,,ii  #aai? ,,congress  #a/ ,session    
,s4 #gje  ,,9 ,,! ,,s5ate ,,( ,,!       
,,unit$ ,,/ates  ,febru>y #be1 #bjbe,mr4
 ,re$ 7= himself & ,mrs4 ,capito7       
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 !   
,innovation 9 ,p$iatric ,drugs ,act (   
#bjbe4  #b4 ,ensur+ completion (        
p$iatric /udy requirem5ts  7a7 ,equal   
acc\ntability = p$iatric /udy           
requirem5ts ,section #eje;,b7d7 ( !     
,f$]al ,food1 ,drug1 & ,cosmetic ,act   
7#ba ,u4,s4,c4 #cee;c7d77 is am5d$--    
7#a7 9 p>agraph 7#a71 by /rik+ ,beg9n+  
#bgj & ins]t+ ,noncompliance            
lett]4--,beg9n+ #bgj2  7#b7 9 p>agraph  
7#b7-- 7,a7 by /rik+ ,! drug or & ins]t+
 ,effect ( noncompliance4--,! drug or2 &
  7,b7 by /rik+ 7except t ! drug or     

biological product %all n be subject to 
action "u section #cjc7 & ins]t+ 7except
 t ! drug or biological product %all be 
subject to action "u section #cjc only  
if su* p]son demon/rat$ a lack ( due    
dilig5ce 9 satisfy+ ! applicable        
requirem5t72 &  7#c7 by add+ at ! end ! 
foll[+3  7#c7 ,limitation ,! ,secret>y  
%all n issue en=cem5t actions "u section
 #cjc = failures "u ?is subsection 9 !  
case ( a drug or biological product t is
 no long] m>ket$4  4  7b7 ,due dilig5ce 
,section #eje;,b7d7 ( ! ,f$]al ,food1   
,drug1 & ,cosmetic ,act 7#ba ,u4,s4,c4  
#cee;c7d771 z am5d$ by subsection 7a71  
is fur!r am5d$ by add+ at ! end !       
foll[+3  7#d7 ,due dilig5ce ,be=e !     
,secret>y may conclude t a p]son fail$  
to submit or o!rwise meet a requirem5t z
 describ$ 9 ! matt] prec$+ p>agraph     
7#a71 ! ,secret>y %all-- 7,a7 issue a   
noncompliance lett] pursuant to p>agraph
 7#a72  7,b7 provide su* p]son ) a      
#de-;day p]iod beg9n+ on ! date (       
receipt ( su* noncompliance lett] to    

respond 9 writ+ z set =? 9 su* p>agraph2
 &  7,c7 aft] review+ su* writt5        
response1 det]m9e :e!r ! p]son          
demon/rat$ a lack ( due dilig5ce 9      
satisfy+ su* requirem5t4  4  7c7 ,con=m+
 am5dm5ts ,section #cjc7;f77#d77;,a7 ( !
 ,f$]al ,food1 ,drug1 & ,cosmetic ,act  
7#ba ,u4,s4,c4 #ccc7;f77#d77;,a77 is    
am5d$ by /rik+ or #eje-a & ins]t+       
#eje-a1 or #eje;,b4  7d7 ,transition    
rule ,! ,secret>y ( ,heal? & ,human     
,s]vices may take en=cem5t action "u    
section #cjc ( ! ,f$]al ,food1 ,drug1 & 
,cosmetic ,act 7#ba ,u4,s4,c4 #ccc7 =   
failures describ$ 9 section #eje;,b7d7 (
 su* ,act 7#ba ,u4,s4,c4 #cee;c7d77 only
 if su* failures occur on or aft] ! date
 t is #ahj days aft] ! date ( enactm5t (
 ?is ,act4  #c4 ,,fda report on ,,prea  
en=cem5t ,section #ejh7;b7 ( ! ,food &  
,drug ,adm9i/ration ,safety &           
,innovation ,act 7#ba ,u4,s4,c4         
#cee;c-#a7;b77 is am5d$-- 7#a7 9        
p>agraph 7#aa71 by /rik+ ! semicolon at 
! end & ins]t+ 1 includ+ an evaluation (

 compliance ) deadl9es provid$ = 9      
def]rals & def]ral ext5sions22  7#b7 9  
p>agraph 7#ae71 by /rik+ & at ! end2    
7#c7 9 p>agraph 7#af71 by /rik+ ! p]iod 
at ! end & ins]t+ 2 &2 &  7#d7 by add+  
at ! end ! foll[+3  7#ag7 a li/+ (      
p5alties1 settlem5ts1 or paym5ts "u     
section #cjc ( ! ,f$]al ,food1 ,drug1 & 
,cosmetic ,act 7#ba ,u4,s4,c4 #cec7 =   
failure to comply ) requirem5ts "u su*  
section #eje;,b1 includ+1 = ea* p5alty1 
settlem5t1 or paym5t1 ! "n ( ! drug1 !  
sponsor !re(1 & ! am\nt ( ! p5alty1     
settlem5t1 or paym5t impos$4  4  #d4    
,program = p$iatric /udies ( drugs      
,section #dji;,i7d7 ( ! ,public ,heal?  
,s]vice ,act 7#db ,u4,s4,c4 #bhdm7d77 is
 am5d$ to read z foll[s3  7d7 ,fund+ ,( 
! am\nt made available = p$iatric rese>*
 to ea* national rese>* in/itute &      
national c5t] "u ?is title = ea* (      
fiscal ye>s #bjbf ?r\gh #bjcj1 !        
,director ( ,,nih is au?oriz$ to make   
available up to "o p]c5t ( su* am\nt =  
p$iatric rese>* "u ?is section4  4  #e4 

,p$iatric /udies ( orphan drugs  7a7    
,application ( p$iatric rese>*          
requirem5ts to orphan drugs  7#a7 ,9    
g5]al ,section #eje;,b7k7 ( ! ,f$]al    
,food1 ,drug1 & ,cosmetic ,act 7#ba     
,u4,s4,c4 #cee;c7k77 is am5d$ to read z 
foll[s3  7k7 ,relation to orphan drugs  
7#a7 ,9 g5]al ,?is section does n apply 
to a drug or biological product = an    
indication = :i* orphan designation has 
be5 grant$ "u section #ebf unless !     
,secret>y det]m9es t p$iatric assessm5ts
 ( su* drug or biological product       
requir$ "u ?is section c\ld repres5t a  
mean+ful !rapeutic b5efit z describ$ 9  
subsection 7c74  7#b7 ,def]rals & waiv]s
 ,def]rals & waiv]s "u subsections      
7a77#d7 & 7a77#e7 %all apply to         
assessm5ts describ$ 9 ?is subsection to 
! same ext5t & 9 ! same mann] z su*     
def]rals & waiv]s apply ) respect to !  
assessm5ts "u subsection 7a77#a71 &     
waiv]s "u subsection 7b77#b7 %all apply 
to assessm5ts describ$ 9 ?is subsection 
to ! same ext5t & 9 ! same mann] z su*  

waiv]s apply ) respect to ! assessm5ts  
requir$ pursuant to subsection 7b77#a74 
4  7#b7 ,applicability ,! am5dm5t made  
by p>agraph 7#a7 applies only to        
applications describ$ 9 subp>agraph 7,a7
 or 7,b7 ( section #eje;,b7a77#a7 ( !   
,f$]al ,food1 ,drug1 & ,cosmetic ,act   
7#ba ,u4,s4,c4 #cee;c7a77#a77 t are     
submitt$ on or aft] ! lat] (-- 7,a7 !   
date t is #ah mon?s aft] ! date (       
issuance ( ! f9al guidance "u subsection
 7b72 &  7,b7 su* lat] date z may be    
specifi$ by ! ,secret>y ( ,heal? &      
,human ,s]vices 7ref]r$ to 9 ?is section
 z ! ,secret>y7 by regulation4  7b7     
,guidance  7#a7 ,issuance ,! ,secret>y  
%all-- 7,a7 n lat] ?an #a ye> aft] !    
date ( enactm5t ( ?is ,act1 issue draft 
guidance describ+ h[1 ^u ! applicability
 ( ! am5dm5t made by subsection 7a77#a71
 ! requirem5ts ( section #eje;,b ( !    
,f$]al ,food1 ,drug1 & ,cosmetic ,act   
7#ba ,u4,s4,c4 #cee;c7 w apply ) respect
 to any drug or biological product = an 
indication )9 a disease or condition =  

:i* orphan designation has be5 grant$ "u
 section #ebf ( su* ,act 7#ba ,u4,s4,c4 
#cfj;bb72 &  7,b7 n lat] ?an #ah mon?s  
aft] ! date ( ! public meet+ requir$ by 
subsection 7c77#a71 f9alize su* draft   
guidance4  7#b7 ,cont5ts ,! guidance "u 
subsection 7b7 %all address ! foll[+3   
7,a7 ,in=mation reg>d+ h[ full & p>tial 
waiv]s "u subsections 7a77#e77;,a71     
7a77#e77;,b71 & 7b77#b7 ( section       
#eje;,b ( ! ,f$]al ,food1 ,drug1 &      
,cosmetic ,act 7#ba ,u4,s4,c4 #cee;c7 = 
any drug or biological product = an     
indication )9 a disease or condition =  
:i* orphan designation has be5 grant$ "u
 section #ebf ( su* ,act 7#ba ,u4,s4,c4 
#cfj;bb7 w be grant$4  7,b7 ,application
 ( ! requirem5ts ( section #eje;,b7e7 ( 
su* ,act 7#ba ,u4,s4,c4 #cee;c7e77 to   
drugs or biological products = an       
indication )9 a disease or condition =  
:i* orphan designation has be5 grant$ "u
 section #ebf ( su* ,act 7#ba ,u4,s4,c4 
#cfj;bb71 includ+ submission & tim+ (   
plann$ reque/s = full or p>tial waiv]s &

 responses by ! ,food & ,drug           
,adm9i/ration to ^? reque/s4  7,c7 ,r>e 
diseases & conditions 7z def9$ 9 section
 #ebf7;a77#b7 ( su* ,act 7#ba ,u4,s4,c4 
#cfj;bb7a77#b77 t %\ld be add$ to ! li/s
 "u section #eje;,b7a77#e77;,e7 &       
#eje;,b7b77#b77;,e7 ( su* ,act1 z add$  
by subsection 7f71 & a process =        
regul>ly updat+ su* li/s4  7,d7         
,situations :]e ! initial p$iatric /udy 
plan "u section #eje;,b7e7 ( su* ,act   
7#ba ,u4,s4,c4 #cee;c7e77 includes a    
plan to fulfill ! requirem5ts ( section 
#eje;,b7a7 ( su* ,act 7#ba ,u4,s4,c4    
#cee;c7a77 )\t reque/+ waiv]s 9 any age 
gr\p4  7,e7 ,consid]ation ( h[ !        
,secret>y w balance ! unique sci5tific  
*all5ges ( r>e disease drug developm5t )
 ! ne$ = improv$ p$iatric label+ ( drugs
 & biological products = indications )9 
diseases or conditions = :i* orphan     
designation has be5 grant$ "u section   
#ebf ( su* ,act 7#ba ,u4,s4,c4 #cfj;bb74
  7,f7 ,consid]ations ( ! /r5g?s1       
weaknesses1 appropriat5ess1 &           

limitations ( diff]5t types ( real-_w   
evid5ce specific to ! fulfillm5t (      
requirem5ts "u section #eje;,b ( su*    
,act 7#ba ,u4,s4,c4 #cee;c74  7,g7      
,consid]ation ( input receiv$ f ! public
 meet+ set =? 9 subsection 7c74  7c7    
,public meet+ ,! ,secret>y %all-- 7#a7 n
 lat] ?an #f mon?s aft] ! date (        
issuance ( ! draft guidance "u          
subsection 7b77#a77;,a71 hold a public  
meet+ to in=m ! f9al guidance to be     
issu$ "u subsection 7b77#a77;,b72 &     
7#b7 publi% prior notice ( su* meet+ 9 !
 ,f$]al ,regi/]4  7d7 ,,gao /udy ,n lat]
 ?an #d ye>s aft] ! applicability date  
describ$ 9 subsection 7a77#b71 !        
,comptroll] ,g5]al ( ! ,unit$ ,/ates    
%all submit to ! ,committee on ,en]gy & 
,comm]ce & ! ,committee on ,ways &      
,means ( ! ,h\se ( ,repres5tatives & !  
,committee on ,heal?1 ,education1       
,labor1 & ,p5sions ( ! ,s5ate a report  
t-- 7#a7 addresses ! impacts ( ?is ,act 
on-- 7,a7 r>e disease drug developm5t 9 
! ,unit$ ,/ates2 &  7,b7 ! availability 

( p$iatric in=mation on drugs &         
biological products )9 diseases or      
conditions = indications = :i* orphan   
designation has be5 grant$ "u section   
#ebf ( ! ,f$]al ,food1 ,drug1 &         
,cosmetic ,act 7#ba ,u4,s4,c4 #cfj;bb72 
&  7#b7 includes-- 7,a7 ! f9d+s ( a     
survey ( companies ( v>y+ sizes engag$ 9
 ! developm5t ( orphan drugs1 :i* %all  
include que/ions reg>d+ ! feasibility & 
o!r *all5ges ( conduct+ p$iatric /udies 
= su* indications2  7,b7 input f pati5t 
gr\ps & m$ical provid] associations2 &  
7,c7 an assessm5t ( ! impact *anges to  
requir$ p$iatric /udies _h on drug      
developm5t = r>e diseases4  7e7 ,rule ( 
con/ruction ,no?+ 9 ?is section %all be 
con/ru$ to limit requirem5ts =          
inve/igations1 z describ$ 9 section     
#eje;,b7a77#c7 ( ! ,f$]al ,food1 ,drug1 
& ,cosmetic ,act 7#ba ,u4,s4,c4         
#cee;c7a77#c771 ( molecul>ly t>get$     
p$iatric canc] drugs = :i* orphan       
designation has be5 grant$ "u section   
#ebf ( su* ,act 7#ba ,u4,s4,c4 #cfj;bb74

  7f7 ,c]ta9ty reg>d+ waiv]s ,section   
#eje;,b ( ! ,f$]al ,food1 ,drug1 &      
,cosmetic ,act 7#ba ,u4,s4,c4 #cee;c7 is
 am5d$-- 7#a7 9 subsection 7a77#e71 by  
add+ at ! end ! foll[+3  7,e7 ,automatic
 full waiv] li/ ,! ,secret>y %all ma9ta9
 a li/1 po/$ on ! website ( ! ,food &   
,drug ,adm9i/ration1 ( adult-relat$     
diseases & conditions-- 7i7 ) respect to
 :i* ! necess>y /udies are impossible or
 highly impracticable1 z describ$ 9     
subp>agraph 7,a77i72 or  7ii7 = :i* a   
drug or biological product = su* disease
 or condition o!rwise meets ! crit]ia   
describ$ 9 subp>agraph 7,a74  2  7#b7 9 
subsection 7b77#b71 by add+ at ! end !  
foll[+3  7,e7 ,automatic full waiv] li/ 
,! ,secret>y %all ma9ta9 a li/1 po/$ on 
! website ( ! ,food & ,drug             
,adm9i/ration1 ( adult-relat$ diseases &
 conditions ) respect to :i* ! necess>y 
/udies w\ld meet ! crit]ia = a full     
waiv] "u subp>agraph 7,a74  2 &  7#c7 9 
subsection 7e77#d71 by add+ at ! end !  
foll[+3 ,if1 at ! "t ( an applicant's   

submission ( ! initial p$iatric /udy    
plan1 ! disease or condition = :i* !    
drug is int5d$ to treat appe>s on ! li/ 
"u subsection 7a77#e77;,e71 !n !        
assessm5ts = su* disease or condition   
%all be waiv$ "u subsection 7a77#e744