Garrett fitzgerald - Clinical and Translational Research in the USA - 2009
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The document discusses challenges and responses in clinical and translational research in the US. It notes that basic, translational, and clinical research are interdependent and need to be integrated. It highlights issues like the need for improved human capital and infrastructure to support this research continuum. The creation of the Penn Institute for Translational Medicine and Therapeutics (ITMAT) is presented as addressing some of these challenges through initiatives like educational programs, funding, and cores for various research resources. Sustained funding across this full spectrum of research is argued to be crucial for improving health outcomes.
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Garrett fitzgerald - Clinical and Translational Research in the USA - 2009
- 2. Basic and Clinical Research are Interdependent Translation from basic science to human studies Translation of new knowledge into clinical practice Basic Research Clinical Research Improved Health Adapted from Sung et al. (2003) JAMA, 289, 1278-89. Copyright © (2003) American Medical Association. All Rights reserved.
- 3. Financial Times Apr. 11th 2009 Merger: A Response to Crisis Short Term Response - Merger
- 5. Fixing the Problem • Addressing the deficit in human capital with interdisciplinary transla,onal skill sets per,nent to drug ac,on • Enlarging the precompe,,ve space by addressing outmoded and unrealis,c expecta,ons based on IP • Crea,ng the informa,cs substrate for secure, compliant, heterogeneous data sharing across sectors and countries
- 6. An Appeal to Reason “If we don’t change the business models, we’re not going to survive as an industry”. Richard Clarke, Merck CEO Financial Times, Oct 22nd 2008
- 7. Research Continuum: BASIC TRANSLATIONAL CLINICAL POPULATION OUTCOMES Basic Science Departments CCEB LDI Disease-based Centers and Institutes Clinical Departments Basic Science Centers/Institutes ITMAT Creation of the Penn Institute for Translational Medicine and Therapeutics (ITMAT)
- 8. MT WY ID WA OR NV UT CA AZ ND SD NE CO NM TX OK KS AR LA MO IA MN WI IL IN KY TN MS AL GA FL SC VA WV MI NY PA MD DE NJ CT RI MA ME VT NH AK HI Building a Na+onal CTSA Consor+um New members 2009 Members Participating Institutions NC OH
- 9. NIH Spending on Clinical Research http://officeofbudget.od.nih.gov/ui/2008/tabular%20data.pdf http://www.nih.gov/news/fundingresearchareas.htm#content Total NIH Budget FY 2008 $29.5 Billion Non-Clinical Research $19.8 Billion (67.3%) Other Clinical Research $6.1 Billion (20.6%) Clinical Trials $3.6 Billion (12.1%)
- 10. Sponsors of Biomedical Research (Basic and Clinical) Moses et al. (2005) JAMA, 294, 1333-42
- 11. Challenges to Academia 1. HUMAN CAPITAL • Disintegra,on of Transla,onal Therapeu,cs • Incen,ves poorly aligned for team science • Few incen,ves to interdisciplinary research • Time lines of promo,on and funding • Specific deficits in medicinal chemistry • Trainee/Exper,se mismatch
- 12. ITMAT Response • Diversified educa,onal offerings in clinical and transla,onal research; masters and cer,ficate programs, farm teams, workshops • Mul,ple funding ini,a,ves to foster truly interdisciplinary transla,onal research • Fostering commercializa,on and addressing IP barriers
- 14. Challenges to Academia INFRASTRUCTURE • Access to HTS and medicinal chemistry • Access to seed funding for commercial POC • Informa,cs infrastructure – data sharing and mining, sample tracking, scanning for collabora,on • Analy,cal biochemistry, kine,cs and modeling • POC in humans
- 15. ITMAT Response • Clinical and Transla,onal Research Center and Laboratory • Inves,ga,onal Drug Service and Kine,cs and Modeling Core • Co‐investment in informa,cs, proteomics, genomics, imaging and biotherapeu,cs infrastructure • Discounted access to exper,se – stats, trial design, databases • Accelerated CTRC/IRB approval
- 17. US Academic Centers and Industry Sponsored Randomized Clinical Trials • Study ini+a+on at academic centers is 49 days longer than at professional and community sites • Once up and running, academic sites are roughly 10% slower in enrollment than dedicated and professional sites • Many centers fail to enroll a single pa+ent 17 Source: Harvard Business School/ RapidTrials Consortium 2.5 01.0 PA to FSFV 82 480200 239 088 243 95th perc. mean %of Enrollment Goal Enrollment Index .89 75.8 Enrollment Rate (wk) .02.82 Screen Failure Rate 78.131.130.7 Drop Out Rate 0 83.3.26 37.3 5th perc. .9 Academic Medical Centers Summary Metrics Report Academic Medical Centers 0 2.9
- 18. Challenges to CTSA CONSORTIUM 1. Fes+na Lente – ShiLing Emphasis from Speed to Quality • Detailed phenotyping in small numbers in single Centers closely integrated with preclinical research • Much more emphasis mechanism and variability in drug response as a basis for personalized dose selec,on • Projected to scale amongst similar centers • Tradi,onal Phase 3 – some CTSAs, but mostly non CTSA environments
- 19. 2. LAUNCHING A NEW DISCIPLINE • Develop and project mechanism based quan,ta,ve biomarkers from model systems into humans. • Evoking phenotypic responses in humans to guide individualiza,on of ra,onal dose selec,on • Harness the unbiased technologies to select amongst molecules directed against a single target • An unintended consequence of nomenclature
- 21. 4. Enhance interac+on with the FDA A JPL for the FDA • Resource FDA to incent academic centers to afford exper,se in emerging science and a site to pursue ques,ons relevant to drug ac,on before and a_er approval • Foster collabora,ve and educa,onal interac,ons between of FDA and Academic scien,sts • Reward innova,on • Staggered approval and drug withdrawl FDA Science and Mission at Risk Science Board Report Nov. 2007
- 23. 5. Show me the Money • CTSA cuts compounded by ins,tu,onal retrenchment • Shi_ to consor,al ac,vi,es at expense of individual center resource • Complement with investment in basic and inves,gator ini,ated science • Sustain and expand public funding for the Transla,onal enterprise; the 17 year rule
- 24. Summary The Health and Wealth of a Na,on • The quality of clinical research depends on basic and transla,onal research. • This is par,cularly so as we move towards a more personalized approach to medicine. • Sustained funding is crucial for all these interdependent elements; it’s not a zero sum game. • Pa,ents will and should play an increasing role as partners in this process.