,i  #aag? ,,congress  #b;d ,session  ,h4
 ,r4 #fhhh  ,,9 ,,! ,,h\se ,,(          
,,repres5tatives  ,m>* #a1 #bjbb ,mr4   
,tonko 7= himself & ,mr4 ,mc,kinley7    
introduc$ ! foll[+ bill2 :i* 0 ref]r$ to
 ! ,committee on ,en]gy & ,comm]ce  ,a  
,,bill  ,to am5d ! ,f$]al ,food1 ,drug1 
& ,cosmetic ,act to improve ! treatm5t (
 r>e diseases & conditions1 & = o!r     
purposes4  #a4 ,%ort title ,?is ,act may
 be cit$ z ! ,help+ ,exp]ts ,accel]ate  
,r>e ,treatm5ts ,act ( #bjbb4  #b4      
,improv+ ! treatm5t ( r>e diseases &    
conditions  7a7 ,annual report on orphan
 drug program ,sub*apt] ,b ( *apt] ,v ( 
! ,f$]al ,food1 ,drug1 & ,cosmetic ,act 
7#ba ,u4,s4,c4 #cfj;aa et seq47 is am5d$
 by add+ at ! end ! foll[+ new section3 
#ebi;,b4 ,annual report on orphan drug  
program  7a7 ,9 g5]al ,n lat] ?an ! end 
( ea* ( ! #d ye>s foll[+ ! date (       
enactm5t ( ! ,help+ ,exp]ts ,accel]ate  
,r>e ,treatm5ts ,act ( #bjbb1 !         
,secret>y %all submit to ! ,congress a  
report summ>iz+ ! activities ( ! ,food &

 ,drug ,adm9i/ration relat$ to designat+
 drugs "u section #ebf = a r>e disease  
or condition & approv+ su* drugs "u     
section #eje ( ?is ,act or lic5s+ su*   
drugs "u section #cea ( ! ,public ,heal?
 ,s]vice ,act1 includ+-- 7#a7 ! numb] ( 
applications = su* drugs "u section #eje
 ( ?is ,act & section #cea ( ! ,public  
,heal? ,s]vice ,act receiv$ by ! ,food &
 ,drug ,adm9i/ration1 ! numb] ( su*     
applications accept$ & reject$ = fil+1 &
 ! numb] ( su* applications p5d+1       
approv$1 & disapprov$ by ! ,food & ,drug
 ,adm9i/ration1 array$ by ! review      
division assign$ to ! application2 &    
7#b7 assess ! ext5t to :i* ! ,food &    
,drug ,adm9i/ration is consult+ )       
ext]nal exp]ts pursuant to section      
#efi7;a77#b7 on topics p]ta9+ to drugs =
 a r>e disease or condition1 includ+ h[ 
& :5 any su* consultation is occurr+4   
7b7 ,public availability ,! ,secret>y   
%all make ea* report "u subsection 7a7  
available to ! public1 includ+ by po/+ !
 report on ! website ( ! ,food & ,drug  

,adm9i/ration4  7c7 ,def9ition ,9 ?is   
section1 ! t]m r>e disease or condition 
means a disease or condition affect+    
few] ?an #bjj1jjj p]sons 9 ! ,unit$     
,/ates4  4  7b7 ,/udy on ,european      
,union safety & efficacy reviews ( drugs
 = r>e diseases & conditions  7#a7 ,9   
g5]al ,! ,comptroll] ,g5]al ( ! ,unit$  
,/ates %all ent] into a contract ) an   
appropriate entity to conduct a /udy on 
! ,european ,union process = evaluat+ ! 
safety & efficacy ( drugs = r>e diseases
 or conditions1 includ+--  7,a7 any     
flexibilities1 au?orities1 or me*anisms 
available 9 ! ,european ,union specific 
to r>e diseases or conditions2 &  7,b7  
consid]ation & use ( supplem5tal data   
submitt$ dur+ ! review process1 includ+ 
data associat$ ) op5 label ext5sion     
/udies & exp&$ access programs specific 
to r>e diseases or conditions4  7#b7    
,consultation ,! contract "u p>agraph   
7#a7 %all provide = consultation )      
relevant /akehold]s1 includ+-- 7,a7 r>e 
disease or condition pati5ts2 &  7,b7   

pati5t gr\ps t-- 7i7 repres5t r>e       
disease or condition pati5ts2 &  7ii7 h 
int]national pati5t outrea*4  7#c7      
,report ,! contract "u p>agraph 7#a7    
%all provide =--  7,a7 n lat] ?an #b    
ye>s aft] ! date ( enactm5t ( ?is ,act--
 7i7 ! completion ( ! /udy "u p>agraph  
7#a72 &  7ii7 ! submission ( a report on
 ! results ( su* /udy to ! ,congress2 & 
7,b7 ! inclusion 9 ! report "u          
subp>agraph 7,a77ii7 ( recomm5dations = 
*anges to ! processes & au?orities ( !  
,food & ,drug ,adm9i/ration to          
facilitate developm5t (1 & access to1   
treatm5ts = r>e diseases or conditions4 
7#d7 ,public availability ,! contract "u
 p>agraph 7#a7 %all provide = !         
appropriate entity ref]r$ to 9 p>agraph 
7#a7 to make ! report "u p>agraph 7#c7  
available to ! public1 includ+ by po/+ !
 report on ! website ( ! appropriate    
entity4  7c7 ,public meet+  7#a7 ,9     
g5]al ,n lat] ?an ,decemb] #ca1 #bjbc1 !
 ,secret>y ( ,heal? & ,human ,s]vices1  
act+ ?r\gh ! ,commission] ( ,food &     

,drugs1 %all conv5e "o or m public      
meet+s to solicit input f /akehold]s    
reg>d+ ! approa*es describ$ 9 p>agraph  
7#b74  7#b7 ,approa*es ,! public meet+  
or meet+s "u p>agraph 7#a7 %all address 
approa*es to increas+ & improv+         
engagem5t ) r>e disease or condition    
pati5ts1 gr\ps repres5t+ su* pati5ts1   
r>e disease or condition exp]ts1 &      
exp]ts on small population /udies1 9    
ord] to improve ! und]/&+ ) respect to  
r>e diseases or conditions (-- 7,a7     
pati5t burd52  7,b7 treatm5t options2 & 
7,c7 side effects ( treatm5ts1 includ+--
  7i7 comp>+ ! side effects ( treatm5ts2
 &  7ii7 und]/&+ ! risks ( side effects 
relative to ! heal? /atus ( ! pati5t & !
 progression ( ! disease or condition4  
7#c7 ,public docket ,! ,secret>y (      
,heal? & ,human ,s]vices %all e/abli% a 
public docket to receive writt5 comm5ts 
relat$ to ! approa*es address$ dur+ ea* 
public meet+ "u p>agraph 7#a74 ,su*     
public docket %all rema9 op5 = #fj days 
foll[+ ! date ( ea* su* public meet+4   

7#d7 ,reports ,n lat] ?an #ahj days aft]
 ea* public meet+ "u p>agraph 7#a71 !   
,commission] ( ,food & ,drugs %all      
develop & publi% on ! website ( ! ,food 
& ,drug ,adm9i/ration a report on--     
7,a7 ! approa*es discuss$ at ! public   
meet+2 &  7,b7 any relat$               
recomm5dations4  7d7 ,consultation on ! 
sci5ce ( small population /udies        
,section #efi7;a77#b7 ( ! ,f$]al ,food1 
,drug1 & ,cosmetic ,act 7#ba ,u4,s4,c4  
#cfj;bbb-#h7;b77 is am5d$ by add+ at !  
end ! foll[+3  7,c7 ,small population   
/udies ,! ext]nal exp]ts on ! li/       
ma9ta9$ pursuant to subp>agraph 7,a7 may
 include exp]ts on ! sci5ce ( small     
population /udies4  4  7e7 ,/udy on     
suffici5cy & use ( ,,fda me*anisms =    
incorporat+ ! pati5t & cl9ician         
p]spective 9 ,,fda processes relat$ to  
applications conc]n+ drugs = r>e        
diseases or conditions  7#a7 ,9 g5]al ,!
 ,comptroll] ,g5]al ( ! ,unit$ ,/ates   
%all conduct a /udy on ! use ( ,food &  
,drug ,adm9i/ration me*anisms & tools to

 ensure t pati5t & physician p]spectives
 are consid]$ & incorporat$ ?r\gh\t !   
processes ( ! ,food & ,drug             
,adm9i/ration-- 7,a7 = approv+ or lic5s+
 "u section #eje ( ! ,f$]al ,food1      
,drug1 or ,cosmetic ,act 7#ba ,u4,s4,c4 
#cee7 or section #cea ( ! ,public ,heal?
 ,s]vice ,act 7#db ,u4,s4,c4 #bfb7 a    
drug designat$ z a drug = a r>e disease 
or condition "u section #ebf ( ! ,f$]al 
,food1 ,drug1 & ,cosmetic ,act 7#ba     
,u4,s4,c4 #cfj;bb72 &  7,b7 9 mak+ any  
det]m9ation relat$ to su* a drug's      
approval1 includ+ assessm5t ( ! drug's--
 7i7 safety or effectiv5ess2 or  7ii7   
po/approval safety monitor+4  7#b7      
,topics ,! /udy "u p>agraph 7#a7 %all-- 
7,a7 id5tify & comp>e ! processes t !   
,food & ,drug ,adm9i/ration has =mally  
put 9 place & utiliz$ to ga!r ext]nal   
exp]tise 7includ+ pati5ts1 pati5t gr\ps1
 & physicians7 on specific applications 
= diseases or conditions affect+ #bj1jjj
 or few] pati5ts 9 ! ,unit$ ,/ates &    
specific applications = diseases or     

conditions affect+ #bjj1jjj or few]     
pati5ts 9 ! ,unit$ ,/ates2  7,b7 exam9e 
tools or me*anisms to improve ef=ts &   
initiatives ( ! ,food & ,drug           
,adm9i/ration to collect & consid] su*  
ext]nal exp]tise ) respect to           
applications = diseases or conditions   
affect+ #bj1jjj or few] pati5ts 9 !     
,unit$ ,/ates comp>$ to applications =  
diseases or conditions affect+ #bjj1jjj 
or few] pati5ts 9 ! ,unit$ ,/ates       
?r\gh\t ! application review & approval 
or lic5sure processes1 includ+ )9       
int]nal b5efit-risk assessm5ts1 advisory
 committee processes1 & po/approval     
safety monitor+2 &  7,c7 exam9e         
processes or alt]natives to address or  
resolve conflicts ( int]e/ t imp$e !    
,food & ,drug ,adm9i/ration 9 ga9+      
ext]nal exp]t input on r>e diseases or  
conditions ) a limit$ set ( cl9ical &   
rese>* exp]ts4  7#c7 ,report ,n lat] ?an
 #b ye>s aft] ! date ( enactm5t ( ?is   
,act1 ! ,comptroll] ,g5]al ( ! ,unit$   
,/ates %all--  7,a7 complete ! /udy "u  

p>agraph 7#a72  7,b7 submit a report on 
! results ( su* /udy to ! ,congress2 &  
7,c7 include 9 su* report               
recomm5dations1 if appropriate1 = *anges
 to ! processes & au?orities ( ! ,food &
 ,drug ,adm9i/ration to improve !       
collection & consid]ation ( ext]nal     
exp]t op9ions ( pati5ts1 pati5t gr\ps1 &
 physicians ) exp]tise 9 r>e diseases or
 conditions1 includ+ any specific       
recomm5dations = diseases or conditions 
affect+ #bj1jjj or few] pati5ts 9 !     
,unit$ ,/ates4  7f7 ,def9ition ,9 ?is   
section1 ! t]m r>e disease or condition 
means a disease or condition affect+    
few] ?an #bjj1jjj p]sons 9 ! ,unit$     
,/ates4