Systematic collection of patient reported outcome research data: A checklist for clinical research professionals

Contemporary Clinical Trials

Volumn 48, Issue , 2016, Pages 21-29

  Wehrlen, Leslie ^a   Krumlauf, Mike ^a   Ness, Elizabeth ^b   Maloof, Damiana ^c   Bevans, Margaret ^a  

^a NATIONAL INSTITUTES OF HEALTH   (United States)

^b NATIONAL CANCER INSTITUTE   (United States)

^c NEWTON WELLESLEY HOSPITAL   (United States)

Author keywords

Clinical trials; Guidance; Patient diary; Patient reported outcome; Questionnaire; Self reported data

Indexed keywords

ARTICLE; CHECKLIST; CLINICAL RESEARCH; FEEDBACK SYSTEM; FOOD AND DRUG ADMINISTRATION; HUMAN; OUTCOME ASSESSMENT; PRACTICE GUIDELINE; SYSTEMATIC REVIEW; CLINICAL TRIAL (TOPIC); INFORMATION PROCESSING; MEDICAL RESEARCH; PATIENT-REPORTED OUTCOME; PERSONNEL; SELF REPORT; STANDARDS;

BIOMEDICAL RESEARCH; CHECKLIST; CLINICAL TRIALS AS TOPIC; DATA COLLECTION; HUMANS; PATIENT REPORTED OUTCOME MEASURES; RESEARCH PERSONNEL; SELF REPORT;

EID: 84962206401     PISSN: 15517144     EISSN: 15592030     Source Type: Journal    
DOI: 10.1016/j.cct.2016.03.005     Document Type: Article

References (64)

1. Basch E., et al. Adverse symptom event reporting by patients vs clinicians: relationships with clinical outcomes. J. Natl. Cancer Inst. 2009, 101(23):1624-1632.
2. Patrick D.L., et al. Content validity-establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO good research practices task force report: part 1-eliciting concepts for a new PRO instrument. Value Health 2011, 14(8):967-977.
3. 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for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance.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.
4. Services, U.S.D.o.H.a.H. Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims 2009, F.a.D. Administration (Ed.).
5. Gralla R.J. Quality-of-life considerations in patients with advanced lung cancer: effect of topotecan on symptom palliation and quality of life. Oncologist 2004, 9(Suppl. 6):14-24.
6. Johnson J.R., Temple R. Food and Drug Administration requirements for approval of new anticancer drugs. Cancer Treat. Rep. 1985, 69(10):1155-1159.
7. Congress, t. The Patient Protection and Affordable Care Act. Public Law 111-148 2010, U.S.G.P. Office (Ed.).
8. Vodicka E., et al. Inclusion of patient-reported outcome measures in registered clinical trials: evidence from ClinicalTrials.gov (2007-2013). Control. Clin. Trials 2015, 43:1-9.
9. Gnanasakthy A., et al. Potential of patient-reported outcomes as nonprimary endpoints in clinical trials. Health Qual. Life Outcomes 2013, 11:83.
10. Ganz P.A., Gotay C.C. Use of patient-reported outcomes in phase III cancer treatment trials: lessons learned and future directions. J. Clin. Oncol. 2007, 25(32):5063-5069.
11. Olschewski M., et al. Quality of life assessment in clinical cancer research. Br. J. Cancer 1994, 70(1):1-5.
12. Calvert M., et al. Patient-reported outcome (PRO) assessment in clinical trials: a systematic review of guidance for trial protocol writers. PLoS One 2014, 9(10).
13. Gerrish K., Clayton J. Promoting evidence-based practice: an organizational approach. J. Nurs. Manag. 2004, 12(2):114-123.
14. Bryant D., Fernandes N. Measuring patient outcomes: a primer. Injury 2011, 42(3):232-235.
15. Lipscomb J.G., C., Snyder C. Outcomes Assessment in Cancer Measures, Methods, and Applications 2005, Cambridge University Press, Cambridge, UK.
16. Calvert M.J., Freemantle N. RESEARCH NOTE Use of health-related quality of life in prescribing research. Part 2: methodological considerations for the assessment of health-related quality of life in clinical trials. J. Clin. Pharm. Ther. 2004, 29(1):85-94.
17. Braido F., et al. Specific recommendations for PROs and HRQoL assessment in allergic rhinitis and/or asthma: a GA(2)LEN taskforce position paper. Allergy 2010, 65(8):959-968.
18. Osoba D. The Quality of Life Committee of the Clinical Trials Group of the National Cancer Institute of Canada: organization and functions. Qual. Life Res. 1992, 1(3):211-218.
19. Kyte D.G., et al. Patient reported outcomes (PROs) in clinical trials: is 'in-trial' guidance lacking? A systematic review. PLoS One 2013, 8(4).
20. Patrick D. Reporting of patient-reported outcomes in randomized trials: the CONSORT PRO extension. Value Health 2013, 16(4):455-456.
21. Eremenco S., et al. PRO data collection in clinical trials using mixed modes: report of the ISPOR PRO mixed modes good research practices task force. Value Health 2014, 17(5):501-516.
22. Calvert M., et al. Reporting of patient-reported outcomes in randomized trials: the CONSORT PRO extension. JAMA 2013, 309(8):814-822.
23. U.S.Department of Health and Human Services, F.a.D.A. Roadmap to Patient-Focused Outcome Measurement in Clinical Trials 3/4/2016]; Available from. http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/ucm370177.htm.
24. Agency, E.M. Draft Reflection Paper on the Use of Patient Reported (PRO) Measures in Oncology Studies 2014, 1-9.
25. Institute, C.P. Patient-Reported Outcome Consortium Available from. http://c-path.org/programs/pro/.
26. Policy, C.f.M.T Transforming Clinical Research Available from. http://www.cmtpnet.org/our-work/transforming-clinical-research/.
27. Forum, N.Q. About Us Available from. http://www.qualityforum.org/story/About_Us.aspx.
28. Kyte D., et al. Inconsistencies in quality of life data collection in clinical trials: a potential source of bias? Interviews with research nurses and trialists. PLoS One 2013, 8(10).
29. Luo X., Cappelleri J.C. A practical guide on incorporating and evaluating patient-reported outcomes in clinical trials. Clin. Res. Regul. Aff. 2008, 25(4):197-211.
30. Fletcher A., et al. Quality of life measures in health care. II: design, analysis, and interpretation. BMJ 1992, 305(6862):1145-1148.
31. Halyard M.Y. The use of real-time patient-reported outcomes and quality-of-life data in oncology clinical practice. Expert Rev. Pharmacoecon. Outcomes Res. 2011, 11(5):561-570.
32. Schipper H. Guidelines and caveats for quality of life measurement in clinical practice and research. Oncology (Williston Park) 1990, 4(5):51-57. (discussion 70).
33. Jackowski D., Guyatt G. A guide to health measurement. Clin. Orthop. Relat. Res. 2003, 413:80-89.
34. Gotay C.C., et al. Quality-of-life assessment in cancer treatment protocols: research issues in protocol development. J. Natl. Cancer Inst. 1992, 84(8):575-579.
35. Cella D.F. Methods and problems in measuring quality of life. Support Care Cancer 1995, 3(1):11-22.
36. Hao Y. Patient-reported outcomes in support of oncology product labeling claims: regulatory context and challenges. Expert Rev. Pharmacoecon. Outcomes Res. 2010, 10(4):407-420.
37. Gnanasakthy A., DeMuro C., Boulton C. Integration of patient-reported outcomes in multiregional confirmatory clinical trials. Control. Clin. Trials 2013, 35(1):62-69.
38. Rock E.P., et al. Challenges to use of health-related quality of life for Food and Drug Administration approval of anticancer products. J. Natl. Cancer Inst. Monogr. 2007, 37:27-30.
39. Snyder C.F., et al. Implementing patient-reported outcomes assessment in clinical practice: a review of the options and considerations. Qual. Life Res. 2012, 21(8):1305-1314.
40. Wiklund I. Assessment of patient-reported outcomes in clinical trials: the example of health-related quality of life. Fundam. Clin. Pharmacol. 2004, 18(3):351-363.
41. Gralla R.J., Thatcher N. Quality-of-life assessment in advanced lung cancer: considerations for evaluation in patients receiving chemotherapy. Lung Cancer 2004, 46(Suppl. 2):S41-S47.
42. Bruner D.W., et al. Issues and challenges with integrating patient-reported outcomes in clinical trials supported by the National Cancer Institute-sponsored clinical trials networks. J. Clin. Oncol. 2007, 25(32):5051-5057.
43. Kyte D., Draper H., Calvert M. Patient-reported outcome alerts: ethical and logistical considerations in clinical trials. JAMA 2013, 310(12):1229-1230.
44. U.S.Code of Federal Regulations 21 CFR 50.20 2012.
45. Hopwood P., et al. Survey of the administration of quality of life (QL) questionnaires in three multicentre randomised trials in cancer. The Medical Research Council Lung Cancer Working Party the CHART Steering Committee. Eur. J. Cancer 1998, 34(1):49-57.
46. Lipscomb J., et al. Patient-reported outcomes assessment in cancer trials: taking stock, moving forward. J. Clin. Oncol. 2007, 25(32):5133-5140.
47. Calvert M.J., Freemantle N. Use of health-related quality of life in prescribing research. Part 2: methodological considerations for the assessment of health-related quality of life in clinical trials. J. Clin. Pharm. Ther. 2004, 29(1):85-94.
48. Land S.R., et al. Compliance with patient-reported outcomes in multicenter clinical trials: methodologic and practical approaches. J. Clin. Oncol. 2007, 25(32):5113-5120.
49. Fayers P.M. Applying item response theory and computer adaptive testing: the challenges for health outcomes assessment. Qual. Life Res. 2007, 16(Suppl. 1):187-194.
50. Young T., Maher J. Collecting quality of life data in EORTC clinical trials-what happens in practice?. Psychooncology 1999, 8(3):260-263.
51. U.S.Department of Health and Human Services, F.a.D.A. Guidance for Industry, E6 Good Clinical Practice: Consolidated Guidance 1996, (Rockville, MD).
52. Lipscomb J., Gotay C.C., Snyder C. Outcomes Assessment in Cancer: Measures, Methods and Applications 2005.
53. Jahagirdar D., et al. Using patient reported outcome measures in health services: a qualitative study on including people with low literacy skills and learning disabilities. BMC Health Serv. Res. 2012, 12:431.
54. Morita S., et al. Assessment and data analysis of health-related quality of life in clinical trials for gastric cancer treatments. Gastric Cancer 2006, 9(4):254-261.
55. Basch E., et al. Recommendations for incorporating patient-reported outcomes into clinical comparative effectiveness research in adult oncology. J. Clin. Oncol. 2012, 30(34):4249-4255.
56. Atkinson T.M., et al. Development and preliminary evaluation of a training workshop for the collection of patient-reported outcome (PRO) interview data by research support staff. J. Cancer Educ. 2013, 28(1):33-37.
57. Fayers P.M., et al. Quality of life assessment in clinical trials-guidelines and a checklist for protocol writers: the U.K. Medical Research Council experience. MRC Cancer Trials Office. Eur. J. Cancer 1997, 33(1):20-28.
58. Kyte D., et al. Management of patient-reported outcome (PRO) alerts in clinical trials: a cross sectional survey. PLoS One 2016, 11(1).
59. Testa M.A., Nackley J.F. Methods for quality-of-life studies. Annu. Rev. Public Health 1994, 15:535-559.
60. Basch E.M., et al. Development of a guidance for including patient-reported outcomes (PROS) in late-phase adult clinical trials of oncology drugs for comparative effectiveness research (CER). Value Health 2012, 15(4):A226-A227.
61. Cappelleri J.C., Bushmakin A.G. Interpretation of patient-reported outcomes. Stat. Methods Med. Res. 2014, 23(5):460-483.
62. Research, I.S.f.Q.o.L. Committees: Best Practices for PROs in Randomized Clinical Trials Available from. http://www.isoqol.org/about-isoqol/committees/best-practices-for-pros-in-randomized-clinical-trials.
63. Chan A.W., et al. SPIRIT 2013 statement: defining standard protocol items for clinical trials. Ann. Intern. Med. 2013, 158(3):200-207.
64. The White House, O.o.t.P.S. FACT SHEET: President Obama's Precision Medicine Initiative 2015.