DEPARTMENT OF HEALTH AND HUMAN SERVICES NATIONAL INSTITUTES OF HEALTH NATIONAL HEART, LUNG, AND BLOOD ADVISORY COUNCIL

MEETING SUMMARY OF THE NATIONAL HEART, LUNG, AND BLOOD ADVISORY COUNCIL

June 13, 2012

I. CALL TO ORDER AND REPORT OF THE ACTING DIRECTOR, NHLBI
II. BIOMEDICAL RESEARCH: HOW DO WE KNOW WE ARE ON TRACK?
III. NHLBI AIDS PROGRAM OVERVIEW
IV. REPORT FROM THE PUBLIC INTEREST ORGANIZATION (PIO) MEETING
V. BACKGROUND AND SUMMARY OF THE EXPERT PANEL REPORT ON MANAGMENT OF SICKLE CELL DISEASE
VI. REPORT OF THE COUNCIL WORKING GROUP ON CLINICAL PRACTICE GUIDELINES
VII. RECOGNITION OF DR. SHURIN
VIII. REPORT OF THE BOARD OF EXTERNAL EXPERTS AND INITIATIVE CONCEPTS
IX. CLOSED PORTION

The 246th meeting of the National Heart, Lung, and Blood Advisory Council (NHLBAC) was convened on Wednesday, June 13, 2012, in Conference Room 10, C Wing, Building 31, National Institutes of Health (NIH), Bethesda, Maryland. The meeting was open to the public from 8:05 a.m. until 12:40 p.m. The meeting was closed to the public from 1:15 p.m. until adjournment at 3:00 p.m. Dr. Susan B. Shurin, Acting Director of the National Heart, Lung, and Blood Institute (NHLBI), presided as Chair.

COUNCIL MEMBERS PRESENT:

Mr. Jonathan R. Alger
Ms. Coletta C. Barrett
Dr. Ivor J. Benjamin
Dr. Ingrid B. Borecki
Dr. Barry S. Coller
Dr. Pamela S. Douglas
Dr. Jack A. Elias
Dr. Robert L. Jesse (ex officio)
Dr. Lanetta B. Jordan
Dr. Talmadge E. King
Dr. Barbara A. Konkle
Dr. Leslee J. Shaw
Dr. Gilbert C. White II

COUNCIL MEMBERS ABSENT:

Ms. Beverly W. Hogan
Dr. Naomi L.C. Luban

VIDEO CONFERENCE:

Dr. Ron G. King
Dr. Michael S. Parmacek

TELECONFERENCE:

Dr. Polly E. Parsons (via telephone)

INVITED GUESTS:

Dr. George Buchanan
Professor of Pediatrics
University of Texas Southwestern Medical
Center, and
Co-Chair, Sickle Cell Guidelines Expert Panel

Dr. Joylene John-Sowah
Medical Officer, NHLBI

Dr. Monica Shah
NHLBI AIDS Coordinator

Dr. Lawrence Tabak
Deputy Director, NIH

MEMBERS OF THE PUBLIC PRESENT:

Ms. Janet Choy
IQ Solutions

Ms. Molly Maguire
Lewis-Burke Associates

Ms. Susan Sepelak
Social & Scientific Systems, Inc.

CSR EMPLOYEES PRESENT:

Dr. Julia Krushkal Adkins
Dr. Larry Boerboom
Dr. Dianne Camp
Dr. Joy Gibson
Dr. Khalid Masood

NHLBI EMPLOYEES PRESENT:

Numerous NHLBI staff members were in attendance or were able to view the meeting via closed circuit broad cast.

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I. CALL TO ORDER AND 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 June, after 21 years with the Institute and 12 as the Financial Officer. She will be replaced by Mr. Alex Hawkins, who comes to the NHLBI from the Security Science and Technology Directorate of the U.S. Department of Homeland Security. He has more than 20 years of budget analysis and business leadership in both the public and private sectors.
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 as an Assistant Professor in the Department of Epidemiology and Public Health.

Dr. Shurin discussed the new policy at NIH for Special Council Review for well funded investigators. Accordingly, the NHLBI will provide information to Council on investigators with 1.5 million or more in annual direct costs in NIH research support. This policy will be a requirement of the FY 2013 budget.

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 additional 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.

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II. 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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III. 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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IV. 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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V. 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' four major topic areas are:

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

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VI. 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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VII. RECOGNITION OF DR. SHURIN

Dr. Shurin was recognized for her outstanding service as Acting Director since November 2009, by Dr. Lawrence A. Tabak, Principal Deputy Director, NIH, Incoming Director Dr. Gary H. Gibbons, and the entire Council. Dr. Carl A. Roth was also applauded for his leadership efforts as Acting Deputy Director for the past two-and-a-half years.

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VIII. 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 to the Council.

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 emphasis 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 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.

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IX. CLOSED PORTION

This portion of the meeting was closed to the public in accordance with the determination that it concerned matters exempt from mandatory disclosure under Sections 552b(c)(4) and 552b(c)(6), Title 5, U.S. Code and Section 10(d) of the Federal Advisory Committee Act, as amended (5 U.S.C. appendix 2).

REVIEW OF APPLICATIONS

The session included a discussion of procedures and policies regarding voting and confidentiality of application materials, committee discussions and recommendations. Members absented themselves from the meeting during discussion of and voting on applications from their own institutions, or other applications in which there was a potential conflict of interest, real or apparent. Members were asked to sign a statement to this effect.

The Council considered and recommended 1,284 applications requesting $1,975,121,550 in total direct costs.