NA TIO NA L C ENTER FO R A DV A NC ING TRA NSLA TIO NA L SC IENC ES - - PowerPoint PPT Presentation

A C D W O RKIN G G RO UP A C TIV ITIES:

NA TIO NA L C ENTER FO R A DV A NC ING TRA NSLA TIO NA L SC IENC ES TRA NSLA TIO NA L SC IENC ES

M a ria Fre ire , Ph. D. , C ha ir

NIH Advisor y Committe e to the NIH Dir e c tor

ACD- NCAT S: Me mbe r ship

MARIA F RE IRE , PhD (Chair )

Pr e side nt Albe r t and Mar y L aske r F

• undation

HE L E N HOBBS, MD

Dir e c tor

• f the Mc De r

mott Ce nte r Unive r sity of T e xas

• 2 -

Pr e side nt, Albe r t and Mar y L aske r F

• undation

JUL IAN ADAMS, PhD

Pr e side nt of Re se ar c h and De ve lopme nt, Infinity Phar amc e utic als, Inc . Dir e c tor

• f the Mc De r

mott Ce nte r, Unive r sity of T e xas at Southwe ste r n

ROBE RT L ANGE R, Sc D

David H. Koc h Institute Pr

• fe ssor

, Massac huse tts Institute of T e c hnology

L E E BABISS, PhD

E xe c utive Vic e Pr e side nt of Global L abor ator y Se r vic e s, PPD, Inc

BROOK BYE RS, MBA

Institute of T e c hnology

ST E L IOS PAPADOPOUL OS, PhD

Dir e c tor and Chair man of the Boar d, E xe lixis

,

Se nior Par tne r , Kle ine r Pe r kins Caufie ld & Bye r s

WIL L IAM CHIN, MD

E xe c utive De an for Re se ar c h, Har var d Me dic al Sc hool

MARY PE NDE RGAST , JD

Pr e side nt, Pe nde r gast Consulting

MONCE F SL AOUI, PhD

Chair man of Re se ar c h and De ve lopme nt,

SUSAN DE SMOND- HE L L MANN, MD, MPH

Chanc e llor , Unive r sity of Califor nia- San F r anc isc o

DAVID GINSBURG, MD

Jame s V. Ne e l Distinguishe d Unive r sity Pr

• fe ssor
• f

I t l M di i d H G ti U i it GlaxoSmithKline

MARC T E SSIE R- L AVIGNE , PhD

Pr e side nt, Roc ke fe lle r Unive r sity Inte r nal Me dic ine and Human Ge ne tic s, Unive r sity

• f Mic higan

VICT ORIA HAL E , PhD

Chie f E xe c utive Offic e r , Me dic ine s360

DAVID VAL L E , MD

Pr

• fe ssor

and Dir e c tor

• f the Institute of Ge ne tic

Me dic ine , Johns Hopkins Unive r sity Sc hool of Me dic ine

ACD- NCAT S: Char ge

1 Ide ntify ar e as in whic h NIH c an c ontr ibute to

• 3 -

1. Ide ntify ar e as in whic h NIH c an c ontr ibute to str e amlining the r ape utic and diagnostic de ve lopme nt nationally and globally; 2. Re c omme nd possible ways in whic h NCAT S c an maximally tap the str e ngths of e xtant pr

• gr

ams, the author itie s unde r the Cur e s Ac c e le r ation Ne twor k author itie s unde r the Cur e s Ac c e le r ation Ne twor k (CAN), and the vast c apabilitie s of par tne r s; 3. Pr

• pose ne w mode ls for

how NCAT S c ould build p par tne r ships with e xte r nal e ntitie s, inc luding biote c hnology and phar mac e utic al c ompanie s, to ac hie ve its mission of ac c e le r ating tr anslational ac hie ve its mission of ac c e le r ating tr anslational r e se ar c h;

ACD- NCAT S: Char ge (c ont.)

4 Re c omme nd the subse t of sc ie ntific and te c hnic al

• 4 -

4. Re c omme nd the subse t of sc ie ntific and te c hnic al c halle nge s along the dr ug disc ove r y pipe line that NCAT S should addr e ss; 5. Re c omme nd pote ntial ar e as of tr anslational r e se ar c h that fall outside the dr ug de ve lopme nt pr

• c e ss that

NCAT S should addr e ss; and 6. Sugge st a fr ame wor k for me tr ic s and time line s by gg y whic h suc c e ss of NCAT S c an be me asur e d.

ACD- NCAT S: Pr

• c e ss and De live r

able s

• Hold de libe r

ations at le ast on a quar te r ly basis with the

• 5 -
• Hold de libe r

ations at le ast on a quar te r ly basis, with the inte nt of me e ting at le ast thr e e time s be for e Oc tobe r 1, 2011;

• T

he Chair

• f the Wor

king Gr

• up will pr

e se nt Wor king Gr

• up findings to the full ACD (pr

e liminar y findings pr e se nte d at the ACD me e ting on June 9- 10, 2011); and,

• Wor

k will be c omple te whe n the Advisor y Counc il for p y NCAT S is for mally in plac e (e xpe c te d by Oc tobe r 2011).

ACD- NCAT S: Me e tings

• F

e br uar y 4 2011 r e c e ipt of c har ge (he ld in

• 6 -
• F

e br uar y 4, 2011 – r e c e ipt of c har ge (he ld in c onjunc tion with the NIH Institute and Ce nte r Dir e c tor s Wor king Gr

• up on NCAT

S)

• May 24, 2011 – e xisting hur

dle s and ar e as ame nable to r e e ngine e r ing g g

• July 15, 2011 – pr
• je c t manage me nt and c r
• ss- se c tor

par tne r ships (inc lude d two e xpe r t pane ls) par tne r ships (inc lude d two e xpe r t pane ls)

• Se pte mbe r

14, 2011 – r e vie w of findings

ACD- NCAT S: E xpe r t Pane lists

Je ffr e y Blue stone , Ph.D. Be th Se ide nbe r g, M.D.

• 7 -

Je ffr e y Blue stone , Ph.D.

E xe c utive Vic e Chanc e llor and Pr

• vost

Unive r sity of Califor nia, San F r anc isc o

Je nnife r Cook, M.S., M.B.A.

Se nior Vic e Pr e side nt Ge ne nte c h Immunology

Be th Se ide nbe r g, M.D.

Par tne r Kle ine r Pe r kins Caufie ld & Bye r s

De e pak Sr ivastava, M.D.

T he Younge r F amily Dir e c tor Gladstone Institute Se nior Vic e Pr e side nt, Ge ne nte c h Immunology and Ophthalmology Ge ne nte c h

John Mc Ke w, Ph.D.

Chi f T h ti D l t B h d T he Younge r F amily Dir e c tor , Gladstone Institute

• f Car

diovasc ular Dise ase ; Pr

• fe ssor

, De par tme nts of Pe diatr ic s and Bioc he mistr y & Biophysic s; and Wilma and Ade line Pir ag Distinguishe d Pr

• fe ssor

in Pe diatr ic De ve lopme ntal Car diology Chie f, T he r ape utic De ve lopme nt Br anc h; and Dir e c tor

• f Che mistr

y NIH Ce nte r for T r anslational T he r ape utic s National Institute s of He alth in Pe diatr ic De ve lopme ntal Car diology Unive r sity of Califor nia, San F r anc isc o

L e wis T . “Rusty” Williams, M.D., Ph.D.

E xe c utive Chair man, F

• unde r

, Pr e side nt, and CE O F i P i

Jac k D. Ne wman, Ph.D.

Co- F

• unde r

and Chie f Sc ie ntific Offic e r Amyr is

T

• r

be n Str aight Nisse n, M.Sc ., Ph.D.

F ive Pr ime

Paul G. Yoc k, M.D.

Mar tha Me ie r We iland Pr

• fe ssor
• f Me dic ine ; and

Dir e c tor , Biode sign Managing Dir e c tor ; and He ad of Por tfolio Manage me nt and De ve lopme nt Str ate gy Pfize r Inc . Stanfor d Unive r sity

R l ti i T l ti

F INDINGS

Re vo lutio nize Tra nsla tio n

NCAT S c an be a le ade r in r e e ngine e r ing the tr anslational pr

• c e ss by ide ntifying and

tr anslational pr

• c e ss by ide ntifying and
• ve r

c oming hur dle s that de c r e ase the pr

• bability of suc c e ss.

p y

Re volutionize the Pr

• c e ss of T

r anslation

Cr itic al ne e d to c atalyze e nable and imple me nt

• 9 -

Cr itic al ne e d to c atalyze , e nable , and imple me nt gr

• und- br

e aking advanc e s in tr anslational sc ie nc e s. NIH posse ss r ic h e xpe r tise and has signific antly NIH posse ss r ic h e xpe r tise and has signific antly advanc e d our unde r standing of human biology.

By c omple me nting e xisting e ffor ts at NIH and ac r

• ss se c tor

s, NCAT S c an fill gaps in and ac r

• ss se c tor

s, NCAT S c an fill gaps in

• ur

unde r standing of the tr anslational pr

• c e ss.

Goals for NCAT S

• Catalyze tr

anslation by pr

• moting innovative
• 10 -
• Catalyze tr

anslation by pr

• moting innovative

r e se ar c h

• Suppor

t and e nable high r isk high r e war d pr

• je c ts
• Suppor

t and e nable high- r isk, high- r e war d pr

• je c ts

that e xpe r ime nt with nove l and innovative str ate gie s.

• Se r

ve as an inc ubator spac e for innovative sc ie nc e Se r ve as an inc ubator spac e for innovative sc ie nc e that is too r isky or too e ar ly in de ve lopme nt for c omme r c ial inve stme nt.

• Re c r

uit dive r se e xpe r tise to addr e ss bar r ie r s and gaps.

Goals for NCAT S (cont.)

• Galvanize and suppor

t ne w par tne r ships

• 11 -
• Galvanize and suppor

t ne w par tne r ships

• Utilize c onve ning powe r

to stimulate c ommunic ation and pr

• mote par

tne r ships ac r

• ss se c tor

s to addr e ss and pr

• mote par

tne r ships ac r

• ss se c tor

s to addr e ss bar r ie r s and pr

• vide solutions.
• Pr
• mote c r
• ss- se c tor

par tne r ships by le ve r aging

• Pr
• mote c r
• ss- se c tor

par tne r ships by le ve r aging funds and pr

• viding ac c e ss to tools, te c hnologie s,

and innovative platfor ms.

Goals for NCAT S (c ont.)

• Suppor

t and augme nt the disc ipline of r e gulator y

• 12 -
• Suppor

t and augme nt the disc ipline of r e gulator y sc ie nc e and its applic ation

• Collabor

ate with F DA to de sign/ unde r take studie s

• Collabor

ate with F DA to de sign/ unde r take studie s infor ming r e gulator y appr

• val pr
• c e sse s.
• He lp de ve lop r

e gulator y pathways inc or por ating He lp de ve lop r e gulator y pathways inc or por ating innovative de signs.

• Str

e ngthe n c ommunic ation be twe e n r e gulator y age nc ie s and r e se ar c he r s ac r

• ss se c tor

s to de ve lop a mor e nimble r e gulator y pr

• c e ss.

Goals for NCAT S (c ont.)

• E

xpand the pr e c ompe titive spac e

• 13 -
• E

xpand the pr e c ompe titive spac e

• Pr
• mote and fac ilitate ope n e xc hange of infor

mation and le ssons le ar ne d and le ssons le ar ne d.

• E

nc our age gr ante e s to submit data r e gar ding failur e s.

• E

nc our age the c olle c tion of infor mation on failur e s, and analyze data to c aptur e le ssons le ar ne d.

• E

stablish an ope n- ac c e ss r e positor y for c olle c ting infor mation.

Goals for NCAT S (c ont.)

• Har

ne ss the powe r

• f the CT

SA Pr

• gr

am

• 14 -
• Har

ne ss the powe r

• f the CT

SA Pr

• gr

am

• Capitalize on the c apabilitie s of the CT

SA pr

• gr

am.

• Affor

d individual CT SAs fle xibility to c ultivate unique str e ngths.

• Pr
• vide inc e ntive s for

for ming a str

• ng national CT

SA c onsor tium.

• De ve lop pr
• gr

ams to tr ain c linic ians in tr anslational sc ie nc e .

Goals for NCAT S (c ont.)

• T

r ansfor mation thr

• ugh tr

aining

• 15 -
• T

r ansfor mation thr

• ugh tr

aining

• Catalyze e duc ational pr
• gr

ams in tr anslational sc ie nc e s e spe c ially in unde r- r e pr e se nte d fie lds sc ie nc e s, e spe c ially in unde r- r e pr e se nte d fie lds.

• Pr
• mote nove l tr

aining me c hanisms suc h as a dr ug de ve lopme nt appr e ntic e ship for e ar ly- stage de ve lopme nt appr e ntic e ship for e ar ly- stage inve stigator s.

• Pr
• vide inc e ntive s for

physic ian sc ie ntists to se e k c r

• ss-
• Pr
• vide inc e ntive s for

physic ian sc ie ntists to se e k c r

• ss-

tr aining in human biology and ne w dr ug, diagnostic , and de vic e disc ove r y; e xplor e c r

• ss- tr

aining of physic ians and sc ie ntists be twe e n and among industr y physic ians and sc ie ntists be twe e n and among industr y, ac ade mia, and gove r nme nt labs.

Goals for NCAT S (c ont.)

• Str

e amline Administr ative Pr

• c e sse s
• 16 -
• Str

e amline Administr ative Pr

• c e sse s
• Ide ntify and ove r

c ome r

• adbloc ks that hinde r

the ability to r apidly and e ffe c tive ly fund manage ability to r apidly and e ffe c tive ly fund, manage , and te r minate pr

• je c ts.

E stablish c le ar pathways for de ve loping

• E

stablish c le ar pathways for de ve loping agr e e me nts be twe e n NIH and othe r se c tor s to allow for pr

• mpt tur

nar

• und and time ly funding.

F INDINGS

P i t t C id Po ints to C o nsid e r

NCAT S should foc us its e ffor ts on ar e as of r e se ar c h that br

• adly and pr
• foundly impac t

r e se ar c h that br

• adly and pr
• foundly impac t

the e ntir e tr anslational disc ipline .

Points to Conside r

• 18 -
• NCAT

S should not duplic ate tr anslational r e se ar c h e ffor ts

• NCAT

S should not duplic ate tr anslational r e se ar c h e ffor ts e xisting in and/ or funde d by NIH Institute s and Ce nte r s or alr e ady unde r way thr

• ughout industr

y.

• NCAT

S should le ve r age its infr astr uc tur e to for m str ate gic c ollabor ations with dive r se se c tor s to minimize sc ie ntific and administr ative r e dundanc y and administr ative r e dundanc y.

• Pr
• je c ts should be suppor

te d only until the y attr ac t c omme r c ial inve stme nt and not ac r

• ss the e ntir

e pipe line c omme r c ial inve stme nt and not ac r

• ss the e ntir

e pipe line .

• NCAT

S le ade r ship will be r e sponsible for pr ior itizing goals and ac tivitie s and ac tivitie s.

Ar e as Ame nable to Re e ngine e r ing - E xample s

• T

ar ge t Validation

• 19 -
• T
• xic ology
• T

ar ge t Validation

• Phar

mac ology

• T
• xic ology
• Biomar

ke r s gy

• Imaging
• Ne w Use s for

E stablishe d

• De vic e s and

Diagnostic s E stablishe d Compounds g

• Che mic al Spac e
• Imple me ntation

Sc ie nc e

E i S

F INDINGS

Ensuring Suc c e ss

NCAT S le ade r ship – its staff and its gove r ning body – will play a de fining r

• le in the ne w

body will play a de fining r

• le in the ne w

Ce nte r ’s suc c e ss.

Qualitie s of E ffe c tive L e ade r ship Gove r nanc e and Staff

• NCAT

S Dir e c tor

• 21 -

L e ade r ship, Gove r nanc e , and Staff

• NCAT

S Dir e c tor

• Has a unique ble nd of e xpe r

tise and e xpe r ie nc e that tr ansc e nds a singe fie ld or disc ipline . g p

• Has e xpe r

ie nc e in both ac ade mia and the pr ivate se c tor .

• Can ide ntify and ove r

c ome hur dle s.

• De monstr

ate s politic al se nsitivity whe n wor king with di t k h ld d tit i dive r se stake holde r s and c onstitue nc ie s.

• Can c onve ne multiple par

tne r s wor king towar ds c ommon goals. c ommon goals.

• A visionar

y willing to e ngage in “disr uptive innovation.”

Qualitie s of E ffe c tive L e ade r ship Gove r nanc e and Staff (

t )

• Re se ar

c h Staff

• 22 -

L e ade r ship, Gove r nanc e , and Staff (cont.)

• Re se ar

c h Staff

• Innovative thinke r

s, te c hnic ally sophistic ate d, and able to wor k inde pe nde ntly and c ollabor ative ly in te ams. p y y

• Pr

imar y r e sponsibility should be foc use d on te am pr

• je c ts and not daily administr

ation of NCAT S.

• Advisor

y Boar d

• Compose d of stake holde r

r e pr e se ntative s with Compose d of stake holde r r e pr e se ntative s with e xpe r tise in se c tor s of r e le vanc e to the NCAT S mission.

M i F d

CONCL UDING RE MARKS

M o ving Fo rwa rd

With visionar y le ade r ship, Age nc y suppor t, and in c ollabor ation with the NIH Institute s and in c ollabor ation with the NIH Institute s and Ce nte r s, the F DA, and industr y, NCAT S c an tr ansfor m the way we de ve lop ne w me dic ine s, y p , diagnostic s, and de vic e s.

Q UESTIO NS A ND DISC USSIO N

M a ria Fre ire , Ph. D. , C ha ir

NIH Advisor y Committe e to the NIH Dir e c tor