Mr. Speaker, before giving my remarks, I include in the Record a statement by Senator Johnson explaining the intent of S. 204. Statement of Legislative Intent (By Sen. Ron Johnson on S. 204 (as considered by the House of Representatives))…
Mr. Speaker, before giving my remarks, I include in the Record a statement by Senator Johnson explaining the intent of S. 204.
Statement of Legislative Intent
(By Sen. Ron Johnson on S. 204 (as considered by the House of
Representatives))
In a recent article about pending right to try legislation,
FDA Commissioner Scott Gottlieb was quoted as saying: ``In
terms of making sure that it balances [access to experimental
drugs] against appropriate patient protections . . . with [S.
204], we'd have to do a little bit more . . . in guidance and
perhaps in regulation to achieve some of those goals, and I
think those are the goals that Congress wants us to
achieve.'' The article went on to quote Commissioner Gottlieb
as saying: ``We felt that there were certain aspects of [S.
204] that could be modified to build in additional patient
protections, but if you weren't able to do that
legislatively, that there [was] a pathway by which you do
that administratively and still remain consistent with the
letter and the spirit of this law.''
In response to this article, Commissioner Gottlieb tweeted
the ``FDA . . . stands ready to implement [right to try] in a
way consistent with the intent of Congress.''
As S. 204's primary author and lead sponsor, I want to make
this legislation's intent absolutely clear and remove any
ambiguity that the FDA could use to implement right to try in
a way contrary to its aim.
S. 204, as originally introduced, applied to patients
``with a terminal illness,'' as defined by State law. In
discussion with the FDA, the agency suggested it would prefer
a uniform federal definition, especially one that already
existed in federal statute or regulation, because an existing
federal definition would facilitate implementation of the
law. The FDA suggested defining terminal illness as an
``immediately life-threatening disease or condition.'' The
FDA disclosed that its suggested definition would exclude,
for example, patients with Duchenne muscular dystrophy--an
illness explicitly intended to be covered by the legislation.
To be clear, I rejected this proposed definition because I
believed it would inappropriately exclude patients with
certain diseases from accessing treatments. By contrast, the
legislation instead defines terminal illness as ``life-
threatening disease or condition'' (which exists in current
federal regulation), which the FDA confirmed would include
patients diagnosed with Duchenne muscular dystrophy.
Contrary to the preference of FDA official Dr. Janet
Woodcock, who expressed the FDA's desire to draft the
legislation ``to make sure we don't include patients we (the
FDA) doesn't intend to include,'' I replied and rejected that
notion by stating my intent was completely opposite hers:
``I wanted to make sure we didn't exclude any one we didn't
intend to exclude.'' My aim from the beginning was to be as
inclusive as possible such that as many patients as possible
who are facing no available alternatives could potentially
qualify.
S. 204 is fundamentally about empowering terminally-ill
patients and their doctors who, together with the cooperation
of the developers of potentially life-saving therapies,
should be in charge of making a determination about their own
course of treatment. The bill is not intended to further
empower any federal agency, including the FDA, to limit in
any way the ability of an individual facing a life-
threatening disease or condition from accessing treatment. S.
204 is about preserving a right to hope and about expanding
individual freedom. It is not meant to empower the FDA to
limit the right to hope by regulation or guidance.
S. 204 includes a provision ensuring the Secretary may not
use a clinical outcome associated with the use of an eligible
investigational drug to delay or adversely affect review or
approval of the drugs, unless use of such clinical outcome is
critical to determining safety. This language is in no way
intended to enable the FDA to expand the scope of existing
safety determinations regarding investigational drugs.
S. 204 requires, in certain circumstances, that an eligible
investigational drug be under investigation in a clinical
trial that is intended to form the primary basis of a claim
of effectiveness in support of approval or licensure.
According to the FDA, this language simply incorporates the
standard definition of a clinical trial. This language is not
in any way intended to enable the FDA to exclude any clinical
trial as a basis for precluding access to treatments under
right to try.
Mr. Speaker, I rise today in strong support of the Right to Try Act and on behalf of the patients who are fighting each and every day to try to save their own lives.
It has been a long ride, but we are in sight of our destination.
Mr. Speaker, I would like to take a brief moment to thank my friend and colleague, Representative Fitzpatrick, for working with me on this cause from the moment we both entered office last year, and to extend my appreciation to Senator Johnson, whose efforts on behalf of right to try have been extraordinary.
Mr. Speaker, I also thank Chairman Walden for his efforts and the leadership of President Trump and Vice President Pence.
Mr. Speaker, I acknowledge and thank my predecessor, Congressman Matt Salmon, for his tireless efforts to pass right to try.
But it is the patients themselves and their tireless advocates who deserve the most recognition. I have said this before and I will continue to say it: when the Right to Try Act passes this Chamber and is signed into law by the President, it will be them, not us, who deserve the most credit for this remarkable victory.
Everyone here has heard me speak about the Right to Try Act more than a few times already and everyone here is aware of the widespread support that this legislation has garnered. Forty States have already passed right-to-try legislation, often with unanimous or overwhelming support from Republicans and Democrats alike.
If we can't come together to support a commonsense cause such as this one, I am not sure what effort we can unite behind.
Those on the other side of this debate--and they are a shrinking minority--argue that this legislation is unnecessary. Well, if it is so unnecessary, why do I receive phone calls and letters from patients each week urging me to do everything in my power to get this legislation passed?
I have no doubt the FDA's expanded access program helps patients, but I also know that the agency's personnel, including Director Gottlieb himself, want to help as many patients as possible, but their efforts simply are not enough.
The Right to Try Act doesn't eliminate the expanded access program. Far from it. We are merely providing another, more direct avenue for patients to acquire potentially lifesaving medications from pharmaceutical companies that don't require them to ask permission from a bureaucratic middleman.
Another argument I hear from the naysayers, one that makes me angry, is that we are peddling false hope. False hope? What is that?
Mr. Speaker, to this tired argument, I respond that there is no such thing as false hope. You either have hope or you don't.
I, for one, want those brave men and women who are fighting every day against terrible illnesses and almost insurmountable odds to have a choice, even if it is the last choice many of them will ever have the opportunity to make. I trust them to weigh the pros and cons and choose for themselves whether they wish to take a risk to try to save their own lives.
Make no mistake: it is a choice. We are not offering a mandate, merely an option.
Mr. Speaker, I urge all of my colleagues to vote ``yes'' on this legislation.