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Session 3: Rare Disease I
Session Chair(s)
Mariette Boerstoel-Streefland, MD, MBA, MS
Senior Vice President, Patient Safety Officer , Bristol Myers Squibb, 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
Senior Vice President, Patient Safety Officer , Bristol Myers Squibb, United States
Larry Bauer, MA, RN
Senior Regulatory Drug Expert, Hyman, Phelps & McNamara, P.C., United States
Rare Diseeases and Orphan Drugs
William Haddock
Dr, Ovid Rx, United States
Safety Management and Benefit-Risk Assessment for Rare Disease