,i  #aaf? ,,congress  #b;d ,session  ,h4
 ,r4 #gefg  ,,9 ,,! ,,h\se ,,(          
,,repres5tatives  ,july #i1 #bjbj ,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 ( #bjbj4  #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 #a ye>
 aft] ! date ( enactm5t ( ! ,help+      
,exp]ts ,accel]ate ,r>e ,treatm5ts ,act 
( #bjbj1 & n less ?an annually !reaft]1 
! ,secret>y %all submit to ! ,congress a
 report summ>iz+ ! activities ( ! ,food 

& ,drug ,adm9i/ration = 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 sections    
#eje ( ?is ,act & section #cea ( !      
,public ,heal? ,s]vice ,act receiv$ by !
 ,food & ,drug ,adm9i/ration1 ! numb] ( 
su* applications consid]$ by ea* ,food &
 ,drug ,adm9i/ration division1 & ! numb]
 ( su* applications p5d+ & approv$2     
7#b7 ! numb] ( applications describ$ 9  
p>agraph 7#a7 disaggregat$ by-- 7,a7 !  
disease or condition to be address$2 &  
7,b7 ! t>get subpopulations id5tifi$ 9  
su* applications2  7#c7 ! preval5ce 9 ! 
,unit$ ,/ates ( ea* disease or condition
 address$ by an application describ$ 9  
p>agraph 7#a72  7#d7 integration ( ,food
 & ,drug ,adm9i/ration /aff ) exp]tise 9
 r>e diseases & conditions 9 ea* review 
( an application describ$ 9 p>agraph    
7#a72  7#e7 id5tification ( all ext]nal 

exp]ts ) exp]tise 9 r>e diseases &      
conditions :o are consult$ 9 connection 
)1 or :o o!rwise p>ticipate 91 ea*      
review ( an application describ$ 9      
p>agraph 7#a72 &  7#f7 ! /atus ( ea*    
application describ$ 9 p>agraph 7#a74   
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  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 conduct a
 /udy on ,european ,union safety &      
efficacy reviews ( drugs = r>e diseases 
& conditions1 includ+-- 7,a7 any        
diff]5tial preval5ce-bas$ me*anisms2 &  
7,b7 consid]ation & use ( supplem5tal   
data submitt$ dur+ ! review process1    
includ+ data associat$ ) op5 label      
ext5sion /udies & exp&$ access programs4
  7#b7 ,report ,n lat] ?an #a ye> aft] !
 date ( enactm5t ( ?is ,act1 !          

,comptroll] ,g5]al %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 
recomm5dations = *anges to ! processes &
 au?orities ( ! ,food & ,drug           
,adm9i/ration to facilitate developm5t  
(1 & access to1 treatm5ts = r>e diseases
 & conditions4  7#c7 ,public            
availability ,! ,comptroll] ,g5]al ( !  
,unit$ ,/ates %all make ea* report "u   
p>agraph 7#b7 available to ! public1    
includ+ by po/+ ! report on ! website ( 
! ,gov]nm5t ,acc\ntability ,(fice4  7c7 
,review process ,subp>agraph 7,a7 (     
section #efi7;a77#b7 ( ! ,f$]al ,food1  
,drug1 & ,cosmetic ,act 7#ba ,u4,s4,c4  
#cfj;bbb-#h7;a77#b77 is am5d$ to read z 
foll[s3  7,a7 ,9 g5]al  7i7 ,li/ ,!     
,secret>y %all develop & ma9ta9 a li/ ( 
ext]nal exp]ts :o1 because ( _! special 
exp]tise1 are qualifi$ to provide advice
 on r>e disease & condition issues1     
includ+ topics describ$ 9 subsection    
7b74  7ii7 ,availability at meet+s ,!   

,secret>y %all ensure availability1 =   
consultation purposes1 ( an ext]nal     
exp]t on r>e diseases1 z describ$ 9     
subp>agraph 7,b71 9 connection ) ea*    
drug product advisory committee meet+   
conc]n+ a drug or biological product = a
 r>e disease or condition4  7iii7       
,review ( applications ,! ,secret>y %all
 ensure t ea* review ( an application   
submitt$ "u section #eje ( ?is ,act or  
section #cea ( ! ,public ,heal? ,s]vice 
,act = an indication associat$ ) a r>e  
disease or condition includes ,food &   
,drug ,adm9i/ration /aff ) exp]tise on  
r>e diseases & conditions z an integral 
"p ( ! review team4  4  7d7 ,risk       
evaluation & mitigation /rategy elem5ts 
,subsection 7a7 ( section #eje-a ( !    
,f$]al ,food1 ,drug1 & ,cosmetic ,act   
7#ba ,u4,s4,c4 #cee-a7 is am5d$ by add+ 
at ! end ! foll[+ new p>agraph3  7#e7   
,orphan drugs ,9 mak+ any det]m9ation   
reg>d+ ! inclusion1 9 ! risk evaluation 
& mitigation /rategy = a drug t has be5 
designat$ z a drug = a r>e disease or   

condition pursuant to section #ebf1 ( an
 elem5t t requires pati5t action1       
re/rictions on use ( ! drug1 or pati5t  
p>ticipation1 ! ,secret>y %all consult )
 pati5ts impact$ by ! r>e disease or    
condition 9 consid]+ ! burd5 (          
p>ticipation & likely pati5t compliance4
  4