[Congressional Record Volume 162, Number 114 (Thursday, July 14, 2016)]
[Extensions of Remarks]
[Page E1133]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




URGING IMMEDIATE REAUTHORIZATION OF THE RARE PEDIATRIC DISEASE PRIORITY 
                         REVIEW VOUCHER PROGRAM

                                 ______
                                 

                         HON. MICHAEL T. McCAUL

                                of texas

                    in the house of representatives

                        Thursday, July 14, 2016

  Mr. McCAUL. Mr. Speaker, I rise today to urge immediate 
reauthorization of the Rare Pediatric Disease Priority Review Voucher 
Program before it expires at the end of this fiscal year. Simply put, 
this program has proven to save the lives of children. My colleague and 
dear friend from North Carolina, Mr. Butterfield, and I have introduced 
legislation that would make it permanent. Last July, thanks to the 
leadership of Committee on Energy and Commerce Chairman Fred Upton and 
his staff and advocates like Nancy Goodman, who is the founder and 
Executive Director of Kids v. Cancer, this Chamber passed an amended 
version of this initiative as part of H.R. 6, the 21st Century Cures 
Act. This program is a crucial incentive--in addition to the Orphan 
Drug Act--for drug manufacturers to make the significant investment in 
developing therapies for rare pediatric diseases, including pediatric 
cancers and lysosomal storage disorders.
   When we were considering H.R. 6 last July and again in recognition 
of National Neuroblastoma Awareness Day last September, I came to this 
Floor to speak about the positive impact the Rare Pediatric Disease 
Priority Review Voucher Program has had on children with neuroblastoma. 
Because of the limited market incentives available prior to the 
creation of the program, biopharmaceutical companies had been unwilling 
to risk investing in research and development for a therapy that treats 
this extremely rare and devastating pediatric cancer. The Rare 
Pediatric Disease Priority Review Voucher Program proved to be the 
necessary incentive for United Therapeutics to satisfy this unmet need 
with the development of Unituxin. In March 2015, this therapy received 
Food and Drug Administration (``FDA'') approval for children with high-
risk neuroblastoma. Unituxin was the first drug that FDA approved for 
this condition and only the second FDA-approved therapy for pediatric 
cancer. Upon its approval, FDA awarded Unituxin the second voucher from 
this program.
   As the chairman of the Childhood Cancer Caucus, I recognize the 
importance of this therapy to the community, especially to children 
like four-year-old Rex Ryan from Austin in the 10th District of Texas. 
Diagnosed with high-risk neuroblastoma at 17 months old, Rex's parents 
Leslie and Casey enrolled him in the clinical trial for Unituxin at 
Children's Blood and Cancer Center at Dell Children's Medical Center of 
Central Texas in Austin. Rex is a neuroblastoma survivor because of 
this new drug, which would not have been developed without the voucher 
program. After witnessing the direct impact Unituxin has had on Rex and 
his parents, it would be unconscionable for Congress to allow this 
program to expire. As I have previously described, the hope afforded by 
the Rare Pediatric Disease Priority Review Voucher Program extends to 
other conditions as well.
   Nearly two years ago, I came to this Floor to speak about the value 
of the program just after BioMarin Pharmaceuticals sold the first 
voucher from this program for $67.5 million, which the company 
immediately reinvested to build a clinical laboratory on its campus in 
San Rafael, California. The laboratory is a critical component of its 
development of a gene therapy platform for hemophilia A, which is 
showing promising early data in eight patients. BioMarin received this 
first voucher for developing a therapy for Morquio A syndrome, which is 
also known as mucopolysaccharidosis (``MPS'') type IV. Unfortunately 
for the millions of children affected by one of the nearly 7,000 rare 
diseases without a treatment, such as several of the other types and 
subtypes of MPS, including Sanfilippo syndrome and Sly syndrome, 
politics have hampered Senate negotiations on its larger innovation 
package, which is putting the program in jeopardy.
   Patients like eight-year-old Beckett Weldon, who is from Cypress in 
the 10th District of Texas, cannot afford for Congress to allow this 
program to lapse. Beckett suffers from SYNGAP1-related intellectual 
disability, which is an ultra-rare and severely disabling, genetic 
neurological disorder. Approximately two-thirds of the children with 
this condition have some form of epilepsy. Other symptoms include 
hypotonia, behavioral disorders, language impairment, and oral 
dyspraxia. Beckett began his four-year diagnostic odyssey at 4 months 
old, when he began missing milestones his twin sister Pyper was 
meeting. After visiting 19 specialists and several diagnostic tests, 
Beckett received his diagnosis.
   Only recently identified, SYNGAP1-related intellectual disability 
has no FDA-approved treatment. Beckett's parents Monica and Chris hope 
to change that for this community. Less than two years after Beckett's 
diagnosis in 2012, Monica Weldon--with the help of Global Genes--
founded Bridge the Gap-SYNGAP Education and Research Foundation, which 
is a member of the National Organization for Rare Disorders (``NORD''). 
Due to Monica's efforts, the Foundation was one of twenty rare disease 
patient organizations selected in April to develop natural history 
studies with the assistance of NORD and supported in part by a 
cooperative agreement with the FDA. The Bridge the Gap-SYNGAP Education 
and Research Foundation is also hosting the first ever SYNGAP1 
International Conference November 30-December 1, 2016 at Texas 
Children's Hospital in Houston in an effort to bring together families, 
researchers, and clinicians to foster a collaborative environment that 
will lead to the development of treatments and ultimately a cure for 
the condition.
   As the SYNGAP1 community initiates these efforts toward treatments 
and a cure, it is critical that the Rare Pediatric Disease Priority 
Review Voucher Program be available as an incentive for manufacturers 
to consider investing in therapies for this condition. Congress must 
continue to help sick children and their families find treatments by 
extending this valuable program.

                          ____________________