165
Oral Formulation Roadmap from Early Drug Discovery to Development,
First Edition. Edited by Elizabeth Kwong.
© 2017 John Wiley & Sons, Inc. Published 2017 by John Wiley & Sons, Inc.
7
Formulation, Analytical, and Regulatory Strategies
for First‐in‐Human Clinical Trials
Lorenzo Capretto
*
, Gerard Byrne
*
, Sarah Trenfield, Lee Dowden
and Steven Booth
Merck Sharpe & Dohme, Hoddesdon, Hertfordshire, UK
*
These authors contributed equally to this work.
7.1 Introduction, 166
7.2 Planning and Executing the FIH Trial, 167
7.2.1 Study Population, 169
7.2.2 Site and Investigator Selection, 170
7.2.3 FIH Trial Design, 171
7.2.3.1 Initial Dose Determination, 172
7.2.3.2 Dose Increment and Escalation Strategies, 173
7.2.3.3 Blinding, 174
7.2.3.4 Single Ascending Dose (SAD) Studies, 175
7.2.3.5 Multiple Ascending Dose (MAD) Studies, 177
7.2.3.6 Multi‐Part Flexible Trial Designs, 177
7.2.4 Oncology‐Specific FIH Trial Designs, 177
7.2.4.1 Rule‐Based Designs, 179
7.2.4.2 Model‐Based Designs, 181
7.2.5 Other Considerations, 181
7.2.6 Sample Acquisition and Data Analysis, 182
7.2.7 Clinical Trial Completion, 183
7.3 Formulation Development, 183
7.3.1 Fit‐for‐Purpose versus Commercial Approach, 184
7.3.2 On‐Site Manufactured Clinical Trial Materials, 187
7.3.3 Solubility, 189
7.3.3.1 Conventional Formulations, 190
7.3.3.2 Enabled Formulations, 190
7.3.4 Trial Design Considerations, 191
7.3.4.1 Placebo Controlled and Blinding, 192