Review Article

Innovation in Oncology Drug Development

Figure 4

FIH Protocol (PN001) for pembrolizumab—Multiple Expansion Cohorts. Illustration Merck FIH pembrolizumab clinical development program. The initial IND submitted in 2010 was to enroll 18 patients with melanoma plus 14 additional patients in an EC with melanoma and renal cell cancer. Over the next 2.5 years, 8 protocol amendments were filed, enabling the FIH study to be expanded to 9 distinct ECs enrolling a planned 1,100 patients [2123]. Successful implementation of this strategy resulted in accelerated approval for refractory, unresectable or metastatic melanoma and was supportive for approval in NSCLC.