[Congressional Record Volume 158, Number 83 (Tuesday, June 5, 2012)]
[Extensions of Remarks]
[Pages E974-E975]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




            FOOD AND DRUG ADMINISTRATION REFORM ACT OF 2012

                                 ______
                                 

                               speech of

                           HON. CLIFF STEARNS

                               of florida

                    in the house of representatives

                        Wednesday, May 30, 2012

  Mr. STEARNS. Mr. Speaker, the Food and Drug Administration Reform 
Act, H.R. 5651, is based on user fee negotiations between FDA and the 
prescription drug, generic drug, biologic, and medical device industry. 
This reauthorization of the FDA user fees will provide stability with 
FDA's new product review as companies submit new and innovative devices 
and drugs for approval.
  In codifying the User Fee Agreement, this committee has included 
additional provisions designed to address some of the defects of the 
regulatory structure and overreach by the FDA. Under my Chairmanship of 
the Oversight and Investigation Subcommittee, we held a hearing into 
FDA's regulatory efforts in the medical device space. During our 
hearing, many of the witnesses talked about the reluctance of FDA to 
approve devices and how FDA continually moved the goalposts for 
approval. I am glad that Title VII of this bill includes a significant 
number of reform provisions designed to bring certainty to the medical 
device field.
  In addition to reforming approaches to medical devices through Title 
VII, the FDA's approach to rare diseases must also be modernized. I'm 
happy the Committee included the Faster Access to Specialized 
Treatments Act, FAST Act, H.R. 4132, which I introduced with my friend 
and colleague, Representative Ed Towns. FAST updates and modernizes 
Section 506 of the Food, Drug & Cosmetic Act, and updates the 
Accelerated Approval statute to reflect two decades worth of medical 
sciences that has occurred since Accelerated Approval was first 
created. FAST will help FDA implement broadly effective processes for 
the expedited development and review of innovative new medicines 
intended to address unmet medical needs for serious or life-threatening 
diseases by using modern scientific tools.
  The use of surrogate endpoints may result in fewer, smaller or 
shorter clinical trials without compromising FDA's existing high 
standards for safety or efficacy. Surrogate and clinical endpoints only 
need to be reasonable predictors of clinical benefit to support 
accelerated approval. They do not need to be validated or proven first. 
The changes made to current law permitting the Secretary to require 
validation of surrogates following accelerated approval is not intended 
to change FDA's long history of granting accelerated approval based on 
unvalidated, but predictive, surrogate endpoints.

[[Page E975]]

  Additionally, FAST includes explicit language for FDA to think about 
the challenges of rare diseases when developing their guidance and 
gives the rare disease community an opportunity to publically comment 
on FDA's draft guidance. FAST ensures that the voices of the 30 million 
Americans with a rare disease will be heard by FDA. There are about 
7,000 rare diseases and only about 250 have any treatment. FAST will 
save lives, and give a voice to the voiceless; and I am glad it is in 
the bill.
  Lastly, the committee included the Expanding and Promoting Expertise 
in Review of Rare Treatments, EXPERRT Act, H.R. 4156, a bill my fellow 
Co-Chairs of the Cystic Fibrosis Caucus and I introduced. EXPERRT will 
have the FDA consult with experts in rare diseases. This will ensure 
that FDA has access to the knowledge needed when dealing with drug 
approvals for diseases where FDA may lack subject matter expertise. As 
one of the Co-Founders of the Cystic Fibrosis Caucus, I am glad that we 
are giving this tool to the FDA.
  I'd like to submit this letter of support for FAST signed by over 150 
rare disease groups into the Record.
  H.R. 5651 is a good bill that will help new drugs and new medicines 
get into the market and be available to patients. I support passage of 
the FDA Reform Act.
                                                   March 23, 2012.
     Hon. Cliff Stearns,
     House of Representatives, Rayburn House Office Building, 
         Washington, DC.
     Hon. Edolphus Towns,
     House of Representatives, Rayburn House Office Building, 
         Washington, DC.
       Dear Congressmen Stearns & Towns: On behalf of patients, 
     physicians, and other members of the health advocacy 
     community we are writing to express our support for H.R. 
     4132, the Faster Access to Specialized Treatments (FAST) Act. 
     This legislation will modernize and expand the FDA's 
     Accelerated Approval pathway to encompass a broader range of 
     diseases and leverage 21st century drug development tools and 
     strategies. This reform will speed the approval of much-
     needed therapies and cures to patients who are facing serious 
     and life-threatening conditions, including Alzheimer's 
     disease, autoimmune diseases, multiple sclerosis, Parkinson's 
     disease, neuromuscular disease and hundreds of rare diseases 
     that remain untreated.
       We commend you for championing legislation that maintains 
     the FDA's high standard for approval while at the same time 
     ensuring the Agency can help facilitate the development of 
     new and novel therapies to patients in a more timely manner. 
     In many cases our patients have no available treatment for 
     their diseases, or they are using a therapy that is older and 
     may not work as effectively and safely. This is not 
     acceptable. We believe that this legislation will ensure 
     patients receive the best, modern treatment as soon as 
     possible and we applaud your efforts on their behalf.
       Thank you for your leadership on this important bill and we 
     look forward to working with you as it moves forward.
           Sincerely,
         Abigail Alliance for Better Access to Developmental 
           Drugs; Advocacy for Patients with Chronic Illness, 
           Inc.; Affiliated American CSA Foundation; Alliance for 
           Aging Research; Alliance for Patient Access; American 
           Autoimmune Related Diseases Association; American Brain 
           Tumor Association; American Childhood Cancer 
           Organization; American College of Medical Genetics; 
           American Institute for Medical and Biological 
           Engineering; American Society of Clinical 
           Psychopharmacology; Batten Disease Support and Research 
           Association; Break Through Cancer Coalition; 
           Californians for Cures.
         Celiac Disease Center at Columbia University; Celiac 
           Sprue Association; Charcot-Marie-Tooth Association 
           (CMTA); Children's Cardiomyopathy Foundation, Inc.; 
           Chinese American Association of Greater Chicago; 
           Coalition Duchenne; Coalition for Pulmonary Fibrosis; 
           Colon Cancer Alliance; Cooleys Anemia Foundation; 
           Crohn's and Colitis Foundation of America; 
           Cryoglobulinemia Vasculitis Organization; CureDuchenne; 
           CurePSP; Digestive Disease National Coalition; Erik 
           Metzler Foundation.
         EveryLife Foundation for Rare Diseases; Fabry Support & 
           Information Group; Georgia PKU Connect; GIST Support 
           International; Hadley Hope Fund; Hannah's Hope Fund; 
           Hayden's Batten Disease Foundation Inc.; HealthHIV; 
           Hope4Bridget Foundation; ICE Epilepsy Alliance; I Have 
           IIH; In Need of Diagnosis, Inc. (INOD); Inspire; 
           International Cancer Advocacy Network (ICAN); Jacob's 
           Cure, Inc.
         Jain Foundation Inc.; Jonah's Just Begun--Foundation to 
           Cure Sanfilippo Inc. LAM Treatment Alliance; LGS 
           Foundation; Liddy Shriver Sarcoma Initiative; Little 
           Miss Hannah Foundation; Lung Cancer Alliance; Lupus 
           Foundation of America; Lymphangiomatosis & Gorham's 
           Disease Alliance (LGDA); Lymphatic Malformation 
           Institute (LMI); Macular Degeneration Support, Inc. 
           Madisons Foundation; Midwest Asian Health Association 
           (MAHA); MLD Foundation; Mpdsupport.org--
           Myeloproliferative Disease Support; Muscular Dystrophy 
           Association.
         National Family Caregivers Association; National MPS 
           Society; National MS Society; National Niemann-Pick 
           Disease Foundation, Inc.; National PKU Alliance; 
           National Tay-Sachs & Allied Diseases Association; 
           National Venture Capital Association; NBIA Disorders 
           Association; New Jersey Association for Biomedical 
           Research; NKH International Family Network; Noah's 
           Hope--Batten Disease Fund; Oxalosis and Hyperoxaluria 
           Foundation; Pachyonychia Congenita Project.
         Parkinson's Action Network; Parry-Romberg Syndrome 
           Resource, Inc.; Partnership for Cures; Polycystic 
           Kidney Disease Foundation; RARE Project; Russell-Silver 
           Syndrome Support; Scleroderma Research Foundation; 
           Sickle Cell Disease Association of America, Inc.; 
           Society for Women's Health Research; Solving Kids' 
           Cancer; Student Society for Stem Cell Research; Sudden 
           Arrhythmia Death Syndromes (SADS) Foundation; Taylor's 
           Tale.
         The Association for Frontotemporal Degeneration (AFTD); 
           The Children's Medical Research Foundation, Inc.; The 
           Erythromelalgia Association; The Focus Foundation; The 
           Manton Center for Orphan Disease Research, Children's 
           Hospital Boston; The Reflex Sympathetic Dystrophy 
           Syndrome Association (RSDSA); The Stop ALD Foundation; 
           Tuberous Sclerosis Alliance; Veterans Health Council; 
           VHL Family Alliance; Vietnam Veterans of America; 
           ZERO--The Project to End Prostate Cancer.

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