Patient-Reported Outcomes: Statistical Considerations in Seeking a Labeling Claim

• Activity Number: 564
• Type: Contributed
• Date/Time: Wednesday, August 6, 2014 : 2:00 PM to 3:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Isaac Nuamah*+
• Companies: Janssen
• Keywords: Patient Reported Outcome ; Quality of Life ; Regulatory claim ; totality of evidence

Abstract

A patient-reported outcome (PRO) is any report on the status of a patient's health condition that comes directly from the patient, without interpretation by anyone else. PROs are standard tools for eliciting the patient's experience.

We consider here the statistical considerations that come into play when a sponsor wants to make a labeling claim on a PRO when evidence of clinical efficacy has/has not been established using data from the same clinical trial. According to the FDA PRO guidance, a claim is defined as a statement of treatment benefit which can appear in any section of a product's FDA-approved labeling or in advertising and promotional labeling.

We consider the recently proposed evidential approach that does not require adjustment of p-values or their significance levels, while still strongly controlling the multiple type 1 error rates. The approach allows testing the secondary endpoint even if the primary endpoint fails. Can such an approach result in regulatory approval of a secondary PRO endpoint? How about if there is prior evidence of the efficacy of the primary endpoint? This is a matter of regulatory review but can the totality of evidence carry the day?