The Expanding Role of RWE in Rare Studies

Presented by

Premier Consulting – Shari Medendorp, MPH, President, and Nach Davé, RPh, MS, Vice President, Development Strategy

About this talk

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 Questions? Contact

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Premier Research is a clinical research company, dedicated to helping biotech, specialty pharma, and device innovators transform life-changing ideas and breakthrough science into new medical treatments. As a global company, Premier specializes in the use of innovative technologies for smart study design and trial management to deliver clean, conclusive data to sponsors. Whether it’s developing product lifecycle strategies, reducing clinical development cycle times, securing access to patients, navigating global regulations, maximizing the impact of limited rare disease data, or providing expertise in specific therapeutic areas, Premier is committed to helping its customers answer the unmet needs of patients across a broad range of medical conditions.