A framework for assessing the impact of accelerated approval

Area of Science:

• Regulatory Science
• Health Economics
• Decision Analysis

Background:

• The U.S. Food and Drug Administration's (FDA) Accelerated Approval (AA) program aims to speed up access to treatments for serious conditions lacking alternatives.
• Debate exists regarding the sufficiency of evidence supporting AA to meet the needs of diverse stakeholders, including patients, physicians, regulators, and health technology assessment (HTA) agencies.
• The increasing prevalence of AA necessitates objective and reproducible methods for evaluating its impact and ensuring consistent assessment of therapeutic options.

Purpose of the Study:

• To describe an approach for evaluating the impact of the FDA's Accelerated Approval (AA) program.
• To accommodate stakeholder-specific perspectives on benefits, risks, and costs associated with AA-approved products.
• To provide a foundation for communicating the value of AA treatments in a manner relevant to various stakeholders.

Main Methods:

• Development of a formal decision-analytic framework.
• Integration of predictive distributions for therapeutic outcomes (efficacy and safety) derived from AA trial data.
• Incorporation of stakeholder assessments regarding potential actions and their implications.

Main Results:

• The proposed framework allows for objective and reproducible evaluation of AA impact.
• It accommodates diverse stakeholder viewpoints on the value of AA-granted treatments.
• The approach facilitates consistent comparison of therapeutic decision alternatives.

Conclusions:

• A formal decision-analytic framework can objectively evaluate the impact of Accelerated Approval (AA).
• This approach integrates predictive outcome modeling with stakeholder-specific value assessments.
• It offers a starting point for transparently communicating the value of AA treatments to all relevant parties.