,,ii  #aaith ,,congress  #ast ,session  
,s4 #ceea  ,,in ,,the ,,senate ,,of     
,,the ,,united ,,states  ,december #ag1 
#bjbe,mr4 ,tillis 7for himself and ,mr4 
,heinrich7 introduced the following     
bill2 which was read twice and referred 
to the ,committee on ,finance  ,a ,,bill
  ,to amend titles ,,xviii and ,,xix of 
the ,social ,security ,act and title    
,,xxvii of the ,public ,health ,service 
,act to provide for coverage of certain 
drugs used in the treatment or          
management of a rare disease or         
condition1 and for other purposes4  #a4 
,short title ,this ,act may be cited as 
the ,providing ,realistic ,opportunity  
,to ,equal and ,comparable ,treatment   
for ,rare ,act or the ,,protect for     
,rare ,act4  #b4 ,coverage of certain   
drugs used in treatment or management of
 a rare disease or condition  7a7       
,medicare  7#a7 ,definition  7,a7 ,in   
general ,section #ahfa7t77#b7 of the    
,social ,security ,act 7#db ,u4,s4,c4   
#aciex7t77#b77 is amended-- 7i7 in      

subparagraph 7,a71 by inserting after   
regimen the following3 1 or used in the 
treatment or management of a disease or 
condition affecting #bjj1jjj or fewer   
individuals in the ,united ,states12 and
  7ii7 in subparagraph 7,b77ii7-- 7,i7  
in subclause 7,i71 by striking 1 or at  
the end and inserting a semicolon2      
7,,ii7 in subclause 7,,ii71 by striking 
the period at the end and inserting 2   
or2 and  7,,iii7 by adding at the end   
the following new subclause3  7,,iii7 in
 the case of a drug that is used in the 
treatment or management of a disease or 
condition affecting #bjj1jjj or fewer   
individuals in the ,united ,states1 such
 use is supported by peer-reviewed      
medical literature1 including clinical  
guidelines1 and is not specifically     
listed as not indicated in one or more  
of the compendia described in section   
#aibg7;g77#a77;,b77i71 or listed as     
contraindicated in the labeling approved
 by the ,food and ,drug ,administration4
  4  7,b7 ,effective date ,the          

amendments made by subparagraph 7,a7    
shall apply to items and services       
furnished on or after ,january #a1      
#bjbg44  7#b7 ,medically accepted uses  
of covered part ,d drugs in treating    
rare conditions  7,a7 ,in general       
,section #ahfj;,d-#b7;e77#d77;,a77i7 of 
the ,social ,security ,act 7#db         
,u4,s4,c4 #aciew-#ajd7;e77#d77;,a77i77  
is amended1 in the matter preceding     
subclause 7,i71 by inserting or in the  
case of a covered part ,d drug used in  
the treatment or management of a disease
 or condition affecting #bjj1jjj or     
fewer individuals in the ,united        
,states1 after regimen1  7,b7 ,effective
 date ,the amendment made by            
subparagraph 7,a7 shall apply to plan   
years beginning on or after ,january #a1
 #bjbg4  7b7 ,medicaid  7#a7 ,in general
 ,section #aibg7k77#f7 of the ,social   
,security ,act 7#db ,u4,s4,c4           
#acifr-#h7k77#f77 is amended to read as 
follows3  7#f7 ,medically accepted      
indication ,the term medically accepted 

indication means any use for a covered  
outpatient drug-- 7,a7 which is approved
 under the ,federal ,food1 ,drug1 and   
,cosmetic ,act2  7,b7 which is supported
 by one or more citations included or   
approved for inclusion in any of the    
compendia described in subsection       
7g77#a77;,b77i72 or  7,c7 which1 in the 
case of a drug used to treat or manage a
 disease or condition affecting #bjj1jjj
 or fewer individuals in the ,united    
,states-- 7i7 is a use of such drug that
 is supported by peer-reviewed medical  
literature1 clinical guidelines1 or an  
expert in such disease or condition1 as 
identified by a medical society involved
 in the treatment or management of such 
disease or condition2 and  7ii7 is a use
 of such drug that is not listed as not 
indicated in the compendia described in 
subsection 7g77#a77;,b77i71 or listed as
 contraindicated in the labeling        
approved by the ,food and ,drug         
,administration4  4  7#b7 ,conforming   
amendment ,section #aibg7;d77#d77;,c7 of

 the ,social ,security ,act 7#db        
,u4,s4,c4 #acifr-#h7;d77#d77;,c77 is    
amended by striking compendia and       
inserting sources4  7#c7 ,effective date
 ,the amendments made by this subsection
 shall apply with respect to covered    
outpatient drugs furnished on or after  
,january #a1 #bjbg4  7c7 ,private health
 insurance  7#a7 ,in general  7,a7      
,,phsa ,part ,d of title ,,xxvii of the 
,public ,health ,service ,act 7#db      
,u4,s4,c4 #cjj;gg-#aaa et seq47 is      
amended by adding at the end the        
following new section3  #bgii;,a-#aa4   
,expedited process for review associated
 with certain drugs used in treatment or
 management of a rare disease or        
condition ,a group health plan or a     
health insurance issuer offering group  
or individual health insurance coverage 
shall provide an expedited process      
pursuant to section #bgai by which a    
participant1 beneficiary1 or enrollee1  
or a designee or prescribing physician  
7or other prescriber1 as appropriate7 of

 the participant1 beneficiary1 or       
enrollee1 may appeal any denial of      
coverage for a drug or biological       
product-- 7#a7 approved under section   
#eje of the ,federal ,food1 ,drug1 and  
,cosmetic ,act or licensed under section
 #cea2  7#b7 for which the use is       
related to treatment or management of a 
disease or condition affecting #bjj1jjj 
or fewer individuals in the ,united     
,states2 and  7#c7 the use of which is  
supported by-- 7,a7 the labeling for the
 drug or biological product approved    
pursuant to such section #eje or #cea2  
or  7,b7 peer-reviewed literature1      
including clinical guidelines1 and that 
is not reviewed unfavorably in the      
compendia described in section          
#aibg7;g77#a77;,b77i7 of the ,social    
,security ,act and is not listed as a   
contraindication in such approved       
labeling4  4  7,b7 ,,erisa  7i7 ,in     
general ,subpart ,b of part #g of       
subtitle ,b of title ,i of the ,employee
 ,retirement ,income ,security ,act of  

#aigd 7#bi ,u4,s4,c4 #aahe et seq47 is  
amended by adding at the end the        
following new section3  #gcf4 ,expedited
 process for review associated with     
certain drugs used in treatment or      
management of a rare disease or         
condition ,a group health plan or a     
health insurance issuer offering group  
health insurance coverage shall provide 
an expedited process pursuant to section
 #bgai of the ,public ,health ,service  
,act 7#db ,u4,s4,c4 #cjj;gg-#ai7 by     
which a participant or beneficiary1 or a
 designee or prescribing physician 7or  
other prescriber1 as appropriate7 of the
 participant or beneficiary1 may appeal 
any denial of coverage for a drug or    
biological product-- 7#a7 approved under
 section #eje of the ,federal ,food1    
,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4
 #cee7 or licensed under section #cea of
 the ,public ,health ,service ,act 7#db 
,u4,s4,c4 #bfb72  7#b7 for which the use
 is related to treatment or management  
of a disease or condition affecting     

#bjj1jjj or fewer individuals in the    
,united ,states2 and  7#c7 the use of   
which is supported by-- 7,a7 the        
labeling for the drug or biological     
product approved pursuant to such       
section #eje or #cea2 or  7,b7          
peer-reviewed literature1 including     
clinical guidelines1 and that is not    
reviewed unfavorably in the compendia   
described in section                    
#aibg7;g77#a77;,b77i7 of the ,social    
,security ,act 7#db ,u4,s4,c4           
#acifr-#h7;g77#a77;,b77i77 and is not   
listed as a contraindication in such    
approved labeling4  4  7ii7 ,clerical   
amendment ,the table of contents in     
section #a of the ,employee ,retirement 
,income ,security ,act of #aigd 7#bi    
,u4,s4,c4 #ajja et seq47 is amended by  
inserting after the item relating to    
section #gbe the following new item3    
,sec4 #gbf4 ,expedited process for      
review associated with certain drugs    
used in treatment or management of a    
rare disease or condition4  4  7,c7     

,,irc  7i7 ,in general ,subchapter ,b of
 chapter #ajj of the ,internal ,revenue 
,code of #aihf is amended by adding at  
the end the following new section3      
#ihbf4 ,expedited process for review    
associated with certain drugs used in   
treatment or management of a rare       
disease or condition ,a group health    
plan shall provide an expedited process 
pursuant to section #bgai of the ,public
 ,health ,service ,act 7#db ,u4,s4,c4   
#cjj;gg-#ai7 by which a participant or  
beneficiary1 or a designee or           
prescribing physician 7or other         
prescriber1 as appropriate7 of the      
participant or beneficiary1 may appeal  
any denial of coverage for a drug or    
biological product-- 7#a7 approved under
 section #eje of the ,federal ,food1    
,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4
 #cee7 or licensed under section #cea of
 the ,public ,health ,service ,act 7#db 
,u4,s4,c4 #bfb72  7#b7 for which the use
 is related to treatment or management  
of a disease or condition affecting     

#bjj1jjj or fewer individuals in the    
,united ,states2 and  7#c7 the use of   
which is supported by-- 7,a7 the        
labeling for the drug or biological     
product approved pursuant to such       
section #eje or #cea2 or  7,b7          
peer-reviewed literature1 including     
clinical guidelines1 and that is not    
reviewed unfavorably in the compendia   
described in section                    
#aibg7;g77#a77;,b77i7 of the ,social    
,security ,act 7#db ,u4,s4,c4           
#acifr-#h7;g77#a77;,b77i77 and is not   
listed as a contraindication in such    
approved labeling4  4  7ii7 ,clerical   
amendment ,the table of sections for    
subchapter ,b of chapter #ajj of the    
,internal ,revenue ,code of #aihf is    
amended by adding at the end the        
following new item3 ,sec4 #ihbf4        
,expedited process for review associated
 with certain drugs used in treatment or
 management of a rare disease or        
condition44  7#b7 ,effective date ,the  
amendments made by this subsection shall

 apply with respect to plan years       
beginning on or after ,january #a1      
#bjbg4