[Congressional Record (Bound Edition), Volume 149 (2003), Part 9]
[House]
[Pages 12113-12115]
[From the U.S. Government Publishing Office, www.gpo.gov]




    COMMEMORATING 20TH ANNIVERSARY OF ORPHAN DRUG ACT AND NATIONAL 
                    ORGANIZATION FOR RARE DISORDERS

  Mr. BILIRAKIS. Mr. Speaker, I move to suspend the rules and agree to 
the concurrent resolution (H. Con. Res. 147) commemorating the 20th 
Anniversary of the Orphan Drug Act and the National Organization for 
Rare Disorders, as amended.
  The Clerk read as follows:

                            H. Con. Res. 147

       Whereas Congress passed, and President Ronald Reagan signed 
     into law on January 4, 1983, the Orphan Drug Act;
       Whereas there are many diseases and conditions, such as 
     Huntington's disease, myoclonus, ALS (Lou Gehrig's disease), 
     Tourette syndrome, and muscular dystrophy, that affect such 
     small numbers of individuals--populations under 200,000--
     residing in the United States that the diseases and 
     conditions are considered rare;
       Whereas there are 6,000 known rare diseases and conditions, 
     affecting approximately 25,000,000 Americans;

[[Page 12114]]

       Whereas these rare diseases and conditions are sometimes 
     referred to as ``orphan'' diseases and conditions, and drugs 
     (including biological products) to treat them are sometimes 
     referred to as ``orphan drugs'';
       Whereas, prior to 1983, because so few individuals were 
     affected by any one orphan disease or condition, a 
     pharmaceutical company that developed an orphan drug may have 
     reasonably expected the drug to generate relatively small 
     sales in comparison to the cost of developing the drug and, 
     therefore, to incur a financial loss;
       Whereas the object of the Orphan Drug Act was to stimulate 
     the development of orphan drugs;
       Whereas the Orphan Drug Act is one of the most successful 
     Federal laws enacted in the last 20 years;
       Whereas, prior to 1983, less than 40 therapies existed to 
     treat orphan diseases and conditions as compared today to the 
     more than 238 drugs, foods, and devices that now exist to 
     treat an estimated 11,000,000 patients in the United States;
       Whereas 85 percent of orphan drugs are being used to treat 
     serious or life-threatening diseases, 31 percent treat rare 
     forms of cancer, and approximately 50 percent are approved 
     for pediatric uses;
       Whereas the National Organization for Rare Disorders, 
     established in 1983, is a federation of voluntary health 
     organizations dedicated to helping people with orphan 
     diseases and conditions and assisting the organizations that 
     serve them; and
       Whereas the National Organization for Rare Disorders is 
     committed to the identification, treatment, and cure of 
     orphan diseases and conditions through programs of education, 
     advocacy, research, and service: Now, therefore, be it
       Resolved by the House of Representatives (the Senate 
     concurring), That the Congress celebrates the 20th 
     anniversary of the Orphan Drug Act and the National 
     Organization for Rare Disorders, and recognizes the great 
     contributions the Orphan Drug Act has made to the rare 
     disease community.

  The SPEAKER pro tempore. Pursuant to the rule, the gentleman from 
Florida (Mr. Bilirakis) and the gentleman from Maine (Mr. Allen) each 
will control 20 minutes.
  The Chair recognizes the gentleman from Florida (Mr. Bilirakis).


                             General Leave

  Mr. BILIRAKIS. 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 H. Con. Res. 147.
  The SPEAKER pro tempore. Is there objection to the request of the 
gentleman from Florida?
  There was no objection.
  Mr. BILIRAKIS. Mr. Speaker, I yield myself such time as I may 
consume.
  Mr. Speaker, I rise in support of H. Con. Res. 147 which commemorates 
the 20th anniversary of the Orphan Drug Act and the National 
Organization for Rare Disorders. This legislation, introduced by the 
gentleman from Florida (Mr. Foley), was unanimously approved by the 
Committee on Energy and Commerce on April 30 of this year.
  H. Con. Res. 147 recognizes the great contribution the Orphan Drug 
Act has made to the rare disease community. In the decade before the 
Orphan Drug Act was enacted, only 10 new treatments for orphan diseases 
were developed. In the 20 years since it was passed, more than 200 new 
drugs, foods and devices have been approved by the Food & Drug 
Administration, and an additional 900 are in various stages of 
development.
  These treatments for rare disorders treat an estimated 11 million 
patients in the United States. Eighty-five percent of orphan drugs are 
being used to treat serious or life-threatening diseases, 31 percent 
treat rare forms of cancer, and approximately 50 percent are approved 
for pediatric uses.
  Last year President Bush signed into law two important bills dealing 
with rare diseases. The Rare Diseases Act of 2002 established an Office 
of Rare Diseases at the National Institutes of Health, while the Rare 
Diseases Orphan Product Development Act of 2002 increased funding for 
the Food and Drug Administration's Orphan Product Research Grants 
Program. I am glad we are following the enactment of these important 
bills by recognizing both the original Orphan Drug Act and the National 
Organization for Rare Disorders, which has advocated tirelessly for the 
victims of rare diseases.
  Mr. Speaker, I urge all of my colleagues to support this important 
resolution.
  Mr. Speaker, I reserve the balance of my time.
  Mr. ALLEN. Mr. Speaker, I yield myself such time as I may consume.
  Mr. Speaker, I thank the gentleman from Florida (Mr. Bilirakis) for 
his work on this bill. I am proud to support H. Con. Res. 147, 
legislation which commemorates the 20th anniversary of the Orphan Drug 
Act and the National Organization for Rare Disorders.
  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 on 
January 4, 1983, which allows for incentives, such as market 
exclusivity, for products developed for rare diseases. Additionally, 
the Food and Drug Administration has been able to support small 
clinical trials through Orphan Products Research Grants. The Orphan 
Drug Act has been effective, leading to the development of more than 
183 drugs and four medical devices for rare diseases.
  I also note that the gentleman from California (Mr. Waxman) was a key 
leader in the development of the Orphan Drug Act. The success and 
longevity of the Orphan Drug Act are a tribute to his skillful work on 
this legislation 2 decades ago.
  We are also here today to recognize and commemorate the National 
Organization for Rare Disorders. NORD is a unique federation of 
voluntary health organizations dedicated to helping people with rare, 
also known as ``orphan'', diseases and assisting the organizations that 
serve them. NORD is committed to the identification, treatment and cure 
of rare disorders through programs of education, advocacy, research and 
service.
  The purpose of this legislation is to recognize the contributions 
that the Orphan Drug Act has made to the rare disease community and to 
commemorate the 20th anniversary of both the Orphan Drug Act and the 
National Organization of Rare Disorders. I urge all of my colleagues to 
join me in support of H. Con. Res. 147.
  Mr. Speaker, I reserve the balance of my time.
  Mr. BILIRAKIS. Mr. Speaker, I yield such time as he may consume to 
the gentleman from Florida (Mr. Foley), the writer of this resolution.
  Mr. FOLEY. Mr. Speaker, I thank the gentleman from Florida (Mr. 
Bilirakis), who is known as Mr. Health in Florida, for the gentleman's 
help on this important issue.
  I rise today in support of H. Con. Res. 147, a bill recognizing the 
20th anniversary of the enactment of the Orphan Drug Act and the 
creation of the National Organization for Rare Disorders.
  For many years, 25 million plus Americans suffering from more than 
6,000 rare diseases and disorders, including Huntington's disease, ALS, 
known as Lou Gehrig's disease, Tourettes syndrome and muscular 
dystrophy, were denied access to effective medicines because 
prescription drug manufacturers could rarely make a profit from 
marketing drugs for such small groups of patients.
  A rare disease is a disease that affects a population of less than 
200,000 people. Currently, one in nine Americans have a rare disease. 
Due to the low number of affected patients, the prescription drug 
industry did not adequately fund research into such treatments. Despite 
the urgent health need for these medicines, they became known as orphan 
drugs because no company would commercialize them.
  On January 4, 1983, Congress passed and President Ronald Reagan 
signed into law the Orphan Drug Act. This legislation created financial 
incentives for research and production of such orphan drugs. In 
addition, a new Federal program 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.
  As a result of this incredible law, which was produced in 
coordination

[[Page 12115]]

with Congress, the White House and the pharmaceutical industry, over 
238 drugs, biologics, foods and devices now exist, as compared to less 
than 40 therapies that existed before the law was enacted. Undoubtedly, 
this can easily be described as one of the most effective laws this 
Chamber has ever passed.
  Mr. Speaker, I would like to recognize an organization which has been 
committed to bringing a voice to the most underserved medical 
population in the world, those suffering from rare diseases, the 
National Organization for Rare Diseases.
  NORD, established soon after the signing of the Orphan Drug Act, is a 
federation of voluntary health organizations dedicated to helping 
people with orphan diseases and conditions and assisting the 
organizations that serve them.
  NORD has been and will continue to be committed to the 
identification, treatment, and cure of orphan diseases and conditions 
through programs of education, advocacy, research and service.
  Mr. Speaker, I would also like to take a moment to thank the 
gentleman from Louisiana (Chairman Tauzin), the gentleman from Florida 
(Mr. Bilirakis), the ranking member, the gentleman from California (Mr. 
Waxman), and, of course, the gentleman from Illinois (Mr. Shimkus) for 
their effective work and advocacy of this issue. I would also like to 
thank Bradley Schrieber, my legislation counsel, who joins me on the 
floor today, who first brought this legislation to my attention.
  For me, it is a personal sense of pride, because it was the first 
bill that was a freestanding bill that I authored that was signed into 
law, and I have the momentous pen in my office. I can look back on my 
career in Congress and know that in some small way we played a critical 
role in helping those voices who may be in the minority but are 
suffering just as those who are suffering from better-known diseases, 
if you will. I am delighted to be part of this, and I am glad it was 
brought to the floor today, and I encourage all of my colleagues to 
join me in recognizing this very important year for the millions of men 
and women and children in this medical minority.
  Mr. ALLEN. Mr. Speaker, I yield 2 minutes to the gentleman from 
California (Mr. Waxman).
  Mr. WAXMAN. Mr. Speaker, I ask my colleagues to join me in voting for 
H. Con. Res. 147, commemorating the 20th anniversary of the Orphan Drug 
Act and the National Organization for Rare Diseases. As the principal 
author of the Orphan Drug Act, I am extremely proud of what has been 
achieved as a result of this landmark legislation.
  Almost one in 10 of us is afflicted with what are called rare 
diseases, diseases that affect fewer than 200,000 people. And yet 
before 1983 if you were unlucky enough to suffer from such a disease, 
you had little or no hope that a treatment or cure would be developed. 
The market for a drug for an individual with a rare disease simply was 
not large enough for the pharmaceutical companies to justify the high 
cost of developing a potential treatment.
  The Orphan Drug Act was the first in a line of important laws that 
utilized creative new market incentives to encourage pharmaceutical 
innovation. No one was more important in the fight to pass orphan drug 
legislation than Abbey Meyers and the group she founded, the National 
Organization for Rare Disorders, or NORD. For the last 20 years, NORD 
has fought tirelessly for people with rare diseases, supported 
research, pushed for Federal funding and spread information about rare 
diseases. NORD's work, and the strides we have made against rare 
diseases thanks to the Orphan Drug Act, justly deserve to be honored in 
this resolution. I want to join my colleagues in urging that this 
matter be passed by the House.
  Mr. ALLEN. Mr. Speaker, I have no further requests for time, and I 
yield back the balance of my time.
  Mr. BILIRAKIS. Mr. Speaker, I have no further requests for time, and 
I yield back the balance of my time.
  The SPEAKER pro tempore. The question is on the motion offered by the 
gentleman from Florida (Mr. Bilirakis) that the House suspend the rules 
and agree to the concurrent resolution, H. Con. Res. 147, as amended.
  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. BILIRAKIS. Mr. Speaker, on that I demand the yeas and nays.
  The yeas and nays were ordered.
  The SPEAKER pro tempore. Pursuant to clause 8 of rule XX and the 
Chair's prior announcement, further proceedings on this motion will be 
postponed.

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