From KOLs To Patient Advocates, Using Every Tool To Fight A Rare Disease

Finding patients for trials in very rare conditions is difficult under any circumstances, and in this case the odds were really stacked high against success.

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.

Our recruiting target was 18 patients, but even that was a challenge for a condition that afflicts just one in 31,000 people. It required making full use of what few resources were available.