,,ii  #aadth ,,congress  #ast ,session  
,s4 #bjcj  ,,in ,,the ,,senate ,,of     
,,the ,,united ,,states  ,september #ae1
 #bjae ,mr4 ,bennet 7for himself1 ,mr4  
,burr1 ,ms4 ,warren1 and ,mr4 ,hatch7   
introduced the following bill2 which was
 read twice and referred to the         
,committee on ,health1 ,education1      
,labor1 and ,pensions  ,a ,,bill  ,to   
allow the sponsor of an application for 
the approval of a targeted drug to rely 
upon data and information with respect  
to such sponsor's previously approved   
targeted drugs4  #a4 ,short title  ,this
 ,act may be cited as the ,advancing    
,targeted ,therapies for ,rare ,diseases
 ,act of #bjae4  #b4 ,targeted drugs for
 rare diseases ,title ,v of the ,federal
 ,food1 ,drug1 and ,cosmetic ,act 7#ba  
,u4,s4,c4 #cea et seq47 is amended by   
inserting after section #ejf;,f the     
following3  #ejf;,g4 ,targeted drugs for
 rare diseases  7a7 ,purpose ,the       
purpose of this section1 through the    
approach provided for in subsection 7b71

 is to-- 7#a7 facilitate the            
development1 review1 and approval of    
genetically targeted drugs to address an
 unmet medical need in one or more      
patient subgroups 7or gene variant      
subpopulations7 with respect to rare    
diseases or conditions that are serious 
or life-threatening2 and  7#b7 maximize 
the use of scientific tools or methods1 
including surrogate endpoints and other 
biomarkers for such purposes4  7b7      
,leveraging of data from previously     
approved drug application or            
applications ,the ,secretary may1       
consistent with applicable standards for
 approval under this ,act or section    
#cea of the ,public ,health ,service    
,act1 allow the sponsor of a genetically
 targeted drug to rely upon data and    
information--  7#a7 previously developed
 by the same sponsor 7or another sponsor
 that has provided the sponsor with a   
contractual right of reference to such  
data and information72 and  7#b7        
submitted by a sponsor described in     

paragraph 7#a7 in support of one or more
 applications previously approved under 
this ,act or section #cea of the ,public
 ,health ,service ,act1  for a drug that
 incorporates or utilizes the same or   
similar genetically targeted technology1
 or the same variant protein targeted   
technology1 as the drug or drugs that   
are the subject of an application or    
applications described in paragraph     
7#b74  7c7 ,definitions ,for purposes of
 this section-- 7#a7 the term           
genetically targeted drug means a drug  
which--  7,a7 is the subject of an      
application under section #eje7;b77#a7  
of this ,act or section #cea7;a7 of the 
,public ,health ,service ,act for the   
treatment of a rare disease or condition
 7as such term is defined in section    
#ebf7 that is serious or                
life-threatening2  7,b7 incorporates or 
utilizes a genetically targeted         
technology or a variant protein targeted
 technology2 and  7,c7 may result in the
 modulation 7including suppression1     

up-regulation1 or activation7 of the    
function of a gene or its associated    
gene product2  7#b7 the term genetically
 targeted technology means a technology 
comprising non-replicating nucleic acid 
or analogous compounds with a common or 
similar chemistry that is intended to   
treat one or more subsets of patients   
with the same disease1 including due to 
other variants in the same gene2 and    
7#c7 the term variant protein targeted  
technology means a technology or        
compound that modulates the function of 
a variant protein1 due to a gene        
variant1 intended to treat one or more  
subsets of patients with the same       
disease1 due to other variants in the   
same gene4  7d7 ,rule of construction   
,nothing in this section shall be       
construed to-- 7#a7 alter the authority 
of the ,secretary to approve drugs      
pursuant to this ,act or section #cea of
 the ,public ,health ,service ,act 7as  
authorized prior to the date of         
enactment of the ,advancing ,targeted   

,therapies for ,rare ,diseases ,act of  
#bjae71 including the standards of      
evidence1 and applicable conditions1 for
 approval under such ,act2 or  7#b7     
confer any new rights1 beyond those     
authorized under this section1 with     
respect to the permissibility of        
referencing information contained in    
another application submitted under     
section #eje7;b77#a7 of this ,act or    
section #cea7;a7 of the ,public ,health 
,service ,act4  4