Why observational studies should be among the tools used in comparative effectiveness research.

Doctors, patients, and other decision makers need access to the best available clinical evidence, which can come from systematic reviews, experimental trials, and observational research. Despite methodological challenges, high-quality observational studies have an important role in comparative effectiveness research because they can address issues that are otherwise difficult or impossible to study. In addition, many clinical and policy decisions do not require the very high levels of certainty provided by large, rigorous randomized trials. This paper provides insights and a framework to guide good decision making that involves the full range of high-quality comparative effectiveness research techniques, including observational research.

[1]  H. Krumholz,et al.  Association of physician certification and outcomes among patients receiving an implantable cardioverter-defibrillator. , 2009, JAMA.

[2]  Ralph D'Agostino,et al.  Stent thrombosis in randomized clinical trials of drug-eluting stents. , 2007, The New England journal of medicine.

[3]  Francis S Collins,et al.  Using science to improve the nation's health system: NIH's commitment to comparative effectiveness research. , 2010, JAMA.

[4]  Neville Kukreja,et al.  Early and late coronary stent thrombosis of sirolimus-eluting and paclitaxel-eluting stents in routine clinical practice: data from a large two-institutional cohort study , 2007, The Lancet.

[5]  Jerry A Krishnan,et al.  Acting on comparative effectiveness research in COPD. , 2010, JAMA.

[6]  David Atkins,et al.  Good research practices for comparative effectiveness research: defining, reporting and interpreting nonrandomized studies of treatment effects using secondary data sources: the ISPOR Good Research Practices for Retrospective Database Analysis Task Force Report--Part I. , 2009, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[7]  Milton C Weinstein,et al.  Comparative effectiveness: asking the right questions, choosing the right method. , 2005, Health affairs.

[8]  A. Farb,et al.  Stent thrombosis redux--the FDA perspective. , 2007, The New England journal of medicine.

[9]  Mark McClellan,et al.  Comparative effectiveness research: Policy context, methods development and research infrastructure , 2010, Statistics in medicine.

[10]  S. Pocock,et al.  Safety and efficacy of sirolimus- and paclitaxel-eluting coronary stents. , 2007, The New England journal of medicine.

[11]  Jeffrey W Moses,et al.  Sirolimus-eluting stents versus standard stents in patients with stenosis in a native coronary artery. , 2003, The New England journal of medicine.

[12]  Elliott S Fisher,et al.  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. , 2007, JAMA.

[13]  Bryan R Luce,et al.  Rethinking Randomized Clinical Trials for Comparative Effectiveness Research: The Need for Transformational Change , 2009, Annals of Internal Medicine.

[14]  Johan Lindbäck,et al.  Long-term outcomes with drug-eluting stents versus bare-metal stents in Sweden. , 2007, The New England journal of medicine.

[15]  P. Lindenauer,et al.  Association of corticosteroid dose and route of administration with risk of treatment failure in acute exacerbation of chronic obstructive pulmonary disease. , 2010, JAMA.

[16]  Jan P Vandenbroucke,et al.  Observational Research, Randomised Trials, and Two Views of Medical Science , 2008, PLoS medicine.

[17]  J. Granada,et al.  Clinical outcomes and stent thrombosis following off-label use of drug-eluting stents. , 2007, JAMA.

[18]  Gregg W Stone,et al.  A polymer-based, paclitaxel-eluting stent in patients with coronary artery disease. , 2004, The New England journal of medicine.

[19]  M. Rawlins.  De testimonio: on the evidence for decisions about the use of therapeutic interventions , 2008, The Lancet.

[20]  D. Stryer,et al.  Practical clinical trials: increasing the value of clinical research for decision making in clinical and health policy. , 2003, JAMA.

[21]  N. Dreyer.  Making observational studies count: shaping the future of comparative effectiveness research. , 2011, Epidemiology.

[22]  K. Popper,et al.  Conjectures and refutations;: The growth of scientific knowledge , 1972 .

[23]  Uwe Siebert,et al.  Good research practices for comparative effectiveness research: approaches to mitigate bias and confounding in the design of nonrandomized studies of treatment effects using secondary data sources: the International Society for Pharmacoeconomics and Outcomes Research Good Research Practices for Retr , 2009, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[24]  K. Rothman,et al.  Extra scrutiny for industry funded trials , 2005, BMJ : British Medical Journal.

[25]  Richard E Gliklich,et al.  GRACE principles: recognizing high-quality observational studies of comparative effectiveness. , 2010, The American journal of managed care.

[26]  M. Schatz,et al.  Impact of asthma controller medications on clinical, economic, and patient-reported outcomes. , 2009, Mayo Clinic proceedings.

[27]  S. Martino,et al.  Conventional-dose chemotherapy compared with high-dose chemotherapy plus autologous hematopoietic stem-cell transplantation for metastatic breast cancer. Philadelphia Bone Marrow Transplant Group. , 2000, The New England journal of medicine.

[28]  Michael L. Johnson,et al.  Good research practices for comparative effectiveness research: analytic methods to improve causal inference from nonrandomized studies of treatment effects using secondary data sources: the ISPOR Good Research Practices for Retrospective Database Analysis Task Force Report--Part III. , 2009, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[29]  William Wijns,et al.  A Cause for Concern , 2007 .