Clinical trial design

Clinical trial design is a key aspect of the successful conduct of clinical trials. It involves deciding parameters such as the number of patients, the length of the trial, the comparator arm (in the case of randomized controlled trials) using information on current treatments, the anticipated effect of the therapeutic intervention being studied and applying biostatistics methods.


Latest Research and Reviews

News and Comment

  • Comments and Opinion |

    With only a limited number of clinical trials of artificial intelligence in medicine thus far, the first guidelines for protocols and reporting arrive at an opportune time. Better protocol design, along with consistent and complete data presentation, will greatly facilitate interpretation and validation of these trials, and will help the field to move forward.

    • Eric J. Topol
    Nature Medicine 26, 1318-1320
  • News and Views |

    Liver biopsy sample evaluation is an essential part of clinical studies in nonalcoholic steatohepatitis (NASH) and is key in excluding confounding morbidities. Current scoring systems, which are decisive for study inclusion, rely on imprecisely defined histological features, leading to a high observer variability of disease categorization. In this News & Views, measures to overcome these limitations are discussed.

    • Thomas Longerich
    •  & Peter Schirmacher
  • Comments and Opinion |

    Single-arm phase II trials can provide compelling results that facilitate the approval of a new therapy. Designing and interpreting single-arm studies based on four principles — instinct, comparative analysis, statistical soundness and like-for-like comparisons — can provide indications as to which drugs are most likely to provide improved therapeutic options for patients.

    • Robert H. Glassman
    • , Grace Kim
    •  & Marc J. Kahn