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Compassionate Use Programs Or Phase IV Trials Of Innovative Molecular Targeted Drugs In Lung Cancer: Deal Or No Deal?
Published 2010 · Medicine
PHASE IV OPEN-LABELED STUDY WITH ERLOTINIB IN SOUTH-EAST ASIANS WITH NON-SMALL CELL LUNG CANCER In this month’s issue of the journal, we are faced with yet another large compassionate use program (expanded access program [EAP/phase IV trial]) with a molecular targeted drug in patients with non-small cell lung cancer (NSCLC).1 This time, Mok et al.1 present the results of a large South-East Asian patient cohort treated within an expanded access program of erlotinib for NSCLC in multiple-line situations after chemotherapy and/or radiotherapy or upfront in patients unsuitable for standard combination chemotherapy. Interestingly, 1242 patients were investigated for safety and efficacy of the treatment with the EGF-R tyrosine kinase inhibitor erlotinib in this mature analysis of the clinical trials results. The median overall survival with more than 14 months and the median progression-free survival (PFS) of nearly 6 months seen with erlotinib in South-East Asian patients were found to be significantly better than the results observed in the parallel TRUST program with erlotinib in white/non-Asian patients reported in the same issue of this journal.2 The overall safety profile of drug administration among South-East Asian patients was excellent and did not show any specific toxicities and does not imply any note of caution as serious toxicities were registered extremely rare with this agent—especially when considering the poor prognostic groups selected here with multiple prior treatments. Probably based on learning effects, only 14% of patient had to be dose reduced in this EAP comparable with what had already been observed with other EGF-R tyrosine kinase inhibitor such as gefitinib and vandetanib in large phase II, phase III studies, or phase IV trials.3–10 Thus, both toxicity profiles and efficacy results are in line to what has already been reported for the even larger EAP for gefitinib performed from 2002 to 2007.6