[Congressional Record Volume 140, Number 142 (Tuesday, October 4, 1994)]
[Extensions of Remarks]
[Page E]
From the Congressional Record Online through the Government Printing Office [www.gpo.gov]


[Congressional Record: October 4, 1994]
From the Congressional Record Online via GPO Access [wais.access.gpo.gov]

 
    INTRODUCTION OF THE OFFICE FOR RARE DISEASE RESEARCH ACT OF 1994

                                 ______


                             HON. RON WYDEN

                               of oregon

                    in the house of representatives

                        Tuesday, October 4, 1994

  Mr. WYDEN. Mr. Speaker, I am introducing today a companion bill to 
legislation sponsored in the Senate by my colleague from the Oregon 
delegation, Mr. Hatfield. This bill would formally establish within the 
National Institutes of Health an Office for Rare Disease Research 
utilizing existing NIH personnel, and would authorize no new spending.
  The Office for Rare Disease Research would coordinate NIH research 
activities in the crucial area of diagnosis and treatment of rare 
diseases, in the same manner as the existing Office for Women's Health 
and Office for AIDS Research. This coordination will help ensure that 
the various institutes working in this area will cooperate with each 
other, avoid wasted and duplicative effort, and focus scarce 
governmental resources on high priority research.
  Congress has taken action in the past to motivate the private 
pharmaceutical industry to invest in treatments for rare disorders that 
represent too small a potential market to provide a reasonable return 
to investors who might otherwise invest in a cure. Enactment of the 
Orphan Drug Act in 1983 has proved to be a very successful venture in 
public policy, focusing private dollars and intellect on these vexing 
and often fatal diseases.
  During the past 10 years, more than 500 orphan drugs have been 
designated by the FDA, and more than 100 such products are now 
available for use by patients. This is 10 times the number of orphan 
products approved for use in the decade preceding enactment of the 
Orphan Drug Act.
  The Office for Rare Disease Research created by this legislation 
would provide an analogous focus for public sector investment in cures 
for these poorly understood diseases, which number in the thousands.
  One of the reasons why this legislation is needed is that rare 
disorders affect the human body in many ways that don't respect the 
institutional pigeonholes that govern how NIH funds are divided up and 
spent. For example, a rare disease often will simultaneously affect 
several organ systems, ranging from the brain to the skin to the 
kidneys, each the special focus of a different institute at NIH. The 
Office for Rare Disease Research would act to coordinate the expertise 
of the separate institutes, getting the most bang for the taxpayers' 
research buck.

  In addition, a tragic and preventable fact of life for people 
afflicted with rare diseases is that they often must see many doctors 
about their symptoms, but go without a proper diagnosis--let alone an 
effective treatment plan--for a year or more. One of the important 
contributions of this new office would be to establish and maintain a 
rare disease clinical database, to help scientists and physicians 
understand and treat rare diseases more promptly and appropriately.
  Mr. Speaker, in closing I would like to point out that this 
legislation is supported by the key consumer organization representing 
those with rare disorders, the National Organization for Rare Disorders 
[NORD]. NORD's president, Ms. Abbey Meyers, worked closely with 
Chairman Waxman, Chairman Dingell, and Senator Hatch in securing 
enactment of the original Orphan Drug Act. Ms. Meyers tells me that she 
has high hopes that this legislation will provide another major boost 
to the search for effective treatments for rare disorders.
  Without objection, I would request that a letter of support for the 
Office of Rare Disease Research Act of 1994 from Ms. Meyers be 
incorporated in the record at the conclusion of my remarks.
                                         National Organization for


                                          Rare Disorders Inc.,

                                  New Fairfield, CT, Oct. 4, 1994.
     Representative Ron Wyden,
     Washington, DC
       Dear Congressman Wyden: We fully support the Office for 
     Rare Diseases Research Act, which you are sponsoring in the 
     House of Representatives. As you know, this legislation would 
     establish an Office for Rare Diseases at the National 
     Institutes of Health [NIH].
       The Office for Rare Diseases at NIH would fulfill one of 
     the primary recommendations of the National Commission on 
     Orphan diseases. As you know, the Commission found that there 
     is an absence of coordination between the many Institutes of 
     NIH which have responsibilities for orphan disease research, 
     and that precious resources are being wasted through 
     duplicated efforts. The NIH office will create a home for 
     rare disorders at NIH, which will have authority to 
     coordinate activities among the many Institutes.
       The fact is, many rare disorders affect different body 
     systems so research on each disease may be in the realm of 
     several Institutes. For example, one disease may have skin 
     manifestations that are the responsibility of NIAMS, 
     neurological symptoms that fall under the NINDS umbrella, 
     renal complications that encompass NIDDK, and genetic 
     research may be pursued at the Human Genome Center. A rare 
     disease center at NIH would be able to coordinate the 
     activities of all these Institutes and Centers so they can 
     plan and execute programs with minimal waste of time and 
     funds. If a treatment is being developed and the FDA must 
     become involved, the office could coordinate FDA's activities 
     with NIH early in the process. This addresses a specific need 
     identified by the Commission when it found that NIH and FDA 
     do not coordinate their activities satisfactorily.
       We support your effort for this legislation with the hope 
     that it will spur productive research on orphan diseases, 
     minimize delays and save needed resources. The astonishing 
     advancements of the Human Genome Project make it imperative 
     that the Office be established quickly to coordinate policies 
     on more than 5,000 rare diseases, in a similar manner to 
     Offices on Women's Health, Minority Health, AIDS and 
     Alternative Therapies.
       Thank you for your support of this important effort to 
     develop a coordinated federal program for orphan diseases.
           Very truly yours,
                                                  Abbey S. Meyers,
     President.

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