garrett fitzgerald - clinical and translational research in the usa - 2009
TRANSCRIPT
![Page 1: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/1.jpg)
ClinicalandTransla,onalResearchintheUSA
G.A.FitzGeraldIns,tuteforTransla,onalMedicineandTherapeu,cs
www.itmat.upenn.edu
IPPOSI SYMPOSIUM DUBLIN 2010
![Page 2: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/2.jpg)
BasicandClinicalResearchareInterdependent
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.
![Page 3: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/3.jpg)
Financial Times Apr. 11th 2009
Merger: A Response to Crisis
Short Term Response - Merger
![Page 4: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/4.jpg)
The Academic Gerontocracy
![Page 5: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/5.jpg)
FixingtheProblem
• Addressingthedeficitinhumancapitalwithinterdisciplinarytransla,onalskillsetsper,nenttodrugac,on
• Enlargingtheprecompe,,vespacebyaddressingoutmodedandunrealis,cexpecta,onsbasedonIP
• Crea,ngtheinforma,cssubstrateforsecure,compliant,heterogeneousdatasharingacrosssectorsandcountries
![Page 6: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/6.jpg)
AnAppealtoReason
“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
![Page 7: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/7.jpg)
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)
![Page 8: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/8.jpg)
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
BuildingaNa+onalCTSAConsor+um
New members 2009 Members
Participating Institutions
NC
OH
![Page 9: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/9.jpg)
NIHSpendingonClinicalResearch
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%)
![Page 10: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/10.jpg)
SponsorsofBiomedicalResearch(BasicandClinical)
Moses et al. (2005) JAMA, 294, 1333-42
![Page 11: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/11.jpg)
ChallengestoAcademia
1.HUMANCAPITAL
• Disintegra,onofTransla,onalTherapeu,cs• Incen,vespoorlyalignedforteamscience
• Fewincen,vestointerdisciplinaryresearch• Timelinesofpromo,onandfunding
• Specificdeficitsinmedicinalchemistry• Trainee/Exper,semismatch
![Page 12: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/12.jpg)
ITMATResponse
• Diversifiededuca,onalofferingsinclinicalandtransla,onalresearch;mastersandcer,ficateprograms,farmteams,workshops
• Mul,plefundingini,a,vestofostertrulyinterdisciplinarytransla,onalresearch
• Fosteringcommercializa,onandaddressingIPbarriers
![Page 13: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/13.jpg)
*Half year
*Half year
TemporaldynamicsofITMAT
![Page 14: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/14.jpg)
ChallengestoAcademia
INFRASTRUCTURE
• AccesstoHTSandmedicinalchemistry
• AccesstoseedfundingforcommercialPOC
• Informa,csinfrastructure–datasharingandmining,sampletracking,scanningforcollabora,on
• Analy,calbiochemistry,kine,csandmodeling
• POCinhumans
![Page 15: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/15.jpg)
ITMATResponse• ClinicalandTransla,onalResearchCenterandLaboratory
• Inves,ga,onalDrugServiceandKine,csandModelingCore
• Co‐investmentininforma,cs,proteomics,genomics,imagingandbiotherapeu,csinfrastructure
• Discountedaccesstoexper,se–stats,trialdesign,databases
• AcceleratedCTRC/IRBapproval
![Page 16: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/16.jpg)
Physician‐Scien,st
BasicResearch
ClinicalResearch
ResearchPlan
LC‐MS/MSCore
Developmentofunbiasedmarkers
ResearchPlan
IACUC
SampleAnalysis
inmice
inhumans
BiomedicalLab
AnimalFacili,es
ExternalScien,ficAdviserandIndustry
Bionutri,onalResearchCore
ClinicalTransla,onalResearchCenter
BiochemistryCoreLabResearchNurseCore
Biosta,s,csCore
Ins,tu,onalReviewBoard
CTRCAdvisoryCouncil
Administra,on/OfficeofHumanResearch
ITMATClinicalTrialsUnit
FoodandDrugAdministra,on(IND)
Bioinforma,csCoreCRCUDataManagement
CenterforClinicalEpidemiologyandBiosta,s,cs
Inves,ga,onalDrugService
Post‐Doc
Kine,csModelingandSimula,on
![Page 17: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/17.jpg)
USAcademicCentersandIndustrySponsoredRandomizedClinicalTrials
• Studyini+a+onatacademiccentersis49dayslongerthanatprofessionalandcommunitysites
• Onceupandrunning,academicsitesareroughly10%slowerinenrollmentthandedicatedandprofessionalsites
• Manycentersfailtoenrollasinglepa+ent
17 Source: Harvard Business School/
RapidTrials Consortium
2.5 0 1.0
PA to FSFV 82 480 200
239 0 88
243
95th perc. mean
%of Enrollment Goal
Enrollment Index .89
75.8
Enrollment Rate (wk) .02 .82
Screen Failure Rate 78.1 31.1 30.7
Drop Out Rate 0 83.3 .26 37.3
5th perc.
.9
Academic Medical Centers Summary Metrics Report
Academic Medical Centers
0
2.9
![Page 18: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/18.jpg)
ChallengestoCTSACONSORTIUM1.Fes+naLente–ShiLingEmphasisfromSpeedto
Quality
• DetailedphenotypinginsmallnumbersinsingleCenterscloselyintegratedwithpreclinicalresearch
• Muchmoreemphasismechanismandvariabilityindrugresponseasabasisforpersonalizeddoseselec,on
• Projectedtoscaleamongstsimilarcenters• Tradi,onalPhase3–someCTSAs,butmostlynonCTSAenvironments
![Page 19: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/19.jpg)
2.LAUNCHINGANEWDISCIPLINE
• Developandprojectmechanismbasedquan,ta,vebiomarkersfrommodelsystemsintohumans.
• Evokingphenotypicresponsesinhumanstoguideindividualiza,onofra,onaldoseselec,on
• Harnesstheunbiasedtechnologiestoselectamongstmoleculesdirectedagainstasingletarget
• Anunintendedconsequenceofnomenclature
![Page 20: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/20.jpg)
3.ExpandthePrecompe++veSpace
• ExampleoftheNotforProfitSector
• FDA:createasafehavenforSystemsPharmacologyandPhysiology
![Page 21: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/21.jpg)
4.Enhanceinterac+onwiththeFDAAJPLfortheFDA
• ResourceFDAtoincentacademiccenterstoaffordexper,seinemergingscienceandasitetopursueques,onsrelevanttodrugac,onbeforeanda_erapproval
• Fostercollabora,veandeduca,onalinterac,onsbetweenofFDAandAcademicscien,sts
• Rewardinnova,on• Staggeredapprovalanddrugwithdrawl
FDA Science and Mission at Risk Science Board Report Nov. 2007
![Page 22: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/22.jpg)
It’sallabouttheMoney
![Page 23: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/23.jpg)
5.ShowmetheMoney
• CTSAcutscompoundedbyins,tu,onalretrenchment
• Shi_toconsor,alac,vi,esatexpenseofindividualcenterresource
• Complementwithinvestmentinbasicandinves,gatorini,atedscience
• SustainandexpandpublicfundingfortheTransla,onalenterprise;the17yearrule
![Page 24: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/24.jpg)
SummaryTheHealthandWealthofaNa,on• Thequalityofclinicalresearchdependsonbasicandtransla,onalresearch.
• Thisispar,cularlysoaswemovetowardsamorepersonalizedapproachtomedicine.
• Sustainedfundingiscrucialforalltheseinterdependentelements;it’snotazerosumgame.
• Pa,entswillandshouldplayanincreasingroleaspartnersinthisprocess.
![Page 25: Garrett fitzgerald - Clinical and Translational Research in the USA - 2009](https://reader033.vdocuments.us/reader033/viewer/2022042907/5886f8361a28ab4e3a8b4e49/html5/thumbnails/25.jpg)
ProgressinTransla,onalMedicineandTherapeu,cs