NIH at the Crossroads: Myths, Realities and Strategies for the Future

1

NIH at the Crossroads Myths, Realities and
Strategies for the Future Elias A. Zerhouni,
M.D. Director, National Institutes of Health
2
NIH Budget Facing a Perfect Storm

• Federal Deficit, Defense and Homeland Security
  priority requirements, Katrina, Pandemic flu and
  domestic budget cuts (-2.7 for HHS)
• Congressional support for Physical Sciences for
  competitiveness
• Overall support for NIH is still strong
• Biomedical research inflation in 2004 5.5

3
NIH Budget Myths and Realities
4
Why Are Success Rates So Low?

• Is NIH placing too much emphasis on translational
  science at the expense of basic research?
• Is NIH over-investing in big projects and
  initiatives at the expense of investigator-initiat
  ed research?
• Is it due to the Roadmap?

5
Basic and Applied Research
60.0
56.4
55.8
55.2
55.2
55.2
53.9
56.6
52.1
56.1
53.0
50.0
43.5
41.0
40.8
40.5
41.0
39.8
39.2
40.0
40.8
38.5
38.4
30.0
20.0
10.0
7.0
5.7
5.0
5.5
5.2
4.8
3.7
3.1
3.1
3.6
0.0
FY 1998
FY 1999
FY 2000
FY 2001
FY 2002
FY 2003
FY 2004
FY 2005
FY 2006
FY 2007
Basic Research
Applied Research
Other
6
Why Are Success Rates So Low?

• Is NIH placing too much emphasis on translational
  science at the expense of basic research?
• Is NIH over-investing in big projects and
  initiatives at the expense of investigator-initiat
  ed research?
• Is it due to the Roadmap?

7
NIH is Committed to Investigator-Initiated
Research
14.0
35
12.0
30
12.2
11.2
11.1
10.8
10.9
10.0
25
10.8
10
8.8
8.7
8.0
20
8.6
8
Appropriations in Billions
8.1
8.2
8.3
Number of Announcements/Billion
7.6
8
7
6.6
6.0
15
6.3
5.9
5.8
5.8
4.0
10
2.0
5
0.0
0
1995
1996
1997
1998
1999
2000
2001
2002
2003
2004
2005
Fiscal Year
8
Grants Unsolicited Far Outnumber Solicited
100
90
80
70
60
Percentage of Grants
Unsolicited
Solicited
50
40
30
20
10
0
1994
1996
1998
2000
2002
2004
2006
Fiscal Year
9
Why Are Success Rates So Low?

• Is NIH placing too much emphasis on translational
  science at the expense of basic research?
• Is NIH over-investing in big projects and
  initiatives at the expense of investigator-initiat
  ed research?
• Is it due to the Roadmap?

10
NIH Roadmap for Medical Research

• Developed to
• Increase synergy across NIH
• Respond to concerns about the perceived
  balkanization of NIH (Congress, IOM report)
• Developed with wide extramural input
• Beyond the scope of a single Institute or Center
  and benefits all
• Emerging areas of science
• High risk/high impact research
• Enabling science infrastructure

11
NIDA and the NIH Roadmap

• Co-lead on an Interdisciplinary Research
  Initiative
• Development of innovative methodology in social
  and behavioral sciences research
• Involved in the development of clinical trials
  networks
• Supports the Molecular Libraries and Imaging
  Roadmap Initiative

12
Roadmap in FY05 0.8 of Total Budget
FY2005 Request 28,757M
Non-Roadmap 99.2 (28,520 Million)
Roadmap 0.8 (237 Million)

• Roadmap in FY06
• 1.2 of budget
• 133 institutions, 33 states

13
NIH Roadmap for Medical Research

• Not a single initiative but over 345 individual
  awards in FY05
• 40 basic
• 40 translational
• 20 high risk
• Creates Incubator Space to accelerate critical
  research efforts that address major cross-cutting
  NIH priorities
• This Incubator Space is now a permanent part of
  NIH Office of Portfolio analysis and Strategic
  Initiatives (OPASI)

Roadmap Funding
600
1.2 - 1.7 NIH Budget
500
400
300
200
100
0
FY04
FY05
FY06
FY07
FY08
FY09
14
NIH Directors Pioneer Award Program

• New program designed to support individuals with
  high-risk, potentially groundbreaking ideas
• Example Sunney Xie of Harvard University
• First to show translation of a gene at the level
  of a single protein molecule in a live cell
• His work published in Science and Nature
  simultaneously

Nature, 440358-362 (March 16, 2006)
Science, 3311600-1603 (March 17, 2006)
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What Is Really Happening?3 Fundamental Drivers

• Large capacity building throughout U.S. research
  institutions and dramatic increase in number of
  tenure-track faculty
• Large increase in applicants and applications
  occurring after 2003
• Budgets
• Appropriations below inflation after 2003 ( 3
  in 04, 2.2 in 05 and 0 in 06 ) while BRDPI in
  2004 was 5.5
• 4-year budget cycling phenomenon

16
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Comparing the of Grant Applications Funded (or
Success Rate) with the Number of New
Applications
35
60,000
32
32
30
32
31
50,000
31
30
49,656
25
46,501
25
40,000
43,069
Projected
40,862
22
20
Number of Applications
19
Success Rate of Grants Funded
30,000
19
34,710
15
28,368
27,798
30,069
26,407
20,000
24,154
10
10,000
5
0
0
1998
1999
2000
2001
2002
2003
2004
2005
2006
2007
Success Rates
Applications
18
As Many New Applications in the Last 2 years as
During the Doubling

8359
43069 - 34710
(2005) (2003)
19
As Many New Applicants in the Last 2 years as
During the Doubling

20
What Funds are Really Available in Any One Year?
From current year to previous year
Budget Increase
Uncommitted Funds
From ending grants started 4-5 years ago
Continuing grants
Committed Funds
NIH Appropriations
21
NIH Congressional Appropriations
30
28.6
28.6
28.6
28.0
27.1
25
?
23.3
20
20.5
17.8
15
Billions of Dollars
15.6
13.7
10
5
0
FY
FY
FY
FY
FY
FY
FY
FY
FY
FY
1998
1999
2000
2001
2002
2003
2004
2005
2006
2007
DOUBLING
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Why is the Transition so Painful?

• Demand for grants took off (2002- 2006) just as
  the NIH budget was landing (1999-2003)
• More increase in demand for grants in last 2
  years than in the full 5 years of the doubling
• NIH budget increases below inflation
• Budget cycle effects- uncommitted pool in 2006
  comes from 2001-2002 when doubling did not yet
  occur
• 2003 commitments will become available in 2007,
  allowing for a 3 increase in competing grants
  (even with a flat budget)

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The Question on Everyones MindWhat are my
chances of being funded?

• Because number of applications per applicant has
  increased from 1.2 to 1.5, success rate
  understates funding rate
• Translates to approximately 15,000 extra
  applications
• FY 05- 22.3 success rate for applications, but
  27.6 for individual applicants
• FY 06- 19.8 for applications, but 25 for
  individual applicants
• Payline is not the funding cut-off line!

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Where Do We Go From Here? The Vision for the
Future
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Strategies

• First Know the facts
• Second Develop adaptive strategies
• Protect the essential Knowledge and Discovery
• Support new investigators
• New Pathway to Independence Program
• Institute and Center efforts to assist new
  investigators
• Increase number of competing grants
  (supply/demand management)
• Third Convey a unified message
• Increase communications about positive impact of
  NIH on science and health at local, regional and
  national levels
• Communicate NIHs vision for the future

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Coronary Heart Disease Today

• 63 decrease in mortality
• 1 million early deaths averted per year
• 2.6 trillions in economic return
• New effective treatments
• More effective prevention
• Successfully reduced cholesterol levels in the
  population
• New discoveries being developed by industry

500
1,329,000 Projected Deaths in 2000
400
300
Deaths per 100,000
200
514,000 Actual Deaths in 2000
100
95
50
55
60
65
70
75
80
85
90
00
Year
35
30
25
20
population with high serum cholesterol
15
10
5
0
1960-62
1971-74
1976-80
1988-94
1999-02
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Cancer Today

• For the first time annual cancer deaths in the
  United States have fallen
• 10 million survivors
• Early detection and screening are more effective
• New targeted, minimally invasive treatments for
  cancer have multiplied
• New discoveries make it possible for the first
  time to personalize cancer treatment

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Need to Transform Medical Research in the 21st
Century
21st Century
20th Century
Intervene before symptoms appear and preserve
normal function for as long as possible
Treat disease when symptoms appear and normal
function is lost
Understanding preclinical molecular events and
ability to detect patients at risk
Did not understand the molecular and cellular
events that lead to disease
Orders of magnitude more effective
Expensive in financial and disability costs
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The Future Paradigm Transform Medicine from
Curative to Preemptive
Predictive
Preemptive
Personalized
30
The Future ParadigmPreempt Disease
Intolerable
Tolerable
Disease Burden
Cost
Preclinical
Time
31
Transforming medicine through discovery
NIH
32
NIH Competing Funds Remain Relatively Constant
Amount of NIH Extramural Awards (Excluding RD
Contracts)
25,000
Competing
Noncompeting
20,000
15,000
Dollars Awarded (Millions)
10,000
5,000
0
1998
1999
2000
2001
2002
2003
2004
2005
Fiscal Year
33
Predictive End Stage Renal Disease

• End Stage Renal Disease (ESRD)
• 22.8 billion in U.S. public and private spending
  (2001)
• In the past decade, the absolute number of ESRD
  patients more than doubled and the incidence rate
  doubled
• More than 85,000 new cases per year
• Apolipoprotein E (APOE)
• Variation predicts kidney disease progression
• Prediction independent of diabetes, race, lipid
  and non-lipid risk factors

34
Personalize Cancer Treatment

• Impact
• 100,000 women each year can make a more informed
  choice
• 70,000 women do not have to undergo chemotherapy
• Reduces routine cost of treating these patients

Identified 16 informative genes
Test tumor samples for mutations in these genes
Predict which patients need chemotherapy
35
Preemptive HPV Vaccine

• Human Papillomavirus (HPV) infects over 80 of
  15-50 year old women and can cause cervical
  cancer
• Prevent sexually transmitted HPV infection
  prevent cervical cancer
• Anti-viral vaccines are among the most cost
  effective public health interventions (e.g.,
  smallpox, polio, measles)
• NIH has two vaccines currently in clinical trials