[Congressional Record Volume 148, Number 126 (Tuesday, October 1, 2002)]
[House]
[Pages H6812-H6815]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]
RARE DISEASES ACT OF 2002
Mr. SHIMKUS. Mr. Speaker, I move to suspend the rules and pass the
bill (H.R. 4013) to amend the Public Health Service Act to establish an
Office of Rare Diseases at the National Institutes of Health, and for
other purposes.
The Clerk read as follows:
H.R. 4013
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 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. The Office of Rare
Diseases at the National Institutes of Health was created in
1993, but lacks a statutory authorization.
(7) The National Institutes of Health has received a
substantial increase in research funding from Congress for
the purpose of expanding the national investment of the
United States in behavioral and biomedical research.
(8) Notwithstanding such increases, funding for rare
diseases and disorders at the National Institutes of Health
has not increased appreciably.
(9) To redress this oversight, the Department of Health and
Human Services has proposed the establishment of a network of
regional centers of excellence for research on rare diseases.
(b) Purposes.--The purposes of this Act are to--
(1) amend the Public Health Service Act to establish an
Office of Rare Diseases at the National Institutes of Health;
and
(2) increase the national investment in the development of
diagnostics and treatments for patients with rare diseases
and disorders.
SEC. 3. NIH OFFICE OF RARE DISEASES AT NATIONAL INSTITUTES OF
HEALTH.
Title IV of the Public Health Service Act (42 U.S.C. 281 et
seq.), as amended by Public Law 107-84, is amended by
inserting after section 404E the following:
``office of rare diseases
``Sec. 404F. (a) Establishment.--There is established
within the Office of the Director of NIH an office to be
known as the Office of Rare Diseases (in this section
referred to as the `Office'), which shall be headed by a
Director (in this section referred to as the `Director'),
appointed by the Director of NIH.
``(b) Duties.--
``(1) In general.--The Director of the Office shall carry
out the following:
``(A) The Director shall recommend an agenda for conducting
and supporting research on rare diseases through the national
research institutes and centers. The agenda shall provide for
a broad range of research and education activities, including
scientific workshops and symposia to identify research
opportunities for rare diseases.
``(B) The Director shall, with respect to rare diseases,
promote coordination and cooperation among the national
research institutes and centers and entities whose research
is supported by such institutes.
``(C) The Director, in collaboration with the directors of
the other relevant institutes and centers of the National
Institutes of Health, may enter into cooperative agreements
with and make grants for regional centers of excellence on
rare diseases in accordance with section 404G.
``(D) The Director shall promote the sufficient allocation
of the resources of the National Institutes of Health for
conducting and supporting research on rare diseases.
``(E) The Director shall promote and encourage the
establishment of a centralized clearinghouse for rare and
genetic disease information that will provide understandable
information about these diseases to the public, medical
professionals, patients and families.
``(F) The Director shall biennially prepare a report that
describes the research and education activities on rare
diseases being conducted or supported through the national
research institutes and centers, and that identifies
particular projects or types of projects that should in the
future be conducted or supported by the national research
institutes and centers or other entities in the field of
research on rare diseases.
``(G) The Director shall prepare the NIH Director's annual
report to Congress on rare disease research conducted by or
supported through the national research institutes and
centers.
``(2) Principal advisor regarding orphan diseases.--With
respect to rare diseases, the Director shall serve as the
principal advisor to the Director of NIH and shall provide
advice to other relevant agencies. The Director shall provide
liaison with national and international patient, health and
scientific organizations concerned with rare diseases.
``(c) Definition.--For purposes of this section, the term
`rare disease' means any disease or condition that affects
less than 200,000 persons in the United States.
``(d) Authorization of Appropriations.--For the purpose of
carrying out this section, there are authorized to be
appropriated such sums as already have been appropriated for
fiscal year 2002, and $4,000,000 for each of the fiscal years
2003 through 2006.''.
SEC. 4. RARE DISEASE REGIONAL CENTERS OF EXCELLENCE.
Title IV of the Public Health Service Act (42 U.S.C. 281 et
seq.), as amended by section 3, is further amended by
inserting after section 404F the following:
``rare disease regional centers of excellence
``Sec. 404G. (a) Cooperative Agreements and Grants.--
``(1) In general.--The Director of the Office of Rare
Diseases (in this section referred to as the `Director'), in
collaboration with the directors of the other relevant
institutes and centers of the National Institutes of Health,
may enter into cooperative agreements with and make grants to
public or private nonprofit entities to pay all or part of
the cost of planning, establishing, or strengthening, and
providing basic operating support for regional centers of
excellence for clinical research into, training in, and
demonstration of diagnostic, prevention, control, and
treatment methods for rare diseases.
``(2) Policies.--A cooperative agreement or grant under
paragraph (1) shall be entered into in accordance with
policies established by the Director of NIH.
``(b) Coordination With Other Institutes.--The Director
shall coordinate the activities under this section with
similar activities conducted by other national research
institutes, centers and agencies of the National Institutes
of Health and by the Food and Drug Administration to the
extent that such institutes, centers and agencies have
responsibilities that are related to rare diseases.
``(c) Uses for Federal Payments Under Cooperative
Agreements or Grants.--Federal payments made under a
cooperative agreement or grant under subsection (a) may be
used for--
``(1) staffing, administrative, and other basic operating
costs, including such patient care costs as are required for
research;
``(2) clinical training, including training for allied
health professionals, continuing
[[Page H6813]]
education for health professionals and allied health
professions personnel, and information programs for the
public with respect to rare diseases; and
``(3) clinical research and demonstration programs.
``(d) Period of Support; Additional Periods.--Support of a
center under subsection (a) may be for a period of not to
exceed 5 years. Such period may be extended by the Director
for additional periods of not more than 5 years if the
operations of such center have been reviewed by an
appropriate technical and scientific peer review group
established by the Director and if such group has recommended
to the Director that such period should be extended.
``(e) Authorization of Appropriations.--For the purpose of
carrying out this section, there are authorized to be
appropriated such sums as already have been appropriated for
fiscal year 2002, and $20,000,000 for each of the fiscal
years 2003 through 2006.''.
The SPEAKER pro tempore. Pursuant to the rule, the gentleman from
Illinois (Mr. Shimkus) and the gentleman from Texas (Mr. Green) 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 include extraneous material on this legislation.
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, I would like to commend the distinguished gentleman from
Louisiana (Chairman Tauzin) and my good friend, the gentleman from
Florida (Mr. Foley), for their work in bringing attention to rare
diseases and disorders.
In the United States today, one in nine Americans suffer from a known
rare disease or disorder for which there is often no good treatment or
cure. The legislation sponsored by my colleagues works to correct that
problem. Taken together, the Rare Diseases Act and its companion bill,
the Rare Disease Orphan Product Development Act, encourage the
development of better treatments, diagnostic procedures, and cures for
large numbers of rare diseases and disorders.
There are over 6,000 known rare diseases, and although each of them
individually affects less than 200,000 people, the total number of
Americans affected is over 25 million people. These acts build on the
success of the Orphan Drug Act of 1983, which has led to the
development of over 220 treatments for rare diseases and disorders,
including Huntington's disease, Lou Gehrig's disease, and Tourette
syndrome.
Still, patients with rare diseases continue to face challenges in
receiving appropriate and adequate treatment. The National Commission
on Orphan Diseases estimated that only one-third of patients receive an
accurate diagnosis in the 3 to 5 years after the onset of symptoms, and
50 percent of the population is not accurately diagnosed until 7 or
more years after the onset of symptoms.
Research into rare diseases and disorders provides hope for thousands
of Americans and their families. This legislation does not detract from
other worthy congressional research priorities of the NIH, such as the
Children's Health Act of 2000. Instead, these bills increase funding
for two programs that have already had a direct and positive impact on
this community. They expand and enhance existing research under way at
various institutes of the NIH.
Again, I am pleased to support passage of these two pieces of
legislation and stand ready to work with my esteemed colleagues to
ensure that they are enacted into law.
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 rise in support of H.R. 4013, the Rare Diseases Act of
2002. I am proud to have introduced this piece of legislation with my
colleague, the gentleman from Illinois (Mr. Shimkus). This is a bill
which would work to the benefit of those suffering from rare diseases
both by establishing an Office of Rare Diseases at the National
Institutes of Health, and by providing for rare disease regional
centers of excellence.
A rare disease is defined in the United States as one affecting fewer
than 200,000 Americans. There are around 6,000 known rare diseases, and
it is estimated that about 25 million Americans are affected by them.
Over 220 treatments have been developed in the last two decades for
rare diseases, but many more are needed.
The Office of Rare Diseases at the National Institutes of Health was
established in 1993 to promote research and provide information.
However, this office was not given any authority. Also, although
Congress has substantially increased research funding for NIH, funding
for rare diseases has only increased slightly. This legislation
increases to $25 million the Orphan Product Research Grant Program
administered by the Office of Orphan Product Development at the Food
and Drug Administration, thus encouraging more extensive research,
testing, and attention.
The Rare Diseases Act of 2002 establishes the Office of Rare Diseases
as a Federal office, including its ability to coordinate research and
establish regional centers of excellence for clinical research.
This is an important piece of legislation, and I urge all my
colleagues to join me in supporting its passage.
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), who has been an ardent spokesperson
and supporter of this legislation.
Mr. FOLEY. Mr. Speaker, I thank the gentleman for yielding time to
me. I thank the gentleman from Illinois (Mr. Shimkus) for his
leadership on these very important pieces of legislation today, as well
as the gentleman from California (Mr. Waxman) for his many, many years
of outstanding service on the Subcommittee on Health.
Mr. Speaker, many people are unfamiliar with some of the diseases
that were mentioned earlier. Today in Palm Beach County, there are a
lot of diseases that have tremendous charity organizations helping to
support them, to raise money. In fact, there is a ball a day that is
pretty much dedicated to raising resources and to not only find
research dollars, but hopefully find cures for diseases that ravage
mankind, but most all of them are popular and well known. They may be
AIDS, Alzheimer's, things that people are well familiar with.
Regrettably, the rare diseases do not have the same fan club. They do
not have the same outreach, and they certainly do not have the same
support network as some of the bigger charities are fortunate to have
in my district and throughout the country. That is why it is critically
important to pass both of these pieces of legislation today, because
they create the framework to bring about an educational process, to
create the framework to channel resources into the National Institutes
of Health and other entities in order to find the potential cures, as
well as the subsequent bill we will talk about that helps to provide,
if you will, the kind of dollars necessary for pharmaceutical companies
and others to be able to pursue what is not a profitable research path,
but is a research path, nonetheless, that yields great results to the
person suffering.
So again, I commend the gentleman from Illinois (Mr. Shimkus), and I
am certain the citizens of Illinois appreciate the fact that he is on
this very important subcommittee of the Committee on Commerce dealing
with the health care of many millions of Americans who are silent on
the floor today, but are watching with great anticipation as we
hopefully unlock the key to one of the many doors that block some of
the research available.
Hopefully with these bills we will see an outpouring of support not
only into research endeavors, but also into long-term sustainability of
the lives of these very important Americans we are speaking about
today.
Mr. WAXMAN. Mr. Speaker, I am pleased to yield such time as he may
consume to the gentleman from Illinois (Mr. Davis).
Mr. DAVIS of Illinois. Mr. Speaker, I want to, first of all, thank
the gentleman from California (Mr. Waxman) for yielding time to me, and
commend him and the gentleman from Illinois (Mr. Shimkus) for
introducing this legislation.
[[Page H6814]]
I also want to thank the former commissioner of the Chicago
Metropolitan Water Reclamation District, the distinguished JoAnn Alter,
for bringing this matter to my attention. Therefore, I rise in support
of H.R. 4013, the Rare Diseases Act of 2002.
Mr. Speaker, with a large low-income population, 24 hospitals, 5
medical schools, and several research institutions in my district, I
know firsthand the heartbreak faced by people who struggle to find the
appropriate medical treatment for themselves and their families.
We have made tremendous strides in education, research, and medical
protocols for individuals with diseases that affect large populations.
Much funding has been dedicated and continues to be directed to
treatment of diseases such as cancer, heart disease, and diabetes, and
this is all good.
There are, however, a number of rare diseases which affect 200,000 or
fewer Americans which continue to go underresourced. While
statistically 200,000 people may be a small number, it is a large
number when we consider it represents people needing medical treatment.
However, if we aggregate the number of people suffering from at least 1
of the 6,000 known rare diseases and disorders, we are talking about 25
million Americans, 1 in 9, suffering from a rare disease.
Several months ago a mother and her young son, who suffers from
Crohn's disease, traveled hundreds of miles from Virginia to
Northwestern Memorial Hospital in my district to see if he could be
accepted into a special treatment program that was offered nowhere near
his home. He wrote a letter to me thanking me for the fact that he was
indeed able to get into Northwestern and to be considered for treatment
for his very rare disease.
This bill, H.R. 4013, which establishing an Office of Rare Diseases
at the National Institutes of Health, by increasing the national
investment in the development of diagnostics and treatment for patients
with rare diseases and disorders, and by allowing for rare disease
regional centers of excellence, is a quantum leap in the right
direction. I again commend my colleagues for its introduction and urge
swift passage of this resolution.
{time} 1330
Mr. WAXMAN. Mr. Speaker, I have no further requests for time, and I
yield back the balance of my time.
Mr. KIRK. Mr. Speaker, I rise today in strong support of H.R. 4013,
the Rare Disease Act of 2002. Currently, more than 6,000 rare diseases
affect 25 million Americans each year. While some progress has been
made to fight rare diseases over the last twenty years, we must commit
greater resources to this effort, particularly through the National
Institutes of Health.
My own constituents have been very active in this fight. Families of
Spinal Muscular Atrophy, based in Libertyville, Illinois, have been
working to enhance research efforts of this and other rare diseases
since 1984. I commend Audrey Lewis and all of the Families of SMA's
staff and volunteers for their continued dedication and hard work in
this field.
SMA is just one of the thousands of rare diseases that continues to
impact American families, and is the number one genetic killer of
children under the age of two. A child of parents who both carry the
SMA gene has a one in four chance of developing this devastating
disease. Inflicted children are forced to live with neuromuscular
deterioration that can affect crawling, walking, head control and
swallowing.
We must make every effort to expand research efforts so that those
with SMA and other rare diseases have a change to live long and healthy
lives. I hope that Congress can send a united message of the importance
of enhanced research efforts in this field by supporting this bill. An
Office of Rare Diseases at the National Institutes of Health is an
essential element in our efforts to raise awareness and research for
SMA and the thousands of other rare diseases affecting Americans. I
urge my colleagues to support H.R. 4013.
Mr. PITTS. Mr. Speaker, the legislation before us today represents
the latest steps in Congress' twenty year commitment to Americans with
rare ``orphan'' diseases. Since its passage in 1983, the Orphan Drug
Act has stimulated the development of 231 new marketed orphan products,
with several hundred more in the pipeline for which we all have great
hopes. Yet, there are still more than 5,000 rare diseases with no
specialized treatment at all. For this reason, H.R. 4013 and H.R. 4014
represent major advances in stimulating new therapies for those with
rare diseases, as well as improved care.
It is important to note, however, that even those 231 marketed orphan
drugs, biologics, foods and devices are not always readily available to
patients because of geographical and insurance barriers. One of the
unfinished pieces of business facing this Congress is to make sure that
Medicare is not contributing to this problem.
Three years ago, when Congress created the Medicare Hospital
Outpatient Prospective Payment System, known as HOPPS, Congress placed
all orphan products into a pass-through category where they would be
paid at a higher rate. Even still, many hospitals have lost money when
they stocked orphan drugs to treat patients with rare diseases.
Now we are faced with a situation where CMS has proposed a regulation
for the 2003 HOPPS program that leaves most orphan drugs, biologicals
and blood plasma therapies and their recombinant analogs such as
clotting factors for individuals with Hemophilia without adequate
reimbursement. Many hospitals will refuse to stock these drugs because
of the large loss they will incur for treating a small number of
patients. Without appropriate reimbursement, patients may be turned
away from hospital emergency rooms or directed to alternative
facilities if the location does not stock their product. The
consequences of inadequate or non-existent access would be devastating,
particularly in rural areas where the nearest hospital to stock a
particular orphan drug may be a hundred or more miles away.
Mr. Speaker, it is critical that the House of Representatives act
this year to ensure that orphan drugs are properly reimbursed.
Mr. KIND. Mr. Speaker, I rise in support of both the Rate Diseases
Act (H.R. 4013) and Rare Diseases Orphan Product Development Act (H.R.
4014). A rare disease is defined as one that afficts fewer than 200,000
individuals. Our country has over 6,000 rare diseases that affect more
than 25 millions Americans.
Both of these measures will provide necessary incentives to find a
cure for these ailments. H.R. 4013 establishes an Office of Rare
Diseases within the National Institutes of Health director's office. In
addition, the bill creates Rare Disease Regional Centers of Excellence
to conduct research and training in the diagnosis, prevention, control
and treatment of rare diseases. H.R. 4014 would double the funding to
$25 million for the successful FDA grant program for research on orphan
drugs. Since 1983, the FDAa has approved more than 200 treatments for
rare diseases, this additional funding will be critical to increasing
the number of treatments available.
A wonderful family in my hometown, the Kirches, brought the issue of
rare diseases to my attention. I have had the opportunity to meet with
Larry, Susan and their daughter Allyson to hear about their struggles
and triumphs with Allyson's battle with Mucopolysachharidosis (MPS)
III. MPS III is a genetic disorder that results in the body's inability
to produce certain enzymes. This lack of enzyme production interrupts
the usual breakdown of complex carbohydrates that are stored in almost
every cell in the body. Without the breakdown, storage progressively
builds in each cell causing damage in multiple systems within the body
including respiratory, bones, internal organs, and nervous system. The
results of this damage include mental retardation, short stature,
cornea damage, loss of mobility, and most importantly a drastically
shortened life span. At present there is no cure for MPS III.
Allyson's future depends on investment in scientific and biomedical
research by the public and private sector and, we owe it to all
children with these disorders to make every effort to improve their
quality of life and ultimately contribute to efforts in developing
effective treatments. I urge my colleagues to support these measures
and assist families like the Kirches all across our country.
Mr. DINGELL. Mr. Speaker, I rise in strong support of H.R. 4013, the
``Rare Diseases Act of 2002,'' of which I am proud to be an original
cosponsor. I would like to thank my colleagues on both sides of the
aisle for working together to introduce this important legislation and
for working on behalf of the 25 million American people who suffer from
rare diseases.
Rare, or orphan diseases affect fewer than 200,000 individuals in
America. Nearly one million people in my home state of Michigan are
afflicted with a rare disease. There are more than 6,000 rare diseases.
Enactment of the Orphan Drug Act of 1983 provided incentives for drug
and biological manufacturers to invest in treatment for rare diseases.
While the Orphan Drug Act has achieved dramatic increases in research
into, and treatments for rare diseases, more still needs to be done.
One positive step includes authorizing, in statute, the Office of Rare
Diseases.
In addition, H.R. 4013 authorizes regional centers of excellence for
rare disease research. This will enable the National Institutes of
Health (NIH) to select sites to concentrate on finding cures and
treatment methods for rare diseases.
I urge all of my colleagues to join me in support of H.R. 4013.
[[Page H6815]]
Mr. BILIRAKIS. Mr. Speaker, I rise today in support of H.R. 4013, the
Rare Diseases Act of 2002. This bill, which was introduced by the
gentleman from Illinois, Mr. Shimkus, would help improve research on
rare diseases at the National Institutes of Health. I urge my
colleagues to join me today in supporting this important piece of
legislation.
A rare or ``orphan'' disease affects fewer than 200,000 people in the
United States. There are more than 6,000 rare disorders that, taken
together, affect approximately 25 million Americans. One in every 10
individuals in this country has received a diagnosis of a rare disease.
H.R. 4013 will help focus research on rare diseases at NIH. The bill
also specifically gives NIH the authority to support regional centers
of excellence in rare disease research. This bill will help strengthen
our national research infrastructure in this area and improve our
ability to treat and hopefully cure numerous rare diseases.
The Energy and Commerce Committee approved H.R. 4013 in late June,
and I again urge my colleagues to support this important bill.
Mr. SHIMKUS. Mr. Speaker, I have no further requests for time, 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. 4013.
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.
____________________