Chapter 1 26 3. Due to the sensitivity of the disease, photographic confirmation of the lesion was done on a voluntary basis. We relied on the physician assessment primarily when photographic outcome confirmation was lacking. Conclusion At the time of data cut-off, 79 treatment rounds were administered via the expanded access program to 53 patients. The outcomes from the expanded access programs (subgroups) are comparable to the outcomes in the primary randomized controlled trial, where 83% (19/23, 95% CI: [60%, 94%]) healed upon pritelivir initiation. Hence, the expanded access program provides some evidence of the effectiveness of pritelivir in a broad patient population set. Nonetheless, these results have to be interpreted with caution due to the uncontrolled nature of the program, the potential impact of COVID-19 on data quality, and loss of follow-up. The large number of patients requesting re-treatment when lesions re-occurred is an important proxy measure of efficacy that could only be captured in the expanded access program, and not in the ongoing phase III trial.
RkJQdWJsaXNoZXIy MTk4NDMw