[Congressional Record Volume 162, Number 115 (Friday, July 15, 2016)]
[Extensions of Remarks]
[Pages E1139-E1140]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




  RECOGNIZING THE IMPORTANCE OF EXTENDING THE RARE PEDIATRIC DISEASE 
                    PRIORITY REVIEW VOUCHER PROGRAM

                                 ______
                                 

                          HON. JOSEPH CROWLEY

                              of new york

                    in the house of representatives

                        Thursday, July 14, 2016

  Mr. CROWLEY. Mr. Speaker, I want to take this opportunity today to 
urge my colleagues to not let the Rare Pediatric Disease Priority 
Review Voucher Program lapse. As the co-chair of the House 
Congressional Rare Disease Caucus, I know first-hand how successful 
this program has been for children and their families throughout the 
country and I am determined for it to continue. Unfortunately, the 
program expires September 30th, which could curb continued research and 
development in the areas of rare pediatric conditions.
  One condition that very much relies on the incentives of the Rare 
Pediatric Disease Priority Review Voucher Program is epidermolysis 
bullosa (``EB''), and those suffering from the condition are in 
desperate need of treatments and cures. EB is a rare, debilitating, and 
potentially fatal connective tissue disorder that manifests through 
severe

[[Page E1140]]

blisters on the skin, eyes, throat, and internal organs. Children with 
EB are often called ``butterfly children'' because their skin so 
fragile. As with most rare diseases, EB can vary in progression and 
severity among its various types and subtypes.
  The most severe form of EB, recessive dystrophic EB, puts children at 
especially high risk for infection. Typically, even a mild localized 
trauma can cause skin to fall off, leading to open sores that do not 
heal, which cause indescribable pain and agony. In addition to the risk 
of infection, other conditions associated with EB include cancer, heart 
failure, malnutrition, and foot and hand deformities.
  I have heard the heartbreaking stories of children with this form, 
such as Rafi Kopelan and Jackson Silver of New York City and Mikey 
Fullmer of Irvine, California. Imagine being eight years old, unable to 
play soccer or swim, covered in bandages, enduring regular painful 
vinegar and bleach baths, and fighting through the discomfort to eat 
and breathe. This has been a reality for kids like Rafi, Jackson, and 
Mikey.
  New York City is home to two of the three leading patient advocacy 
and research organizations representing the EB community--debra of 
America and the EB Research Partnership. I would like to recognize the 
tremendous impact both organizations, as well as the Los Angeles, 
California based EB Medical Research Foundation, are having on the 
community. Due to their efforts, several drug manufacturers are 
currently in the research and development phase for various treatments 
for EB--from topical creams to protein replacement therapies.
  The Rare Pediatric Disease Priority Review Voucher Program has been 
instrumental in creating an incentive for these companies to finally 
allocate resources to investigating potential solutions for children 
with EB beyond pain management and wound care. But more progress is 
needed. Further research is necessary, for example, to develop 
interventions against a particularly virulent form of squamous cell 
carcinoma in EB patients, which has a mortality rate approaching 90 
percent.
  Congress has long supported EB research funding through the National 
Institutes of Health, which has helped lay the foundation for these 
existing development programs. Reauthorizing the Rare Pediatric Disease 
Priority Review Voucher Program, however, is a critical way of 
providing the necessary incentives to ensure children like Rafi, 
Jackson, and Mikey will soon benefit from this important research in 
the form of FDA-approved therapies.
  We must not let this deadline slip and this important program expire, 
and I hope all my colleagues recognize the urgency of this issue.

                          ____________________