NATIONAL HEART, LUNG, AND BLOOD ADVISORY COUNCIL

This is a brief summary of the June 13, 2012, meeting of the National Heart, Lung, and Blood Advisory Council (NHLBAC). It will be replaced by the full minutes of the meeting when they become available.

Report of the Acting Director, NHLBI
Biomedical Research: How Do We Know We Are On Track?
NHLBI AIDS Program Overview
Report from the PIO Meeting
Background and Summary of the Expert Panel Report on Management of Sickle Cell Disease
Report of the Council Working Group on Clinical Practice Guidelines
Recognition of Dr. Shurin
Report of the Board of External Experts and NHLBI Initiative Concepts

REPORT OF THE ACTING DIRECTOR, NHLBI

Dr. Susan B. Shurin, Acting Director of the National Heart, Lung, and Blood Institute (NHLBI), welcomed members to the 246th meeting of the National Heart, Lung, and Blood Advisory Council (NHLBAC).

Dr. Shurin updated the Council on NHLBI and NIH leadership appointments:

Dr. Gary H. Gibbons has been appointed the next NHLBI Director. He is expected to start in early August.
Ms. Sandy Gault, NHLBI Financial Management Officer, is retiring at the end of the month.
Mr. Alex Hawkins is the new NHLBI Financial Management Officer.
Ms. Debra C. Chew has been appointed Director, NIH Office of Equal Opportunity and Diversity.

Dr Shurin updated the Council on member news:

Mr. Jonathan Alger has been named President of James Madison University in Harrisonburg, Virginia.
Dr. Lanetta Jordan joined the faculty of the Miller School of Medicine at the University of Miami.

Dr. Shurin discussed the FY 2013 President's Budget. The proposed NHLBI budget remains relatively flat overall, with a total increase of 0.02%, or approximately $700 thousand. However, the Research Project Grant (RPG) line would decrease slightly, in part due to a mandated increase in Small Business Innovation Research (SBIR)/Small Business Technology Transfer Research (STTR) funding. Despite the RPG funding decrease, a slight increase in numbers of competing RPGs is expected because of variation in grant cycles.

The FY 2012 funding payline will remain at the 10th percentile for established investigator R01s and R21s. Pending availability of funds, the Institute intends to make use of the select pay option to fund some grants.

Dr. Shurin discussed the SBIR/STTR programs. Their reauthorization mandated increasing the set-aside percentages over the course of the next 6 years. Currently, a Scientific Management Review Board (SMRB) subcommittee is analyzing the SBIR/STTR program at the NIH. Its charge is to recommend strategies for how the NIH can optimize its use of the SBIR/STTR programs in keeping with the NIH mission. The NHLBI convenes a Topic Review Advisory Committee with a charge to assist NHLBI staff in identifying, assessing, and developing SBIR/STTR ideas for commercial development.

Dr. Shurin updated the Council on two other SMRB recommendations--(1) translational medicine and therapeutics, and (2) substance use, abuse, and addiction research at NIH.

The National Center for Advancing Translational Sciences (NCATS) was created based on the SMRB recommendations. Programs in clinical and translational science (CTSAs), rare diseases research and therapeutics, and re-engineering translational sciences have been imported into NCATS. It has new initiatives in tissue chips for drug screening, rescuing and repurposing drugs, and target validation. Moving forward, NCATS is looking to recruit permanent leadership, convene an advisory council and Cures Acceleration Network board, and issue new RFAS for the CTSAs and the Therapeutic Discoveries Program.
The SMRB recommended that the NIH establish a new institute that encompasses all substance use, abuse, and addiction-related research and dissolve the independent existence of NIAAA and NIDA. The new National Institute of Substance Use and Addiction Disorders is expected to be included in the FY 2014 budget and be launched in October 2013.

Dr. Shurin discussed the proposed policy at NIH for Special Council Review--extra consideration before awarding a grant to a PI with $1.5 million or more annual total costs in NIH research support. This policy will likely be a requirement of the FY 2013 budget.

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BIOMEDICAL RESEARCH: HOW DO WE KNOW WE ARE ON TRACK?

Dr. Michael Lauer, Director, Division of Cardiovascular Sciences, NHLBI presented an update of ongoing activities in the Institute's program divisions on assessing impact and productivity of research investments. He also discussed developing Results Based Accountability as a part of the NHLBI culture.

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NHLBI AIDS PROGRAM OVERVIEW

Dr. Monica Shah, NHLBI AIDS Coordinator, discussed the NHLBI AIDS program. It has an independent budget ($68 million in FY 2012), a separate payline that is approximately 10 percentile points higher than the regular payline, specific receipt dates, and specific review. Success rates of AIDS applications are much higher than those of non-AIDS applications--42% versus 18% in FY 2011. The NHLBI hopes to encourage more submissions of AIDS projects and hopes to understand the barriers to submission.

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REPORT FROM THE PUBLIC INTEREST ORGANIZATION (PIO) MEETING

Dr. Lanetta Jordan, Assistant Professor, Department of Epidemiology and Public Health, University of Miami, Miller School of Medicine, and Council member, reported on the 13th annual NHLBI PIO meeting held June 11-12, 2012. Four Council members attended the meeting in addition to representatives from PIOs and professional societies, and NHLBI staff.

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BACKGROUND AND SUMMARY OF THE EXPERT PANEL REPORT ON MANAGMENT OF SICKLE CELL DISEASE

Dr. Joylene John-Sowah, Medical Officer, NHLBI, and Dr. George Buchanan, Professor of Pediatrics, University of Texas Southwestern Medical Center, and Co-Chair, Sickle Cell Guidelines Expert Panel, presented background and a summary of the Panel's report on the management of sickle cell disease. Dr. John-Sowah discussed the reasons why sickle cell disease management guidelines are needed:

Effective therapies are underutilized
The patient community has clearly stated a need
Guideline-based care improves outcomes and reduces costs
A health care disparity requires addressing
Improvement in quality of life for those affected is desired
Comprehensive evidence-based guidelines are lacking for this disease
Systematic review will inform the research agenda

At the request of the NHLBAC, the NHLBI convened a multi-disciplinary panel to develop evidence-based sickle cell disease guidelines for children and adults, targeting primary care providers, to enhance sickle cell disease management. Dr. John-Sowah outlined the guideline development steps and the review and approval process. Release of the guidelines is expected in December 2012.

Dr. Buchanan discussed the goals of the panel--to generate implementable evidence-based recommendations on managing sickle cell disease in clinical practice and to identify gaps in knowledge for additional research. The guidelines' five major topic areas are:

Health maintenance
Managing acute complications
Managing chronic complications
Use of hydroxyurea therapy
Use of transfusion therapy

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REPORT OF THE COUNCIL WORKING GROUP ON CLINICAL PRACTICE GUIDELINES

Dr. Pamela Douglas, Ursula Geller Professor or Research in Cardiovascular Diseases and Director, Cardiovascular Imaging Program, Duke University School of Medicine, and Council member, presented a report of the Council Working Group on Clinical Practice Guidelines. The working group was asked whether the NHLBI should create guidelines. Dr. Douglas noted that part of the NHLBI strategic plan is, "To promote the development and implementation of evidence-based guidelines." She offered a snapshot of the cost of guideline creation and dissemination and presented pros and cons for creation of guidelines by the NHLBI.

Pros:

A small portfolio of highly respected guidelines is maintained
The guidelines are directed to general practitioners, not just specialists
The guidelines fill important gaps in care
Guideline development is part of the Institute's dissemination and public health missions
Guidelines can help define research needs

Cons :

Many other organizations produce guidelines
The focus of NHLBI guidelines is becoming narrower
Rapid changes in care can lead to guideline obsolescence
The complexity of the guideline development process is increasing
Guideline development has potential clinical and reputational liability
Cost of guideline creation is high

The working group recommended that the NHLBI:

Continue to participate in the production of select clinical practice guidelines, primarily by partnering with professional societies
Continue to create very few stand-alone guidelines if the need is great and no other sponsoring organization is available
Establish a mechanism for topic selection and prioritization

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RECOGNITION OF DR. SHURIN

Dr. Larry Tabak, Principal Deputy Director, NIH, and the Council recognized Dr. Shurin's outstanding service as Acting Director for the past 2.5 years.

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REPORT OF THE BOARD OF EXTERNAL EXPERTS AND INITIATIVE CONCEPTS

NHLBI staff presented 7 new initiatives and 5 renewals, all of which had been reviewed in May by the Board of External Experts (BEE). Initiative development at the NHLBI is a two-cycle process. First, staff within each extramural division develop ideas and potential initiatives, which they present to the trans-NHLBI Idea Forum. Sufficiently developed initiatives are subsequently considered by the BEE, which ranks each and provides accompanying advice.

The Council was mostly supportive of the initiatives presented, but made a number of specific recommendations for consideration prior to their release. The Acting Director, NHLBI, will consider the recommendations of the BEE and the Council and other budgetary and programmatic issues in determining which of the proposed initiatives, if any, to implement.

Strategic Plan Goal I: To improve understanding of the molecular and physiological basis of health and disease, and to use that understanding to develop improved approaches to disease diagnosis, treatment, and prevention

Initiative	Purpose
Building a National Resource to Study Myelodysplastic Syndromes (MDS): The MDS Natural History Cohort Study (N01), RFP	To establish a natural history cohort study and create a prospective collection of genetically and phenotypically well-characterized biospecimens from patients with MDS to advance the understanding of disease progression.
Functional Assays to Screen Genomic Hits (R21, R33), RFA	To conduct functional analysis of identified genetic variations related to heart, lung, blood, and sleep phenotypes, using amenable in vitro or animal model systems.
Mechanisms of Human HIV-Related Lung Disease Research (R01), RFA	To define cellular and molecular events underlying the pathogenesis of HIV-associated lung diseases using biospecimens and clinical data from both HIV-infected patients and controls with an emphasis on clinical translation.
Molecular Atlas of Lung Development (U01), RFA	To build a molecular atlas of late-stage lung development to serve as a critical platform for discovery research to better understand critical events, including alveologenesis.
Topics in Transfusion Medicine (R01, R21, Renewal), PAR	To allow for continuation of review by a special emphaisis panel of applications focused on topics in transfusion.
Trans-Agency Research Consortium for Trauma-Induced Coagulopathy (TIC) (U01), RFA	To enhance an existing NHLBI/Department of Defense collaboration by creating a novel trans-agency program to coordinate research efforts and specimen and data sharing in TIC, allowing for a comprehensive study of the pathophysiological mechanisms underlying TIC and for the translation of emerging mechanistic data into new early diagnosis and effective treatment strategies.

Strategic Plan Goal II: To improve understanding of the clinical mechanisms of disease and thereby enable better prevention, diagnosis, and treatment

Initiative	Purpose
Biologic Specimen and Data Repositories Information Coordinating Center (BioLINCC) (N01, Renewal), RFP	To promote and expand the existing BioLINCC infrastructure to enhance the utility and scientific value of the NHLBI Biologic Specimen Repository and the NHLBI Data Repository.
Clinical Trial Pilot Studies (R34, Renewal), PAR	To facilitate the development of clinical trials by investigators who already have identified a testable hypotheses, a preliminary trial design, and a plan for trial implementation, but lack critical information about the intervention, outcome, recruitment criteria, or other elements necessary to complete the design of the trial.
NHLBI Studies in Systems and Model-Guided Approaches to Pharmacogenomics (SysMAP) (U01), RFA	To develop a research program incorporating systems medicine and quantitative approaches to assess adverse human drug response and foster translational research.
Secondary Dataset Analyses in Heart, Lung, and Blood Diseases and Sleep Disorders (R21), PAR	To stimulate well-focused secondary analyses using existing human datasets to test innovative hypotheses concerning the epidemiology, pathophysiology, prevention, or treatment of diseases and conditions relevant to the NHLBI mission.
Strong Heart Study Core Support (N01, Renewal), RFP	To support core functions of the Strong Heart Study to: (1) enhance the scientific productivity of the study by expanding and promoting use of the database, promoting use of stored samples, facilitating development of ancillary studies, and maintaining cooperation with the participating communities; (2) extend identification and validation of morbidity and mortality events to significantly increase the power of analyses of lower frequency clinical events and younger age groups; (3) enhance the use of biological material through support of the central laboratory to maintain and distribute stored study samples; and (4) promote training and career development opportunities for early career investigators, particularly American Indian investigators.
Translational Programs in Lung Diseases (P01, Renewal), PAR	To encourage collaborative, translational science that will foster a research continuum from basic to applied biomedical research to improve diagnosis and treatment of lung and sleep diseases.