Optimizing adaptive design for Phase 2 dose-finding trials incorporating long-term success and financial considerations: A case study for neuropathic pain

Contemp Clin Trials. 2017 Jun;57:69-86. doi: 10.1016/j.cct.2017.03.011. Epub 2017 Apr 4.

Abstract

In this paper, we propose an adaptive randomization design for Phase 2 dose-finding trials to optimize Net Present Value (NPV) for an experimental drug. We replace the traditional fixed sample size design (Patel, et al., 2012) by this new design to see if NPV from the original paper can be improved. Comparison of the proposed design to the previous design is made via simulations using a hypothetical example based on a Diabetic Neuropathic Pain Study.

MeSH terms

  • Analgesics / administration & dosage*
  • Analgesics / economics
  • Analgesics / therapeutic use
  • Clinical Trials, Phase II as Topic / methods*
  • Cost-Benefit Analysis
  • Diabetic Neuropathies / drug therapy*
  • Diabetic Neuropathies / economics
  • Dose-Response Relationship, Drug
  • Endpoint Determination / methods
  • Humans
  • Models, Theoretical
  • Pain Measurement
  • Randomized Controlled Trials as Topic / methods*
  • Treatment Outcome

Substances

  • Analgesics