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

Life Sciences, Clinical Trials, Drug Discovery & Development,
  • Tuesday, September 20, 2022

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.

Read Less...

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.


Nadine Kraft, Mapi Research Trust

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.

Message Presenter
Roya Sherafat, Mapi Research Trust

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.

Message Presenter

Who Should Attend?

This webinar will be of interest to:

  • Clinical Scientists, Epidemiologists and those involved in cancer clinical trial and comparative effectiveness research
  • Clinical Outcome Assessment (COA) Scientist and Psychometricians involved in development and validation of instruments for patient-reported outcomes (PROs) related to oncology and palliative care

What You Will Learn

Attendees will:

  • Discover an up-to-date information source about use of PROs in oncology drug approval and labeling claims between the FDA and the EMA — case of breast and prostate cancer
  • Become familiar with key requirements for PROs consideration specific to oncology trials and current challenges in PROs strategy design and implementation for clinical trials
  • Understand how the ePROVIDE centralized database platform can help screening and selecting appropriate clinical outcome assessments

Xtalks Partner

Mapi Research Trust

Mapi Research Trust is a non-profit organization that promotes the use of Clinical Outcomes Assessments (COAs) in health research and practice and encourages exchanges of Patient-Centered Outcomes and COA information among academics, pharmaceutical companies, health care organizations and health authorities. With the web-based platform ePROVIDE™ that includes the databases PROQOLID™, PROLABELS™, and PROINSIGHT™, Mapi Research Trust has become the preeminent source of reliable, up-to-date and comprehensive information about COAs.

You Must Login To Register for this Free Webinar

Already have an account? LOGIN HERE. If you don’t have an account you need to create a free account.

Create Account