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.

Value of a Quantitative Approach to Benefit–Risk Assessment

Dr. Tollman explained that federal agencies, such as the Environmental Protection Agency (EPA) and the Federal Motor Carrier Safety Administration (FMCSA) use quantitative evaluations to more rigorously assess benefit and risk, as well as cost. While parallels to pharmaceuticals are not conclusive, they suggest that quantitative approaches to drug benefit–risk assessment may be viable. Quantification, however, has its own set of challenges:

  • 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;

  • Addressing 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.

Dr. Tollman suggested that adopting a quantitative approach could be beneficial in that it could objectively combine clinical trial data and information on patient preferences. Dr. Tollman noted that the academic community has a number of simple, powerful frameworks and utility weighting methods that could feasibly be adapted to the drug approval process. He cautioned, however, against quantitative elements being too simplistically or narrowly interpreted.

Dr. Tollman further argued that a more structured, transparent, and quantitative approach would be advantageous for all constituents— patients, regulators, and industry. For patients, advantages include the fact that the approval decision incorporates patient preferences, ultimate drug choice is based on individual response and preferences, and more differentiated treatment options become available to patients. For regulators, advantages include decisions that are grounded in a preagreed



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