PROs and Drug Labeling Undergo Scrutiny

In a Monday morning session entitled "Patient-Reported Outcome Instruments: Overview and Comments on the FDA Guidance," CDER officials stressed the importance of including PROs early in the clinical trial planning process, rather than at data analysis stages. Edwin P. Rock, MD, PhD, medical officer, division of drug oncology products, described the endpoint model/conceptual framework that organizes the amount/type of data for various uses.

"This approach provides clarity for FDA as well as the sponsor in terms of benefit and feasibility of labeling goals." John H. Powers, MD, FACP, lead medical officer, office of antimicrobial products, said "This is an exciting time to develop novel ways to find drugs to benefit patients." However, he cautioned that clinical researchers must be careful to measure what they think they are measuring in a valid and precise way.

And addressing the issue of multiple endpoints, Robert T. O'Neill, PhD, director, office of biostatistics, said this is an issue FDA is currently examining in the context of the Critical Path Initiative. He also said the issue of Minimum Important Differences and their inclusion in PROs is a hot topic that also warrants further analysis.