Communicating
the Patient Voice with PROs: Making the Most of PRO Data from Objectives to Estimands
Organized By: Statistics SIG
Series description
There is substantial heterogeneity in PRO analyses in trials. PRO objectives are often vaguely defined, making interpretation difficult. Better definition of PRO objectives and their connection to analyses can improve interpretation. The estimand framework provides a clear process for defining and interpreting PRO endpoints and has been encouraged by regulatory agencies. This webinar series will begin with a basic introduction to PRO endpoints in clinical trials, with examples from multiple disease areas. It will then introduce the estimand framework and demonstrate how to use this framework to construct PRO estimands in which trial design and analysis are connected, and the analysis is interpretable and communicable.
Intended Audience
The intended audience are PRO researchers and clinical trialists, especially those who are non-statisticians.
Level of Knowledge Needed: Intermediate
Some prior training or work experience relevant to the topic is recommended to aid in understanding of webinar content.
The first webinar is basic and the second is advanced; the overall level is intermediate.
Audience Benefits
This series will provide the audience with:
- An understanding of the importance of connecting trial design, analysis and interpretation for PROs in clinical trials
- An understanding of how to interpret the results of different analytic choices for PROs in different scenarios, and the implication of these choices
- An overview of the estimand framework and its relevance in the trial setting
- A clear and accessible demonstration of the value of the estimand framework for patient-reported endpoints, and how these can be applied in the regulatory setting
Learning Outcomes/Objectives
At the end of this series, attendees will be able to:
- Develop a PRO objective
- Understand how different PRO analyses of the same data may lead to different results & impacts on interpretation
- Understand how using estimands provide a framework connecting the PRO objective, with the analytic approach, and interpretation
- Describe different analytic approaches of interest for PRO measures
- Describe the 5 components of estimands for PROs
- Evaluate the strengths and limitations of different estimand strategies for trials in different clinical areas
- Describe evaluable PRO estimands for a clinical trial
- Understand post-randomisation events and how to plan for them using estimands
- Understand the patient-relevance in applying estimands to evaluate trials
Webinar #1: How to Develop PRO Objectives for Trials
PROs are encouraged in trials, but vaguely defined objectives that are not connected to PRO analyses means that these results are not easy to interpret. This webinar will show how to develop a good PRO objective and demonstrate the possibilities and implications of different analytic choices, using examples from multiple disease areas.
Speakers:
- Corneel Coens, MSc, Biostatistician, EORTC HQ, Belgium
- Rachael Lawrance, BSc, Director and Principal Statistician, Adelphi Values, United Kingdom
- Jessica Roydhouse, PhD, Select Foundation Senior Research Fellow in Health Services Research, Menzies Institute for Medical Research, University of Tasmania, Australia
Webinar #2: What’s an Estimand and Why Should I Use It?
This webinar will introduce estimands, and show how estimands can be used to plan for and address post-randomisation challenges to make the most of PRO data. It will demonstrate how to use this framework to construct, evaluate and communicate PRO estimands using examples from multiple disease areas.
Speakers:
- Libby Floden, PhD MPH, Senior Director, Head of Endpoints, Evinova, United States
- Antoine Regnault, PhD, Partner and Global Lead – Statistics, Modus Outcomes, a Division of THREAD, France
- Rachael Lawrance, BSc, Director and Principal Statistician, Adelphi Values, United Kingdom
- Patient Representative
Purchase this 2 webinar series below by 1 May 2024.
Individual webinars also available for purchase below and here.
