,,ii #aaf? ,,congress #b;d ,session ,s4 #dhfg ,,9 ,,! ,,s5ate ,,( ,,! ,,unit$ ,,/ates ,octob] #bf 7legislative day1 ,octob] #ai71 #bjbj,mr4 ,coons 7= himself & ,ms4 ,murk[ski7 introduc$ ! foll[+ bill2 :i* 0 read twice & ref]r$ to ! ,committee on ,heal?1 ,education1 ,labor1 & ,p5sions ,a ,,bill ,to direct ! ,secret>y ( ,heal? & ,human ,s]vices to support rese>* on1 & exp&$ access to1 inve/igational drugs = amyotrophic lat]al scl]osis1 & = o!r purposes4 #a4 ,%ort title ,?is ,act may be cit$ z ! ,accel]at+ ,access to ,critical ,!rapies = ,,als ,act4 #b4 ,grants = rese>* on & exp&$ access to exp]im5tal !rapies = ,,als 7a7 ,9 ,g5]al ,! ,secret>y ( ,heal? & ,human ,s]vices %all aw>d grants to eligible entities = purposes ( support+ rese>* on1 & exp&$ access = individuals to1 inve/igational drugs = ! prev5tion1 diagnosis1 mitigation1 treatm5t1 or cure ( amyotrophic lat]al scl]osis pursuant to an exp&$ access reque/ submitt$1 & all[$ to proce$ by ! ,secret>y1 "u section #efa ( ! ,f$]al ,food1 ,drug1 & ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb74 7b7 ,application ,an eligible entity seek+ a grant "u ?is section %all submit to ! ,secret>y an application at su* "t1 9 su* mann]1 & conta9+ su* in=mation z ! ,secret>y %all specify4 ,su* application %all specify a rese>* objective relat+ to exp&+ access to inve/igational drugs 7z describ$ 9 subsection 7a77 t w\ld be support$ by ! aw>d ( su* grant4 7c7 ,selection ,n lat] ?an #ij days aft] ! date ( submission ( an application = a grant "u ?is section1 ! ,secret>y %all det]m9e :e!r to aw>d ! grant1 tak+ into consid]ation :e!r aw>d+ su* grant w support a rese>* objective relat+ to exp&+ access to inve/igational drugs 7z describ$ 9 subsection 7a771 consi/5t ) ! mission ( ! ,national ,in/itutes ( ,heal?4 7d7 ,use ( funds ,an eligible entity may use funds receiv$ ?r\gh ! grant-- 7#a7 to pay ! manufactur] or sponsor = ! direct co/s ( su* drug 7z au?oriz$ "u section #cab4h7;d7 ( title #ba1 ,code ( ,f$]al ,regulations 7or successor regulations771 if su* co/s are ju/ifi$ z "p ( pe] review ( ! grant2 7#b7 = ! entity's direct co/s incurr$ 9 provid+ su* drug consi/5t ) ! rese>* mission ( ! grant2 or 7#c7 = ! direct & indirect co/s ( ! entity 9 conduct+ rese>* ) respect to ! drug involv$4 7e7 ,def9itions ,9 ?is section3 7#a7 ,! t]m eligible entity means a p>ticipat+ cl9ical trial site or sites sponsor$ by a small bus9ess conc]n 7z def9$ 9 section #c7;a7 ( ! ,small ,bus9ess ,act 7#ae ,u4,s4,c4 #fcb7;a777 t is ! sponsor ( a drug t is ! subject ( an inve/igational new drug application "u section #eje7;i7 ( ! ,f$]al ,food1 ,drug1 & ,cosmetic ,act 7#ba ,u4,s4,c4 #cee7;i774 7#b7 ,! t]m p>ticipat+ cl9ical trial means a phase #c cl9ical trial conduct$ pursuant to an exemption "u section #eje7;i7 ( ! ,f$]al ,food1 ,drug1 & ,cosmetic ,act 7#ba ,u4,s4,c4 #cee7;i77 or section #cea7;a7 ( ! ,public ,heal? ,s]vice ,act 7#db ,u4,s4,c4 #bfb7;a77 to inve/igate a drug int5d$ to prev5t1 diagnose1 mitigate1 treat1 or cure amyotrophic lat]al scl]osis4 7#c7 ,! t]m p>ticipat+ cl9ical trial site means a nonpr(it or public heal? c>e facility1 or network ( facilities1 at :i* pati5ts p>ticipat+ 9 a p>ticipat+ cl9ical trial receive an inve/igational drug ?r\gh su* trial4 #c4 ,,hhs ,collaborative = ,neurodeg5]ative ,diseases 7a7 ,e/abli%m5t ,n lat] ?an "o ye> aft] ! date ( ! enactm5t ( ?is ,act1 ! ,secret>y ( ,heal? & ,human ,s]vices %all e/abli% & implem5t a ,collaborative = ,neurodeg5]ative ,diseases betwe5 ! ,national ,in/itutes ( ,heal? & ! ,food & ,drug ,adm9i/ration 7to be kn[n & ref]r$ to 9 ?is section z ! ,collaborative71 :i* %all-- 7#a7 ent] into a coop]ative agreem5t1 contract1 or o!r in/rum5t ) a private entity or entities "u :i* su* private entity or entities w op]ate ! ,collaborative2 7#b7 focus on advanc+ regulatory improvem5ts & sci5tific rese>* t w support & accel]ate ! developm5t & approval ( drugs = pati5ts ) amyotrophic lat]al scl]osis2 7#c7 fo/] ! developm5t ( effective drugs t improve ! lives ( p t suff] f r>e neurodeg5]ative diseases2 & 7#d7 %>e in=mation ) ! ,secret>y to advance ! purposes specifi$ 9 p>agraph 7#c71 su* z ?r\gh c>ry+ out ! grant programs "u sections #b & #e & develop+ ! action plan "u section #d4 7b7 ,gifts 7#a7 ,9 g5]al ,! ,collaborative may solicit & accept gifts1 grants1 & o!r donations1 e/abli% acc\nts1 & inve/ & exp5d funds 9 support ( pre-competitive rese>* & rese>* associat$ ) phase #c & phase #d cl9ical trials conduct$ ) respect to inve/igational drugs t are ! subjects ( exp&$ access applications "u section #efa ( ! ,f$]al ,food1 ,drug1 & ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb74 7#b7 ,use ,9 addition to any am\nts appropriat$ = purposes ( c>ry+ out ?is section1 ! ,collaborative may use1 )\t fur!r appropriation1 any funds d]iv$ f a gift1 grant1 or o!r donation accept$ pursuant to p>agraph 7#a74 7c7 ,advisory panel 7#a7 ,9 g5]al ,! ,collaborative %all conv5e an advisory panel to conduct a review ( ! design & implem5tation ( ! programs au?oriz$ by ?is ,act4 ,su* panel %all include repres5tatives ( pati5ts1 treat+ physicians1 national organizations t facilitate provision ( c>e s]vices1 rese>*]s1 drug sponsors1 drug manufactur]s1 & ,f$]al ag5cies4 7#b7 ,report ,! advisory panel conv5$ "u p>agraph 7#a7 %all1 n lat] ?an #ah mon?s aft] ! date ( ! enactm5t ( ?is ,act1 submit to ! ,committee on ,en]gy & ,comm]ce ( ! ,h\se ( ,repres5tatives & ! ,committee on ,heal?1 ,education1 ,labor1 & ,p5sions ( ! ,s5ate a report t conta9s-- 7,a7 ! f9d+s & conclusions ( ! review conduct$ "u p>agraph 7#a72 & 7,b7 recomm5dations = c>ry+ out ! programs "u ?is ,act dur+ ! #b-ye> p]iod foll[+ ! submission ( su* report1 includ+ recomm5dations relat+ to ! inclusion ( additional neurodeg5]ative diseases or disease areas )9 ! grant programs "u sections #b & #e4 7d7 ,duties & au?orities ,! ,collaborative %all id5tify & implem5t /rategies = ! ,secret>y-- 7#a7 = purposes ( exp$it+ ! approval ( drugs to treat amyotrophic lat]al scl]osis1 includ+ ?r\gh coord9ation among ! c5t]s ( ! ,food & ,drug ,adm9i/ration to a*ieve ! goals specifi$ 9 ! draft guidance = drug sponsors entitl$ ,amyotrophic ,lat]al ,scl]osis3 ,develop+ ,drugs = ,treatm5t ,guidance = ,indu/ry publi%$ 9 ,septemb] #bjai2 7#b7 to facilitate access to inve/igational drugs = amyotrophic lat]al scl]osis pursuant to an exp&$ access reque/ "u section #efa ( ! ,f$]al ,food1 ,drug1 & ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb7 9 a simil> mann] z inve/igational drugs = canc] are provid$ ?r\gh ,project ,facilitate ( ! ,c5t] ( ,excell5ce = ,oncology2 7#c7 ) respect to ! r>e neurodeg5]ative disease grant program e/abli%$ "u section #e2 7#d7 to def9e or develop ! regulatory & translational pa?way = em]g+ !rapeutic categories2 7#e7 to %>e1 )9 ! ,collaborative1 f9d+s & insights relat$ to pre-competitive rese>* & rese>* associat$ ) phase #c & phase #d cl9ical trials conduct$ ) respect to inve/igational drugs t are ! subjects ( exp&$ access applications "u section #efa ( ! ,f$]al ,food1 ,drug1 & ,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bbb72 & 7#f7 to develop & implem5t an ongo+ me*anism to %>e fe$back & in=mation & develop /rategies ) ! neurodeg5]ative disease community1 includ+ pati5ts1 treat+ physicians1 national organizations t facilitate provision ( c>e s]vices1 access1 & rese>*1 rese>*]s1 drug sponsors1 drug manufactur]s1 & ,f$]al ag5cies4 #d4 ,neurodeg5]ative disease action plan 7a7 ,9 g5]al ,n lat] ?an #f mon?s aft] ! date ( ! enactm5t ( ?is ,act1 ! ,secret>y ( ,heal? & ,human ,s]vices %all publi% an action plan describ+ actions ! ,dep>tm5t ( ,heal? & ,human ,s]vices1 ?r\gh ! ,national ,in/itutes ( ,heal? & ,food & ,drug ,adm9i/ration1 int5d to take dur+ ! #e-ye> p]iod foll[+ publication ( ! plan ) respect to program enhancem5ts1 policy developm5t1 regulatory sci5ce initiatives1 & o!r appropriate initiatives to-- 7#a7 fo/] ! developm5t ( safe & effective drugs t improve ! lives ( p liv+ ) r>e neurodeg5]ative diseases z quickly z possible2 & 7#b7 facilitate access to inve/igational drugs4 7b7 ,cont5ts ,! action plan publi%$ "u subsection 7a7 %all-- 7#a7 id5tify appropriate repres5tation f )9 ! ,food & ,drug ,adm9i/ration & ,national ,in/itutes ( ,heal? to be responsible = implem5tation ( su* action plan2 & 7#b7 include elem5ts to facilitate-- 7,a7 int]actions & collaboration betwe5 ! ,food & ,drug ,adm9i/ration1 includ+ ! review c5t]s !re(1 & /akehold]s includ+ pati5ts1 sponsors1 & ext]nal biom$ical community2 7,b7 consid]ation ( cross-cutt+ cl9ical & regulatory policy issues1 includ+ consi/5cy ( regulatory advice & decision mak+2 7,c7 id5tification ( key regulatory sci5ce & policy issues critical to advanc+ developm5t ( safe & effective drugs2 & 7,d7 engagem5t by /aff ( ! relevant c5t]s ( ! ,food & ,drug ,adm9i/ration & o!r relevant (fices ( ! ,food & ,drug ,adm9i/ration & ,national ,in/itutes ( ,heal? ) ! designat$ lead]%ip ( ! ,collaborative4 #e4 ,r>e ,neurodeg5]ative ,disease ,grant ,program ,! ,secret>y ( ,heal? & ,human ,s]vices1 act+ ?r\gh ! ,commission] ( ,food & ,drugs1 %all c>ry out a program ( aw>d+ grants to1 & contracts ent]$ into )1 public & private entities to cov] ! co/s ( rese>* on & developm5t ( int]v5tions int5d$ to prev5t1 diagnose1 mitigate1 treat1 or cure amyotrophic lat]al scl]osis & o!r life-?reat5+ or sev]ely debilitat+ neurodeg5]ative diseases1 includ+ co/s incurr$ ) respect to ! developm5t & critical evaluation ( tools1 me?ods1 & processes-- 7#a7 to *>act]ize su* neurodeg5]ative diseases & _! natural hi/ory2 7#b7 to id5tify molecul> t>gets = su* neurodeg5]ative diseases2 & 7#c7 to increase effici5cy & productivity ( cl9ical developm5t ( !rapies1 includ+ advanc+ rational !rapeutic developm5t & work+ to e/abli% cl9ical trial networks4 #f4 ,au?orization ( appropriations ,= purposes ( c>ry+ out ?is ,act1 !re are au?oriz$ to be appropriat$ #ajj1jjj1jjj = ea* ( fiscal ye>s #bjbb ?r\gh #bjbf4