,,ii  #aag? ,,congress  #a/ ,session    
,s4 #fgj  ,,9 ,,! ,,s5ate ,,( ,,!       
,,unit$ ,,/ates  ,m>* #aj1 #bjba,ms4    
,klobu*> 7= h]self & ,mr4 ,wick]7       
introduc$ ! foll[+ bill2 :i* 0 read     
twice & ref]r$ to ! ,committee on       
,heal?1 ,education1 ,labor1 & ,p5sions  
,a ,,bill  ,to am5d ! ,f$]al ,food1     
,drug1 & ,cosmetic ,act to accel]ate    
developm5t ( !rapies across ! spectrum (
 r>e diseases & conditions & facilitate 
pati5t access to su* !rapies1 & = o!r   
purposes4  #a4 ,%ort title ,?is ,act may
 be cit$ z ! ,spe$+ ,!rapy ,access      
,today ,act ( #bjba4  #b4 ,table (      
cont5ts ,! table ( cont5ts ( ?is ,act is
 z foll[s3 ,sec4 #a4 ,%ort title4,sec4  
#b4 ,table ( cont5ts4,sec4 #c4 ,int]c5t]
 ,in/itute on ,r>e ,diseases &          
,conditions4,sec4 #d4 ,r>e ,disease &   
,condition ,drug ,advisory              
,committee4,sec4 #e4 ,grants & contracts
 = developm5t ( drugs = r>e diseases &  
conditions4  #c4 ,int]c5t] ,in/itute on 
,r>e ,diseases & ,conditions  7a7       

,e/abli%m5t requir$ ,! fir/ s5t5ce (    
section #ajad7;a7 ( ! ,f$]al ,food1     
,drug1 & ,cosmetic ,act 7#ba ,u4,s4,c4  
#cii;g7a77 is am5d$ by ins]t+ 1 at lea/ 
"o ( :i* %all be focus$ on r>e diseases 
& conditions be=e ! p]iod at ! end ( !  
s5t5ce4  7b7 ,tim+ ( e/abli%m5t         
,subsection 7c7 ( section #ajad ( !     
,f$]al ,food1 ,drug1 & ,cosmetic ,act   
7#ba ,u4,s4,c4 #cii;g7 is am5d$ to read 
z foll[s3  7c7 ,tim+ ,n lat] ?an ! date 
t is #a ye> aft] ! date ( enactm5t ( !  
,spe$+ ,!rapy ,access ,today ,act (     
#bjba1 ! ,secret>y %all e/abli%1 9      
accordance ) ?is section & section      
#ebi;,b1 an ,in/itute "u subsection 7a7 
focus$ on r>e diseases & conditions1 to 
be kn[n z ! ,int]c5t] ,in/itute on ,r>e 
,diseases & ,conditions4  4  7c7        
,responsibilities ,sub*apt] ,b ( *apt]  
,v ( ! ,f$]al ,food1 ,drug1 & ,cosmetic 
,act 7relat+ to drugs = r>e diseases or 
conditions7 is am5d$ by ins]t+ aft]     
section #ebi;,a ( su* ,act 7#ba         
,u4,s4,c4 #cfj;ff-#a7 ! foll[+ new      

section3  #ebi;,b4 ,int]c5t] ,in/itute  
on ,r>e ,diseases & ,conditions  7a7    
,responsibilities ,9 addition to c>ry+  
out activities li/$ 9 section #ajad7;a71
 ! ,int]c5t] ,in/itute on ,r>e ,diseases
 & ,conditions %all-- 7#a7 s]ve z !     
,food & ,drug ,adm9i/ration's coord9at+ 
(fice = engagem5t ) r>e disease &       
condition /akehold]s1 complem5t+ b n    
supplant+ engagem5t activities betwe5   
/akehold]s & ! review divisions2  7#b7  
build1 )9 ! ,food & ,drug ,adm9i/ration1
 k & und]/&+ associat$ ) ! review (     
m$ical products to treat r>e diseases & 
conditions1 includ+ advancem5ts 9 trial 
design1 /ati/ical analysis1 regulatory  
sci5ce1 product manufactur+1 & o!r      
topics z det]m9$ by ! ,secret>y2  7#c7  
implem5t cross-c5t] r>e disease &       
condition-focus$ meet+s & policy        
developm5t2  7#d7 coord9ate r>e disease 
& condition-specific regulatory sci5ce  
initiatives2  7#e7 facilitate /akehold] 
engagem5t to ! ext]nal community &      
int]national regulatory ag5cies on r>e  

disease & condition product developm5t2 
7#f7 e/abli% & implem5t ! ,accel]at+    
,lifesav+s ,!rapies 9 ,treat+ ,ultra-r>e
 ,disease ,entities ,program "u         
subsection 7b72 &  7#g7 e/abli% & c>ry  
out ! r>e disease & condition ?ird-p>ty 
payor program "u subsection 7d74  7b7   
,,altitude ,program  7#a7 ,9 g5]al ,!   
,int]c5t] ,in/itute %all e/abli% &      
implem5t a program1 to be kn[n z !      
,accel]at+ ,lifesav+s ,!rapies 9 ,treat+
 ,ultra-r>e ,disease ,entities ,program1
 to id5tify & make recomm5dations to    
address curr5t & em]g+ regulatory sci5ce
 & public policy *all5ges associat$ )   
develop+ m$ical products to treat r>e   
diseases or conditions 9 an individual  
or v small populations4  7#b7 ,issues ,!
 program "u p>agraph 7#a7 %all focus on 
issues includ+-- 7,a7 manufactur+ /&>ds 
= !rapies describ$ 9 su* p>agraph1      
includ+ 9 non-indu/ry sett+s2  7,b7     
trial designs & metrics2  7,c7          
regulatory flexibilities = abbreviat$   
toxicology /udies1 ov]lapp+ animal      

/udies1 & pati5t dos+2  7,d7 regulatory 
sci5ce1 *emi/ry1 manufactur+1 & o!r ne$s
 associat$ ) develop+ su* !rapies2 &    
7,e7 o!r issues z det]m9$ by !          
,secret>y4  7c7 ,proposals = am5d+      
labels  7#a7 ,/akehold] gr\p ,n lat] ?an
 #ahj days aft] ! date ( enactm5t ( ?is 
section1 ! ,int]c5t] ,in/itute %all     
conv5e a meet+ ( /akehold]s f ! r>e     
disease community1 includ+ pati5ts1     
c>egiv]s1 product manufactur]s1         
?ird-p>ty payors1 & o!rs1 to consid]    
pot5tial am5dm5ts to labels = m$ical    
products to treat r>e diseases or       
conditions approv$ pursuant to a pa?way 
"u section #ejf4  7#b7 ,guidance ,n lat]
 ?an #ij days aft] ! date ( ! meet+ "u  
p>agraph 7#a71 ! ,secret>y %all issue   
guidance to propose *anges to h[ !      
labels ( m$ical products to treat r>e   
diseases or conditions demon/rate       
cl9ical b5efits & reflect relevant      
sci5tific data includ+ surrogate        
endpo9ts4  7d7 ,r>e ,disease &          
,condition ,?ird-,p>ty ,payor ,program  

7#a7 ,9 g5]al ,! ,int]c5t] ,in/itute    
%all e/abli% & c>ry out a volunt>y r>e  
disease & condition e>ly ?ird-p>ty payor
 fe$back program-- 7,a7 to in=m cov]age 
policies = r>e disease !rapies2 &  7,b7 
to in=m cl9ical trial design1 pati5t    
engagem5t1 & o!r data collections4  7#b7
 ,program requirem5ts ,! program "u     
p>agraph 7#a7 %all-- 7,a7 facilitate    
volunt>y communication betwe5 sponsors (
 m$ical products to treat r>e diseases &
 conditions & ?ird-p>ty payors2 &  7,b7 
require p>ticipation ( ! ,c5t]s =       
,m$ic>e  ,m$icaid ,s]vices )            
repres5tation f-- 7i7 ! ,c5t] = ,m$ic>e2
 &  7ii7 ! ,c5t] = ,m$icaid & ,,*ip     
,s]vices4  7#c7 ,annual report ,!       
,int]c5t] ,in/itute %all-- 7,a7 on an   
annual basis1 submit a report to t      
,congress on-- 7i7 ! p>ticipation )9 !  
program "u p>agraph 7#a72 &  7ii7 !     
impacts ( ! program "u p>agraph 7#a72 & 
7,b7 po/ ea* su* report on ! public     
website ( ! ,int]c5t] ,in/itute4  7#d7  
,bullet9 to ,m$icaid directors ,foll[+ !

 approval1 cle>ance1 or au?orization by 
! ,food & ,drug ,adm9i/ration ( a m$ical
 product to treat a r>e disease or      
condition1 ! ,secret>y %all issue a     
bullet9 to ,/ate ,m$icaid directors     
conta9+ in=mation to help in=m cov]age  
decisions on ! product by ,/ate ,m$icaid
 & ,*ildr5's ,heal? ,insurance programs4
  7e7 ,def9ition ,9 ?is section1 ! t]ms 
,int]c5t] ,in/itute on ,r>e ,diseases & 
,conditions & ,int]c5t] ,in/itute ref]  
to ! ,int]c5t] ,in/itute on ,r>e        
,diseases & ,conditions e/abli%$        
pursuant to section #ajad4  4  #d4 ,r>e 
,disease & ,condition ,drug ,advisory   
,committee ,sub*apt] ,b ( *apt] ,v ( !  
,f$]al ,food1 ,drug1 & ,cosmetic ,act is
 fur!r am5d$ by ins]t+ aft] section     
#ebi;,b ( su* ,act1 z ins]t$ by section 
#c1 ! foll[+ new section3  #ebi;,c4 ,r>e
 ,disease & ,condition ,drug ,advisory  
,committee  7a7 ,9 g5]al ,! ,secret>y   
%all e/abli% & ma9ta9 a committee1 to be
 kn[n z ! ,r>e ,disease & ,condition    
,drug ,advisory ,committee 79 ?is       

section ref]r$ to z ! ,advisory         
,committee74  7b7 ,duty ( ,committee ,! 
,advisory ,committee %all advise !      
,secret>y on issues associat$ )         
developm5t ( !rapies to treat r>e       
diseases or conditions4  7c7 ,specific  
issues ,9 advis+ ! ,secret>y1 !         
,advisory ,committee may address issues 
includ+-- 7#a7 modifi$ or new regulatory
 pa?ways to support review ( !rapies2   
7#b7 cl9ical trial design ne$s1 includ+ 
developm5t ( innovative approa*es to    
cl9ical trials2  7#c7 qualifications (  
biom>k]s or o!r drug developm5t tools = 
use 9 reviews2  7#d7 modifi$ or new     
/&>ds to support ! review ( already     
m>ket$ drugs be+ evaluat$ = repurpos+ to
 treat a r>e disease or condition2 &    
7#e7 issues-- 7,a7 t p]ta9 to an        
application = approval ( a !rapy to     
treat a r>e disease or condition2 &     
7,b7 ) respect to :i* a review division 
has reque/$ t ! ,advisory ,committee    
provide advice4  7d7 ,memb]%ip  7#a7 ,9 
g5]al ,! ,advisory ,committee %all      

consi/ (-- 7,a7 n m ?an #ae memb]s      
appo9t$ by ! ,secret>y 9 accordance )   
p>agraph 7#b72 &  7,b7 ! nonvot+ ex     
(ficio memb]s "u p>agraph 7#c74  7#b7   
,appo9t$ memb]s  7,a7 ,special ,gov]nm5t
 employees ,memb]s ( ! ,advisory        
,committee appo9t$ pursuant to p>agraph 
7#a77;,a7 %all s]ve z special ,gov]nm5t 
employees 7z def9$ 9 section #bjb7;a7 ( 
title #ah1 ,unit$ ,/ates ,code74  7,b7  
,eligibility ,to be eligible = appo9tm5t
 pursuant to p>agraph 7#a77;,a71 an     
individual %all-- 7i7 be eligible to    
s]ve z special ,gov]nm5t employee 7z    
def9$ 9 section #bjb7;a7 ( title #ah1   
,unit$ ,/ates ,code72 &  7ii7 h exp]tise
 9 ! fields ( public policy1 law1       
regulatory policy1 economics1           
pati5t-focus$ product developm5t1 or    
pati5t advocacy4  7,c7 ,composition ,( !
 memb]s ( ! ,advisory ,committee appo9t$
 pursuant to p>agraph 7#a77;,a7-- 7i7 up
 to #aj %all be select$ f among exp]ts 9
 ! discipl9es relevant to ! activities (
 ! ,int]c5t] ,in/itute on ,r>e ,diseases

 & ,conditions1 to include at lea/ "o   
exp]t 9 ea* (-- 7,i7 r>e disease product
 developm5t2  7,,ii7 conduct+ cl9ical   
trials ) respect to r>e diseases &      
conditions1 includ+ ) respect to v small
 pati5t populations2  7,,iii7 r>e       
disease & condition natural hi/ory &    
relat$ /udies2  7,,iv7 heal? economics  
p]ta9+ to ! developm5t ( m$ical products
 = r>e diseases or conditions2  7,v7    
manufactur+ & relat$ ne$s associat$ )   
m$ical products = r>e diseases or       
conditions2 &  7,,vi7 pati5t exp]i5ce   
data collection2 &  7ii7 up to #e %all  
be select$ f ! public1 to include-- 7,i7
 at lea/ #d individuals :o are          
repres5tatives ( ! r>e disease pati5t   
community2  7,,ii7 at lea/ "o individual
 :o is directly impact$ by a r>e disease
 or condition2 &  7,,iii7 at lea/ "o    
p]son :o s]ves z a family c>egiv] to a  
p]son diagnos$ ) a r>e disease or       
condition4  7#c7 ,nonvot+ ex (ficio     
memb]s ,! nonvot+ ex (ficio memb]s ( !  
,advisory ,committee "u p>agraph        

7#a77;,b7 %all consi/ ( ! foll[+3 7,a7  
,! ,secret>y 7or ! ,secret>y's          
designee74  7,b7 ,! ,director ( !       
,int]c5t] ,in/itute on ,r>e ,diseases & 
,conditions4  7,c7 ,! ,director ( !     
,c5t] = ,biologics ,evaluation & ,rese>*
 7or ! ,director's designee74  7,d7 ,!  
,director ( ! ,c5t] = ,drug ,evaluation 
& ,rese>* 7or ! ,director's designee74  
7,e7 ,! ,director ( ! ,c5t] = ,devices &
 ,radiological ,heal? 7or ! ,director's 
designee74  7,f7 ,! ,director ( !       
,national ,c5t] = ! ,advanc+            
,translational ,sci5ces ( ! ,national   
,in/itutes ( ,heal? 7or ! ,director's   
designee74  7,g7 ,! ,adm9i/rator ( !    
,c5t]s = ,m$ic>e  ,m$icaid ,s]vices 7or 
! ,adm9i/rator's designee74  7,h7 ,any  
additional (fic]s or employees ( !      
,dep>tm5t ( ,heal? & ,human ,s]vices z !
 ,secret>y det]m9es necess>y = !        
,advisory ,committee to effectively c>ry
 out its functions4  7#d7 ,*air ,! ,*air
 ( ! ,advisory ,committee %all be !     
,director ( ! ,int]c5t] ,in/itute = ,r>e

 ,diseases & ,conditions4  7#e7 ,t]ms   
7,a7 ,memb]s  7i7 ,9 g5]al ,! t]m ( a   
memb] ( ! ,advisory ,committee appo9t$  
pursuant to p>agraph 7#a77;,a7 %all be  
#d ye>s1 except t any memb] appo9t$ to  
fill a vacancy 9 an unexpir$ t]m %all be
 appo9t$ = ! rema9d] ( t t]m4  7ii7     
,cont9u$ s]vice ,a memb] appo9t$        
pursuant to p>agraph 7#a77;,a7 may      
cont9ue s]v+ z a memb] ( ! ,advisory    
,committee = up to #ahj days aft] !     
expiration ( t memb]'s t]m if a         
successor has n be5 appo9t$4  7,b7      
,reappo9tm5t ,a memb] ( ! ,advisory     
,committee :o has be5 appo9t$ pursuant  
to p>agraph 7#a77;,a7 = a t]m ( #d ye>s 
may n be reappo9t$ to s]ve z a memb] ( !
 ,advisory ,committee be=e ! date t is  
#b ye>s aft] ! date ( expiration ( t    
memb]'s t]m4  7e7 ,quorum ,a majority ( 
! appo9t$ memb]s ( ! ,advisory          
,committee %all con/itute a quorum = !  
conduct ( bus9ess4  4  #e4 ,grants &    
contracts = developm5t ( drugs = r>e    
diseases & conditions  7a7 ,au?ority (  

,secret>y ,section #e7;a7 ( ! ,orphan   
,drug ,act 7#ba ,u4,s4,c4 #cfj;ee7a77 is
 am5d$-- 7#a7 9 p>agraph 7#b71 by /rik+ 
& at ! end2 &  7#b7 by ins]t+ be=e !    
p]iod at ! end 1 & 7#d7 develop+        
practices p]ta9+ to ! *emi/ry1          
manufactur+1 regulatory approval (1 &   
controls ( individualiz$ !rapies or     
!rapies to treat v small populations4   
7b7 ,,altitude ,program ,9 support+     
grants & contracts "u section #e7;a77#d7
 ( ! ,orphan ,drug ,act1 z add$ by      
subsection 7a71 ! ,secret>y ( ,heal? &  
,human ,s]vices %all consult ) !        
,director ( ! ,int]c5t] ,in/itute on    
,r>e ,diseases & ,conditions reg>d+ !   
,accel]at+ ,lifesav+s ,!rapies 9 ,treat+
 ,ultra-r>e ,disease ,entities ,program 
e/abli%$ "u section #ebi;,b7b7 ( !      
,f$]al ,food1 ,drug1 & ,cosmetic ,act1 z
 add$ by section #c7;c7 ( ?is ,act1 to--
 7#a7 id5tify ! regulatory sci5ce &     
relat$ *all5ges & ne$s associat$ )      
develop+ individualiz$ !rapies or       
!rapies to treat v small pati5t         

populations2 &  7#b7 support rese>* to  
address su* *all5ges4