[Congressional Record Volume 161, Number 107 (Friday, July 10, 2015)]
[Extensions of Remarks]
[Pages E1036-E1038]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




                         21ST CENTURY CURES ACT

                                 ______
                                 

                               speech of

                         HON. GUS M. BILIRAKIS

                               of florida

                    in the house of representatives

                         Thursday, July 9, 2015

       The House in Committee of the Whole House on the state of 
     the Union had under consideration the bill (H.R. 6) to 
     accelerate the discovery, development, and delivery of 21st 
     century cures, and for other purposes:

  Mr. BILIRAKIS. Mr. Chair, on a personal level, I have family members 
who have suffered with Parkinson's--I witness this debilitating disease 
through them. It is hard to see. Few things in America are truly 
ubiquitous--diseases, sadly, are one of those things.
  In addition to the struggle chronic and rare disease patients face, 
physicians, researchers, clinicians, and medical device companies 
(among others) deal with an outdated and overly burdensome regulatory 
structure. These regulations stifle the development of new cures and 
treatments, whether they are drugs, biologics, or devices.
  Given the reality, we have to ask: how can we get cures and 
treatments to the people who desperately need them?
  That is the question the 21st Century Cures Initiative was created to 
answer. The 21st Century Cures Initiative is a bipartisan undertaking 
by members of the Energy and Commerce Committee to help our healthcare 
innovation infrastructure thrive and deliver more hope for all 
patients. This is a tremendous undertaking, and is much easier said 
than done.
  It is about finding new ways to drive innovation. In addition to 
adequate funding and resources, we need to think critically about 
structural changes to streamline and modernize our health care system. 
We need to rethink what we have been doing and how we are doing it for 
the 21st Century.
  This is what 21st Century Cures Initiative is giving us: an 
opportunity to address some of the structural barriers to new cures and 
promote new ways to incentivize developments. The 21st Century Cures 
Initiative has examined and seeks to accelerate the complete cycle of 
cures--from discovery to development to delivery and back again to 
discovery. This has resulted in the 21st Century Cures Act--a 
culmination of over a year's worth of engaging with patients, 
researchers, physicians, government, and private entities.
  This year included numerous hearings and roundtables in Washington 
D.C. As legislators, we worked tirelessly to engage all stakeholders 
from across the spectrum. The only way we can answer the question--how 
do we get better cures and treatments?--is to work with everyone 
involved in the American health care system.
  I am proud that I was able to have several provisions that were 
included in the final version of the Cures Act. These provisions will 
help to change the lives of patients in small to larger ways. I want to 
take a moment and highlight some of the provisions and some of the 
people that helped shape the policy.

[[Page E1037]]

  Rare diseases are not a rare problem. Nearly 30 million Americans--1 
in every 10 people--are living with a rare disease.
  That is why I introduced the OPEN Act--the Orphan Product Extensions 
Now Act. It was included as a provision of the 21st Century Cures Act.
  My bipartisan bill has the potential to help millions of people by 
incentivizing the testing of mainstream drugs--or repurposing them--to 
treat rare diseases and pediatric cancers, and it was included as a 
major provision in the 21st Century Cures Act.
  The OPEN Act would unlock a new world of potential treatments--it 
would put FDA-approved, safe, and effective treatments ``on-label.''
  Through the 21st Century Cures Act, Congress has a chance to come 
together to make a real difference in the lives of the 160 million 
Americans who suffer from a rare or chronic condition, as well as the 
family members and friends of all those afflicted.
  The OPEN Act is one provision in the 21st Century Cures Act, but it 
is one I am proud to have authored, and one I believe will make a 
substantial difference in the lives of a lot of people.
  I want to take this opportunity to thank all the people who helped 
make the OPEN Act a reality, and who fought for this legislation to be 
in the 21st Century Cures Act.
  Julia Jenkins, Max Bronstein, Andy Russell, Harry Sporidis, Tim 
Perrin, everyone at the EveryLife Foundation for Rare Diseases, and the 
other 155 rare disease groups that supported the OPEN Act:

       National MPS Society, With Purpose, National PKU Alliance, 
     Taylor's Tale, RASopathies Network USA, Kids v Cancer, Let 
     Them Be Little X2 Inc., Info and Resources for Idiopathic 
     Pulmonary Hemosiderosis (IPH-NET), Noah's Hope, Mary Payton's 
     Miracle Foundation, Hope4Bridget Foundation, Batten Disease 
     Support & Research Association, Cure Sanfilippo Foundation, 
     Beyond Batten Disease Foundation, Drew's Hope Scientific 
     Research Foundation, International Pemphigus and Pemphigoid 
     Foundation (IPPF), Cure AHC, Autoinflammatory Alliance, MLD 
     Foundation, Fabry Support & Information Group, Children's PKU 
     Network, FMD Chat;
       National Tay-Sachs & Allied Diseases Association (NTSAD), 
     Little Miss Hannah Foundation, Rare Disease United 
     Foundation, Global Genes Project, Fibromuscular Dysplasia 
     Society of America (FMDSA), Lymphatic Malformation Institute, 
     Mastocytosis Society, EB Research Partnership, BRBN Alliance, 
     Jonah's Just Begun, Abigail Alliance for Better Access to 
     Developmental Drugs, Hannah's Hope Fund, GNE Myopathy 
     International, The Ryan Foundation, Organic Acidemia 
     Association, Cardio-Facio-Cutaneous International, NGLY1.org, 
     Gwendolyn Strong Foundation, POMC Island One boy an Ocean of 
     friends, Gene Giraffe Project, International FOP Association, 
     Aware of Angels;
       CureCADASIL, GT23 FOUNDATION, Desmoid Tumor Research 
     Foundation (DTRF), The Association for Glycogen Storage 
     Disease, Gene Spotlight Inc., Amyloidosis Foundation, 
     Heriditary Neuropathy Foundation, Relapsing Polychondritis, 
     Klippel-Feil Syndrome Freedom, CureDuchenne, Prader-Willi 
     Syndrome Association, Bert's Big Adventure, Parent Project 
     Muscular Dystrophy, Sarcoma Foundation of America, The 
     Nicholas Conor Institute, Luck2Tuck Foundation, Team 
     Sanfilippo Foundation, The Rally Foundation for Childhood 
     Cancer Research, CARES Foundation, Inc., Help Extinguish 
     Hunter Syndrome, Sephardic Health Organization for 
     Referral & Education, Hunter Syndrome Research Coalition;
       The Kortney Rose Foundation, Saving Case & Friends, Phelan-
     McDermid Syndrome Foundation, The Children's Medical Research 
     Foundation, Inc., Cure SMA, Narcolepsy Network, Celiac 
     Support Association, Caleb's Crusade Against Childhood 
     Cancer, International Waldenstrom's Macroglobulinemia 
     Foundation (IWMF), PKD Foundation, EDSers United Foundation, 
     Choroideremia Research Foundation, Inc., Genetic Alliance, 
     The Life Raft Group, The Will Luthcke Foundation, Angioma 
     Alliance, Smashing Walnuts Foundation, Castleman Disease 
     Collaborative Network/Castleman's Awareness & Research 
     Effort, The GIST Cancer Awareness Foundation, The Truth 365, 
     The Arms Wide Open Childhood Cancer Foundation, Sophia's 
     Fund;
       Journey4ACure, Princesses on a Mission, Inc., Noah's Light 
     Foundation, Pediatric Cancer Foundation, West Virginia Kids 
     Cancer Crusaders, Inc., Bear Necessities Cancer Foundation, A 
     Kids' Brain Tumor Cure, RARE Science, Inc., ISMRD (the 
     International Advocate for Glycoprotein Storage Diseases), 
     Hermansky-Pudlak Syndrome Network Inc., Run4Rare, A-T 
     Children's Project, The Global Foundation for Peroxisomal 
     Disorders, The Adult Polyglucosan Body Disease Research 
     Foundation (APBDRF), Alexa Nawrocki Pediatric Cancer 
     Foundation, Beckwith-Wiedemann Children's Foundation 
     International, The Brooke Healey Foundation, Talia's Legacy 
     Children's Cancer Foundation, The Rare Childhood Cancer 
     Advocacy Group, Alex's Army Childhood Cancer Foundation, The 
     Catherine Elizabeth Blair Memorial Foundation, Stillbrave 
     Childhood Cancer Foundation;
       Cures Within Reach, ALL4Trey, Team Sabrina, Sofia's Hope, 
     Inc., ALL4Trey, Delainee's Battle, Joey's Wings Foundation, 
     The Bozeman 3, Team Ashley Bragg, Cole vs Cancer, Dominick 
     One in a Million, Samuel Szabo Foundation, Wilms Tumor 
     Survivor Group, Aiden's Army, Sofia's Hope, Inc., Mikey's Way 
     Foundation, Team Serena, Supporting Our Cancer Kids, The 
     Champ's Corner, Habitat for Hope, Ali's Angels Foundation, 
     Gold Rush Cure Foundation;
       Sickle Cell Warriors, Inc., The Rare Cancer Research 
     Foundation, Carson Leslie Foundation, Amyloidosis Research 
     Consortium, Pulmonary Fibrosis Advocates, The Coalition for 
     Pulmonary Fibrosis, Mytonic Dystrophy Foundation, LMSarcoma 
     Direct Research Foundation, BioPontis Alliance for Rare 
     Diseases, Foundation for Ichthyosis & Related Skin Types, 
     Inc., 5p-Society, The Santonio Holmes III & Long Foundation, 
     National Fragile X Foundation, National Organization for Rare 
     Disorders (NORD), OsteoPETrosis Society, Curing Retinal 
     Blindness Foundation, The MAGIC Foundation, Cure HHT, DEFY 
     Foundation, Chase After a Cure, DC Outreach Inc., Children's 
     Cardiomyopathy Foundation, and the Bridget the Gap--SYNGAP 
     Education and Research Foundation.

  These groups' grassroots efforts were instrumental in the effort to 
get the OPEN Act in the 21st Century Cures Act.
  I would like to thank all the participants of the two 21st Century 
Roundtables I held in my District in August of 2014. Your input was 
vital in the early stages of drafting the 21st Century Cures Act, and I 
will be forever appreciative:
  Dr. Wayne Taylor, on behalf of the Leukemia and Lymphoma Society;
  Mrs. Colleen Labbadia, on behalf of the Parent Project for Muscular 
Dystrophy (PPMD);
  Ms. Patricia Stanco, MHS, on behalf of the ALS Association Florida 
Chapter;
  Ms. Ashleigh Pike and Ms. Beth Pike--dysautonomia patient advocates;
  Dr. Samantha Lindsay, on behalf of the Alpha-1 Foundation;
  Ms. Janice Starling, on behalf of the American Association of Kidney 
Patients;
  Mr. and Mrs. Michael and Gretchen Church, on behalf of the 
Parkinson's Action Network;
  Dr. Clifton Gooch, FAAN, Professor and Chair, Department of 
Neurology, University of South Florida;
  Dr. Dave Morgan, CEO and Director of USF's Byrd Alzheimer's 
Institute;
  Dr. Richard Finkel, Chief Neurologist at Nemour's Children's 
Hospital;
  Mr. Geary A. Havran, President of NDH Medical, Inc., and Chairman, 
Florida Medical Manufacturers Consortium (FMMC);
  Ms. Lisa Novorska, CFO, Rochester Electro-Medical, Inc.;
  Dr. Thomas Sellers, MPH., Center Director and Executive Vice 
President for Moffit Cancer Center;
  Dr. Glen Hortin, Clinical Pathology Medical Director for the 
Southeast Region, for Quest Diagnostics.
  Additionally, I want to thank Nick Manetto from PPMD and USAgainst 
Alzheimer's, Miriam O'Day from the Alpha-1 Foundation, John DeMuro from 
Moffitt, Monica Richter from USF, Jennifer Sheridan from PAN, Gary 
Dessatti, John Ray from FMMC, and Erin O'Malley and Virginia Biggar 
from USAgainstAlzheimer's.
  I would also like to thank Candace Lerman, Laura Milford, and Max 
Schill. They are all rare disease patients and advocates who I have had 
the pleasure of meeting. Their in-person advocacy and their dedication 
to improve the lives of everyone with a rare disease is admirable. I am 
truly grateful to their contribution and support of this legislation.
  I also want to recognize Noah Coughlan and Jonny Lee Miller. Noah is 
a young man who ran across the country--over 3,000 miles--three times 
to raise awareness for rare diseases. This is a feat achieved by very 
few, and is a tremendous physical feat demonstrating his dedication to 
this cause. Jonny Lee Miller is an actor and advocate who runs ultra-
marathons to raise money and awareness for rare diseases.
  To everyone else who was involved, supported the OPEN Act, tweeted 
about it, posted about it on Facebook, or advocated on behalf of the 30 
million Americans with rare diseases, I sincerely thank you from the 
bottom of my heart.
  Additionally, the 21st Century Cures Act includes another one of my 
bills, H.R. 2298, the Patient Safety and Prescription Drug Abuse 
Prevention Act. I began work on this bill three years ago, after a 
prescription drug abuse hearing. The problem was apparent, and a fix 
was desperately needed.
  This provision will create a drug management program within Medicare 
to use the same tools used in Medicaid, TRICARE, and private insurance 
to deal with the growth in prescription drug abuse.
  The Substance Abuse and Mental Health Administration (SAMHSA) 
estimates that there are 15.3 million Americans over the age of 12 that 
``used prescription drugs non-medically in the last year.'' USA Today 
reported that in 2012, the average number of seniors misusing

[[Page E1038]]

or dependent on prescription pain relievers in the past year grew to an 
estimated 336,000, up from 132,000 a decade earlier, based on data from 
SAMHSA. Addiction does not recognize age, race, ethnicity, or income. 
Anyone could be susceptible including seniors.
  The U.S. Department of Health and Human Services' Office of the 
Inspector General has recommended that Medicare have this type of a 
program. In a hearing, the Center for Medicare and Medicaid Services' 
Principal Deputy Administrator stated that they supported this policy, 
but needed a statutory change in the law to create such a program.
  A change to the Medicare program is a herculean task. I want to thank 
some of the people that supported this provision and helped get this 
legislation over the finish line. Lindsay Berman from the Pew 
Charitable Trusts, Jerry Steffl, Jonathan Heafitz, Gary Kline, Sergio 
Santiviago, Richard Hoar, Heather Cutler, Nelson Bunn from the Major 
County Sheriffs Association, and Chuck DeWitt from the Major Cities 
Chiefs Association.

                          ____________________