Update from NLHBI - NHLBI Update...CV, lung, and blood diseases accounted for 41% of deaths in the U.S. in 2012 or ~1 million lives lost Charting the Future Together: The NHLBI Strategic
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Update from NLHBI Gail D. Pearson, MD, ScD Associate Director, Division of Cardiovascular Sciences Director, Office of Clinical Research NHLBI/NIH Congenital Heart Surgeons’ Society November 18, 2016
Gail D. Pearson, MD, ScD Associate Director, Division of Cardiovascular
Sciences Director, Office of Clinical Research
NHLBI/NIH
Congenital Heart Surgeons’ Society November 18, 2016
National Heart, Lung, and Blood Institute
Dr. Gary Gibbons Director
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NHLBI is one of 27 Institutes and Centers that make up the National Institutes of Health
The Financial Picture
FASEB, NIH Budget Office
NHLBI 10%
Rest of NIH 90%
Majority – investigator initiated research
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NHLBI is the 3rd largest Institute, and receives ~10% of the NIH budget NIH appropriations overall have lost about 25% in spending power since the end of the doubling of the NIH budget in 2003 The majority of the budgets of all NIH Institutes goes to fund investigator-initiated research
Shaping Today’s Investments Toward the NHLBI of 2025: Imagine a World in Which…
What IF… We were able to prevent and pre-empt
the burden of cardiovascular, lung, and blood diseases?
We achieved a stroke-free generation of patients with Sickle Cell Disease?
We realized options for precision and personalized medicine for HLBS diseases?
CV, lung, and blood diseases accounted for 41% of deaths in the U.S. in 2012 or ~1 million lives lost
Charting the Future Together: The NHLBI Strategic Vision
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The National Heart, Lung, and Blood Institute (NHLBI), together with its partners, has released a new Strategic Vision that will chart the course for scientific discovery in heart, lung, blood and sleep research in the next decade. Many of NHLBI’s research goals that once seemed remote are now within reach. For example, in the next decade, preventable cardiovascular disease deaths could decline by more than 30 percent with greater adoption of proven prevention and treatment. A stroke-free era for all children with sickle cell disease is imaginable. The NHLBI Strategic Vision will serve as a roadmap to identify and prioritize the most compelling science that is critical to closing existing gaps in knowledge and breaking down barriers to research progress. The four goals: understand human biology, reduce human disease, advance translational research and develop workforce and resources, will enable us to accelerate our journey towards scientific and health advances over the next decade.
Strategic Visioning Engagement
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Through a dynamic and iterative process between Fall 2014 and Summer 2015, we engaged diverse stakeholders from across the U.S. and around the globe and received an unprecedented number of ideas that have collectively informed the development of this Strategic Vision. NHLBI sought to: anticipate and capitalize on emerging scientific opportunities, foresee and identify approaches to overcome emerging barriers to progress, and acknowledge transformational scientific advances that are emerging at an accelerated pace. The effort resulted in a remarkable response from the heart, lung, blood, and sleep community that exceeded expectations. Nearly 4,500 of our peers from all 50 states and 42 countries provided ideas and feedback in the form of 1,235 compelling questions and critical challenges that identified the greatest gaps in knowledge and barriers to research progress facing HLBS research today. With extensive input from the National Heart, Lung, and Blood Advisory Council and Board of External Experts, the NHLBI reviewed the submitted ideas and associated feedback and selected a set of Strategic Research Priorities that resonated as high priorities for the Institute based on timeliness, feasibility, and potential to advance the fields of study. This engagement process used to develop these research priorities was a first for NHLBI and a collective approach to setting a research agenda; the results reflect the diversity of ideas from the public and the research communities.
Strategic Visioning Engagement
Presenter
Presentation Notes
Through a dynamic and iterative process between Fall 2014 and Summer 2015, we engaged diverse stakeholders from across the U.S. and around the globe and received an unprecedented number of ideas that have collectively informed the development of this Strategic Vision. NHLBI sought to: anticipate and capitalize on emerging scientific opportunities, foresee and identify approaches to overcome emerging barriers to progress, and acknowledge transformational scientific advances that are emerging at an accelerated pace. The effort resulted in a remarkable response from the heart, lung, blood, and sleep community that exceeded expectations. Nearly 4,500 of our peers from all 50 states and 42 countries provided ideas and feedback in the form of 1,235 compelling questions and critical challenges that identified the greatest gaps in knowledge and barriers to research progress facing HLBS research today. With extensive input from the National Heart, Lung, and Blood Advisory Council and Board of External Experts, the NHLBI reviewed the submitted ideas and associated feedback and selected a set of Strategic Research Priorities that resonated as high priorities for the Institute based on timeliness, feasibility, and potential to advance the fields of study. This engagement process used to develop these research priorities was a first for NHLBI and a collective approach to setting a research agenda; the results reflect the diversity of ideas from the public and the research communities.
The NHLBI Strategic Vision: Objectives
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Investigator-initiated research remains a top priority for the Institute The Strategic Research Priorities will help shape Institute-solicited research, and, because the objectives were developed with community input, it is our wish that they inspire the research community at large. Flowing from these goals are eight objectives. These objectives provide an organizing framework and focus for the Strategic Research Priorities. While over the next decade, these objectives will serve as the Institute’s guide for moving HLBS science forward, exploring research opportunities, and determining funding opportunities, it is important to note the Strategic Vision is not meant to be a comprehensive picture of the science needed to advance health.
The NHLBI Strategic Vision: Objectives
Presenter
Presentation Notes
Investigator-initiated research remains a top priority for the Institute The Strategic Research Priorities will help shape Institute-solicited research, and, because the objectives were developed with community input, it is our wish that they inspire the research community at large. Flowing from these goals are eight objectives. These objectives provide an organizing framework and focus for the Strategic Research Priorities. While over the next decade, these objectives will serve as the Institute’s guide for moving HLBS science forward, exploring research opportunities, and determining funding opportunities, it is important to note the Strategic Vision is not meant to be a comprehensive picture of the science needed to advance health.
What underlies secondary resilience, such that some people are protected from complications of HLBS diseases?
Which phenotypic, biomarker, and molecular characteristics predict outcome and differential responses to therapy in individuals and populations?
What are the optimal blood product transfusion strategies in pediatric and adult patients?
How should management of congenital heart disease be modified in the transition to adulthood?
Compelling Questions
Integration of registry data and research datasets is needed to facilitate research on the molecular genomics and pathobiology of CHD, including the natural history of CHD across the lifespan
A variety of ‘smart’ devices are needed to monitor physiology and assist, adjust, or intervene automatically to treat acute complications of cardiovascular disease
Creative and innovative methods are needed to integrate and analyze data from population and cohort research to generate hypotheses and to expedite bedside-to-basic ‘reverse translation’
Critical Challenges
Join the conversation and share our Vision! #NHLBIVision @nih_nhlbi
See the full report and resources: https://www.nhlbi.nih.gov/about/documents/strategic-vision
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Strategic Visioning will remain a “living” process. As NHLBI moves forward with implementation, our engagement with the community will continue. We anticipate an ongoing dialogue to continue to gather inputs from our community regarding our scientific directions while also keeping our strategic research priorities nimble and adaptive to scientific advancements and opportunities. We encourage you to share the NHLBI Strategic Vision with your networks and use the internal email signature. NHLBI is grateful for all the input received to help develop this Strategic Vision. We look forward to working with you and welcome comments on all that we do together.
NHLBI funds research in two major ways: Ideas can come from the investigator or the institute. Investigator initiated applications begin with a great idea from a bright scientist with a particular area of interest. The majority of our research is funded through investigator initiated grants. Based on our Strategic Vision or newly-identified gaps, the Institute may release requests for applications or program announcements that solicit applications from particular areas of science. The Institute may also design large consortia or networks to address these gaps.
www.nhlbi.nih.gov
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The “Researchers” section of the NHLBI web site, which can be accessed from the home page, has a wealth of information to guide investigators to the right mechanism for them.
Overview of the Grant Process
Have a great idea. Write a great application. Send it in.
• Receive it. • Study section. • Council. • Award.
Conferences critical to the mission of NHLBI Must receive permission to submit 3 receipt dates/year Generally $5K - $20K https://www.nhlbi.nih.gov/research/funding/r13
Spectrum of Research Training & Career Development Awards
Graduate and Medical School
Postdoctoral Training
Transition to Independence
Established Investigator
T32 Institutional award T32 Minority Institutions
F30 Dual-Degree F31 Minority students &
individuals with disabilities
T35/R25 Short-term training institutional award for minority
students
F32 Individual award
T32 Institutional
award
T32 Minority Institutional award
K01 K08 K23 K25
K99/R00
K12 & KL2 Programs
F33
K24
K25
Diversity and Re-entry Supplements to Grants and Contracts
Mentored Career Development Awards Goal: To transition post-docs/ junior faculty
into successful R01 investigators by: Providing training needed for a research
career Supporting career development activities Protecting time and effort in early stages of
research career
3-5 years Up to $100K per year, plus benefits Research development support up to $50K 75% effort 50% effort for CV surgery, interventional
cardiology, EP
Mentored Career Development Awards
Mentored Career Development Awards
K08: Mentored Clinical Scientist Development Award Clinical doctoral degree Basic or applied research
K23: Mentored Patient-Oriented Research Career Development Award Clinical doctoral degree Research conducted with human subjects Training in areas related to human subjects research Does not include analysis of existing databases
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5 years Phase I Mentored research (K99) 1-2 years Phase II Independent research (R00) 1-3 years, contingent on
securing an independent research/faculty position
U.S. citizenship/permanent residency not required
Ph.D. or clinical degree Max 4 years post-doc research experience
Minimum 75% effort K99: $75K salary, $25K research support/year R00 component , up to $249K/year
K99-R00 Pathway to Independence Award
K 24 Mid-Career Patient-Oriented Research Award Associate or Full Professor
25% effort to protect time More time to augment capabilities in patient-oriented
research
Mentoring time for new clinical investigators
Support: up to 5 years; one renewal
Salary: Subject to NIH cap
Research development support: up to $50,000
US citizen or permanent resident
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If full professor, must be within 10 years of appointment to this rank
Primary Children’s Medical Center Salt Lake City, UT
Medical University of SC Charleston, SC
North Carolina Consortium
Toronto Gen. Hospital Toronto, CA
Hospital for Sick Children Toronto, CA
New England Research Inst Watertown, MA Children’s Hospital
Boston, MA
Mt. Sinai Hospital New York, NY
Presbyterian Hospital New York, NY
Children’s Hospital New York, NY
Children’s Hospital Philadelphia, PA
Johns Hopkins Baltimore, MD
NHLBI Bethesda, MD
Children’s Hospital Pittsburgh, PA
Children’s Hospital Seattle, WA
Cedars-Sinai Los Angeles, CA
Vanderbilt University Nashville, TN
Children’s Hospital Minneapolis, MN
University of Ghent Belgium
Children’s Memorial Chicago, IL
Washington University St. Louis, MO
Prairieland Consortium Cincinnati Children’s, OH Riley Children’s, IN
Nemours Children's Clinic Wilmington DE
University of Michigan Ann Arbor, MI
Children’s Hospital Milwaukee, WI
All Children's Hospital St. Petersburg, FL
Funding Agency (NHLBI)
Clinical Sites
Auxiliary Sites
Data Coordinating Center
Protocol Chair
Children’s Hospital Los Angeles, CA
Children’s Hospital of Atlanta Atlanta, GA
Protocol Chair, UTSW Dallas, TX
Rady Children’s Hospital San Diego, CA
Children’s National Medical Center, Washington, DC
Pediatric Heart Network 2016
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The Pediatric Heart Network (PHN) was created by NIH in 2001 as an infrastructure to support clinical research in pediatric acquired and congenital heart disease. Today, the PHN is made up of nine main clinical sites, 23 auxiliary sites, a data coordinating center, and the NHLBI Program Office.
The Next Seven Years
Industry Non-PHN Investigators
PHN Investigators
DCC Clinical Sites
Proposals
High Quality Clinical Research
Proposals
Industry Funding Grants
PHN Funding
Propose a Study
Idea
PHN Leadership Triage
Steering Committee Presentation
Executive Committee Vote
Protocol Development
1 page synopsis OR <10 page proposal Support from PHN site PI
Funding: PHN External Sources
AHA, NIH R01, etc.
Standard study: All PHN Sites will participate Pilot study: Fewer sites permissible
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Any investigator can also propose an idea for a PHN study. For full studies, all 9 clinical sites are expected to participate; however, for pilot studies, participation of fewer sites is permissible. If you have an idea, this should be communicated to the PHN leadership in the form of a 1 page synopsis or a 10 page proposal (max!). The forms and instructions are available on the PHN website. It is necessary to have support from a PHN main site PI when proposing a study. After triage by the PHN leadership, study proposals are presented to the larger PHN community at the twice a year steering committee meeting for information and discussion. After presentation at the SC meeting, the executive committee will vote on whether to put the trial forward for protocol development. Funding for PHN studies can either come from within the PHN or from external sources such as NIH R01 applications, AHA awards, or foundation grants.
Location: One of the 9 clinical centers Expertise: Pediatric cardiovascular subspecialty Level: Junior faculty (<5 years out of training)
Senior fellows Pre-doctoral/doctoral nurses
Career Plan: Pursue formal research funding
Apply to be a PHN Scholar
Who
What
How Much
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$75,000 for 1 year
New pilot studies Ancillary studies to PHN studies
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For those junior faculty, senior fellows, and doctoral level nurses at MAIN PHN sites, you have the opportunity to apply to be a PHN Scholar. These scholars propose pilot studies or ancillary studies to existing PHN studies and receive $75,000 of funding from the PHN to do such research. We are currently in our 2nd year of the PHN Scholars program and have funded 13 promising investigators doing great science.
Integrate data across current CHD sources Method: link data locally, create distributed
data network across sites Method: capture longitudinal outcomes Method: standardize data
Circulation 2016
Data Integration
Local Data Center
Warehouse
CHSS
STS
IMPACT
PHTS
EHR
Distributed Research Data Sets
Local Data
Leverage existing data sources to plan, implement and conduct studies Health economics Heath services research
Support data standardization, integration and sharing across sites and data sources
Mentor young investigators
Integrated CARdiac Data and Outcomes Collaborative (iCARD)
New Paradigms: Registry-Based Trials
35 Ness C et al. AHJ 2013 Frobert O et al. AHJ 2010
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Another approach is trials based in registries. Randomize within registry, then follow using data already being collected in registry, plus perhaps some additional data (SAFE-PCI) This solves the problem of difficulty of enrolling – the patients are already there, and also in TASTE, there was no loss to follow-up. SAFE-PCI: National Cardiovascular Data Registry, NCDR, developed by the ACC Radial approach did not significantly reduce bleeding or vascular complications in women undergoing PCI TASTE: Thrombus aspiration did not reduce mortality in patients with STEMI Also, no loss to follow-up, 7244 patients, $50/patient (above registry cost)
Harness genomics strength of PCGC Conduct whole exome sequencing in PHN cohorts
for genotype-phenotype correlation Outcomes include cardiac function, arrhythmias,
