PRO Task Force: Use of Existing Instruments and their Modification

Goal:
Development of good research practices for assessing existing instruments for use in label claim studies, identifying the concepts contained in existing instruments, obtaining patient input if not used in original instrument, development of instruments used in a battery and modification of these instruments including use in new measurement concept, use in new population, change in item content, and assessment of measurement properties for revised instruments.

Chair: Margaret Rothman, PhD,
Executive Director, WW Patient-reported Outcomes Center of Excellence, Johnson & Johnson Pharmaceutical Services, LLC, Washington, GA, USA

13th Annual International Meeting Presentation

Third Plenary:  Patient Reported Outcomes: Implementing Good Research Practices
Plenary Presentation
Plenary Program

Workshop 10: Patient Reported Outcomes – Selecting, Evaluating and Documenting Support for Existing Instruments for Labeling Claims: Content Validity
Wednesday, May 7 8:30 – 9:30 AM in the Essex Ballroom (Mezzanine Level)
Workshop Presentation
Workshop Program

Leadership Team:
Laurie B. Burke, RPh, MPH, Director, Study Endpoints and Label Developments, Office of New Drugs, CDER, FDA, Washington, DC
Pennifer Erickson, PhD, Founder, OLGA, State College, PA
Nancy Kline Leidy, PhD, Senior Vice President of Scientific Affairs, United BioSource, Bethesda, MD
Donald Patrick, PhD, MSPH, Professor, University of Washington, Seattle, WA
Charles Petrie, PhD, Senior Director/Group Leader, Worldwide Outcomes Research – Neurosciences, Groton, CT

Background:
A wide variety of Health Technology Assessors at different levels of the health system and a number of other stakeholders in medical product use consider PRO studies of efficacy and effectiveness of medical products. Although formal guidelines may not be available, good research practices in PRO development are important in improving the value of this information to decision-makers.

In July 2005, the Committee for Medicinal Products for Human Use of the European Medicines Agency issued a Reflection Paper on the Regulatory Guidance for the Use of Health Related Quality of Life (HRQL) Measures in the Evaluation of Medicinal Products. Recommendations of this Paper came into effect January 2006. The scope of the EMEA document was to discuss the place that a HRQL type of PRO may have in the drug evaluation process and to give some broad recommendations on its use in the context of already existing guidance documents. The EMEA reflection paper and existing documents also point out the need for standardization of research practices in the evaluation of medical products using PROs.

In February 2006, the U.S. Food and Drug Administration issued a draft version of Guidance for Industry titled “Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims.”

Draft FDA Guidance for Industry - Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims Draft Guidance

Chapter IV: Evaluating PRO Instruments, Part D: Modification of an Existing Instrument:

The adequacy of a PRO instrument depends on the developmental history and demonstrated measurement properties. Sponsors need to identify all endpoint measurement goals to provide the basis for approval or claim substantiation. If sponsors determine that no existing instrument is applicable or adequate for their product, they can develop or modify an existing instrument. The FDA generally plans to evaluate the modified instrument in the same way that it evaluates a new instrument. It is important to have documentation supporting the original development process, modifications made and updated assessment of the measurement properties.

The FDA identifies 5 specific issues to address when modifying an existing PRO instrument:

1. Revised Measurement Concept
2. Application to a New Population or Condition
3. Changed Item Content or Instrument Format
4. Changed Mode of Administration
5. Changed Culture or Language of Application

The final two issues are addressed by the PRO Task Forces bearing those names.

Task Forces Index

Use of Existing Instruments and their Modification TF Members Access Only

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