Mr. Speaker, I thank the ranking member for his leadership and for yielding me time. Mr. Speaker, S. 204 is harmful legislation that offers a false hope of access to investigational therapies and endangers patients who have serious and…
Mr. Speaker, I thank the ranking member for his leadership and for yielding me time.
Mr. Speaker, S. 204 is harmful legislation that offers a false hope of access to investigational therapies and endangers patients who have serious and life-threatening diseases. The bill establishes a dangerous and unnecessary alternative pathway that is void of any FDA review or oversight. It is opposed overwhelmingly by the patient community.
Mr. Speaker, I include in the Record a letter to the Speaker and minority leader from 104 patient advocacy groups. It includes such groups as the American Cancer Society Cancer Action Network, the American Lung Association, the Cystic Fibrosis Foundation--all opposed to this bill--the Leukemia & Lymphoma Society, and about 100 more.
May 21, 2018.
Hon. Paul Ryan,
Speaker, House of Representatives, Washington, DC.
The Hon. Nancy Pelosi,
Minority Leader, House of Representatives, Washington, DC.
Dear Speaker Ryan and Leader Pelosi: The undersigned
organizations collectively represent millions of patients
with serious and life-threatening diseases. We write to
express our strong opposition to the Trickett Wendler, Frank
Mongiello, Jordan McLinn, and Matthew Bellina Right to Try
Act (S. 204).
On March 21st, The House of Representatives passed a
version of the Right to Try Act (H.R. 5247), that
incorporated important patient safeguards such as more robust
informed consent and public reporting requirements,
additional Food and Drug Administration (FDA) oversight, and
a narrower definition of eligibility for this pathway. The
Senate version does not include these safeguards and
therefore could greatly increase the likelihood of our
patients being harmed by unsafe and ineffective experimental
therapies. Therefore, this version is substantially worse for
patients.
We reiterate our concern with creating a secondary pathway
for accessing investigational therapies outside of clinical
trials. This pathway removes FDA approval and consultation
and would not increase access to promising therapies for our
patients because it does not address the primary barriers to
access.
FDA's expanded access program, though imperfect,
facilitates access to investigational therapies for over a
thousand patients facing serious and life-threatening
conditions each year. FDA repeatedly approves over 99 percent
of requests while sometimes making important dosing and
safety improvements to proposed expanded use. Conversely, it
is often times the pharmaceutical company that denies access
to its investigational therapy outside of its clinical trials
for any number of reasons.
The Senate version of the legislation is less safe than the
pathway proposed in the House version and is dangerous
compared to the current expanded access process. The Senate's
bill would allow unproven therapies to be given to patients
without FDA notification for up to a full year and would not
establish any standards for informed consent.
Additionally, both versions prohibit FDA from halting
access to these experimental therapies short of placing a
clinical hold on all clinical research on the therapy in
question. Both House and Senate versions would also remove
FDA's consultation on dosing, route of administration, dosing
schedule, and other important safety measures available under
FDA's current expanded access program.
While we did not support the recent House passed version of
this legislation, the House legislation includes improved
patient safeguards compared to the Senate version. The Senate
version would negatively impact patient safety substantially,
and our collective organizations are strongly opposed. We
appreciate past efforts in the House to consider stakeholder
perspectives and desire to continue the dialogue, but
returning to the Senate version is simply not the way
forward.
Sincerely,
A Twist of Fate-ATS; ADNP Kids Research Foundation; Adult
Polyglucosan Body Disease Research Foundation; AIDS Action
Baltimore; Alliance for Aging Research; Alliance of Dedicated
Cancer Centers; American Cancer Society Cancer Action
Network; American Lung Association; American Society of
Clinical Oncology; American Syringomyelia and Chiari Alliance
Project; Amyloidosis Support Groups; APS Type 1 Foundation;
Association for Creatine Deficiencies; Association of
American Medical Colleges; Benign Essential Blepharospasm
Research Foundation; Bonnie J. Addario Lung Cancer
Foundation; Bridge the Gap-SYNGAP Education and Research
Foundation; CancerCare; Charlotte and Gwenyth Gray Foundation
to Cure Batten Disease; Children's Cardiomyopathy Foundation.
Congenital Hyperinsulinism International; cureCADASIL;
CurePSP; Cutaneous Lymphoma Foundation; Cystic Fibrosis
Foundation; Defeat MSA; The Desmoid Tumor Research
Foundation; The Disability Rights Legal Center; Dupl5q
Alliance; Dysautonomia Foundation; Dyskeratosis Congenita
Outreach, Inc.; Equal Access for Rare Disorders; Fight
Colorectal Cancer; FORCE: Facing Our Risk of Cancer
Empowered; Friedreich's Ataxia Research Alliance (FARA);
Friends of Cancer Research; The Global Foundation for
Peroxisomal Disorders; Glutl Deficiency Foundation; The
Guthy-Jackson Charitable Foundation; Hemophilia Federation of
America.
HLRCC Family Alliance; Hope for Hypothalamic Hamartomas;
Hyper IgM Foundation, Inc.; Incontinentia Pigmenti
International Foundation; Indian Organization for Rare
Disorders; International Fibrodysplasia Ossificans
Progressiva (FOP) Association; International Myeloma
Foundation; International Pemphigus and Pemphigoid
Foundation; International Society for Stem Cell Research;
International Waldenstrom's Macroglobulinemia Foundation
(IWMF); The Isaac Foundation; Jack McGovern Coats' Disease
Foundation; The LAM Foundation; The Leukemia & Lymphoma
Society; Li-Fraumeni Syndrome Association (LFS
Association/LFSA); LUNGevity Foundation; Lymphangiomatosis
& Gorham's Disease Alliance; M-CM Network; Mattie Miracle
Cancer Foundation; MitoAction.
MLD Foundation; Moebius Syndrome Foundation; The MSA
Awareness Shoe; Mucolipidosis Type IV Foundation; The Myelin
Project; Myotonic Dystrophy Foundation; National Brain Tumor
Society; National Comprehensive Cancer Network; National
Consumers League; National Health Council; National MPS
Society; National Niemann-Pick Disease Foundation; National
Organization for Rare Disorders (NORD); National Patient
Advocate Foundation; National PKU Alliance; National PKU
News; Neurofibromatosis Northeast; The Oley Foundation;
Operation ASHA; Organic Acidemia Association.
PSC Partners Seeking a Cure; Platelet Disorder Support
Association; PRP Alliance, Inc.; Pulmonary Fibrosis
Foundation; Rare and Undiagnosed Network (RUN); Rothmund-
Thomson Syndrome Foundation; The Snyder-Robinson Foundation;
Sofia Sees Hope; SSADH Association; Susan G. Komen;
TargetCancer Foundation; Tarlov Cyst Disease Foundation; Team
Audrey; Treatment Action Group; The Turner Syndrome Society;
United Leukodystrophy Foundation; United Mitochondrial
Disease Foundation (UMDF); Vasculitis Foundation; Veterans
Health Council; Vietnam Veterans of America; VHL Alliance;
Wilhelm Foundation; Worldwide Syringomyelia & Chiari Task
Force; The XLH Network, Inc.
Mr. Speaker, this bill is a bill in search of a problem. FDA has approved 99 percent of the expanded access requests it receives. FDA's expanded access program approves nearly all requests for investigational drugs or biologics it receives.
Physicians at the FDA are available 24 hours a day to approve any emergency expanded access requests that the agency receives, and it typically grants those emergency requests immediately over the phone and nonemergency requests in a median time of 4 days and generally no longer than 30 days. FDA has also taken actions to streamline this entire process.
The process of clinical trials at FDA is vital to the protection of the health of all of our neighbors and the folks we represent. In 11 percent of expanded access applications, FDA has raised a red flag and said: Do you know what? You have got to change this.
That is who we are trying to protect here: the actual patients. The patient groups across the country agree with us.
Many States have tinkered with right-to-try laws, but this is different. Forty States have enacted right-to-try laws, but there is no evidence that anyone has obtained the type of therapy via these laws that couldn't have been obtained through the FDA's expanded access program.
Right-to-try laws do not compel companies to provide patient access to these treatments. Therefore, under these laws, patients still do not have a right to try, only the right to request it from the company.
Sometimes those insurance companies will say: Do you know what? We are not going to pay for it.
So that is going to be another barrier.
Mr. Speaker, in the end, these right-to-try laws put patients at higher risk by prohibiting and weakening the FDA oversight, leaving our neighbors on the hook to cover the cost of unproven treatments.
For all of these reasons, I urge a ``no'' vote on the bill. Join with the patient advocates across America, who, in this letter, called this a dangerous proposal.