Real-World Science and Late Phase

The Expanding Role of RWE in Rare Studies

Real-world evidence is increasingly important in drug development as the medical and pharma communities recognize the data’s potential for predicting the benefits of treatment interventions. Accelerating this trend is the Food and Drug Administration’s growing understanding of how RWE has helped researchers better understand and use COVID-19 treatments.

While placebo-controlled trials continue to have a place in evaluating new therapies, many sponsors, regulators, physicians, payers, and patients recognize that an alternative approach may involve using a synthetic control arm in place of a placebo arm when designing studies for treatment of rare diseases.

This webinar examines the utility of real-world evidence in development of orphan drugs — how and when RWE (and real-world data) can be applied in rare disease studies and what obstacles researchers need to overcome. Premier Consulting experts discuss three key challenges surrounding the use of RWE.

Key Learning Objectives

  • Identification of relevant data and how such data can be leveraged for regulatory submission
  • Standardization of the data: converting unstructured data into structured data
  • Using RWE as a complement to clinical trial data


Sheri Medendorp, MPH, President, Premier Consulting

Nach Davé, RPh, MS, Vice President, Development Strategy, Premier Research