Fourth Annual Public Interest Organization Meeting

Concurrent Community Forums
February 5, 2003 - Bethesda, Maryland

Three concurrent forums were held during the afternoon to foster interaction and exchange in NHLBI areas of interest:

• cardiovascular health and diseases
• lung and sleep health and diseases
• blood diseases and resources

In these community forums, PIO representatives, NHLBI research administrators, and leaders of key professional societies and voluntary health agencies had an opportunity to suggest and discuss specific research needs and issues, from a patient's perspective, and potential areas of collaboration. Each forum consisted of brief presentations by resource panels to provide a framework for in-depth discussions that followed.

Organizations interested in cardiovascular health and disease participated in a discussion led by:

• Dr. Robert Bonow, President, American Heart Association
• Dr. Cary Sennett, Vice-President of Science and Quality Improvement, American College of Cardiology
• Dr. Sonia Skarlatos, Director, Vascular Biology Research Program, Division of Heart and Vascular Diseases, NHLBI
• Dr. Denise Simons-Morton, Acting Director, Clinical Applications and Prevention Program, Division of Epidemiology and Clinical Applications, NHLBI

Dr. Bonow described the organization, mission, and activities of the AHA. A broad grassroots organization, the AHA comprises a central office, affiliates, local divisions, and local and national lay and science volunteers. It is a national voluntary health agency with a mission to reduce disability and death from cardiovascular diseases and stroke. Heart disease is the leading cause of death among Americans, and stroke is the third most common cause. Both are largely preventable, and major disparities exist in the population groups affected. The AHA's goal is to reduce the risk of CHD by 25 percent by 2010. As in all its endeavors, the AHA works with many partners, including the NHLBI, to realize this goal.

Dr. Bonow highlighted the following key factors for a voluntary health organization and provided examples of each factor based on the experience of the AHA.

• Define your mission and goals.
• Determine your scope of activities (i.e., what you will and will not do)—for example, patient support, public education, research funding, linking patients with research.
• Include advocacy for research support
.
• Establish strategic partnerships (e.g., with government agencies, community institutions, health care providers, schools, workplaces, and other organizations).
• Leverage your activities in conjunction with the goals of government agencies (e.g., the Healthy People 2010 partnership).
• Define your role and function with other partners that support research in your disease area.

Dr. Sennett briefly described the organization, mission, and activities of the ACC. With a headquarters staff of 200 located in Bethesda, Maryland, the ACC represents more than 28,000 cardiologists around the world and supports 40 chapters. About 95 percent of U.S. cardiologists belong to the ACC. Its mission is "to foster optimal cardiovascular care and disease prevention through professional education, promotion of research, leadership in the development of standards and guidelines and the formulation of health care policy." ACC activities include establishing evidence-based care guidelines through the consensus of experts and literature reviews, equipping physicians to deliver evidence-based care through education programs, enabling systems to monitor the gap between science and practice (e.g., using performance measures), and remodeling the health care environment through advocacy to improve the quality of care.

Dr. Sonia Skarlatos described the structure of DHVD and discussed some of the funding mechanisms used by the Institute to support research-related activities. The division consists of three programs: the Heart Research Program, the Clinical and Molecular Medicine Program, and the Vascular Biology Research Program. In addition to the research grants and contracts that the NHLBI funds, it also supports a wide variety of training and career development awards at all stages of a research career as well as programs with other ICs, workshops, and scientific working groups.

Dr. Denise Simons-Morton described the function of DECA and the different types of studies and trials that the division funds. DECA focuses on the clinical and public health aspects of cardiovascular, lung, and blood diseases through two programs: the Clinical Applications and Prevention Program and the Epidemiology and Biometry Program. Studies include epidemiological investigations of the incidence and prevalence of disease, population-based research, clinical trials, demonstration and education research, disease prevention and health promotion research, and basic and applied research in behavioral medicine. Emphasizing the need for research, Dr. Simons-Morton gave examples of the many studies and initiatives funded through DECA. The division works closely with the NHLBI Office of Prevention, Education, and Control to ensure that research findings are translated into clinical practice and that educational materials are available to the public.

The participants expressed several concerns, commented on research needs, and offered suggestions for collaboration.

Early Diagnosis and Specialized Care. For many diseases, early diagnosis is critical to patient survival. Participants expressed concern about:

• The gap between science and clinical practice, especially as it relates to diagnosis.
• The failure of many patients to get help before their condition is such that they may no longer be eligible for the most effective treatments.
• The reluctance of some physicians or health care systems to refer complex cases (e.g., congenital heart disease, Barth's syndrome) to specialists.

Accurate Estimates of Disease Prevalence. Lack of epidemiological data for many diseases can be a problem for clinical research and can impede potential collaborations with the pharmaceutical industry.

The participants considered the following ways their organizations could improve physician awareness of a disease, increase research on a disease, and influence clinical practice.

• Collaborate with other PIOs to
• Support conferences focused on the disease.
• Support investigators who are developing new research programs.
• Collaborate with government agencies, professional societies, and voluntary health agencies that disseminate research findings and treatment guidelines to
• Recommend changes to medical school curricula.
• Encourage research sponsors to monitor physician compliance with guidelines for clinical research programs.
• Partner with the NHLBI, other ICs, and other government agencies such as the FDA and Centers for Disease Control and Prevention to improve physician awareness of a disease
.
• Provide materials (e.g., brochures, fact sheets) directly to physicians, hospitals, and patients.

Dr. Sennett suggested two specific ways for PIOs to interact with the ACC.

• Collaborative communications to foster high-quality practice and patient education.
• Involvement in clinical trials to address cardiovascular care and disease prevention.

NHLBI staff encouraged PIOs to submit specific suggestions of research needs in writing to the NHLBI.

Organizations interested in lung and sleep health and disease participated in a discussion led by:

• Dr. Carl Hunt, Director, National Center on Sleep Disorders Research, NHLBI
• Dr. Thomas Martin, President, American Thoracic Society
• Dr. Gail Weinmann, Director, Airway Biology and Disease Program, Division of Lung Diseases, NHLBI

Dr. Hunt described the activities of the NCSDR, which is an integral part of the NHLBI. Both the center and the Sleep Disorders Research Advisory Board were established by law in 1993. The center conducts and supports research, scientist training, dissemination of health information, and other activities on sleep disorders and related concerns. It also coordinates sleep research activities with other Federal agencies and with public and nonprofit organizations. The center fulfills four key functions: research, training, technology transfer, and coordination.

The NIH also has a Trans-NIH Sleep Research Coordinating Committee, which was established in 1986 by the NIH Director to facilitate interchange of information on sleep and sleep-related research. Ten ICs are members of this committee.

In January 2003, the NCSDR issued its revised National Sleep Disorders Research Plan. The plan highlights three categories of problems addressed at the center: sleep restriction, primary sleep disorders, and secondary sleep disorders. The center has developed and disseminated many publications, including information pamphlets and fact sheets, on sleep disorders. The NCSDR 5-year Star Sleeper Educational Campaign was highlighted at the 2002 PIO meeting.

Dr. Martin briefly described the organization and activities of the ATS, which focuses on pulmonary and critical care medicine and sleep. The ATS consists of 12 assemblies that reflect the interests of society's approximately 13,500 members, 25 percent of whom are from outside the United States. The society's long-range goal is "to decrease morbidity and mortality from respiratory disorders and life-threatening acute illnesses." Its interests are similar to those of the PIOs: advocacy, treatment, science, and training.

Dr. Weinmann said that DLD supports research, training, and career development in lung diseases. Its goal is to promote understanding of the cause, pathogenesis, diagnosis, treatment, and prevention of all diseases of the pulmonary system, except for lung cancer and certain infectious diseases that are addressed by other NIH components. The division consists of two programs: the Airway Biology and Disease Program and the Lung Biology and Disease Program.

Dr. Weinmann described the following stages for developing a research program at the NHLBI:

1. Initial identification of a research need by scientists and the public (including PIOs).
2. Discussion of the research need by scientists and advocates at a conference or meeting to advise on the direction of research, training, and/or education.
3. Design and development of a research program, based on this advice, at the NHLBI.

Drs. Martin and Weinmann encouraged the PIOs to assume a role in advocating for research. The potential of research programs cannot be fulfilled without the involvement of the public and patients. Both the NHLBI and the ATS are eager to interact with PIOs.

The participants identified and discussed several actions to improve clinical research and patient care and highlighted collaborative steps that the PIOs and the NHLBI could take.

• Provide avenues for health-care providers to
• Help patients who are receiving the same treatment meet each other and connect patients to professional societies and PIOs.
• Participate, with health maintenance organizations (HMOs), in building patient communities and forums—to promote communication and education.
• Enroll in continuing education programs that include patients who are invited to convey their personal experiences—to help keep physicians in tune with patient communities.
• Establish rural support groups. Because of the scarcity of patients with any particular disease, support groups that are not disease-specific are needed in scarcely populated areas.
• Encourage physician-scientists to study pulmonary diseases.
• Combine funds with one another and with those of the NIH or other funding agencies to sponsor research or career development grants.
• Help new researchers who are studying a disease by contacting the NIH to identify a possible mentor that could guide the researcher through NIH funding mechanisms and the application process.
• Develop patient registries.
• Foster sympathetic interest in Congress.
• Develop and provide tools to help patients obtain information about available and effective drugs and treatments for their condition.
• Collect and disseminate information about hospitals and doctors to enable patients to research and select their health care providers (e.g., a clearinghouse or Web site that lists physicians who treat specific or rare diseases would be a useful resource for patients).

The participants considered how the NIH and the ATS could help organizations carry out their missions. Suggestions included:

• Develop and disseminate educational materials for patients and physicians.
• Involve organization representatives when developing information.
• Ask the PIOs to help distribute messages.
• Facilitate PIO interactions across the ICs.
• Establish liaison personnel for trans-NIH research projects
• Encourage each IC to designate a staff person that patients and PIOs could contact to address issues related to the collaboration.
• Increase the visability of patient and public organizations with the research and clinical communities by encouraging PIO involvement in scientific conferences.
• Find ways for the PIOs to raise awareness of and encourage participation in clinical studies.

Dr. Martin encouraged greater dialogue between ATS and PIOs, and between PIOs and the NHLBI, to "connect" better with patients. He noted that, through advocacy, PIOs can energize, catalyze, stimulate, and start research "where none was done before."

Organizations interested in blood diseases and the safety and availability of blood resources participated in a discussions led by:

• Dr. Liana Harvath, Deputy Director, Division of Blood Diseases and Resources (DBDR), NHLBI
• Dr. George Dover, American Society of Hematology

Dr. Harvath reviewed the structure of DBDR and some major advances achieved by investigators with support from DBDR. The division consists of two programs: the Blood Diseases Program and the Blood Resources Program. It supports research to discover treatments and improve the quality of life for persons with blood diseases and disorders, and ensure the adequacy and safety of the nation's blood supply. Through this support, the NHLBI has improved the length and quality of life of persons with sickle cell disease and Cooley's anemia and has developed new and more effective treatments for thrombosis associated with heart attacks and stroke and for sepsis. In addition, the NHLBI support is advancing the possibility of gene therapy for patients with blood diseases, harnessing the potential curative powers of hematopoietic stem cells, and continuing to reduce or eliminate infectious agents from the U.S. blood supply.

Dr. George Dover reviewed the mission of the ASH and described some new directions it plans to pursue. The ASH is a professional society whose mission is "to further the understanding, diagnosis, treatment, and prevention of disorders affecting the blood, bone marrow, and the immunologic, hemostatic and vascular systems, by promoting research, clinical care, education, training, and advocacy in hematology." Its members are hematologists and oncologists. The ASH currently is focusing attention on taking a more active educational role (e.g., recruiting young persons into hematology), increasing interaction with patient groups, and supporting the development of "best practices" for specific diseases.

The participants raised specific concerns and suggested steps that the PIOs, the NHLBI, and the ASH could take to foster collaboration.

Genetic Aspects of Disease. The participants noted that diagnosis, counseling, and therapy are more complicated for diseases involving more than one gene mutation. They also noted that genetic screening of patients involves important ethical questions that need to be addressed and resolved in cooperation with patients.

Blood Safety. NHLBI staff explained that research to enable the safety of blood (i.e., by reducing or eliminating infectious agents) is funded by the NHLBI. The FDA has regulatory authority for actually ensuring the safety of blood in the United States.

Information Exchange. Participants expressed different views about the potential effects of the Federal Health Insurance Portability and Accounting Act (HIPAA) regulations, which become effective in spring 2003, on the exchange of information between patients and physicians. One view is that the HIPAA regulations will deter physicians from interacting with patient groups and may hinder clinical trial recruitment; another view is that the regulations will foster positive changes (e.g., development of registries of patients with rare diseases). The uncertainty surrounding the HIPAA regulations and the potential for disrupting information sharing among research institutions and patient organizations are significant concerns for PIOs and the patients they serve.

Pharmaceutical Resources. The participants noted that a listing of drug companies and their products, organized by disease, would be a useful resource for PIOs. The Physicians' Desk Reference is one source of this information. Information on drugs for rare diseases, which often are prescribed "off label," is more difficult to find. NHLBI staff noted that development and dissemination of information on drugs is beyond the research mission of the Institute.

The participants suggested the following ways that PIOs could foster collaboration and communicate their messages to both patients and researchers:

• Enlist the help of PIO medical advisory boards in attracting specialists (e.g., hematologists) to PIO conferences and meetings.
• Enhance communications between PIOs and professional societies (e.g., the ASH) and their members.
• Develop conferences in collaboration with the NHLBI, ASH, and other PIOs.
• Encourage PIO medical advisory boards to help develop accurate messages that can give hope to patients.
• Team up with the National Organization for Rare Disorders (NORD) to get messages to the public.

The participants suggested the following ways that the NHLBI could help in disseminating information to patients:

• Continue to collaborate with PIOs to sponsor forums for interaction with their constituencies.
• Convene experts to develop evidence-based guidelines for state-of-the-art treatments (e.g., bone marrow transplantation) that could benefit patients with certain diseases.
• Continue to support scientific meetings and workshops that address areas of doubt or controversy in science and that include representation from PIOs.

The PIOs should identify and communicate to NHLBI staff any issues or areas that could potentially be addressed in an NIH workshop or conference.

Dr. Dover encouraged the PIOs to provide feedback to ASH regarding the impact of the association's new programs to broaden research conferences.