Methodological standards and patient-centeredness in comparative effectiveness research: The PCORI perspective

JAMA

Volumn 307, Issue 15, 2012, Pages 1636-1640

  Basch, Ethan ^a   Aronson, Naomi ^b   Berg, Alfred ^c   Flum, David ^c   Gabriel, Sherine ^d   Goodman, Steven N ^e   Helfand, Mark ^f   Ioannidis, John P A ^e   Lauer, Michael ^g   Meltzer, David ^h   Mittman, Brian ⁱ   Newhouse, Robin ^j   Normand, Sharon Lise ^k   Schneeweiss, Sebastian ^k   Slutsky, Jean ^l   Tinetti, Mary ^m   Yancy, Clyde ⁿ  

^a MEMORIAL SLOAN KETTERING CANCER CENTER   (United States)

^b Blue Cross and Blue Shield Association   (United States)

^c UNIVERSITY OF WASHINGTON   (United States)

^d MAYO CLINIC   (United States)

^e STANFORD UNIVERSITY   (United States)

^f PORTLAND VA MEDICAL CENTER   (United States)

^g NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

^h UNIVERSITY OF CHICAGO   (United States)

ⁱ VETERANS AFFAIRS MEDICAL CENTER   (United States)

^j UNIVERSITY OF MARYLAND   (United States)

^k HARVARD MEDICAL SCHOOL   (United States)

^l AGENCY FOR HEALTHCARE RESEARCH AND QUALITY   (United States)

^m YALE UNIVERSITY   (United States)

ⁿ NORTHWESTERN UNIVERSITY   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

CLINICAL RESEARCH; COMPARATIVE EFFECTIVENESS; DECISION MAKING; HEALTH CARE QUALITY; HUMAN; LAW; MEDICAL RESEARCH; METHODOLOGY; OUTCOME ASSESSMENT; PATIENT CARE; PRACTICE GUIDELINE; PRIORITY JOURNAL; PROPENSITY SCORE; REVIEW; STANDARDIZATION; STUDY DESIGN;

EID: 84859863807     PISSN: 00987484     EISSN: 15383598     Source Type: Journal    
DOI: 10.1001/jama.2012.466     Document Type: Review

References (46)

1. Altman DG. The scandal of poor medical research. BMJ. 1994;308(6924):283-284.
2. Gluud LL. Bias in clinical intervention research. Am J Epidemiol. 2006;163(6):493-501.
3. Weiss NS, Koepsell TD, Psaty BM. Generalizability of the results of randomized trials. Arch Intern Med. 2008;168(2):133-135. (Pubitemid 351185878)
4. Zwarenstein M, Oxman A; Pragmatic Trials in Health Care Systems (PRACTIHC). Why are so few randomized trials useful, and what can we do about it? J Clin Epidemiol. 2006;59(11):1125-1126. (Pubitemid 44485433)
5. Luce BR, Kramer JM, Goodman SN, et al. Rethinking randomized clinical trials for comparative effectiveness research: the need for transformational change. Ann Intern Med. 2009;151(3):206-209.
6. Patient Protection and Affordable Care Act, Pub L No. 111-148, 124 Stat 727. http://www.gao.gov/about/hcac/pcor-sec-6301.pdf. Accessed March 6, 2012.
7. Selby JV, Beal AC, Frank L. The Patient-Centered Outcomes Research Institute (PCORI) national priorities for research and initial research agenda. JAMA. 2012;307(15):1583-1584.
8. Centers for Medicare&Medicaid Services.Outcome measures. https://www.cms.gov/HospitalQualityInits/20-OutcomeMeasures.asp. Accessed February 20, 2012.
9. Riegel B, Moser DK, Anker SD, et al American Heart Association Council on Cardiovascular Nursing; American Heart Association Council on Cardiovascular Nursing; American Heart Association Council on Clinical Cardiology; American Heart Association Council on Nutrition, Physical Activity, and Metabolism; American Heart Association Interdisciplinary Council on Quality of Care and Outcomes Research. State of the science: promoting self-care in persons with heart failure: a scientific statement from the American Heart Association. Circulation. 2009;120(12):1141-1163.
10. Ioannidis JP, Haidich AB, Pappa M, et al. Comparison of evidence of treatment effects in randomized and nonrandomized studies. JAMA. 2001; 286(7):821-830. (Pubitemid 33015576)
11. Ioannidis JP. Effectiveness of antidepressants: an evidence myth constructed from a thousand randomized trials? Philos Ethics Humanit Med. 2008;3:14-22.
12. Wisniewski SR, Rush AJ, Nierenberg AA, et al. Can phase III trial results of antidepressant medications be generalized to clinical practice? a STAR*D report. Am J Psychiatry. 2009;166(5):599-607.
13. Dickersin K. The existence of publication bias and risk factors for its occurrence. JAMA. 1990;263 (10):1385-1389. (Pubitemid 20069945)
14. Turner EH, Matthews AM, Linardatos E, Tell RA, Rosenthal R. Selective publication of antidepressant trials and its influence on apparent efficacy. N Engl J Med. 2008;358(3):252-260.
15. Cipriani A, Furukawa TA, Salanti G, et al. Comparative efficacy and acceptability of 12 newgeneration antidepressants: a multiple-treatments meta-analysis. Lancet. 2009;373(9665): 746-758.
16. Gartlehner G, Gaynes BN, Hansen RA, et al. Comparative benefits and harms of second-generation antidepressants: background paper for the American College of Physicians. Ann Intern Med. 2008;149 (10):734-750.
17. Dwan K, Altman DG, Arnaiz JA, et al. Systematic review of the empirical evidence of study publication bias and outcome reporting bias. PLoS One. 2008; 3(8):e3081.
18. Clarke M, Hopewell S, Chalmers I. Clinical trials should begin and end with systematic reviews of relevant evidence: 12 years and waiting. Lancet. 2010; 376(9734):20-21.
19. Clarke M, Chalmers I. Discussion sections in reports of controlled trials published in general medical journals: islands in search of continents? JAMA. 1998; 280(3):280-282.
20. Claxton KP, Sculpher MJ. Using value of information analysis to prioritise health research: some lessons from recent UK experience. Pharmacoeconomics. 2006;24(11):1055-1068. (Pubitemid 44683363)
21. Sutton AJ, Cooper NJ, Jones DR. Evidence synthesis as the key to more coherent and efficient research. BMC Med Res Methodol. 2009;9:29.
22. Tunis SR, Stryer DB, Clancy CM. Practical clinical trials: increasing the value of clinical research for decision making in clinical and health policy. JAMA. 2003; 290(12):1624-1632. (Pubitemid 37430387)
23. Rubin DB. Estimating causal effects from large data sets using propensity scores. Ann Intern Med. 1997; 127(8 pt 2):757-763. (Pubitemid 27446562)
24. Schneeweiss S, Rassen JA, Glynn RJ, Avorn J, Mogun H, Brookhart MA. High-dimensional propensity score adjustment in studies of treatment effects using health care claims data. Epidemiology. 2009; 20(4):512-522.
25. Stuart EA. Matching methods for causal inference: a review and a look forward. Stat Sci. 2010; 25(1):1-21.
26. McClellan M, McNeil BJ, Newhouse JP. Does more intensive treatment of acute myocardial infarction in the elderly reduce mortality? analysis using instrumental variables. JAMA. 1994;272(11):859-866. (Pubitemid 24282095)
27. Stukel TA, Fisher ES, Wennberg DE, Alter DA, Gottlieb DJ, Vermeulen MJ. Analysis of observational studies in the presence of treatment selection bias: effects of invasive cardiac management on AMI survival using propensity score and instrumental variable methods. JAMA. 2007;297(3):278-285. (Pubitemid 46143305)
28. Kuss O, Legler T, Börgermann J. Treatments effects from randomized trials and propensity score analyses were similar in similar populations in an example from cardiac surgery. J Clin Epidemiol. 2011;64 (10):1076-1084.
29. Toh S, Hernández-Díaz S, Logan R, Robins JM, Hernán MA. Estimating absolute risks in the presence of nonadherence: an application to a follow-up study with baseline randomization. Epidemiology. 2010;21(4):528-539.
30. Schneeweiss S, Gagne JJ, Glynn RJ, Ruhl M, Rassen JA. Assessing the comparative effectiveness of newly marketed medications: methodological challenges and implications for drug development. Clin Pharmacol Ther. 2011;90(6):777-790.
31. Cai T, Tian L, Wong PH, Wei LJ. Analysis of randomized comparative clinical trial data for personalized treatment selections. Biostatistics. 2011;12 (2):270-282.
32. Rothwell PM. Treating individuals 2: subgroup analysis in randomised controlled trials: importance, indications, and interpretation. Lancet. 2005;365 (9454):176-186. (Pubitemid 40091809)
33. Basch E, Abernethy AP, Reeve BB. Assuring the patient centeredness of patient-reported outcomes: content validity in medical product development and comparative effectiveness research. Value Health. 2011;14(8):965-966.
34. Wu AW, Snyder C, Clancy CM, Steinwachs DM. Adding the patient perspective to comparative effectiveness research. Health Aff (Millwood). 2010; 29(10):1863-1871.
35. Basch E, Abernethy AP, Mullins CD, et al. Effectiveness guidance document: recommendations for incorporating patient-reported outcomes (PROs) into clinical comparative effectiveness research (CER) in adult oncology, from the Center for Medical Technology Policy. http://cmtpnet.org/wp-content/uploads/ downloads/2011/12/PRO-EGD-FINAL.pdf. Accessed March 26, 2012.
36. Craig P, Dieppe P, Macintyre S, Michie S, Nazareth I, Petticrew M; Medical Research Council. Developing and evaluating complex interventions: new guidance. http://www.mrc.ac.uk/Utilities/Documentrecord/index.htm?d=MRC004871. Accessed February 24, 2012.
37. Davidoff F. Heterogeneity is not always noise: lessons from improvement. JAMA. 2009;302(23): 2580-2586.
38. Green LW, Glasgow RE. Evaluating the relevance, generalization, and applicability of research: issues in external validation and translation methodology. Eval Health Prof. 2006;29(1):126-153. (Pubitemid 43797500)
39. Shekelle PG, Pronovost PJ, Wachter RM, et al. Advancing the science of patient safety. Ann Intern Med. 2011;154(10):693-696.
40. Kaplan HC, Provost LP, Froehle CM, Margolis PA. The Model for Understanding Success in Quality (MUSIQ): building a theory of context in healthcare quality improvement. BMJ Qual Saf. 2012;21(1): 13-20.
41. Stetler CB, Legro MW, Wallace CM, et al. The role of formative evaluation in implementation research and the QUERI experience. J Gen Intern Med. 2006; 21(suppl 2):S1-S8.
42. Walshe K, Freeman T. Effectiveness of quality improvement: learning from evaluations. Qual Saf Health Care. 2002;11(1):85-87. (Pubitemid 35265309)
43. Berwick DM. The stories beneath. Med Care. 2007; 45(12):1123-1125.
44. Atkins D, Kupersmith J. Implementation research: a critical component of realizing the benefits of comparative effectiveness research.AmJMed. 2010; 123(12)(suppl 1):e38-e45.
45. Glasziou P, Chalmers I, Altman DG, et al. Taking healthcare interventions from trial to practice. BMJ. 2010;341:c3852.
46. Curran GM, Bauer M, Mittman B, Pyne JM, Stetler C. Effectiveness- implementation hybrid designs: combining elements of clinical effectiveness and implementation research to enhance public health impact. Med Care. 2012;50(3):217-226.