WHAT TO CHOOSE? CREATING A PATIENT-REPORTED OUTCOMES (PRO) ASSESSMENT STRATEGY WITH EORTC MEASURES AND ITEM LIBRARY THAT CAN MEET THE NEEDS OF REGULATORS

Author(s)

Kulis D1, Piccinin C1, Bottomley A1, Darlington AS2, Grønvold M3
1EORTC, Brussels, Belgium, 2University of Southampton, Southampton, UK, 3University of Copenhagen, Copenhagen, Denmark

Recent papers and workshops from the FDA have proposed focusing on measurement of three concepts in cancer clinical trials for label claims: symptomatic adverse effects (AEs), physical function (PF), and disease-related symptoms. Responding to these recommendations for use of PROs, the EORTC proposes a new model allowing for an assessment strategy within one measurement system. Within the FDA’s framework, one should select key endpoints with well-defined objectives, and determine disease-related symptoms and AEs from previous research, literature, and qualitative methods, before choosing appropriate PRO measures. This abstract shows how the EORTC’s tools can meet this selection. The EORTC QLQ-C30 core questionnaire measures PF, as well as other well-defined functions (role, emotional, social, cognitive), and includes 11 out of 12 core set cancer symptoms (Reeve 2014). As a holistic instrument to measure HRQOL, it covers some symptoms and AEs (appetite loss, fatigue, nausea, vomiting, dyspnea, diarrhea, constipation, insomnia), and additional domains: overall QOL and financial difficulties. A shorter 17-item QLQ-F17 is available, focusing on function only. Alternatively, all QLQ-C30 domains (including PF) can be measured with an IRT-based Computer Adaptive Testing (CAT) system with full item banks or short forms for higher precision or more flexibility. Other disease-related symptoms and most frequent AEs are part of the validated disease-specific modules. Any missing issues can be included in a supplemental item list created with the Item Library’s over 950 items. Through mixing static questionnaires with item lists to address possible gaps, EORTC’s one system solution allows for a consistent measurement strategy with less cognitive burden than when various instruments are combined. The wide range of over 40 validated EORTC instruments and Item Library enables a flexible assessment tailored to each trial’s objectives. We believe such a system will fulfill the FDA’s and other regulators’ strategy for selecting robust endpoints in clinical trials.

Conference/Value in Health Info

2019-05, ISPOR 2019, New Orleans, LA, USA

Value in Health, Volume 22, Issue S1 (2019 May)

Code

PCN45

Topic

Health Policy & Regulatory, Methodological & Statistical Research, Patient-Centered Research

Topic Subcategory

Approval & Labeling, Instrument Development, Validation, & Translation, Patient-reported Outcomes & Quality of Life Outcomes, PRO & Related Methods

Disease

Oncology

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