[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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