smart talk

Smart Talk

October 10, 2019

Accelerated Drug Development in the Era of Breakthrough Therapies


Join representatives from SCiAN and Biocatalyst 4 Development as they explore the drug development process; from selecting the lead indication to designing/executing a trial under accelerated timelines. 

Drugs designated as "breakthrough therapies" benefit from expedited drug development and regulatory reviews.  A study may start off as a first-in-human (FiH) trial and evolve to include expansion cohorts that address phase 2 and/or phase 3 objectives, all within a single integrated protocol.  Expanded cohorts lead to rapid patient enrolment and increases the risk of patients exposed to a drug of unknown efficacy or minimally characterized toxicity profiles. To mitigate these risks, drug development processes must address challenges in trial logistics, data collection and analysis/reporting of data.  This talk walks the audience through the drug development process for a breakthrough therapy—from selecting the lead indication to designing/executing a trial under accelerated timelines.


Considerations in selecting a lead indication for oncology

  • Mechanism of action: cytotoxins, targeted drugs, immuno-oncology
  • Regulatory considerations: Fast Track, Breakthrough and Priority Review designations, Accelerated Approvals
  • Clinical development feasibility: market size, competing programs, patient availability, clinical program duration

Overview of clinical development programs in oncology

  • Traditional programs (sequential approach): Safety phase 1, followed by multiple phase 2 studies for efficacy/exploration , and 1-2 pivotal phase 3 studies
  • Accelerated programs (Integrated Protocols): First in Human (FiH) MTD safety study with expansion cohort(s) to characterize therapeutic activity / efficacy, with potential extension to phase 3
  • Bucket trials (multiple histologies), (Adaptive), seamless phase 2/3 designs

Challenges associated with accelerated programs

  • Risk - benefit assessment / risk mitigation
  • Operational challenges
  • Change to clinical trial infrastructure


3675 Market Street

Philadelphia, PA 19104

11:30am - 1:00pm