,,ii  #aahth ,,congress  #ast ,session  
,s4 #bhig  ,,in ,,the ,,senate ,,of     
,,the ,,united ,,states  ,september #ba1
 #bjbc,mr4 ,bennet 7for himself and ,mr4
 ,rubio7 introduced the following bill2 
which was read twice and referred to the
 ,committee on ,health1 ,education1     
,labor1 and ,pensions  ,a ,,bill  ,to   
amend the ,federal ,food1 ,drug1 and    
,cosmetic ,act with respect to          
molecularly targeted pediatric cancer   
investigations1 and for other purposes4 
#a4 ,short title ,this ,act may be cited
 as the ,give ,kids a ,chance ,act of   
#bjbc4  #b4 ,research into pediatric    
uses of drugs2 additional authorities of
 ,food and ,drug ,administration        
regarding molecularly targeted cancer   
drugs  7a7 ,in general  7#a7 ,additional
 active ingredient for application drug2
 limitation regarding novel-combination 
application drug ,section #eje;,b7a77#c7
 of the ,federal ,food1 ,drug1 and      
,cosmetic ,act 7#ba ,u4,s4,c4           
#cee;c7a77#c77 is amended-- 7,a7 by     

redesignating subparagraphs 7,b7 and    
7,c7 as subparagraphs 7,c7 and 7,d71    
respectively2 and  7,b7 by striking     
subparagraph 7,a7 and inserting the     
following3  7,a7 ,in general ,for       
purposes of paragraph 7#a77;,b71 the    
investigation described in this         
paragraph is 7as determined by the      
,secretary7 a molecularly targeted      
pediatric cancer investigation of-- 7i7 
the drug or biological product for which
 the application referred to in such    
paragraph is submitted2 or  7ii7 such   
drug or biological product in           
combination with-- 7,i7 an active       
ingredient of a drug or biological      
product-- 7aa7 for which an approved    
application under section #eje7;j7 under
 this ,act or under section #cea7k7 of  
the ,public ,health ,service ,act is in 
effect2 and  7bb7 that is determined by 
the ,secretary to be the standard of    
care for treating a pediatric cancer2 or
  7,,ii7 an active ingredient of a drug 
or biological product-- 7aa7 for which  

an approved application under section   
#eje7;b7 of this ,act or section        
#cea7;a7 of the ,public ,health ,service
 ,act to treat an adult cancer is in    
effect and is held by the same person   
submitting the application under        
paragraph 7#a77;,b72 and  7bb7 that is  
directed at a molecular target that the 
,secretary determines to be             
substantially relevant to the growth or 
progression of a pediatric cancer4  7,b7
 ,additional requirements  7i7 ,design  
of investigation ,a molecularly targeted
 pediatric cancer investigation referred
 to in subparagraph 7,a7 shall be       
designed to yield clinically meaningful 
pediatric study data that is gathered   
using appropriate formulations for each 
age group for which the study is        
required1 regarding dosing1 safety1 and 
preliminary efficacy to inform potential
 pediatric labeling4  7ii7 ,limitation  
,an investigation described in          
subparagraph 7,a77ii7 may be required   
only if the drug or biological product  

for which the application referred to in
 paragraph 7#a77;,b7 contains either--  
7,i7 a single new active ingredient2 or 
7,,ii7 more than one active ingredient1 
if an application for the combination of
 active ingredients has not previously  
been approved but each active ingredient
 has been previously approved to treat  
an adult cancer4  7iii7 ,results of     
already-completed preclinical studies of
 application drug ,the ,secretary may   
require that reports on an investigation
 required pursuant to paragraph         
7#a77;,b7 include the results of all    
preclinical studies on which the        
decision to conduct such investigation  
was based4  7iv7 ,rule of construction  
regarding inactive ingredients ,with    
respect to a combination of active      
ingredients referred to in subparagraph 
7,a77ii71 such subparagraph shall not be
 construed as addressing the use of     
inactive ingredients with such          
combination4  4  7#b7 ,determination of 
applicable requirements ,section        

#eje;,b7e77#a7 of the ,federal ,food1   
,drug1 and ,cosmetic ,act 7#ba ,u4,s4,c4
 #cee;c7e77#a77 is amended by adding at 
the end the following3 ,the ,secretary  
shall determine whether subparagraph    
7,a7 or 7,b7 of subsection 7a77#a7 shall
 apply with respect to an application   
before the date on which the applicant  
is required to submit the initial       
pediatric study plan under paragraph    
7#b77;,a744  7#c7 ,clarifying           
applicability ,section #eje;,b7a77#a7 of
 the ,federal ,food1 ,drug1 and         
,cosmetic ,act 7#ba ,u4,s4,c4           
#cee;c7a77#a77 is amended by adding at  
the end the following3  7,c7 ,rule of   
construction ,no application that is    
subject to the requirements of          
subparagraph 7,b7 shall be subject to   
the requirements of subparagraph 7,a71  
and no application 7or supplement to an 
application7 that is subject to the     
requirements of subparagraph 7,a7 shall 
be subject to the requirements of       
subparagraph 7,b74  4  7#d7 ,conforming 

amendments ,section #eje;,b7a7 of the   
,federal ,food1 ,drug1 and ,cosmetic    
,act 7#ba ,u4,s4,c4 #cee;c7a77 is       
amended-- 7,a7 in paragraph 7#c77;,c71  
as redesignated by paragraph 7#a77;,a7  
of this subsection1 by striking         
investigations described in this        
paragraph and inserting investigations  
referred to in subparagraph 7,a72 and   
7,b7 in paragraph 7#c77;,d71 as         
redesignated by paragraph 7#a77;,a7 of  
this subsection1 by striking the        
assessments under paragraph 7#b77;,b7   
and inserting the assessments required  
under paragraph 7#a77;,a74  7b7         
,guidance ,the ,secretary shall-- 7#a7  
not later than #a year after the date of
 enactment of this ,act1 issue draft    
guidance on the implementation of the   
requirements in subsection 7a72 and     
7#b7 not later than #a year after       
closing the comment period on such draft
 guidance1 finalize such guidance4  7c7 
,applicability ,the amendments made by  
this section apply with respect to any  

application under section #eje7;b7 of   
the ,federal ,food1 ,drug1 and ,cosmetic
 ,act 7#ba ,u4,s4,c4 #cee7;b77 and any  
application under section #cea7;a7 of   
the ,public ,health ,service ,act 7#db  
,u4,s4,c4 #bfb7;a771 that is submitted  
on or after the date that is #c years   
after the date of enactment of this     
,act4  7d7 ,reports to ,congress  7#a7  
,secretary of ,health and ,human        
,services ,not later than #b years after
 the date of enactment of this ,act1 the
 ,secretary of ,health and ,human       
,services shall submit to the ,committee
 on ,energy and ,commerce of the ,house 
of ,representatives and the ,committee  
on ,health1 ,education1 ,labor1 and     
,pensions of the ,senate a report on the
 ,secretary's efforts1 in coordination  
with industry1 to ensure implementation 
of the amendments made by subsection    
7a74  7#b7 ,,gao study and report  7,a7 
,study ,not later than #c years after   
the date of enactment of this ,act1 the 
,comptroller ,general of the ,united    

,states shall conduct a study of the    
effectiveness of requiring assessments  
and investigations described in section 
#eje;,b of the ,federal ,food1 ,drug1   
and ,cosmetic ,act 7#ba ,u4,s4,c4       
#cee;c71 as amended by subsection 7a71  
in the development of drugs and         
biological products for pediatric cancer
 indications4  7,b7 ,findings ,not later
 than #g years after the date of        
enactment of this ,act1 the ,comptroller
 ,general shall submit to the ,committee
 on ,energy and ,commerce of the ,house 
of ,representatives and the ,committee  
on ,health1 ,education1 ,labor1 and     
,pensions of the ,senate a report       
containing the findings of the study    
conducted under subparagraph 7,a74