Quality Over Quantity: How Do Clinical Trial Sponsors Balance the Evidence Needs of Regulatory and Reimbursement Bodies When Assessing Patient-Reported Outcomes (PRO) in Oncology Trials?
Author(s)
Moderator: Peter Trask, PhD, Genentech, South San Francisco, CA, USA
Panelists: Claire Piccinin, MSc., European Organisation for Research and Treatment of Cancer (EORTC), Brussels, Belgium; Kirsten Janke, PhD, Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG), Cologne, Germany; Vishal Bhatnagar, MD, Oncology Center of Excellence, U.S. Food and Drug Administration, Silver Spring, MD, USA
ISSUE: Assessing the health-related quality of life (HRQoL) of treated cancer patients remains paramount for sponsors, regulators, reimbursement bodies, clinicians and patients. Patients are the best source of data on the frequency and severity of disease-related symptoms, treatment side effects, and the impact of treatment on functioning and well-being; measuring these issues in a way that minimizes patient burden and limits missing data is paramount. Doing this in a way that satisfies all stakeholders can be challenging, as there appear to be differing evidence requirements among review bodies, leading to alternative approaches of using full generic and disease-specific PRO questionnaires vs. targeted use of specific scales and items. These contrasting perspectives will be discussed in this plenary session.
OVERVIEW: Oncology clinical trials have historically collected PRO data through standard generic HRQoL questionnaires and disease-specific modules. Treatment paradigms have evolved since these instruments were developed, meaning current clinical trials may include questions that have little or no relevance, while missing important disease or treatment-related concepts.
FDA’s patient-focused drug development (PFDD#3) guidance describes the importance of starting with a clear understanding of the disease or condition in order to select, modify or develop validated PRO measures. The outcomes measurement community (including the EORTC), based on feedback from patients, is moving towards assessments that are tailored, through the use of modular item libraries, to maximize relevance to the person's disease and treatments. In contrast, some reimbursement bodies require complete validated, standardized generic and disease-specific questionnaires. This plenary discussion aims to: 1) Clarify positions on the requirements for oncology clinical trial PRO evidence across these critical groups in drug development 2) Identify areas of agreement among them 3) Brainstorm on actions that can be taken to bridge the remaining gaps. Presentation time totals 40 minutes with 20 minutes for audience questions and discussion.Conference/Value in Health Info
Code
314
Topic
Patient-Centered Research