Patient-reported outcomes supporting anticancer product approvals.

Area of Science:

• Oncology
• Regulatory Science
• Clinical Trial Design

Background:

• The US Food and Drug Administration (FDA) issued draft guidance in 2006 for patient-reported outcome (PRO) measures used in drug development.
• PRO measures are crucial for substantiating treatment benefit claims in product labeling, especially for anticancer therapies.
• Reviewing past FDA approvals provides context for the application of PRO guidance in oncology.

Purpose of the Study:

• To summarize and discuss FDA approvals of anticancer products in relation to the 2006 draft PRO guidance.
• To identify anticancer product approvals where efficacy claims were based partly or wholly on PRO data.
• To analyze the limitations of PRO instruments submitted for regulatory review between 2004 and 2006.

Main Methods:

• Identification of FDA-approved anticancer products with efficacy claims supported by PRO data.
• Collating limitations of PRO instruments submitted for regulatory review during a specific two-year period.
• Analysis of the role and characteristics of PRO data in supporting treatment benefit claims.

Main Results:

• From 1995 onwards, nine indications for seven anticancer products were approved based partly on PROs.
• In most approvals (8/9), PRO data supplemented other clinical evidence of benefit.
• In seven approvals, PROs measured single symptoms or functional domains directly linked to treatment effects.

Conclusions:

• The FDA's draft PRO guidance reflects principles already utilized in anticancer product approvals.
• PRO endpoints commonly support claims related to patient symptoms and functional abilities.
• Internally consistent PRO data, aligned with other clinical evidence, is essential for regulatory acceptance.