Clinical Research: Phase 1 - Phase 4

Rare Disease Studies: How to Run Successful Programs


Patricia Nowowieski, Head of Global Clinical Operations for Alexion Pharmaceuticals.

Patricia’s work in global clinical trials involves specialized populations and complex logistical and regulatory challenges.

Angi Robinson, Executive Director and Scientific Account Leader for Premier Research.

Angi’s extensive experience includes oversight of global studies in pediatrics and rare diseases.

Mallory Bissett, Associate Director of Global Clinical Operations for Alexion Pharmaceuticals.

Mallory focuses solely on rare diseases, including late-stage, ultra-rare disease products.

Topics to be addressed in this webinar include:

  • Program design — understanding natural history, reducing sources of bias, and validating endpoints.
  • Protocol design — reducing the need for protocol amendments.
  • Patient recruitment and retention — determining the number of study sites, managing patient travel, incorporating international patients, and knowing what questions to expect.
  • Preparing for site inspections by the FDA.