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Pharmacovigilance and Risk Management Strategies Conference

NEW! The 2019 meeting will be a full three days, allowing maximum learning opportunities!


Session 3: Rare Disease I

Session Chair(s)

Mariette  Boerstoel-Streefland, MD, MBA, MS

Mariette Boerstoel-Streefland, MD, MBA, MS

  • Senior Vice President Global Drug Safety
  • Alexion Pharmaceuticals, Inc., United States
There is considerable unmet medical need for rare disease treatment options. Clinical development and regulatory hurdles are difficult to navigate. The mandate to assess the benefit-risk balance in order to make a decision about any new product before market approval is challenging when data to assess the safety of a new product is very limited. The traditional benefit-risk assessment does not necessarily apply, in case of fatal, or severely debilitating conditions with no alternative treatment options. The typical strong focus on avoiding risks and uncertainties concerning new medicines might not apply to rare diseases and the benefit risk assessment may differ between regulators, prescribers, and patients and their caregivers. This session explores the regulatory, ethical, and patient perspective aspects of benefit-risk assessment and managing remaining uncertainty about safety for orphan drugs.

Speaker(s)

Mariette  Boerstoel-Streefland, MD, MBA, MS

Mariette Boerstoel-Streefland, MD, MBA, MS

  • Senior Vice President Global Drug Safety
  • Alexion Pharmaceuticals, Inc., United States
Larry  Bauer, MA, RN

Rare Diseeases and Orphan Drugs

Larry Bauer, MA, RN

  • Senior Regulatory Drug Expert
  • Hyman, Phelps & McNamara, P.C., United States
William  Haddock

Safety Management and Benefit-Risk Assessment for Rare Disease

William Haddock

  • Dr
  • Ovid Rx, United States