Development programs in rare diseases are becoming more common and a major focus for small biotech and pharmaceutical companies.  A crucial consideration in clinical study and protocol design in these indications is the choice of control group used to assess the efficacy of an intervention.  Standard study designs and control are often not feasible due to limitations in the number of prospective patients as well as ethical considerations.  Non-standard study designs, including studies relying on external historical control groups, can be used to evaluate the efficacy of an intervention. This webinar will discuss:

  • Factors to consider when evaluating the use of an external control in a planned clinical study
  • Appropriate analysis and interpretation of externally controlled studies
  • The critical role of externally controlled studies in supporting marketing applications for orphan/rare disease products

Featured Speakers

Rob Woolson

Chief Strategist, Biostatistics & Standards for Regulatory Submissions

Rob Woolson has over 18 years of experience as an applied statistician. He brings an extensive background of statistical and project leadership experience on US and ex-US regulatory submissions, having led the biostatistical and technical aspects of CDISC-compliant marketing applications, guided the creation of ISS/ISE statistical analysis plans; integrated analysis dataset design and production; integrated display design and production; and submission-related documentation development. He has conducted statistical analyses in all phases of drug development and has led SDTM/ADaM dataset conversion projects in multiple therapeutic areas.

Kevin Barber, PhD

Vice President, Regulatory Strategy & Submissions

Kevin Barber has more than 20 years of experience in regulatory affairs and product development, working for both sponsor companies and CROs, across all stages of development from pre-clinical through product launch and post-approval life cycle management. He has lead the preparation and execution of integrated regulatory strategy and clinical development plans for drug, biologic, and medical device products in multiple therapeutic areas. Dr. Barber has significant experience preparing and filing regulatory submissions, including more than 40 US INDs and more than 35 marketing applications in multiple countries including the US and Canada.


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