Validating Select Physical Performance Outcome (PerfO) Assessments to Support Labelling Claims in the US: Methods and Lessons Learned

As a type of Clinical Outcome Assessment (COA), Performance Outcome assessments (PerfOs) specifically refer to task(s) performed by a patient according to instructions administered by a health care professional. They thus require patient cooperation and motivation (FDA, COA Glossary of terms). While PerfO assessments such as walk and stair climbing tests are widely used to assess the effectiveness of interventions, and there is some evidence on the measurement properties of the tests, there is no regulatory guidance in terms of assessment development and validation specific to PerfOs. While the Patient Reported Outcome guidance (FDA/SEALD Guidance for Industry 2009) provides a useful framework, PerfOs do present specific challenges.

This webinar draws on experience in validating a select group of PerfO assessments in orthopaedics, an area with a strong history of performance measurement, with the intention of supporting future labelling claims. The PerfO assessments were Timed Up and Go, Stair Climbs and Repeated Chair Stands which were administered in hip fracture and elective hip and elective knee replacement patients in a multi-site study.

The webinar will present conceptual and logistical challenges of both qualitative and quantitative studies to assess the content validity and measurement properties of these PerfO assessments in these patient groups, and the results from these studies. We will also reflect on the value of FDA Guidance on the use of PRO measures (FDA/SEALD Guidance for Industry 2009) for PerfOs, and review the FDA Clinical Outcome Assessment Compendium and other sources for indication of the extent and nature of use of PerfOs to support labelling claims in conditions more widely.

The objectives of this webinar are to:

• Briefly describe PerfOs and related terminology
• Describe how PerfO assessments compare with other COAs and the breadth of PerfOs
• Provide an in-depth orthopedic case study
• Highlight practical and logistical issues of assessing select physical PerfO assessments
• Demonstrate the approach taken to validating selected PerfO assessments
• Share validation study results
• Provide clear take-away messages to help inform approach to selecting and validating PerfO assessments for physical mobility-type conditions
• Provide an indication as to the extent and nature of PerfOs used to support labelling claims more widely

Speakers

Elizabeth (Nicki) Bush, Research Scientist, Eli Lilly and Company

Nicki has been with Eli Lilly and Company’s Global Patient Outcomes and Real World Evidence (GPORWE) group for five years. In this role, she is responsible for developing and executing patient focused outcomes and health economics strategies in a number of therapeutic areas including pain and psychiatry. Prior to joining Lilly, Ms. Bush was a researcher at United BioSource Corporation’s Center for Health Outcomes Research, specializing in developing and validating instruments to measure patient reported outcomes in various disease states. Ms. Bush earned her BA degree in the liberal arts (philosophy) from St. John’s College, her master’s degree in epidemiology from Johns Hopkins Bloomberg School of Public Health, and is currently pursuing her PhD in epidemiology at Indiana University’s Fairbanks School of Public Health.

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Rachel Ballinger, Lead Outcomes Researcher, ICON

Rachel has been with ICON since 2011 and has worked on a large range of COA studies, including validation of physical performance outcomes in a multi-site study. Before joining ICON, Rachel worked as a Research Fellow at the Psychosocial Oncology Group at the University of Sussex, where she conducted numerous quality of life studies including assessment of patient and clinical decision making. She has also undertaken an extensive study of public policy and carcinogenic risk assessment of Pharmaceuticals. She received her PhD in medical sociology and her BSc in Psychology and Sociology from Brunel University, West London.

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Helen Doll, Senior Principal, Clinical Outcomes Assessments, ICON

Helen joined ICON in 2012 to lead the quantitative analysis work undertaken by the Clinical Outcome Assessments group. By training, Helen is a medical statistician with particular experience of statistical and psychometric issues associated with measuring patient reported outcomes (PROs). Helen worked for 20 years as a senior medical statistician in the University of Oxford, collaborating with researchers and clinicians in a variety of other specialties, most notably in orthopaedics, where she worked on the development and validation of a number of PROs. Helen was educated first at Warwick University where she took a BSc degree in Biological Sciences (1984) and then at Oxford University where she received a post-graduate diploma in Applied Statistics (1986), an MSc in Clinical Medicine by research (1992) and a DPhil in Clinical Medicine (2005). Helen’s main research interests are in the use of item response theory, and Rasch methodology in particular, in the development of PROs.

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