The Expanding Role of RWE in Rare Studies

Identification of relevant data and how such data can be leveraged for regulatory submission

  • Free

Why attend?

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:

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

Target Audience

  • Small to mid-size pharma companies
  • Diagnostics & Drug professionals
  • Medical device companies & manufacturers

Speakers

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Shari Medendorp, MPH
President, Premier Consulting

Shari Medendorp leverages her more than 20 years of industry knowledge and experience to lead Premier Consulting, a highly integrated business unit of Premier Research that specializes in strategic product development and global regulatory consulting for emerging biotech and specialty pharma companies.

Ms. Medendorp’s experience includes leading Premier Research’s biometrics operations, holding positions as Senior Vice President of Biometrics at CliniRx, Reliance Clinical Research Services, and SCIREX Corporation, among other executive roles. She started her career as a Biostatistician working with medical staff at the Cleveland Clinic Foundation on the design and analysis of clinical studies. Ms. Medendorp holds a master’s degree in public health in biostatistics from the University of Michigan and a bachelor’s degree in mathematics from Calvin University.

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

Nach Davé provides strategic and commercial input for Premier Consulting. He brings more than 20 years of experience in the pharmaceutical and contract research industries to the position. He previously served as the Vice President of Regulatory at Premier and has been in leadership positions at both CROs and sponsor companies.

Mr. Davé previously led clinical and regulatory affairs at Maxx Orthopedics, a developer of orthopedic medical devices, and has held roles in clinical operations, business development, strategic consulting and medical affairs at companies such as Merck, Bristol-Myers Squibb, Aventis Pharmaceuticals and Mitsubishi Pharma America.

In his current role, Mr. Davé brings innovative solutions and grow Premier’s footprint in the areas of medical device development, real-world evidence, and government relations. He is keen to explore how innovative technology like AI, ML, and other developments can best support the growth of Premier’s business.

Mr. Davé holds a master’s degree in drug regulatory affairs from Long Island University and a bachelor’s degree in pharmacy from the University of Sciences-Philadelphia. He is a registered pharmacist.

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