OBJECTIVE: The US Food and Drug Administration (FDA) gives guidance at various times during the drug development process at the request of the sponsor, but perhaps the most critical discussion occurs at the end of Phase II. At that time, the sponsor will submit for FDA’s review draft protocols for what they hope will be convincing studies of the product’s benefit/risk profile that will support an approval decision. FDA’s insight into this advisory process as it pertains to study end point review may facilitate the efficiency of future discussions of this type, DISCUSSION: We suggest ways to increase efficiency in these discussions, including proposed labeling to provide context to the study end point discussion, submission of full documentation of end point development and validation in the meeting package, clear methodology for the estimation of effect sizes, and a detailed analysis plan that explains criteria for end point interpretation.
#7 The Patient’s Assessments
Cynthia Chauhan,l Wayland Eppard,2 Marlene H. Frost, RN, PhD,3 Michele Halyard, MD,4 and Jeff A. Sloan, PhD5 IPatient advocate, Wichita, Kansas, 2Patient advocate, Rochestel;Minnesota, 3Mayo Clinic Women? Cancer Program, Rochestel;Minnesota, 4Mayo Clinic Scottsdale,Scottsdale,Arizona, and 5Mayo Clinic and Foundation, Rochestel;Minnesota What do quality-of-life (QOL) assessments mean to patients, and how do their interpretations compare to others? This interactive session involves a dynamic assessment of a cancer patient’s QOL by an oncology nurse specialist, an oncologist, a QOL tool developer/researcher, a QOL researcher/analyst, and a peer patient advocate. The patient volunteer also has completed a series of QOL assessments prior to the presentation. Each of the clinical and research professionals will provide their interpretation of the QOL assessment results provided by the patient. The patient will then provide feedback regarding the concurrence and discrepancies between these assessments and the patient’s own interpretation. A general discussion will follow with respect to the amount and importance of the discrepancies. This case study is intended to cast light, in an exemplary fashion, on how the perspectives of a consumer of QOL assessments can affect the interpretation of the clinical significance of the results.