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APPTT

CLIN

PHARM

PTAC/CFTR
Symposium

S22--Finding an “Optimal” Dose in Drug Development: The Importance of Exposure/Response Relationships for Advancing Chronic CF Therapies

Friday, September 27, 2024
2:30 PM – 4:30 PM ET
Location:210 A-C

Recorded Session

Our comprehension of efforts needed to identify new CFTR therapies is greater at either end of the process: whether they be pre-clinical laboratory observations of in vitro activity on the front end or Phase 3 safety and efficacy studies on the back end. A less well appreciated but critical component of CF drug development lies in the middle: drug exposure and response studies to support/justify which dose(s) are brought forward for Phase 3 evaluation. Information gleaned from these studies can also assist clinicians in considering empiric dose adjustments for marketed therapies to address intolerability or ineffectiveness in individual patients.
Lessons learned from past CF exposure/response studies (when conducted) supporting current standards of CF respiratory care help inform current/future study designs. However, as we enter an era of CFTR therapeutic development for smaller CF subpopulations who are ineligible or intolerant for CFTR modulators, dose justification strategies have become more challenging, but remain critical components of safety and efficacy dossiers.

Learning Objectives:

  • Describe regulatory guidance for establishing dose-response relationships during drug development.
  • Review results of previous exposure/response studies for available chronic CF therapies.
  • Describe the rationale and benefits of empiric post-marketing dose adjustment.
  • Explain the potential and limitations of biomarkers as exposure/response endpoints in smaller study populations.

Presentations:

  • 2:30 PM – 4:30 PM ET
    S22.1- Establishing Exposure/Response Relationships During CF Drug Development

    Speaker: Tao Liu, PhD (he/him/his) – U.S. Food and Drug Administration

  • 2:30 PM – 4:30 PM ET
    S22.2- Previous Exposure/Response Observations for CF Respiratory Therapies

    Speaker: Charles R. Esther, Jr., MD, PhD – UNC

  • 2:30 PM – 4:30 PM ET
    S22.3- ETI Exposure Adjustment in the Real World: Implications for Safety & Efficacy

    Speaker: Jennifer Guimbellot – Arkansas Children's Hospital/Arkansas Children's Research Institute

  • 2:30 PM – 4:30 PM ET
    S22.4- Establishing Exposure/Response Relationships for New Respiratory Therapies

    Speaker: John Clancy, MD (he/him/his) – Cystic Fibrosis Foundation