Interpretation of credibility evaluation tools for minimal important difference in patient-reported outcomes based on anchoring methods_Chinese Journal of Evidence-Based Medicine

Authors：

LI Meichen ¹ ,  WEN Zehuai ^2,3 ,  ZHOU Li ²

1. Second Clinical School, Guangzhou University of Traditional Chinese Medicine, Guangzhou 510405, P. R. China;
2. Clinical Research Center of the Second Affiliated Hospital, Guangzhou University of Traditional Chinese Medicine (Guangdong Provincial Hospital of Traditional Chinese Medicine), Guangzhou 510120, P. R. China;
3. Science and Technology Innovation Center, Guangzhou University of Traditional Chinese Medicine, Guangzhou 510405, P. R. China;

Corresponding author：

WEN Zehuai, Email: wenzh@gzucm.edu.cn; ZHOU Li, Email: zhouli@gzucm.edu.cn

Keywords：

Anchoring method; Patient-reported outcome; Minimal important difference; Credibility evaluation

DOI：

10.7507/1672-2531.202410066

Video：

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The estimation of the minimal important difference (MID) in patient-reported outcomes (PRO) relies on various selection principles and statistical methodologies, resulting in varying degrees of credibility among studies. When applying these findings, it is crucial to consider their evaluation outcomes. In the context of widely accepted MID studies based on the anchoring method, the credibility of the MID of PRO is influenced by the selection of anchors and the statistical methods employed for estimation. Variations in the anchors utilized, differences in clinical trial designs, disparities in the characteristics of measurement subjects and environment, as well as the control of biases in studies, can all contribute to inconsistencies in the MID of PRO. In response to this, McMaster University in Canada has developed a credibility evaluation tool specifically for MID studies in PRO. The tool comprises five core items and four additional items. The five core items encompass an evaluation framework that assesses: (1) Is the patient or necessary proxy responding directly to both the PRO and the anchor? (2) Is the anchor easily understandable and relevant for patients or necessary proxy? (3)Has the anchor shown good correlation with the PRO? (4) Is the MID precise? (5) Does the threshold or difference between groups on the anchor used to estimate the MID reflect a small but important difference? The four additional items concerning transition-rated anchors assess: (1) Is the amount of elapsed time between baseline and follow-up measurement for MID estimation optimal? (2) Does the transition item have a satisfactory correlation with the PRO score at follow-up? (3) Does the transition item correlate with the PRO score at baseline? (4) Is the correlation of the transition item with the PRO change score appreciably greater than the correlation of the transition item with the PRO score at follow-up? Given the relative weights of each item in the tool are uncertain and environment-dependent, items are not scored; instead, an overall judgment is made using a qualitative rating approach. This article introduces the specific items of this tool and illustrates the evaluation process through a case study to improve its use in optimizing PRO results presentation and interpretation in clinical trials, reviews, assessments, and guidelines.

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2.	Acquadro C, Berzon R, Dubois D, et al. Incorporating the patient's perspective into drug development and communication: an ad hoc task force report of the Patient-Reported Outcomes (PRO) Harmonization Group meeting at the Food and Drug Administration, February 16, 2001. Value Health, 2003, 6(5): 522-531.
3.	Weinfurt KP, Reeve BB. Patient-reported outcome measures in clinical research. JAMA, 2022, 328(5): 472-473.
4.	U. S. Department of Health and Human Services FDA Center for Drug Evaluation and Research. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance. Health Qual Life Outcomes, 2006, 4: 79.
5.	Schünemann HJ, Guyatt GH. Commentary-goodbye M(C)ID! Hello MID, where do you come from. Health Serv Res, 2005, 40(2): 593-597.
6.	Guyatt GH, Osoba D, Wu AW, et al. Methods to explain the clinical significance of health status measures. Mayo Clin Proc, 2002, 77(4): 371-383.
7.	杨丽虹, 刘少南, 吴大嵘, 等. 最小临床意义差值的概念及其估算方法. 中国循证医学杂志, 2020, 20(11): 1345-1352.
8.	Devji T, Guyatt GH, Lytvyn L, et al. Application of minimal important differences in degenerative knee disease outcomes: a systematic review and case study to inform BMJ Rapid Recommendations. BMJ Open, 2017, 7(5): e015587.
9.	Devji T, Carrasco-Labra A, Qasim A, et al. Evaluating the credibility of anchor based estimates of minimal important differences for patient reported outcomes: instrument development and reliability study. BMJ, 2020, 369: m1714.
10.	Escobar A, García Pérez L, Herrera-Espiñeira C, et al. Total knee replacement; minimal clinically important differences and responders. Osteoarthritis Cartilage, 2013, 21(12): 2006-2012.
11.	Hägg O, Fritzell P, Nordwall A, et al. The clinical importance of changes in outcome scores after treatment for chronic low back pain. Eur Spine J, 2003, 12(1): 12-20.
12.	Terwee CB, Roorda LD, Dekker J, et al. Mind the MIC: large variation among populations and methods. J Clin Epidemiol, 2010, 63(5): 524-534.
13.	Hayes RP, Schultz EM, Naegeli AN, et al. Test-retest, responsiveness, and minimal important change of the ability to perform physical activities of daily living questionnaire in individuals with type 2 diabetes and obesity. Diabetes Technol Ther, 2012, 14(12): 1118-1125.
14.	Colwell HH, Mathias SD, Turner MP, et al. Psychometric evaluation of the FACT Colorectal Cancer Symptom Index (FCSI-9): reliability, validity, responsiveness, and clinical meaningfulness. Oncologist, 2010, 15(3): 308-316.
15.	Devillier P, Chassany O, Vicaut E, et al. The minimally important difference in the Rhinoconjunctivitis Total Symptom Score in grass-pollen-induced allergic rhinoconjunctivitis. Allergy, 2014, 69(12): 1689-1695.
16.	Wang Y, Devji T, Carrasco-Labra A, et al. A step-by-step approach for selecting an optimal minimal important difference. BMJ, 2023, 381: e073822.
17.	Bird SB, Dickson EW. Clinically significant changes in pain along the visual analog scale. Ann Emerg Med, 2001, 38(6): 639-643.
18.	Lopez BL, Flenders P, Davis-Moon L, et al. Clinically significant differences in the visual analog pain scale in acute vasoocclusive sickle cell crisis. Hemoglobin, 2007, 31(4): 427-432.
19.	Kelly AM. The minimum clinically significant difference in visual analogue scale pain score does not differ with severity of pain. Emerg Med J, 2001, 18(3): 205-207.
20.	Gallagher EJ, Liebman M, Bijur PE. Prospective validation of clinically important changes in pain severity measured on a visual analog scale. Ann Emerg Med, 2001, 38(6): 633-638.
21.	Gallagher EJ, Bijur PE, Latimer C, et al. Reliability and validity of a visual analog scale for acute abdominal pain in the ED. Am J Emerg Med, 2002, 20(4): 287-290.
22.	Bijur PE, Latimer CT, Gallagher EJ. Validation of a verbally administered numerical rating scale of acute pain for use in the emergency department. Acad Emerg Med, 2003, 10(4): 390-392.
23.	Mark M, Au T, Choi Y, et al. The minimum clinically significant difference in visual analogue scale pain score in a local emergency setting. J Emerg Med, 2009, 16(4): 233-236.
24.	Ward MM, Guthrie LC, Alba MI. Clinically important changes in individual and composite measures of rheumatoid arthritis activity: thresholds applicable in clinical trials. Ann Rheum Dis, 2015, 74(9): 1691-1696.
25.	Wang Y, Devji T, Qasim A, et al. A systematic survey identified methodological issues in studies estimating anchor-based minimal important differences in patient-reported outcomes. J Clin Epidemiol, 2022, 142: 144-151.
26.	Ingelsrud LH, Terwee CB, Terluin B, et al. Meaningful change scores in the knee injury and osteoarthritis outcome score in patients undergoing anterior cruciate ligament reconstruction. Am J Sports Med, 2018, 46(5): 1120-1128.
27.	Devji T, Johnston BC, Patrick DL, et al. Presentation approaches for enhancing interpretability of patient-reported outcomes (PROs) in meta-analysis: a protocol for a systematic survey of Cochrane reviews. BMJ Open, 2017, 7(9): e017138.
28.	Zhang Y, Zhang S, Thabane L, et al. Although not consistently superior, the absolute approach to framing the minimally important difference has advantages over the relative approach. J Clin Epidemiol, 2015, 68(8): 888-894.
29.	Busse JW, Craigie S, Sadeghirad B, et al. Management of acute musculoskeletal pain (excluding low back pain): protocol for a systematic review and network meta-analysis of randomised trials. BMJ Open, 2019, 9(4): e024441.
30.	Carrasco-Labra A, Devji T, Qasim A, et al. Serious reporting deficiencies exist in minimal important difference studies: current state and suggestions for improvement. J Clin Epidemiol, 2022, 150: 25-32.

1. Deshpande PR, Rajan S, Sudeepthi BL, et al. Patient-reported outcomes: a new era in clinical research. Perspect Clin Res, 2011, 2(4): 137-144.
2. Acquadro C, Berzon R, Dubois D, et al. Incorporating the patient's perspective into drug development and communication: an ad hoc task force report of the Patient-Reported Outcomes (PRO) Harmonization Group meeting at the Food and Drug Administration, February 16, 2001. Value Health, 2003, 6(5): 522-531.
3. Weinfurt KP, Reeve BB. Patient-reported outcome measures in clinical research. JAMA, 2022, 328(5): 472-473.
4. U. S. Department of Health and Human Services FDA Center for Drug Evaluation and Research. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance. Health Qual Life Outcomes, 2006, 4: 79.
5. Schünemann HJ, Guyatt GH. Commentary-goodbye M(C)ID! Hello MID, where do you come from. Health Serv Res, 2005, 40(2): 593-597.
6. Guyatt GH, Osoba D, Wu AW, et al. Methods to explain the clinical significance of health status measures. Mayo Clin Proc, 2002, 77(4): 371-383.
7. 杨丽虹, 刘少南, 吴大嵘, 等. 最小临床意义差值的概念及其估算方法. 中国循证医学杂志, 2020, 20(11): 1345-1352.
8. Devji T, Guyatt GH, Lytvyn L, et al. Application of minimal important differences in degenerative knee disease outcomes: a systematic review and case study to inform BMJ Rapid Recommendations. BMJ Open, 2017, 7(5): e015587.
9. Devji T, Carrasco-Labra A, Qasim A, et al. Evaluating the credibility of anchor based estimates of minimal important differences for patient reported outcomes: instrument development and reliability study. BMJ, 2020, 369: m1714.
10. Escobar A, García Pérez L, Herrera-Espiñeira C, et al. Total knee replacement; minimal clinically important differences and responders. Osteoarthritis Cartilage, 2013, 21(12): 2006-2012.
11. Hägg O, Fritzell P, Nordwall A, et al. The clinical importance of changes in outcome scores after treatment for chronic low back pain. Eur Spine J, 2003, 12(1): 12-20.
12. Terwee CB, Roorda LD, Dekker J, et al. Mind the MIC: large variation among populations and methods. J Clin Epidemiol, 2010, 63(5): 524-534.
13. Hayes RP, Schultz EM, Naegeli AN, et al. Test-retest, responsiveness, and minimal important change of the ability to perform physical activities of daily living questionnaire in individuals with type 2 diabetes and obesity. Diabetes Technol Ther, 2012, 14(12): 1118-1125.
14. Colwell HH, Mathias SD, Turner MP, et al. Psychometric evaluation of the FACT Colorectal Cancer Symptom Index (FCSI-9): reliability, validity, responsiveness, and clinical meaningfulness. Oncologist, 2010, 15(3): 308-316.
15. Devillier P, Chassany O, Vicaut E, et al. The minimally important difference in the Rhinoconjunctivitis Total Symptom Score in grass-pollen-induced allergic rhinoconjunctivitis. Allergy, 2014, 69(12): 1689-1695.
16. Wang Y, Devji T, Carrasco-Labra A, et al. A step-by-step approach for selecting an optimal minimal important difference. BMJ, 2023, 381: e073822.
17. Bird SB, Dickson EW. Clinically significant changes in pain along the visual analog scale. Ann Emerg Med, 2001, 38(6): 639-643.
18. Lopez BL, Flenders P, Davis-Moon L, et al. Clinically significant differences in the visual analog pain scale in acute vasoocclusive sickle cell crisis. Hemoglobin, 2007, 31(4): 427-432.
19. Kelly AM. The minimum clinically significant difference in visual analogue scale pain score does not differ with severity of pain. Emerg Med J, 2001, 18(3): 205-207.
20. Gallagher EJ, Liebman M, Bijur PE. Prospective validation of clinically important changes in pain severity measured on a visual analog scale. Ann Emerg Med, 2001, 38(6): 633-638.
21. Gallagher EJ, Bijur PE, Latimer C, et al. Reliability and validity of a visual analog scale for acute abdominal pain in the ED. Am J Emerg Med, 2002, 20(4): 287-290.
22. Bijur PE, Latimer CT, Gallagher EJ. Validation of a verbally administered numerical rating scale of acute pain for use in the emergency department. Acad Emerg Med, 2003, 10(4): 390-392.
23. Mark M, Au T, Choi Y, et al. The minimum clinically significant difference in visual analogue scale pain score in a local emergency setting. J Emerg Med, 2009, 16(4): 233-236.
24. Ward MM, Guthrie LC, Alba MI. Clinically important changes in individual and composite measures of rheumatoid arthritis activity: thresholds applicable in clinical trials. Ann Rheum Dis, 2015, 74(9): 1691-1696.
25. Wang Y, Devji T, Qasim A, et al. A systematic survey identified methodological issues in studies estimating anchor-based minimal important differences in patient-reported outcomes. J Clin Epidemiol, 2022, 142: 144-151.
26. Ingelsrud LH, Terwee CB, Terluin B, et al. Meaningful change scores in the knee injury and osteoarthritis outcome score in patients undergoing anterior cruciate ligament reconstruction. Am J Sports Med, 2018, 46(5): 1120-1128.
27. Devji T, Johnston BC, Patrick DL, et al. Presentation approaches for enhancing interpretability of patient-reported outcomes (PROs) in meta-analysis: a protocol for a systematic survey of Cochrane reviews. BMJ Open, 2017, 7(9): e017138.
28. Zhang Y, Zhang S, Thabane L, et al. Although not consistently superior, the absolute approach to framing the minimally important difference has advantages over the relative approach. J Clin Epidemiol, 2015, 68(8): 888-894.
29. Busse JW, Craigie S, Sadeghirad B, et al. Management of acute musculoskeletal pain (excluding low back pain): protocol for a systematic review and network meta-analysis of randomised trials. BMJ Open, 2019, 9(4): e024441.
30. Carrasco-Labra A, Devji T, Qasim A, et al. Serious reporting deficiencies exist in minimal important difference studies: current state and suggestions for improvement. J Clin Epidemiol, 2022, 150: 25-32.

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