,i  #aah? ,,congress  #a/ ,session  ,h4 
,r4 #fffd  ,,9 ,,! ,,h\se ,,(           
,,repres5tatives  ,decemb] #g1 #bjbc,ms4
 ,e%oo 7= h]self & ,mr4 ,mc,caul7       
introduc$ ! foll[+ bill2 :i* 0 ref]r$ to
 ! ,committee on ,en]gy & ,comm]ce  ,a  
,,bill  ,to enc\rage innovation 9 !     
developm5t ( p$iatric drugs1 & = o!r    
purposes4  #a4 ,%ort title ,?is ,act may
 be cit$ z ! ,innovation 9 ,p$iatric    
,drugs ,act ( #bjbc4  #b4 ,p$iatric     
/udies ( orphan drugs  7a7 ,application 
( p$iatric rese>* requirem5ts to orphan 
drugs  7#a7 ,9 g5]al ,section #eje;,b ( 
! ,f$]al ,food1 ,drug1 & ,cosmetic ,act 
7#ba ,u4,s4,c4 #cee;c7 is am5d$ by /rik+
 subsection 7k74  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]
 ! date t is #ah mon?s aft] ! date (    
enactm5t ( ?is ,act4  7b7 ,guidance ,!  

,secret>y ( ,heal? & ,human ,s]vices    
7ref]r$ to 9 ?is ,act z ! ,secret>y7    
%all--  7#a7 n lat] ?an #f mon?s aft] ! 
date ( enactm5t ( ?is ,act1 issue draft 
guidance describ+ h[1 ^u ! applicability
 ( ! am5dm5t made by subsection 7a77#a71
 ! requirem5ts ( section #eje;,b ( !    
,f$]al ,food1 ,drug1 & ,cosmetic ,act   
7#ba ,u4,s4,c4 #cee;c7 w apply ) respect
 to any drug or biological product = an 
indication = :i* orphan designation has 
be5 grant$ "u section #ebf ( su* ,act   
7#ba ,u4,s4,c4 #cfj;bb72 &  7#b7 n lat] 
?an #ah mon?s aft] ! date ( enactm5t (  
?is ,act1 f9alize su* draft guidance4   
7c7 ,cont5t ( guidance ,! 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$]al ,food1 ,drug1 &      
,cosmetic ,act 7#ba ,u4,s4,c4 #cee;c7 = 
any drug or biological product = an     
indication = :i* orphan designation has 
be5 grant$ "u section #ebf ( su* ,act   

7#ba ,u4,s4,c4 #cfj;bb7 w be grant$     
7includ+ a description ( impossible or  
highly impracticable /udies =           
indications = :i* orphan designation has
 be5 grant$ "u su* section #ebf74  7#b7 
,application ( ! requirem5ts ( section  
#eje;,b7e7 ( su* ,act 7#ba ,u4,s4,c4    
#cee;c7e77 to drugs or biological       
products = an indication = :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      
,process = regul>ly updat+ conditions t 
automatically qualify = a waiv] "u      
subsection 7a77#e77;,a7 or 7b77#b77;,a7 
( section #eje;,b ( su* ,act 7#ba       
,u4,s4,c4 #cee;c74  7#d7 ,situations :]e
 ! initial p$iatric /udy plan "u section
 #eje;,b7e7 ( su* ,act 7#ba ,u4,s4,c4   
#cee;c7e77 includes a plan to /udy !    
drug or biological product 9 all        
relevant p$iatric age gr\ps4  7#e7      

,consid]ation ( h[ ! ,secret>y ( ,heal? 
& ,human ,s]vices w balance ! unique    
sci5tific *all5ges ( r>e disease drug   
developm5t ) ! ne$ = improv$ p$iatric   
label+ ( drugs & biological products =  
indications = :i* orphan designation has
 be5 grant$ "u section #ebf ( su* ,act  
7#ba ,u4,s4,c4 #cfj;bb74  7#f7          
,applicability ( real-_w evid5ce 9 !    
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 7d74  7d7 ,public meet+ ,!   
,secret>y %all-- 7#a7 n lat] ?an #a ye> 
aft] ! date ( enactm5t ( ?is ,act1 hold 
a public meet+ to in=m ! f9al guidance  
to be issu$ "u subsection 7b77#b72 &    
7#b7 publi% prior notice ( su* meet+ 9 !
 ,f$]al ,regi/]4  7e7 ,,gao /udy ,n lat]
 ?an #d ye>s aft] ! date ( enactm5t (   
?is ,act1 ! ,comptroll] ,g5]al ( !      
,unit$ ,/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 ! increas$ availability 
( p$iatric in=mation on drugs &         
biological products = indications = :i* 
orphan designation has be5 grant$ "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 drugs2 &  7,b7   
input f pati5t gr\ps & m$ical provid]   
associations4  7f7 ,rule ( con/ruction  
,no?+ 9 ?is section %all be con/ru$ 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  #c4 ,reau?oriz+ !  

program = p$iatric /udies ( drugs       
,section #dji;,i7d77#a7 ( ! ,public     
,heal? ,s]vice ,act 7#db ,u4,s4,c4      
#bhdm7d77#a77 is am5d$ 9 p>agraph 7#a7  
by /rik+ #be1jjj1jjj = ea* ( fiscal ye>s
 #bjbc ?r\gh #bjbg & ins]t+ #ej1jjj1jjj 
= ea* ( fiscal ye>s #bjbc ?r\gh #bjbg4  
#d4 ,ensur+ completion ( p$iatric /udy  
requirem5ts  7a7 ,equal acc\ntability = 
p$iatric /udy requirem5ts ,section      
#eje;,b7d77#b7 ( ! ,f$]al ,food1 ,drug1 
& ,cosmetic ,act 7#ba ,u4,s4,c4         
#cee;c7d77#b77 is am5d$ by /rik+ 7except
 t ! drug or biological product %all n  
be subject to action "u section #cjc74  
7b7 ,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  7c7
 ,transition rule ,! ,secret>y ( ,heal? 
& ,human ,s]vices %all take no en=cem5t 
actions "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 be=e ! date ?is is 
#ahj days aft] ! date ( enactm5t ( ?is  
,act4  #e4 ,,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$--  7#a7 9       
p>agraph 7#aa71 by /rik+ ! semicolon at 
! end & ins]t+ 1 includ+ an assessm5t ( 
! appropriat5ess ( def]rals & def]ral   
ext5sions grant$ = su* products & an    
evaluation ( compliance ) deadl9es      
provid$ = 9 su* 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
 = failure to comply ) requirem5ts "u   
section #eje;,b ( su* ,act1 includ+ =   
ea* p5alty1 settlem5t1 or paym5t ! "n ( 
! drug1 ! sponsor !re(1 & ! am\nt ( !   

p5alty1 settlem5t1 or paym5t impos$4  4