Skip to main content
Key Specialties
Cardiology Diabetology Endocrinology Gastroenterology Geriatrics and Gerontology Gynecology and Obstetrics Hematology Infectious Disease Internal Medicine Nephrology Neurology Oncology Pediatrics Respiratory Medicine Rheumatology Surgery
Congress Coverage
2024 ACC Congress 2023 ESMO Congress 2023 EASD Congress 2023 ERS Congress 2023 ESC Congress 2023 EHA Congress 2023 ASCO Annual Meeting
Clinical Collections
Advances in Alzheimer’s

At a glance: The ONWARDS insulin icodec trials

A round-up of the ONWARDS phase 3 clinical trials evaluating the novel weekly insulin icodec in people with diabetes.
ADVANCED SEARCH
Log in
Top
PharmacoEconomics
Published in: PharmacoEconomics 5/2008

01-05-2008 | Leading Article

Perspectives on Patient-Reported Outcomes

Content Validity and Qualitative Research in a Changing Clinical Trial Environment

Authors: Dr Nancy Kline Leidy, Margaret Vernon

Published in: PharmacoEconomics | Issue 5/2008

Login to get access

Abstract

Patient-reported outcomes (PROs) represent the voice of the patient in drug and device evaluation. As such, the outcomes selected for evaluation must be relevant to the patient, and the instruments used to capture them must have sound measurement properties. This paper discusses the role of qualitative research methods in assuring PRO clarity and content validity in a clinical trial environment characterized by evolving regulatory policy, new advances in technology and increasingly diverse samples within global clinical trials. Three important PRO measurement issues influenced by these changes are addressed: (i) endpoint selection and instrument development; (ii) the adaptation of instruments for electronic administration; and (iii) conceptual equivalence of measures across diverse samples. These are viewed as interrelated issues of content validity that can be addressed through the appropriate and rigorous application of qualitative research methods. Focus groups, cognitive debriefing, user acceptance testing and translation methodologies are discussed as ways to address and document the content validity of PRO instruments and ensure the empirical data representing the voice of the patient is sound.
Literature
1.
2.
3.
4.
Hays RD, Anderson R, Revicki D. Psychometric considerations in evaluating health-related quality of life measures. Qual Life Res 1993 Dec; 2 (6): 441–449PubMedCrossRef
5.
Nunnally JC, Bernstein I. Psychometric theory. 3rd ed. New York: McGraw-Hill, 1994
6.
Revicki DA. EDA draft guidance and health-outcomes research. Lancet 2007 Feb 17; 369 (9561): 540–542PubMedCrossRef
7.
8.
Dicicco-Bloom B, Crabtree BF. The qualitative research interview. Med Educ 2006 Apr; 40 (4): 314–321PubMedCrossRef
9.
Draper AK. The principles and application of qualitative research. Proc Nutr Soc 2004 Nov; 63 (4): 641–646PubMedCrossRef
10.
Tobin GA, Begley CM. Methodological rigour within a qualitative framework. J Adv Nurs 2004 Nov; 48 (4): 388–396PubMedCrossRef
11.
Krueger RA, Casey MA. Focus groups: a practical guide for applied research. 3rd ed. Thousand Oaks (CA): Sage Publications, Inc., 2000
12.
Morgan DL. Focus groups as qualitative research. 2nd ed. Thousand Oaks (CA): Sage Publications, Inc., 1997
13.
Stewart DW, Shamdasani PN, Rook DW. Focus groups: theory and practice. 2nd ed. Thousand Oaks (CA): Sage Publications, Inc., 2006
14.
Creswell JW. Qualitative inquiry and research design: choosing among five methods. Thousand Oaks (CA): Sage Publications, Inc., 1998
15.
Graneheim UH, Lundman B. Qualitative content analysis in nursing research: concepts, procedures and measures to achieve trustworthiness. Nurse Educ Today 2004 Feb; 24 (2): 105–112PubMedCrossRef
16.
Willis G. Cognitive interviewing. Thousand Oaks (CA): Sage Publications, Inc., 2005
17.
Aquilino WS. Interview mode effects in surveys of drug and alcohol use: a field experiment. Public Opin Q 1994; 58 (2): 210–240CrossRef
18.
Dillman D. Mail and internet surveys. 2nd ed. New York: Wiley, 2000
19.
McCabe SE, Boyd CJ, Couper MP, et al. Mode effects for collecting alcohol and other drug use data: web and US mail. J Stud Alcohol 2002 Nov; 63 (6): 755–761PubMed
20.
Newman JC, Des Jarlais DC, Turner CF, et al. The differential effects of face-to-face and computer interview modes. Am J Public Health 2002 Feb; 92 (2): 294–297PubMedCrossRef
21.
Tourangeau R, Smith W. Asking sensitive questions: the impact of data collection mode, question format, and question context. Public Opin Q 1996; 60 (2): 275–304CrossRef
22.
23.
Fayers PM. Applying item response theory and computer adaptive testing: the challenges for health outcomes assessment. Qual Life Res 2007; 16 Suppl. 1: 187–194PubMedCrossRef
24.
Holland PW, Wainer H. Differential item functioning. London: Lawrence Erlbaum, 1993
25.
Rogers HJ, Swaminathan H, Hambleton RK. Fundamentals of item response theory. Thousand Oaks (CA): Sage Publications, Inc., 1991
26.
Cella D, Lloyd S, Wright B. Cross-cultural instrument equating: current research and future directions. In: Spilker B, editor. Quality of life and pharmacoeconomics in clinical trials. 2nd ed. Philadelphia (PA): Lippincott-Raven, 1996: 707–715
27.
Flaherty JA, Gaviria FM, Pathak D, et al. Developing instruments for cross-cultural psychiatric research. J Nerv Ment Dis 1988 May; 176 (5): 257–263PubMedCrossRef
28.
Herdman M, Fox-Rushby J, Badia X. ‘Equivalence’ and the translation and adaptation of health-related quality of life questionnaires. Qual Life Res 1997 Apr; 6 (3): 237–247PubMedCrossRef
29.
Van de Vijver F. Towards a theory of bias and equivalence. ZUMA Nachrichten Spezial 1998; 42: 41–65
30.
Zheng YP, Wei LA, GoaL G, et al. Applicability of the Chinese beck depression inventory. Compr Psychiatry 1988 Sep–Oct; 29 (5): 484–489PubMedCrossRef
31.
Willgerodt MA. Using focus groups to develop culturally relevant instruments. West J Nurs Res 2003 Nov; 25 (7): 798–814PubMedCrossRef
32.
Cull A, Sprangers M, Bjordal K, et al. EORTC Quality of Life Group translation procedure. 2nd ed. Brussels: EORTC Quality of Life Group, 2002
33.
Eremenco SL, Cella D, Arnold BJ. A comprehensive method for the translation and cross-cultural validation of health status questionnaires. Eval Health Prof 2005 Jun; 28 (2): 212–232PubMedCrossRef
34.
Guillemin F, Bombardier C, Beaton D. Cross-cultural adaptation of health-related quality of life measures: literature review and proposed guidelines. J Clin Epidemiol 1994; 47 (12): 1465–1466CrossRef
35.
Sartorius N, Kuyken W. Translation of health status instruments. In: Orley J, Kuyken W, editors. Quality of life assessment: international perspectives. Berlin Heidelberg: Springer Verlag, 1994: 3–18
36.
Wild D, Grove A, Martin M, et al. Principles of good practice for the translation and cultural adaptation process for patient-reported outcomes (PRO) measures: report of thelSPOR Task Force for Translation and Cultural Adaptation. Value Health 2005 Mar-Apr; 8 (2): 94–104PubMedCrossRef
37.
Hunt S. Cross-cultural issues in the use of quality of life measures in randomized controlled trials. In: Staquet M, Hays R, Fayers P, editors. Quality of life assessment in clinical trials. Oxford: Oxford University Press, 1998: 51–68
38.
Lenderking WR. Comments on the ISPOR Task Force report on translation and adaptation of outcomes measures: guidelines and the need for more research. Value Health 2005 Mar-Apr; 8 (2): 92–93PubMedCrossRef
39.
Swaine-Verdier A, Doward LC, Hagell P, et al. Adapting quality of life instruments. Value Health 2004 Sep–Oct; 7 Suppl. 1: S27–S30PubMedCrossRef
Metadata
Title
Perspectives on Patient-Reported Outcomes
Content Validity and Qualitative Research in a Changing Clinical Trial Environment
Authors
Dr Nancy Kline Leidy
Margaret Vernon
Publication date
01-05-2008
Publisher
Springer International Publishing
Published in
PharmacoEconomics / Issue 5/2008
Print ISSN: 1170-7690
Electronic ISSN: 1179-2027
DOI
https://doi.org/10.2165/00019053-200826050-00002

Other articles of this Issue 5/2008

PharmacoEconomics 5/2008 Go to the issue

Review Article

Economic Evaluation of Interventions to Treat Opiate Dependence

Editorial

Cost-Effectiveness Analysis

Review Article

Economic Evaluations in Rheumatoid Arthritis

Original Research Article

Updated Estimates of Survival and Cost Effectiveness for Imatinib versus Interferon-a Plus Low-Dose Cytarabine for Newly Diagnosed Chronic-Phase Chronic Myeloid Leukaemia

Original Research Article

A Multicentre, Prospective Study to Evaluate Costs of Septic Patients in Brazilian Intensive Care Units

Review Article

Methodological Issues in Evaluating Cost Effectiveness of Adjuvant Aromatase Inhibitors in Early Breast Cancer