Gaining Ground in Translation Francis S. Collins, M.D., Ph.D. Director, National Institutes of Health

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1 Gaining Ground in Translation Francis S. Collins, M.D., Ph.D. Director, National Institutes of Health

2 Some Key Challenges in Biomedical Research Providing robust methods and tools for translation Conducting efficient/effective clinical research Managing and analyzing BigData Generating reproducible research findings Ensuring the capability and size of the biomedical research workforce What Role Can be Played by the CTSAs?

3 Translation Challenges Deluge of new discoveries of potential targets Unmet therapeutic needs for many conditions, especially rare and neglected diseases Need to view drug development pipeline as a scientific problem ripe for experimentation and process engineering 3

4 Translation Challenges: Some CTSA Solutions RESEARCH MATCH Free, secure, Web-based registry to improve research participation Enables investigators and volunteers to find a match Database of more than 28,000 registrants REDCap Allows users to build and manage online surveys and databases quickly and securely Is composed of 547 active institutional partners in 50 countries

5 Translation Challenges: Some CTSA Solutions RESEARCH MATCH Free, secure, Web-based registry to improve research participation Enables investigators and volunteers to find a match Database of more than 28,000 registrants REDCap Allows users to build and manage online surveys and databases quickly and securely Is composed of 547 active institutional partners in 50 countries In their new NCATS home, CTSAs will be encouraged to: Innovate translational research methods, resources, and services Develop and build upon unique institutional strengths

6 Clinical Research Challenges Takes too long Costs too much Success rate too low Process too bureaucratic

7 Clinical Research Challenges: Some CTSA Solutions Establishing regional consortia example, UC BRAID Sites defer to each others IRBs Negotiate common master clinical trials agreement Share EMR data allowing identification of potential trial participants Slide from Clay Johnston, UCSF

8 BigData Challenges Recent explosion of biomedical data Genome sequence data Public health databases Challenge: find new ways to make the most of this data, to Speed discovery and innovation Improve nation s health and economy

9 BigData Challenges: Some CTSA Solutions Convening leaders to share and discuss the best technologies and practices Informatics for Integrating Biology and the Bedside (i2b2) Developing a scalable informatics framework for bridging clinical research data and the vast data banks arising from basic science research Consists of: Infrastructure for security, access rights, and managing the data repository Tools for mining the data and asking questions

10 Reproducibility Challenges Dissemination of research findings is critical and accuracy is imperative Struggle to find balance between timely release of research results to allow for further scrutiny and delaying publication until they are thoroughly validated Academics gravitate towards early release and vetting through publications Industry hold research findings as confidential for IP Opportunity costs associated with time and money are considerable For every drug that reaches the market, nearly 10,000,000 compounds are screened and nearly $2B is spent

11 Reproducibility Challenges Some CTSA Solutions Features information on biostatistics, ethics, research design, data management, grants and recruitment Users can search for statistical tools, download software, and learn more about reproducible research Identify challenges and solutions to reproducibility Create educational materials on reproducible research methodology Develop technical solutions for data manipulation, analysis, and reporting Present short courses and materials on how to implement reproducible research practices

12 Workforce Challenges Too long to become independent Discouraging budget climate Lack of diversity Need for interdisciplinary capabilities

13 Workforce Challenges: Some CTSA Solutions Training is essential to the CTSA program

14 Enhancing the Success of the CTSAs How the IOM Can Help ALIGNMENT How can NCATS help the CTSAs experiment with innovative new approaches to the process of translation, especially in light of current fiscal constraints? SCOPE The CTSA mission includes support for the full spectrum of translational research. Is this mission best achieved by: Homogeneity, where each site supports all stages of translation? Heterogeneity, where each site capitalizes on its unique strengths? Groups of CTSAs specializing in discrete areas? ROLE What is the distinct contribution of the CTSAs to the broader clinical research enterprise?

15 Final Thoughts 1 steering committee 5 strategic goal committees 14 key function committees (~12 mtgs/yr, ~60 voting members = over 10,080 person hours!) Numerous subcommittees and working groups A committee is a cul-de-sac down which ideas are lured and then quietly strangled. ~Sir Barnett Cocks