Development and validation of eleven symptom indexes to evaluate response to chemotherapy for advanced cancer: Measurement compliance with regulatory demands

Abstract

Recent guidance from the United States Food and Drug Administration discusses patient-reported outcomes as endpoints in clinical trials (FDA, 2006). Using methods consistent with this guidance, we developed symptom indexes for patients with advanced cancer. Input on the most important symptoms was obtained from 533 patients recruited from National Comprehensive Cancer Network (NCCN) member institutions and four non-profit social service organizations. Diagnoses included the following 11 primary cancers: bladder, brain, breast, colorectal, head/neck, hepatobiliary/pancreatic, kidney, lung, lymphoma, ovarian and prostate. Physician experts in each of 11 diseases were also surveyed to differentiate symptoms that were predominantly disease-based from those that were predominantly treatment-induced. Results were evaluated alongside previously published indexes for 9 of these 11 advanced cancers that were created based on expert provider surveys, also at NCCN institutions (Cella et al., 2003). The final results are 11 symptom indexes that reflect the highest priorities of people affected by these 11 advanced cancers and the experienced perspective of the people who provide their medical treatment. Beyond the clinical value of such indexes, they may also contribute significantly to satisfying regulatory requirements for a standardized tool to evaluate drug efficacy with respect to symptomatology.

Citation

Rosenbloom, S., Yount, S., Yost, K., Hampton, D., Paul, D., Abernethy, A., Jacobsen, P.B., Syrjala, K., Von Roenn, J. and Cella, D. (2007), "Development and validation of eleven symptom indexes to evaluate response to chemotherapy for advanced cancer: Measurement compliance with regulatory demands", Farquhar, I., Summers, K.H. and Sorkin, A. (Ed.) The Value of Innovation: Impact on Health, Life Quality, Safety, and Regulatory Research (Research in Human Capital and Development, Vol. 16), Emerald Group Publishing Limited, Leeds, pp. 53-66. https://doi.org/10.1016/S0194-3960(08)16003-6