[Congressional Record Volume 148, Number 126 (Tuesday, October 1, 2002)]
[House]
[Pages H6815-H6818]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]
RARE DISEASES ORPHAN PRODUCT DEVELOPMENT ACT OF 2002
Mr. SHIMKUS. Mr. Speaker, I move to suspend the rules and pass the
bill (H.R. 4014) to amend the Federal Food, Drug, and Cosmetic Act with
respect to the development of products for rare diseases.
The Clerk read as follows:
H.R. 4014
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 ``Rare Diseases Orphan Product
Development Act of 2002''.
SEC. 2. FINDINGS AND PURPOSES.
(a) Findings.--Congress makes the following findings:
(1) Rare diseases and disorders are those which affect
small patient populations, typically populations smaller than
200,000 individuals in the United States. Such diseases and
conditions include Huntington's disease, amyotrophic lateral
sclerosis (Lou Gehrig's disease), Tourette syndrome, Crohn's
disease, cystic fibrosis, cystinosis, and Duchenne muscular
dystrophy.
(2) For many years, the 25,000,000 Americans suffering from
the over 6,000 rare diseases and disorders were denied access
to effective medicines because prescription drug
manufacturers could rarely make a profit from marketing drugs
for such small groups of patients. The prescription drug
industry did not adequately fund research into such
treatments. Despite the urgent health need for these
medicines, they came to be known as ``orphan drugs'' because
no companies would commercialize them.
(3) During the 1970s, an organization called the National
Organization for Rare Disorders (NORD) was founded to provide
services and to lobby on behalf of patients with rare
diseases and disorders. NORD was instrumental in pressing
Congress for legislation to encourage the development of
orphan drugs.
(4) The Orphan Drug Act created financial incentives for
the research and production of such orphan drugs. New Federal
programs at the National Institutes of Health and the Food
and Drug Administration encouraged clinical research and
commercial product development for products that target rare
diseases. An Orphan Products Board was established to promote
the development of drugs and devices for rare diseases or
disorders.
(5) Before 1983, some 38 orphan drugs had been developed.
Since the enactment of the Orphan Drug Act, more than 220 new
orphan drugs have been approved and marketed in the United
States and more than 800 additional drugs are in the research
pipeline.
(6) Despite the tremendous success of the Orphan Drug Act,
rare diseases and disorders deserve greater emphasis in the
national biomedical research enterprise.
(7) The Food and Drug Administration supports small
clinical trials through Orphan Products Research Grants. Such
grants embody successful partnerships of government and
industry, and have led to the development of at least 23
drugs and four medical devices for rare diseases and
disorders. Yet the appropriations in fiscal year 2001 for
such grants were less than in fiscal year 1995.
(b) Purposes.--The purpose of this Act is to increase the
national investment in the development of diagnostics and
treatments for patients with rare diseases and disorders.
SEC. 3. FOOD AND DRUG ADMINISTRATION; GRANTS AND CONTRACTS
FOR THE DEVELOPMENT OF ORPHAN DRUGS.
Subsection (c) of section 5 of the Orphan Drug Act (21
U.S.C. 360ee(c)) is amended to read as follows:
``(c) For grants and contracts under subsection (a), there
are authorized to be appropriated such sums as already have
been appropriated for fiscal year 2002, and $25,000,000 for
each of the fiscal years 2003 through 2006.''.
SEC. 4. TECHNICAL AMENDMENT.
Section 527(a) of the Federal Food, Drug, and Cosmetic Act
(21 U.S.C. 360cc(a)) is amended in the matter following
paragraph (2)--
(1) by striking ``, of such certification,''; and
(2) by striking ``, the issuance of the certification,''.
The SPEAKER pro tempore. Pursuant to the rule, the gentleman from
Illinois (Mr. Shimkus) and the gentleman from California (Mr. Waxman)
each will control 20 minutes.
The Chair recognizes the gentleman from Illinois (Mr. Shimkus).
General Leave
Mr. SHIMKUS. Mr. Speaker, I ask unanimous consent that all Members
may have 5 legislative days within which to revise and extend their
remarks and to insert extraneous material on the bill, H.R. 4014.
The SPEAKER pro tempore. Is there objection to the request of the
gentleman from Illinois?
There was no objection.
Mr. SHIMKUS. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, this is really my first day ever managing a bill on the
floor of the House. I have done a lot of other things from speaking to
presiding but never actually managing a bill; and it is really
appropriate that this legislation that we just took up, H.R. 4013, and
this piece of legislation, H.R. 4014, I cannot think of a better bill
to have a chance to manage. And so I thank my chairman and the
subcommittee chairman for doing that.
Mr. Speaker, I also want to take this time to say that it is an honor
to be managing with my friend, the gentleman from California (Mr.
Waxman), because many times we are opponents on the legislative battles
and the agenda. One of the great things about this institution is when
we can work together from across the political divide and ideological
divide to find issues that we become impassioned about and we join in
forces that really help move legislation. So I extend my thanks to my
friend from California (Mr. Waxman) and this will be a memorable day
for me as I think back on my congressional career.
Mr. Speaker, I rise today in support of this legislation, H.R. 4014,
the Rare Disease Orphan Product Development Act of 2002. This bill is
sponsored by my good friend, the gentleman from Florida (Mr. Foley),
who will join us in a minute, and the gentleman from California (Mr.
Waxman), and will increase the authorization for grants given to
researchers who are developing cures and treatments for rare diseases.
With more money available to these researchers, we will be better able
to find cures for the 6,000 rare diseases affecting nearly 25 million
Americans. And when you know a family who has someone affected by rare
diseases, it does not seem that rare because it is time consuming, it
is costly, and it makes you really be passionate about making sure
everybody has some help in trying to find cures and drugs to help them
alleviate the onset of their disease.
Prior to the passage of the Orphan Drug Act in 1983, only a handful
of drug and biologics had been developed to treat rare diseases. The
reasons for this were simple. There was very little economic incentive
for drug companies to spend the hundreds of millions of dollars it
takes to develop a drug for a patient population totalling in the
thousands. That is why prior to 1983 only 38 drugs had been developed
for rare diseases.
[[Page H6816]]
The Orphan Drug Act changes this by doing three things: offering
greater exclusivity for drugs designed to treat diseases affecting
fewer than 200,000 Americans, the establishing of a grant program for
researchers performing clinical trials on orphan drugs, and providing a
tax incentive program.
The Orphan Drug Act has been a resounding success. Whereas fewer than
40 drugs have been developed in the past for rare diseases, in the past
few decades more than 200 drugs have been developed and approved to
treat these diseases. The bill before us today reauthorizes a grant
program contained within the bill. Presently the government funds fewer
than 100 researchers performing clinical trials into rare diseases,
cures and therapies. While the demand is much higher, funding has been
limited. This bill reauthorizes the grant program at 25 million in
fiscal years 2003 through 2006, meaning more monies will be available
to finding cures for these diseases.
Mr. Speaker, I rise in strong support of this bill and commend the
gentleman from Florida (Mr. Foley) and the gentleman from California
(Mr. Waxman) for their sponsorship of this legislation.
Mr. Speaker, I reserve the balance of my time.
Mr. WAXMAN. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, I also rise in support of this bill, and I am honored to
be a co-sponsor with the gentleman from Florida (Mr. Foley) and the
gentleman from Illinois (Mr. Shimkus) in this legislation.
This bill and the previous one are two bills that are furthering the
cause of developing drugs for people with rare diseases. It is very
important for the progress of research on treatments and to find cures
for rare diseases. There are 6,000 rare diseases that affect
approximately 25 million Americans. These diseases include cystic
fibrosis, Lou Gehrig's disease and muscular dystrophy, to mention three
of the well-known diseases that affect less than 200,000 people, and,
therefore, are designated as rare.
The availability of safe and effective treatments for rare diseases
has historically been limited due to the lack of incentive for
pharmaceutical firms to commercialize such medications. To address this
problem, Congress passed the Orphan Drug Act, which allows for market
exclusivity for products developed for rare diseases, as well as
special tax treatment for the companies that are willing to make that
investment.
In addition, the Food and Drug Administration supports small clinical
trials through orphan products research grants. These grants have led
to the development of at least 23 drugs and four medical devices for
rare diseases. The purpose of this legislation is to increase the
national investment in the development of diagnostics and treatments
for people with rare diseases.
H.R. 4014 is for the funding of the Orphan Product Research Grant
Program, and increases the national investment in the development of
diagnostics and treatment for patients with these rare diseases. It is
a good piece of legislation. I am pleased and honored to join with my
colleagues on a bipartisan basis. There should be no partisanship or
conflict that we see on other issues when it comes to trying to help
Americans overcome the terror of diseases that afflict them and is such
a burden to their families. I urge all of my colleagues to join all of
us in supporting H.R. 4014.
Mr. Speaker, I reserve the balance of my time.
Mr. SHIMKUS. Mr. Speaker, I yield such time as he may consume to the
gentleman from Florida (Mr. Foley), a major sponsor of the legislation.
Mr. FOLEY. Mr. Speaker, I congratulate the gentleman on his
management of this important piece of legislation.
If I were Speaker of the House, I would probably declare that the
only business on the House floor would be health care legislation
because the bipartisanship displayed in this very important legislation
is really indicative of the heart and soul of this Chamber.
Many people see us in vigorous debate over issues and why they cannot
get along. Yet today you see the gentleman from Illinois (Mr. Shimkus)
and the gentleman from California (Mr. Waxman) and myself all really
focused on people who have been heard by us but have not been heard by
society in general.
Today I rise in strong support of our mutually agreed-upon bill, H.R.
4014, the Rare Disease Orphan Product Development Act of 2002. This
important piece of bipartisan legislation will encourage better
treatment, diagnostic procedures, and cures for large numbers of rare
diseases and disorders.
The gentleman from California (Mr. Waxman) mentioned the statistics,
25 million people suffering from more than 6,000 rare diseases. A rare
disease, to underscore, is one that affects the population under
200,000 people, or about one in 11 Americans.
Mr. Speaker, I would like to mention just one of those Americans, a
little girl fighting for her life. Her name is Madison, but her parents
call her Maddy. She is a 5-year-old constituent of mine who contracted
MPS 1, Hurler Syndrome, one year after she was born. This horrible
disease causes shortness of stature, mental retardation, speech and
hearing impairments, heart disease, and worst of all a shortened life
span.
As with most persons suffering from rare diseases, her situation is
made worse because there may be only one or two doctors in the world
working on a cure for her disease. Our bill would double the amount
currently authorized for the Orphan Product Development Grant Program
from $12 million to $25 million per year. This grant program is
considered one of the most successful programs at the Food and Drug
Administration. To date, 23 drugs and four medical devices have been
developed as a direct result of this medical program, 23 drugs and four
medical devices. This is an extraordinary achievement, given these
products are generally not financially profitable for the companies
that make them.
Let me stop there and go just a bit off text because during the
political season it is very, very easy to beat up the pharmaceutical
industry. It seems to be a target on both sides of the aisle. And yet
today we are talking about companies that truly do God's work here on
Earth. They are working hard to develop the kind of resources and
procedures, medications and things necessary to save lives. So while we
can have our quarrel and disagreement with the industry over certain
items, I do think it appropriate that on occasion we speak up for their
great research. We are the envy of the world in development of
products, pharmaceutical aids, and other things in this country. We are
the envy of the world, and we should not lose sight that scientists are
working collaboratively with some of these well-known brand-name
manufacturers who are in fact shining some light and hopefully some
hope for the people suffering.
Mr. Speaker, this medical minority cannot be ignored any longer. We
cannot afford to keep paying 50 cents on the dollar when these patients
are feeling 100 percent of the pain. It is time to raise the level of
awareness of these diseases once and for all.
Mr. Speaker, I want to thank the gentleman from Illinois (Mr.
Shimkus) for introducing H.R. 4013, the Rare Disease Act of 2002, which
would permanently establish the Office of Rare Diseases at the National
Institutes of Health. His bill will allow for continued research and
information-sharing among those scientists and doctors who are in the
front lines of finding cures with these horrible diseases.
I would like to thank the gentleman from Louisiana (Mr. Tauzin), the
gentleman from Florida (Mr. Bilirakis), and the gentleman from
California (Mr. Waxman), the ranking member, for their extraordinary
efforts for bringing this issue to the national spotlight and for
bringing these bills to the floor. I also want to commend my staff
legislative counsel, Bradley Shieber, who is here on the floor with me
today who brought these bills to my attention months ago. It is
actually the fastest piece of legislation in my 7\1/2\-plus years in
Congress that has come from a drafting, to a conclusion, to an
introduction on the House floor, to hopefully a successful passage
today.
Mr. Speaker, I would also like to thank Senator Edward Kennedy of
Massachusetts and Senator Orrin Hatch of Utah for their leadership on
our companion measures that reside in the other Chamber.
Again, I would thank everyone for participating in the debate. I urge
my
[[Page H6817]]
colleagues' strong approval of both bills before us, H.R. 4013 and H.R.
4014, as we proceed on these important measures today.
Mr. WAXMAN. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, I want to use this opportunity to give some historical
perspective on some of these issues with regard to the pharmaceutical
industry.
Mr. Speaker, I think that the pharmaceutical industry could be
credited with the enormous contribution they make in curing diseases.
But I think we need to recognize that when the Orphan Drug Act was
first talked about, it was due to the fact that the pharmaceutical
industry did not pay attention to people who had rare diseases because
the profit potential was not there for them to make the investment.
They were much more interested, as any business would be, in trying to
manufacture drugs that could have a widespread audience, so to speak,
to buy their product.
A lot of the work they do is based on the government investment and
research. We give money to the National Institutes of Health, and they
work with grants and contracts with leading researchers all around the
country to do the basic work. The pharmaceutical industry then takes
the benefit of that public investment and finds an application which
leads to products that they are able to market. They then get a patent
on the product. I have always regretted the fact that the public does
not get its share of the return on our investment for some of these
very same products.
But in the Orphan Drug Act we said, look, we will give you every
incentive in the pharmaceutical industry to make the investment because
we want people with rare diseases not to be ignored. So we gave them an
exclusivity. If they developed a drug for patients with rare diseases,
we gave them tax breaks. We funded research as this bill and the
previous one will do at the NIH and at the FDA, but we found that while
in most cases it barely offered any real profit numbers to be
attractive to pharmaceutical industries, they responded well to the
incentives.
{time} 1345
In some cases, there were diseases that were classified as rare
diseases which became a windfall for the pharmaceutical industry. The
pharmaceutical industry, for example, when the HIV/AIDS epidemic hit,
were able to classify their drugs as orphan drugs because the patient
population was not that large at that time.
Later it mushroomed, of course, as the epidemic progressed, and the
Congress revisited the issues through hearings as to whether we were
being much too generous to the pharmaceutical industry in giving them
the exclusivity which meant they could block competition. We were
willing to give them exclusivity for a disease that did not offer much
profit potential, but when it was extremely profitable, there was not
really any justification for that exclusivity over and above their
patents and other rights that pharmaceutical manufacturers have. But we
have never been able to take anything back from the drug companies once
they have gotten it in law, even when it was not justified for them to
have it.
There was another example of this, by the way, earlier this year. I
was involved in the original legislation to say to the manufacturers,
do the research on children when they get a drug approved, do that
research so that we can know what the needs are for children, if they
could use a certain pharmaceutical product. We tried to use a carrot
and a stick. A stick would be if they were coming up with a new drug,
FDA should require those tests before it approved the new drug, but a
carrot for those drugs that are already on the market, we gave them an
exclusivity of 6 months. Does not sound like a lot of time.
Then when we revisited the issue, it turned out that the companies
were using that exclusivity in a way to enhance their monopoly over
drugs that are widely used even though the studies for the children
required a minimal amount of investment. Not only that, they were doing
the minimal amount investment on the use for children, on drugs that
were rarely used by children, so they could get the monopoly on the
pharmaceuticals that were used by adults. And monopoly is a real
incentive for research, but it can be abusive, because after a while
monopolies are simply a way to keep out competition, and we know what
happens when there is no competition. It means consumers pay the
highest prices.
So we have some pharmaceuticals where there are wonderful drugs, the
public investment in research paid off when they were applied by the
pharmaceutical industry to get these drugs, but it meant that some
consumers could not even afford the drugs that were developed.
This bill before us today is a good one. We want to encourage the
development of drugs for rare diseases, and I commend the drug
companies for their work, but we need to keep it in perspective, that
sometimes we have to come back and review these special breaks that we
give to the companies because they are willing to take a loophole and
expand it so enormously that it outprices many consumers for their
product. We want to give them the incentive to develop the product, but
we want to let the public be able to purchase the product as well.
I take these few minutes to give some expansion of the historical
perspective on the Orphan Drug Act, the pediatric exclusivity, and we
will save for another time the abuses the Hatch-Waxman Act, which we,
hopefully we are this year going to try to end by following the example
of the U.S. Senate in stopping the loopholes that have been so abused
by pharmaceutical companies, far beyond anything that any of us ever
envisioned when we adopted the original Hatch-Waxman Act.
I ask my colleagues to join me in supporting this legislation.
Mr. Speaker, I yield back the balance of my time.
Mr. SHIMKUS. Mr. Speaker, I yield myself such time as I may consume.
I appreciate my colleague's historical background. I am a relatively
new Member still, in my sixth year, and I know there is a lot of water
underneath the bridge on a lot of these issues, and it is always good
to look back.
He has been a leader and has been helpful on orphan drugs and
pediatric exclusivity, and I am proud to have a chance to work with him
on this legislation. I look forward to the opportunity to work with him
more in the future as we try to make sure that all our citizens in this
country have access to affordable health care.
Mr. DINGELL. Mr. Speaker, I am proud to be an original cosponsor of
the bill before us today, H.R. 4014, the ``Rare Diseases Orphan Product
Development Act of 2002.'' I would like to thank my colleagues,
particularly Representatives Waxman, Brown, and Rush, for their work on
this legislation.
Approximately 25 million Americans suffer from more than 6,000 rare
diseases. These diseases include Huntington's disease, Lou Gehrig's
disease, cystic fibrosis, and Duchenne muscular dystrophy. Because of
the relatively small patient populations associated with rare diseases,
pharmaceutical firms are concerned about receiving an adequate return
on their investment in developing medications to treat them.
In response to this problem, Congress passed the Orphan Drug Act,
which allows for market exclusivity for products developed for rare
diseases. Additionally, the Food and Drug Administration (FDA) has been
able to support small clinical trials through Orphan Products Research
Grants. These grants have been effective, leading to the development of
more than 23 drugs and four medical devices for rare diseases.
The purpose of this legislation is to increase the national
investment in the development of diagnostics and treatments for
patients suffering from rare diseases. H.R. 4014 continues the Orphan
Products Research Grant program for clinical research needed to
evaluate the safety and efficacy of therapies to treat rare diseases.
Specifically, this legislation authorizes such sums as already have
been appropriated for fiscal year 2002, and $25 million for each of the
fiscal years 2003 through 2006.
This is good legislation and I urge all of my colleagues to join me
and support H.R. 4014.
Mr. BILIRAKIS. Mr. Speaker, I am also pleased today to support H.R.
4014, the Rare Diseases Orphan Product Development Act of 2002. This
bill, which was introduced by our colleague from Florida, Mr. Foley,
will ensure that cutting-edge treatments are available for a myriad of
rare diseases.
Specifically, H.R. 4014 will increase funding for the Food and Drug
Administration's Orphan Product Research Grants Program. This crucial
program provides funding to academic scientists and small companies to
conduct clinical trials on new orphan drugs, medical devices, and
medical foods for rare diseases.
[[Page H6818]]
By definition, ``orpahn products'' are treatments for rare conditions
that have small potential markets and thus are not attractive
investments for the private sector. Such treatments were not being
developed for rare diseases until the Orphan Drug Act was enacted in
1983, and it has become a highly successful government/industry
partnership. Prior to 1983, only ten orphan products had come to the
market, while more than 200 drugs and biological products for rare
diseases have been brought to market since passage of the Orphan Drug
Act.
H.R. 4014 ensures that adequate funding is available for the
development of orphan products. I commend my colleagues for their
bipartisan efforts in this area and look forward to voting for this
legislation.
Mr. SHIMKUS. Mr. Speaker, I have no other speakers on my side, and I
yield back the balance of my time.
The SPEAKER pro tempore (Mr. Boozman). The question is on the motion
offered by the gentleman from Illinois (Mr. Shimkus) that the House
suspend the rules and pass the bill, H.R. 4014.
The question was taken.
The SPEAKER pro tempore. In the opinion of the Chair, two-thirds of
those present have voted in the affirmative.
Mr. WAXMAN. Mr. Speaker, I object to the vote on the ground that a
quorum is not present and make the point of order that a quorum is not
present.
The SPEAKER pro tempore. Pursuant to clause 8, rule XX and the
Chair's prior announcement, further proceedings on this motion will be
postponed.
The point of no quorum is considered withdrawn.
____________________