2022 - 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 ePROVIDETM 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 (PROQOLIDTM) 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.
Previous reports show that there is high convergence in drug approval and labeling between the two agencies, however, oncology is one area where there are more differences due to divergent strategies. In this webinar, the speakers summarize the FDA and EMA requirements in terms of: endpoint selection and labeling claim; instrument properties and psychometric validation; and considerations based on clinical trial study design and PRO implementation.
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.