Clinical Outcome Assessment

Mission

To provide leadership in the use of clinical outcome assessments (patient reported, clinician reported, observational reported, and performance outcomes) in HEOR and to encourage harmonization between all stakeholders of interest.

Goal

• Provide a centralized platform to exchange ongoing ideas relating to the development, implementation and validation of clinical outcomes assessments (COA) measures
• Discuss novel ideas by outlining existing evidence gaps in COA measurement science
• To synthesize COA development practices that allow for the use of scientifically rigorous endpoints.
• Expand the current knowledge base and develop new industry standards for COA use as primary or secondary endpoints in a regulated research environment
• Identify new evidence generation opportunities that will benefit the research, clinical, and patient communities 

Background

The FDA defines a clinical outcome assessment (COA) as “a measure that describes or reflects how a patient feels, functions, or survives.” The four types of COAs are: clinician-reported outcome (ClinRO), observer-reported outcome (ObsRO), patient-reported outcome (PRO), and performance outcome (PerfO). To see the FDA’s and NIH’s definition of each type, click here.

The growing use and increasing scrutiny of COA measures in pharmaceutical research has led to a need for more precision in the generation and interpretation of such data.  Indeed, if the outcomes research literature is reviewed, it demonstrates an almost exponential growth in the development and use of COA measures in drug/medical device research. Those working in the pharmaceutical, medical devices, and diagnostics industries rely upon robust and valid ways to collect COA data. Yet there are few standardized COA measures across stakeholders, due to the ongoing challenges that surround robust COA data capture, analyses, and interpretation.

Leadership

Working Groups:

Key Project

Value of Clinical Outcome Assessment Data to All Stakeholders

Co-Chairs:

• Lynda Doward, MRES, European Head, RTI-Health Solutions, UK
• Bryan Bennett, PhD, WWHEOR Asset/Indication Lead, Bristol-Myers Squibb, UK

Member Engagement Project: Standardizing Clinical Outcome Assessments for Real World Studies

Co-Chairs:

• Angela Rylands, PhD, Senior International Outcomes Research Manager, Kyowa Kirin, UK
• Ana Maria Rodriguez, PhD, Director, RWE Patient-Centered Endpoints, Center of Excellence, IQVIA

Webinars

1. Patient-Reported Outcomes Webinar 1: What are PROs and HRQOL Tools? (September 2020)
2. Patient-Reported Outcomes Webinar 2: Generating PRO Data – and Using It (October 2020)
3. Patient Reported Outcomes Webinar 3: Developing New Patient-Reported Outcomes Instruments (October 2020)

Work Products

1. ISPOR Virtual 2021 Forum: “15% of the scale range” Universally Applicable to Define “MID” and Clinical Relevance of Patient-Reported Treatment Benefits?
2. COA SIG Virtual Workshop:  Patient-focused Endpoints: Developing and Analyzing Patient-Reported Outcomes for Optimal Interpretability (April 2021)
3. ISPOR Europe 2020 Issue Panel: How Will New Guidance Impact the Statistical Analyses and Interpretation for Patient-Reported Outcomes (PRO) in the Context of HA and HTA Submissions
4. COA SIG Virtual Roundtable: Guiding Principles for Using COAs in Real World Studies with representatives from EMA, US FDA, and the PRO Consortium (October 2019)

Join this Special Interest Group