Case Study Cardiovascular Disease

The global head of clinical development of a mid-size pharmaceutical company hired L&C to:

  • Provision of a highly comprehensive and objective data analysis for definition of the key study design parameters of a phase II/III program in a cardiovascular disease:
    • increment of the patient population for the respective indication in order to gain the broadest possible label
  • Deliver a fact-based, objective basis for discussions with internal stakeholders and external experts (KOL, regulators, market access)
Case Study V – Study Design Optimization

Method

  • Assessment of 80 phase II/III studies
  • Calculation of a possible correlation between all demographics or inclusion/exclusion criteria and study outcome: numerous endpoints and side effects
    (see anonymized example on the left)
  • Systematic assessment of potentially co-founding factors with reference to all underlying data

Results

  • Quantitative assessment of the impact of past inclusion/exclusion criteria and demographics on study outcome (safety, efficacy):
    • > 50 inclusion/exclusion criteria
    • > 40 demographics analyzed
  • Fact-based and quantifiable arguments for industry/government authorities and key opinion leaders

Key Benefits

  • Revised FDA opinion regarding one inclusion criterion, leading to broader potential label
  • Change of several inclusion/exclusion criteria, selection of a different comparator for a future study
  • Definition of the scientific aspects of the target product profile
  • Basis for the conception of the market access strategy