NHLBI Working Group
Clinical Heart Failure Research Network
Executive Summary

The National Heart, Lung, and Blood Institute convened a Working Group (WG) of experts in heart failure on November 18, 2003 by teleconference. Its charge was to consider the need for and utility of an organized network of clinical centers to foster research on heart failure and to advise the Institute on the types of studies and scope of research that might be conducted through such a network.

There was unanimous, enthusiastic support from the WG for NHLBI to proceed with development of a heart failure research network modeled after the existing NHLBI Networks. Aspects of the NHLBI network model that were particularly attractive to the WG included: a standing organizational structure with the flexibility to evaluate and institute new protocols; network governance by a steering committee; an independent protocol review committee; the potential for ancillary studies of network trials; and the potential for involvement of industry.

The Working Group provided the following comments and recommendations to NHLBI outlining five major areas considered pivotal for establishing heart failure network:

1. Utility and advantages of a clinical research network dedicated to heart failure research include:

• The network offers the opportunity to do important proof-of-concept studies that industry is not likely to pursue.
• The pace of protocol development would be accelerated by the established infrastructure of a standing network.
• Trial investigators would be involved in the selection and design of protocols in the earliest stages, thereby strengthening their engagement and commitment.
• Establishing a standing support mechanism would reduce operational costs, especially those related to turnover of study coordinators (i.e., costs of retraining, data loss) and allow selection and retention of the most qualified staff.
• The network, through its steering committee, would entertain ideas for protocols from many sources.
• Patient and physician education, and evaluation of quality of life and cost-effectiveness questions, could be built into every study.
• The network offers opportunities for both training a new generation of clinical investigators and strengthening the base of existing clinical researchers.

2. Considerations regarding the size of network and type, duration, and scope of intervention, which could be studied in a network setting include:
• The final size of the network would be determined by the availability of funds. The WG anticipated the network would include approximately 5-10 clinical centers, a Data Coordinating Center, Data Safety and Monitoring Board, and relevant core facilities.
• Proof of concept trials would be favored over Phase III trials that require long timelines and large numbers of patients.
• Protocols selected by the network should have a public health impact; should test treatment strategies rather than single agent interventions (e.g., interventions employing several classes of drugs rather than a single drug in that class); and, when appropriate, should have multiple arms rather than being limited to the traditional two-arm, randomized clinical trial design.
• Duration of clinical studies would vary from short- to long-term, but all protocols must be completed within the five year funding cycle.
• The spectrum of studies would be broad and could include cell-based therapies, devices, pharmaceuticals, surgical procedures, angiogenesis, and others.
• The broad spectrum of potential interventions would require multidisciplinary teams.
• Consideration should be given to attempting to streamline and centralize main protocol clearances, such as from the IRB, PRC, and DSMB, to reduce logistical burden and cost.

3. Strategies for maximizing clinician participation should include:
• Selection of research questions that are important to public health which anticipate a changing clinical environment.
• Targeting patient populations that reflect "real" clinical practice.
• Providing stability by means of a steady flow of trials in which sites can participate.
• Designing trials that are straightforward and comprehensible.
• Providing opportunities prior to the end of the trial for recognition of investigators and coordinators through opportunities for authorship and for presentation of results at national scientific meetings.
• Ensuring that trial budgets allow for adequate support of coordinator staff and infrastructure in the clinical sites, including start-up funds in addition to capitated reimbursement.

4. Bridging the gap between evidence-based knowledge and clinical practice requires:
• That information derived from trials not only be credible, but applicable to, and feasible in real world clinical practice.
• That the study test strategies that simulate the resources available to the community.
• That the network educate community-based physicians and patients on the role of research in advancing the treatment of heart failure.
• That quality of life issues be addressed.

5. Fostering research in practice patterns and out-of-hospital patient management requires:
• Targeted involvement of primary care physicians for participation in the network and its protocols.
• Building on the foundation of trust that exists between the patient and his/her primary care physician.

6. Other considerations include:
• The eligible centers should have expertise in at least some minimum number of the areas of expected study, including devices, cell-based therapies, surgery, angiogenesis, medical management of heart failure, and other relevant therapies.
• The principal investigators of network centers need to be open to new ideas from their community partners.
• A long-term goal should be to create a three-way partnership among the clinical community (both academic and private physicians), NHLBI, and industry, for conducting patient-oriented heart failure research.

Working Group Members

Chair:

• Eugene Braunwald, M.D., Chairman, TIMI Study Group Brigham and Women's Hospital, Faculty Dean for Academic Programs in the Partners HealthCare System, Boston, MA

Members:

• Lynne Warner Stevenson, M.D., Brigham and Women's Hospital, Director, Cardiomyopathy and Heart Failure Program, Boston MA
• Christopher O'Connor, M.D., Duke University, Director of the Heart Failure Program, Durham, NC
• Sidney Goldstein, M.D., Henry Ford Health System, Division Head, Emeritus, Cardiovascular Medicine Heart & Vascular Institute, Detroit, MI
• Patrick McCarthy, M.D., Cleveland Clinic Foundation, Cardiothoracic Surgery, Cleveland, OH 44195
• Susan J. Bennett, D.N.S., R.N.,Indiana University Center for Aging, Professor, Dept. of Adult Health School of Nursing, Indianapolis, IN

Absent:

• Douglas Losordo, M.D., Chief of Cardiology, Boston University, Boston, MA. Input provided after the conference call.

Last updated: March 18, 2004