MR RESCUE
(Merci Retriever and Recanalization of Stroke Clots using Embolectomy) |
An NIH(NINDS)-funded trial of a device (MERCI™ Retriever) to remove intracranial cerebrovascular clots Purpose: To compare the effectiveness of treating acute ischemic stroke with the Merci Retriever within 8 hours of symptom onset to standard medical treatment, and to identify people who might benefit from the MR device Enrollment goal: 120 patients in ten centers. Enrollment began in 2004; as of June 2008, the study is still recruiting.
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Device is being used off-trial at a high rate (approximately 5,000 procedures in July 2007 and 6,000 in August 2007). Availability of and coverage for the device off-trial has made it very difficult to recruit patients to the trial.
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| CORAL (Cardiovascular Outcomes in Renal Atherosclerotic Lesions) |
An NIH(NHLBI)-funded study of a device to treat renal artery stenosis Purpose: To compare medical therapy plus stenting of hemodynamically significant renal artery stenoses to medical therapy alone in patients with systolic hypertension and renal artery stenosis Enrollment goal: 1,080 patients. Enrollment began in 2005 at multiple sites; as of June 2008, the study is still recruiting.
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While 35,000 stenting procedures are performed annually in the United States, the trial had managed to enroll only 290 patients as of September 2007. Investigators attribute slow accrual to CMS coverage for stents off-trial.
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| CREST (Carotid Revascularization Endarterectomy versus Stenting Trial) |
An NIH(NINDS)-sponsored trial of a relatively new procedure to prevent stroke Purpose: To compare stent-assisted carotid angioplasty (CAS) to the traditional and accepted surgical approach of carotid endarterectomy (CEA) for treatment of carotid artery stenosis to prevent recurrent strokes in patients who had a TIA (transient ischemic attack) or a mild stroke within the past 6 months (symptomatic) and in patients who have not had any symptoms within the past 6 months (asymptomatic). Enrollment goal: 2,500 patients at multiple sites. Enrollment began in 2001, and is predicted to be completed in 2008.
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This trial encountered initial difficulty enrolling because CMS reimbursed for one arm (CEA) but not the other (CAS). Physicians are implanting stents in many low-risk patients off-label before RCTs have generated evidence to support this practice. Surgeons appear reluctant to refer patients to the trial, due to potential loss of income (i.e., patient has 50% chance of being randomized to CAS and therefore not undergo surgery). Many non-CMS payers will not cover cost of stenting for low-risk patients on clinical trial.
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| Multi MERCI™ Trial (Mechanical Embolus Removal in Cerebral Ischemia) |
An industry-sponsored (Concentric Medical) trial of a new generation device to restore blood flow in the neurovasculature of ischemic stroke patients by removing blood clots Purpose: To test the safety and efficacy of newer generation (L5) MERCI™ Retriever Device Enrolled 164 patients from 15 sites. The study has been completed.
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This controlled registry study rather than an RCT. Because the device was both available and reimbursable, radiologists and vascular surgeons rapidly adopted it despite lack of evidence from RCTs. Patients are eager to receive this device, and may have no incentive to enroll in an RCT where they might be randomized to a non-device arm.
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CATT
(Comparison of Age-Related Macular Degeneration Treatments Trials: Lucentis-Avastin Trial) |
An NIH(NEI)-funded trial of two drug therapies for age-related macular degeneration. Purpose: to evaluate the relative efficacy and safety of treatment of neovascular AMD with Lucentis on a fixed schedule, Avastin on a fixed schedule, Lucentis on a variable schedule, and Avastin on a variable schedule. Enrollment goal: 1,200 patients. Enrollment began in February 2008, and is still open as of June 2008.
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Cost of Avastin, but not cost of Lucentis, was covered by the sponsor. Lucentis was approved for AMD, but was nonetheless deemed investigational in this context. CMS could not legally pay 80% of those costs without changes to the Medicare CTP. This trial thus became an important stimulus for the Proposed CRP. Billing practices posed a threat to blinding, but the cost implications of central purchase of drugs was prohibitive.
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SCOT Trial
(Scleroderma: Cyclophosphamide or Transplantation) |
An NIH(NIAID)-funded trial of two treatments for systemic sclerosis Purpose: To compare high-dose immunosuppressive therapy followed by hematopoietic stem cell transplant to high-dose pulse IV cyclophosphamide for treating systemic sclerosis Enrollment goal: 226 patients from multiple sites. Enrollment began in June 2005. As of June 2008, 113 patients had been enrolled and screened on SCOT but only 40 randomized.
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This Phase randomized III trial was preceded by a pilot study which enrolled 36 patients at 4 sites. By study design, all costs of clinical care were assigned to the insurance carriers; study budget includes no funds for clinical care costs. Research costs (e.g. blood samples)are covered by the grant. The study had difficulty enrolling patients due to insurance coverage denials. Primary reason for denial was the reimbursement was requested for “investigational” or “experimental” procedures or items. The PI has engaged in “hand-to-hand combat” with individual carriers to advocate for coverage of patients wishing to enroll in the trial.
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NETT
(National Emphysema Treatment Trial) |
A trial funded by NIH (NHLBI), CMS, and AHRQ of interventions to treat emphysema Purpose: To evaluate the long term efficacy, morbidity and mortality associated with medical therapy with lung volume reduction surgery (LVRS) as compared to medical therapy alone and to define patient selection criteria. Enrollment goal: 2,500 patients. The study is ongoing but enrollment closed; 1,218 patients were randomized.
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Initially (per DHHS Advisory Opinion No. 98-6), the target enrollment for the study sample was 4,700. This goal was dropped to 2,500 due, in part, to difficulties encountered by sites in recruiting patients to the study (per Advisory Opinion No. 00-5). A waiver of Medicare copayment and deduction for participants was requested on the grounds that it would promote patient compliance with data collection and promote enrollment of additional patients. This waiver was denied in DHHS Office of the Inspector General (OIG) Advisory Opinion No. 00-5.
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BARI 2D
(Bypass Angioplasty Revascularization Investigation in Type 2 Diabetes |
A 2x2 factorial NIH(NHLBI)-funded trial of (1) elective revascularization, and (2) glycemic control strategies for patients with Type 2 diabetes and stable coronary artery disease. Purpose: To compare elective revascularization with aggressive medical therapy to aggressive medical therapy alone, and simultaneously an insulin providing to an insulin sensitizing strategy of glycemic control. Enrollment goal: 2,400 patients. As of June 2008, the study is ongoing but no longer recruiting patients.
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The supplier requested a waiver of Medicare cost-sharing expenses of blood glucose self-monitoring supplies Desire to encourage adequate enrollment in the study was cited as a reason for requesting this waiver. OIG Advisory Opinion No. 04-01 ruled in favor of the request.
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| Chicago Healthy Aging Study |
An NIH(NHLBI)-funded observational study examining how risk factors for heart disease in young and middle aged people affect people's health as they age. Purpose: To gather follow-up data on individuals who participated in the CHAS between 1967–1973. Enrollment goal: 1,500 patients, 600 of whom formerly had low-risk of heart disease and 900 who were at high risk.
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Participation entails many tests and services, including: a physical exam, blood pressure measurements, blood and urine collection, electrocardiogram, physical function tests, and computed tomography chest scan. Many of these items are covered by Medicare. Patients have had little incentive to enroll, because they can get the services reimbursed off-trial. Even offering services for free has not been an incentive. Recruitment has been challenging. Patients have expressed concern that participation may impact their Medicare coverage.
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| Women’s Health Initiative (WHI) |
An NIH(NHLBI)-funded trial studying cardiovascular disease, cancer, and osteoporosis in postmenopausal women Purpose: To evaluate, through a randomized controlled clinical trial, three primary prevention strategies: hormone replacement therapy (HRT), dietary modification, and calcium/vitamin D supplementation Enrollment: 68,132 patients enrolled in the RCT component. The study was terminated due to results indicating that risks of HRT outweighed benefits.
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The study budget did not include funds for mammograms, which were expected to be covered by Medicare and third-party payers. Participants were displeased that they were expected to file insurance claims. This became a retention issue, and eventually the study began paying for mammograms in order to retain participants.
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