Efficient Early Drug Development for Ulcerative Colitis.

69 70 71 72 73 74 75 76 77 78 79 80 81 82 83 84 85 86 87 88 89 90 91 92 93 94 95 96 97 98 99 100 101 102 103 104 Aapproved for the treatment of ulcerative colitis (UC), new agents are required for patients who are unresponsive to conventional management (Supplementary Appendix 1). Moreover, some patients who have responded to currently available therapies may benefit from improved approaches to treatment. Although innovative therapies hold promise, important barriers to development programs are recognizable. First, the cost of drug development continues to increase, which restricts the number of new therapies that can be evaluated. Second, limited numbers of patients are available for enrollment into competing programs. Third, current study designs that mandate multiple arms for dose finding require large patient numbers and are time consuming. Fourth, the regulatory environment has become increasing complex. Finally, new technologies have led to evolution of highly rigorous trial endpoints. Consequently, fewer drugs successfully complete the registration process than in the past. Because regulatory and patientrelated factors are unlikely to change, new strategies for drug development are needed. Accordingly, trial design must evolve to detect efficacy signals with limited resources. In this article, we describe a novel approach to early drug development in UC.