Transcript Executive Committee 9-13 - Institute for Clinical and

Building Capacity in Clinical and
Translational Science at the UI
Gary E. Rosenthal, MD
Professor of Internal Medicine and
Health Management and Policy
Director, Institute for Clinical & Translational Science
September 23, 2013
Overview of Presentation
1. National context for prioritizing developmental
efforts in clinical and translational research (CTR)
at the UI
 Recent IOM Report on CTSA Program
 Major concerns of NIH regarding clinical and
translational research
2. ICTS priority areas over the coming 12-18 months
3. Key future issues for clinical and translational
research at the UI
IOM Report on CTSA Program:
Major Recommendations
1. Strengthen NCATS Leadership of the CTSA Program
 Set measurable goals and objectives for the program that
address the full spectrum of CTR
 Form strategic partnerships with NIH institutes and centers
and with other research networks and industry
2. Reconfigure and streamline the efforts of the CTSA
Consortium and establish a new multi-stakeholder
Steering Committee that would provide direction
IOM Report on CTSA Program:
Major Recommendations (cont.)
3. Build on strengths of individual CTSAs in ways that:
 Drive innovation in methods across the spectrum of CTR
 Emphasize interdisciplinary team-based approaches to
research
 Build partnerships with industry, other research networks, &
community groups
4. Standardize evaluation processes for individual CTSAs
and the CTSA Program as a whole that are built around
clear, consistent, and innovative metrics and go
beyond publications & number of grant awards
IOM Report on CTSA Program:
Major Recommendations (cont.)
5. Advance innovation in education & training
 Emphasize training models which focus on team science,
leadership, community engagement, and entrepreneurship
and which offer flexible and personalized training
experiences across the CTR spectrum
 Utilize on line approaches that can be broadly disseminated
6. Ensure community engagement in all phases of CTR
 Ensure active community stakeholder participation in
priority setting and decision making
 Define and clearly communicate goals and expectations for
community engagement at individual CTSA programs
IOM Report on CTSA Program:
Major Recommendations (cont.)
7. Strengthen clinical and translational research relevant
to child health
 identify and designate CTSAs with expertise in child health
research as leaders in advancing clinical and translational
research and as coordinators for CTSA program-wide efforts
 Promote and increase community engagement specific to
child health by raising awareness of opportunities for children
and families to participate in research & by involving patients
and family members in identifying priorities and setting
research agendas.
Major Concerns of NIH
About Clinical Research
(Josephine Briggs, MD, CRF, 12/4/12)
 Studies cost too much
 Studies fail to recruit adequate numbers of patients
to yield interpretable results
 Studies fail to start in a timely fashion
 Studies may not be asking the right questions or
studying the right endpoints
Major Concerns of NIH
About Clinical Research
 Studies cost too much
 Studies fail to recruit adequate numbers of patients
to yield interpretable results
 Studies fail to start in a timely fashion
 Studies may not be asking the right questions or
studying the right endpoints
Studies Cost Too Much
 Costs of conducting clinical trials are 60% of total drug
development costs, compared to 30% in 1980s (Pfizer)
 High costs makes US less competitive worldwide 
Costs of conducting trials 1.5 to 3 times higher in the
US than in India, China, Russia, or Brazil
 E.g., recently funded NHLBI ISCHEMIA Trial
- Randomize 8000 patients with moderate angina on
stress testing to cath & possible revasc or medical
management with 4 year follow-up
- Total cost $84 million  $10,500 per subject
Major Concerns of NIH
About Clinical Research
 Studies cost too much
 Studies fail to recruit adequate numbers of patients
to yield interpretable results
 Studies fail to start in a timely fashion
 Studies may not be asking the right questions or
studying the right endpoints
Failures to Recruit Adequate Numbers of
Patients & Start in a Timely Fashion
 Overall, clinical trials enrollment rates dropped from
75% in 2000 of what was initially planned to 59% in
2006, while subject retention fell from 69% to 48%.
 80% of trials are delayed at least one month because
of unfulfilled enrollment.
 Industry perspective: For each day a drug is delayed
from market, sponsors lose up to $8 M
 Clinical trials suffer from low MD participation with
less than 4% of US MDs participating in trials and 3.5%
relative decline annually since 2001 in the number of
active investigators in the US
Major Concerns of NIH
About Clinical Research
 Studies cost too much
 Studies fail to recruit adequate numbers of patients
to yield interpretable results
 Studies fail to start in a timely fashion
 Studies may not be asking the right questions or
studying the right endpoints
Studies May Not Ask the Right Questions
or Study the Right Endpoints
 Many trials often use placebo as the comparator &
don’t examine if new treatment better than existing
treatments  key question for clinical practice
 Orientation often around postulated biological
mechanisms & measurement of intermediate outcomes
less relevant to patients  e.g., changes in blood
markers or imaging results rather than health or
symptoms
 Often enroll homogeneous patients with limited
comorbidity to reduce response variation
ICTS Priority Areas Over Next 1218 Months
1. Improve subject recruitment for clinical trials
2. Increase UI involvement in multi-site CTR consortia
3. Build capacity to conduct lower cost pragmatic trials
4. Develop functional research data warehouse
incorporating Epic EMR & billing data
5. Promote drug discovery research & entrepreneurship
6. Work with IRB, Sponsored Programs & Compliance to
improve research & contract approval process
7. Support “K to R” transition of junior investigators
ICTS Priority Areas Over Next 1218 Months
1. Improve subject recruitment for clinical trials
 Capitalize on Epic  provide investigators lists of
eligible patients for trials & use BPAs to inform
clinicians of patients’ eligibility at point of care
 Working with UI Marketing Office to more actively
promote clinical trials  new interactive website
 Relationships with Mercy Des Moines to enable
recruitment of subjects in Des Moines for UI trials
 Increase recruitment statewide by participation in
ResearchMarch national registry with transition to
Iowa Heroes Research Registry
ICTS Priority Areas Over Next 1218 Months (cont.)
2. Increase UI involvement in multi-site CTR consortia
 Increasing amount of funding for clinical research
administered through U awards (i.e., contracts)
 Assist UI investigators in competing for
opportunities to participate in clinical research
networks (e.g. NINDS stroke & NHLBI ICU networks)
 Establish regional collaborations with other AMCs
to facilitate recruitment & share best practices
 Promote unique UI resources (e.g., Animal Histology
core, GMP drug facilities)
ICTS Priority Areas Over Next 12-18
Months (cont.)
3. Build capacity to conduct lower cost pragmatic
practice-based trials
 Funding obtained in 2012 from NIH Common Fund
Healthcare Systems Collaboratory initiative
 Development of innovative approaches to facilitate
pragmatic trials  online platforms for informed
consent & collecting patient reported outcomes data
 Create multi-institutional consortium to respond to
new opportunities from PCORI for large pragmatic
comparative effectiveness trials based on EMR data
 Develop model “learning clinic”
ICTS Priority Areas Over Next 1218 Months (cont.)
4. Develop functional research data warehouse
incorporating Epic EMR & GE/IDX billing data
 Working with HCIS on Park Street repository project
 Developing I2B2 warehouse to facilitate linkages
with other institutions and create user friendly
interface for investigators to conduct their own
queries
 Will be critical to success of the CTSA grant and in
responding to new RFAs from PCORI, AHRQ, and
NIH on pragmatic trials
ICTS Priority Areas Over Next 1218 Months (cont.)
5. Promote drug discovery research & entrepreneurship
 New pilot program that will support projects using the
recently established HTS facility
 Recruitment of project manager (Kate Holt) with
biotech start-up experience to help investigators move
projects through developmental pipeline
 Collaborations with other CTSAs to fill gaps in expertise
− Collaboration with KU CTSA – CDA for discovery mining
and early phase drug repurposing trials
− Participation in Stanford SPARK Program
ICTS Priority Areas Over Next 1218 Months (cont.)
5. Promote drug discovery research & entrepreneurship
 Actively nurture collaborations with industry
− Visiting professorships from leaders at Eli Lilly, Cook
Medical, and other companies
− Creation of external advisory board to help identify
promising research and promote investigators
 New tracks in Translational Biomedicine MS program
 Promote changes in institutional culture such that
investigators pursuing product development are
recognized & rewarded
ICTS Priority Areas Over Next 1218 Months (cont.)
6. Work with IRB, Sponsored Programs and Compliance
Office to improve research and contract approval
processes
 Increase UI involvement in shared and reciprocal
IRB review processes for multi-site studies
 Promote adoption of standard contract language to
improve approval turnaround times
 Ensure that UI interpretation of HIPAA and OHRP
regulations is similar to peer institutions so as not to
disadvantage UI investigators
ICTS Priority Areas Over Next 1218 Months (cont.)
7. Increase efforts to support “K to R” transition of
junior investigators through K Club (~50 NIH K,
institutional K12, VA, and professional society career
development awardees)
 Quarterly professional development seminars
 Semi-annual R01 workshops to guide trainees
through the development of different components
of grant
 Semi-annual mock study sections
Key Future Institutional Issues in
Clinical and Translational Science
1. Matching investigative capacity to emerging funding
opportunities (e.g., CER, IPS cells)
2. Identifying institutional priorities for capacity
building & targeting departmental recruiting around
those areas
3. Building research informatics capacity (bio &
medical) to respond to new Big Data opportunities
4. Increasing number of investigators who are national
leaders in clinical trials research and leading trials
5. Conservative institutional attitudes towards risk that
adversely impact IRB & contract approval processes