Release Date: December 24, 2003
Department of Health and Human Services (DHHS)
National Institutes of Health (NIH) (http://www.nih.gov)
COMPONENTS OF PARTICIPATING ORGANIZATIONS
National Heart, Lung, and Blood Institute (NHLBI) (http://www.nhlbi.nih.gov/)
Response Due Date: January 15, 2004
The NHLBI plans to release a Request for Proposals for a Clinical and Data Coordinating Center to establish a patient and device data registry to track patients receiving mechanical circulatory support for end-stage heart failure. The registry will also establish a tissue and blood repository. The registry will benefit the research and practice communities as well as federal agencies concerned with patient safety and oversight.
The NHLBI would like information from academic institutions, professional groups and private industries that develop and apply mechanical circulatory support devices to identify optimum parameters to establishing a registry and ways to design and implement an effective program. Specifically, NHLBI seeks advice concerning core information to be collected, concerns and solutions to protect rights of patients, facilities, providers and support teams and manufacturers. Also important are independence of data analysis, access to data and data sharing, and potential strategies for the registry to become at least partially self-supporting. Ways to involve private industry without undermining the independence of the program governance are also sought.
Over the past two decades, mechanical circulatory support devices, such as ventricular assist devices and total artificial hearts, have been developed to augment or supplant failing myocardial performance. This therapy has been used successfully as a bridge to heart transplantation, a bridge to recovery, and, most recently, as destination therapy for intractable heart failure. Although cardiac transplantation offers life-saving therapy for selected patients, its use is limited by a supply of donor organs that currently meets less than one-tenth the need, and which is steadily decreasing. As a consequence, the number of mechanical circulatory support device implantations has increased from a cumulative total U.S. experience of 1,000 in 1990, to approximately 1,000 per year in 2000. The latter figure is expected to increase greatly following the 2002 Food and Drug Administration (FDA) approval of a destination therapy device and the October 1, 2003, CMS approval for reimbursement for this indication. Despite favorable survival and quality of life outcomes, mechanical devices have severe and sometimes life-threatening complications associated with infection, thrombosis, and device failure. Development of new procedures and devices to reduce these complications would be expedited by a systematic, independent analysis of mechanical support device implantation procedures and outcomes. Cooperation among the federal agencies involved in the development, regulation, and use of circulatory support devices, including NHLBI, FDA and Center for Medicare and Medicaid Services (CMS), will permit development of standard reporting of patient characteristics, indications, implantation procedures, and adverse events. Such cooperation will facilitate clinical evaluation and patient management and aid better device development.
Heart failure is a malignant cardiac disorder affecting over 4.5 million people in the U.S.; it is the most common hospital discharge diagnosis, exacting substantial morbidity and mortality, and health care resources and costs. Current therapies improve symptoms, but have only a modest impact on long-term survival. The NHLBI-supported Randomized Evaluation of Mechanical Assistance for the Treatment of Congestive Heart Failure (REMATCH) study evaluated the implantation of left ventricular assist devices and optimal medical therapy, versus optimal medical therapy alone, in randomly assigned end-stage heart failure patients ineligible for cardiac transplantation. REMATCH results demonstrated better survival and quality of life in patients who received the assist devices. Because the cost of mechanical assistance in REMATCH was similar to transplantation, mechanical assistance offers an important therapeutic alternative to transplantation. Nevertheless, device therapy can be improved. Better patient selection, device design and patient management are possible, but require careful evaluation that would be facilitated by data centralization and independent analysis. A tissue and blood repository would encourage studies of heart failure using cutting-edge approaches, such as genomics and proteomics, the response to mechanical assistance, and the mechanisms underlying the reported improvement in native heart function with these devices. Information obtained by the registry will aid in the development of improved surgical implantation procedures and patient management paradigms, and also point the way for future research studies.
The registry will support a Clinical and Data Coordinating Center, a tissue and blood repository, and a scientific advisory group to develop protocols for standard procedures to collect patient and device data, tissue, and blood samples for genomic and proteomic investigations. Registry data would be analyzed to permit understanding of clinical outcomes, complications, and associated therapies. The registry would serve as a resource to encourage research investigations using centrally collected tissue or data, and would provide information critical to the design of clinical investigations or trials of specific therapeutic interventions. The proposed registry would build on the progress of the existing registries, and facilitate transfer of federally required data sets without compromising confidentiality of proprietary information.
NHLBI seeks comments and suggestions as requested below.
1. Patient outcomes are determined by patient characteristics, surgical provider and facilities support, and device performance. Please suggest data elements you consider essential for collection by the registry.
2. Abstracting hospital and out-patient data and checking accuracy of submitted data will be required. Please provide concerns you have about the registry collecting data on patients, devices, hospitals and medical providers. Please suggest ways the NHLBI can design registry protocols to minimize those concerns.
3. Please identify ways for NHLBI to encourage industry to participate and support the registry.
4. Please suggest ways to design the registry so that participants do not have duplicate reporting requirements.
5. Please provide methods to ensure accuracy and security of data in the registry.
6. Please suggest strategies for the registry to become at least partially
7. We would appreciate any additional views or opinions that you think would be useful to develop a registry that will improve outcomes for patients receiving mechanical circulatory support.
RESPONSE AND PROCESS:
Responses in any of the areas are welcome; respondents should not feel compelled to address all items. Please respond no later than December 30, 2003. We look forward to your thoughts, opinions and suggestions and hope that you will share this document with your colleagues. All responses will be kept confidential. Thank you for your assistance.
To respond please link to the online form in the “What’s New” section on the NHLBI home page (http://www.nhlbi.nih.gov/new/whatsnew.htm.) or send a letter, fax or email to the following address:
Clinical and Molecular Medicine Program
Division of Heart and Vascular Diseases
National Heart, Lung, and Blood Institute
National Institutes of Health
6701 Rockledge Drive, Rm. 9153, MSC-7940
Bethesda, MD 20892-7940 (Courier 20817)
Tel. 301-435-0515 / Fax. 301-480-1336