National Heart, Lung, and Blood Advisory Council September 2016 Meeting Summary

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

September 20, 2016

The 269th meeting of the National Heart, Lung, and Blood Advisory Council (NHLBAC) was convened on Tuesday, September 20, 2016, in Building 35A, the Porter Neuroscience Center, National Institutes of Health (NIH), Bethesda, Maryland and included the Advisory Council’s Board of External Experts (BEE) Working Group members. Strategic Visioning working groups met in the morning, and the Council meeting began at 12:30 p.m. and ended at 3:41 p.m. Dr. Gary H. Gibbons, Director of the National Heart, Lung, and Blood Institute (NHLBI), presided as Chair.

Council Members attending

Dr. Bradford C. Berk
Dr. James D. Crapo
Dr. George Q. Daley
Dr. Michael R. DeBaun
Dr. Jonathan A. Epstein
Dr. Serpil C. Erzurum
Dr. Fernando Martinez
Dr. Diane Nugent
Dr. Pilar Ossorio
Dr. Bruce M. Psaty
Dr. Véronique Lee Roger
Dr. Anna Maria Siega-Riz
Dr. Jeffrey A. Whitsett
Dr. Phyllis C. Zee

BEE Members attending

Dr. Nancy Berliner
Dr. Eric Boerwinkle
Dr. Barry Coller
Dr. Allen Cowley
Dr. Ronald Crystal
Dr. Karina Davidson
Dr. Christopher Granger
Dr. Philip Greenland
Dr. Judith Hochman
Dr. Deborah Nickerson
Dr. Daniel Radar
Dr. David Scadden

Council Members unable to attend

Dr. Nancy Brown 
Dr. Richard Schofield (ex officio)

Public Attendees

Dr. Reagan Bailey, Purdue University
Ms. Julie Croxford, RTI International
Mr. Dale Dirks, Health and Medicine Counsel of Washington
Dr. Jessica Ellis, Purdue University
Ms. Nuala Moore, American Thoracic Society
Ms. Haley Payne, Health and Medicine Counsel of Washington

Ad Hoc Members attending via teleconference

Dr. Robert Robbins
Dr. Kim Smith-Whitley

NHLBI employees attending 
A number of NHLBI staff members were in attendance.

Other NIH employees attending

Dr. Katherine Malinda, CSR
Dr. Eugene Carstea, CSR
Dr. Irwin Feuerstein, NIH OD
Dr. Vivien Bonazzi, NIH OD

I. CALL TO ORDER AND OPENING REMARKS – Dr. Gary H. Gibbons

Dr. Gary H. Gibbons, Director of the National Heart, Lung, and Blood Institute (NHLBI), welcomed members and called the 269th meeting of the National Heart, Lung, and Blood Advisory Council (NHLBAC) to order at 12:30 p.m.

II. REVIEW OF CONFIDENTIALITY AND CONFLICT OF INTEREST – Dr. Valerie L. Prenger

Dr. Valerie L. Prenger, Acting Director, Division of Extramural Research Activities (DERA), NHLBI, made the required announcements for the Council meeting, which included the fact that a notice of the meeting was published in the Federal Register, that Council members may not engage in lobbying activities while attending Council meetings or sponsored events, and that the open session was being recorded.

III. CLINICAL TRIALS UPDATE

Dr. Amy Patterson, Chief Science Advisor, Office of the Director, NHLBI, discussed new NIH
clinical trial policies and highlights of proposed clinical trial Funding Opportunity Announcements (FOAs).

NHLBI’s vision is to address major compelling scientific questions that will inform the diagnosis,
treatment, and/or prevention of heart, lung, blood, and sleep (HLBS) disorders. Prerequisites to achieve this vision include trials that launch successfully, and reach completion. NHLBI–funded trials shape clinical practice, extending and improving the health of millions. However, challenges to the success of these trials occur with startup, regulatory processes, and accrual of sufficient patients. To address these challenges, the NHLBI Clinical Trial Working Group deliberations began with a Request for Information (RFI) in the fall of 2015.

One initial activity is to optimize funding solicitations and peer review of NHLBI clinical trials so that operational feasibility is addressed. The NHLBI currently offers multiple pathways for investigator-initiated clinical trials, including: R01 FOAs; P01 FOAs; Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) programs parent FOAs; Kaward FOAs; and pilot study FOAs (R34). For future submissions of clinical trial applications, the Institute is launching a suite of FOAs aimed at optimizing trial conduct and execution. Two such FOAs have recently been published and one is forthcoming:

•  The FOA for multisite clinical trials in Phase II and beyond uses a phased, milestone driven approach involving cooperative agreements for Clinical Coordinating Centers (CCC) and Data Coordinating Centers (DCC).
• The phased, milestone-driven plan for single-site clinical trials in Phase II and beyond goes into effect next fiscal year.

To address investigators’ needs for “on-ramps,” NHLBI developed an Early Phase Clinical Trials for Diagnostics and Therapeutics FOA which is anticipated to be published in Summer 2017. Important new changes in NIH clinical trial policies will take effect next year including that all clinical trial applications must be submitted to a clinical trial–specific FOA (https://grants.nih.gov/grants/guide/notice-files/NOT-OD-16-147.html). Additional FOAs are being planned to complete the Institute’s suite of funding opportunities specific to clinical trials. Dr. Traci Mondoro, Branch Chief, Division of Blood Diseases and Resources, NHLBI discussed a proposed phased mechanism for mechanistic and safety trials. The proposed R61/R33 mechanism would allow 2 years for preclinical activities (R61) and 3 years for clinical trials (R33). Agreed-upon milestones will determine the readiness to transition from the R61 and the R33. Dr. Donna DiMichele, Deputy Director of the Division of Blood Diseases and Resources, NHLBI discussed a clinical trials planning mechanism that could prove to be especially important for late-phase clinical trials (Phase II and beyond) as well as for innovative trial designs.

To assist potential researchers with navigating through these new FOAs, NHLBI is developing
websites specifically for investigator resources, including direct links to FOAs.

IV. REPORTS FROM WORKING GROUP DISCUSSIONS

Dr. Jonathan Epstein, Council member, facilitated this portion of the meeting. The Chair of each
Working Group presented their charge, suggestions, conclusions, and additional questions. Each presentation was followed by a discussion to refine and identify the ideas.

Early Translational Research Working Group

Dr. Serpil Erzurum (Council member) presented this working group’s deliberations. The group
discussed empowering investigator-initiated translation of discoveries; facilitating translation of disease-specific therapeutics, devices, and diagnostics; developing resources and innovative
techniques to facilitate translation of findings; and training a diverse scientific workforce fluent in
translation and commercialization issues.

Below are some examples of topics that arose during the working group discussion:

•  Linking genotypic/phenotypic data with electronic health record data
•  Identifying specific topics that cross many of the compelling questions and critical challenges in the Strategic Vision for deeper focus

Data Science Working Group

Dr. Veronique Roger (Council member) and Dr. Nancy Zhou (NHLBI staff member) presented
this working group’s deliberations. The group discussed short- and mid-term needs of the HLBS
community in data science and how NHLBI can help facilitate broad accessibility and sharing of
existing HLBS data. The group provided input and considerations for an NHLBI data commons
strategy, platforms, and tools to facilitate research and analyses that leverage rich HLBS data
sources.
Below are some examples of topics that arose during the working group discussion:

• Critical nature of workforce development including training and mentoring to enhance the success of scientists
• Importance of ongoing engagement of the public
• Enhancing loan repayment programs

Physician Scientist Workforce Working Group

Dr. Karina Davidson (BEE member) presented this working group’s deliberations. The group brainstormed innovative ideas and novel mechanisms to promote a diverse and ample cadre of well-trained physician scientists who attain productive careers in HLBS research.

The group noted that the nature of science and training has changed. Some examples of ideas
discussed by this working group include the following:

• Research opportunities in residency
• Exposure of clinician scientists in training to successful clinician educators and role models
• Importance of consideration of all stages of training from medical school through residency, fellowship, and research training

V. NEXT STEPS FOR IMPLEMENTATION OF THE STRATEGIC VISION

Dr. James Kiley, Director of the Division of Lung Diseases, NHLBI, summarized the concepts presented by the working groups and outlined some of the next steps in the implementation plan. He indicated that staff wanted to hear the Council and working groups impressions about the entire strategic visioning and thoughts about how to move it from development to implementation and wanted to see where the members thought the most pressing priorities are.
Dr. Kiley pointed out the benefit of bringing the Council and working groups into the process earlier so their input can be used to help shape the ideas, instead of reviewing and commenting on the finished product. The ideas still need to be brought through the appropriate review within the Institute before they are ready. He concluded with telling the members that if they thought something was missing they could let staff know.

VI. CONCLUDING REMARKS

Dr. Gibbons highlighted the progress that the Council had made in advancing the Institute’s strategic visioning process and thanked the members for their contributions prior to adjourning the meeting.

IV. ADJOURNMENT

The meeting was adjourned at 3:41 p.m.