Dissemination and Adoption of Comparative Effectiveness Research Findings When Findings Challenge Current Practices. Case-Study Research Approach

01/01/2013

Identifying Case-Study Topics

In selecting case studies, our preliminary intent was to identify and include CER trials whose results challenged existing clinical practices. To identify such trials, we conducted an environmental scan of the peer-reviewed and grey literatures. We examined summaries of key clinical-research studies published in peer-reviewed journals, such as the Updates in General Internal Medicine series published by Annals of Internal Medicine, and we consulted with the project’s expert panel, staff at ASPE, and RAND CER experts. We developed a preliminary list of case study topics (shown in Table 1.2) based on the following considerations:

·        Conditions must have a high overall burden of illness (life expectancy, quality of life, cost)

·        Conditions must be highly prevalent

·        Studies must provide high-quality evidence (e.g., randomized designs).

To attain diversity across the case-study topics, we applied two additional considerations:

·        Diversity of treatment modalities (e.g., drugs, devices, procedures, delivery system interventions)

·        Diversity of treatment settings (e.g., inpatient and outpatient care settings).

While CER results always have the potential to challenge current practices, they do not invariably do so. In some instances, the results simply reinforce predominant practice; in others, the results may be ambiguous. For example, if CER provides estimates of risks and benefits in a population where treatment effects may be heterogeneous, the “right answer” may vary across subgroups or by variants on treatment. The results of some CER studies may suggest modifications to some aspects of practice while reinforcing the status quo for other aspects. For our case studies, we sought to include CER studies that challenged at least some aspect of practice in at least one population.

Table 1.2

Preliminary Case-Study Topics

Topic

CER Finding

Reference

Medications

 

 

High-dose versus usual-dose statin for secondary prevention after myocardial infarction (MI)

Intensive lowering of LDL-C did not significantly reduce the primary outcome
of major coronary events.

Pedersen et al., 2005

Beta-blockers versus other drugs for primary hypertension

Beta blockers are less than optimal compared with other antihypertensive drugs.

Lindholm et al., 2005

Diuretics versus calcium channel blockers versus angiotensin-converting enzyme (ACE) inhibitors versus alpha-adrenergic blockers for hypertension (ALLHAT)

Thiazide-type diuretics are superior in preventing one or more major forms of cardiovascular disease (CVD) and are less expensive.

Officers, 2002

Tiotropium versus ipratropium for chronic obstructive pulmonary disease (COPD)

Tiotropium improves health outcomes and is associated with higher costs than ipratropium.

Oostenbrink et al., 2004

Warfarin versus low-molecular-weight (LMW) heparin for outpatient treatment of acute venous thromboembolism

High-dose heparin is more effective than low-dose heparin.

Kovacs et al., 2003

Salmeterol versus fluticasone for COPD

Combination treatment improved symptoms and lung function better than either component alone.

Calverley et al., 2003

Rituximab versus usual care for follicular lymphoma

Rituximab improved outcomes for patients.

Hiddemann et al., 2005

Atypical antipsychotic drugs versus conventional antipsychotic drugs for schizophrenia

Older antipsychotic medications were similar in effectiveness and had lower costs than newer antipsychotic medications.

Lieberman, Stroup, et al., 2005

Procedures

 

 

Carotid artery stenting versus carotid endarterectomy

Rates of death and stroke were lower with endarterectomy than with stenting.

Mas et al., 2006

Surgical versus nonsurgical treatment for lumbar spinal stenosis

Surgery had better outcomes than nonsurgical treatment in the observational data analysis.

 

Weinstein, Tosteson, et al., 2008

 

Optimal medical therapy versus cardiac resynchronization therapy versus combined cardiac resynchronization therapy and defibrillator therapy for patients with moderate to severe heart failure (HF)

Cardiac resynchronization reduced hospitalization and improved functional status and survival in patients with moderate to severe HF.

Bristow, Saxon, et al., 2004

Coronary-artery revascularization versus none prior to elective major vascular surgery

Coronary-artery revascularization before elective vascular surgery did not improve long-term outcomes.

McFalls et al., 2004

Preventive screening and diagnostic testing

 

 

Screening for abdominal aortic aneurysm

Screening for abdominal aortic aneurysms reduced mortality.

Lindholt et al., 2005

Virtual colonoscopy versus optical colonoscopy for colorectal cancer screening

Virtual colonoscopy compared favorably with optical colonoscopy.

Pickhardt et al., 2003

Computed tomography for coronary angiography (CTCA) versus usual care for acute coronary syndrome

Use of CTCA for acute coronary syndrome should be avoided because of significant radiation exposure.

Einstein et al., 2007

Medication versus procedure

 

 

Percutaneous coronary intervention (PCI) versus optimal medical therapy for chronic stable angina

PCI and optimal medical therapy provided equivalent survival benefit and relief of angina symptoms.

Boden et al., 2007

Primary PCI versus thrombolytic therapy for acute coronary syndrome

PCI was superior to thrombolytic therapy for acute coronary syndrome.

Aversano et al., 2002

Delivery-system interventions

 

 

Computerized physician order entry (CPOE) to prevent serious medication errors

Introduction of CPOE system and a team intervention into the hospital reduced the incidence of serious medication errors.

Bates et al., 1998

Wrong-site, wrong-procedure, wrong-person surgery prevention techniques versus usual care

 

Implementation of the Joint Commission’s “Universal Protocol” reduced the incidence of wrong-site, wrong-procedure, and wrong-person surgeries relative to usual care.

NQF Report on Safe Practices, 2009

Use of critical-care-certified physicians versus usual care

 

Patients treated by physicians who have specific training and certification in critical-care medicine had better outcomes than those treated by non-critical-care-certified physicians.

NQF Report on Safe Practices, 2009

 

Working from our preliminary list of potential CER studies and using the considerations described above as a guide, we narrowed the list to seven case studies that addressed these considerations. Five topics were selected for investigation (Table 1.3), and the other two were kept as backups.

The Clinical Antipsychotic Trials of Intervention Effectiveness (CATIE) trial was selected as one of the most prominent CER studies to involve comparison of two classes of pharmaceutical agents and because of findings that were somewhat controversial. The Spine Patient Outcomes Research Trial (SPORT) and Clinical Outcomes Utilizing Revascularization and Aggressive Drug Evaluation (COURAGE) studies were selected to illustrate the comparison of surgical versus nonsurgical (or procedural versus nonprocedural) management of a chronic clinical condition. The Comparison of Medical Therapy, Pacing, and Defibrillation in Heart Failure (COMPANION) study was chosen to represent the study of a device with demonstrated efficacy in a subgroup of the potentially affected population. The CPOE study was selected to represent the class of CER involving delivery-system interventions.

 

Table 1.3

Final Case-Study Topics

Topic

CER Finding

Reference

Type of Comparison

Clinical Antipsychotic Trials of Intervention Effectiveness (CATIE):
atypical antipsychotic drugs versus conventional antipsychotic drugs for schizophrenia

Older antipsychotic medications were similar in effectiveness and had lower costs than newer antipsychotic medications.

Lieberman, Stroup, et al., 2005

Medications

Clinical Outcomes Utilizing Revascularization and Aggressive Drug
Evaluation (COURAGE): percutaneous coronary intervention versus optimal medical therapy for chronic stable angina

PCI and optimal medical therapy provided equivalent survival benefit and relief of angina symptoms.

 

Boden et al., 2007

Medication versus procedure

Spine Patient Outcomes Research Trial (SPORT): surgical versus
nonsurgical treatment for lumbar spinal stenosis

Surgery had better outcomes than nonsurgical treatment in the observational data analysis.

 

Weinstein, Tosteson, et al., 2008

 

Procedures

Comparison of Medical Therapy, Pacing, and Defibrillation in Heart Failure (COMPANION): optimal medical therapy versus cardiac resynchronization therapy versus combined cardiac resynchronization therapy and
defibrillator therapy for patients with moderate to severe HF

Cardiac resynchronization reduced hospitalization and improved functional status and survival in patients with moderate to severe HF.

Bristow, Saxon, et al., 2004

Procedures

Computerized physician order entry (CPOE): interventions to prevent
serious medication errors

Introduction of CPOE system and a team intervention into the hospital reduced the incidence of serious medication errors.

Bates et al., 1998

Delivery-system intervention

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