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 NIH Advisor y Committe e M a ria Fre ire , Ph. D. , C ha ir to the NIH Dir e c tor

ACD- NCAT S: Me mbe r ship - 2 - MARIA F RE IRE , PhD (Chair ) HE L E N HOBBS, MD Pr Pr e side nt Albe r e side nt, Albe r t and Mar t and Mar y L y L aske r aske r F F oundation oundation Dir Dir e c tor e c tor of the Mc De r of the Mc De r mott Ce nte r Unive r mott Ce nte r, Unive r sity of T sity of T e xas e xas at Southwe ste r n JUL IAN ADAMS, PhD Pr e side nt of Re se ar c h and De ve lopme nt, Infinity ROBE RT L ANGE R, Sc D amc e utic als, Inc . Phar David H. Koc h Institute Pr ofe ssor , Massac huse tts Institute of T Institute of T e c hnology e c hnology L E E BABISS, PhD E xe c utive Vic e Pr e side nt of Global L abor ator y ST E L IOS PAPADOPOUL OS, PhD Se r vic e s, PPD, Inc Dir e c tor and Chair man of the Boar d, E xe lixis BROOK BYE RS, MBA , MARY PE NDE RGAST , JD Se nior Par tne r , Kle ine r Pe r kins Caufie ld & Bye r s Pr e side nt, Pe nde r gast Consulting 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 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 GlaxoSmithKline Chanc e llor , Unive r sity of Califor nia- San F r anc isc o MARC T E SSIE R- L AVIGNE , PhD DAVID GINSBURG, MD Pr e side nt, Roc ke fe lle r Unive r sity Jame s V. Ne e l Distinguishe d Unive r sity Pr ofe ssor of I t Inte r nal Me dic ine and Human Ge ne tic s, Unive r l M di i d H G ti U i sity it DAVID VAL L E , MD of Mic higan Pr ofe ssor and Dir e c tor of the Institute of Ge ne tic Me dic ine , Johns Hopkins Unive r sity Sc hool of VICT ORIA HAL E , PhD Me dic ine Chie f E xe c utive Offic e r , Me dic ine s360

ACD- NCAT S: Char ge - 3 - 1 1. Ide ntify ar Ide ntify ar e as in whic h NIH c an c ontr e as in whic h NIH c an c ontr ibute to 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 ogr ams, the author author itie s unde r itie s unde r the Cur the Cur e s Ac c e le r e s Ac c e le r ation Ne twor ation Ne twor k k (CAN), and the vast c apabilitie s of par tne r s; 3. Pr opose ne w mode ls for p how NCAT S c ould build 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 ac hie ve its mission of ac c e le r ating tr ating tr anslational anslational r e se ar c h;

ACD- NCAT S: Char ge (c ont.) - 4 - 4 4. Re c omme nd the subse t of sc ie ntific and te c hnic al 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 oc e ss that NCAT S should addr e ss; and 6. Sugge st a fr gg ame wor k for me tr ic s and time line s by y whic h suc c e ss of NCAT S c an be me asur e d.

ACD- NCAT S: Pr oc e ss and De live r able s - 5 -  Hold de libe r  Hold de libe r ations at le ast on a quar ations at le ast on a quar te r te r ly basis with the 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 of the Wor king Gr oup will pr e se nt Wor king Gr oup 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 p y Counc il for 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 - 6 -  F  F e br e br uar uar y 4 2011 y 4, 2011 – r ge (he ld in ge (he ld in r e c e ipt of c har e c e ipt of c har c onjunc tion with the NIH Institute and Ce nte r Dir e c tor s Wor king Gr oup on NCAT S)  May 24, 2011 – e xisting hur dle s and ar e as ame nable to r e e ngine e r g ing g  July 15, 2011 – pr oje c t manage me nt and c r oss- se c tor par par tne r tne r ships (inc lude d two e xpe r ships (inc lude d two e xpe r t pane ls) t pane ls)  Se pte mbe r 14, 2011 – r e vie w of findings

ACD- NCAT S: E xpe r t Pane lists - 7 - Je ffr Je ffr e y Blue stone , Ph.D. e y Blue stone , Ph.D. Be th Se ide nbe r Be th Se ide nbe r g, M.D. g, M.D. E xe c utive Vic e Chanc e llor and Pr ovost Par tne r Unive r sity of Califor nia, San F r anc isc o Kle ine r Pe r kins Caufie ld & Bye r s Je nnife r Cook, M.S., M.B.A. De e pak Sr ivastava, M.D. Se nior Se nior Vic e Pr Vic e Pr e side nt Ge ne nte c h Immunology e side nt, Ge ne nte c h Immunology T T he Younge r he Younge r F F amily Dir amily Dir e c tor e c tor Gladstone Institute , Gladstone Institute and Ophthalmology of Car diovasc ular Dise ase ; Ge ne nte c h Pr ofe 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 ofe ssor John Mc Ke w, Ph.D. in Pe diatr in Pe diatr ic De ve lopme ntal Car ic De ve lopme ntal Car diology diology Chie f, T Chi f T he r h ape utic De ve lopme nt Br ti D l t B anc h; and h d Unive r sity of Califor nia, San F r anc isc o Dir e c tor of Che mistr y NIH Ce nte r for T r anslational T he r ape utic s L e wis T . “Rusty” Williams, M.D., Ph.D. National Institute s of He alth E xe c utive Chair man, F ounde r , Pr e side nt, and CE O F F i ive Pr P i ime Jac k D. Ne wman, Ph.D. Co- F ounde r and Chie f Sc ie ntific Offic e r Paul G. Yoc k, M.D. Amyr is Mar tha Me ie r We iland Pr ofe ssor of Me dic ine ; and T or be n Str aight Nisse n, M.Sc ., Ph.D. Dir e c tor , Biode sign Managing Dir e c tor ; and Stanfor d Unive r sity He ad of Por tfolio Manage me nt and De ve lopme nt Str ate gy Pfize r Inc .

F INDINGS R Re vo lutio nize Tra nsla tio n l ti i T l ti NCAT S c an be a le ade r in r e e ngine e r ing the tr tr anslational pr anslational pr oc e ss by ide ntifying and oc e ss by ide ntifying and ove r c oming hur dle s that de c r e ase the pr p obability of suc c e ss. y

Re volutionize the Pr oc e ss of T r anslation - 9 - Cr Cr itic al ne e d to c atalyze e nable and imple me nt itic al ne e d to c atalyze , e nable , and imple me nt gr ound- br e aking advanc e s in tr anslational sc ie nc e s. NIH posse ss r NIH posse ss r ic h e xpe r ic h e xpe r tise and has signific antly 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 and ac r oss se c tor oss se c tor s, NCAT s, NCAT S c an fill gaps in S c an fill gaps in our unde r standing of the tr anslational pr oc e ss.

Goals for NCAT S - 10 -  Catalyze tr  Catalyze tr anslation by pr anslation by pr omoting innovative omoting innovative r e se ar c h  Suppor  Suppor t and e nable high r t and e nable high- r isk high r isk, high- r e war e war d pr d pr oje c ts oje c ts that e xpe r ime nt with nove l and innovative str ate gie s.  Se r Se r ve as an inc ubator ve as an inc ubator spac e for spac e for innovative sc ie nc e 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.) - 11 -  Galvanize and suppor  Galvanize and suppor t ne w par t ne w par tne r tne r ships ships  Utilize c onve ning powe r to stimulate c ommunic ation and pr and pr omote par omote par tne r tne r ships ac r ships ac r oss se c tor oss se c tor s to addr s to addr e ss e ss bar r ie r s and pr ovide solutions.  Pr  Pr omote c r omote c r oss- se c tor oss- se c tor par par tne r tne r ships by le ve r ships by le ve r aging aging funds and pr oviding ac c e ss to tools, te c hnologie s, and innovative platfor ms.

Goals for NCAT S (c ont.) - 12 -  Suppor  Suppor t and augme nt the disc ipline of r t and augme nt the disc ipline of r e gulator e gulator y y sc ie nc e and its applic ation  Collabor  Collabor ate with F ate with F DA to de sign/ unde r DA to de sign/ unde r take studie s take studie s infor ming r e gulator y appr oval pr oc e sse s.  He lp de ve lop r He lp de ve lop r e gulator e gulator y pathways inc or y pathways inc or por por ating 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 oss se c tor s to de ve lop a mor e nimble r e gulator y pr oc e ss.