Results from expanded access programs: a review of academic literature 157 7♦ Comparison with other data When comparing the expanded access program with clinical phase II and III studies, the authors conclude that: • ‘The relatively liberal entry criteria for this expanded access study allowed treatment of 3 patient groups not eligible or available for previous vemurafenib trials: (1) patients with poor PS (ECOG PS 2 and 3), (2) patients with previously treated brain metastases, and (3) patients previously treated with ipilimumab.’ • ‘The efficacy of vemurafenib has been remarkably consistent in the major studies reported to date. In the phase II study (BRIM-2) in which patients with previously treated BRAFV600- mutant metastatic melanoma received vemurafenib, a confirmed ORR of 53% was reported.' • 'In the pivotal phase III (BRIM-3) study, the confirmed ORR with vemurafenib therapy was 48% in patients with previously untreated metastatic melanoma, compared with 5% for dacarbazine.’' • ‘The documentation of a 42% ORR in patients with poor PS is arguably the most important clinical finding derived from this study. In the phases II and III trials of vemurafenib, such patients were excluded’ Conclusion The authors conclude that: ‘Despite limitations due to the nonrandomized design and short treatment duration, the results of this expanded access study confirm the previously reported rapid and high tumor response rate achieved by vemurafenib in the treatment of metastatic BRAFV600 mutation– positive melanoma. In addition, this study provides new data suggesting the efficacy of vemurafenib in several groups of patients with metastatic melanoma not addressed in previous trials.’ The authors do not provide specific suggestions for future studies.
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