesirable side effects for patients as an oral medication, and the only other alternative available was to undergo numerous surgeries to physically remove the tumors.