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OpportunityAnalyzer: Acute Myeloid Leukemia (AML) - Opportunity Analysis and Forecasts to 2017

7.2.6 Unmet Need: Guidelines Directing the Optimum Course of Therapy 123
7.2.7 Unmet Need: Therapies with More Convenient Dosing Regimens 123
7.2.8 Unmet Need: Lower Cost of Therapy 124
7.3 Opportunity Analysis 124
7.3.1 Opportunity: Targeting Minimal Residual Disease (Leukemic Stem Cells) 124
7.3.2 Opportunity: Targeting Driver Molecular Mutations with Combination Therapy 125
7.3.3 Opportunity: Development of Immunotherapies for AML 126
7.3.4 Opportunity: Differentiation Agents 126
7.3.5 Opportunity: Development of Oral Maintenance Therapies 127
8 R&D Strategies 128
8.1 Overview 128
8.1.1 Targeting Multiple Patient Segments 128
8.1.2 Developing Novel Drugs in Combination with Established Therapies 129
8.1.3 Targeting Elderly AML Patients 130
8.1.4 Investigating New Targets Involved in the Pathogenesis of AML 130
8.1.5 Alliances with Academic Groups 131
8.2 Clinical Trial Design 133
8.2.1 Selection of the Appropriate Efficacy Endpoints to Support Regulatory Approval 133
8.2.2 Selection of Active Comparator 136
8.2.3 Randomization and Stratification of Patients 137
8.2.4 Current Clinical Trial Design 139
8.2.5 Future Trends in Clinical Trial Design 141
9 Pipeline Assessment 144
9.1 Overview 144
9.2 Promising Drugs in Clinical Development 145
9.2.1 CC-486 (oral azacitidine) 146
9.2.2 CPX-351 149
9.2.3 Midostaurin 154
9.2.4 Quizartinib 158
9.2.5 Sapacitabine 164
9.2.6 Volasertib 168
9.2.7 Vosaroxin 172
9.3 Innovative Early-stage Approaches 176
9.3.1 Biologic-Targeted Therapy 177
9.3.2 Immunotherapies 179
9.3.3 Epigenetic Approaches 181
10 Pipeline Valuation Analysis 183
10.1 Clinical Benchmark of Key Pipeline Drugs 184
10.2 Commercial Benchmark of Key Pipeline Drugs 187
10.3 Competitive Asse
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