Clinical Research: Phase 1 - Phase 4

The Expanding Role of RWE in Rare Studies

August 4th, 2022
10:00am EST

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 will examine 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 will discuss three key challenges surrounding the use of RWE:

  • 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

The presenters also will summarize the latest FDA guidance on use of RWE and provide a case study on use of this evidence in a successful regulatory submission.

Featured Speakers:

Shari Medendorp, MPH, President, Premier Consulting

Nach Davé, RPh, M.S., Vice President, Development Strategy, Premier Consulting