Data collection in expanded access: real-world examples 25 The vast majority of requests 65% (79/122, 95% CI: [56%, 73%]) initiated treatment with pritelivir. These requests concerned 53 unique patients. Requests were received from 11 different countries across Europe, North America, and Africa. Safety Zero (0) serious adverse events that were drug-related were reported through the program. Efficacy The efficacy of all treatment cycles (including re-treatments) can be seen below (N=69) in Table 1. The high percentage of patients that re-initiated treatments when novel lesions appeared (36%) could be seen as a surrogate indicator of response. Table 1: Binary efficacy outcomes for pritelivir for all evaluable treatment cycles. Variable N = 691 95% CI2 Healed after 28-day course (cumulative) 37 (54%) [41%, 66%] Healed after 42-day course (cumulative) 42 (61%) [48%, 72%] 1n (%), 2CI = Confidence Interval Furthermore, we were also interested in the outcomes of patients that were naïve to pritelivir (i.e., the ‘trial’ population. Hence, we removed all patients that were re-treated in the EAP (N=23), and we removed patients that were treated in the EAP once but were also treated in the trial (N=4). For all naïve patients (N=69 – 23 – 4 =42), the cumulative outcomes are provided in Table 2. Table 2: Binary efficacy outcomes for pritelivir for all unique patients that were naïve to pritelivir. Variable N N = 421 95% CI2 Healed after 28-day course (cumulative) 42 21 (50%) [36%, 64%] Healed after 42-day course (cumulative) 42 25 (60%) [43%, 74%] 1n (%),2CI = Confidence Interval Limitations 1. Due to COVID, patients could not go to the hospital and lesion assessments were made through telephone visit confirmation via the physician, patient self-assessed lesion healing, or sent-through photographs made by patients or family members 2. Although ‘perfect’ trial patients may have exact squared or circles lesions, we faced a number of patients where the size of lesions was nearly impossible to measure, or certainly not through width times height. We relied on the physician assessment whether or not lesions were healed when exact measurements were unavailable.
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