Generic Patient Self-Report and Investigator Report Instruments of Therapeutic Safety and Tolerability

MARISSA N.D. LASSERE, KENT R. JOHNSON, SUSANNE VAN SANTEN, KERRI CARLTON, JOY RAPPO, RACHEL MICHAEL, JOHN R. KIRWAN, and JOHN P. EDMONDS

ABSTRACT.

A patient self-report instrument was designed as a patient event index that maps to a parallel investigator instrument. Event importance (a composite of severity, frequency, and duration) was reported, but attribution was not required. The patient instrument used a checklist but also allowed for spontaneous reporting for new or unusual events. The investigator instrument (also a checklist) includes all events reported by the patient, as well as events such as signs, investigations, and diagnoses that would not generally be known to the patient. Presently, both patient and investigator instruments are to be used alongside current methods of adverse event reporting in clinical trials. The patient instrument would serve as a safety/tolerability index, whereas the investigator instrument would be a fully quantifiable (appropriately weighted), standardized adverse event index. As in many methodological projects in medicine, the overriding problem was the tradeoff between validity (comprehensiveness and accuracy) and feasibility (clarity and short administration time) in instrument development. A summary of pilot studies and results of instrument reliability and validity are presented. (J Rheumatol 2005;32:2033-6)

Key Indexing Terms:

ADVERSE EVENT
PATIENT QUESTIONNAIRE
RANDOMIZED CONTROLLED TRIALS

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From the Department of Rheumatology, St. George Hospital, University of New South Wales, Sydney, Australia; Department of Clinical Pharmacology, Mater Hospital, University of Newcastle, Newcastle, Australia; Amsterdam Medical School, Amsterdam, The Netherlands; and the Academic Rheumatology Unit, Bristol Royal Infirmary, Bristol, England.

M.N.D. Lassere MB, BS, Grad Dip Epi, PhD, FRACP, FAFPHM, Associate Professor in Medicine, Conjoint Appointee University of NSW, Department of Rheumatology, St. George Hospital; K.R. Johnson, MD, Senior Research Officer, Department of Clinical Pharmacology, Mater Hospital, University of Newcastle; S. Van Santen, Amsterdam Medical School; K. Carlton, BSc, MSc, Research Officer; J. Rappo, BSc, M Epi, Research Nurse; R. Michael, BCom, Research Officer, St. George Hospital; J.R. Kirwan, Consultant and Reader in Rheumatology, Academic Rheumatology Unit, Bristol Royal Infirmary; J.P. Edmonds, Professor of Rheumatology, University of NSW, Director, Department of Rheumatology, St. George Hospital.

Address reprint requests to Dr. M. Lassere, Department of Rheumatology, St. George Hospital, Kogarah, 2217 NSW, Australia. E-mail: lasserem@sesahs.nsw.gov.au

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