[Congressional Record Volume 166, Number 195 (Tuesday, November 17, 2020)]
[House]
[Pages H5807-H5809]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]
FAIRNESS IN ORPHAN DRUG EXCLUSIVITY ACT
Mr. PALLONE. Mr. Speaker, I move to suspend the rules and pass the
bill (H.R. 4712) to amend the Federal Food, Drug, and Cosmetic Act with
respect to limitations on exclusive approval or licensure of orphan
drugs, and for other purposes, as amended.
The Clerk read the title of the bill.
The text of the bill is as follows:
H.R. 4712
Be it enacted by the Senate and House of Representatives of
the United States of America in Congress assembled,
SECTION 1. SHORT TITLE.
This Act may be cited as the ``Fairness in Orphan Drug
Exclusivity Act''.
SEC. 2. LIMITATIONS ON EXCLUSIVE APPROVAL OR LICENSURE OF
ORPHAN DRUGS.
(a) In General.--Section 527 of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 360cc) is amended--
(1) in subsection (a), by striking ``Except as provided in
subsection (b)'' and inserting ``Except as provided in
subsection (b) or (f)''; and
(2) by adding at the end the following:
``(f) Limitations on Exclusive Approval, Certification, or
License.--
``(1) In general.--For a drug designated under section 526
for a rare disease or condition pursuant to the criteria set
forth in subsection (a)(2)(B) of such section, the Secretary
shall not grant, recognize, or apply exclusive approval or
licensure under subsection (a), and, if such exclusive
approval or licensure has been granted, recognized, or
applied, shall revoke such exclusive approval or licensure,
unless the sponsor of the application for such drug
demonstrates--
``(A) with respect to an application approved or a license
issued after the date of enactment of this subsection, upon
such approval or issuance, that there is no reasonable
expectation at the time of such approval or issuance that the
cost of developing and making available in the United States
such drug for such disease or condition will be recovered
from sales in the United States of such drug, taking into
account all sales made or reasonably expected to be made
within 12 years of first marketing the drug; or
``(B) with respect to an application approved or a license
issued on or prior to the date of enactment of this
subsection, not later than 60 days after such date of
enactment, that there was no reasonable expectation at the
time of such approval or issuance that the cost of developing
and making available in the United States such drug for such
disease or condition would be recovered from sales in the
United States of such drug, taking into account all sales
made or reasonably expected to be made within 12 years of
first marketing the drug.
``(2) Considerations.--For purposes of subparagraphs (A)
and (B) of paragraph (1), the Secretary and the sponsor of
the application for the drug designated for a rare disease or
condition described in such paragraph shall consider sales
from all drugs that--
``(A) are developed or marketed by the same sponsor or
manufacturer of the drug (or a licensor, predecessor in
interest, or other related entity to the sponsor or
manufacturer); and
``(B) are covered by the same designation under section
526.
``(3) Criteria.--No drug designated under section 526 for a
rare disease or condition pursuant to the criteria set forth
in subsection (a)(2)(B) of such section shall be eligible for
exclusive approval or licensure under this section unless it
met such criteria under such subsection on the date on which
the drug was approved or licensed.''.
(b) Rule of Construction.--The amendments made in
subsection (a) shall apply to any drug that has been or is
hereafter designated under section 526 of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 360bb) for a rare disease
or condition pursuant to the criteria under subsection
(a)(2)(B) of such section regardless of--
(1) the date on which such drug is designated or becomes
the subject of a designation request under such section;
(2) the date on which such drug is approved under section
505 of such Act (21 U.S.C. 355) or licensed under section 351
of the Public Health Service Act (42 U.S.C. 262) or becomes
the subject of an application for such approval or licensure;
and
(3) the date on which such drug is granted exclusive
approval or licensure under section 527 of the Federal Food,
Drug, and Cosmetic Act (21 U.S.C. 360cc) or becomes the
subject of a request for such exclusive approval or
licensure.
SEC. 3. DETERMINATION OF BUDGETARY EFFECTS.
The budgetary effects of this Act, for the purpose of
complying with the Statutory Pay-As-You-Go Act of 2010, shall
be determined by reference to the latest statement titled
``Budgetary Effects of PAYGO Legislation'' for this Act,
submitted for printing in the Congressional Record by the
Chairman of the House Budget Committee, provided that such
statement has been submitted prior to the vote on passage.
The SPEAKER pro tempore. Pursuant to the rule, the gentleman from New
Jersey (Mr. Pallone) and the gentleman from Oregon (Mr. Walden) each
will control 20 minutes.
The Chair recognizes the gentleman from New Jersey.
General Leave
Mr. PALLONE. Mr. Speaker, I ask unanimous consent that all Members
may have 5 legislative days in which to revise and extend their remarks
and include any extraneous material on H.R. 4712.
The SPEAKER pro tempore. Is there objection to the request of the
gentleman from New Jersey?
There was no objection.
Mr. PALLONE. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, today I rise in support of H.R. 4712, the Fairness in
Orphan Drug Exclusivity Act, a bill that will close a loophole in the
orphan drug program to ensure generic drugs are not unfairly being
blocked from entering the market.
Since it was first passed in 1983, the Orphan Drug Act has been
successful in driving research and discovery of new therapies to treat
and even cure rare diseases. The law creates two pathways for
manufacturers to be designated as an orphan drug and to gain certain
incentives, including 7 years of market exclusivity.
The first and most commonly used pathway is for developing drugs
approved to treat diseases with patient populations of 200,000 or
fewer. There is also the rarely used cost-recovery pathway, where the
drug research and development costs are not expected to be recouped by
sales of the underlying drug.
Now, under certain circumstances, a manufacturer may also receive
additional rounds of exclusivity for drugs in their portfolio if they
treat the same conditions and have the same active ingredient, even if
the second drug does not meet the orphan drug qualifications. This
provision has allowed some manufacturers to circumvent the original
intent of the Orphan Drug Act, which was to incentivize creation of
novel drugs for small populations, all the while blocking generic
competitors from coming to market.
An example of this recently occurred when a formulation of
Buprenorphine, a drug to treat opioid use disorder, was approved in
2017. It was allowed to carry the orphan drug designation granted to
its manufacturer's original Buprenorphine drug more than 20 years
earlier, in 1994.
When the original 1994 orphan drug designation was granted, it was
expected that Buprenorphine would not be prescribed frequently;
however, as the opioid crisis worsened and our response to the crisis
evolved, millions were eventually prescribed the drug, generating
billions of dollars in sales.
Clearly, we knew in 2017 that Buprenorphine was not an orphan drug.
Nevertheless, the drug was granted orphan drug status and exclusivity,
delaying additional forms of generic competition. So while the Food and
Drug Administration eventually recognized this issue with this
particular drug and revoked its orphan drug designation, its
exclusivity delayed generic competition that otherwise would have been
on the market.
We need every tool available to us to combat the opioid epidemic, and
loopholes like this one should not be allowed to limit access to
treatment, Mr. Speaker.
H.R. 4712 will stop this from happening again in the future by
requiring drug manufacturers to demonstrate in their application to the
FDA that each drug application considered under the cost recovery
pathway would fail to recoup development costs.
This bill is narrowly tailored. It is a fix for a small but very real
loophole in the law, and I want to thank Representative Dean for
introducing the legislation.
Mr. Speaker, I urge all of my colleagues to support it, and I reserve
the balance of my time.
[[Page H5808]]
{time} 1230
Mr. WALDEN. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, I too rise in support of H.R. 4712. This is the Fairness
in Orphan Drug Exclusivity Act as you have heard. I want to thank
Representatives Carter, McKinley, Dean, and Veasey for their work and
leadership on this important legislation.
The Orphan Drug Act was enacted to incentivize the development of
drugs for rare diseases by providing products that receive an orphan
drug designation 7 years of market exclusivity. That means a drug
produced by another manufacturer that contains the same active
ingredient to treat the same condition is barred from entering the
market during this time period.
One way a drug can receive an orphan designation and subsequent
marketing exclusivity is by the manufacturer's demonstration that there
is no reasonable expectation that the cost of developing the drug will
be recovered.
However, we have seen in recent years that some drug manufacturers,
in an effort to block competitors from entering the market, have taken
advantage of a loophole in this law. Existing law allows an orphan drug
designation and marketing exclusivity to carry forward to future
versions of the same drug without requiring the manufacturer to
demonstrate the drug has not been, and remains unlikely to be,
profitable. This legislation closes that loophole. It requires
manufacturers to demonstrate there is no reasonable expectation that
the costs of research and development will be recovered for each
successor drug, while still preserving incentives for orphan drug
development.
While disagreements do remain, Mr. Speaker, on whether these
amendments should apply retroactively, those differences should not
prevent us from addressing this important issue today.
So I look forward to continuing negotiations on these differences as
we work with the Senate and get a bill down to the President's desk for
signature.
Mr. Speaker, I reserve the balance of my time.
Mr. PALLONE. Mr. Speaker, I yield 3 minutes to the gentlewoman from
Pennsylvania (Ms. Dean), who is the sponsor of the legislation.
Ms. DEAN. Mr. Speaker, I rise in support of H.R. 4712, the Fairness
in Orphan Drug Exclusivity Act.
This legislation would close a current loophole that is used to block
competition in the pharmaceutical marketplace. This could deny
innovative treatments for opioid use disorder and limit the types of
treatments for those in recovery and what they can access.
The Orphan Drug Act of 1983 has provided incentives for prescription
drug manufacturers to develop products to treat rare diseases. This
includes an exclusive 7-year marketing right for therapies that receive
an orphan drug designation.
For a drug to qualify, it must either be a treatment for a disease or
condition that affects fewer than 200,000 people in the United States;
Or a drug intended for diseases that there is no reasonable
expectation to recoup research and development costs.
For the latter criterion, this legislation would require all drug
manufacturers who obtain orphan drug status to prove that they have no
reasonable expectation that they will recover their R&D costs.
Importantly, this legislation is narrowly tailored and would not affect
any product that does receive orphan drug status under the first
criterion.
The scenario this legislation works to prevent, as the chairman has
said, is companies continuing to use orphan drug exclusivity status for
a newly approved drug with an identical ingredient to the former
version without having to prove the inability to recoup costs. Closing
this loophole would ensure that a product does not receive unfair
market advantage and, therefore, remains consistent with the spirit and
the intent of the Orphan Drug Act.
In addition, H.R. 4712 clears barriers for innovative medication-
assisted treatments, or MATs, coming to market that will help treat
those with substance use disorder. Substance use disorder is by no
means a rare disease and should not be treated as such. Medication-
assisted treatments can and do save lives.
According to the National Institute on Drug Abuse, in 2016 more than
2.1 million Americans were living with opioid use disorder, but just
over 17 percent of people received specialty treatment. Medication-
assisted treatment is one of those personalized options. We must work
to ensure more people can gain access to newer therapies and MAT
treatments that are currently blocked due to an orphan designation.
Mr. Speaker, I thank the bipartisan group of legislators who
introduced this bill with me: Congressmen Buddy Carter, Marc Veasey,
and David McKinley, as well as Chairman Pallone and Ranking Member
Burgess, for supporting the bill, and passing it unanimously out of the
Energy and Commerce Committee.
Mr. Speaker, I urge all Members to support this bill.
Mr. WALDEN. Mr. Speaker, I yield 2 minutes to the gentleman from
Georgia (Mr. Carter), who is one of the coauthors of this very
important legislation and who is the only--I think still only--
pharmacist in the United States House of Representatives.
Mr. CARTER of Georgia. Mr. Speaker, I thank the gentleman for
yielding.
Mr. Speaker, I am grateful for the time today to let me speak on this
important legislation, and I thank Congresswoman Dean for introducing
it.
I am glad to be a lead Republican on this bill, as it corrects a
loophole in the Orphan Drug Act that has been and very well could be
taken advantage of at the expense of the American people's health.
As you know, Mr. Speaker, the Orphan Drug Act provides incentives for
drug manufacturers to invest in research to bring innovative drugs to
market that may not become profitable or that treat a small portion of
the population.
Unfortunately, a loophole exists that allows some drugs to obtain
market exclusivity even though they can easily recoup their R&D costs
and turn a profit. This exact problem took place in just the past few
years when a drug treating opioid abuse disorders got FDA approval--
orphan status--and a new 7-year exclusivity period, despite the active
ingredient remaining the same, all based on the drug's original 1994
orphan designation. This subsequently blocked a new, innovative opioid
abuse drug from coming to market during the opioid epidemic--a drug
that would help save lives.
The Orphan Drug Fairness Act would stop some drugs from obtaining
exclusivity, in turn allowing more competition and innovation in the
marketplace, benefiting consumers' health and lowering costs.
Mr. Speaker, I urge passage of this legislation.
Mr. WALDEN. Mr. Speaker, I do not have any more speakers on my side
of the aisle on this matter.
Mr. Speaker, I urge colleagues to approve the bill, and I yield back
the balance of my time.
Mr. PALLONE. Mr. Speaker, I have no additional speakers, I urge
passage of the bill, and I yield back the balance of my time.
Ms. ESHOO. Mr. Speaker, I rise in support of H.R. 4712, the Fairness
in Orphan Drug Exclusivity Act. I'm proud to have advanced this
bipartisan bill through my Health Subcommittee and I'm proud to support
it on the Floor today.
The Fairness in Orphan Drug Exclusivity Act was introduced by Reps.
Madeline Dean (D-PA), Marc Veasey (D-TX), Buddy Carter (R-GA), and
David McKinley (R-WV).
The bill will close a loophole so that orphan drug exclusivity cannot
be used to deny access to certain drugs, especially drugs for opioid
use disorder.
This is a narrowly drawn bill to fix a technical problem without
hurting the original intention of the Orphan Drug Act. It requires drug
companies to show that they will not recoup costs each year in order to
achieve the orphan drug designation.
I urge my colleagues to support this legislation.
Mr. McKINLEY. Mr. Speaker, I rise in support of H.R. 4712.
Understandably, our nation has focused on COVID, but the opioid
epidemic still ravishes across America. During this crisis overdose
rates have increased dramatically. In fact, in West Virginia more
people have died from drug abuse than from COVID.
We have a duty to our constituents to ensure that all possible
treatment options are available. MAT (Medication Assisted Treatment),
has been proven to be effective in treating opioid addiction. Yet, drug
companies are holding new treatments hostage through a
[[Page H5809]]
loophole in the Orphan Drug Act, which was created to encourage drug
companies to research treatments for rare diseases.
It was not intended to prevent competition. With millions of
Americans suffering from opioid addiction, it is vital we give them and
health care providers every option available. The Fairness in Orphan
Drug Exclusivity Act will help expand access for those suffering from
addiction by making innovative treatments available.
I urge my colleagues to support the passage of H.R. 4712.
The SPEAKER pro tempore. The question is on the motion offered by the
gentleman from New Jersey (Mr. Pallone) that the House suspend the
rules and pass the bill, H.R. 4712, as amended.
The question was taken; and (two-thirds being in the affirmative) the
rules were suspended and the bill, as amended, was passed.
A motion to reconsider was laid on the table.
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