Author of

• 11745

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  P2.11 - Poster Session 2 - NSCLC Novel Therapies (ID 209)

  • Event: WCLC 2013
  • Type: Poster Session
  • Track: Medical Oncology
  • Presentations: 1
  • Moderators:
  • Coordinates: 10/29/2013, 09:30 - 16:30, Exhibit Hall, Ground Level

  • 11791

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    P2.11-046 - A Novel Approach to Increase the Efficiency of Survival-Endpoint<br /> Phase II Trials. (ID 3153)

    09:30 - 09:30  |  Author(s): G. Han
    • Abstract

    Background
    Lung cancer is the second most common cancer in United States. However, new molecular information whether arising from identification of key mutations, or Next Gen Sequencing (NGS) has lead to the fragmentation of this common disease into multiple and often very rare molecular subtypes. Even though this has enabled us to substantially improve survival in specific molecular subtypes with targeted agents, having sufficient numbers of patients to demonstrate the effectiveness of novel targeted therapies for each subtype has therefore become challenging, essentially impeding progress.

    Methods
    Use of sample size determination software allows for comparison of phase II trials with a target survival proportion at a given time T based upon Kaplan-Meier (KM) versus exponential (E) survival methods. This comparison was made in instances where the exponential distribution assumption is valid.

    Results
    The use of exponential survival fitting methods, in lieu of the currently implemented KM trial designs, to single arm phase II studies can routinely reduce patient numbers by 25-40% (Table) without compromising the statistical power. Another substantial advantage is that the variability in the survival information for the exponential fit estimate both before and after the median survival points is reduced. Since patient accrual is staggered ,this reduced variability allows for obtaining robust survival information with shorter follow up times T. Any additional follow-up beyond T only further improves the E estimate, but not the KM estimate. Thus, compared to the KM approach, the E approach will allow us to do single arm phase II trials with smaller numbers of patients without compromising statistical power. Robust survival estimates can also be achieved with shorter follow up periods. Figure 1

    Conclusion
    When conducting single arm phase II clinical trials with rare molecular entities of lung cancer, the exponential survival fitting method could replace the current standard approach. Robust survival information can be obtained with smaller numbers of patients and with shorter survival times. Detailed examples and analysis will be presented.