Pharmacovigilance and Risk Management Strategies Conference

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.