This webinar will focus on the methodology for developing and validating Clinician-Reported Outcomes (ClinROs) for implementation in clinical trials and to support labeling claims. In the broader family of Clinical Outcomes Assessments (COAs), ClinROs are appropriate for outcomes which cannot be reported by a patient, and are observable but require judgment or interpretation by a trained health professional. As there are no regulatory guidelines specific to ClinROs, we will share how we apply the Patient Reported Outcome guidance (FDA/SEALD Guidance for Industry 2009) as a framework for ClinRO development and validation.
We will share two recent case examples to illustrate the challenges in developing and validating ClinROs and share lessons learned in the development of a novel ClinRO intended to measure functional status of extremely premature infants in intensive care, and in the validation of a ClinRO following the completion of the clinical trial, where the ClinRO was included as a secondary endpoint to assess joint arthropathy in adult hemophilia patients
With the first case example, ICON will focus on the methodology leading to instrument development, including a targeted literature review, expert interviews, a Delphi panel, cognitive interviewing, and an online physician survey. In the second example, we will describe the design of a hybrid study to evaluate both the reliability and content validity of an already-developed ClinRO that was adapted from a pediatric measure for the adult patient population. Psychometric validity was also supported by analyses conducted with the trial dataset.
- What is a ClinRO, and when is it appropriate to use one over other types of COAs?
- What are best practices for developing and validating ClinROs?
- What are some of the key challenges and potential solutions in ClinRO development and validation?
- What are some of the key considerations for implementing ClinROs in a clinical study?
Magdi Vanya, Lead Outcomes Researcher, ICON
Magdi is a project director at ICON COA overseeing the development, selection, and validation of outcomes instruments from concept elicitation through psychometric validation, as well as preparing FDA evidence dossiers. Magdi has developed expertise in autoimmune disease, hematology, rare diseases, neurology, and renal disease, with specific focus on defining and measuring the concept of fatigue across different therapeutic conditions. Prior to joining theICON COA team in 2011, Magdi worked on a NIH-funded multi-method project in the field of substance use and violence prevention. Magdi has over 20 years of qualitative research and project management experience. Magdi completed her PhD in Sociology at the University of California, Davis.
Olga Moshkovich, Outcomes Researcher, ICON
Olga has been with ICON’s COA group for three years. In her role, Olga is responsible for managing studies to support the development and validation of COAs. Prior to joining ICON, Olga conducted epidemiological research at an academic center specializing in youth health and development. Olga is experienced in the design and implementation of qualitative research, survey design, and qualitative interviewing and analysis. Her research experience has spanned various disease areas including lupus, lung cancer, hemophilia, sickle cell, substance use, and health care quality improvement. Olga earned her BA degree in biology from Cornell University, and her Master’s in Public Health degree in epidemiology from University of Maryland.
Katy Benjamin, Principal, ICON
Katy has been a Principal at ICON COA since 2012. She is a health services researcher with extensive experience in rare diseases and outcomes research, including the qualitative and quantitative development of patient reported outcomes measures. Currently, she is the Chair of the ISPOR Task Force on PROs for Rare Disease Research in Clinical Trials. Prior to joining ICON, Katy was the Senior Health Services Researcher at MedAssurant, Inc. She served as a Research Scientist at United BioSource Corporation for three years, and spent eight year as a Social Science Analyst with the Agency for Healthcare Research and Quality’s Center for Outcomes and Effectiveness Research.
Who Should Attend?
COA scientists and those with interest in COAs, including senior level decision makers in:
- Late Phase Research
- Project Management
- Patient Registries
- Risk Management
- Market Access
- Regulatory Affairs
ICON Commercialisation & Outcomes optimises the value of drugs and medical devices through innovative strategies and tactics that reflect evolving evidentiary, regulatory, and reimbursement requirements. Our expert team establishes and communicates a product’s unique clinical and economic outcomes to achieve success in today’s dynamic and patient-centric healthcare environment.