during the workshop focused on the following: (1) premarket assessment, during which clinical trial data are used to assess benefit and risk; (2) communication of that information to prescribing physicians and their patients; (3) healthcare decisions made by prescribing physicians and their patients; and (4) the accumulation of benefit–risk information from postmarketing experience, which feeds back into the other phases.


Workshop participants identified several challenges that industry and the FDA must overcome as they make their premarket assessment of pharmaceutical benefit and risk:

  • Lack of a systematic, consistent, and transparent approach to benefit–risk analysis;

  • Uncertainty regarding how to balance risk and benefits;

  • Insufficient knowledge about the risks of drugs at the time of their launch;

  • Conflict of interest (for example, experts consult with industry and government on the same products) and involvement of stakeholders (for example, scientists, industry) in evaluating benefit and risk;

  • Lack of involvement of prescribing physicians and the public in the FDA regulatory process; and

  • Confusing and inconsistent terminology in benefit–risk assessment.

As panelists considered how to construct a systematic, consistent, and transparent approach to benefit–risk analysis, discussion focused on whether and how such a framework should be quantitatively based. Creating a quantitative system has many challenges, including the following:

  • Quantitatively capturing a complex drug benefit–risk profile;

  • Quantitatively characterizing drug benefit–risk for individuals because of variation among patients in terms of both physiology and preferences;

  • Updating benefit–risk assessments with new information through the drug life cycle;

  • Addressing the inherent uncertainty in benefit–risk measurement;

  • Resolving disagreement about the role that cost should play in benefit–risk calculations;

  • Addressing the cost of adopting a quantitative framework and its potential adverse effect on innovation; and

  • Effectively presenting and communicating quantitative information (see more below).

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