[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.

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