The challenges and opportunities for rare disease drug development


Rare disease clinical trials have several challenges that make them more difficult to conduct than clinical trials for more common diseases.  Small patient populations, pediatric populations and the desire by patients and their caregivers to receive active therapy while on a clinical trial make it much more likely that rare disease trials will be conducted without placebo control groups. An alternative to a placebo control group is the use of existing medical history information on a group of similar patients to provide a historical control.

 Recent developments by regulatory agencies have resulted in increasing use of historical control groups to provide comparative evidence to establish the efficacy and safety of rare disease therapies.  This webinar provides an overview of the use of historical control populations in rare disease drug development and approval.

Join us to learn the challenges and opportunities of this approach, including:

  • Sources for historical control information, including prospective and retrospective data
  • The advantages and implications on study enrollment, study budget and time to market
  • How to identify and address data consistency, quality and outcomes assessment methods
  • The current regulatory landscape in the US and Europe and a review of recent approvals using historical controls

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William C. Maier, MPH, PhD

Chief Scientific Officer & Head of Rare Disease Research, Commercialisation and Outcomes, ICON

William has over 30 years of experience with pharmaceutical companies in Europe, Canada, the United States and Asia. At ICON he works with pharmaceutical companies to provide regulatory, strategic and scientific guidance on medical treatment development and commercialisation. He is a member of the EMEA’s European Network of Centres for Pharmacoepidemiology and Pharmacovigilance. He is a frequent speaker at medical conferences and is a member of the Royal Society of Medicine in the UK.