,,ii #aah? ,,congress #a/ ,session ,s4 #aijf ,,9 ,,! ,,s5ate ,,( ,,! ,,unit$ ,,/ates ,june #h1 #bjbc,mr4 ,braun 7= himself1 ,mrs4 ,gillibr&1 ,mr4 ,wick]1 ,mr4 ,cram]1 & ,ms4 ,murk[ski7 introduc$ ! foll[+ bill2 :i* 0 read twice & ref]r$ to ! ,committee on ,heal?1 ,education1 ,labor1 & ,p5sions ,a ,,bill ,to am5d ! ,f$]al ,food1 ,drug1 & ,cosmetic ,act to e/abli% a "t-limit$ provisional approval pa?way1 subject to specific obligations1 = c]ta9 drugs & biological products1 & = o!r purposes4 #a4 ,%ort title ,?is ,act may be cit$ z ! ,promis+ ,pa?way ,act4 #b4 ,provisional approval ( new human drugs 7a7 ,9 g5]al ,sub*apt] ,a ( *apt] ,v ( ! ,f$]al ,food1 ,drug1 & ,cosmetic ,act 7#ba ,u4,s4,c4 #cea et seq47 is am5d$ by add+ at ! end ( ! foll[+3 #ebd;,c4 ,provisional approval ( new human drugs 7a7 ,priority review & evaluation ( applications 7#a7 ,9 g5]al ,! ,secret>y %all e/abli% a priority review sy/em to evaluate applications submitt$ "u ?is section = provisional approval )9 #ij days ( receipt ( a complet$ application4 7#b7 ,o!r designations ,if a drug submitt$ = review "u ?is section is eligible = a special designation by ! ,secret>y "u ?is ,act1 includ+ z a drug = a r>e disease or condition "u section #ebf1 all b5efits ( su* o!r designation %all be available = use "u provisional approval1 includ+ any tax cr$its & waiv+ ( fees "u *apt] ,,vii4 7b7 ,eligibility ,a drug may be eligible = provisional approval "u ?is section if ! ,secret>y det]m9es t ! drug is int5d$ = ! treatm5t1 prev5tion1 or m$ical diagnosis ( a s]i\s or life-?reat5+ disease or condition = :i* !re is a reasonable likelihood t premature dea? w occur )\t e>ly m$ical int]v5tion = an individual contract+ or be+ diagnos$ ) su* disease or condition4 7c7 ,/&>d ( review = provisional approval 7#a7 ,requirem5ts ,an application = provisional approval "u ?is section may be approv$ only if ! ,secret>y det]m9es t-- 7,a7 !re is sub/antial evid5ce ( safety = ! drug1 su* t !re is evid5ce consi/+ ( adequate & well-controll$ inve/igations1 includ+ cl9ical inve/igations1 by exp]ts qualifi$ by sci5tific tra9+ & exp]i5ce to evaluate ! safety ( ! drug involv$1 on ! basis ( :i* x c\ld fairly & responsibly be conclud$ t ! drug w h ! effect x purports or is repres5t$ to h "u ! conditions ( use prescrib$1 recomm5d$1 or sugge/$ 9 ! label+ or propos$ label+2 & 7,b7 !re is relevant e>ly evid5ce bas$ on adequate & well-controll$ inve/igations1 includ+ e>ly-/age cl9ical inve/igations1 to e/abli% t-- 7i7 ! drug provides a positive !rapeutic outcome2 & 7ii7 ! outcome ( ! drug is consi/5t ) or great] ?an curr5tly m>ket$ on-label !rapies1 ) equal or few] side effects1 if !re are curr5tly m>ket$ on-label !rapies4 7#b7 ,protocols ,! ,secret>y %all promulgate rules t e/abli% ! appropriate protocols to enable roll+1 real-"t1 mid-trial submission :ile pres]v+ ! integrity ( ! ongo+ trial & )\t p5aliz+ ! sponsor = seek+ provisional approval "u ?is section4 7#c7 ,real _w evid5ce ,! ,secret>y %all all[ ! use ( real _w evid5ce 7z def9$ 9 section #eje;,f7b771 includ+ real _w data us$ to g5]ate real _w evid5ce1 to support an application = provisional approval "u ?is section1 & to fulfill ! foll[-up requirem5ts & support applications = approval "u section #eje or section #cea ( ! ,public ,heal? ,s]vice ,act1 z applicable4 7#d7 ,use ( sci5tifically sub/antiat$ surrogates 7,a7 ,9 g5]al ,! sponsor ( an application = provisional approval "u ?is section may use sci5tifically sub/antiat$ surrogates to support su* application4 7,b7 ,def9ition ,9 subp>agraph 7,a71 ! t]m sci5tifically sub/antiat$ surrogates means surrogate endpo9ts to pr$ict cl9ical b5efit o!r ?an su* endpo9ts previ\sly validat$ by ! ,secret>y1 bas$ on-- 7i7 epidemiologic1 !rapeutic1 pa?ophysiologic1 or o!r evid5ce2 or 7ii7 an effect on a cl9ical endpo9t o!r ?an survival or irrev]sible morbidity ( int]e/4 7d7 ,transp>5cy & pati5t monitor+ requirem5ts 7#a7 ,regi/ries 7,a7 ,9 g5]al ,! sponsor ( a drug provisionally approv$ "u ?is section %all require t all pati5ts :o use su* drug p>ticipate 9 an obs]vational regi/ry & cons5t to ! sponsor's collection1 & submission to ! regi/ry1 ( data relat$ to ! pati5t's use ( su* drug until su* drug receives approval "u section #eje or section #cea ( ! ,public ,heal? ,s]vice ,act1 or ! provisional approval is resc9d$4 7,b7 ,requirem5ts = regi/ries ,an obs]vational regi/ry describ$ 9 subp>agraph 7,a7 may be run by a ?ird p>ty1 su* z a gov]nm5t1 = pr(it1 or nonpr(it organization1 & %all track all pati5ts :o use ! provisionally approv$ drug4 7,c7 ,accessibility ,an obs]vational regi/ry describ$ 9 subp>agraph 7,a7 %all be easily accessible =-- 7i7 all pati5ts :o are p>ticipat+ 9 any regi/ry relat$ to a provisionally approv$ drug t all[s = easy1 unre/rict$ 7or transp>5t7 access = su* pati5ts to _! pati5t data & relat$ in=mation reg>d+ _! usage ( ! provisionally approv$ drug2 & 7ii7 approv$ rese>*]s & m$ical pr(essionals :o may access data ma9ta9$ 9 ! regi/ry1 :i* access %all be = public heal? rese>* & only 9 a de-id5tifi$1 aggregat$ mann]4 7#b7 ,fund+ ,an obs]vational regi/ry "u ?is subsection %all be ma9ta9$1 z applicable-- 7,a7 by ! sponsor ( ! drug provisionally approv$ "u ?is section t is ! subject ( ! regi/ry2 or 7,b7 by a ?ird p>ty1 su* z a gov]nm5t1 = pr(it1 or nonpr(it organization4 7#c7 ,sponsor requirem5ts 7,a7 ,9 g5]al ,= any drug application provisionally approv$ "u ?is section1 ! ,secret>y %all notify ! sponsor ( ! exact data su* sponsor is requir$ to submit to an obs]vational regi/ry4 7,b7 ,annual review ( ! regi/ry2 p5alties ,! ,secret>y %all conduct an annual review ( obs]vational regi/ries e/abli%$ "u ?is subsection4 ,if1 at su* an annual review1 few] ?an #ij p]c5t ( pati5ts are p>ticipat+ 9 an obs]vational regi/ry ) respect to a drug approv$ "u ?is section1 ! ,secret>y %all issue to ! sponsor ( su* drug a civil monet>y p5alty ( n m ?an #ajj1jjj4 ,if a violation ( ?is section is n correct$ )9 ! #cj-;day p]iod foll[+ notification1 ! sponsor %all1 9 addition to any p5alty "u ?is subp>agraph be subject to a civil monet>y p5alty ( n m ?an #aj1jjj = ea* day ( ! violation aft] su* p]iod until ! violation is correct$4 ,if application pati5t p>ticipation 9 an obs]vational regi/ry is n at or above #ij p]c5t )9 #f mon?s ( issuance ( su* p5alty1 ! provisional approval %all be )drawn4 7#d7 ,annual report to ,congress ,! ,secret>y %all submit an annual report to ,congress on all drugs grant$ provisional approval "u ?is section4 ,su* report %all include-- 7,a7 ! numb] ( pati5ts treat$ ) ea* su* drug1 & ! numb] ( pati5ts track$ 9 an obs]vational regi/ry ) respect to ea* su* drug2 7,b7 a discussion ( ! m9imum am\nt ( data requir$ 9 ! regi/ries1 includ+ pati5t treatm5ts & uses1 l5g? ( use1 side effects enc\nt]$1 relevant biom>k]s or sci5tifically sub/antiat$ surrogates1 scan results1 cause ( dea? & h[ long ! pati5t liv$1 & adv]se drug effects2 7,c7 a li/ ( all su* drugs = :i* an application = approval "u section #eje ( ?is ,act or section #cea ( ! ,public ,heal? ,s]vice ,act1 or an application = an ext5sion ( provisional approval "u ?is section1 has be5 submitt$2 & 7,d7 a li/ ( all applications d5i$ provisional approval "u ?is section1 toge!r ) an explanation = ! decisions to d5y ea* su* application4 7e7 ,)drawal ( provisional approval 7#a7 ,9 g5]al ,! ,secret>y %all )draw provisional approval "u ?is section if !re are a significant numb]s ( pati5ts :o exp]i5ce s]i\s adv]se effects1 comp>$ to ! o!r curr5tly m>ket$ on-label !rapies t are available = ! applicable disease or condition4 7#b7 ,effect ( )drawal ,if a provisional approval is )drawn "u ?is subsection1 ! sponsor may n make ! drug available to any new pati5ts1 b may be all[$ to cont9ue to make su* drug available to pati5ts :o />t$ tak+ ! drug prior to ! date ( )drawal1 = z long a p]iod z dictat$ by pati5t ne$1 z det]m9$ by ! ,secret>y4 7f7 ,transp>5cy ,any sci5tific1 m$ical1 academic1 or heal? c>e j\rnal publi%+ an article expla9+1 releas+1 convey+1 or ann\nc+ rese>* f9d+s :i* 7 fund$ by ! ,dep>tm5t ( ,heal? & ,human ,s]vices %all be prohibit$ f publi%+ su* rese>* unless-- 7#a7 su* article convey+ rese>* f9d+s is made publicly available on ! j\rnal's int]net website )\t a paywall or *>ge n lat] ?an #c mon?s aft] ! date on :i* su* article 0 fir/ provid$ to subscrib]s ( su* j\rnal 7or fir/ made available = pur*ase72 & 7#b7 ! article's au?or or rese>*] or au?or's in/itution 7or1 9 ! case ( multiple au?ors1 rese>*]s1 or in/itutions1 all su* au?ors1 rese>*]s1 or in/itutions7 receiv$ less ?an #cj p]c5t ( fund+ = su* rese>* f ! ,dep>tm5t ( ,heal? & ,human ,s]vices ?r\gh\t ! p]iod ( "t ! rese>* 0 conduct$4 7g7 ,in=m$ ,cons5t ,prior to receiv+ a drug provisionally approv$ "u ?is section1 ! sponsor ( ! drug %all receive f ea* pati5t1 or ! pati5t's repres5tative1 in=m$ cons5t1 ?r\gh a sign$ in=m$ cons5t =m1 ackn[l$g+ t su* pati5t und]/&s t ! drug did n und]go ! usual process = approval ( a drug by ! ,food & ,drug ,adm9i/ration1 & t su* pati5t is will+ to accept ! risks involv$ 9 tak+ su* drug4 7h7 ,po/m>ket controls & label+ 7#a7 ,,fda annual review ( regi/ry data ,! ,secret>y %all annually review ! data made available ?r\gh ! obs]vational regi/ries "u subsection 7d7 & make a det]m9ation reg>d+ :e!r ! side effect pr(ile ( any drug provisionally approv$ "u ?is section does n support ! b5efit provid$1 or ! data %[s ! b5efit is less ?an ! b5efits (f]$ ?r\gh o!r1 fully approv$ drugs4 7#b7 ,label+ ,! sponsor ( ! provisionally approv$ drug %all ensure t all label+ & promotional mat]ials = ! drug be> ! /atem5t provisionally approv$ by ! ,,fda p5d+ a full demon/ration ( effectiv5ess "u application numb] 7specify+ ! application numb] assign$ by ! ,secret>y 9 place ( ! blank74 ,all promotional1 educational & m>ket+ mat]ials = provisionally approv$ products %all be review$ & approv$ by ! ,secret>y be=e su* mat]ials are di/ribut$4 7#c7 ,rescission ( provisional approval ,if ! ,secret>y det]m9es t ! side effect pr(ile ( any drug includ$ 9 su* obs]vational regi/ries does n support ! b5efit provid$ by su* drug1 or t ! data %[s t ! b5efit is less ?an ! b5efits (f]$ ?r\gh o!r1 drugs approv$ "u section #eje ( ?is ,act or section #cea ( ! ,public ,heal? ,s]vice ,act1 ! ,secret>y %all resc9d su* provisional approval4 7i7 ,duration ( provisional approval2 requirem5t ,to br+ drug to m>ket 7#a7 ,duration2 r5ewals ,! provisional approval = a drug "u ?is section is effective = a #b-ye> p]iod4 ,! sponsor may reque/ r5ewal = provisional approval /atus = up to #c subsequ5t #b-ye> p]iods4 ,provisional approval /atus ) respect to a drug %all n exce$ a total ( #h ye>s f ! initial date ! sponsor 0 aw>d$ provisional approval /atus4 7#b7 ,m>ket+ requirem5t ,if any drug t receives provisional approval "u ?is section is n br\ght to m>ket )9 #ahj days ( ! provisional approval1 su* provisional approval %all be resc9d$4 7j7 ,limitation on liability ,) respect to any claim "u ,/ate law alleg+ t a drug sold or o!rwise made available pursuant to a grant ( provisional approval "u ?is section is unsafe or ineffective1 no liability 9 a cause ( action %all lie aga9/ a sponsor or manufactur]1 unless ! relevant conduct con/itutes reckless or willful misconduct1 gross neglig5ce1 or an int5tional tort "u any applicable ,/ate law4 7k7 ,"r ,to petition an advisory committee = approval 7#a7 ,9 g5]al ,! sponsor ( a drug grant$ provisional approval pursuant to ?is section may reque/1 at any "t aft] provisional approval is grant$ "u ?is section1 a meet+ ) ! appropriate advisory committee 7or advisory committees7 to pres5t safety & efficacy data = ! purposes ( receiv+ a recomm5dation f su* an advisory committee = approval "u section #eje ( ?is ,act or section #cea ( ! ,public ,heal? ,s]vice ,act ( ! provisionally approv$ drug4 ,su* a reque/$ meet+ %all be grant$ n lat] ?an #ij days aft] a reque/ is made4 ,no?+ 9 ?is p>agraph %all be con/ru$ to alt] ! processes & timeframes = recomm5dation = approval by su* an advisory committee ( ! provisionally approv$ drug or = approval ( ! provisionally approv$ drug "u section #eje ( ?is ,act or section #cea ( ! ,public ,heal? ,s]vice ,act4 7#b7 ,waiv] ( adequate & well-controll$ /udy requirem5ts 7,a7 ,9 g5]al ,9 consid]+ :e!r to recomm5d a drug t 0 provisionally approv$ "u ?is section = approval "u section #eje1 ! ,director ( ! ,c5t] = ,drug ,evaluation & ,rese>* %all consid] ! option to waive requirem5ts = adequate & well-controll$ /udies 9 accordance ) ! process describ$ 9 section #cad4abf7;c7 ( title #ba1 ,code ( ,f$]al ,regulations 7or successor regulations74 7,b7 ,biological products ,9 consid]+ :e!r to recomm5d a biological product t 0 provisionally approv$ "u ?is section = lic5sure "u section #cea ( ! ,public ,heal? ,s]vice ,act1 ! ,director ( ! ,c5t] = ,biologics ,evaluation & ,rese>* may1 & %all consid] ! option to1 waive requirem5ts1 z applicable1 = adequate & well-controll$ /udies = su* biological product 9 accordance ) ! process describ$ 9 section #cad4abf7;c7 ( title #ba1 ,code ( ,f$]al ,regulations 7or successor regulations74 4 7b7 ,con=m+ am5dm5t ,section #eje7;a7 ( ! ,f$]al ,food1 ,drug1 & ,cosmetic ,act 7#ba ,u4,s4,c4 #cee7;a77 is am5d$ by ins]t+ 1 or !re is 9 effect a provisional approval "u section #ebd;,c ) respect to su* drug be=e ! p]iod4 7c7 ,reimbursem5t 7#a7 ,private heal? insur]s ,section #bgai;,a ( ! ,public ,heal? ,s]vice ,act 7#db ,u4,s4,c4 #cjj;gg-#ai;a7 is am5d$ by add+ at ! end ! foll[+3 7f7 ,treatm5t ( c]ta9 drugs ,a gr\p heal? plan or heal? insurance issu] ( gr\p or individual heal? insurance cov]age %all n d5y cov]age ( any drug provisionally approv$ "u section #ebd;,c ( ! ,f$]al ,food1 ,drug1 & ,cosmetic ,act on ! basis ( su* drug be+ exp]im5tal4 ,9 det]m9+ cov]age "u ! applicable plan or cov]age1 a gr\p heal? plan or heal? insurance issu] %all treat a drug provisionally approv$ "u su* section 9 ! same mann] z su* plan or cov]age w\ld treat a drug approv$ "u section #eje ( ! ,f$]al ,food1 ,drug1 & ,cosmetic ,act or section #cea ( ?is ,act4 ,no?+ 9 ?is subsection %all be con/ru$ to require a gr\p heal? plan or heal? insurance issu] to cov] any specific drug provisionally approv$ "u su* section #ebd;,c4 4 7#b7 ,f$]al heal? c>e programs ,! requirem5t "u subsection 7f7 ( section #bgai;,a ( ! ,public ,heal? ,s]vice ,act 7z add$ by p>agraph 7#a77 %all apply ) respect to cov]age det]m9ations "u a ,f$]al heal? c>e program 7z def9$ 9 section #aabh;,b7f7 ( ! ,social ,security ,act 7#db ,u4,s4,c4 #acbj;a-#g;b7f777 9 ! same mann] su* requirem5t applies "u su* subsection 7f74 7#c7 ,con=m+ am5dm5t ,section #aibg7k77#b77;,a77i7 ( ! ,social ,security ,act 7#db ,u4,s4,c4 #acifr-#h7k77#b77;,a77i77 is am5d$-- 7,a7 by /rik+ or :i* & ins]t+ 1 :i*2 & 7,b7 by ins]t+ 1 or :i* is provisionally approv$ "u section #ebd;,c ( su* ,act be=e ! semicolon4