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

  • 11759

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    P2.11-014 - Biomarkers reduce clinical trial risk and the cost of drug development in non-small cell lung cancer (NSCLC) (ID 1525)

    09:30 - 09:30  |  Author(s): A. Falconi
    • Abstract

    Background
    We evaluated the risk of clinical trial failure and the cost of drug development for patients with NSCLC between 1998 and 2012

    Methods
    For assessment of drug development we used trial disclosures from publically available resources, such as clinicaltrials.gov and others. Compounds were excluded from the analysis if they began phase I clinical testing before 1998 and if they did not use clinically significant, treatment-relevant endpoints, such as response rate, progression-free and overall survival. Analysis was conducted in regards to treatment indication, compound classification and mechanism of action. A compound that had completed a phase I clinical trial was classified as successful if there was an ongoing, completed, or currently recruiting phase II clinical trial found. A compound that had completed a phase II clinical trial was similarly classified as successful if there was an ongoing, completed or currently recruiting phase III found. A compound that had completed a phase III clinical trial was classified as successful if there was an official FDA approved indication in our study population. In addition, a phase I/II trial was classified as a phase II trial and a phase II/III trial was classified as a phase III trial for analysis purposes. Follow up was completed until January 1, 2012. Costs of clinical drug development for advanced NSCLC were calculated using industry data and assumptions, a 9% yearly discount rate and assuming a clinical trial length of 2.5 years for phase I trials, 4 years for phase II trials, 5 years for phase III trials and an average of 5 phase I trials, 7 phase II trials, and 4 phase III trials per approved drug. All funding costs are in US dollars (USD).

    Results
    2,407 clinical trials met search criteria. 676 trials and 199 unique compounds met our inclusion criteria. The likelihood, or cumulative clinical trial success rate, that a new drug would pass all phases of clinical testing and be approved was found to be 11% (less than the expected industry aggregate rates of 16.5%). The biggest obstacle for approval was the success of phase III trials: success rate was only 28%. Biomarker-guided targeted therapies (with a success rate of 62%) and receptor targeted therapies (with a success rate of 31%) were found to have the highest likelihood of success in clinical trials. The risk-adjusted cost for NSCLC clinical drug development with biomarkers was US$1.4 billion, a 26% reduction when compared to overall lung cancer drug development costs of US$1.89 billion, which is within industry expectations. Potential savings may be even higher if fewer clinical trials are required for successful development.

    Conclusion
    Physicians that enroll patients in NSCLC trials should prioritize their participation in programs that involve either a biomarker or receptor targeted therapy, which appear to carry the best chances for a successful treatment response. Given the high adjusted cost of clinical testing alone in NSCLC, efforts to mitigate the risk of trial failure need to explore these factors more fully.