1. The Role of the Indiana CTSI: Impacting the Life Sciences Sector, Translating Science Into Therapies November 12, 2015

2. Chris Eckerle, Project Director, BioCrossroads

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4. The Role of the Indiana CTSI: Impacting the Life Sciences Sector, Translating Science Into Therapies November 12, 2015

5. Indiana’s Position in the Past, Present & Future November 12, 2015

6. GCRCs described by Congress as “critical” infrastructure for POR. GCRCs expected to be a driving force behind future biomedical research. 80 GCRCs. 2004 2005 2006 2012 Following the first round of awards, NIH added between 5-14 new sites each year until the program reached level of 62 sites nationwide in 31 states and the District of Columbia. NCRR dissolved, and National Center for Advancing Translational Science (NCATS) founded. Implementation of the NIH Roadmap and efforts to revitalize clinical and translational research lead to phasing out of GCRC program. Funding consolidated and redirected to launch CTSA program. Evolution of the Translational Science at NIH 1970

7. 2004: The NIH Roadmap 2005: Conceiving the Program 2007: Anticipating the New Era Evolution of NIH CTSA

8. CTSA Today

9. NCATS Mission To catalyze the generation of innovative methods and technologies that will enhance the development, testing and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions.

10. Some of the scientific translational problems on NCATS’ to-do list ► Predictive toxicology ► Predictive efficacy ► Derisking undruggable targets/untreatable diseases ► Data interoperability ► Biomarker qualification process ► Clinical trial networks ► Patient recruitment ► Electronic Health Records for research ► Harmonized IRBs ► Clinical diagnostic criteria ► Clinical outcome criteria (e.g., PROs) ► Adaptive clinical trial designs ► Shortening time of intervention adoption ► Adherence ► Methods to better measure impact on health…

11. Some of the organizational translational problems on NCATS’ to-do list… ► Data transparency/release ► IP management ► Integration of project management ► Incentives/credit for team science ► Incentives/credit for health improvements ► Education/Training (scientific and cultural) ► Collaborative structures ► Public-private partnership models

12. Recent Developments  In its 2013 report, the Institute of Medicine (IOM) recommended the creation of a Working Group within NCATS.  In 2014, the Working Group set forth measurable goals for the CTSA program including:  Workforce development  Collaboration and engagement of stakeholder communities  Integration of translational science across lifespan  Method and process improvement to enable hubs to function individually and collectively  The 2014 CTSA RFP was revised to emphasize patient and community engagement, and collaboration between CTSA hubs

13. CTSA 2015  The 2015 CTSA RFP is expected to be refashioned even more significantly in response to IOM and Working Group recommendations, and feedback over the last year  Anticipated changes include:  A focus on funding program priorities  Expanding to fund more sites using smaller grants  Emphasis on interdisciplinary team science  Recognizing the value of non-traditional contributions of CTSA hubs to the national platform  Creating new models for POR  It is clear that CTSA remains a priority for the Administration  President Obama’s FY2016 budget request asked for 72% of total NCATS funding to be allocated to CTSA

14. Evolving the CTSA Program to Transform Clinical Translational Science RIC: Recruitment Innovation Centers Feasibility Assessment Recruitment Plan and Implementation TIC: Trial Innovation Centers Central IRB Contracting Budgeting Other support PRN Multi-site Study funded by NIH IC or others CTSA Hubs Clinical Lead Stats/Data Management No need to re-build trial components each time

15. Domain Task Forces

16. The Indiana CTSI

17. Indiana’s CTSI: Report Methodology 1. Several metrics were developed to measure CTSA site performance. Data collection methods included: a) Literature review b) Interview with site leaders c) Publicly-reported data d) Insights from key opinion-leaders 2. Six candidate CTSA hubs were selected to inform the report based on eight key measures: Three “comparable” hubs were chosen with similar funding levels to Indiana University’s, and two “outlier” hubs were chosen with much higher levels of NIH funding than Indiana University 3. Three key areas of CTSA activity were identified: a) Translational Science b) Community engagement and impact c) Education and workforce development 4. Measurable variables and qualitative data were used to generate individual site reports, summaries, and key comparisons

18. Selected CTSA Hubs Institution Funding Level for CTSA PIs (FY14) Total NIH Funding (FY14) Total Bioscience Industry Employment Population of the Region School of Public Health Health System Health Ranking* Indiana University $6,715,523$147,332,68457,6441,953,961Yes 41 Albert Einstein College of Medicine $5,217,637$157,391,49976,07019,949,502NoYes15 Emory $6,229,144$265,282,29627,6675,522,942Yes 38 University of Texas $6,451,913$156,565,93680,7926,810,913NoYes36 Washington University $10,722,640$371,946,94928,1092,801,056NoYes39 Stanford $10,497,336$384,340,065235,8641,919,641NoYes21 * America's Health Rankings: http://www.americashealthrankings.org/about/annual

19. Key Findings Across Hubs  Positive shift in relationships within universities  Increased collaboration and interaction within the university, as well as with university leadership  Blurring the lines between research program and clinical care  The extent to which the CTSA hubs are informing and partnering with local responses to health care reform pressures is striking  Concern about the future direction of CTSA program  The use of a new CTSA hub funding formula tied to the host institution’s overall NIH funding level  Rising emphasis on standardizing processes around translation and especially in the conduct of clinical trials

20. Key Findings Across Hubs  Incentivizing Team Science  Program has maintained emphasis on enabling multi-disciplinary and multi-organizational approaches to research and development  However, while there was a consistent acknowledgement of the need for this strategic shift, the traditional mechanisms for professional promotion and recognition at the hubs remain largely intact  Industry Remains Substantially at Arm’s Length  For the majority of sites contacted for this report, external industry relationships do not play a central role in the CTSA hub  Community Engagement  All hub leaders were quick to acknowledge the importance of community engagement  But, it is more difficult to plan and execute in a rigorous manner as both the nature and degree of CTSA program expectations are in flux

21. Summary of the Indiana CTSI Structure

22. Key Findings – Indiana CTSI  The Indiana CTSI is a leading CTSA program hub when compared to peers across the nation.  Very few comparable mechanisms exist in the region with the skills, flexibility, and mission to bridge the academic, industry, and clinical components of the sector:

23. Where does Indiana’s comparative strength come from?  A founding principal investigator who is uniformly recognized as a translational science thought leader  Industry commitment at the highest levels  Multitude of organizations that are co-invested with the Indiana CTSI across translation, commercialization, and health impact  Perhaps most importantly, a supportive regional life sciences sector that increasingly values and rewards collaboration among its participants with the Indiana CTSI Key Findings - Indiana CTSI

24. CTSA Future Challenges Challenges ► People are unhealthier and funders of biomedical research enterprise (private and public) are impatient ► The number of new drugs approved by the FDA per billion dollars spent on R&D has halved every year since 1950 Congress & the Public: What are we getting for our nation’s investment in the NIH? 2008: 800,000 research papers published. 2011: 21 Drugs approved. NYT. David Borenstein

25. CTSA Future Opportunities Opportunities – We are entering a pivotal stage of development for the program as it grows from a definitional phase to maturity – Expectations for the CTSA program to deliver will continue to mount and continue to place pressure on the NIH and recipient institutions – The question will remain: Is the CTSA bringing more innovation into clinical practice and improving health outcomes for Americans to make it worth the investment? – If hubs continue to rise to the challenge as Indiana has, then the answer is yes

26. The CTSI as the translational HUB in Indiana Accelerating Clinical and Translational Research SPARC Indiana CTSI launches multi-state consortium to spark translational medicine collaborations

27. @BioCrossroads #CTSI #INlifesciences

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