In February 2006, the United States Food and Drug Administration (FDA) issued a draft Guidance for the Industry on Patient-Reported Outcome (PRO) Measures: Use in Medical Product Development to support Labeling Claims. This document described how PRO measures are used as effectiveness end points and how PRO study results are used to support product label claims and advertising. This document included discussions on PRO instrument evaluation, study design, and data analysis.

ISPOR membership, coordinated by the PRO / QOL Information in Regulatory and Health Decision Working Group of the ISPOR PRO Special Interest Group, provided comments to the FDA on this draft guidance. The preponderance of comments and concerns regarded evaluation of PRO instruments, specifically creation of the PRO instrument, choice of methods for interpretation, modification of existing instruments, development of PRO instruments for specific populations, changing culture or language of application, and changing mode of administration (specifically electronic PRO instruments). These comments by ISPOR members identified a need for clarity on specific issues. Repeatedly the need for standardization of instrument development, process of administration, and methods for assessment and use was encouraged.

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.

Finally 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.

ISPOR, through its Good Research Practice Task Force Reports (e.g. Modeling Task Force Report CEA in Clinical Trials Task Force Report, Budget Impact Task Force Report) has helped to ‘standardize’ methodologies for outcomes research. Thus, it seems appropriate that key issues not addressed by other bodies outside ISPOR and issues already being addressed within ISPOR be considered for ISPOR action.

It is recommended that ISPOR Task Forces on Patient Reported Outcomes Measures on the following topics with the indicated objectives be developed.

PRO GOOD RESEARCH PRACTICES TOPICS

PRO Instrument Creation
Objective: Development of good research practices for creation of a new PRO instrument including identification of claims, selection of concepts, generation of items, choice of data collection method, choice of recall period, choice of response options, evaluation of patient understanding, development of format / instructions / training, identification of preliminary scoring of items / domains, assessment of respondent and administrator burden, confirmation of conceptual framework, assessment of measurement properties (reliability, validity, and ability to detect change) and finalization of the instrument.

Use of Existing PRO Instruments and their Modification (POSSIBLE PRIORITY)
Objective: 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, change in mode of administration. Assessment of measurement properties for revised instruments.  

Changing Culture or Language of PRO Application (Diane Wild already has a group)
Objective: Development of good research practices for adapting or translating an instrument from one language/culture to another including translation measurement properties and acceptability of pooling data in multi-language trials

Changing Mode of Administration of PRO Instrument including ePRO (Stephen Coons)
Objective: Development of good research practices for changing the mode of administration (e.g. paper to computer, supervised to self questionnaire) and use of electronic PRO instruments.

Development of PRO Instruments for Children and Youth
Objective: Development of good research practices for development of PRO instruments for children and youth including age-related vocabulary, language, comprehension, duration of recall and the cognitively impaired including proxy reporting.

PRODUCTS

The following PRO Good Research Practices would be published in Value in Health, at the ISPOR website, and in a future ISPOR publication on good research practices.

• Good Research Practices for Creation of a new PRO Instrument
• Good Research Practices for Assessing Existing PRO Instruments
• Good Research Practices For Adapting Or Translating An Instrument
• Good Research Practices For Changing The Mode Of Administration
• Good Research Practices For Development Of PRO Instruments For Children And Youth

PRO GOOD RESEARCH PRACTICES MANAGEMENT

Management
The PRO / QOL Information in Regulatory and Health Decision Working Group of the ISPOR PRO Special Interest Group would have oversight responsibility in the development of each PRO Good Research Practice Task Force including identifying the Task Force chairs. Each Task Force chair would be responsible for the selection of the Task Force members. Interested ISPOR members would be asked to participate on either the Task Force or the Task Force Review Group (who will review & comment on the draft good research practices).

Timeline
The development of these PRO good research practices by each Task Force would be approximately 18 months.

Resource
Requirements Resources needed are staff coordination, teleconferences, and one face-to-face meeting per task force