Patient-Reported Outcomes as Endpoints in Oncology Trials and Marketing Claims: An FDA/EMA Perspective

There is growing interest in inclusion of patient voice in the drug discovery and approval processes. Patient-reported outcomes (PROs) in clinical studies can capture the patients’ subjective symptoms, function and quality of life. These are used as tools to monitor drug effects on outcomes that might be more tangible or as important for the patients as objective clinical outcomes. Oncology drug development can uniquely benefit from use of PROs because disease symptoms and adverse effects of treatment both can substantially affect patients’ quality of life and other attributes that can be only monitored and quantified through appropriate PROs.

This webinar will present the current status of PRO use in labeling claims under the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) demonstrating how researchers can use ePROVIDE^TM databases – Mapi Research Trust centralized comprehensive database platform – to review drug labels and identify key endpoints in drug trials in a specific therapeutic area.

The speakers performed a search in the Patient-Reported Outcomes and Quality of Life Instrument Database (PROQOLID^TM)  for two selected clinical indications: breast and prostate neoplasms looking for clinical outcome assessment tools and clinical trials endpoints, and labeling claims under the two regulatory agencies.  They will detail the PRO instruments used in drug labeling for these indications, types of instruments, endpoint position and the study design while describing the regulatory requirements underlying these differences.

Register to explore examples (among the top-rated instruments used in clinical trials) of challenges and issues in endpoint strategy such as complex, multi-dimensional concepts and issues surrounding implementation of PROs such as sources of bias.

Speakers

Nadine Kraft, MSc, Team Lead, Database Unit, Mapi Research Trust

Nadine Kraft is the Team Lead of Mapi Research Trust’s Database Unit (DB). Prior to her work with the DB unit, side by side with her studies, she worked in an international medical technology company and assisted in the development of an outcome assessment. At the University of Salzburg, she graduated with a MSc in psychology and a specialization in cog. neuroscience and psychological diagnostic, including psychometric properties.

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Roya Sherafat, MD, PhD, Scientific Director, Mapi Research Trust

Roya Sherafat is a physician scientist and epidemiologist. She has worked in academic clinical research for more than a decade designing and conducting interventional and observational studies in the field of cardiovascular health and obesity. During the past nine years she worked as a health services researcher and health policy expert on patient-centered value-based healthcare focusing on performance assessment and measuring quality of care in complex healthcare systems. Her recent work focused on psychometric properties and implications of incorporating patient-reported outcome measures in such performance assessment tools. She is currently leading the scientific activities at Mapi Research Trust in dissemination of patient-reported outcomes and providing support and access to clinical outcome assessment tools.

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