,,ii  #aai? ,,congress  #a/ ,session    
,s4 #ccjb  ,,9 ,,! ,,s5ate ,,( ,,!      
,,unit$ ,,/ates  ,decemb] #b1 #bjbe,mr4 
,mull9 7= himself1 ,mr4 ,b5net1 ,ms4    
,klobu*>1 ,mrs4 ,%ahe51 ,mr4 ,m>%all1   
,mr4 ,scott ( ,florida1 ,ms4 ,coll9s1   
,mr4 ,coons1 ,mrs4 ,capito1 ,mr4 ,kelly1
 ,mr4 ,hu/$1 ,mr4 ,kim1 ,mr4 ,%eehy1    
,mrs4 ,moody1 ,mr4 ,hick5loop]1 ,ms4    
,duckwor?1 ,mr4 ,boozman1 ,mr4 ,s*iff1  
,mr4 ,k5n$y1 ,mr4 ,re$1 ,mr4 ,s*mitt1   
,mr4 ,m>key1 ,mr4 ,book]1 & ,mr4        
,ju/ice7 introduc$ ! foll[+ bill2 :i* 0 
read twice & ref]r$ to ! ,committee on  
,heal?1 ,education1 ,labor1 & ,p5sions  
,a ,,bill  ,to am5d ! ,f$]al ,food1     
,drug1 & ,cosmetic ,act ) respect to    
molecul>ly t>get$ p$iatric canc]        
inve/igations1 & = o!r purposes4  #a4   
,%ort title ,?is ,act may be cit$ z !   
,mikaela ,naylon ,give ,kids a ,*ance   
,act ( #bjbe4  #b4 ,rese>* into p$iatric
 uses ( drugs2 additional au?orities (  
,food & ,drug ,adm9i/ration reg>d+      
molecul>ly t>get$ canc] drugs  7a7 ,9   

g5]al  7#a7 ,additional active +r$i5t = 
application drug2 limitation reg>d+     
novel-comb9ation application drug       
,section #eje;,b7a77#c7 ( ! ,f$]al      
,food1 ,drug1 & ,cosmetic ,act 7#ba     
,u4,s4,c4 #cee;c7a77#c77 is am5d$-- 7,a7
 by r$esignat+ subp>agraphs 7,b7 & 7,c7 
z subp>agraphs 7,c7 & 7,d71             
respectively2 &  7,b7 by /rik+          
subp>agraph 7,a7 & ins]t+ ! foll[+3     
7,a7 ,9 g5]al ,= purposes ( p>agraph    
7#a77;,b71 ! inve/igation describ$ 9 ?is
 p>agraph is a molecul>ly t>get$        
p$iatric canc] inve/igation (-- 7i7 !   
drug or biological product = :i* !      
application ref]r$ to 9 su* p>agraph is 
submitt$2 or  7ii7 su* drug or          
biological product us$ 9 comb9ation )-- 
7,i7 an active +r$i5t ( a drug or       
biological product-- 7aa7 = :i* an      
approv$ application "u section #eje7;j7 
"u ?is ,act or "u section #cea7k7 ( !   
,public ,heal? ,s]vice ,act is 9 effect2
 &  7bb7 t is det]m9$ by ! ,secret>y1   
aft] consultation ) ! applicant1 to be  

"p ( ! /&>d ( c>e = treat+ a p$iatric   
canc]2 or  7,,ii7 an active +r$i5t ( a  
drug or biological product-- 7aa7 = :i* 
an approv$ application "u section       
#eje7;b7 ( ?is ,act or section #cea7;a7 
( ! ,public ,heal? ,s]vice ,act to treat
 an adult canc] is 9 effect & is held by
 ! same p]son submitt+ ! application "u 
p>agraph 7#a77;,b72 &  7bb7 t is direct$
 at a molecul> t>get t ! ,secret>y      
det]m9es to be sub/antially relevant to 
! gr[? or progression ( a p$iatric      
canc]4  7,b7 ,additional requirem5ts    
7i7 ,design ( inve/igation ,a molecul>ly
 t>get$ p$iatric canc] inve/igation     
ref]r$ to 9 subp>agraph 7,a7 %all be    
design$ to yield cl9ically mean+ful     
p$iatric /udy data t is ga!r$ us+       
appropriate =mulations = ea* age gr\p = 
:i* ! /udy is requir$1 reg>d+ dos+1     
safety1 & prelim9>y efficacy to in=m    
pot5tial p$iatric label+4  7ii7         
,limitation ,an inve/igation describ$ 9 
subp>agraph 7,a77ii7 may be requir$ only
 if ! drug or biological product = :i* !

 application ref]r$ to 9 p>agraph       
7#a77;,b7 conta9s ei!r-- 7,i7 a s+le new
 active +r$i5t2 or  7,,ii7 m ?an "o     
active +r$i5t1 if an application = !    
comb9ation ( active +r$i5ts has n       
previ\sly be5 approv$ b ea* active      
+r$i5t is 9 a drug product t has be5    
previ\sly approv$ to treat an adult     
canc]4  7iii7 ,results (                
already-complet$ precl9ical /udies (    
application drug ,) respect to an       
inve/igation requir$ pursuant to        
p>agraph 7#a77;,b71 ! ,secret>y may     
require ! results ( any complet$        
precl9ical /udies relevant to ! initial 
p$iatric /udy plan be submitt$ to !     
,secret>y at ! same "t t ! initial      
p$iatric /udy plan requir$ "u subsection
 7e77#a7 is submitt$4  7iv7 ,rule (     
con/ruction reg>d+ inactive +r$i5ts ,)  
respect to a comb9ation ( active +r$i5ts
 ref]r$ to 9 subp>agraph 7,a77ii71 su*  
subp>agraph %all n be con/ru$ z address+
 ! use ( inactive +r$i5ts ) su*         
comb9ation4  4  7#b7 ,det]m9ation (     

applicable requirem5ts ,section         
#eje;,b7e77#a7 ( ! ,f$]al ,food1 ,drug1 
& ,cosmetic ,act 7#ba ,u4,s4,c4         
#cee;c7e77#a77 is am5d$ by add+ at ! end
 ! foll[+3 ,! ,secret>y %all det]m9e    
:e!r subp>agraph 7,a7 or 7,b7 (         
subsection 7a77#a7 applies ) respect to 
an application be=e ! date on :i* !     
applicant is requir$ to submit ! initial
 p$iatric /udy plan "u p>agraph         
7#b77;,a744  7#c7 ,cl>ify+ applicability
 ,section #eje;,b7a77#a7 ( ! ,f$]al     
,food1 ,drug1 & ,cosmetic ,act 7#ba     
,u4,s4,c4 #cee;c7a77#a77 is am5d$ by    
add+ at ! end ! foll[+3  7,c7  ,rule (  
con/ruction  ,no application t is       
subject to ! requirem5ts ( subp>agraph  
7,b7 %all be subject to ! requirem5ts ( 
subp>agraph 7,a71 & no application 7or  
supplem5t to an application7 t is       
subject to ! requirem5ts ( subp>agraph  
7,a7 %all be subject to ! requirem5ts ( 
subp>agraph 7,b74  4  7#d7 ,con=m+      
am5dm5ts ,section #eje;,b7a7 ( ! ,f$]al 
,food1 ,drug1 & ,cosmetic ,act 7#ba     

,u4,s4,c4 #cee;c7a77 is am5d$-- 7,a7 9  
p>agraph 7#c77;,c71 z r$esignat$ by     
p>agraph 7#a77;,a7 ( ?is subsection1 by 
/rik+ inve/igations describ$ 9 ?is      
p>agraph & ins]t+ inve/igations ref]r$  
to 9 subp>agraph 7,a72 &  7,b7 9        
p>agraph 7#c77;,d71 z r$esignat$ by     
p>agraph 7#a77;,a7 ( ?is subsection1 by 
/rik+ ! assessm5ts "u p>agraph 7#b77;,b7
 & ins]t+ ! assessm5ts requir$ "u       
p>agraph 7#a77;,a74  7b7 ,guidance ,!   
,secret>y ( ,heal? & ,human ,s]vices1   
act+ ?r\gh ! ,commission] ( ,food &     
,drugs1 %all-- 7#a7 n lat] ?an #ab mon?s
 aft] ! date ( enactm5t ( ?is ,act1     
issue draft guidance on ! implem5tation 
( ! am5dm5ts made by subsection 7a72 &  
7#b7 n lat] ?an #ab mon?s aft] clos+ !  
comm5t p]iod on su* draft guidance1     
f9alize su* guidance4  7c7              
,applicability ,! am5dm5ts made by ?is  
section apply ) respect to any          
application "u section #eje7;b7 ( !     
,f$]al ,food1 ,drug1 & ,cosmetic ,act   
7#ba ,u4,s4,c4 #cee7;b77 & any          

application "u section #cea7;a7 ( !     
,public ,heal? ,s]vice ,act 7#db        
,u4,s4,c4 #bfb7;a771 t is submitt$ on or
 aft] ! date t is #c ye>s aft] ! date ( 
enactm5t ( ?is ,act4  7d7 ,reports to   
,congress  7#a7 ,secret>y ( ,heal? &    
,human ,s]vices ,n lat] ?an #f ye>s aft]
 ! date ( enactm5t ( ?is ,act1 !        
,secret>y ( ,heal? & ,human ,s]vices    
%all submit to ! ,committee on ,en]gy & 
,comm]ce ( ! ,h\se ( ,repres5tatives & !
 ,committee on ,heal?1 ,education1      
,labor1 & ,p5sions ( ! ,s5ate a report  
on ! ,secret>y's ef=ts1 9 coord9ation ) 
indu/ry1 to ensure implem5tation ( !    
am5dm5ts made by subsection 7a74  7#b7  
,,gao /udy & report  7,a7 ,/udy ,n lat] 
?an #h ye>s aft] ! date ( enactm5t ( ?is
 ,act1 ! ,comptroll] ,g5]al ( ! ,unit$  
,/ates %all conduct a /udy ( !          
effectiv5ess ( requir+ assessm5ts &     
inve/igations describ$ 9 section #eje;,b
 ( ! ,f$]al ,food1 ,drug1 & ,cosmetic   
,act 7#ba ,u4,s4,c4 #cee;c71 z am5d$ by 
subsection 7a71 9 ! developm5t ( drugs &

 biological products = p$iatric canc]   
indications1 includ+ consid]ation ( any 
b5efits to1 or burd5s on1 p$iatric canc]
 drug developm5t4  7,b7 ,f9d+s ,n lat]  
?an #aj ye>s aft] ! date ( enactm5t (   
?is ,act1 ! ,comptroll] ,g5]al %all     
submit to ! ,committee on ,en]gy &      
,comm]ce ( ! ,h\se ( ,repres5tatives & !
 ,committee on ,heal?1 ,education1      
,labor1 & ,p5sions ( ! ,s5ate a report  
conta9+ ! f9d+s ( ! /udy conduct$ "u    
subp>agraph 7,a74  #c4 ,ext5sion (      
au?ority to issue priority review v\*]s 
to enc\rage treatm5ts = r>e p$iatric    
diseases  7a7 ,ext5sion ,section        
#ebi7;b77#e7 ( ! ,f$]al ,food1 ,drug1 & 
,cosmetic ,act 7#ba ,u4,s4,c4           
#cfj;ff7b77#e77 is am5d$ by /rik+       
,decemb] #bj1 #bjbd1 unless & all t     
foll[s ?r\gh ! p]iod at ! end & ins]t+  
,septemb] #cj1 #bjcj44  7b7 ,us] fee    
paym5t ,subsection #ebi7;c77#d7 ( !     
,f$]al ,food1 ,drug1 & ,cosmetic ,act   
7#ba ,u4,s4,c4 #cfj;ff7c77#d77 is am5d$ 
by /rik+ subp>agraph 7,a7 & ins]t+ !    

foll[+3  7,a7 ,9 g5]al ,! priority      
review us] fee requir$ by ?is subsection
 %all be due ^u ! submission ( a human  
drug application "u section #eje7;b77#a7
 or section #cea7;a7 ( ! ,public ,heal? 
,s]vice ,act = :i* ! priority review    
v\*] is us$4 ,all o!r us] fees associat$
 ) ! human drug application %all be due 
z requir$ by ! ,secret>y or "u          
applicable law4  4  7c7 ,,gao report on 
effectiv5ess ( r>e p$iatric disease     
priority v\*] aw>ds 9 inc5tiviz+ r>e    
p$iatric disease drug developm5t  7#a7  
,,gao /udy  7,a7 ,/udy ,! ,comptroll]   
,g5]al ( ! ,unit$ ,/ates %all conduct a 
/udy ( ! effectiv5ess ( aw>d+ r>e       
p$iatric disease priority v\*]s "u      
section #ebi ( ! ,f$]al ,food1 ,drug1 & 
,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;ff71 
z am5d$ by subsection 7a71 9 !          
developm5t ( human drug products t treat
 or prev5t r>e p$iatric diseases 7z     
def9$ 9 su* section #ebi74  7,b7        
,cont5ts ( /udy ,9 conduct+ ! /udy "u   
subp>agraph 7,a71 ! ,comptroll] ,g5]al  

%all exam9e ! foll[+3 7i7 ,! indications
 = ea* drug or biological product t--   
7,i7 is ! subject ( a r>e p$iatric      
disease product application 7z def9$ 9  
section #ebi ( ! ,f$]al ,food1 ,drug1 & 
,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;ff77 
= :i* a priority review v\*] 0 aw>d$2 & 
7,,ii7 0 approv$ "u section #eje ( !    
,f$]al ,food1 ,drug1 & ,cosmetic ,act   
7#db ,u4,s4,c4 #cee7 or lic5s$ "u       
section #cea ( ! ,public ,heal? ,s]vice 
,act 7#db ,u4,s4,c4 #bfb74  7ii7 ,:e!r1 
& to :at ext5t1 an unmet ne$ relat$ to !
 treatm5t or prev5tion ( a r>e p$iatric 
disease 0 met ?r\gh ! approval or       
lic5sure ( su* a drug or biological     
product4  7iii7 ,! size ( ! company to  
:i* a priority review v\*] 0 aw>d$ "u   
section #ebi ( ! ,f$]al ,food1 ,drug1 & 
,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;ff7 =
 su* a drug or biological product4  7iv7
 ,! value ( su* priority review v\*] if 
transf]r$4  7v7 ,id5tification ( ea*    
drug = :i* a priority review v\*] aw>d$ 
"u su* section #ebi 0 us$4  7vi7 ,! size

 ( ! company us+ ea* priority review    
v\*] aw>d$ "u su* section #ebi4  7vii7  
,! l5g? ( ! p]iod ( "t betwe5 ! date on 
:i* a priority review v\*] 0 aw>d$ "u   
su* section #ebi & ! date on :i* x 0    
us$4  7viii7 ,:e!r1 & to :at ext5t1 an  
unmet ne$ relat$ to ! treatm5t or       
prev5tion ( a r>e p$iatric disease 0 met
 ?r\gh ! approval "u section #eje ( !   
,f$]al ,food1 ,drug1 & ,cosmetic ,act   
7#db ,u4,s4,c4 #cee7 or lic5sure "u     
section #cea ( ! ,public ,heal? ,s]vice 
,act 7#db ,u4,s4,c4 #bfb7 ( a drug = :i*
 a priority review v\*] 0 us$4  7ix7    
,:e!r1 & to :at ext5t1 companies 7      
motivat$ by ! availability ( priority   
review v\*]s "u section #ebi ( ! ,f$]al 
,food1 ,drug1 & ,cosmetic ,act 7#ba     
,u4,s4,c4 #cfj;ff7 to attempt to develop
 a drug = a r>e p$iatric disease4  7x7  
,:e!r1 & to :at ext5t1 p$iatric review  
v\*]s aw>d$ "u su* section 7 successful 
9 /imulat+ developm5t & exp$it$ pati5t  
access to drug products = treatm5t or   
prev5tion ( a r>e p$iatric disease t    

w\ldn't o!rwise take place )\t !        
inc5tive provid$ by su* v\*]s4  7xi7 ,! 
impact ( su* priority review v\*]s on ! 
workload1 review process1 & public heal?
 prioritization ef=ts ( ! ,food & ,drug 
,adm9i/ration4  7xii7 ,any o!r inc5tives
 9 ,f$]al law t exi/ = companies        
develop+ drugs or biological products   
describ$ 9 clause 7i74  7#b7 ,report on 
f9d+s ,n lat] ?an #e ye>s aft] ! date ( 
! enactm5t ( ?is ,act1 ! ,comptroll]    
,g5]al ( ! ,unit$ ,/ates %all submit to 
! ,committee on ,en]gy & ,comm]ce ( !   
,h\se ( ,repres5tatives & ! ,committee  
on ,heal?1 ,education1 ,labor1 &        
,p5sions ( ! ,s5ate a report conta9+ !  
f9d+s ( ! /udy conduct$ "u p>agraph     
7#a74